1. Consider, as part of a national policy framework, a wide range of public and private strategies and innovations, such as:

    • enhancing multidisciplinary collaboration and government-university-industry partnerships in basic and translational research;

    • expanding public engagement and enhancing the roles of patient organizations;

    • facilitating research data and biomaterials collection and dissemination, including the use of bio-repositories and registries;

    • strengthening training of investigators;

    • disseminating information to clinicians, patients, and families;

    • revising policies and regulations;

    • encouraging alternative research financing mechanisms; and

    • developing research agendas and coordinating resources and development efforts throughout the product development pathways.

  1. Make recommendations for an integrated national rare disease policy on research and development, including responding to the proposals included in a white paper that will be prepared by the sponsors and provided to the committee approximately six months after the contract begins.

NIH and FDA concluded after further consideration that it was not feasible to present the white paper with proposals for the committee, but NIH and FDA staff made presentations to the committee and answered numerous committee questions. The committee reviewed a broad range of government, industry, and academic resources. It commissioned two background papers that appear as Appendixes B and C.

The study committee met five times between August 2009 and May 2010. Three of these meetings included public sessions during which the committee heard from a range of interested parties, including government officials, basic and clinical researchers, representatives of advocacy groups that are engaged in supporting research and product development, and representatives of industry trade associations and individual companies involved in developing drugs and devices. (The agendas for the public sessions follow this overview of study activities.) The committee also worked with the National Organization for Rare Disorders and the Genetic Alliance to solicit views from member organizations that are involved in research. (A list of organizations that submitted written statements follows the November meeting agenda below.)

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