MBMH Issue 7

My Blood My Health

ISSN 2819-2486

A Heal Canada Signature Program

"Bridging Health with Knowledge and Advocacy"

Understanding Blood Counts: A Patient’s Guide to Lab Results What makes up your Complete Blood Count Test The Role of Real-World Evidence in Blood Cancer Advocacy Why Patient Advocacy Is Essential in the Healthcare Ecosystem The Power of the Patient Voice in Clinical Trials

May 2025 Issue #7

Table of Contents

My Blood My Health

Welcome Understanding Blood Counts: A Patient’s Guide to Lab Results What makes up your Complete Blood Count Test The Role of Real-World Evidence in Blood Cancer Advocacy Legislative Advocacy for Blood Health Why Patient Advocacy Is Essential in the Healthcare Ecosystem Clinical Trials and Patient Surveys Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens Clinical Trial Search - Canada The Power of the Patient Voice in Clinical Trials: Shaping the Future of Hematological Research List of Hematology Advocacy Groups References Alliances and collaborations Partners My Blood, My Health Team

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Visit My blood, My Health.ca Healcanada.org to download the current issue 2

Welcome to My Blood, My Health Digital Magazine Welcome to this edition of My Blood My Health, where we shine a spotlight on two of the most vital forces in hematology: the Complete Blood Count (CBC) and the Patient Voice in Clinical Trials.

At first glance, the CBC may seem like just another lab test—a standard line item in a patient’s chart. But for individuals living with blood cancers and other hematological disorders, this humble test holds profound meaning. It’s often the first clue, the ongoing monitor, and the silent storyteller of what’s happening deep within. Whether tracking white cells, red cells, or platelets, a CBC paints the picture of disease status, treatment impact, and overall well-being. Understanding this test empowers patients to ask better questions, anticipate changes, and partner more effectively in their care journey.

But data alone doesn’t change outcomes— people do. That’s why this issue also elevates the essential role of advocacy in clinical trials. As research evolves to be more inclusive, innovative, and responsive, the patient voice is no longer a “nice-to-have”—it is a must-have.

From designing meaningful endpoints to building trust in underrepresented communities, patients and advocates are helping to shape the future of hematology research. They are not just participants in clinical trials; they are co-creators of better science.

Together, knowledge and advocacy form a powerful alliance. We hope this issue equips you with both and inspires you to use your voice, ask questions, and lead with courage.

Cheryl Petruk, MBA B.Mgt. CEO & Founder of Heal Canada Heal Canada's mission is to empower patients to access better and equitable services. We're deeply grateful to our readers for their continued interest and support. Your engagement drives us to produce insightful and valuable content that encourages patientcentricity in healthcare.

Thank you for participating in our journey, and welcome to this enlightening issue!

With great humility for reading out digital magazine, My Blood My Health Cheryl.

Publisher Heal Canada www.healcanada.org Email: admin@healcanada.org Executive Publisher & Editor in Chief Cheryl Petruk Editorial Team Heal Canada Staff/Consultants Scientific Advisors MBMH Scientific Consultants Marketing & Outreach Heal Canada Published By Heal Canada – A national not-for-profit organization dedicated to advancing patient advocacy through education, empowerment, and engagement.

Publication Frequency Bi-Monthly | Digital Only

Copyright © 2025 My Blood My Health Digital Magazine. All rights reserved. No part of this publication may be copied, reproduced, or transmitted in any form without prior written consent of the publisher.

Disclaimer: The Patient Advocacy Digital Magazine provides general information and resources to promote patient empowerment and awareness. The content is not a substitute for professional medical advice or treatment. Always consult with qualified healthcare professionals for personalized guidance regarding your specific medical condition or situation.

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Understanding Blood Counts: A Patient’s Guide to Lab Results

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Educating Patients on CBCs, Bone Marrow Biopsies, and Diagnostic Insights —and the Need for Stronger Health Literacy Tools Receiving blood test results can be overwhelming, especially when unfamiliar medical terms like “hematocrit,” “neutrophils,” or “reticulocyte count” appear on a report with no explanation. For many patients, lab work is an essential part of understanding what’s happening inside their body, particularly in cases involving blood disorders, cancers, or chronic illnesses. Yet far too often, patients receive these results with little context or clarity.

Understanding blood counts is more than a technical skill — it's a powerful step toward advocacy, self-awareness, and better health outcomes. This article offers patients and caregivers a practical, plain-language guide to interpreting common blood tests, including Complete Blood Count (CBC), bone marrow biopsies, and other vital diagnostics. It also explores how improved health literacy tools can empower patients, foster better communication with healthcare providers, and support more informed decisions about treatment and care.

Part 1: Why Blood Counts Matter The human body relies on blood to deliver oxygen, fight infections, and regulate essential functions.

When something changes in your blood, such as low red cells or an excess of white cells, it can be a clue that something deeper is occurring, ranging from infections to cancers to autoimmune diseases.

Blood counts are often the first signal that something isn’t right. A single lab result may not tell the whole story, but trends over time can reveal patterns that help doctors diagnose, monitor, and manage conditions.

Understanding the “why” behind your lab work can be a life-changing step.

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Part 2: Decoding the Complete Blood Count (CBC) A Complete Blood Count (CBC) is one of the most commonly ordered blood tests. It provides information about the different cells in your blood: red blood cells, white blood cells, and platelets. Here's what you need to know: Red Blood Cells (RBCs) Function: Carry oxygen from your lungs to the rest of your body.

Key terms: Hemoglobin (Hgb): Measures the amount of oxygen-carrying protein.

Hematocrit (Hct): Percentage of blood volume made up by RBCs. MCV (Mean Corpuscular Volume): The average size of red blood cells.

What to watch for: Low RBCs, hemoglobin, or hematocrit may suggest anemia, which can result from blood loss, iron deficiency, or bone marrow disorders.

High levels might be seen in conditions like polycythemia vera or chronic dehydration.

White Blood Cells (WBCs) Function: Defend the body against infections.

Differential count: Breaks down types of WBCs: Neutrophils: First responders to infection.

Lymphocytes: Include T-cells and B-cells, which help fight viruses and produce antibodies.

Monocytes, eosinophils, and basophils: Less common, but play roles in immune responses.

What to watch for: High WBCs may indicate infection, inflammation, or leukemia.

Low WBCs may signal bone marrow problems or effects of chemotherapy.

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Platelets (PLTs) Function: Help blood to clot and stop bleeding.

Normal range: Typically between 150,000–400,000 per microliter of blood.

What to watch for: Low platelets (thrombocytopenia): Risk of bruising and bleeding.

High platelets (thrombocytosis): May be reactive (due to inflammation) or a sign of a myeloproliferative neoplasm.

Part 3: Beyond the CBC—Other Key Blood Tests While the CBC is foundational, other tests provide further clues: Reticulocyte Count Measures young red blood cells. High counts may suggest blood loss or hemolysis; low counts may point to bone marrow failure.

Peripheral Blood Smear A microscope view of blood cells that can show abnormal shapes or inclusions, useful for diagnosing leukemia or hemoglobinopathies.

Erythropoietin Levels A hormone that stimulates RBC production—used to differentiate causes of anemia or polycythemia.

Iron Studies, B12, and Folate Help determine if nutrient deficiencies are contributing to anemia.

Part 4: Understanding Bone Marrow Biopsies When blood tests raise concern about how your blood cells are being made, your doctor may recommend a bone marrow biopsy.

What Is It? A bone marrow biopsy removes a sample of bone marrow (usually from the hip) to examine under a microscope. It provides a direct look at your blood cell factory.

Why It’s Done Diagnosing blood cancers (like leukemia, lymphoma, or myelofibrosis).

Evaluating unexplained anemia, low platelets, or abnormal white cell counts.

Staging or monitoring certain cancers.

What Patients Should Know It’s often done under local anesthesia.

You may feel pressure, but pain is usually brief.

Results take several days and are reviewed by a pathologist.

Understanding what the test is looking for—and asking your healthcare provider to explain the findings—is critical for your comfort and confidence.

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Part 5: Advocating for Health Literacy Too many patients receive test results without guidance. A lack of clear, accessible health information leads to confusion, fear, and unnecessary anxiety. It can also delay care or lead to poor decision-making.

Why Health Literacy Tools Are Needed Interpretation matters. Numbers don’t speak for themselves.

Clear explanations reduce fear.

Patients who understand are more engaged in their care.

Common Gaps in Communication Lab reports often use abbreviations or ranges with no explanation.

Portals may provide “normal” vs “abnormal” results without context.

Providers may assume patients understand terminology.

Part 6: Tips for Patients—How to Read and Ask About Your Lab Results Here are some practical steps patients can take: Ask for a copy of your lab results.

Always request your CBC and other diagnostic results so you can track trends over time.

Highlight what’s outside the normal range.

If something is marked “high” or “low,” ask your provider what that means for your health.

Write down your questions.

Before your appointment, prepare questions like: “What does this test result mean?”

“Is this a significant change from my last test?”

“What could be causing this result?”

“Do we need to follow up with more tests?”

Use trusted resources.

Check reliable sources like: The Leukemia & Lymphoma Society (LLS) Mayo Clinic Canadian Cancer Society My Blood My Health by Heal Canada

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Part 7: Empowering the Blood Disorders Community For those living with blood cancers or rare hematological diseases, like myeloproliferative neoplasms (MPNs), aplastic anemia, or sickle cell disease, understanding lab work is a key part of navigating care. Yet access to personalized explanations remains uneven.

Many Patient Organizations are working to assist their patient populations with: Simplify diagnostic language.

Create patient-centred education materials.

Advocate for improved communication between providers and patients.

Support digital tools that visualize lab trends over time.

Patient advocates are playing a vital role in shifting the system by pushing for better communication and clearer tools.

Part 8: The Role of Digital Platforms and Patient Portals Many healthcare systems now use online portals to share test results. While these tools are helpful, they often present data without explanation. To make these more patient-friendly, there’s a growing call for: Integrated definitions and tooltips on what each lab measure means.

Patient videos walking through common test types.

Visual graphs showing how results change over time.

Color-coded alerts that offer guidance without creating panic.

Patients can advocate for better digital experiences by providing feedback to hospitals and care providers.

Understanding as a Path to Empowerment Blood counts are more than numbers—they’re clues about how your body is working and how your care should proceed. For patients, especially those with chronic or serious health conditions, interpreting these lab results can be confusing without support.

But knowledge is power. The more patients understand, the more confidently they can participate in decisions, ask the right questions, and advocate for themselves. Healthcare systems, providers, and advocacy organizations must continue working toward a world where health literacy is a right, not a privilege, and where no one is left to decode their diagnosis alone.

Takeaway Messages A CBC is a critical tool in evaluating health, especially for patients with blood disorders.

Bone marrow biopsies provide insight into the production of blood cells and help diagnose serious conditions.

Patients should never be afraid to ask for explanations or second opinions on test results.

Health literacy resources should be simple, accessible, and widely available to all.

Patient advocacy can change the system by pushing for clearer communication, better tools, and inclusive care.

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Know Your Tests Complete Blood Count (CBC) A CBC is a routine blood test that evaluates your overall health and detects a variety of disorders.

Component

What It Measures

What It Might Indicate

Red Blood Cells (RBC)

Oxygen delivery capability

Low = Anemia; High = Polycythemia or dehydration

Hemoglobin (Hgb)

Oxygen-carrying protein in RBCs

Low = Anemia; High = Lung/heart disease, dehydration

Hematocrit (Hct)

Proportion of RBCs in blood

Similar to Hemoglobin interpretations

Mean Corpuscular Volume (MCV)

Size of RBCs

High = B12/Folate deficiency; Low = Iron deficiency

White Blood Cells (WBC)

Immune system defense

High = Infection or leukemia; Low = Immune suppression

Platelets (PLT)

Clotting ability of blood

Low = Bleeding risk; High = Inflammation or MPNs

Additional Lab Tests Test

Why It’s Done

Reticulocyte Count

Measures new red cells to assess bone marrow function

Iron Studies, B12, Folate

Detects nutrient deficiencies that may cause anemia

Peripheral Blood Smear

Identifies abnormal shapes or features of blood cells

Erythropoietin Level

Evaluates hormone that stimulates red cell production

Bone Marrow Biopsy

Examines marrow for blood cancers or cell production disorders

Your Lab Report — How to Read It Most lab reports include: Reference ranges (normal values) Your result Flagged items (marked as “H” for high or “L” for low)

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Example Table: Test

Your Result

Normal Range

Flag

What It Might Mean

Hemoglobin

9.5 g/dL

12–16 g/dL

L

Mild anemia—may need further tests

White Blood Cell

11.2 x10⁹/L

4.0–10.0 x10⁹/L

H

Possible infection/inflammation

Questions to Ask Your Doctor Bring your lab report to your next appointment and consider asking: What do these results mean for my condition?

Are these changes significant since my last test?

Could any of these results be caused by my medications?

What follow-up tests or actions are needed?

Should I be referred to a hematologist?

Tracking Your Lab Trends Use this template to record and compare your results over time: Date

Test Name

Result

Normal Range

Notes (e.g., symptoms, treatment changes)

Tip: Use color-coding (green for normal, red for flagged) to visualize changes.

Understanding Abbreviations Abbreviation

Full Term

Meaning

Hgb

Hemoglobin

Oxygen-carrying protein in red blood cells

Hct

Hematocrit

Volume percentage of red blood cells

WBC

White Blood Cell count

Immune system activity indicator

PLT

Platelets

Cells involved in clotting

MCV

Mean Corpuscular Volume

Average size of red blood cells

ANC

Absolute Neutrophil Count

Measures infection-fighting capability

RDW

Red Cell Distribution Width

Variation in red cell size

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd Trusted Resources Educational Websites Leukemia & Lymphoma Society of Canada (LLSc) Canadian Cancer Society My Blood My Health – Heal Canada Lab Tests Online Mobile Apps MyChart – Access and track lab results.

CareClinic – Track symptoms, labs, and medication.

Blood Counts Explained – App for understanding test meanings (if available regionally).

Advocate for Yourself You have the right to access your test results.

It’s OK to ask for a second opinion.

You can request a referral to a hematologist.

Ask your healthcare team to explain results in plain language.

Being informed is a key part of your treatment plan. Don’t be afraid to speak up.

When to Seek Medical Attention Immediately Call your healthcare provider or go to the ER if you experience: Sudden bruising or bleeding Unexplained fatigue or weakness Fever with low white cell counts Shortness of breath or chest pain Dizziness with low hemoglobin Final Thoughts Understanding your blood work can feel like learning a new language, but you’re not alone. With the right tools, explanations, and support, you can take charge of your health. Use this guide as a companion, ask questions, and connect with patient advocacy organizations who can help you every step of the way.

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Understanding Blood Counts: A Patient’s Guide to Lab Results cont'd 15 Questions to Ask Your Doctor A handy guide for patients to bring to their healthcare appointments.

General understanding 1. Can you explain what these test results mean in plain language?

2. Which of my results are outside the normal range, and should I be concerned?

3. How do these results compare to my previous tests? Are there any trends?

4. What could be causing any abnormal results?

5. Are these results typical for someone with my condition or treatment plan?

Follow-up and next steps 1. Do any of these results suggest I need more tests or a referral to a specialist?

2. Would you recommend a bone marrow biopsy or other diagnostics based on this?

3. Should I be monitoring any of these values regularly? How often?

4. Do these results explain the symptoms I’ve been experiencing?

5. Is this a one-time issue, or does it indicate a chronic condition?

Medication & Lifestyle Impact 1. Could any of my medications or supplements be affecting these results?

2. Are there lifestyle changes I should consider based on these results?

3. What steps can I take to improve my blood counts? (if applicable) Learning & Support 1. Can you walk me through what each of the CBC values means for my health?

2. Where can I find trusted resources to help me understand my lab results better?

Check out our website for some printer friendly resources to use in your physician appointments. www.healcanada.org

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What makes up your Complete Blood Count Test Brigitte Leonard, PHD.

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You can be asked to do a blood test, also known in medical jargon as a complete blood count (CBC). CBC is a set of tests that provides information about a person's blood cells, size, and quantity.

This test is relatively easy for patients. The analysis is performed by an automated hematology analyzer, which makes the results fast and cheap to obtain. The CBC is one of the most commonly performed medical tests because it provides much information on a person's general health.

How can CBC be used?

CBC can be done to monitor general health during annual checkups or to explore possible causes of symptoms. It can help diagnose medical conditions and check the health of your immune system. It can detect blood cancers, anemia, infections and other conditions.

Anemia is a good example of the importance of CBC. It can have several causes, including nutritional deficiency (iron or vitamin) or a genetic disorder (sickle cell disease, thalassemia).

Bone marrow failure or blood cancer can also cause anemia. Combining different elements of the CBC can help physicians understand what causes anemia.

Blood count can also help to assess the impact of some medications. CBC analysis before surgical procedures can minimize complications.

What CBC measure?

The blood is composed of plasma and blood cells. In a healthy person, plasma accounts for 55% of the blood. Plasma is the liquid portion of the blood, consisting of water, essential blood proteins, and nutrients.

The CBC will measure the number and size of all blood cells: Red blood cells (erythrocytes) White blood cells (leukocytes) Platelets (thrombocytes) Red Blood Cells: Red Blood cells are the blood's most abundant cells, representing 45% of the blood volume. Mature red blood cells lack a nucleus, giving them a concave shape. They mainly contain a protein (hemoglobin) which transports oxygen and carbon dioxide.

Hemoglobin needs iron to work correctly, which is why iron deficiency can cause anemia. Hemoglobin gives the blood its red colour.

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What makes up your Complete Blood Count Test cont'd Red Blood cells are the blood's most abundant cells, representing 45% of the blood volume. Mature red blood cells lack a nucleus, giving them a concave shape. They mainly contain a protein (hemoglobin) which transports oxygen and carbon dioxide.

Hemoglobin needs iron to work correctly, which is why iron deficiency can cause anemia.

Hemoglobin gives the blood its red colour.

In the CBC, several parameters are used to evaluate red blood cells: In the CBC, several parameters are used to evaluate red blood cells: Red blood cell count Hemoglobin (Hg): This is the amount of hemoglobin in your blood Hematocrit (Hct): This is the percentage of your blood made up of red blood cells.

- Mean corpuscular volume (MCV): This is the average size of your red blood cells.

Mean corpuscular hemoglobin (MCH): This is the average quantity of hemoglobin in each red blood cell.

Red cell distribution width (RDW): This is the degree of difference in red blood cell size. If the red blood cells are close to the same size, the RDW will be low.

White Blood Cells: White blood cells are part of the immune system and help your body fight germs. They include several types of cells, all of which help protect you from diseases.

A typical CBC counts all types of white blood cells.

However, physicians can also request a white blood cell differential analysis, which counts all white blood cells by type.

Neutrophils constitute 60-70% of the circulating white blood cells. They are the first responder when a pathogen is detected; they prevent infections by ingesting and releasing enzymes to destroy invading bacteria and fungi (2).

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What is part of your Complete Blood Count Test cont'd Neutrophils constitute 60-70% of the circulating white blood cells. They are the first responder when a pathogen is detected; they prevent infections by ingesting and releasing enzymes to destroy invading bacteria and fungi (2).

Monocytes share neutrophils' "vacuum cleaner" function to fight infections, help remove damaged tissues, and destroy cancer cells. Still, they can also present pathogens to lymphocytes to stimulate the immune system's memory, which is essential to improve response over time.

Lymphocytes are much more common in the lymphatic system than in blood. There are three sub-groups of lymphocytes.

B cells make antibodies against pathogens to block invasion and facilitate their destruction.

T cells destroy cells presenting pathogen molecules and coordinate the immune response.

Natural killer cells can destroy infected and cancerous cells that escape T cells.

Eosinophils compose about 2-4% of white blood cells in circulating blood.

They primarily deal with parasitic infections. They secrete chemicals that destroy large parasites, such as hookworms and tapeworms, that are too big for any white blood cell to ingest. Eosinophils are also the predominant inflammatory cells in allergic reactions (3).

Basophils the rarest white blood cells (less than 0.5%), are chiefly responsible for allergic response by releasing histamine, which causes the dilation of blood vessels. They also secrete heparin, which inhibits blood clotting. Both substances promote the movement of other white blood cells into an area Platelets Platelets are cell fragments that help your blood clot and control bleeding. They gather at the injured site, attach themselves to the blood vessel, and attract other platelets to connect to each other to plug the hole. They also initiate the coagulation process and facilitate wound healing.

What are the normal values?

Abnormal values will fall below or above the reference ranges. The reference range varies depending on your age, sex, and where you live. Each parameter of the CBC can indicate a possible issue that needs further investigation. In Table 1, you can see a few examples of potential causes.

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What is part of your Complete Blood Count Test cont'd

Parameters

Reference Range

Platelets

150000 to 450000

Lower value

Higher value

Thrombocytopenia

(tone of causes)

Thrombocytosis (Blood cancer) Leukocytosis (inflammation, Blood cancer)

WBC

4500 to 11000

Leucopenia (Infection, Bone marrow disease)

RBC

M: 4.5 to 5.9 W: 4.1 to 5.1

Anemia

Blood cancer

Hematocrit

M: 41.5% to 50.4% W: 35.9% to 44.6%

Iron deficiency

Deshydratation, blood cancer

Hemoglobin

M: 14 to 17.5 W: 12.3 to 15.3

Anemia, Hyperhydratation

Deshydratation

CMV

80 to 96

Iron deficiency, Thalassemia

Vitamin B12 & Folate deficiency

Each parameter of the CBC can indicate a possible issue that needs further investigation. In Table 1, you can see a few examples of potential causes.

As discussed earlier, combining CBC parameters information can help pinpoint underlying causes and improve diagnoses. The average red blood cell size (MCV) versus the width distribution (RDW) can provide additional information to identify the cause of blood disorders and direct the intervention precisely. See Table 2 that the combination of two CBC parameters can help the investigation and direct follow-up tests.

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What is part of your Complete Blood Count Test cont'd

We hope this summary will help you understand the importance of blood tests and how to track CBC results for you and your loved ones.

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The Role of Real-World Evidence in Blood Cancer Advocacy Cheryl Petruk, MBA

The Role of Real-World Evidence in Blood Cancer Advocacy explores how realworld evidence (RWE)—data collected from patients outside of traditional clinical trials—can transform the landscape of blood cancer care and patient advocacy.

The article highlights how RWE bridges gaps in clinical understanding, supports equitable access to treatment, and strengthens health policy decisions by reflecting diverse patient experiences. Through examples such as patient-led registries, real-world surveys, and partnerships with regulators and industry, the article underscores the growing importance of RWE in shaping research priorities, drug approvals, and advocacy strategies. It concludes by positioning RWE as both a scientific tool and a human rights mechanism for advancing better outcomes for individuals living with blood cancers.

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The Role of Real-World Evidence in Blood Cancer Advocacy

By Cheryl Petruk, MBA | My Blood My Health Digital Magazine

In the complex and rapidly evolving landscape of healthcare, especially within the field of hematology, the patient voice is no longer a peripheral input—it is central to innovation, access, and equity. Real-world evidence (RWE), which captures how therapies perform outside the confines of randomized clinical trials (RCTs), is playing an increasingly crucial role in supporting this paradigm shift. For patients with blood cancers such as leukemia, lymphoma, myeloma, or myeloproliferative neoplasms (MPNs), real-world data (RWD) can mean the difference between theoretical efficacy and practical, life-improving outcomes.

RWE is not just a scientific concept; it is an advocacy tool. Blood cancer patient advocacy organizations are harnessing RWE to push for better drug approvals, improve patient access to therapies, and reshape healthcare policies to reflect lived experiences. This article explores the transformative potential of RWE in blood cancer advocacy, the ethical considerations it raises, and the collaborative responsibilities among patients, advocates, clinicians, industry partners, and policymakers.

Defining Real-World Evidence in Context Real-world evidence is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from the analysis of real-world data. Unlike clinical trials, which operate under controlled environments with strict inclusion/exclusion criteria, RWE reflects the outcomes of treatments in broader, more diverse patient populations—patients of varying ages, comorbidities, socioeconomic backgrounds, and racial identities.

Sources of RWD include: Electronic health records (EHRs) Insurance claims and billing data Patient-reported outcomes (PROs) Patient registries Mobile health apps and wearable devices Together, these sources provide a holistic picture of a patient's health journey—one that can often highlight gaps, barriers, and opportunities for better care that controlled trials may overlook.

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The Role of Real-World Evidence in Blood Cancer Advocacy cont'd Why RWE Matters in Blood Cancer Blood cancers are complex, heterogeneous, and frequently rare in their subtypes, making RCTs challenging to execute with broad applicability. Many patients are excluded from trials due to age, coexisting conditions, or geographic limitations, creating an evidence gap that RWE is uniquely positioned to fill.

For instance: Older adults with acute myeloid leukemia (AML) are frequently underrepresented in trials but are often the majority affected by the disease.

Black and Indigenous patients with lymphoma or multiple myeloma face systemic disparities in diagnosis, treatment, and outcomes. RWE can help quantify and correct these inequities.

Patients with MPNs, such as myelofibrosis, often endure years of underdiagnosis. RWE can track long diagnostic journeys and support earlier interventions.

By leveraging real-world data, advocacy groups can identify unmet needs, highlight disparities, and support health technology assessment (HTA) submissions with patientcentred perspectives.

RWE as an Advocacy Catalyst Blood cancer advocacy has historically been rooted in storytelling, giving voice to those facing uncertain futures, often navigating fragmented care. RWE gives statistical weight to those stories, making them actionable in policy, funding, and research.

Here are five ways RWE is catalyzing advocacy efforts: 1. Empowering Patient-Led Research Organizations like Heal Canada, Leukemia & Lymphoma Society, and MPN Research Foundation are initiating and funding studies that integrate patient-reported outcomes, quality of life metrics, and social determinants of health. These studies provide nuanced insights that can be translated into treatment guidelines or recommendations.

2. Improving Access to Treatments RWE supports reimbursement decisions by demonstrating how therapies perform in realworld settings. In Canada, for example, CADTH and provincial drug programs increasingly consider real-world effectiveness data to make funding decisions, especially for costly therapies like CAR T-cell treatments or JAK inhibitors.

3. Accelerating Regulatory Decisions Regulatory agencies like Health Canada, the FDA, and the European Medicines Agency are integrating RWE into accelerated pathways for rare and serious conditions. This is crucial in blood cancers where timely access can significantly alter prognosis.

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The Role of Real-World Evidence in Blood Cancer Advocacy cont'd 4. Fostering Inclusion and Equity RWE helps reveal patterns of exclusion in traditional research. When blood cancer data includes more diverse patient voices, it enables advocacy groups to push for culturally competent care, translation services, and equitable clinical trial designs.

5. Building Global Advocacy Networks RWE enables patient advocacy groups to connect across borders. Global registries, such as the Myelofibrosis Registry or the Global Leukemia Registry, help map treatment patterns internationally, creating shared platforms for advocacy and policy change.

Ethical Considerations in Using RWE As the use of real-world evidence expands, so do the ethical considerations, particularly around patient data, consent, and privacy.

1. Informed Consent: Patients must understand how their data will be used, and consent should be clear, ongoing, and easily withdrawable.

2. Data Ownership and Transparency: Advocates must fight for transparency in how RWD is collected and used. Who owns the data —the patient, the provider, or a third-party registry? Transparency builds trust and accountability.

3. Representation and Equity: If RWD does not represent the full spectrum of patient experiences—rural patients, lowincome individuals, marginalized communities—then RWE risks perpetuating existing inequities.

4. Industry Collaboration: Advocacy groups working with pharmaceutical companies to generate RWE must navigate potential conflicts of interest. Strong governance frameworks are critical to maintaining credibility and ethical rigour.

The Advocate’s Role in RWE Patient advocates are essential stakeholders in the generation, interpretation, and application of RWE. Here’s how they can engage: 1. Championing Patient Registries Encourage patients to participate in disease-specific registries. Advocate-led registries often offer patients a chance to guide research priorities, ensuring the data collected is meaningful to them.

2. Co-Designing Studies Work with researchers and industry partners to co-design protocols that include real-world endpoints, such as days spent at home, emotional well-being, and treatment burden.

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The Role of Real-World Evidence in Blood Cancer Advocacy cont'd 3. Mobilizing Communities Use digital platforms, community forums, and social media to educate patients on the importance of RWE and how participation in RWD initiatives supports collective change.

4. Translating the Science Advocates act as “bridge builders”—translating complex RWE findings into actionable insights for patients, caregivers, and policymakers. This includes simplifying infographics, policy briefs, and explainer videos.

5. Holding Systems Accountable Use RWE findings to hold healthcare systems accountable for disparities in care delivery. Push for policy interventions when real-world data reveals avoidable harm or systemic gaps.

Success Stories: RWE in Action CAR T-Cell Therapy Access in Canada Real-world studies showed improved survival outcomes in patients with relapsed/refractory diffuse large B-cell lymphoma treated with CAR T therapy, especially in populations underrepresented in trials. These findings contributed to provincial funding decisions across Canada.

MPN Patient RWE/Surveys by Heal Canada Heal Canada’s real-world survey on MPN patients uncovered diagnostic delays averaging three years and highlighted significant unmet needs in fatigue management and mental health. These findings are included in reports and to advocate for better specialist referral pathways.

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The Role of Real-World Evidence in Blood Cancer Advocacy cont'd UK’s Blood Cancer Alliance Data Collaboration Patient organizations partnered with NHS Digital to identify disparities in treatment initiation times between different ethnic groups. The findings prompted policy changes to standardize early interventions.

Looking Ahead: Future Opportunities for RWE in Blood Cancer Advocacy As digital health tools become more sophisticated, the opportunity to generate high-quality RWE expands: Wearables & Apps: Devices that track symptoms, mobility, and adherence can provide real-time RWD. AI and Predictive Analytics: Machine learning models can identify patterns in large datasets that suggest earlier intervention points or treatment optimization pathways.

Global Data Collaboration: Cross-border data pooling can offer broader insights into rare hematologic cancers, improving global standards of care.

Advocacy organizations must remain at the forefront of these changes—ensuring that RWE doesn’t just serve scientific progress, but human dignity and equity.

Making Evidence Matter The shift toward real-world evidence in hematology reflects a broader truth: that evidence is not just data —it is people. Behind every metric is a patient who deserves to be seen, heard, and healed. Advocacy organizations have an unparalleled opportunity— and responsibility—to guide the ethical use of RWE in ways that center lived experience and drive real change.

By fostering collaboration between patients, clinicians, researchers, and policymakers, the blood cancer community can harness the full potential of RWE to shape a more just, inclusive, and effective healthcare system.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes Cheryl Petruk, MBA

Blood health policies play a critical role in ensuring that patients with hematological malignancies and blood disorders receive timely diagnoses, effective treatments, and comprehensive care. From funding for research to equitable access to life-saving therapies, legislative decisions shape the healthcare landscape for millions of patients worldwide. Legislative advocacy is a powerful tool for influencing these policies, ensuring that patient voices are heard and that systemic improvements lead to better outcomes for blood cancer and blood disorder patients.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes

By Cheryl Petruk, MBA

Blood health is an essential component of overall wellness, yet it is often overlooked in broader healthcare policy conversations. For the millions of individuals living with blood disorders—ranging from iron deficiency anemia and hemophilia to blood cancers such as leukemia, lymphoma, and myelofibrosis—public policy decisions directly influence access to care, timely diagnosis, treatment affordability, and long-term outcomes. Legislative advocacy has emerged as a powerful force to close gaps in the healthcare system, address inequities, and ensure that blood health becomes a policy priority.

This article explores how policy change and legislative advocacy can significantly enhance patient outcomes in the realm of blood health. We examine the key policy levers affecting blood disorder patients, highlight examples of successful advocacy efforts, and provide a roadmap for patient advocates to engage in legislative change. Whether it’s ensuring funding for rare disease research or influencing drug reimbursement pathways, legislative advocacy is not just a supplementary effort—it is central to systemic change.

Understanding Legislative Advocacy in Healthcare Legislative advocacy refers to efforts aimed at influencing laws and regulations through engagement with policymakers, including elected officials and government agencies. It often involves: Educating legislators on healthcare issues Proposing new laws or amendments Supporting or opposing proposed legislation Testifying at legislative hearings Participating in public consultations In the context of blood health, legislative advocacy focuses on improving the system of care for individuals with hematological conditions by addressing issues like research funding, diagnostic pathways, equitable drug access, reimbursement criteria, and patient rights.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes cont'd Why Blood Health Needs a Strong Legislative Voice Blood disorders are often underfunded and misunderstood, especially in rare and chronic categories. Some reasons why blood health advocacy is so urgent include: Delayed Diagnoses: Many blood disorders, such as myeloproliferative neoplasms (MPNs), are underdiagnosed or misdiagnosed due to a lack of awareness and limited access to hematologists.

Treatment Inequity: Access to medications can vary dramatically by province or insurance plan in Canada and state-by-state in the U.S., leaving patients in limbo.

Lack of Data Collection: Rare diseases often fall outside traditional health information systems, hindering population health planning.

Underrepresentation in Research: Certain populations—ethnic minorities, rural residents, and low-income individuals—are often left out of clinical research and policymaking.

Legislative advocacy has the power to change all of this by ensuring policy frameworks recognize and support the unique needs of individuals with blood disorders.

Key Legislative Priorities for Blood Health Advocates Access to Timely and Equitable Diagnosis Early diagnosis is critical for effective treatment. Legislative advocacy can push for standardized diagnostic protocols, ensure appropriate genetic and molecular testing is covered, and mandate shorter referral timelines to specialists. In Canada, wait times for hematologists can extend from weeks to months, too long for patients with aggressive blood cancers or complex disorders.

Funding for Rare Disease Research and Registries Advocates can influence budget allocations toward blood disorder research, including funding for disease-specific registries that support epidemiological insights and clinical trial recruitment.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes cont'd Drug Approval and Reimbursement Reform Many blood disorder patients wait years for new treatments to be approved by Health Canada, then face additional delays for reimbursement decisions by provincial or private drug plans.

Legislative reform is needed to: Speed up health technology assessments (HTAs) Incorporate real-world evidence in decision-making Ensure patient voices are included in pricing decisions Workplace Protections and Leave Policies Blood cancer treatments often require long-term leave.

Advocacy can lead to legislative changes that expand sick leave, protect job security, and provide income replacement.

Legislative efforts have successfully expanded Employment Insurance (EI) benefits for critically ill Canadians and should be extended further for chronic disease patients.

National Blood Strategy Canada lacks a unified national blood health strategy. A coordinated federal plan could align provincial resources, guide research investments, and improve patient access across the spectrum of blood disorders. This has been a top legislative priority for groups like the Canadian Hemophilia Society and other blood focused patient advocacy organizations.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes cont'd Case Study: Advocating for MPN Patient Needs In 2023–2024, Heal Canada and its signature program My Blood My Health conducted advocacy outreach based on findings from the MPN Patient Survey. Results revealed: 67% experienced a diagnostic delay of more than two years 82% had never received mental health support despite fatigue and depression being common

Important conclusions from patient surveys and information are: Inclusion of blood cancers in rare disease drug funding frameworks Mental health screening as part of standard care Funding for molecular diagnostic testing at the primary care level International Perspectives: Legislative Wins Around the Globe United States: Accelerated Approvals and the 21st Century Cures Act The U.S. has implemented legislation like the 21st Century Cures Act, which supports faster drug approvals and strengthens the inclusion of patient perspectives in FDA decision-making. Blood cancer organizations such as the Leukemia & Lymphoma Society have been instrumental in lobbying for these reforms.

Europe: EU Rare Disease Strategy The European Union’s strategy on rare diseases includes mandates for member countries to develop national plans.

Many countries have implemented pathways for early access and patient involvement in HTA processes, influencing care for rare blood cancers and bleeding disorders.

United Kingdom: All-Party Parliamentary Group on Blood Cancer This group has advocated for a national strategy on blood cancer in the UK. Their 2018 report influenced NHS England’s long-term plan to recognize blood cancer as a priority area and improve access to care, particularly for marginalized groups.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes cont'd

The Advocacy Toolbox: How to Influence Policy Legislative advocacy can feel daunting, but a structured approach helps patient advocates make a measurable impact.

Step 1: Identify the Issue Define the specific policy problem affecting patients. Is it delayed access to a therapy? Lack of coverage for diagnostics? Inconsistent referral patterns?

Step 2: Collect Evidence Support your issue with data—clinical studies, patient surveys, economic analysis, or testimonials. Real-world evidence is especially powerful in underscoring gaps between guidelines and lived experiences.

Step 3: Build a Coalition Partner with other patient organizations, medical societies, and industry stakeholders. Unified voices carry more weight in legislative meetings and public consultations.

Step 4: Engage Legislators Schedule meetings with Members of Parliament, provincial health ministers, or agency directors. Come prepared with: A one-page summary of your ask A personal story to humanize the issue Evidence to show impact on outcomes and costs Step 5: Leverage Public Platforms Use digital campaigns, op-eds, petitions, and social media to raise awareness. Platforms like Twitter, LinkedIn, and advocacy-specific forums help connect with policymakers and amplify your message.

Step 6: Follow Up and Stay Engaged Policy change takes time. Continue following up, provide updates on your advocacy efforts, and keep building momentum until the issue is addressed.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes cont'd Role of Patient Organizations in Legislative Advocacy Patient-led organizations like Heal Canada, the Canadian Organization for Rare Disorders (CORD), and the Canadian Cancer Society are critical catalysts for legislative advocacy.

They provide policy literacy training for patients and caregivers and host advocacy days at provincial legislatures.

Collaborate with researchers to present evidence-based recommendations Develop educational materials for policymakers Organize roundtables between patients, regulators, and pharmaceutical companies.

By equipping patients to be policy participants—not just recipients—these organizations are redefining what meaningful advocacy looks like.

Challenges and Opportunities Challenges Bureaucratic Delays: Legislative timelines are often slow and unpredictable.

Lack of Awareness: Policymakers may not understand the urgency or complexity of blood health issues.

Funding Constraints: Many advocacy organizations operate on limited budgets and rely on volunteer efforts.

Opportunities Digital Advocacy: Virtual meetings and online petitions make it easier than ever to engage decision-makers.

Public Sentiment: There is growing societal support for patient-centred care and equity in healthcare access.

Policy Windows: Government reviews, budget consultations, or elections create opportunities to elevate blood health on political agendas.

Looking Forward: Legislative Priorities for 2025 and Beyond As we look ahead, here are five policy goals for the blood health advocacy community to unite around: A Pan-Canadian Strategy for Blood Health: Develop a unified approach to diagnosis, treatment, and research that ensures consistency across provinces.

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Legislative Advocacy for Blood Health: How Policy Changes Can Improve Patient Outcomes cont'd Inclusion of Patient-Reported Outcomes in HTA Processes Mandate that patient voices and lived experiences are included in every assessment of new therapies.

Legislative Support for Early Molecular Diagnostics Enshrine reimbursement policies that cover next-generation sequencing and molecular tests for blood cancers.

Expanded Compassionate Access Programs Advocate for national frameworks that ensure patients can access life-saving medications while they await funding decisions.

Permanent Government-Funded Patient Advocacy Training Secure funding for patient advocates to receive formal training and certification, ensuring sustained and informed legislative engagement.

Legislative advocacy is not a luxury—it is a lifeline. For individuals living with blood disorders, policy decisions shape every aspect of the care journey—from diagnosis to treatment, to survivorship and beyond. Through structured, sustained advocacy, we can transform individual struggles into collective progress.

Patient stories, when backed by real-world data and delivered with strategy, have the power to rewrite legislation and reshape healthcare systems. By making blood health a legislative priority, we can ensure that future patients face fewer barriers, receive faster diagnoses, and access the innovative therapies they deserve.

Let this be our collective call to action: to advocate boldly, legislate wisely, and ensure that every voice—especially those too often silenced—is heard where it matters most: in the halls of power.

Canadian MPN Network Patient Advocacy and Education Group

Providing MPN Patients Support and Education

Info@canadianmpnnetwork.ca

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Patient Advocacy Training Level One Courses Starting September 2025 Advanced Courses Starting September 2025 Register now! www.cacheducation.org contact: Info@CACHEducation.org

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CACHEducation is evolving to better serve the needs of patient advocates and healthcare professionals with its rebrand to CACHEducation Academy. This transformation reflects an expanded commitment to delivering high-quality, structured learning experiences tailored to the ever-changing landscape of patient advocacy and healthcare education. As part of this rebrand, CACHEducation Academy will introduce Advanced Curriculum offerings starting in September 2025, providing deeper insights, specialized training, and enhanced skill development for those looking to elevate their expertise. This next phase marks a significant step forward in strengthening the capacity and impact of patient advocates through comprehensive and innovative education.

"Enrolling in CACHEducation was a game-changer for me as a patient advocate. The program provided invaluable knowledge, practical skills, and a supportive community that empowered me to make a real impact in healthcare advocacy."

Patient Advocacy Training contact: Info@CACHEducation.org

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Why Patient Advocacy Is Essential in the Healthcare Ecosystem

By Cheryl Petruk, MBA

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Why Patient Advocacy Is Essential in the Healthcare Ecosystem

by Cheryl Petruk, MBA In an increasingly complex and fragmented healthcare system, patient advocacy has emerged as a vital force for ensuring that individuals receive equitable, informed, and compassionate care. Whether through individual advocates, community organizations, or systemic policy efforts, patient advocacy plays a pivotal role in bridging gaps between patients and providers, safeguarding patient rights, and driving improvements in healthcare delivery.

This article explores the multifaceted importance of patient advocacy within the healthcare ecosystem, highlighting its impact on individual outcomes, systemic change, and health equity.

Defining Patient Advocacy Patient advocacy encompasses actions and support aimed at promoting and protecting patients' rights, interests, and well-being within the healthcare system. Advocates may be healthcare professionals, trained individuals, or organizations that assist patients in navigating medical decisions, accessing services, and voicing concerns.

Their responsibilities include ensuring informed consent, facilitating communication between patients and providers, and addressing systemic barriers to care .

Empowering Patients and Enhancing Outcomes One of the core functions of patient advocacy is empowering individuals to actively participate in their healthcare decisions.

By providing education and support, advocates enable patients to make informed choices, leading to improved satisfaction and health outcomes. For instance, the Choosing Wisely Canada campaign encourages conversations between clinicians and patients about unnecessary medical interventions, promoting evidence-based care and reducing potential harm Moreover, patient advocacy can lead to tangible improvements in care quality. A study published in Nursing Ethics found that nurse-led advocacy efforts contributed to better patient safety and satisfaction by ensuring that care decisions aligned with patients' values and preferences Patient advocacy is instrumental in addressing health disparities and promoting equity, particularly among marginalized populations. Advocates work to dismantle systemic barriers that contribute to unequal access to care, such as socioeconomic status, race, and language differences.

For example, health advocates have been pivotal in implementing culturally tailored strategies to improve healthcare access for immigrant populations, addressing critical barriers like language gaps and unfamiliarity with healthcare navigation processes).

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Why Patient Advocacy Is Essential in the Healthcare Ecosystem cont'd Personal narratives also underscore the importance of advocacy in combating healthcare bias. Brooke Abbott Abron, an advocate for individuals with inflammatory bowel disease, shared her experience of navigating the healthcare system as a Black woman facing medical gaslighting and delayed diagnosis. Her story highlights the necessity of patient advocacy in ensuring that marginalized voices are heard and respected within medical settings.

Influencing Health Policy and Systemic Change Beyond individual support, patient advocacy plays a crucial role in shaping health policy and driving systemic change. Advocacy efforts have led to significant policy reforms, such as the AIDS Coalition to Unleash Power (ACT UP) influencing HIV/AIDS treatment policies and the "right to be forgotten" movement impacting cancer survivorship rights (Health Policy Partnership, 2024). The Health Policy Partnership Organizations like the Patient Access Network (PAN) Foundation exemplify how advocacy can lead to broader access to care.

The Role of Healthcare Professionals in Advocacy: Healthcare professionals, particularly nurses, are integral to patient advocacy. The American Nurses Association emphasizes that advocacy is a fundamental aspect of nursing, involving the promotion and protection of patients' rights and interests.

Nurses often serve as intermediaries between patients and the broader healthcare system, ensuring that care is patient-centred and ethically sound However, nurses may face challenges in their advocacy roles, including institutional constraints and potential conflicts between organizational policies and patient needs.

Addressing these barriers requires institutional support and a commitment to fostering an environment where advocacy is valued and facilitated.

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Why Patient Advocacy Is Essential in the Healthcare Ecosystem cont'd

Technological Advancements and Digital Advocacy The advent of digital health technologies has expanded the scope of patient advocacy.

Platforms like Solace connect patients with healthcare advocates to assist with tasks such as appointment scheduling and navigating insurance complexities. The integration of such services into Medicare coverage reflects a growing recognition of the importance of advocacy in enhancing care coordination and patient experience.

Additionally, online communities like Superbloom provide support networks for individuals with chronic illnesses, offering resources and a platform to share experiences. These digital spaces empower patients to advocate for themselves and others, fostering a sense of community and shared purpose .

Patient advocacy is a cornerstone of a responsive and equitable healthcare system. By empowering individuals, addressing disparities, influencing policy, and embracing technological innovations, advocacy ensures that patient voices are central to healthcare delivery. As the healthcare landscape continues to evolve, the role of patient advocacy will remain essential in promoting health, dignity, and justice for all individuals.

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mybloodmyhealth.ca

monsangmasante.ca encouraging Canadians to get their blood checked yearly

My Blood My Health is an awareness campaign encouraging Canadians to look after their blood health at their yearly check-up.

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Clinical Trials and Surveys 41

Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens

By Cheryl Petruk, MBA Clinical trials are the backbone of medical advancement. They bring innovation to life, transforming theoretical possibilities into tangible treatment options. For patients with hematological diseases—ranging from leukemia, lymphoma, and myeloma to rare disorders like myeloproliferative neoplasms (MPNs)—clinical trials are more than just a research mechanism. They are a lifeline, a source of hope, and a pivotal means of accessing cuttingedge therapies. But while their scientific value is well understood, the patient perspective remains underrepresented in the broader discourse. This article explores the crucial role of clinical trials in hematology through the eyes of the patient, addressing the emotional, practical, ethical, and systemic dimensions that define their participation and experience.

The Patient-Centric View of Clinical Trials From the patient's point of view, participating in a clinical trial is not merely a medical decision—it is deeply personal, often complex, and laden with hope, fear, and trust. The decision to join a trial may be driven by several factors: the limitations of existing treatments, the desire for more time, the hope for improved quality of life, or the altruistic motivation to help future patients.

For many with hematologic conditions, standard therapies can become ineffective over time. For example, patients with myelofibrosis may experience progression despite frontline treatments. Others with chronic lymphocytic leukemia (CLL) may relapse after standard regimens. Clinical trials offer access to novel therapies such as targeted agents, CAR T-cell therapy, or bispecific antibodies that might not otherwise be available outside a research setting.

Empowerment Through Participation Clinical trials empower patients by placing them at the center of innovation. Rather than being passive recipients of care, patients become active contributors to scientific discovery.

This empowerment often translates into a sense of purpose, as many patients find meaning in helping to generate knowledge that may benefit others in similar situations.

Access to Innovation For some, clinical trials provide access to cutting-edge therapies not yet available in clinical practice. These may include genetically engineered cell therapies, monoclonal antibodies, or oral inhibitors that address disease pathways with greater specificity. For patients with rare hematological disorders or treatment-resistant disease, trials may offer the only viable option for extending life or improving symptoms

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Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens cont'd Challenges Patients Face in Clinical Trials While the potential benefits are significant, participating in a clinical trial is not without challenges. Patients face logistical, psychological, and informational hurdles that must be acknowledged and addressed.

Understanding the Trial Medical jargon, long consent forms, and complex protocols can overwhelm patients.

Understanding the difference between phases of clinical trials—Phase I (safety) Phase 1 tests initial safety in small groups (20-100 people), Phase II (efficacy) Phase 2 evaluates effectiveness (up to 300 people), Phase III (comparative effectiveness) Phase 3 compares to existing treatments (300-3,000 people)—and interpreting outcomes like progression-free survival or overall response rate can be daunting without adequate support and in some cases Phase 4 monitors long-term effects after FDA, CDA or other regulatory authority approval.

Navigating Eligibility and Access Not every patient is eligible for every trial. Inclusion and exclusion criteria may disqualify patients based on factors such as previous treatments, organ function, or age. Geographic location can also be a barrier. Many trials are conducted in large academic centers, making participation difficult for rural or underserved populations.

Logistical and Financial Strains Although study-related medications and tests are typically covered, indirect costs— transportation, accommodation, childcare, or time off work—can be burdensome. These financial and practical considerations often disproportionately affect low-income patients, limiting their participation.

Emotional and Physical Toll Participating in a trial may require more frequent hospital visits, blood tests, and monitoring, which can be physically exhausting. The emotional toll of not knowing whether the treatment will work or whether one is in a placebo group can be significant, particularly in randomized or blinded trials.

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Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens cont'd Why Patients Still Choose to Participate Despite these challenges, many patients with hematological diseases willingly and courageously enter clinical trials. Why? Hope for Better Outcomes For patients who have exhausted standard therapies or face aggressive disease, clinical trials may offer the only path forward. Even in early-phase trials where therapeutic benefit is uncertain, patients often feel hopeful—if not for themselves, then for others.

Sense of Altruism Many patients report a strong desire to "give back." Knowing that their participation could contribute to future therapies or lead to better understanding of their condition brings purpose and meaning. This altruistic motivation is especially evident in rare disease communities where patient advocates work tirelessly to support research.

Trust in Medical Teams The relationship between patients and their hematologists or oncologists plays a vital role.

Trust in their care team’s recommendation can significantly influence the decision to enroll in a trial. Patients are more likely to participate when they feel their values and preferences are respected.

The Importance of Integrating the Patient Voice in Trial Design For clinical trials to truly serve the patient population, they must be designed with the patient in mind.

Patient-Reported Outcomes (PROs) Including PROs as endpoints ensures that trials measure what truly matters to patients, such as fatigue, pain, cognitive function, and emotional well-being, not just clinical markers like white blood cell counts or blast percentages.

Inclusive Trial Design Diverse representation in trials is critical.

Historically, racial minorities, women, older adults, and people from rural communities have been underrepresented in hematology trials. This lack of diversity limits the generalizability of findings. Designing studies with broader inclusion criteria and decentralized models (e.g., local lab testing, telemedicine visits) can increase participation among underserved populations.

Transparency and Communication Patients need clear, jargon-free information about trial goals, risks, and potential benefits. Involving patient advocates early in trial design ensures that recruitment materials and consent documents are understandable and aligned with patients’ values and concerns.

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Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens cont'd

Hematology-Specific Considerations Hematological diseases are often systemic, chronic, and complex. The nature of these diseases introduces unique considerations for clinical trial participation.

Chronicity and Trial Duration Conditions like chronic myeloid leukemia (CML) or polycythemia vera (PV) may require long-term participation in trials that track molecular markers over time. Patients must weigh their ability to commit over extended periods and the lifestyle adjustments required.

Risk of Transformation Some conditions, such as MPNs, carry the risk of transforming into acute leukemia.

Clinical trials investigating preventative therapies or biomarkers for transformation are essential, and patient participation is key to advancing these efforts.

Immune-Based Therapies Innovations such as CAR T-cell therapy and bispecific T-cell engagers are revolutionizing hematology. These therapies often involve complex trial protocols, including lymphodepletion, hospitalization, and postinfusion monitoring.

Patients need comprehensive education and psychosocial support to navigate these intense regimens.

The Role of Patient Advocacy Patient advocacy organizations play a critical role in bridging the gap between the research community and those living with blood cancers and disorders.

Education and Awareness Advocacy groups help patients understand clinical trial options and their rights. They simplify scientific information and translate it into accessible resources, increasing patients’ confidence in asking questions and making informed decisions.

Support and Navigation Many organizations provide trial navigators —trained individuals who help patients identify suitable trials, understand eligibility, and manage logistics. These services are invaluable, particularly for those without access to a major cancer center.

Shaping Research Priorities Patient advocates are increasingly being invited to sit on research committees, ethics boards, and regulatory advisory panels.

Their input helps ensure that research agendas align with real-world patient priorities, such as improving daily function or reducing symptom burden.

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Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens cont'd Moving Toward Patient-Centric Clinical Research The future of clinical research in hematology must be co-created with patients. Several shifts are underway to enhance the patient experience in trials: Decentralized Trials: Enabling remote monitoring, telehealth visits, and at-home lab testing.

Digital Tools: Mobile apps and wearable devices to track symptoms, side effects, and activity levels.

Real-World Evidence (RWE): Integrating clinical trial data with real-world data to better understand patient trajectories and preferences.

Adaptive Trial Designs: Allowing for modifications in the protocol based on interim results, increasing efficiency and flexibility.

Shared Decision-Making Models: Encouraging open dialogue between clinicians and patients regarding trial options, values, and risks.

Clinical trials in hematological diseases are not just about scientific advancement—they are about people. Every new treatment approved today stands on the shoulders of patients who participated in yesterday’s trials. From hope and empowerment to logistical burdens and ethical dilemmas, the patient perspective adds essential richness and realism to the clinical research landscape.

To honour this contribution, healthcare systems, researchers, and regulators must work together to create a truly patient-centric research ecosystem—one where patients are not just subjects but partners. In doing so, we not only accelerate the discovery of new treatments but also uphold the dignity, autonomy, and voice of every person living with a blood disorder.

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Clinical Trials in Hematological Diseases: A Vital Journey Through the Patient’s Lens cont'd Canada stands at the forefront of hematological research, offering patients access to cutting-edge clinical trials that are shaping the future of blood disorder treatments. From common malignancies like leukemia and lymphoma to rare conditions such as myeloproliferative neoplasms (MPNs), Canadian institutions are actively engaged in studies that aim to improve patient outcomes and quality of life.

The Canadian Clinical Trial Landscape Canada's robust healthcare infrastructure and commitment to research excellence make it an ideal environment for conducting clinical trials. The country boasts over 40 research hospitals and 15 research universities, facilitating a wide range of studies across various medical fields. Health Canada's Clinical Trials Database provides a comprehensive listing of authorized phase I, II, and III clinical trials, serving as a valuable resource for patients and healthcare professionals seeking information on ongoing studies.

Hematological Clinical Trials: A Spectrum of Opportunities Canadian research institutions are conducting numerous clinical trials targeting hematological diseases. The Canadian Cancer Trials Group (CCTG), for instance, is involved in several studies focusing on conditions such as acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), Hodgkin lymphoma, and multiple myeloma. These trials explore various treatment modalities, including novel drug combinations and targeted therapies, aiming to enhance efficacy and reduce side effects.

Patient Participation: Empowering Choices Participation in clinical trials offers patients access to innovative treatments that may not be available through standard care. Engaging in a trial can provide hope for improved outcomes and contribute to the advancement of medical knowledge. Patients are encouraged to discuss trial opportunities with their healthcare providers and consider the potential benefits and risks involved.

Navigating Clinical Trial Options For those interested in exploring clinical trial opportunities in Canada, several resources are available: Health Canada's Clinical Trials Database: Offers information on authorized clinical trials across the country.

Canadian Cancer Trials: Provides a searchable database of cancer-related clinical trials, including those focusing on hematological malignancies.

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Clinical Trials in Hematological Diseases in Canada: Advancing Care Through Innovation cont'd Canada's dedication to advancing hematological research through clinical trials presents significant opportunities for patients seeking alternative treatment options. Interferon-based therapies and other innovative approaches are continually being evaluated, offering hope for improved management of blood disorders. By participating in clinical trials, patients not only access potential new therapies but also contribute to the collective effort to enhance care for future generations.

Note: Patients should consult with their healthcare providers to determine eligibility and suitability for participation in clinical trials.

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Consult your doctor: If you are living with Myleofibrosis,discuss this trial with your healthcare provider to see if this is the right opportunity for you.

For more information contact your local MPN Support Group 49

If you are living with a hematological condition or care for someone who is, consider exploring clinical trials. Talk to your healthcare team, reach out to advocacy organizations and use platforms such as ClinicalTrials.gov or Canadian Cancer Trials to learn more. Your voice and experience matter—more than you know.

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Clinical Trial Search - Canada

Health Canada's Clinical Trials Database - Website Health Canada, through its Clinical Trials Database, is providing the public with a listing of specific information relating to phase I, II, and III clinical trials in patients. The database is managed by Health Canada and provides a source of information about Canadian clinical trials involving human pharmaceutical and biological drugs.

Access the Clinical Trials Database Patients can access the database to determine if a clinical trial has met the regulatory requirements. The database may also assist Canadians in finding clinical trials that might be relevant to their medical condition.

The Clinical Trials Database is not a registry, and therefore, it does not contain comprehensive information about each clinical trial. To maximize the use of the database and available information, users are advised to link to external resources, including publicly available registries, to obtain further information such as trial objectives and patient eligibility.

Note, however, that not all clinical trials are necessarily registered and thus found in these registries.

Health Canada continues to encourage sponsors to register their clinical trials in publicly accessible registries such as ClinicalTrials.gov and ISRCTN. A Canadian-based registry for cancer trials is also available at Canadian Cancer Trials.

Additionally, the search portal provided by the World Health Organization (WHO) can be used to access a central database that contains information about trials registered in several international registries.

Health Canada is the federal regulator responsible for authorizing the importation and sale of drugs for the purpose of clinical trials. This responsibility is fulfilled through the review of clinical trial applications (CTAs) for phase I, II, and III clinical trials, filed by clinical trial sponsors. One of the objectives of Health Canada's review is to ascertain that subjects participating in the trial will not be exposed to undue risks.

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Clinical Trial Search - Canada Every year, Health Canada authorizes approximately 900 clinical trials in patients. The database lists trials that were authorized by Health Canada starting April 1, 2013. The database will be populated with information about each clinical trial after Health Canada issues its No-Objection Letter (see terminology section for a definition). Thus, following the launch, the number of clinical trials available in the database is expected to be small, but the number will increase with time as the database is populated.

Information Listed in the Database

The database will provide the following information on clinical trials for which a CTA has been authorized: Protocol Number; Protocol Title; Drug Name; Medical Condition; Study population; Date of No Objection Letter; Sponsor Name; Control Number; Study Start Date; Study End Date; Trial Status.

Health Canada's Clinical Trials Database serves as an essential resource for patients, healthcare providers, and researchers by offering regulatory insights into authorized clinical trials in Canada. While not a comprehensive registry, the database helps users identify trials that have met Health Canada’s safety and regulatory standards, offering a level of trust and transparency for Canadians exploring participation in clinical research.

By supplementing searches with additional global registries such as ClinicalTrials.gov, ISRCTN, and the Canadian Cancer Trials portal, individuals can gain a more complete understanding of trial options and eligibility. As the database continues to expand over time, it reinforces Health Canada's commitment to safeguarding patient well-being while promoting access to innovation and informed decision-making in clinical trial participation.

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The Power of the Patient Voice in Clinical Trials: Shaping the Future of Hematological Research

By Cheryl Petruk, MBA

In recent years, the clinical trial landscape has undergone a subtle yet powerful transformation—one in which the patient voice has emerged not as a peripheral concern, but as a central, driving force of research innovation. Nowhere is this shift more critical than in hematological trials, where rare and chronic conditions like myelofibrosis, leukemia, and myelodysplastic syndromes (MDS) demand nuanced, patient-centered approaches. The importance of incorporating the patient voice in clinical trials transcends ethical obligation— it is a scientific and strategic imperative.

This article explores why the patient voice is vital in clinical trial design, recruitment, retention, and outcomes; how it enriches the data and impact of hematological research; and what structural changes are necessary to embed patient perspectives at every level of the research process.

Redefining Value: Patients as Co-Creators of Science Traditionally, clinical trials have been designed from the top down, with protocol development, endpoints, and methodologies largely determined by pharmaceutical companies, clinicians, and regulatory agencies. While these stakeholders bring indispensable expertise, they cannot replicate the lived experience of the patient—the dayto-day navigation of symptoms, side effects, uncertainty, and hope.

In hematology, where patients often face long disease trajectories with evolving treatments, the concept of value must reflect what truly matters to patients. Is a 2-month extension of life meaningful if accompanied by unrelenting fatigue? Does a “manageable” side effect profile still feel manageable to someone balancing work, caregiving, and cancer treatment?

Embedding the patient voice ensures that clinical trial outcomes align with real-world needs.

This includes selecting endpoints that reflect quality of life (QoL), functional ability, emotional well-being, and financial toxicity—not just survival statistics or biomarker shifts.

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The Power of the Patient Voice in Clinical Trials cont'd Improving Trial Design Through Lived Experience Patient engagement in trial design improves protocol feasibility, enhances relevance, and reduces protocol amendments—saving time, money, and ethical burdens.

Key contributions patients make to design include: Endpoint Selection: Patients can advise on which outcomes should be prioritized, ensuring that efficacy is balanced with tolerability.

Eligibility Criteria: Overly restrictive criteria often exclude diverse populations or those with co-morbidities. Patients can help ensure that trials reflect the broader disease community.

Visit Schedules & Burden: Patients can highlight logistical and emotional challenges in attending frequent visits, undergoing biopsies, or completing lengthy forms. Protocols can be adjusted to ease this burden.

A study by the Clinical Trials Transformation Initiative (CTTI) found that patient engagement in the protocol design phase led to measurable improvements in recruitment speed and participant satisfaction.

Representation Matters: Equity and Inclusion For hematological trials to produce meaningful and generalizable results, they must include diverse patient populations—racial, ethnic, gender, socioeconomic, and geographic. Yet many trials continue to underrepresent communities most affected by certain blood cancers.

Including patient advocates and grassroots organizations during trial planning can reveal structural barriers to participation: lack of transportation, language limitations, mistrust of medical institutions, or a lack of culturally competent communication.

These insights are critical for designing inclusive recruitment strategies and establishing trust.

Additionally, patient organizations can support communitybased awareness campaigns and peer outreach, humanizing the process and helping individuals feel empowered to ask about clinical trials as care options.

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The Power of the Patient Voice in Clinical Trials cont'd The Role of Patient Advocates: Trusted Intermediaries Patient advocates are not just spokespeople —they are translators, connectors, and educators. They play a critical role in building bridges between researchers and communities by: Explaining clinical trial concepts in accessible language Supporting informed consent processes Helping patients understand their rights and options Advocating for ongoing support throughout participation Their presence within advisory boards, ethics review panels, and steering committees ensures that the patient lens is embedded throughout the trial lifecycle.

In the hematological space, organizations such as the Leukemia & Lymphoma Society, Myeloma Canada, and Heal Canada’s My Blood My Health program are pioneering advocacy-led initiatives that center the patient voice in research design and dissemination.

Real-World Data and Patient-Reported Outcomes One of the most compelling reasons for prioritizing patient input is the growing reliance on Real-World Evidence (RWE) and Patient-Reported Outcomes (PROs) in regulatory decision-making.

Patients can provide granular insights through PROs that reflect how treatments affect their daily lives—pain levels, sleep quality, fatigue, anxiety, social functioning.

These data points are increasingly valued by regulators like Health Canada, the FDA, and EMA, and influence both market approvals and post-marketing surveillance.

Moreover, involving patients in the creation of these measures ensures the tools reflect lived experience rather than abstract clinical definitions.

Recruitment and Retention: Trust, Motivation, and Meaning Recruitment remains one of the biggest challenges in clinical trials, with 80% of studies failing to meet enrollment timelines.

Retention is equally difficult, especially in trials requiring frequent travel, invasive procedures, or extended commitments.

Patients who are engaged early in the process—who understand the trial’s purpose, who feel their contribution is meaningful, and who trust the process—are more likely to enroll and stay.

Storytelling, peer ambassadors, and community-based engagement strategies designed by and for patients significantly enhance recruitment.

Moreover, when participants know their feedback will be valued, and that they will receive post-trial updates, a deeper sense of partnership emerges.

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The Power of the Patient Voice in Clinical Trials cont'd Changing the Culture of Research To make patient voice integration sustainable, it must become part of research culture, not a checkbox. This includes: Institutionalizing Patient Engagement: Require patient input in grant applications, ethics reviews, and protocol approvals.

Compensating Patient Partners: Recognize patient expertise with fair compensation for time and knowledge.

Training Researchers: Provide education on how to work with patient partners respectfully and effectively.

Feedback Loops: Share results, updates, and learnings with trial participants and advocacy groups.

Culture change also means acknowledging power imbalances and creating space for shared decision-making—a difficult but essential shift for academia, industry, and regulators alike.

From Participation to Partnership The future of hematological research depends on a simple but transformative shift: treating patients not as “subjects” but as partners. The value of such partnerships is already visible in trials that have embedded advocacy from the start.

For example, the development of the “Living with MPN” QoL tool was led by patients, who defined what well-being means when living with an incurable condition.

The more we elevate patient perspectives, the more relevant, ethical, and successful our trials will become.

Elevating the Human Side of Science Clinical trials are not just scientific experiments—they are human experiences.

Every protocol is lived out by someone managing not just disease, but dignity, family, fear, and hope.

By integrating the patient voice, we ensure that hematological research is not only effective, but compassionate, inclusive, and ultimately—transformative.

This is not just good science—it’s the right thing to do.

Call to Action: If you're a patient, caregiver, or advocate, your voice matters. Get involved by: Joining advisory boards or patient councils Asking your care team about clinical trials Sharing your experience with advocacy organizations Participating in surveys or focus groups Supporting peers navigating the clinical trial journey Together, we can reshape the future of blood cancer research—one voice at a time.

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for more information how LCA can help you info@latinxcanceralliance.org

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Heal Canada and Pat ADV Hub in the USA have embarked on a collaborative journey, aiming to revolutionize the realm of patient advocacy across North America. This pioneering partnership brings together two influential organizations from neighbouring countries, combining their extensive expertise and resources.

The objective is to expand and enhance the access to critical information for patient advocates, ensuring that individuals across the continent receive the best possible support and guidance in their healthcare journeys.

By bridging the gap between Canadian and American healthcare advocacy, this alliance promises to foster a more informed, empowered, and connected community of patient advocates, significantly contributing to the improvement of healthcare experiences for countless individuals.

patadvhub@gmail.com

www.patadvhub.org

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Hematolgy Advocacy Groups

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List of Hematology Advocacy Groups - Canada National & Regional Hematology Advocacy Organizations Network of Rare Blood Disorder Organizations (NRBDO) A pan-Canadian coalition representing patients with rare blood disorders such as hemophilia, thalassemia, aplastic anemia, and von Willebrand disease. NRBDO focuses on advocacy, healthcare access, and best practices in care delivery.

Canadian Hemophilia Society (CHS) Founded in 1953, CHS supports individuals with inherited bleeding disorders through education, research, and advocacy. It operates chapters in every province

Aplastic Anemia & Myelodysplasia Association of Canada (AAMAC) Provides education, peer support, and research funding for Canadians living with aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH). Offers local support meetings and webinars.

Leukemia & Lymphoma Society of Canada (LLSC) A national charity dedicated to curing leukemia, lymphoma, Hodgkin's disease, and myeloma. LLSC provides patient support services, education, and funds research across Canada.

Lymphoma Canada Focuses on education, support, and advocacy for lymphoma patients. Offers virtual and in-person support groups across provinces, including Ontario.

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List of Hematology Advocacy Groups - Canada Myeloma Canada The only national organization exclusively focused on multiple myeloma. Provides educational resources, promotes clinical research, and advocates for patient access to new therapies.

CLL Canada A national advocacy group supporting Canadians affected by Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL). Offers education and works to improve treatment access.

Alpha-1 Canada Provides peer-to-peer support and disease management services for Canadians diagnosed with Alpha-1 Antitrypsin Deficiency. Engages in national and international advocacy efforts. aHUS Canada Advocates for individuals with atypical Hemolytic Uremic Syndrome (aHUS), focusing on education, support, and access to treatment.

Canadian Hemochromatosis Society Founded by Marie Warder, this organization raises awareness and provides support for those affected by hereditary hemochromatosis. Offers educational materials and advocacy.

The Canadian CML Network is a community of people living with Chronic Myeloid Leukemia. We are dedicated to providing emotional, social and educational support to people living with CML and their families.The Canadian CML Network works with patients and healthcare providers from all across Canada, and is focused on building CML patient groups nationally

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List of Hematology Advocacy Groups - Canada Canadian MPN Network Patient Advocacy Group A Formal Canadian network of patients and caregivers.

Provides a platform for sharing best practices and connecting with local support groups.

Know your Blood A Formal Canadian organization dedicated to the Black/Caribbean community. Provides a platform for sharing and connecting for specific diseases to this community

Informal Groups (Facebook, other social media) CLL/SLL Information & Support (Canada) A Canadian community for individuals living with Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL), their families, and professionals. Members share experiences, ask questions, and offer mutual support. Facebook CML Friends of Canada A group for Canadians living with or supporting someone with Chronic Myeloid Leukemia (CML). It serves as a space for sharing experiences, information, and support among members.

Lymphoma Canada – Facebook Page While not a group, Lymphoma Canada's Facebook page offers resources, updates, and information on virtual support groups, including monthly sessions co-hosted with Wellspring for lymphoma and CLL patients. Lymphoma Canada

If you would like your group listed here, please email us at admin@healcanada.org

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List of Hematology Advocacy Groups - USA Leukemia & Lymphoma Society (LLS) LLS is the largest nonprofit dedicated to curing leukemia, lymphoma, Hodgkin's disease, and myeloma. It provides patient support, funds research, and advocates for policies to ensure access to quality, affordable care.

National Bleeding Disorders Foundation (NBDF) Formerly the National Hemophilia Foundation, NBDF focuses on awareness, care, and treatment of inheritable blood and bleeding disorders like hemophilia and von Willebrand disease.

International Myeloma Foundation (IMF) IMF is dedicated to improving the quality of life for myeloma patients through research, education, support, and advocacy. It offers resources for patients, caregivers, and healthcare professionals Leukemia Research Foundation This foundation's mission is to cure leukemia by funding research and supporting patients and families. It provides educational resources and financial assistance.

Lymphoma Research Foundation As the nation's largest lymphoma-focused health organization, it is devoted to funding lymphoma research and providing patients with education and support services CancerCare CancerCare offers free, professional support services for people affected by cancer, including counselling, support groups, educational workshops, publications, and financial assistance.

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List of Hematology Advocacy Groups - USA National Marrow Donor Program (NMDP) NMDP manages the Be The Match Registry, facilitating bone marrow and umbilical cord blood transplants for patients with life-threatening blood cancers Aplastic Anemia and MDS International Foundation (AAMDSIF) AAMDSIF provides support and education for patients with aplastic anemia, myelodysplastic syndromes (MDS), and related bone marrow failure diseases.

MDS Foundation The MDS Foundation offers support and resources for patients with myelodysplastic syndromes, including information on support groups and treatment options.

International Waldenstrom's Macroglobulinemia Foundation This foundation provides support and education for patients with Waldenström macroglobulinemia, a rare type of non-Hodgkin lymphoma, and funds research for improved treatments.

Children's Cancer and Blood Foundation (CCBF) CCBF is the first and largest charitable organization in the U.S. dedicated to supporting the care of children with cancer and blood diseases MPN Advocacy & Education International This organization offers educational programs, patient and caregiver conferences, and resources tailored to MPN patients. They aim to empower patients through knowledge and support.

MPN Education Foundation The foundation provides information and support for MPN patients, including the MPN-NET online support group, which facilitates discussions among patients, caregivers, and healthcare professionals.

If you would like your group listed here, please email us at admin@healcanada.org 68

List of Hematology Advocacy Groups - Global The Acute Leukemia Advocates Network (ALAN) A global patient advocacy organization dedicated to improving the lives of people affected by acute leukemias. By connecting patients, caregivers, and advocates, ALAN promotes awareness, facilitates access to information, and supports research to ensure patient voices shape leukemia care and policy worldwide.

CML Advocates Network An informal global network moderated by Chronic Myeloid Leukemia (CML) patients and caregivers.

Provides a platform for sharing best practices and connecting with national support groups.

The Global MPN Scientific Foundation (Global MPNSF) A non-profit organization committed to improving the lives of individuals affected by myeloproliferative neoplasms (MPNs), a group of rare blood cancers.

Through education, advocacy, and research support, Global MPNSF raises awareness, connects patients with specialists, and promotes advancements in treatment and care.

List of Hematology Advocacy Groups - Online MPN-NET Online Support Group Hosted by the MPN Education Foundation, MPN-NET is an email-based discussion group where patients and caregivers share experiences, information, and support related to MPNs. Facebook Groups Several Facebook groups offer informal support for MPN patients: Myeloproliferative Neoplasms (MPN) Support Group: A global community where members discuss various MPN-related topics.

Polycythemia Vera (PV) Support Group: Focused on individuals diagnosed with PV, sharing experiences and coping strategies.

Essential Thrombocythemia (ET) Support Group: A space for those with ET to connect and support each other.

Myelofibrosis (MF) Support Group: Dedicated to discussions around living with MF, treatments, and research updates.

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Have your voice heard!

Visit the Heal Canada website and participate in our Various Surveys healcanada.org mybloodmyhealth.ca monsangmasante.ca

Openned Surveys: Blood Cancer Quality of Life (QoL) Impact of Blood Transfusion on QoL Myelofibrosis impact on QoL AML impact on QoL

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References American Society of Hematology. (n.d.). Complete blood count (CBC). Retrieved April 30, 2025, from https://www.hematology.org/education/patients/blood-basics/completeblood-count Mayo Clinic. (2023). CBC (complete blood count). Retrieved April 30, 2025, from https://www.mayoclinic.org/tests-procedures/complete-blood-count/about/pac20384919 Lab Tests Online. (2023). Understanding your complete blood count. American Association for Clinical Chemistry. Retrieved April 30, 2025, from https://labtestsonline.org/tests/complete-blood-count-cbc Leukemia & Lymphoma Society. (2023). Blood and bone marrow tests. Retrieved April 30, 2025, from https://www.lls.org/treatment/blood-and-bone-marrow-tests Canadian Cancer Society. (2022). Bone marrow tests. Retrieved April 30, 2025, from https://cancer.ca/en/cancer-information/diagnosis-and-treatment/tests-andprocedures/bone-marrow-tests Nielsen-Bohlman, L., Panzer, A. M., & Kindig, D. A. (Eds.). (2004). Health literacy: A prescription to end confusion. Institute of Medicine. The National Academies Press. https://doi.org/10.17226/10883 Canadian Institute for Health Information. (2021). Health literacy in Canada: A healthy understanding. Retrieved April 30, 2025, from https://www.cihi.ca/en/health-literacy-incanada Weiss, B. D. (2007). Health literacy and patient safety: Help patients understand (2nd ed.).

American Medical Association Foundation.

Heal Canada. (2025). My Blood My Health: Patient education tools for understanding hematological diseases. Retrieved from https://www.healcanada.ca Canadian Agency for Drugs and Technologies in Health (CADTH). (2023). Real-World Evidence Framework.

U.S. Food & Drug Administration. (2022). Real-World Evidence Program.

MPN Research Foundation. (2023). State of MPNs: Registry Report.

Heal Canada. (2024). My Blood My Health: MPN Quality of Life Survey.

Leukemia & Lymphoma Society. (2022). The Importance of Real-World Data in Blood Cancer.

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References Canadian Agency for Drugs and Technologies in Health (CADTH). (2023). Real-world evidence use and its impact on decision making. https://www.cadth.ca/real-worldevidence-use Canadian Organization for Rare Disorders (CORD). (2023). Rare disease strategy for Canada: 2023 policy update. https://www.raredisorders.ca Canadian Partnership Against Cancer. (2022). Equity in cancer care: A national strategy to improve outcomes. https://www.partnershipagainstcancer.ca Centers for Medicare & Medicaid Services. (2021). Coverage with evidence development: Policy overview. https://www.cms.gov European Commission. (2023). Rare diseases: Policy and data. https://health.ec.europa.eu/non-communicable-diseases/rare-diseases_en Food and Drug Administration. (2022). Framework for FDA’s real-world evidence program. https://www.fda.gov/media/120060/download Heal Canada. (2024). MPN Patient Survey: Lived experiences, diagnostic journeys, and access to care. Internal report.

Health Canada. (2023). Modernization of the Health Canada Special Access Program and priority drug approvals. https://www.canada.ca/en/health-canada/services/drugshealth-products/special-access.html Leukemia & Lymphoma Society. (2023). Policy priorities: Access, equity, and innovation in blood cancer care. https://www.lls.org/advocate National Organization for Rare Disorders. (2022). Legislative wins in rare disease advocacy: An annual review. https://rarediseases.org Office of the Auditor General of Canada. (2021). Access to health services for remote and Indigenous communities. https://www.oag-bvg.gc.ca Pan-Canadian Pharmaceutical Alliance. (2022). Drug negotiation and reimbursement framework. https://www.pcpacanada.ca Public Health Agency of Canada. (2023). Canada’s national strategy for rare diseases – Building pathways to better care. https://www.canada.ca/en/publichealth/services/publications/diseases-conditions/national-strategy-rare-diseases.html UK Parliament. (2018). All-Party Parliamentary Group on Blood Cancer: Inquiry into blood cancer care in the NHS. https://www.appgbloodcancer.org.uk United States Congress. (2016). 21st Century Cures Act. https://www.congress.gov/bill/114th-congress/house-bill/34

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References Abbasinia, M., Ahmadi, F., & Kazemnejad, A. (2019). Patient advocacy in nursing: A concept analysis. Nursing Ethics, 26(6), 1835–1848. https://doi.org/10.1177/0969733017751742 Health Policy Partnership. (2024). Patient advocates: vital voices in shaping health policy and care. https://www.healthpolicypartnership.com/patient-advocates-vital-voices-inshaping-health-policy-and-care/ Very well Health. (2024). How Self-Advocacy Helped Me Navigate Crohn's Disease and Healthcare Bias. https://www.verywellhealth.com/self-advocacy-to-navigate-crohns8730188 Vogue. (2020). Superbloom Is a New Online Community for Women With Chronic Illness. https://www.vogue.com/article/superbloom-is-a-new-online-community-for-womenwith-chronic-illness Wall Street Journal. (2025). Medicare Coverage Fuels Solace’s $60 Million Series B. https://www.wsj.com/articles/medicare-coverage-fuels-solaces-60-million-series-b28eb41b1 Wikipedia. (2024). Patient advocacy. https://en.wikipedia.org/wiki/Patient_advocacy Wikipedia. (2025). Choosing Wisely Canada. https://en.wikipedia.org/wiki/Choosing_Wisely_Canada Wikipedia. (2025). PAN Foundation. https://en.wikipedia.org/wiki/PAN_Foundation Wikipedia. (2024). Health advocacy. https://en.wikipedia.org/wiki/Health_advocacy Canadian Cancer Trials. (2023). Find a clinical trial. https://www.canadiancancertrials.ca Leukemia & Lymphoma Society. (2023). Understanding clinical trials. https://www.lls.org/treatment/clinical-trials ASH Clinical News. (2023). “Patient Engagement in Hematology Research.”

Mayo Clinic. (2022). Understanding the Phases of Clinical Trials.

National Institutes of Health. (2023). ClinicalTrials.gov. https://clinicaltrials.gov American Society of Clinical Oncology. (2022). “Improving Clinical Trial Diversity.”

Health Canada's Clinical Trials Database This database provides information on authorized phase I, II, and III clinical trials in Canada, including those related to hematological diseases.

Canadian Cancer Trials Group (CCTG) CCTG conducts national and international multicentre phase I-III cancer clinical research trials, including those focusing on hematologic malignancies.

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References Basch, E., Abernethy, A. P., Mullins, C. D., Reeve, B. B., Smith, M. L., Coons, S. J., ... & Tunis, S. R. (2012). Recommendations for incorporating patient-reported outcomes into clinical comparative effectiveness research in adult oncology. Journal of Clinical Oncology, 30(34), 4249-4255. https://doi.org/10.1200/JCO.2012.42.5967 Basch, E., Deal, A. M., Kris, M. G., Scher, H. I., Hudis, C. A., Sabbatini, P., ... & Schrag, D. (2017).

Symptom monitoring with patient-reported outcomes during routine cancer treatment: A randomized controlled trial. Journal of Clinical Oncology, 34(6), 557-565. https://doi.org/10.1200/JCO.2015.63.0830 Canadian Cancer Clinical Trials Network. (2021). Patient engagement framework for cancer clinical trials in Canada. https://3ctn.ca/wp-content/uploads/2021/12/3CTNPatient-Engagement-Framework.pdf Clinical Trials Transformation Initiative. (2020). CTTI Patient Engagement Playbook.

Clinical Trials Transformation Initiative. https://www.ctticlinicaltrials.org/projects/patient-engagement Food and Drug Administration (FDA). (2022). Patient-Focused Drug Development: Methods to Identify What is Important to Patients. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders. https://www.fda.gov/media/131230/download Frank, L., Basch, E., & Selby, J. V. (2014). The PCORI perspective on patient-centered outcomes research. JAMA, 312(15), 1513–1514. https://doi.org/10.1001/jama.2014.11100 Patrick, D. L., Burke, L. B., Gwaltney, C. J., Leidy, N. K., Martin, M. L., Molsen, E., & Ring, L. (2011).

Content validity—establishing and reporting the evidence in newly developed patientreported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 2—assessing respondent understanding. Value in Health, 14(8), 978-988. https://doi.org/10.1016/j.jval.2011.06.013 Wesch, N. L., & Lofters, A. (2022). Improving equity and diversity in cancer clinical trials in Canada. Current Oncology, 29(3), 1717–1722. https://doi.org/10.3390/curroncol29030139

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Alliances

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Alliances

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Partners

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My Blood, My Health Team Cheryl Petruk, MBA B.Mgt.

Cheryl A. Petruk is a multifaceted professional whose career spans across patient advocacy, business, and post-secondary education, showcasing her dedication to making a significant impact in each of these areas.

Cheryl's transition into patient advocacy was driven by a passion from her family circumstances and a deep commitment to ensuring patients' rights and access to care. Cheryl has worked tirelessly to bridge the gap between the healthcare system, patients, and pharma stakeholders, ensuring that patients' voices are heard and their needs are met. Her work involves collaborating with Stakeholders and Patient Advocacy Organizations, lobbying for patient centricity, and providing patient support and guidance. Cheryl's empathetic approach and dedication to advocacy have made her a respected figure in this field, admired by patients, healthcare professionals, and fellow advocates.

Cheryl also leads and is the lead faculty member at CACHEducation, Patient Advocacy Training. Cheryl is a certified greenbelt in VBHC, a certified Trainer in VBHC, and is a DBA Student

Heal Canada and its signature initiative, My Blood My Health, collaborate with a diverse array of consultants who bring specialized expertise to support our mission of advancing patient advocacy, education, and engagement in hematological health. These consultants include healthcare professionals, patient engagement strategists, medical writers, clinical research advisors, digital health experts, legal and regulatory specialists, and communications and media consultants. Each contributes uniquely—whether it's crafting patient-centered educational content, guiding ethical and regulatory compliance for clinical outreach, developing digital tools for patient engagement, or ensuring that advocacy messaging resonates across platforms. This multidisciplinary approach ensures our programs are informed, inclusive, and impactful, amplifying the patient voice while driving meaningful change in healthcare.

My Blood My Health Digital Magazine – Copyright Statement © 2025 My Blood My Health Digital Magazine. All rights reserved.

This publication is intended for informational and educational purposes only. No portion of this magazine may be copied, republished, or shared without express written permission, except as allowed under fair use for non-commercial educational purposes.

For inquiries or permissions, contact: admin@healcanada.org My Blood My Health is a patient-focused initiative of Heal Canada, dedicated to raising awareness of blood cancers and supporting the patient voice.

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