IOM Issues Report on Rare Diseases and Orphan Products; Recommends an Integrated National Strategy

By Kurt R. Karst –      

Earlier this week, the Institute of Medicine (“IOM”) issued a 350-page report, titled “Rare Diseases and Orphan Products: Accelerating Research and Development,” which is intended to help refocus attention on accelerating rare diseases research and product development.  The report is the culmination of a series of meetings held between August 2009 and May 2010.

The IOM report calls for implementing an integrated national strategy to promote rare diseases research and product development that would include seven key elements:

• Active involvement and collaboration by a wide range of public and private interests, including government agencies, commercial companies, academic institutions and investigators, and advocacy groups.
• Timely application of advances in science and technology that can make rare diseases research and product development faster, easier, and less expensive.
• Appropriate use and further development of trial design and analytic methods tailored to the special challenges of conducting research on small populations.
• Creative strategies for sharing research resources and infrastructure to make good and efficient use of scarce funding, expertise, data, biological specimens, and participation in research by people with rare conditions.
• Reasonable rewards and incentives for private-sector innovation and prudent use of public resources for product development when the latter appears to be a faster or less costly way to respond to important unmet needs.
• Adequate organization and resources, including staff with expertise on rare diseases research and product development, for the public agencies that fund biomedical 2research on rare diseases and regulate drugs and medical devices.
• Mechanisms for weighing priorities for rare diseases research and product development, establishing collaborative as well as organization-specific goals, and assessing progress toward these goals.

Although “[c]omponents of each of these elements already exist, some more robust than others,” according to a report summary, “it is difficult to achieve coherence, given the array of participants with differing perspectives and priorities, the number and diversity of rare diseases, and the limited and even undocumented resources devoted to them individually and collectively.”   The IOM report goes on to recommend a number of specific steps to aid in developing a more integrated approach to rare diseases research and product development.

Interest in rare disease/orphan product issues has been running high as of late.  As we previously reported (here and here), Congress is considering legislation to streamline the development and regulation of products for rare and neglected diseases and to increase incentives for the development and approval of such products.