Critères de substitution (surrogate endpoints)

La démonstration qu'un critère intermédiaire prédit le critère clinique n'est pas suffisant

En effet ces études montrent des associations statistiques. En déduire une prédiction est abusive (et constitue une sur-interprétation du résultat compte tenu de la méthode).

En particulier il peut toujours y avoir des phénomènes de confusion.

papiers classiques

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méta-analyse [ref] [ref]

acceptabilité [ref] [ref]

Références

Fleming TR. Surrogate End Points in Clinical Trials: Are We Being Misled? Ann Intern Med. 1996 Sep 30;125(7):605–5. Pubmed

Freedman LS, Graubard BI, Schatzkin A. Statistical validation of intermediate endpoints for chronic diseases. Stat Med. 1992;11(2):167–78. Pubmed

Glasser SP, Clark PI, Lipicky RJ, Hubbard JM, Yusuf S. Exposing patients with chronic, stable, exertional angina to placebo periods in drug trials. JAMA. American Medical Association; 1991 Mar 26;265(12):1550–4. Pubmed

Prentice RL. Surrogate endpoints in clinical trials: definition and operational criteria. Stat Med. 1989 Mar 31;8(4):431–40. Pubmed

Buyse M, Molenberghs G. Criteria for the validation of surrogate endpoints in randomized experiments. Biometrics. 1998 Sep;54(3):1014–29. Pubmed

Molenberghs G, Geys H, Buyse M. Evaluation of surrogate endpoints in randomized experiments with mixed discrete and continuous outcomes. Stat Med. 2001 Oct;20(20):3023–38. Pubmed

Molenberghs G, Buyse M, Geys H, Renard D, Burzykowski T, Alonso A. Statistical challenges in the evaluation of surrogate endpoints in randomized trials. Control Clin Trials. 2002 Dec;23(6):607–25. Pubmed

Molenberghs G, Burzykowski T, Alonso A, Buyse M. A perspective on surrogate endpoints in controlled clinical trials. Stat Methods Med Res. 2004 Jun;13(3):177–206. Pubmed

Alonso A, Molenberghs G, Geys H, Buyse M, Vangeneugden T. A unifying approach for surrogate marker validation based on Prentice's criteria. Stat Med. 2006 Jan;25(2):205–21. Pubmed

Kobayashi F, Kuroki M. A new proportion measure of the treatment effect captured by candidate surrogate endpoints. Stat Med. John Wiley & Sons, Ltd; 2014 Apr 29;:n/a–n/a. Pubmed

Buyse M, Molenberghs G, Burzykowski T, Renard D, Geys H. The validation of surrogate endpoints in meta-analyses of randomized experiments. Biostatistics (Oxford, England). 2000 Mar;1(1):49–67. Pubmed

Buyse M. Use of meta-analysis for the validation of surrogate endpoints and biomarkers in cancer trials. Cancer J. 2009 Sep;15(5):421–5. Pubmed

Psaty BM, Lumley T. Surrogate end points and FDA approval: a tale of 2 lipid-altering drugs. JAMA. 2008 Mar 26;299(12):1474–6. Pubmed

Burzykowski T, Buyse M, Piccart-Gebhart MJ, Sledge G, Carmichael J, Luck H-J, et al. Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer. J Clin Oncol. 2008 Apr;26(12):1987–92. Pubmed