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RARE DISEASE WORKSHOP SERIES
Improving the Clinical Development Process
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The development of treatments for rare diseases can be challenging given the complexities and uncertainties regarding these diseases. To manage the effects of small patient populations and variable, irreversible disease requires the use of optimal statistical approaches, alternative study designs and for some diseases, the use of biomarkers. Industry, academia, the Food & Drug Administration, the National Institutes of Health, and other stakeholders must work together to develop strategies, policies and guidances to help improve the predictability and efficiency of the development process to enable more approvals for rare disease treatments.
This Workshop Series is designed to facilitate the process and help guide improvements in the development process for rare disease treatments. It features analyses on approved data from three ultra-rare treatments. The workshops are open to the public, including the media. Space is limited.
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Workshop 1: Optimizing the Choice of Statistical Analysis and Study Design for Ultra Rare Diseases (November 8, 2010, Bethesda, MD)
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Workshop 2: Clinical Evaluation of Rare Disease Treatments (June 14-15, 2011, Washington, DC)
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Workshop 3: : Use of Surrogate Endpoints in Rare Disease Treatment Development (November 8-9, 2011, Washington, DC)
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Workshop 4: Draft Guidance and Policy Recommendations for Rare Disease Treatment Development (Fall 2012)
The EveryLife Foundation for Rare Diseases sponsors the Workshop Series with generous support from outside donations.
- The EveryLife Foundation for Rare Diseases is a 501(c)(3) public charity
- The EveryLife family funds all Foundation operating costs
- Sponsorships go directly to funding the scientific analysis and the Workshop Series
Sponsorship opportunities are available please contact: jjenkins@kakkis.org