DIA/FDA Statistics 2016 Forum

Now in its tenth year, the Forum fosters open discussion of timely topics of mutual theoretical and practical interest to biostatisticians and clinical trialists.

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Session 7: Innovative Designs and Strategies for Rare Disease Clinical Trials

Session Chair(s)

Brenda Crowe, PhD

Associate Vice President, Statistics

Eli Lilly and Company, United States

Cristiana Mayer, DrSc, PhD

Head of Biostatistics

Johnson & Johnson Vision, United States

Yeh-Fong Chen, PhD

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER

United States

Approximately 30 million people worldwide are affected by a rare disease. However, of the nearly 7,000 rare diseases, only about 400 have therapies. Given the limited number of patients with a specific rare disease, enrolling patients in clinical studies and drawing conclusions about the efficacy and safety of a treatment can be challenging. One possible solution is to adjust the general standards in clinical trials that apply well to common diseases for rare diseases, but there is still no consensus on the specifics that can ensure both the quality of good trials and the efficacy of the approved drugs. In this session, three experts from public and private sectors will present their research, challenges, lessons learned and possible solutions.

Speaker(s)

Rare Diseases and Orphan Drug Development: Challenges and Opportunities

Laurie Muldowney, MD

FDA, United States

Deputy Director, OSI, OC CDER

Informational Designs and Potential Applications to Rare Diseases

Robert A. Beckman, MD

Georgetown University Medical Center, United States

Professor of Oncology and of Biostatistics, Bioinformatics, and Biomathematics

Can We Optimize Rare Disease Trials?

Carl-Fredrik Burman, PhD

AstraZeneca R&D, Sweden

Assoc. Prof. in Biostatistics, Chalmers Univ of Tech; Senior Principal Scientist

Q&A Panel Discussion