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Randomized Controlled Trials Versus Real World Evidence: Neither Magic Nor Myth.
- Source :
-
Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2021 May; Vol. 109 (5), pp. 1212-1218. Date of Electronic Publication: 2020 Nov 12. - Publication Year :
- 2021
-
Abstract
- Compared with drugs from the blockbuster era, recently authorized drugs and those expected in the future present a heterogenous mix of chemicals, biologicals, and cell and gene therapies, a sizable fraction being for rare diseases, and even individualized treatments or individualized combinations. The shift in the nature of products entails secular trends for the definitions of "drugs" and "target population" and for clinical use and evidence generation. We discuss that the lessons learned from evidence generation for 20th century medicines may have limited relevance for 21st century medicines. We explain why the future is not about randomized controlled trials (RCTs) vs. real-world evidence (RWE) but RCTs and RWE-not just for the assessment of safety but also of effectiveness. Finally, we highlight that, in the era of precision medicine, we may not be able to reliably describe some small treatment effects-either by way of RCTs or RWE.<br /> (© 2020 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.)
- Subjects :
- Cystic Fibrosis drug therapy
Cystic Fibrosis genetics
Cystic Fibrosis Transmembrane Conductance Regulator genetics
Humans
Information Storage and Retrieval
Mutation
Precision Medicine
Cell- and Tissue-Based Therapy methods
Evidence-Based Medicine
Pharmacology trends
Randomized Controlled Trials as Topic
Subjects
Details
- Language :
- English
- ISSN :
- 1532-6535
- Volume :
- 109
- Issue :
- 5
- Database :
- MEDLINE
- Journal :
- Clinical pharmacology and therapeutics
- Publication Type :
- Academic Journal
- Accession number :
- 33063841
- Full Text :
- https://doi.org/10.1002/cpt.2083