Rare Diseases and Orphan Products Accelerating Research and Development (2010) / Chapter Skim
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8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases
8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases
Pages 241-248
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From page 241... ...
Closing the gap between what has been and what can now be accomplished is the challenge for rare diseases research and product development. A critical question is how to take better advantage of scientific and technological advances and investments in biomedical research in ways that will deliver improved health outcomes for the millions of Americans with rare diseases.
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From page 242... ...
ELEMENTS OF AN INTEGRATED NATIONAL STRATEGY Given the broad scope of this report, the analyses and recommendations focus selectively on the range of issues and activities related to rare diseases research and product development. They emphasize actions to be initiated by the National Institutes of Health (NIH)
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From page 243... ...
A particular focus of the recommended NIH action plan for rare diseases research (Recommendation 4-2) would be training of researchers on rare diseases and methods particularly applicable to rare diseases.
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From page 244... ...
In particular, uncertainty about the application of FDA standards for product approval can discourage companies from considering orphan product development. The analysis of FDA staff reviews of orphan products may identify inappropriate variation in FDA reviews and other information that will aid the development of guidelines for staff reviews as well as for staff assistance to drug sponsors through consultation beginning with the early stages of product development (Recommendation 3-1)
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From page 245... ...
. In addition, it is important that FDA and NIH cooperate to provide those who receive NIH awards for rare diseases product development with the guidance they need to design studies that meet FDA expectations (Recommendation 3-3)
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From page 246... ...
The recommendation below calls for a task force on rare diseases that would extend beyond NIH and contribute to a broader process of setting priorities and assessing progress. RECOMMENDATION The analyses presented in earlier chapters support the emphasis in the policy framework on a coordinated, collaborative approach to rare diseases research and product development.
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From page 247... ...
RECOMMENDATION 8-1: The Secretary of Health and Human Ser vices should establish a national task force on accelerating rare diseases research and product development. The objectives of the task force would be to promote, coordinate, monitor, and assess the implementa tion of NIH, FDA, and other public- and private-sector initiatives on rare diseases and orphan products and to support additional opportuni ties for public-private collaboration.
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From page 248... ...
A task force on rare diseases research and product development will not lessen the need for participants to improve their individual efforts and relationships as outlined in this report. Individual improvement will strengthen the foundation for collaboration.
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