RWE Comments

February 5, 2019

Division of Dockets Management (HFA-305)US Food and Drug Administration5630 Fishers Lane, Rm. 1061Rockville, MD 20852Re: Docket No. FDA-2018-N-4000 for “Framework for a Real-World Evidence Program.”Submitted electronically at www.regulations.govTo Whom It May Concern:The Global Healthy Living Foundation (GHLF) appreciates the opportunity to provide comments on the draft guidance entitled “Framework for a Real-World Evidence Program” published in the Federal Register on December 7, 2018. GHLF is a 501(c)(3) patient advocacy organization that works to improve the quality of life for people living with chronic disease by making sure their voices are heard. GHLF represents more than 100,000 chronically ill patients and their caregivers across the country. GHLF is pleased that the Food and Drug Administration (FDA) recognizes the value of using data collected during regular patient care and is taking steps to provide practical guidance on how medical products can use real-world evidence (RWE) in the evaluation of a product during its entire life cycle. With therapeutics, sometimes safety information may only emerge in real-world settings through patients who are using these productsas opposed to in efficacy trials or in phase 4 trials and early approval pathways. Throughits patient-centered drug development efforts, FDA has already worked to incorporate the patient perspective in the review of medical products, and we believe that use of RWEand this framework,will further enhance the ability of both FDA and manufacturers to incorporate patient feedback. We agree that disease-specific real-world data (RWD) sources sponsored by patient advocacy organizations such as our research registry and affiliated application (“app”), ArthritisPower, can be used to collect patient-reported outcomes and can serve as an important information resource in this capacity. Disease-specific registries and patient-reported data collected by non-profit organizations like ours have served as valuable tools in other government efforts associated with the incorporation of the patient perspective into the discovery, development, and delivery lifecycle of a medical product. As an example, the National Patient-Centered Clinical Research Network (PCORnet) is aimed at incorporating the experiences and concerns of patients through standardized measures and instruments that can be used by clinicians at the point of care to help inform decision making, and we have been honored to contribute to this important initiative through our ownPatient-Powered Research Network (PPRN), ArthritisPower. Our app allows patients with rheumatoid arthritis, lupus and other rheumatic and musculoskeletal disease to actively engage in multi-stakeholder patient-centered health research aimed at advancing the understanding of musculoskeletal and rheumatic conditions while monitoring their disease journey. The primary mission of ArthritisPower is to generate data for research through collaborations with patients, researchers, clinicians and otherhealth care stakeholders. We do this by providing curated patient input, patient-generated data, linkage to other data sources (i.e., EHR, claims) and leadership in designing and conducting projects and disseminating results. A crucial value of patient-reported data is in understanding how patients respond to treatments over time and we are pleased to report that ArthritisPower is already actively collecting patient-generated data that could be useful in regulatory decisions: patient-reported outcomes (PROs), biometrics, patient reports of symptoms, health and treatment history. We appreciate Page2that FDA has acknowledged and agrees that relevant and reliable RWD could include PRO measures when fit-for-purpose.We understand that RWD sources such as registries sponsored by patient advocacy organizations have the potential to be tremendously useful but are not necessarily designed for regulatory purposes from the outset. We are pleased that the framework does reference the work of PCORnet and registry data as suitable for use in robust randomized trials in its summary of current examples. We believe research using RWD could examine both intended and unintended factors related to patient use of atherapyincluding convenience, emotional and psychological effects, adherence, and other factors that we, as a patient organization, consider important. We are confident that working with established and activated patient communities represents a cost-effective way to collect highly relevant and reliable RWD. Our goal is for ArthritisPower to serve this purpose and,therefore, we would like additional information about how patient organizations can prepare to meet the RWDneeds of FDA and medical product manufacturers.Specifically, we would welcome the opportunity to meet with you to identify the metrics FDA will use to evaluate the effectiveness and quality of RWD so that we can better anticipate necessary modifications as we continue to build and expand ArthritisPower. qThank you for your work on this draft guidance and we look forward to assisting you in incorporating patient-generated RWDinto FDA’s regulatory approach. We are ready to help any way we can. For questions regarding GHLF or the above comments, please contact me at [email protected]. Sincerely,Steven NewmarkPolicy Director and General Counsel Global Healthy Living Foundation