Surrogate endpoints and FDA's accelerated approval process

Health Affairs

Volumn 24, Issue 1, 2005, Pages 67-78

  Fleming, Thomas R ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER; CLARITHROMYCIN; ENCAINIDE; FLECAINIDE; FLUOROURACIL; PSEUDOMONIC ACID;

ATYPICAL MYCOBACTERIOSIS; CANCER; CLINICAL TRIAL; DISEASE COURSE; DISEASE MARKER; DOSE RESPONSE; DRUG APPROVAL; DRUG EFFICACY; FOOD AND DRUG ADMINISTRATION; HEART ARRHYTHMIA; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; MYCOBACTERIUM INTRACELLULARE AVIUM; POSTOPERATIVE INFECTION; PUBLIC HEALTH; REVIEW; STAPHYLOCOCCUS AUREUS; STAPHYLOCOCCUS INFECTION; SURGICAL RISK; SURVIVAL RATE; TREATMENT OUTCOME;

BIOLOGICAL MARKERS; DRUG APPROVAL; OUTCOME ASSESSMENT (HEALTH CARE); UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 12944288028     PISSN: 02782715     EISSN: None     Source Type: Journal    
DOI: 10.1377/hlthaff.24.1.67     Document Type: Review

References (26)

1. T.R. Fleming and D.L. DeMets, "Surrogate End Points in Clinical Trials: Are We Being Misled?" Annals of Internal Medicine 125, no. 7 (1996): 605-613.
2. L.A. Guay et al., "Intrapartum and Neonatal Single-Dose Nevirapine Compared with Zidovudine for Prevention of Mother-to-Child Transmission of HIV-1 in Kampala, Uganda: HIVNET 012 Randomised Trial," Lancet 354, no. 9181 (1999): 795-802; and J.B. Jackson et al., "Intrapartum and Neonatal Single-Dose Nevirapine Compared with Zidovudine for Prevention of Mother-to-Child Transmission of HIV-1 in Kampala, Uganda: Eighteen-Month Follow-up of the HIVNET 012 Randomised Trial," Lancet 362, no. 9867 (2003): 859-868.
3. L.A. Guay et al., "Intrapartum and Neonatal Single-Dose Nevirapine Compared with Zidovudine for Prevention of Mother-to-Child Transmission of HIV-1 in Kampala, Uganda: HIVNET 012 Randomised Trial," Lancet 354, no. 9181 (1999): 795-802; and J.B. Jackson et al., "Intrapartum and Neonatal Single-Dose Nevirapine Compared with Zidovudine for Prevention of Mother-to-Child Transmission of HIV-1 in Kampala, Uganda: Eighteen-Month Follow-up of the HIVNET 012 Randomised Trial," Lancet 362, no. 9867 (2003): 859-868.
4. D.S. Echt et al., "Mortality and Morbidity in Patients Receiving Encainide, Flecainide, or Placebo: The Cardiac Arrhythmia Suppression Trial," New England Journal of Medicine 324, no. 12 (1991): 781-788.
5. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; and R. Temple, "Are Surrogate Markers Adequate to Assess Cardiovascular Disease Drugs?" Journal of the American Medical Association 282, no. 8 (1999): 790-795.
6. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; and R. Temple, "Are Surrogate Markers Adequate to Assess Cardiovascular Disease Drugs?" Journal of the American Medical Association 282, no. 8 (1999): 790-795.
7. T.M. Perl et al., "Intranasal Mupirocin to Prevent Postoperative Staphylococcus aureus Infections," New England Journal of Medicine 346, no. 24 (2003): 1871-1877.
8. R. Chaisson et al., "Clarithromycin Therapy for Bacteremic Mycobacterium Avium Complex Disease: A Randomized, Double-Blind, Dose-Ranging Study in Patients with AIDS," Annals of Internal Medicine 121, no. 12 (1994): 905-911.
9. R.L. Prentice, "Surrogate Endpoints in Clinical Trials: Definition and Operational Criteria," Statistics in Medicine 8, no. 4 (1989): 431-440.
10. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; L.S. Freedman, B.L. Graubard, and A. Schatzkin, "Statistical Validation of Intermediate Endpoints for Chronic Diseases," Statistics in Medicine 11, no. 2 (1992): 167-178; V. De Gruttola et al., "Perspective: Validating Surrogate Markers - Are We Being Naïve?" Journal of Infectious Diseases 175, no. 2 (1997): 237-246; D.Y. Lin, T.R. Fleming and V De Gruttola, "Estimating the Proportion of Treatment Effect Explained by a Surrogate Marker," Statistics in Medicine 16, no. 14 (1997): 1515-1527; and M. Buyse et al., "The Validation of Surrogate Endpoints in Meta-Analysis of Randomized Experiments," Biostatistics 1, no. 1 (2000): 49-67.
11. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; L.S. Freedman, B.L. Graubard, and A. Schatzkin, "Statistical Validation of Intermediate Endpoints for Chronic Diseases," Statistics in Medicine 11, no. 2 (1992): 167-178; V. De Gruttola et al., "Perspective: Validating Surrogate Markers - Are We Being Naïve?" Journal of Infectious Diseases 175, no. 2 (1997): 237-246; D.Y. Lin, T.R. Fleming and V De Gruttola, "Estimating the Proportion of Treatment Effect Explained by a Surrogate Marker," Statistics in Medicine 16, no. 14 (1997): 1515-1527; and M. Buyse et al., "The Validation of Surrogate Endpoints in Meta-Analysis of Randomized Experiments," Biostatistics 1, no. 1 (2000): 49-67.
12. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; L.S. Freedman, B.L. Graubard, and A. Schatzkin, "Statistical Validation of Intermediate Endpoints for Chronic Diseases," Statistics in Medicine 11, no. 2 (1992): 167-178; V. De Gruttola et al., "Perspective: Validating Surrogate Markers - Are We Being Naïve?" Journal of Infectious Diseases 175, no. 2 (1997): 237-246; D.Y. Lin, T.R. Fleming and V De Gruttola, "Estimating the Proportion of Treatment Effect Explained by a Surrogate Marker," Statistics in Medicine 16, no. 14 (1997): 1515-1527; and M. Buyse et al., "The Validation of Surrogate Endpoints in Meta-Analysis of Randomized Experiments," Biostatistics 1, no. 1 (2000): 49-67.
13. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; L.S. Freedman, B.L. Graubard, and A. Schatzkin, "Statistical Validation of Intermediate Endpoints for Chronic Diseases," Statistics in Medicine 11, no. 2 (1992): 167-178; V. De Gruttola et al., "Perspective: Validating Surrogate Markers - Are We Being Naïve?" Journal of Infectious Diseases 175, no. 2 (1997): 237-246; D.Y. Lin, T.R. Fleming and V De Gruttola, "Estimating the Proportion of Treatment Effect Explained by a Surrogate Marker," Statistics in Medicine 16, no. 14 (1997): 1515-1527; and M. Buyse et al., "The Validation of Surrogate Endpoints in Meta-Analysis of Randomized Experiments," Biostatistics 1, no. 1 (2000): 49-67.
14. Fleming and DeMets, "Surrogate End Points in Clinical Trials"; L.S. Freedman, B.L. Graubard, and A. Schatzkin, "Statistical Validation of Intermediate Endpoints for Chronic Diseases," Statistics in Medicine 11, no. 2 (1992): 167-178; V. De Gruttola et al., "Perspective: Validating Surrogate Markers - Are We Being Naïve?" Journal of Infectious Diseases 175, no. 2 (1997): 237-246; D.Y. Lin, T.R. Fleming and V De Gruttola, "Estimating the Proportion of Treatment Effect Explained by a Surrogate Marker," Statistics in Medicine 16, no. 14 (1997): 1515-1527; and M. Buyse et al., "The Validation of Surrogate Endpoints in Meta-Analysis of Randomized Experiments," Biostatistics 1, no. 1 (2000): 49-67.
15. D. Sargent, "Disease Free Survival versus Overall Survival as an Endpoint for Adjuvant Colon Cancer Studies: Data from Randomized Trials," Meeting Transcript of the FDA Oncology Drugs Advisory Committee, Washington, D.C., 5 May 2004.
16. R. Nduati et al., "Effect of Breastfeeding and Formula Feeding on Transmission of HIV-1: A Randomized Clinical Trial," Journal of the American Medical Association 283, no. 9 (2000): 1167-1174.
17. Regarding point 3, see Freedman et al., "Statistical Validation"; De Gruttola et al., "Validating Surrogate Markers"; and Lin et al., "Estimating the Proportion of Treatment Effect."
18. Regarding point 3, see Freedman et al., "Statistical Validation"; De Gruttola et al., "Validating Surrogate Markers"; and Lin et al., "Estimating the Proportion of Treatment Effect."
19. Regarding point 3, see Freedman et al., "Statistical Validation"; De Gruttola et al., "Validating Surrogate Markers"; and Lin et al., "Estimating the Proportion of Treatment Effect."
20. 21 CFR, Sees. 314.500-314.560
21. T.R. Fleming and B.A. Richardson, "Some Design Issues in Microbicide HIV Prevention Trials," Journal of Infectious Diseases 190, no. 4 (2004): 666-674; and Meeting Transcript of the FDA Oncology Drugs Advisory Committee, Washington, D.C., 12-13 March 2003.
22. T.R. Fleming and B.A. Richardson, "Some Design Issues in Microbicide HIV Prevention Trials," Journal of Infectious Diseases 190, no. 4 (2004): 666-674; and Meeting Transcript of the FDA Oncology Drugs Advisory Committee, Washington, D.C., 12-13 March 2003.
23. Meeting Transcript of the FDA Oncology Drugs Advisory Committee (12-13 March 2003);
24. and R. Dagher et al., "Accelerated Approval of Oncology Products: A Decade of Experience," Journal of the National Cancer Institute 20, no. 96 (2004): 1500-1509.
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