Agency releases a series of new guidances to advance the reliability of patient-focused tools and methods.
Ten years ago, FDA launched an initiative to expand the role of patients in informing regulators and sponsors on most harmful effects of a disease and primary goals of treatment that can lead to desirable medical approaches. To further advance and refine the process, the agency is highlighting how the patient-focused drug development (PFDD) program has encouraged active involvement of many patient groups in bringing their perspectives into the process of developing, testing, and approving new medicines.
The primary goal of PFDD is to “better incorporate the patient’s voice in drug development and evaluation,” says Theresa Mullin, associate director for strategic initiatives in the Center for Drug Evaluation and Research (CDER), who has led this effort since its establishment. The initial program featured FDA-led meetings but was expanded to include a wider range of sessions organized by patient groups themselves, Mullin explained in a webinar sponsored by the Alliance for a Stronger FDA on Sept. 16.1
The PFDD initiative was established by legislation reauthorizing the Prescription Drug User Fee Act (PDUFA) in 2012. It called for FDA to organize 20 meetings each year to discuss patient experiences, which symptoms have the most significant impact on daily life, and how well current therapies treat those symptoms.2 The program grew to 24 meetings in the first year as more groups recognized the benefits of hearing patient perspectives on disease severity and on potential effects of treatment. Then in 2016, PFDD further expanded to include discussions organized by patient groups. There have been 55 of these externally led meetings, which follow the format of the agency-organized meetings in being open to the public and producing detailed reports to post on the FDA website.
Every one of these meetings “was very powerful,” Mullin commented, noting that physicians at FDA said that they had never heard these perspectives and that reviewers found that patient comments helpful in assessing the risks and benefits of a new therapy. And even though patients did not advocate for any particular therapies, they discussed the impact of available treatments and where new therapy could have an impact, a process that supported the development of new treatments for sickle cell disease complications.
To further improve the PFDD process, FDA has issued a series of guidance documents to advance the reliability of PFDD tools and methods. Two final guidances address how to collect comprehensive and representative input from patients and caregivers3 and how to assess which aspects of disease burden and treatment are most important to patients.4 A third draft guidance outlines processes for selecting, developing, or modifying clinical outcome assessments.5 Still to come is a fourth guidance on incorporating clinical outcome assessments into endpoints to support regulatory decision-making. These advisories aim to encourage manufacturers and researchers to utilize high-quality methods for collecting patient experience information that will be useful in regulatory decisions—and to move away from multiple differing efforts for developing clinical assessment measures and tools that repeat the PFDD process with every manufacturer.
In looking to the future, Mullin envisions some kind of “national catalog” of standard core clinical outcomes and assessments based on patient perspectives. This would provide reliable measures of clinical outcomes for disease areas that are meaningful to patients and could gain support from FDA’s Standard Core Clinical Outcome Assessments Grant Program. The overarching aim is to ensure that clinical trial protocols are reasonable and accessible for patients and that clinical outcome assessment measures compare things that people care about.