Orphan drug development - International program and study design issues

Drug Information Journal

Volumn 32, Issue 1, 1998, Pages 93-99

  Haffner, Marlene E ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Clinical trials; Orphan Drug Act; Rare diseases; Trial design

Indexed keywords

BIOLOGICAL PRODUCT; ORPHAN DRUG;

CLINICAL TRIAL; DRUG DESIGN; DRUG EFFICACY; DRUG INDUSTRY; DRUG LEGISLATION; DRUG RESEARCH; DRUG SAFETY; FOOD AND DRUG ADMINISTRATION; HUMAN; INTERNATIONAL COOPERATION; PRIORITY JOURNAL; REVIEW;

EID: 0031942634     PISSN: 00928615     EISSN: None     Source Type: Journal    
DOI: 10.1177/009286159803200113     Document Type: Review

References (8)

1. U.S. Federal Food, Drug, and Cosmetic Act (FFDCA). 21 USCA, § 201, et seq.
2. FFDCA SEC. 505(d); 21 USCA § 355(d) (Safety and effectiveness requirements).
3. P.L. 97-414, January 4, 1983 (The Orphan Drug Act).
4. FFDCA, SEC. 523; 21 USCA § 360aa (Recommendations for Investigations of drugs for rare diseases).
5. Section 1.28-1, Title 26, CFR (IRS Regulations on Rare disease research tax credit).
6. Orphan Drug Act Section 5 [21 USCA § 360ee] (Orphan Grant Funding Provision).
7. Draft guidance document; docket number 97D-0100; released for comment March 13, 1997.
8. 21CFR Part 316; Orphan Drug Regulations: Final Rule; Federal Register. December 29, 1992.