“I am a cancer survivor, I was treated for cancer during my last tour at FDA, so I k now the importance of what American medicine does — and what the FDA does — for every one of us.”
Scott Gottlieb, M.D., April 5, 2017 – Senate Confirmation Hearing
Three decades ago patient involvement in the development and approval process for new medicines was virtually non-existent, except of course for being the study subjects in clinical trials. The AIDS epidemic drove a wedge in the system that changed that. Many HIV/AIDS activists, while not scientists, were extremely familiar and aware of the clinical aspects of the approval process and were highly conversant in science.
This week marks a new milestone in the arc of progress that has been patient involvement since that time. FDA announced in a blog posting on FDA Voice, and in a Federal Register notice, that the inaugural meeting of the Patient Engagement Advisory Committee (PEAC) will be held in October. The first meeting will take place on October 11 and 12 and the agenda is focused on getting input into getting patient input into medical device trials.
The current roster of the committee has 9 members, two of whom are from disease-specific organizations – the American Association of Kidney Patients and the Arthritis Foundation. Other member slots represent individuals who work in the area of patient engagement. At least one person appears to be experienced in minority outreach, but unlike other FDA Advisory Committees, the CV’s of the members are not yet linked to their names on the current roster listing.
The launch of the committee is an important step, but it is important to note that it is one step in a much longer journey. It is important for FDA to connect the dots so that all the stakeholders – patient advocates, researchers and companies involved in the development of new medicines – understand how the input from patients is going to be integrated into practice. The Regulatory Affairs Professional Society reported in April of this year that Dr. Gottlieb has been an advocate of the use of Patient Reported Outcomes as endpoints in clinical trials – is there a committee role in making that happen and how does it fit into a larger process for achieving that goal?
Also, as FDA is well aware, the inauguration of such a committee is only part of the bigger picture to gain patient involvement. One committee of 9 cannot represent the entire spectrum of input from the vast array of conditions and diseases that need attention. The committee will have to turn its attention to issues related to rare diseases and to extremely important matter of the role of real life patient experience into the assessment of new drugs. The complexity and range of issues means that FDA will have to have a broader system in place beyond the development of the committee as well as broader input into the workings of the committee to extend it beyond the current membership. This committee meets a few times a year and has a two-year remit. The scope of work is bigger than that.
The agency has been making strides, particularly in devices, in getting patient input. Since 2013 FDA has been involved in disease/condition-specific meetings to facilitate Patient-Focused Drug Development. The launch of the committee is an important milestone, but there will need to be others and a perhaps most importantly a holistic approach to patient input needs to be articulated to understand how the efforts at increasing the conversation with patients results in solid outcomes. The dots need to be connected. Discussion about enhanced patient involvement and patient reported outcomes have been under discussion for years – it is time to move to greater implementation. It is good that the committee is established. Now, less talk, more action.
