LAM & TSC Patient-Focused Drug Development Meeting with the FDA

Posted on March 24, 2017   |   

by Steven L. Roberds, PhD, Chief Scientific Officer of the Tuberous Sclerosis Alliance

The TS Alliance, in partnership with The LAM Foundation, is sponsoring a Patient-Focused Drug Development Meeting (PFDD) with the FDA on June 21, 2017, at the Hyatt Regency on Capitol Hill in Washington, DC, from 8:30 a.m. to 4:30 p.m. The purpose of this meeting is for individuals affected by LAM and TSC and caregivers of dependent adults or children to communicate their perspectives on living with LAM and TSC to the FDA. This meeting is free and open to the public, and it will be webcast live online. We hope you will register and make plans to attend in person or online.

As a volunteer advocacy nonprofit organization, this is an outstanding way for our community to have a voice in the development of new potential treatments of TSC,” said Kari Luther Rosbeck, President & CEO of the TS Alliance. “The Patient Focused Drug Development meeting puts our TSC community front and center in sharing how they weigh risks and benefits and what is essential to their or their loved one’s quality of life. The meeting allows our community a unique opportunity to help guide the FDA.

The PFDD meeting will be divided into two parts. The morning will focus on input from parents of children with TSC who have experienced, or are at risk for developing, epilepsy and will be led by Elizabeth Thiele, MD, PhD, head of neurology at Massachusetts General. The afternoon will focus on adults with TSC and/or LAM who have experienced, or are at risk for developing, angiomyolipomas or LAM. The LAM Foundation’s Scientific Director, Frank McCormack, MD, will provide a brief clinical overview of LAM and angiomyolipoma at this afternoon session. The PFDD meeting is designed to communicate to FDA the impacts of LAM and TSC on individuals’ daily lives, what types of treatment benefits make the most impact on peoples’ lives, and individuals’ and caregivers’ perspectives on how well available therapies are working.

The data and information from this meeting will be used in FDA’s risk-benefit analysis of potential new products intended to prevent and treat epilepsy, angiomyolipomas, and LAM in TSC. Additionally, the data and information may be relevant to FDA and researchers designing clinical trials.

This is important because many clinical trials, even those that have led to drug approvals for LAM and TSC, do not capture effects of the disease that directly impact how patients feel or function or the impact of treatment on daily living. Also, as research moves toward the possibility of early treatments to possibly prevent specific manifestations of LAM and TSC from occurring, clinical trials or other methods must capture the risk-benefit considerations of patients (or parents of young patients) including the unknown risk of letting the disease progress vs. the known risks of drug therapies.

The PFDD meeting will be an in-person meeting with a live webcast for remote viewing, including the ability to respond to polling questions in real time. The LAM Foundation and The TS Alliance will identify and invite a small number of affected individuals and caregivers to participate in panel discussions during the meeting. Panel discussions, comments from the audience—in person and online—and polling questions will be facilitated by a former FDA staff member. The meeting is open to the public, and anyone is welcome to attend either in person or via the webcast.

Additionally, surveys will be used to collect a variety of comments and input on priorities from the LAM and TSC communities prior to the PFDD meeting. Immediately after the meeting, a recording of the webcast will be posted and a transcript of the meeting will be developed and posted for people to download and read. A report will also be published summarizing the major outcomes and lessons learned from the meeting. The final meeting report will be submitted to the FDA and posted online. Hardcopies will be made available upon request.

In 2012, a federal law was enacted requiring the FDA to hold patient-focused drug development meetings on a small set of diseases and disorders. According to the FDA’s website, these PFDD meetings enable FDA to learn directly from individuals about “the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application.”

Federal funding for the PFDD program enabled the FDA to hold 25 such meetings over a five-year period, which is a very small portion of the thousands of diseases that affect human beings. In response to public requests for meetings on additional diseases and disorders, the FDA developed a process for externally-led PFDD meetings. This is the process by which The LAM Foundation and the TS Alliance are hosting this externally-led PFDD meeting for TSC.

If you’d like to attend this meeting in person or online, RSVP at

For additional questions contact Anne McKenna, Patient Services & Education Manager at 513-777-6889 or by email at

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