Historical Controls in Randomized Clinical Trials: Opportunities and Challenges.
Journal
Clinical pharmacology and therapeutics
ISSN: 1532-6535
Titre abrégé: Clin Pharmacol Ther
Pays: United States
ID NLM: 0372741
Informations de publication
Date de publication:
02 2021
02 2021
Historique:
received:
10
03
2020
accepted:
11
06
2020
pubmed:
1
7
2020
medline:
22
5
2021
entrez:
1
7
2020
Statut:
ppublish
Résumé
Randomized control trials (RCTs) with placebo are the gold standard for determining efficacy of novel pharmaceutical treatments. Since their inception, over 75 years ago, researchers have amassed a large body of underutilized data on outcomes in the placebo control arms of these trials. Although rare disease indications have used these historical placebo data as synthetic controls to reduce burden on patients and accelerate drug discovery, broad use of historical controls is in its infancy. Large-scale historical placebo data could be leveraged to benefit both drug developers and patients if warehoused and made more available to guide trial design and analysis. Here, we examine challenges in utilizing historical controls related to heterogeneity in trial design, outcome ascertainment, patient characteristics, and unmeasured pharmacogenomic effects. We then discuss the advantages and disadvantages of current approaches and propose a path forward to broader use of historical controls in RCTs.
Substances chimiques
Pharmaceutical Preparations
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
343-351Informations de copyright
© 2020 The Authors Clinical Pharmacology & Therapeutics © 2020 American Society for Clinical Pharmacology and Therapeutics.
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