1. PATIENT FOCUSED DRUG DEVELOPMENT MEETING
Gabrielle Rushing
March 2nd, 2017

2. The fda wants to understand the patient perspective
Providing the FDA an insight into daily living for individuals affected by TSC/LAM
The TS Alliance is sponsoring an externally-led PFDD just prior to this year’s International Research Conference on TSC and LAM
Wednesday, June 21st, 2017
8:30 AM – 4:30 PM
Hyatt Regency on Capitol Hill, Washington DC
Both the patient perspective and the family/caregiver perspective
FDA sponsors 25 of these meetings over a 5 year period ( )

3. Who will be there? Individuals with TSC and/or LAM or their caregivers
Invited panel members
Attending in person
Participating via live web broadcast
FDA Staff
Office of New Drugs
Office of Health and Constituent Affairs
Office of Orphan Products Development
Center for Drug Evaluation and Research

4. What will be discussed? The PFDD will be separated into two parts:
Epilepsy
AML/LAM
Within these topics, we will address:
Impact of these symptoms on an individual’s daily life
Most impactful treatments
How well treatment is working
The TS Alliance has given out a survey to collect a variety of comments and input on priorities from the TSC community and will continue to collect more responses. This Tuesday, we met with a small group to discuss what types of questions should be addressed at the meeting and so that we can truly focus on what affects patient’s and caregivers day to day.

5. EXAMPLE SURVEY Questions
Which 1-3 symptoms have the most significant impact on life?
Are there specific activities that are important that those affected cannot do at all/as fully as they would like because of their condition?
How have their symptoms changed over time? What makes them better or worse?
How are you currently treating the symptoms? Has the treatment regimen changed over time?
How well do these treatments improve the ability to perform specific activities?
What are the downsides to current treatments?
Pre-specified answers on polling questions.

6. How will this be helpful to affected individuals?
The “Voice of the Patient” (VOP) report
Summary of PFDD findings
Submitted to the FDA
Available online
Informs the FDA’s decisions on drug development and review of market applications
This is a very important consideration as many clinical trials do not capture the effects on how patient’s feel or function day to day as a whole (example: one of the current epilepsy treatments, affinitor, although effective has adverse effects).

7. PFDD design Introduction to TSC and treatments
Session 1: Epilepsy in infants with TSC
Session 2: Angiomyolipomas (AML) and lymphangioleiomyomatosis (LAM) in adults with TSC
Topic 1: Living with TSC (disease symptoms/daily impacts that matter most to patients)
Panel discussion and facilitated large group discussion
Topic 2: Current & future approaches to treating TSC

8. What happens after the meeting?
Webcast recording
Downloadable transcript
The Voice of the Patient Report

9. How will this help the development of new therapies?
FDA Risk-Benefit Analysis
Analysis of condition and current treatment options
Potential ‘early intervention’ studies to potentially prevent specific manifestations of TSC
Basic structure of a benefit-risk framework that includes the following categorization of key decision factors: Analysis of Condition, Current Treatment Options, Benefit, Risk, and Risk Management.
Analysis of Condition and Current Treatment Options provide a summary and assessment of the severity of the condition that the product is intended to treat and other therapies available to treat the condition. This represents the context of the decision that can provide useful information for weighing the benefits and risks of the drug under review.

10. Example: early intervention clinical trial
Preventing Epilepsy using Vigabatrin in Infants with Tuberous Sclerosis Complex (PREVeNT) trial- began December 2016
Goal: To test whether early treatment with Vigabatrin before the onset of seizures in TSC infants having an abnormal EEG biomarker can prevent epilepsy and improve developmental outcomes

11. Questions?