Provisions to Promote Approval of Rare and Neglected Disease Products Survive Chopping Block in the Senate’s FY11 Omnibus Approps Bill

By Kurt R. Karst –      

Earlier this week, Senate Appropriations Committee Chair Senator Daniel Inouye (D-HI), released the text of the proposed Fiscal Year 2011 Omnibus Appropriations Act.  The almost 2,000-page bill, if enacted, would fund the government until September 30, 2011.  In addition to an increase in funding for FDA ($37.5 million more than the President’s request) and the FDA Food Safety Modernization Act, buried in the bill are provisions intended to promote the development and approval of products for rare and neglected diseases (as well as an increase in funding for the orphan drug grants program and funding for the Office of the Associate Director for Rare Diseases).  Specifically, Section 743 of the bill provides:

SEC. 743. (a) When implementing the authority provided in paragraphs (2) and (3) of section 740(c) of the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010 (Public Law 111–80) that requires the Commissioner of Food and Drugs to develop updated guidance documents and review standards for the development of safe and effective products to treat rare diseases and neglected tropical diseases, the Commissioner shall —

(1) maximize the use of accelerated approval where feasible and appropriate;

(2) work with sponsors to facilitate expanded access to investigational therapies;

(3) increase coordination and interaction with the World Health Organization, European Medicines Agency, and other international regulatory agencies;

(4) implement mechanisms for enhanced collaboration between the Food and Drug Administration and National Regulatory Authorities in developing countries;

(5) develop guidance on clinical development programs for rare diseases;

(6) develop guidance on the use of surrogate endpoints that are reasonably likely to predict clinical benefit of drugs and biological products under the regulations under subpart H of part 314 of title 21, Code of Federal Regulations and subpart E of part 601 of title 21, Code of Federal Regulations; and

(7) increase coordination among individual drug, biological product, and device review divisions across Food and Drug Administration centers to support the development of safe and effective medical products for rare and neglected diseases.

(b) The Commissioner of Food and Drugs shall submit a report to the Committee on Appropriations of the Senate and the Committee on Appropriations of the House of Representatives not later than 180 days after the report required in section 740(c)(1) of the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010 (Public Law 111–80) is submitted: Provided, That the report submitted in response to this section shall describe in detail how the Food and Drug Administration is implementing subsection (a).

This same provision was included in the FY 2011 Agriculture, Rural Development, FDA, and Related Agencies Appropriations bill (S. 3606) introduced earlier this year (see our previous post here), and has thus far survived the omnibus appropriations chopping block.   The Senate is expected to take up the omnibus appropriations bill this week.  If it passes in the Senate it will go to the House of Representatives as a substitute for the full-year Continuing Resolution the House passed earlier this month.