Advocacy groups crowdsourcing patient views on risk-benefit of drugs

The reauthorization of PDUFA committed the FDA to giving patients a stronger voice in regulatory decisions, particularly when assessing the benefits and risks of new drugs. The FDA is planning a series of meetings, and Genetic Alliance thinks it's found a way to accelerate the gathering of patient feedback--crowdsourcing.

Nonprofit health advocacy group Genetic Alliance has worked with PhRMA and Private Access to create an online platform for gathering patient perspectives on the benefits and risks of medicines. A vanilla version of the platform, called Reg4All, is already online and asks users to complete a series of questions about their health, such as, "how concerned are you about health information privacy?" Genetic Alliance is customizing this framework to suit the needs of its 7 disease-specific partner organizations, which span sickle cell, idiopathic pulmonary fibrosis and irritable bowel syndrome.

Each partner organization will have a version of the platform with questions tailored to its community. By making the survey available on their websites and encouraging members to participate, Genetic Alliance thinks its partners can gather information that will help in future meetings with the FDA. "Using the Platform for Engaging Everyone Responsibly (PEER), there is an opportunity to demonstrate the power of a secure, crowdsourced approach to provide additional insight into patients' experience with a disease or condition," Genetic Alliance CEO Sharon Terry said.

The FDA is holding a meeting to discuss sickle cell in February, and talks about idiopathic pulmonary fibrosis and irritable bowel syndrome are also in the pipeline. In total, the FDA plans to hold 20 disease-focused talks to establish which therapeutic areas will benefit most from systematically incorporating the patient perspective on unmet medical need. Armed with the crowdsourced feedback, the advocacy groups plan to make a data-driven case for their areas of focus being picked by the FDA. Ultimately, this could see the FDA become less risk-averse in their disease areas and help patients receive new drugs sooner. 

- read the press release
- view the project overview
- check out the survey