According to a story from the US Food and Drug Administration (FDA), Dr. Amy Abernethy, who is the current Prinicipal Deputy Commissioner at the agency, was eager to start working there. However, it didn’t take long for her to realize that there were ways in which the perspective of patients, particularly rare disease patients, could improve the impact and effectiveness of the FDA’s operations.
If the FDA is going to conduct its mission successfully, which includes tasks like advancing regulatory science and overseeing the development of new medications and other medical products, then feedback from the people that are greatest affected by disease is a necessity. This includes not just patients, but their caregivers as well. Dr. Abernethy has noticed that the inclusion of these perspectives and feedback has not always been achieved.
As an example, not all research projects are directly addressing patient concerns. Another concern that has gotten attention more recently are clinical trial endpoints; these endpoints are critical for determining if a new therapy is having a useful effect, but sometimes these endpoints simply don’t square up with the problems that a patient is actually facing. This leads to the approval of drugs that appear effective in the clinical setting but actually aren’t that helpful in the real world.
Incorporating Rare Disease Patient Perspectives
A discussion with FDA officials about including patient feedback was recently held at the most recent summit of the National Organization for Rare Disorders (NORD), as this problem is perhaps most pronounced for rare diseases, where research, treatment, and other resources are often scarce. A part of the agency’s efforts to include patient perspectives also include the Rare Disease Patient Listening Sessions, which took place as a pilot program over the last year. There were a dozen of these sessions held in total.
More sessions are currently scheduled for 2020. These sessions represent the latest effort from the FDA to be more inclusive of the patient perspective and the rare disease community as a whole, which has had a contentious relationship with the agency at times.
To learn more about the FDA Patient Listening Sessions, click here.
