FDA Launches Multiple Initiatives for Patient-Focused Clinical Trials 
​[Posted on: Thursday, 14 January, 2016] 

Under PDUFA V, FDA committed to obtain patient perspectives on at least 20 disease areas and their treatment options by the year 2017. The purpose was to include patient perspectives on their conditions, its impact on daily life, and available therapies for better patient-focused and patient-centric clinical trials. FDA committed to hold public meetings to provide patients an opportunity to present their comments directly to the regulators. The regulators are expected to use these comments for defining acceptable endpoints in clinical trials and clinical outcome measures. The process started in 2012 with the selection of disease areas for public meetings. FDA collected about 4500 comments regarding 90 disease areas and selected 20 disease areas for detailed public discussion. The primary criteria for choosing the disease areas were debilitating nature of the symptoms, aspects of disease not normally captured in clinical trials, and diseases for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels or functions. FDA created a web-site for patient-focused drug development (PFDD) discussions to organize these public meetings. Since 2013, FDA organized 17 public meetings. Each public meeting led to a “Voice of the Patient” report summarizing the discussion. Another 7 meetings are planned for this year, taking the total to 24 disease areas. Now FDA is taking the discussion to the next level. This week, FDA announced creation of the Clinical Outcome Assessment (COA) Compendium for collating and summarizing COA information for many different diseases and conditions, which could help drug developers review how certain COA were used in clinical trials to measure patient’s experience and to support labeling claims, how COAs were qualified by CDER, and to foster development of COA for unmet measurement needs. The first COA compendium released provides guidance for for 117 different diseases in 15 disease areas. Since this is a pilot program, FDA is collecting public comments in the utility of the COA compendium and promised to keep in updated. In another initiative, acknowledging that there are several more disease areas of interest and that it practically can conduct only so many public meetings before the 2017 deadline, FDA invited patient organizations to identify and organize patient-focused collaborations to generate public input on those other disease areas, using its own process as a model. FDA expects patient organizations to inform it for such externally-led PFDD meetings, generate reports and surveys. Although, FDA may or may not attend such meetings, and the reports generated from such meetings will not be considered FDA-sponsored or FDA-endorsed, the obvious intent is to encourage systematic patient discussions on additional disease areas. FDA did not state how it will use information generated in such meetings but it can be assumed that well-organized meetings will get FDA-support by participation of FDA reviewers and possibly used along with other PFDD reports. The PFDD reports are expected to add to the COA compendium by inserting advice on outcome measure of importance for patients. Assessment of a product’s benefits and risks should involve ongoing patient feedback and not just post-marketing safety and use data. This information should be a critical aspect of FDA’s decision-making by establishing the context in which the regulatory decision is made. By obtaining the patient perspective on disease severity and current available options, FDA believes that it can improve the drug development and regulatory review process. We should see greater clinical relevance discussions taking center stage in FDA review of clinical trial design and clinical outcome assessment.