107th Congress

Rare Diseases Act

P.L. 107-280 (H.R. 4013)

Impact of Public Law

Title I of the Rare Diseases Act provides statutory authorization for the existing National Institutes of Health (NIH) Office of Rare Diseases (ORD), which was administratively established within the Office of the Director in 1993. The measure requires the Director of ORD to 1) recommend an agenda for research on rare diseases, 2) promote coordination and cooperation among NIH Institutes and Centers (ICs), 3) promote sufficient allocation of NIH resources related to rare diseases, 4) promote the establishment of a centralized rare disease information clearinghouse, 5) prepare a biennial report of rare disease research activities and opportunities, 6) prepare the annual report of the Director of NIH to Congress on rare disease research, and 7) serve as the principal advisor on orphan diseases to the Director of NIH. P.L. 107-280 authorizes such sums as have already been appropriated for fiscal year (FY) 2002, and $4 million for FYs 2003 through 2006.

The legislation also establishes regional Centers of Excellence on rare diseases. The Director of ORD, in collaboration with the Directors of relevant ICs, can enter into cooperative agreements and make grants for these centers. The Centers of Excellence are authorized to receive such sums that have already been appropriated for FY 2002, and $20 million for FYs 2003 through 2006.

Legislative History

H.R. 4013 was introduced on March 20, 2002, and was referred to the House Energy and Commerce Committee. On June 19, the bill was marked up and ordered to be reported. On June 26, the bill was reported (H.R. 107-543) and placed on the Union Calendar. H.R. 4013 passed the House on October 1 and the Senate on October 17. The President signed the bill on November 6 as P.L. 107-280.

S. 1379, the companion bill, was introduced on August 3, 2001, and was referred to the Senate Committee on Health, Education, Labor and Pensions. On October 16, the Senate Committee ordered the bill to be reported with a technical amendment during a markup hearing. On December 18, the Senate Committee reported the bill (S.R. 107-239) and placed it on the Senate Calendar under General Orders. There was no further action on this measure during the 107th Congress.