
From the U.S. Code Online via GPO Access
[wais.access.gpo.gov]
[Laws in effect as of January 2, 2001]
[Document not affected by Public Laws enacted between
  January 2, 2001 and January 28, 2002]
[CITE: 21USC360aa]

 
                        TITLE 21--FOOD AND DRUGS
 
             CHAPTER 9--FEDERAL FOOD, DRUG, AND COSMETIC ACT
 
                     SUBCHAPTER V--DRUGS AND DEVICES
 
              Part B--Drugs for Rare Diseases or Conditions
 
Sec. 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, or
        (2) 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 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, Sec. 525, as added Pub. L. 97-414, Sec. 2(a), 
Jan. 4, 1983, 96 Stat. 2049; amended Pub. L. 99-91, Sec. 3(a)(1), Aug. 
15, 1985, 99 Stat. 387; Pub. L. 105-115, title I, Sec. 125(b)(2)(F), 
(G), Nov. 21, 1997, 111 Stat. 2325, 2326.)


                               Amendments

    1997--Subsec. (a). Pub. L. 105-115, Sec. 125(b)(2)(G), struck out 
``, certification of such drug for such disease or condition under 
section 357 of this title,'' before ``or licensing of such drug'' in 
closing provisions.
    Subsec. (a)(1) to (3). Pub. L. 105-115, Sec. 125(b)(2)(F), inserted 
``or'' at end of par. (1), redesignated par. (3) as (2), and struck out 
former par. (2), which read as follows: ``if the drug is an antibiotic, 
it may be certified for such disease or condition under section 357 of 
this title, or''.
    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, Sec. 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.''
