depositing it into a trust, allowing open access to data, implementing socially responsible licensing, standardizing material transfer agreements, and creating new benefit-sharing arrangements.

  • Chapter 7 describes strategies for facilitating clinical trials. It reviews the regulatory tools available to assist with orphan drug development and approval processes, including fast track designation; accelerated approval; priority review; and early and frequent communication with FDA through such vehicles as Type A, B, or C formal meetings, special protocol assessments, or informal meetings.

  • Chapter 8 outlines areas identified as needing further discussion: (1) business models for the development of drugs for rare and neglected diseases, as it is still too soon to be able to distill broadly applicable lessons and best practices, and new models will continue to be created; (2) the current state of data and resource sharing and public access, with a focus on distilling best practices; (3) intellectual property issues as they relate to orphan drugs and rare and neglected diseases; and (4) policies applied to the review of orphan drug applications, with consideration of what new or revised policies might better facilitate the approval of such drugs.

  • Finally, a number of resources mentioned throughout the workshop are available on the Internet. Appendix C provides a list of websites for those interested in the development of drugs for rare and neglected diseases.

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