Controlling type I error rate for fast track drug development programmes

Statistics in Medicine

Volumn 22, Issue 5, 2003, Pages 665-675

  Shih, Weichung J ^a   Ouyang, Peter ^b   Quan, Hui ^c   Lin, Yong ^a   Michiels, Bart ^b   Bijnens, Luc ^b  

^a RUTGERS CANCER INSTITUTE OF NEW JERSEY   (United States)

^b JOHNSON AND JOHNSON PHARMACEUTICAL RESEARCH AND DEVELOPMENT   (United States)

^c MERCK RESEARCH LABORATORIES   (United States)

Author keywords

Accelerated review; Conditional approval; Food and Drug Administration; New drug application; Surrogate endpoint; Type 1 error rate

Indexed keywords

NEW DRUG;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ANALYTICAL ERROR; CANCER; CONTROLLED STUDY; DRUG APPROVAL; DRUG DEVELOPMENT; DRUG INDUSTRY; DRUG LEGISLATION; DRUG PROGRAM; DRUG SCREENING; FOOD AND DRUG ADMINISTRATION; OSTEOPOROSIS; PRACTICE GUIDELINE; REVIEW; STATISTICAL ANALYSIS; UNITED STATES;

CLINICAL TRIALS, PHASE III; DISEASE PROGRESSION; DRUG APPROVAL; HUMANS; PHARMACEUTICAL PREPARATIONS; PRODUCT SURVEILLANCE, POSTMARKETING; RESEARCH DESIGN; STATISTICS; SURVIVAL ANALYSIS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 0345701538     PISSN: 02776715     EISSN: None     Source Type: Journal    
DOI: 10.1002/sim.1396     Document Type: Review

References (23)

1. U.S. Department of Health and Human Services. Section 112 of the Food and Drug Administration Modernization Act of 1997: Expediting Study and Approval of Fast Track Drugs. http://www.fda.gov cder/guidance/index.htm.
2. U.S. Department of Health and Human Services. Guidance for Industry: Fast Track Drug Development programes - Designation, Development, and Application Review. 1998, http://www.fda.gov/cder/guidance/index.htm.
3. Ellenberg S, Hamilton JM. Surrogate endpoints in clinical trials: cancer. Statistics in Medicine 1989; 8: 405-414.
4. Fleming TR, Prentice RL, Pepe MS, Glidden D. Surrogate and auxiliary endpoints in clinical trials, with potential applications in cancer and AIDS research. Statistics in Medicine 1994; 13:955-968.
5. Fleming TR. Surrogate markers in AIDS and cancer trials (with discussions). Statistics in Medicine 1994; 13:1423-1435.
6. Neaton JD, Wentworth DN, Phame F, Hoagan C, Abrams DI, Deyton L. Considerations in choice of a clinical endpoint for AIDS clinical trials. Statistics in Medicine 1994; 13:2107-2125.
7. Chi G, Hung HMJ, O'Neill RO. Some comments on 'Adaptive trials and Bayesian Statistics in Drug Development' by Donald A. Berry. Biopharmaceutical Report 2001; 9:7-9.
8. Wittes J, Brittan E. The role of internal pilot studies in increasing the efficiency of clinical trials. Statistics in Medicine 1990; 9:65-72.
9. Shih WJ. Commentary: sample size re-estimation - journey for a decade. Statistics in Medicine 2001; 20: 515-518.
10. Yang P, Sridhara R, Chen G, Chi G. Determination of optimal conditional power by controlling the false accelerated approval rate of new drug. Statistics in Medicine 2002 (submitted).
11. Tang DI, Gnecco C, Geller NL. Design of group sequential clinical trials with multiple endpoints. Journal of the American Statistical Association 1989; 84:776-779.
12. Conaway MR, Petroni GR. Bivariate sequential designs for phase II trials. Biometrics 1995; 51:656-664.
13. Wei LJ, Su JQ, Lachin JM. Interim analyses with repeated measurements in a sequential clinical trial. Biometrika 1990; 77:359-364.
14. Lin DY. Nonparametric sequential testing in clinical trials with incomplete multivariate observations. Biometrika 1991; 78:123-131.
15. Williams PL. Sequential monitoring of clinical trials with multiple survival endpoints. Statistics in Medicine 1996; 15:2341-2357.
16. Shih WJ. Group sequential methods. In Encyclopedia of Biopharmaceutical Statistics, Chow SC (ed.). Marcel-Dekker Inc.: New York, 2000.
17. D'Agnostino RB Sr. Editorial: Controlling alpha in a clinical trial: the case for secondary endpoints. Statistics in Medicine 2000; 19:763-766.
18. Moye LA. Alpha calculus in clinical trials: considerations and commentary for a new millennium. Statistics in Medicine 2000; 19:767-779.
19. O'Neill RT. Commentary on 'Alpha calculus in clinical trials: considerations and commentary for a new millennium'. Statistics in Medicine 2000; 19:785-793.
20. Holm S. A simple sequentially rejective multiple test procedure. Scandinavian Journal of Statistics 1976; 6:65-70.
21. Zhang J, Quan H, Ng J, Stepanavage ME. Some statistical methods for multiple endpoints in clinical trials. Controlled Clinical Trials 1997; 18:204-221.
22. Statistical Issues in the Fast Track Approval Program for New Drug Applications, Joint Statistical Meetings, American Statistical Association, Atlanta, GA, 5-9 August 2001.
23. Accelerated Approval. Drug Information Association Workshop on Innovative Statistical Strategies in Today's Environment, Hilton Head Island, SC, 10-12 March 2002.