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Cover Letter
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October 26, 2017
Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research (CDER) Dr. William Dunn, Director, Division of Neurology Products, CDER Dr. Patricia Keegan, Director, Division of Oncology Products 2, CDER Dr. Theresa Mullin, Director, Office of Strategic Programs, CDER Dr. Jonathan Goldsmith, Associate Director, Rare Disease Program, CDER Food and Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20993 via digital transmission Dear Drs. Woodcock, Dunn, Keegan, Mullin, and Goldsmith: The Tuberous Sclerosis Alliance (TS Alliance) is pleased to submit the attached Voice of the Patient report arising from our Externally-Led Patient-Focused Drug Development Meeting on tuberous sclerosis complex (TSC) and lymphangioleiomyomatosis (LAM) held June 21, 2017, in Washington, DC. It is our hope you will distribute this document to the appropriate FDA staff for use in regulatory decision-making and include it in the public collection of externally-submitted Voice of the Patient reports under PDUFA VI. We specifically encourage sharing within the Division of Neurology Products, Division of Oncology Products 2, and the Rare Disease Program. The TS Alliance deeply appreciates the broad support this Externally-Led Patient-Focused Drug Development Meeting received across FDA, especially within CDER. We are grateful for the active participation of Drs. Woodcock, Dunn, Bastings, Donoghue, and Goldsmith, who invested valuable time, experience, and insight to make the meeting a success. This meeting was enabled by acceptance by the Office of Strategic Programs on September 30, 2016, of the TS Alliance’s Letter of Intent submitted on May 31, 2016. Subsequent conversations with Office of Strategic Programs personnel were very helpful to us for planning an effective meeting. Ninety-eight participants joined the meeting in person, including patients, family members, healthcare providers, industry representatives, National Institutes of Health and FDA staff. The live webcast had 666 views from 27 countries. Most livestream viewers were from the United States (72% of views), followed by Israel (4.8%), Guatemala (3.8%), Serbia (3.3%) and India (2.6%). Through September 2017, the archived video recordings of the livestream on YouTube
had been viewed more than 290 times. Video recordings and the Voice of the Patient report are available and will be maintained for future reference at www.tsalliance.org/pfdd. Heartfelt testimony and discussions on June 21 vividly portrayed the clear need for improved treatments for TSC and LAM—a disorder caused by somatic mutations of TSC genes—and the willingness of patients and caregivers to work with researchers and regulators to accelerate the development of new treatments. Following the meeting, the 30-day post-event public comment portal received 60 open-ended responses, 85% from the United States. Additionally, in advance of the meeting, the TS Alliance conducted an international survey in English, Spanish, and French to gather broadly representative data relevant to the topics to be discussed at the meeting. The survey received 1,309 responses from 57 countries, 66.5% of which were from the United States. Survey data are included in the Voice of the Patient report, providing a quantitative data set to complement the rich descriptions of the impact of TSC and LAM best conveyed through personal testimony. We hope that FDA will use this Voice of the Patient report in its benefit-risk analysis when evaluating new or repurposed treatments for TSC. We believe the data and testimonies, taken together, will best help FDA staff understand the needs of the TSC and LAM community. In the report, we have also included a draft of a Structured Benefit-Risk Assessment Framework to help start the process. Thank you again for your participation, without which this meeting could not have been successful, and for your ongoing support and partnership. As you review the attached Voice of the Patient report, feel free to contact us with any comments or questions. We would be more than happy to address them. The TS Alliance sincerely appreciates your commitment to improving the lives of those affected by TSC. We will give everything. But up. With gratitude,
Kari Luther Rosbeck President and CEO
Steven L. Roberds, PhD Chief Scientific Officer
FDA attendees included: Dr. Eric Bastings, Deputy Director, Division of Neurology Products, CDER Mr. Larry Bauer, Regulatory Scientist, Rare Diseases Program, OND, CDER
Dr. Suzanne Demko, Team Lead, Division of Oncology Products 2, CDER Dr. Martha Donoghue, Acting Associate Deputy Director, Division of Oncology Products 2, CDER Dr. Billy Dunn, Director, Division of Neurology Products, CDER Ms. Andrea Furia-Helms, Director, Patient Representative Program, Office of Health and Constituent Affairs, FDA Dr. Jonathan Goldsmith, Associate Director for Rare Diseases, Office of New Drugs, CDER Dr. Paul Kluetz, Acting Deputy Director, Office of Hematology and Oncology Products, CDER Dr. Debra Lewis, Deputy Director, Office of Orphan Products Development, FDA Dr. Heidi Marchand, Associate Commissioner for Health and Constituent Affairs, FDA Dr. Gayatri Rao, Director, Office of Orphan Products Development, FDA Dr. Joohee Sul, Medical Officer, Division of Oncology Products 2, CDER Dr. Pujita Vaidya, Operations Research Analyst, Office of Strategic Programs, FDA Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research Submission: Voice of the Patient report