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Pages 1-14

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From page 1...
... Because the number of people affected with any particular rare disease is relatively small and the number of rare diseases is so large, a host of challenges complicates the development of safe and effective drugs, biologics, and medical devices to prevent, diagnose, treat, or cure these conditions. These challenges include difficulties in attracting public and private funding for research and development, recruiting sufficient numbers of research participants for clinical studies, appropriately using clinical research designs for small populations, and securing adequate expertise at the government agencies that review rare diseases research applications or authorize the marketing of products for rare conditions.
From page 2...
... In response to the difficulties confronting rare diseases research and orphan product development, NIH with support from FDA approached the Institute of Medicine (IOM) about a study to examine the opportunities for and obstacles to the development of drugs and medical devices to treat rare diseases.
From page 3...
... The challenges of doing so are many and will raise difficult questions of affordability and equitable access. As envisioned by the committee, an integrated national strategy to promote rare diseases research and product development has several dimensions (Box S-1)
From page 4...
... Criticisms of FDA procedures related to orphan drug development and approval tend to focus on three issues -- insufficient resources for timely meetings and guidance for sponsors; inconsistency in reviews of applications for orphan drug approvals across CDER divisions; and inadequate resources for the orphan products grants program. In addition, it is sometimes stated that FDA inappropriately requires two phase III, randomized, placebo-controlled, double-blind trials to support orphan drug approvals.
From page 5...
... RECOMMENDATION 3-2: The Center for Drug Evaluation and Re search should evaluate the extent to which studies submitted in support of orphan drugs are consistent with advances in the science of small clinical trials and associated analytic methods. Based on its findings, CDER should work with others at FDA, NIH, and outside organiza tions and experts, as appropriate, to • adjust and expand existing educational programs on the design and conduct of small clinical trials; • specify which CDER and NIH personnel should complete these educational programs; • revise guidance for sponsors on trial design and analysis and on safety and efficacy reviews of products for rare diseases; and • support further work to develop and test clinical research and data analysis strategies for small populations.
From page 6...
... Making the best use possible of research resources calls for arrangements that make existing knowledge and resources more accessible to rare diseases researchers and that also discourage a duplicative infrastructure of, for example, natural history data, animal models of disease, biorepositories, and chemical compound libraries. Although many barriers will have to be overcome, a "rare diseases research commons" with several unlinked or loosely linked elements should yield significant benefits.
From page 7...
... Given the important role that NIH plays in supporting rare diseases research, a comprehensive NIH action plan on rare diseases would be useful to better integrate and expand existing work and attract new resources and investigators to the field. The following recommendation spans all phases of research on rare diseases and orphan products, including research on medical devices for people with rare diseases.
From page 8...
... RECOMMENDATION 5-2: In collaboration with industry, academic researchers, NIH and FDA scientists, and patient organizations, FDA should expand its Critical Path Initiative to define criteria for the evaluation of surrogate endpoints for use in trials of products for rare conditions. The expansion and improvement of patient registries and biorepositories is another important element in a strategy to accelerate rare diseases research and product development.
From page 9...
... Op portunities to be explored include • expanding the Rare Diseases Clinical Research Network to address opportunities for diagnostic and therapeutic advances for a greater number of rare diseases; • setting priorities for rare diseases research within other NIH clinical trials networks; • creating a study group approach to rare diseases, modeled after the Children's Oncology Group; and • building additional capability for rare diseases clinical research within the Clinical and Translational Science Awards program. A new NIH program that is not restricted to rare diseases research but will likely benefit such research is the Cures Acceleration Network.
From page 10...
... The most expensive orphan drugs cost more than $400,000 per year. The committee's analysis focused on Medicare, which covers many individuals with severe, disabling rare conditions.
From page 11...
... Such populations also present the practical challenges of ensuring sufficient research participants for clinical trials to demonstrate safety and effectiveness. Devising meaningful alternative incentives to encourage the development of medical devices for small populations has proved a persistent challenge.
From page 12...
... The options examined might include the additional orphan products grants and NIH awards for the development of devices to meet priority needs; tax credits for certain research and development costs; and the creation of inducement prizes for the design and initial testing of novel devices in areas of unmet need. Changes in the HDE incentives for pediatric devices, including removal of the restriction on profits, may provide an opportunity to gauge whether similar changes could encourage innovative devices for conditions affecting small populations of adults.
From page 13...
... INTEGRATING STRATEGIES FOR RARE DISEASES RESEARCH AND ORPHAN PRODUCT DEVELOPMENT An integrated national strategy for rare diseases research and orphan product development will have many elements. As outlined earlier, such a strategy will actively involve the many parties that play essential roles in the process -- government, industry, academic investigators, advocacy groups, and others.
From page 14...
... 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. A task force on rare diseases research and product development will not lessen the need for all participants to improve their individual efforts and relationships as outlined in this report.


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