Randomized Controlled Trials Versus Real World Evidence: Neither Magic Nor Myth

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. T...

Full description

Saved in:
Bibliographic Details
Published in:Clinical pharmacology and therapeutics 2021-05, Vol.109 (5), p.1212-1218
Main Authors: Eichler, Hans‐Georg, Pignatti, Francesco, Schwarzer‐Daum, Brigitte, Hidalgo‐Simon, Ana, Eichler, Irmgard, Arlett, Peter, Humphreys, Anthony, Vamvakas, Spiros, Brun, Nikolai, Rasi, Guido
Format: Article
Language:English
Subjects:
Review
Reviews
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary: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.
ISSN:0009-9236
1532-6535
DOI:10.1002/cpt.2083