Incorporate the patient voice in real world studies to improve outcomes for patients and caregivers
The U.S. Food and Drug Administration (FDA) has increasingly emphasized the importance of real-world data and real-world evidence in regulatory decision-making, as highlighted in recent FDA Guidances. For patient organizations and research foundations, this evolution in the FDA’s perspective offers new opportunities to serve as the liaison between patients, the healthcare industry, and regulators – centered around patient data.
Patient organizations can work with the FDA to be more receptive to real-world studies in several ways. These include helping the FDA understand the unmet need in specific patient populations, the well-defined natural history of diseases, and the richness of existing real-world data (RWD) sources, such as claims and electronic health records (EHR) data, for their patient populations. This role by these organizations can enable new and strategic partnerships with pharmaceutical and medical device companies that are targeting their specific therapeutic areas.
For organizations that represent patients with rare diseases or cancer, the ability to engage with regulators on real-world studies is particularly valuable, as designing and conducting clinical trials for these populations often proves challenging. By taking advantage of these opportunities, patient organizations can accelerate the development of life-changing therapies, improve quality of life for their patient communities, and further their missions of advocacy, education, and support of patients.
Historically, the FDA has shown a commitment to working with patients and patient organizations. Often, this collaboration came in the form of communication about the specific challenges a patient community may face in accessing care or therapies, such as patients with rare diseases.
Drug development for rare diseases presents several barriers: first, by nature, a relatively small number of people are affected by any given rare disease, making recruitment for clinical trials cumbersome and expensive. Additionally, prospective therapeutics generally lack funding and precedents or analogs in terms of previously approved products.
However, a recent move by the FDA to establish a Rare Disease Innovation Hub highlights the agency’s greater eagerness to work with patient groups. The Hub focuses on developing therapies for rare diseases, with the goal of leveraging lessons learned across the previously siloed regulatory precedents and decision-makers into a more cohesive “hub.”
A critical aspect of the Hub is that it provides an avenue for patients and patient organizations to collaborate and engage with the FDA. At this point, it is unclear exactly what this engagement will look like or how it will work – patient portals or direct engagement with FDA personnel, perhaps – but, in theory, at least, it will allow for a more centralized approach to sharing patient voices with regulators.
Separately, another FDA initiative, the Patient Engagement Collaborative (PEC), also illustrates the FDA’s new focus on obtaining patients’ perspectives. The PEC is a collaborative effort in which the patient community, FDA, and Clinical Trials Transformation Initiative discuss ideas for improving communication, education, and patient engagement related to medical product regulation.
The FDA’s recent changes present a new opportunity for these organizations to engage with FDA or partner with industry to engage FDA to discuss opportunities to use real-world data sources—such as their own patient registries or data assets—to generate evidence supporting the development of drugs, biologics, and medical devices for their communities.
For example, patients with rare diseases or cancers may use drugs off-label or through an expanded access program (EAP) due to a lack of targeted therapeutics. These off-label use data or EAP data may often be captured in real-world data sources such as registries, EHR, and claims. Patient organizations could potentially work together with the pharmaceutical industry and the FDA to discuss if it would be feasible and acceptable to conduct real-world studies to supplement or enhance clinical trials. These studies could support label updates and new approvals to improve patient access and reimbursement of these drugs.
Despite the growing precedent of using real-world data for regulatory approvals, conducting clinical trials remains the gold standard in drug development. However, in certain patient populations, the unmet need, infeasibility of conducting a clinical trial, and well-defined natural history of the disease may enable the use of real-world studies to generate substantial evidence for an FDA approval. While not always the case, real-world studies may allow for faster and cheaper ways to get a drug, biologic, or medical device approved for patients.
For patient organizations seeking greater engagement with the FDA, here are a few tips to get started:
Conclusion
Today, patient organizations have an unprecedented ability to influence regulatory decision-making. This influence may start with patient organizations leveraging their own real-world data in the form of patient registries to support real-world studies. By owning their data sources, patient organizations can move beyond understanding the patient experience to informing regulatory decisions that expedite delivery of life-changing therapies to their patients.
To learn more about recent FDA guidance for patient organizations or how patient organizations could engage with FDA, please reach out to the Regulatory Science and Strategy Team: regulatoryscienceandstrategy@iqvia.com.
Incorporate the patient voice in real world studies to improve outcomes for patients and caregivers
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