To encourage more collaboration and more efficient use of resources and build on the initiatives and recommendations discussed in earlier chapters, the committee proposes the creation of a time-limited task force on accelerating rare diseases research and product development. This task force would bring together leaders of key groups. Recognizing that mobilizing such a task force might be difficult in the private sector and that high-level backing is crucial, the committee concluded that the responsibility for creating the task force should rest with the Secretary of Health and Human Services. Nearly all of the government agencies discussed here report ultimately to the Secretary.
RECOMMENDATION 8-1: The Secretary of Health and Human Services 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 implementation of NIH, FDA, and other public- and private-sector initiatives on rare diseases and orphan products and to support additional opportunities for public-private collaboration.
As envisioned here, the task force would bring together a network of stakeholders for accelerating research and development. In the public sector, it would draw on representatives of the National Institutes of Health, the Food and Drug Administration, the Centers for Disease Control and Prevention, the Department of Defense, and other relevant federal agencies. From the private sector, it would recruit senior participants from the pharmaceutical, biotechnology, and medical device industries; patient advocacy groups; private foundations; and academic and other research institutions. International agencies and other relevant parties would be involved as appropriate.
The committee does not envision the task force as open-ended. It might extend for 4 to 8 years, at which point alternatives would be evaluated.
If this approach is to be effective, identifying and engaging the key public- and private-sector stakeholders is an important first step. The involvement of international counterparts of federal agencies and private organizations will also be desirable, especially for many extremely rare diseases for which global research participation is critical. Creating a venue for meaningful interactions and decision making among these groups will require arrangements for convening task force meetings, conducting analyses and developing strategies, following through on recommendations, and creating a scheme for monitoring progress. One major challenge will be identifying and sustaining a stable funding source for these administrative and analytic activities.
The proposed task force would build on existing initiatives and part-