The Major Role of Clinicians in the Discovery of Off-Label Drug Therapies

28 Pages Posted: 11 Aug 2005 Last revised: 24 Dec 2014

See all articles by Harold J. DeMonaco

Harold J. DeMonaco

Harvard University - Massachusetts General Hospital; Massachusetts Institute of Technology (MIT) - Sloan School of Management

Ayfer Ali

Harvard University - Department of Economics; Wellesley College - Department of Economics

Eric A. von Hippel

Massachusetts Institute of Technology (MIT) - Sloan School of Management

Date Written: August 1, 2005

Abstract

Objective: To determine, through a review of the medical literature and author contact, the role of clinicians in the discovery of off label use of Food and Drug Administration approved prescription drugs.

Data Sources: The literature was accessed through MEDLINE (1999-December 2003). Additional sources accessed included the U.S. Patent Office and Micromedex, Thompson Scientific and Healthcare, Inc.

Data Synthesis: A survey of new therapeutic uses for New Molecular Entity drugs approved in 1998 was conducted for the subsequent 5 years of commercial availability. During that time period, a total of 144 new applications were identified in a computerized search of the literature for the 29 new drugs approved in 1998. Literature and patent searches were conducted to identify the first report of each new application. Authors of the seminal articles were contacted via survey and telephone interview to determine whether they were in fact the originators of the new applications. If they were, examinations of article contents and author surveys were used to explore whether each new application was discovered via clinical practice that was independent of pharmaceutical company or university research (field discovery) or whether the discovery was made by or with the involvement of pharmaceutical firm or university researchers (central discovery).

Conclusions: Post-NDA discoveries of new, off-label uses for new drugs were present in 22 of the 29 drugs in our sample. We found that 59% (85/144) of the drug therapy innovations in our sample were discovered by practicing clinicians via field discovery. The major role of clinicians in the discovery of new, off-label drug therapies has not been previously documented or explored. We propose that this finding has important regulatory and health policy implications.

Keywords: off label drug use, prescription drugs, new molecular entities, lead users

Suggested Citation

DeMonaco, Harold J. and Ali, Ayfer and von Hippel, Eric, The Major Role of Clinicians in the Discovery of Off-Label Drug Therapies (August 1, 2005). MIT Sloan Research Paper No. 4552-05, Available at SSRN: https://ssrn.com/abstract=780544 or http://dx.doi.org/10.2139/ssrn.780544

Harold J. DeMonaco (Contact Author)

Harvard University - Massachusetts General Hospital ( email )

55 Fruit Street
Boston, MA 02114
United States

Massachusetts Institute of Technology (MIT) - Sloan School of Management ( email )

100 Main Street
E62-416
Cambridge, MA 02142
United States

Ayfer Ali

Harvard University - Department of Economics ( email )

Littauer Center
Cambridge, MA 02138
United States

Wellesley College - Department of Economics ( email )

106 Central Street
Wellesley, MA 02181
United States
781-283-2236 (Phone)
781-283-2177 (Fax)

Eric Von Hippel

Massachusetts Institute of Technology (MIT) - Sloan School of Management ( email )

E62-455
Cambridge, MA 02142
United States
617-253-7155 (Phone)
617-253-2660 (Fax)

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