The Institute of Medicine (IOM) produced a new report replete with recommendations as to how to better harnass research and development efforts to attack rare diseases and develop more orphan therapies. Rare diseases are a problem as they affect millions. Unfortunately, those millions afflicted are dispersed among as many as 5000-8000 diseases - some afflicting only a few individuals and some affecting thousands. I never thought about it before - but there may even be a pecking order within rare diseases themselves.
In general, traditional pharma companies have not attacked these diseases as it is very technically challenging and difficult to earn a return. Among the difficulties, one could count funding, access to adequate patient populations, clinical trial design and execution in small populations, etc. With many of these diseases, not even the most basic mechanistic information about the disease is known. If therapies exist, they tend to be very expensive - for obvious reasons. Given these problems the IOM was asked to review the situation and report its findings toward improving the R&D response toward therapies.
The report calls for an integrated national strategy. Let's call it 7 points of light:
- Public-private collaboration of government, commercial companies, academic institutions, and advocacy groups
- Application of science and technology that can make rare disease research and product development faster, easier and less expensive
- More trial design and use of analytical tools tailored to doing research in small populations
- Sharing research resources and infrastructure to better leverage scarce funding, data, biological specimens and rare disease-patient participation
- Reasonable rewards and incentives to private sector innovation and prudent use of public resources to meet targeted goals when appropriate
- Adequate staff with expertise on rare disease research and product development in public agencies funding biomedical research on rare diseases or regulating drugs
- Mechanisms for weighing priorities for rare disease research and measuring progress toward these goals
The IOM recommended the NIH develop a comprehensive action plan that covers all institutes and centers. The plan should cover program planning, grant review, training and coordination of all research. Further the NIH, should work with industry, academia, and voluntary organizations to develop a comprehensive system of shared resources for discovery research on rare diseases. This should include a public repository of available animal models (see Sigma-Aldrich SAGE Labs) for rare disorders that reflect disease mechanisms. It should also include a public repository of biological and molecular mechanism data.
Mechanisms exist to encourage developement of orphan drugs for rare diseases like the Orphan Drug Act 1983 and NIH's Rare Diseases Clinical Research Network. IOM recommends more be done to identify and implement collaborative strategies with an eye toward reducing development costs. The NIH should coordinate the new Cures Acceleration Network with the Rare Diseases Clinical Research Network, the Clinical and Translational Science Awards program, and the grants programs of FDA's Office of Orphan Product Development.
Pulling it all together the IOM calls on the Secretary of DHHS to establish a national Task Force to see it through over the next 4 to 8 years. Time to get busy.
Posted by Bruce Lehr October 7th 2010.
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