107th Congress

Rare Diseases Act
S. 1379/Senate Report 107-239 and H.R. 4013/House Report 107-543

Background

On August 3, 2001, Senator Edward M. Kennedy (D-MA) introduced S. 1379, the Rare Diseases Act of 2001. During his introduction of S. 1379, Senator Kennedy stated that this legislation "promises to greatly enhance the prospects for developing new treatments and diagnostics, and even cures, for literally thousands of rare diseases and disorders." On September 17, 2001, the National Organization for Rare Disorders, Inc., along with 100 disease advocacy organizations, sent a letter to Senate Majority Leader Thomas A. Daschle (D-SD) to encourage the Senate to pass S. 1379. The letter indicated that only 30 percent of patients with rare diseases receive a diagnosis during the first 3 to 5 years after the onset of symptoms.

On March 20, 2002, Representative John Shimkus (R-IL) introduced H.R. 4013, the Rare Diseases Act of 2002. Although he did not have any oral remarks when he introduced the bill, Representative Shimkus made a statement on June 18, during the prescription drug coverage debate. He stated that because rare diseases affect such a small percentage of the population, incentives are needed to encourage research and development. He noted that at least 1 in 9 people in the United States suffer from at least 1 of the 6,000 known rare diseases.

Provisions of the Legislation/Impact on NIH

Title I of the Rare Diseases Act of 2001 would provide 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 would require the Director of ORD to recommend an agenda for research on rare diseases, promote coordination and cooperation among NIH Institutes and Centers (ICs), promote sufficient allocation of NIH resources related to rare diseases, promote the establishment of a centralized rare diseases information clearinghouse, prepare a biennial report of rare disease research activities and opportunities, prepare the annual report of the Director of NIH to Congress on rare disease research, and serve as the principal advisor on orphan diseases to the Director of NIH. H.R. 4013 authorizes such sums that have already been appropriated for fiscal year (FY) 2002, and $4 million for FYs 2003 through 2006. S. 1379 authorizes $4 million for FY 2002 and such sums as necessary for FYs 2003 through 2006.

In addition, the legislation would establish regional Centers of Excellence on rare diseases. The Director of ORD, in collaboration with the Directors of relevant ICs, would enter into cooperative agreements and make grants for these centers. The House bill would make the establishment discretionary, and the Centers of Excellence would be authorized to receive such sums that have already been appropriated for FY 2002, and $20 million for FYs 2003 through 2006. The Senate bill would make establishment mandatory and authorize $20 million for FY 2002, and such sums as may be necessary for each subsequent fiscal year.

Title II of the measure would authorize $25 million to be appropriated to an existing Food and Drug Administration (FDA) Orphan Product Research Grant program.

H.R. 4013 is similar to S. 1379 but does not include the section related to FDA. This section was introduced as a separate piece of legislation (H.R. 4014).

Status and Outlook

S. 1379 was introduced on August 3, 2001, and was referred to the Senate Committee on Health, Education, Labor, and Pensions. The measure currently has 19 cosponsors. On October 16, 2001, the Senate Committee ordered the bill to be reported with a technical amendment during a markup hearing. On December 18, 2001, the Senate Committee reported the bill (Senate Report 107-239) and placed it on the Senate Calendar under General Orders.

H.R. 4013 was introduced on March 20, 2002, and was referred to the House Committee on Energy and Commerce. H.R. 4013 has 54 cosponsors. On June 19, 2002, the bill was marked up and ordered to be reported. On June 26, 2002, the bill was reported (House Report 107-543) and placed on the Union Calendar.