RARE DISEASE WORKSHOP SERIES

Improving the Clinical Development Process

This workshop series is designed to help guide improvements in the development process for rare disease treatments. The Workshops are open to the public, including the media. Space is limited.

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 diseases treatments.

This Workshop Series is intended to fulfil two of the CureTheProcess Campaign’s Goals.  The Campaign is now officially endorsed by more than 160 patient organizations and physician societies.

  • Workshops 1 & 2: Optimizing the Choice of Statistical Analysis and Study Design for Ultra Rare Diseases
  • Workshops 3 & 4: Improving Access to the Accelerated Approval Process by Creating Qualification Criteria for the Effective Use of Surrogate Endpoints in Rare Disease Treatment Development
    – Dates TBA – Please check back soon
  • Workshop 5: Draft Guidance and Policy Recommendations for Rare Diseases
    - September 2011

*All workshops will be completed by September 2011, the deadline for the Food & Drug Administration to fulfill the requirements of the Brownback-Brown Amendment for Rare & Neglected Diseases in the 2010 FDA appropriation bill.

Workshop 1: Optimizing the Choice of Statistical Analysis
and Study Design for Ultra Rare Diseases
November 8, 2010 8:00 a.m. – 3:00 p.m.
Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, MD

Goal: The goal of the workshop is to define improved or optimal statistical methods for randomized placebo-controlled clinical studies of heterogeneous ultra-rare diseases when using clinical endpoints. These small studies involving highly variable patients are challenging to analyze and capture benefit in the face of baseline variation and heterogeneous clinical responses. By evaluating specific case examples of challenges and solutions, the workshop hopes to create the core findings that could inform on the optimal statistical analysis methods for traditionally designed studies in rare diseases. Planned analyses of existing drug data will also be discussed.

A small group of interested leaders from FDA, NIH, academia and industry will be participating in this true workshop. Be ready to think and contribute to a solution or recommendation. The contributions and ideas provided will drive further data analyses of existing drug data. The goal of the conference is purposely limited to this particular topic, as a starting place on statistical issues and will progress in future workshops on alternative study designs and methods.

Draft Agenda:

  • Keynote speaker: Tom Fleming, Ph.D., Professor Biostatistics, University of Washington
  • Ultra rare disease case study presentations from participants -- including representatives from BioMarin, Genzyme and Shire
  • Planned statistical analysis on endpoints for approved ultra rare drugs, including work on control of variation, repeated measures and multiple endpoint analyses will be discussed
  • Discussion with key representatives from FDA, NIH, industry and academia on critical next steps for analysis and discussion

Please note: This workshop is currently at maximum capacity, to be put on the waiting list please contact: [email protected] or 415-884-0223

Speaker Presentations will be posted by Nov. 30, 2010

The Kakkis EveryLife Foundation sponsors the Workshop Series with generous support from outside donations.

  • The Kakkis EveryLife Foundation is a 501(c)(3) public charity
  • Dedicated to pursuing science-driven changes in the health care and regulatory systems to accelerate biotech innovation for rare diseases
  • The Kakkis family funds all Foundation operating costs.
  • Sponsorships go directly to funding the scientific analysis and the Workshop series

Sponsorship opportunities are available please contact: [email protected]

Kakkis EveryLife Foundation Scientific Advisory Committee:

Brent Blumenstein, Ph.D., Principal Statistician, TriArc Consulting
Tony Lachenbruch, Ph.D., Professor, Oregon State University
L.J. Wei, Ph.D., Professor, Harvard University
Larry Friedman, M.D., Independent Consultant
Emil Kakkis, M.D., Ph.D., President, Kakkis EveryLife Foundation