FDA Expects Patients to Play a Bigger Role in Drug Development
[Posted on: Thursday, June 09, 2016]

Patient perspectives could be vastly different from that of the drug developers and regulators in terms of the benefits and risks of existing therapies, the medical need and relevant endpoints for clinical trials. In the recent years, FDA has aggressively courted patients to provide opinions about the regulatory review process through its Patient Focused Drug Development (PFDD) initiative. FDA would like the industry to similarly involve patients in new product development programs. FDAs commissioner, Dr. Califf, emphasized the importance of industry collecting patient perspectives at the BIO meeting in San Francisco. Historically, drug development programs at industry are developed based on internal R&D, and information collected from public sources about diseases, their effects on patients, and potential medical need. Patients had been passive participants whose opinions did not play a significant role in clinical trial design, relevant data elements, risk tolerance matrices, and prioritization of development milestones. This should change. Patients can educate the industry about their experiences with existing treatments, what treatments to develop, and their tolerance for risk. In the past, industry relied to physicians to provide this information but according to FDA, patients could be the direct source of such information. FDA itself values this feedback and plans to use it fine tune its review processes to meet patient needs. It encourages the industry to do the same. There are other obvious benefits of this such as increased recruitment in clinical trials, higher compliance with trial procedures, lower drop-out rate for patients enrolled in trials, and rapid market access once approved. While the merits of this suggestion are obvious, there are some notes of caution both for the FDA and industry. Patient interest is highly emotional in nature and creates challenges when the drug with which they were involved does not meet the scientific merits. There have been instances where patients lobbied company to start compassionate use programs for patients who did not meet the entry criteria for a clinical trial or other reasons. Companies had been lobbied to keep programs alive that did not make business sense or lose public image. Patients have also pushed companies to provide drugs for extended periods of time when regulatory approvals were delayed. On the FDA side, we have seen increasing episodes of activist patient groups pushing FDA to reduce its review integrity to approve products with dubious supporting data. It is not uncommon that a given drug with unacceptable results in clinical trials, getting glowing recommendations from patients at Advisory Committee meetings and other public forums. Increased patient involvement is inevitable but we need to clear guidelines from FDA for acceptable practices. FDA’s PFDD program is a good template for industry to follow. Hope it does.