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  4. Developing Therapies for Primary Mitochondrial Diseases: Bridging the Gaps - 09/06/2019 - 09/06/2019
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Event Title
Developing Therapies for Primary Mitochondrial Diseases: Bridging the Gaps
September 06, 2019

September 06, 2019
08:00 AM - 04:00 PM EDT
Event Location
White Oak Campus: The Great Room
Conference Center

10903 New Hampshire Ave
Building 31, Room 1503
Silver Spring, MD 20993
United States

Mitochondria Images

Given the complexities of primary mitochondrial diseases and, by extension, the challenges of designing informative clinical trials in this group of rare diseases, this scientific symposium will bring together academic physicians, FDA regulatory experts, and experts from related disciplines to exchange ideas on ways to address the challenges and to promote drug development in this challenging field.


Persons interested in attending this public workshop either in-person or by web viewing must register on-line by 5 pm on September 5, 2019.


8:00 - 8:05 am  Welcome/ Introduction | Dragos Roman, FDA

8:05 - 8:30 am  Integrating Primary Mitochondrial Disease Biology into Designing Clinical Trials | RobertNaviaux, UCSD

8:30 - 9:00 am  Lessons Learned from Previous Drug Development Programs | Jim Carr, Stealth & Matt Klein, BioElectron

9:00 - 9:45 am  Leveraging Natural History Knowledge to Design Informative Clinical Trials | Michio Hirano, Columbia University/ Philip Yeske, UMDF / Anita Zaidi, FDA

9:45 - 10:15 am  Patient Population Selection for Clinical Trials: Lumping vs Splitting, Pediatric Considerations | Amel Karaa, MGH & Melanie Bhatnagar, FDA

10:15 - 11:00 am  Panel Discussion, TBD

11:00 - 11:45 am  Open discussion

11:45 - 1:00 pm  Lunch

1:00 - 1:45 pm Defining and Assessing Clinical Benefit: Regulatory, Scientific, and Patient Perspectives | Sophia Hufnagel, FDA | Bruce Cohen, Akron Children’s Hospital & Phil Yeske, UMDF

1:45 - 2:15 pm  Panel Discussion, TBD

2:15 - 2:45 pm  Clinical Trial Design and Statistical Considerations | Frank Sasinowski, Hyman, Phelps & McNamara, P.C./ Jan Wang, FDA

2:45 - 3:15 pm  Panel Discussion, TBD

3:15 - 3:45 pm  Open Discussion

3:45 - 4:00 pm  Summary and Next Steps


Webcast is available the morning of the workshop. Please register and the webcast link will be emailed the day before the workshop. You must register before noon September 5, 2019

*FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with physical disabilities or special needs. If you require special accommodations due to a disability, please contact the Project Manager at least seven days in advance of the meeting.


Laurie-Anne Sayles, Project Manager
Center for Drug Evaluation and Research
Office of New Drugs
Public Meeting & Symposia Project Management Team