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Rare Diseases and Orphan Products: Accelerating Research and Development
the new products and claims submitted to FDA for pre-market review and approval, the emergence of challenging safety problems, and the globalization of the industries that FDA regulates,” whereas “resources have not increased in proportion to the demands … [so] that the scientific demands on the Agency far exceed its capacity to respond” (p. 3). In brief, the numbers of personnel are insufficient, the agency is reactive rather than leading in the development of regulatory science, and its surveillance mission suffers from lack of staff and inadequate information technology.
A group that lobbies for increased resources for FDA has compared FDA funding trends to those for the CDC and reported that the CDC and FDA had roughly equivalent funding in FY 1985 but that the budget for the former has grown at a compounded average rate of 11.4 percent compared to 7.1 percent for FDA. The CDC’s FY 2010 budget was $6.37 billion compared to $2.35 billion for FDA (Alliance for a Stronger FDA, 2009).
The FDA Science Board report identified eight areas of scientific and technological advances that are particular challenging for the agency: “systems biology (including genomics and other “omics”), wireless healthcare devices, nanotechnology, medical imaging, robotics, cell- and tissue-based products, regenerative medicine, and combination products” (p. 4). Although the report did not specify a particular level of increased funding, it suggested that another group’s recommended increase of 15 percent per year for 5 years “would still be insufficient … to initiate and support all the changes necessary” for the agency to fulfill its mission (p. 8).
FDA has recognized the need to take advantage of scientific developments to improve the way medical products are developed and evaluated. For example, the Critical Path Initiative, which was created in 2004 and emphasizes public-private collaborations, has focused on certain areas of particular relevance to products for rare diseases, including improving the development of biomarkers and modernizing the science of clinical trials (FDA, 2009d). In addition, the Advancing Regulatory Science Initiative is intended to strengthen the science base for product evaluation by providing better evaluation tools, standards, and pathways. It includes as one focus the setting of standards for products with people with unmet health needs (Hamburg, 2010b). Of note is that a research grants program to support the initiative is being funded primarily under the auspices of NIH with the NIH Common Fund providing $6 million and the FDA providing $750,000 for FY 2010 to FY 2012. Box 3-5 summarizes the research objectives. The announcement of the initiative included several examples of projects that might be funded and would reinforce elements of the Critical Path Initiative, for example, the development of new or improved biomarkers and the development of clinical trial strategies for more rapidly and efficiently evaluating the safety and efficacy of FDA-regulated products.