In August 2017, the National Consumers League, WomenAgainstAlzheimer’s, AARP, National Association of Nurse Practitioners in Women’s Health, and several other women’s health advocacy organizations met with Dr. Janet Woodcock, FDA’s Director of the Center for Drug Evaluation and Research (CDER), to discuss treatments for Alzheimer’s Disease (AD).
We requested this meeting because of concerns about Alzheimer’s disproportionately affecting women, with two-thirds of the patients diagnosed and the vast majority of caregivers being female, and the unfortunate fact that no new treatments for this terrible disease have been developed and approved in the past 15 years.
The group discussed the need for updating the agency’s Guidance on AD, and Dr. Woodcock indicated that this Guidance would be a good candidate for review. We were pleased that FDA Commissioner Scott Gottlieb, speaking at the September 13, 2017 Blueprint for Breakthroughs meeting sponsored by Friends of Cancer Research (FOCR) and Alexandria Real Estate Equities, confirmed the agency’s intent to revise the Guidance on AD.
As reported in The Pink Sheet, the FDA Commissioner made the following observations at the FOCR meeting:
- We don’t have as many disease-specific guidelines as you would expect on the new drug side.
- The FDA is planning a revised guidance on Alzheimer’s drug development, part of a broader reform of the Office of New Drugs (OND).
- The OND development is being spearheaded by Center for Drug Evaluation and Research Director Dr. Janet Woodcock. As Acting Director of OND, Dr. Woodcock is moving to implement policy and structural changes for OND, including re-evaluating the division-by-division staffing model to determine whether staff can be trained to move between review divisions as needs arise. Dr. Woodcock has cited the need for more policy development, including guidance documents, to ensure that product sponsors receive the most up-to-date advice.
- As part of the OND revamp, Gottlieb said, “we want to develop more time for reviewers to work on specific guidance documents that will help address certain aspects of the review process where we think that the science has evolved to a point where we want to create more modern standards.”
- The disease-specific guidance writing effort is an attempt to go back and look at diseases where there hasn’t been a lot of innovation in different aspects of clinical development, where the FDA hasn’t articulated principles, with the forthcoming documents addressing issues such as use of different trial designs and endpoints.
- The guidance-writing initiative is intended to articulate modern evidentiary and clinical trial design expectations in specific disease areas where the science has evolved.
- The goal, Gottlieb said, is to “create more incentives for people to try to attack these unmet medical needs because we have clear direction that they can shoot against.”
- Gottlieb explained: The new Alzheimer’s guidance will “look at some of the new modalities and points that could become maybe not a primary endpoint, but perhaps secondary and tertiary endpoints, or how you look at cognitive function versus performance as different endpoints. Those are now dual endpoints in a lot of clinical studies.” With the guidance writing effort, “there’s going to be some things that we’re going to update because the science has changed. In most cases, we’re doing these things anyway now in terms of how we’re thinking about these different therapeutic areas, but we just haven’t had the opportunity to articulate the standards in guidance, and that’s what this exercise is for.”
Our coalition is very appreciative of Dr. Woodcock’s time and accessibility for discussion of these issues. Her leadership is renowned. We also appreciate FDA Commissioner Gottlieb’s commitment to developing Guidances to ensure more clarity and direction as the medical and scientific community works to develop safe, effective, and affordable treatments for diseases like Alzheimer’s and so many others.