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Use of surrogate outcomes in US FDA drug approvals, 2003-2012: a survey.

Yu T, Hsu YJ, Fain KM, Boyd CM, Holbrook JT, Puhan MA - BMJ Open (2015)

Bottom Line: To evaluate, across a spectrum of diseases, how often surrogate outcomes are used as a basis for drug approvals by the US Food and Drug Administration (FDA), and whether and how the rationale for using treatment effects on surrogates as predictors of treatment effects on patient-centred outcomes is discussed.For evaluating new drugs, patient-centred outcomes should be chosen whenever possible.If the use of surrogate outcomes is necessary, then a consistent approach is important to review the evidence for surrogacy and consider surrogate's usage in the treatment and population under study.

View Article: PubMed Central - PubMed

Affiliation: Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, USA Epidemiology, Biostatistics, and Prevention Institute, University of Zurich, Zurich, Switzerland.

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Review process. COPD, Chronic obstructive pulmonary disease; FDA, Food and Drug Administration.
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BMJOPEN2015007960F1: Review process. COPD, Chronic obstructive pulmonary disease; FDA, Food and Drug Administration.

Mentions: Sixty-eight of 1043 (7%) drug approvals were about COPD, diabetes, glaucoma or osteoporosis, and 58 (58/1043; 6%) of these were eligible for our study. The reasons for exclusion of approvals are summarised in figure 1. Of the 58 included approvals, 9 were for COPD (16%), 26 (45%) for diabetes, 9 (16%) for glaucoma and 14 (24%) for osteoporosis. For three of the four examined conditions, the drug approvals were mostly based only on a surrogate outcome (COPD (7/9 approvals were based only on a surrogate, 78%), diabetes (26/26 approvals, 100%) and glaucoma (9/9 approvals, 100%), see online supplementary table S1). COPD drug approvals were primarily based on the effects on improving lung function, with the exception of two drug approvals (SPIRIVA HANDIHALER and DALIRESP), which also examined COPD exacerbations. All diabetes drug approvals reviewed were based on lowering blood sugar level and all glaucoma drug approvals reviewed were based on lowering IOP. Most drug approvals for osteoporosis (10/14; 71%) were based on both, surrogate outcomes (bone mineral density) and patient-centred outcome (fractures).


Use of surrogate outcomes in US FDA drug approvals, 2003-2012: a survey.

Yu T, Hsu YJ, Fain KM, Boyd CM, Holbrook JT, Puhan MA - BMJ Open (2015)

Review process. COPD, Chronic obstructive pulmonary disease; FDA, Food and Drug Administration.
© Copyright Policy - open-access
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC4663404&req=5

BMJOPEN2015007960F1: Review process. COPD, Chronic obstructive pulmonary disease; FDA, Food and Drug Administration.
Mentions: Sixty-eight of 1043 (7%) drug approvals were about COPD, diabetes, glaucoma or osteoporosis, and 58 (58/1043; 6%) of these were eligible for our study. The reasons for exclusion of approvals are summarised in figure 1. Of the 58 included approvals, 9 were for COPD (16%), 26 (45%) for diabetes, 9 (16%) for glaucoma and 14 (24%) for osteoporosis. For three of the four examined conditions, the drug approvals were mostly based only on a surrogate outcome (COPD (7/9 approvals were based only on a surrogate, 78%), diabetes (26/26 approvals, 100%) and glaucoma (9/9 approvals, 100%), see online supplementary table S1). COPD drug approvals were primarily based on the effects on improving lung function, with the exception of two drug approvals (SPIRIVA HANDIHALER and DALIRESP), which also examined COPD exacerbations. All diabetes drug approvals reviewed were based on lowering blood sugar level and all glaucoma drug approvals reviewed were based on lowering IOP. Most drug approvals for osteoporosis (10/14; 71%) were based on both, surrogate outcomes (bone mineral density) and patient-centred outcome (fractures).

Bottom Line: To evaluate, across a spectrum of diseases, how often surrogate outcomes are used as a basis for drug approvals by the US Food and Drug Administration (FDA), and whether and how the rationale for using treatment effects on surrogates as predictors of treatment effects on patient-centred outcomes is discussed.For evaluating new drugs, patient-centred outcomes should be chosen whenever possible.If the use of surrogate outcomes is necessary, then a consistent approach is important to review the evidence for surrogacy and consider surrogate's usage in the treatment and population under study.

View Article: PubMed Central - PubMed

Affiliation: Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, USA Epidemiology, Biostatistics, and Prevention Institute, University of Zurich, Zurich, Switzerland.

Show MeSH
Related in: MedlinePlus