§360aa. Recommendations for investigations of drugs for rare diseases or conditions

(a) Request by sponsor; response by Secretary

The sponsor of a drug for a disease or condition which is rare in the States may request the Secretary to provide written recommendations for the non-clinical and clinical investigations which must be conducted with the drug before-

(1) it may be approved for such disease or condition under section 355 of this title,

(2) if the drug is an antibiotic, it may be certified for such disease or condition under section 357 of this title, or

(3) if the drug is a biological product, it may be licensed for such disease or condition under section 262 of title 42.

If the Secretary has reason to believe that a drug for which a request is made under this section is a drug for a disease or condition which is rare in the States, the Secretary shall provide the person making the request written recommendations for the non-clinical and clinical investigations which the Secretary believes, on the basis of information available to the Secretary at the time of the request under this section, would be necessary for approval of such drug for such disease or condition under section 355 of this title, certification of such drug for such disease or condition under section 357 of this title, or licensing of such drug for such disease or condition under section 262 of title 42.

(b) Regulations

The Secretary shall by regulation promulgate procedures for the implementation of subsection (a) of this section.

(June 25, 1938, ch. 675, §525, as added Jan. 4, 1983, Pub. L. 97–414, §2(a), 96 Stat. 2049 ; amended Aug. 15, 1985, Pub. L. 99–91, §3(a)(1), 99 Stat. 387 .)

Amendments

1985-Subsec. (a). Pub. L. 99–91 struck out "or" at end of par. (1), inserted par. (2), redesignated former par. (2) as (3) and struck out "before" after "product,", and in last sentence inserted provisions relating to certification of such drug for disease or condition under section 357 of this title and substituted "licensing of such drug for such disease or condition under section 262 of title 42" for "licensing under section 262 of title 42 for such disease or condition".

Effective Date of 1985 Amendment

Section 8 of Pub. L. 99–91 provided that:

"(a) General Rule.-Except as provided in subsection (b), this Act and the amendments made by this Act [amending this section, sections 360bb, 360cc, and 360ee of this title, and sections 295g–1 and 6022 of Title 42, The Public Health and Welfare, and enacting provisions set out as notes under section 301 of this title and section 236 of Title 42] shall take effect October 1, 1985.

"(b) Exception.-The amendments made by sections 2, 3, and 6(a) [amending this section and sections 360bb and 360cc of this title] shall take effect on the date of the enactment of this Act [Aug. 15, 1985]. The amendment made by section 6(b) [amending section 6022 of Title 42] shall take effect October 19, 1984. The amendments made by section 7 [amending section 295g–1 of Title 42] shall take effect October 1, 1984 and shall cease to be in effect after September 30, 1985."

Study

Pub. L. 100–290, §3(d), Apr. 18, 1988, 102 Stat. 91 , directed Secretary of Health and Human Services to conduct a study to determine whether the application of subchapter B of chapter V of the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. 360aa et seq. (relating to drugs for rare diseases and conditions), and 26 U.S.C. 28 (relating to tax credit) to medical devices or medical foods for rare diseases or conditions or to both was needed to encourage development of such devices and foods and report results of the study to Congress not later than one year after Apr. 18, 1988.

Congressional Findings

Section 1(b) of Pub. L. 97–414 provided that: "The Congress finds that-

"(1) there are many diseases and conditions, such as Huntington's disease, myoclonus, ALS (Lou Gehrig's disease), Tourette syndrome, and muscular dystrophy which affect such small numbers of individuals residing in the United States that the diseases and conditions are considered rare in the United States;

"(2) adequate drugs for many of such diseases and conditions have not been developed;

"(3) drugs for these diseases and conditions are commonly referred to as 'orphan drugs';

"(4) because so few individuals are affected by any one rare disease or condition, a pharmaceutical company which develops an orphan drug may reasonably expect the drug to generate relatively small sales in comparison to the cost of developing the drug and consequently to incur a financial loss;

"(5) there is reason to believe that some promising orphan drugs will not be developed unless changes are made in the applicable Federal laws to reduce the costs of developing such drugs and to provide financial incentives to develop such drugs; and

"(6) it is in the public interest to provide such changes and incentives for the development of orphan drugs."