Hypothesis: A single clinical trial plus causal evidence of effectiveness is sufficient for drug approval

Clinical Pharmacology and Therapeutics

Volumn 73, Issue 6, 2003, Pages 481-490

  Peck, Carl C ^b   Rubin, Donald B ^b   Sheiner, Lewis B ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL PHARMACOLOGY; CLINICAL PRACTICE; CONCENTRATION RESPONSE; CORRELATION ANALYSIS; DOSE RESPONSE; DRUG ACTIVITY; DRUG APPROVAL; DRUG COST; DRUG DESIGN; DRUG EFFECT; DRUG EFFICACY; DRUG INDUSTRY; DRUG LABELING; DRUG MECHANISM; EMPIRICISM; FOOD AND DRUG ADMINISTRATION; HEALTH CARE POLICY; HISTORY; HYPOTHESIS; NOTE; OUTCOMES RESEARCH; PRACTICE GUIDELINE; PRIORITY JOURNAL; SIMULATION; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DRUG LEGISLATION; HUMAN; METHODOLOGY; RANDOMIZED CONTROLLED TRIAL; REVIEW; STATISTICS; UNITED STATES;

BIOLOGICAL MARKER;

BIOLOGICAL MARKERS; CLINICAL TRIALS; DRUG APPROVAL; HUMANS; LEGISLATION, DRUG; RANDOMIZED CONTROLLED TRIALS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 0037534027     PISSN: 00099236     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0009-9236(03)00018-3     Document Type: Note

References (24)

1. Food and Drug Administration Modernization Act of 1997, Pub. L. No. 105-115, 111 Stat. 2295 (1997).
2. Federal Food, Drug, and Cosmetic Act of 1938, Drug Amendments of 1962. Pub. L. No. 87-781, 76 Stat. 780 (1962).
3. Peck CC, Wechsler J. Report of a workshop on confirmatory evidence to support a single clinical trial as a basis for new drug approval. Drug Inf J 2002;36:517-34.
4. Applications for FDA approval to market a new drug, adequate and well-controlled studies, 21 C.F.R. Sect. 314.126.
5. Wardell WM. Introduction of new therapeutic drugs in the United States and Great Britain: an international comparison. Clin Pharmacol Ther 1973;14:773-90.
6. Peck CC. Streamlining and modernizing drug development: hearings before the Subcommittee on Health and Environment of the House Committee on Commerce, 104th Cong., 2nd Sess. (May 2, 1996).
7. Peck CC. Modernizing effectiveness testing in drug development. Hearings before the Senate Labor and Human Resources Committee. (Feb 21, 1996).
8. Peck CC. Clinical drug development may soon be accomplished in less than 3 years: will FDA and the pharmaceutical industry be ready? Hearings before the Subcommittee on Health and Environment of the House Committee on Commerce, 105th Cong., 2nd Sess. (April 23, 1997).
9. Guidance for industry: providing clinical evidence of effectiveness for human drugs and biological products. Dept. of Health and Human Services (US), Food and Drug Administration, Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research; 1998 May.
10. Statement regarding the demonstrations of effectiveness of human drug products and devices. FDA docket No. 95N-0230, Federal Register 60 (147), 1995 Aug 1. p. 39180-1.
11. Temple R. Experiences with single trials leading to section 115 of FDAMA and May 1998 evidence document: the use of the single, adequate and well-controlled efficacy trial (SCT) to support approval. Presented at Drug Information Association, The Use of the Single, Adequate and Well-Controlled Efficacy Study to Support Approval; 2001 Jan 22-23; Washington DC.
12. Prescription Drug User Fee Authorization and Drug Regulation Act of 1997. House Committee on Commerce, House Report No.: 105-310, 105th Cong., 1st Sess. (Oct 7 1997). p. 67-8.
13. Sheiner LB. Learning versus confirming in clinical drug development. Clin Pharmacol Ther 1997;61:275-91.
14. Sheiner LB, Rubin DB. Intention-to-treat analysis and the goals of clinical trials. Clin Pharmacol Ther 1995;57:6-15.
15. Senn S. Statistical issues in drug development. Chichester (UK): John Wiley & Sons; 1997.
16. Sheiner LB, Steimer JL. Pharmacokinetic/pharmacodynamic modelling in drug development. Annu Rev Pharmacol Toxicol 2000;40:67-96.
17. Sanathanan LP, Peck CC. The randomized concentration-controlled trial: an evaluation of its sample size efficiency. Control Clin Trials 1991;12:780-94.
18. Urquhart J. Demonstrating effectiveness in a post-placebo era. Clin Pharmacol Ther 2001;70:115-20.
19. Biomarkers Definitions Working Group. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther 2001;69:89-95.
20. Buyse M, Molenberghs G. Criteria for the validation of surrogate endpoints in randomized experiments [published erratum appears in Biometrics 2000;56:324]. Biometrics 1998;54:1014-29.
21. Frangakis CE, Rubin DB. Principal stratification in causal inference. Biometrics 2002;58:21-9.
22. Holford NH, Kimko HC, Monteleone JP, Peck CC. Simulation of clinical trials. Annu Rev Pharmacol Toxicol 2000;40:209-34.
23. Fisher LD. One large, well-designed multicenter study as an alternative to the usual FDA paradigm. Drug Inf J 1999;33:265-71.
24. FDA Task Force on Risk Management. Managing the risks from medical product use. Washington: Dept. of Health and Human Services (US); 1999.