The Role of Real-World Evidence in Blood Cancer Advocacy

The Role of Real-World Evidence in Blood Cancer Advocacy explores how real-world 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 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.

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),whichcaptureshowtherapiesperform outsidetheconfinesofrandomizedclinicaltrials(RCTs), is playing an increasingly crucial role in supporting this paradigmshift. 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.

DefiningReal-WorldEvidenceinContext

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, including 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.

Why RWE Matters in Blood Cancer

Blood cancers are complex, heterogeneous, and frequently rare in their subtypes, making RCT 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 a cutaneous 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 patient-centred perspectives.

RWE as an Advocacy Catalyst

Blood cancer advocacy has historically been rooted in storytelling, giving voice to those facing uncertain futures and 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 real-world 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.

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 RW 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, low-income individuals, and marginalized communities, then RWE risks perpetuating existing inequities.

4. Industry Collaboration:

Advocacy groups working with pharmaceutical companies to generate RE 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 design protocols that include real-world endpoints, such as days spent at home, emotional well-being, and treatment burden.

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, 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.