The effect of RCTs on drug demand: Evidence from off-label cancer drugs

Rebecca McKibbin

doi:10.1016/j.jhealeco.2023.102779

The effect of RCTs on drug demand: Evidence from off-label cancer drugs

J Health Econ. 2023 Jul:90:102779. doi: 10.1016/j.jhealeco.2023.102779. Epub 2023 Jun 21.

Author

Rebecca McKibbin¹

Affiliation

¹ The University of Sydney, Australia. Electronic address: rebecca.mckibbin@sydney.edu.au.

PMID: 37352724
DOI: 10.1016/j.jhealeco.2023.102779

Abstract

This paper investigates the effect of scientific information from randomized controlled clinical trials (RCTs) on the demand for off-label uses of cancer drugs. This is a unique setting where demand for a drug for a specific use is observable both before and after the first RCT results are released. Using variation in the timing of RCTs across off-label uses of drugs, I find that demand responds asymmetrically to the trial results based on the statistical significance of the clinically relevant endpoint. When this endpoint is statistically significant, there is a large and immediate increase in demand. When this end point is not statistically significant, physicians are relatively slow to abandon use of the drug.

Keywords: Cancer; Healthcare economics; Off-label prescribing; Pharmaceuticals; Regulation.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Antineoplastic Agents* / therapeutic use
Humans
Neoplasms*
Off-Label Use
Physicians*
Practice Patterns, Physicians'
Randomized Controlled Trials as Topic

Substances

Antineoplastic Agents