Critical PEGASYS Shortage, BESREMI Is The Alternative!

Brigitte Leonard, Ph.D

Recently, due to manufacturing disruptions, health authorities worldwide have faced a critical shortage of Pegasys, a peginterferon alfa-2a injection.

Canada is no exception to this stressful situation. This shortage presents serious risks to patients who depend on interferon-based therapy for treating myeloproliferative neoplasms (MPNs).

Health Canada has permitted the exceptional importation of another interferon-based therapy, Besremi, a ropeginterferon alfa-2b injection [1]. Besremi can be used instead of Pegasys to treat MPN. BESREMi® will be available to you regardless of your MPN form. Speak to your doctor to formulate a transition plan that is suitable for you.

Besremi is not an experimental drug and has proven efficient in MPN. The FDA approved Besremi as a PV treatment in 2021[2]. Besremi is still studied extensively in several phase III trials in Polycythemia vera (PV), Essential Thrombocytopenia (ET), and Myelofibrosis. Also, Besremi is part of the treatment recommendation in the USA (NCCN guidelines)[3].

You can find more information on the FORUS website: www.besremiei.com. This website provides information on exceptional importation and is easy to navigate. 0:42min.

Why is the MPN community facing a Pegasys shortage?

When a new company acquired the rights to sell Pegasys in 2017, a 10-year global supply stockpile was planned to allow the new manufacturer time to establish the new production line. Due to increased demand across many diseases, the stockpile has run out much earlier than anticipated. There is a minimal supply of Pegasys® in Canada. If you are experiencing difficulty filling your prescription, work with your doctor and pharmacist to find a pharmacy that still has a supply. Your pharmacist can initiate a pharmacy-topharmacy transfer.

Why can Besrmi be used instead of Pegasys?

Besremi (ropeginterferon alfa-2b) and Pegasys (peginterferon alfa-2a) are interferon therapies used in treating MPN, such as PV. Both Besremi and Pegasys are long-acting forms of interferon alfa, which stimulate the immune system, inhibit the production of abnormal blood cells, and reduce the production of red blood cells in PV. They both target similar pathways but differ in formulation and specific pharmacokinetic properties.

Formulation and Dosing:

Besremi (Ropeginterferon alfa-2b):

Besremi is a longer-acting interferon form, allowing for less frequent dosing. It is typically administered once every 2 weeks during the initial treatment phase and then once a month after the maintenance phase. Its extended duration of action means fewer injections than Pegasys.

Pegasys (Peginterferon alfa-2a):

Pegasys is also a long-acting interferon but requires more frequent administration than Besremi. It is typically injected once a week for the treatment of PV.

The dosage must be adjusted when a patient switches from Pegasys to Besremi. Your healthcareprofessionalwillbeabletoassistyouduringthetransition.

Besremi:

A Proven

Interferon Formulation For Myeloproliferative Neoplasms (MPNs)

Interferon (IFN) has been known for decades to provide interesting results in PV. However, the poor tolerability of the old formulation impacted its adoption in clinical practices. RopegIFN (BESREMI) has been studied in low-risk and high-risk patient populations.

In low-risk patients, the dose can be increased from 100 µg every 2 weeks to 250 → 350 → 500 microgramme every two weeks if the clinical benefits are not observed [4]. RopegIFN provides superior results in hydroxyurea-naive patients Avoiding HU and initiating patients with RopegIFN could improve long-term care. Based on these findings, the National Cancer Network (NCCN) recommended RopegIFN as the preferred cytoreductive regimen in the first-line setting for patients with low-risk PV and a preferred interferon regimen in high-risk PV.

Besremi:

1) Reduces the need for phlebotomy (Figure 1a).

2) Reduces the number of phlebotomy from 3 to 0.5 (Figure 1b), with patient who receive Hydroxyurea (HU) the number of phlebotomy are reduced to 2.4 to 0.5.

3) Normalizes leucocyte and platelet counts, resulting in no thrombotic events (Figure 2).

4) Reduces the JAK2 mutation burden and is considered a disease modifier treatment (Figure 3).

5) Improved the quality of life for PV patients, addressing concerns about drug tolerability and toxicity.

The Role of Real-World Evidence in Blood Cancer Advocacy

Cheryl Petruk, MBA

How Patient-Reported Outcomes and Real-World Data Can Shape Better Treatments and Policies for Blood Cancer Patients.

Blood cancers, including leukemia, lymphoma, myeloma, and myeloproliferative neoplasms, represent a significant health burden, affecting thousands of patients annually. While clinical trials remain the gold standard for evaluating new therapies, they often fail to capture the full spectrum of patient experiences in real-world settings. This is where Real-World Evidence (RWE) and Patient-Reported Outcomes (PROs) play a crucial role in blood cancer advocacy. By leveraging data from diverse sources outside controlled clinical environments, RWE provides a more comprehensive understanding of disease progression, treatment effectiveness, and patient needs. In the healthcare landscape, where public health policies and reimbursement decisions impact patient access to therapies, RWE and PROs can be instrumental in shaping better treatments and policies.

Understanding Real-World Evidence and Patient-Reported Outcomes

Real-world evidence (RWE) is derived from Real-World Data (RWD), which includes patient registries, electronic health records (EHRs), insurance claims, and patient-reported information. Unlike traditional clinical trial data, RWE reflects the experiences of a broader population, encompassing factors like comorbidities, socioeconomic status, and long-term treatment effects.

Patient-reported outcomes (PROs) are a subset of RWE that focus on patients’ self-reported experiences with their disease and treatments. PROs capture elements such as quality of life, symptom burden, treatment side effects, and emotional well-being. PROs are increasingly used to assess patient satisfaction and treatment efficacy in real-world conditions.

The Importance of RWE in Blood Cancer Advocacy

Patient advocacy groups and healthcare policymakers have started to recognize the power of RWE in influencing better care and treatment outcomes for blood cancer patients. Key areas where RWE has an impact include improving access to innovative therapies and enhancing patient-centered Care.

Improving Access to Innovative Therapies

One of the biggest challenges in blood cancer treatment is ensuring timely and equitable access to novel therapies The drug approval and reimbursement process involves multiple stakeholders, including regulatory and health technology assessment agencies. While clinical trial data is crucial for initial approvals, RWE strengthens the case for coverage and reimbursement by demonstrating real-world efficacy and patient benefits beyond clinical trial settings.

For example, RWE has been pivotal in expanding access to targeted therapies, such as CAR-T cell therapy, for patients with relapsed or refractory blood cancers. By showing survival benefits, fewer hospitalizations, and improved quality of life compared to standard chemotherapy, RWE has influenced decisionmakers to prioritize funding for these cuttingedge treatments

Enhancing Patient-Centered Care

The patient’s journey in blood cancer treatment varies significantly depending on factors such as age, genetic mutations, and co-existing conditions. Traditional clinical trials often exclude older patients or those with complex medical histories, leading to gaps in treatment guidance. RWE bridges these gaps by providing insights into how different patient populations respond to treatment in real-world settings.

Influencing Policy and Healthcare Planning

In Canada, where healthcare is publicly funded, policymakers rely on data to make informed decisions about resource allocation and funding priorities. RWE plays a crucial role in shaping policies by providing evidence of unmet needs, treatment gaps, and the costeffectiveness of different interventions. For instance, a study using real-world data may reveal that blood cancer patients in rural communities experience poorer outcomes due to limited access to specialized care. This evidence can drive policy changes such as increased telemedicine services, funding for regional cancer centers, or improved transportation support for patients. Similarly, RWE can support value-based healthcare models, where reimbursement is linked to patient outcomes rather than the volume of services provided. By demonstrating the long-term benefits of specific treatments, RWE helps policymakers optimize healthcare spending for maximum patient benefit.

PROs (patient-reported outcomes) also help healthcare providers tailor treatment approaches based on patient preferences. For example, some patients may prioritize treatments with fewer side effects over those with a slightly higher survival rate. By incorporating PROs into clinical decisionmaking, oncologists can better align treatment plans with individual patient goals.

Advancing Research and Drug Development

Pharmaceutical companies and researchers increasingly rely on RWE to complement traditional clinical trials. In blood cancer research, RWE helps identify treatment patterns, assess long-term safety, and discover new therapeutic targets. For example, post-market surveillance studies use RWE to track side effects and treatment adherence among blood cancer patients. This real-world insight allows researchers to refine therapies and develop better treatment protocols. Furthermore, RWE can help identify subpopulations that respond exceptionally well to specific treatments, leading to more personalized and effective therapies.

Challenges and Considerations in Implementing RWE and PROs in Canada

Despite the many benefits, incorporating RWE and PROs into healthcare decision-making in Canada presents several challenges:

Data Integration and Standardization

Real-world data comes from various sources, including hospital records, patient surveys, and pharmacy databases However, the lack of standardized data collection and integration across provinces creates barriers to its practical use Efforts like the Pan-Canadian Health Data Strategy aim to address these challenges by improving data interoperability and accessibility

Privacy and Ethical Concerns

Patient data must be handled with strict privacy protections under laws such as the Personal Information Protection and Electronic Documents Act (PIPEDA). Patient Advocacy groups must work with policymakers to ensure RWE is collected and shared ethically while maintaining patient confidentiality

Stakeholder Engagement

For RWE to have a meaningful impact, collaboration between patients, researchers, healthcare providers, and policymakers is essential. Patient advocacy organizations play a crucial role in educating stakeholders about the value of RWE and advocating for policies that support its integration into healthcare decision-making.

The Future of RWE in Canadian Blood Cancer Advocacy

The future of RWE in blood cancer advocacy looks promising, with several emerging trends set to enhance its role in improving patient outcomes:

Expanded Use in Health Policy

Artificial Intelligence and Big Data

AI-driven analytics can process vast amounts of RWD to identify patterns, predict treatment responses, and optimize care pathways. Machine learning models can detect early signs of treatment resistance, helping physicians adjust therapies before patients experience severe progression. AI also aids in analyzing large-scale patient-reported outcomes to personalize treatments based on symptom management and quality of life indicators.

Patient-Generated Health Data

Wearable devices and mobile health apps enable patients to contribute real-time data on symptoms and treatment effects, enriching RWE datasets. This allows for continuous monitoring of patient health beyond clinical visits, providing a more holistic view of treatment impact. In Canada, integrating patientgenerated data with electronic health records could bridge gaps in patient care and improve personalized treatment strategies.

As Canadian healthcare moves towards personalized and value-based care models, RWE will become a cornerstone of decision-making. Government agencies are increasingly incorporating RWE into health technology assessments, ensuring policies reflect real-world patient experiences. Additionally, RWE-driven policy decisions will help identify disparities in healthcare access, leading to targeted interventions that improve outcomes for marginalized and underserved patient populations.

Real-world Evidence and Patient-Reported Outcomes are transforming blood cancer advocacy in Canada by providing a deeper understanding of patient needs, treatment effectiveness, and healthcare gaps. By leveraging RWE, advocacy organizations can drive policy changes, improve access to innovative treatments, and promote patientcentered care. As technology and data integration continue to evolve, RWE will play an increasingly vital role in shaping better treatments and policies for blood cancer patients, ultimately improving outcomes and quality of life for those affected by these devastating diseases.

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.

In recent years, significant policy advancements have been made in blood health, including increased investment in rare disease research, improved access to new therapies, and strengthened patient protections. However, continued advocacy is essential to bridge existing gaps, especially for underserved populations. This article explores recent legislative wins, the ongoing challenges in blood health policy, and the ways in which patients and advocates can drive meaningful change.

ThePowerofLegislativeAdvocacyinBloodHealth

Advocacy is a key driver of change in healthcare By influencing policymakers, patient advocatescanhelpsecurefundingforresearch,improvedrugaccessibility,andpushfor policies that protect patient rights Legislative advocacy involves engaging with governmentrepresentatives,regulatoryagencies,andstakeholderstoshapepoliciesthat improvepatientcare

Effectiveadvocacyhasledtomajorpolicymilestonesinbloodhealth,suchas:

Expandedscreeningprogramsforblooddisorders

Fasterapprovalprocessesforinnovativetherapies

Improvedreimbursementpoliciesforblooddiseasetreatments

Thesechangesunderscorethepowerofcollectiveactioninshapingthefutureofblood healthcare.

Recent Legislative Wins in Blood Health

Several key legislative advancements in recent years have positively impacted patients with blood disorders. Below are some noteworthy developments:

The 21st Century Cures Act (United States)

The 21st Century Cures Act passed in 2016 and expanded in subsequent years, has accelerated medical innovation by streamlining the FDA approval process for new treatments, including gene and cell therapies for blood disorders like sickle cell disease and hemophilia. The law has also provided funding for biomedical research, enhancing efforts to develop cures for blood cancers and rare hematologic conditions.

The Rare Disease Drug Development Initiatives (Canada & U.S.)

Canada and the United States have strengthened rare disease frameworks to improve access to life-saving treatments. In Canada, the National Strategy for Drugs for Rare Diseases announced in 2023 that it would allocate significant funding to improve access to new treatments. In the U.S., the Orphan Drug Act continues to incentivize pharmaceutical companies to develop therapies for rare blood disorders.

Expanded Newborn Screening Programs

Many countries have expanded newborn screening programs to include genetic testing for inherited blood disorders such as sickle cell disease and thalassemia. Early detection can lead to timely interventions, significantly improving long-term health outcomes.

Advances in CAR T-Cell Therapy Policy & Reimbursement

Chimeric antigen receptor (CAR) T-cell therapy has revolutionized treatment for blood cancers like leukemia and lymphoma. Legislative efforts in multiple countries have focused on making these therapies more accessible through improved insurance reimbursement and public healthcare coverage. The Ontario government’s recent decision to expand coverage for CAR T-cell therapy is a prime example of policy-driven progress.

Insurance and Affordability Protections

Countries such as the U.K., Canada, and the U.S. have enacted policies to prevent discrimination in insurance coverage for individuals with genetic predispositions to blood disorders. The Genetic Information Nondiscrimination Act (GINA) in the U.S. prevents insurers from using genetic test results to deny coverage, a crucial protection for people at risk of developing blood conditions.

ChallengesThatStillExistinBloodHealthPolicy

Despitetheseadvancements,manychallengesremaininachievingequitableaccessto careforblooddisorderpatients:

DelaysinDrugApprovals:Whileregulatoryagencieshaveacceleratedtheapprovalof breakthrough therapies, some countries still face significant bureaucratic delays, preventingtimelyaccesstolife-savingtreatments.

AffordabilityIssues:Evenwithimprovedreimbursementpolicies,highout-of-pocketcosts continuetoburdenpatients,particularlythoseneedingspecializedtherapieslikeCARTcelltreatmentorgenetherapies.

Inequities in Access: Rural and underserved communities often struggle to access cutting-edge treatments due to geographic barriers and limitations in healthcare infrastructure.

LackofResearchFunding:Whilerarediseaseframeworkshaveimproved,someblood disordersstillreceiveinadequateresearchanddrugdevelopmentfunding.

BloodDonationPolicies:Policiesaroundblooddonationeligibilityhavebeenhistorically restrictiveforcertaingroups,includingindividualsfromLGBTQ+communitiesandthose withspecificmedicalhistories

To address these challenges, sustained advocacy efforts are required to push for policy changesthatprioritizepatientneeds

How Patients and Advocates Can Influence Policy Change

Patients, caregivers, and advocates play a crucial role in shaping blood health policies

Here are some key strategies for influencing legislative change:

Engage with Policymakers

One of the most effective ways to advocate for policy change is to directly engage with lawmakers Patients and advocacy groups can: Request meetings with legislators to share personal stories and highlight policy needs

Write letters or emails advocating for specific bills or funding initiatives

Attend public hearings or town halls to voice concerns about blood health policy gaps

Join or Support Patient Advocacy Groups

Organizations such as the My Blood My Health program, Canadian Organization for Rare Disorders (CORD), National Hemophilia Foundation (NHF), and Leukemia & Lymphoma Society actively work to influence legislative decisions. Becoming a member or supporting their efforts can amplify the collective voice of the patient community.

Leverage Social Media and Public Awareness Campaigns

Social media platforms provide a powerful tool for advocacy. Patients and supporters can: Share personal experiences to humanize policy issues.

Use hashtags like #BloodHealthMatters and #AdvocateForPatients to raise awareness.

Organize online petitions to push for specific legislative changes.

Advocate for Clinical Trial Access and Equity

Many blood disorder patients rely on clinical trials for access to experimental therapies. Advocacy efforts can focus on:

Expanding trial availability in underserved areas.

Ensuring inclusive recruitment practices to represent diverse patient populations. Pushing for policies that improve insurance coverage for trial participants.

Participate in Legislative Advocacy Days

Several organizations host annual advocacy days, where patients and advocates can meet with policymakers to discuss pressing issues in blood health. Events like Rare Disease Day, Blood Cancer Awareness Month, and National Hemophilia Advocacy Days provide key opportunities to influence policy.

LookingAhead:TheFutureofBloodHealthPolicy

As science and medicine advance, so must public policy. Thefutureofbloodhealthadvocacywilllikelyfocuson:

Expanding gene therapy access and ensuring affordability.

Strengthening rare disease frameworks for faster treatmentapprovals.

Promoting global collaborations to align blood health policiesacrossdifferentcountries.

Improving telemedicine services for patients with mobility oraccessibilitychallenges.

Advocating for a patient-first approach in healthcare decision-making.

With continued efforts from the patient community, advocacy groups, and healthcare professionals, future policies can drive more equitable, effective, and innovative solutions for blood healthcare.

Legislative advocacy is a powerful force for improving patient outcomes in blood health While significant progress has been made, continued efforts are necessary to address remaining challenges, such as drug affordability, access inequities, and research funding gaps

By engaging with policymakers, supporting advocacy organizations, raising public awareness, and championing equitable policies, patients and advocates can help shape a future where everyone with a blood disorder has access to the care, treatment, and support they deserve. Now more than ever, your voice matters—join the movement to advocate for better blood health policies today.

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