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BMJ Open. 2017 Feb 8;7(2):e014582. doi: 10.1136/bmjopen-2016-014582.

Assessing the potential clinical impact of reciprocal drug approval legislation on access to novel therapeutics in the USA: a cohort study.

Author information

1
Pharmagellan LLC, Milton, Massachusetts, USA.
2
Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
3
Section of General Internal Medicine, Department of Internal Medicine, Yale University, New Haven, Connecticut, USA.

Abstract

OBJECTIVE:

To quantify the potential effect of reciprocal approval legislation on access to clinically impactful therapeutics in the USA.

DESIGN:

A cohort study.

SETTING:

New therapeutics approved by the Food and Drug Administration (FDA), European Medicines Agency (EMA) and/or Health Canada between 2000 and 2010.

MAIN OUTCOME MEASURES:

Characteristics of new therapeutics approved by the EMA and/or Health Canada before the FDA, including mechanistic novelty, likely clinical impact, size of the affected population and FDA review outcome.

RESULTS:

From 2001 to 2010, 282 drugs were approved in the USA, Europe or Canada, including 172 (61%) first approved in the USA, 24 (9%) never approved in the USA, and 86 (30%) approved in the USA after Europe and/or Canada. Of the 110 new drugs approved in Europe and/or Canada before the USA, 37 (34%) had a novel mechanisms of action compared with drugs already approved by the FDA, but only 10 (9%) were for conditions lacking alternate available therapies in the USA at the time of ex-US approval-of which the majority (9/10; 90%) were indicated for rare diseases. 12 of the 37 agents with novel mechanisms of action approved first in Europe and/or Canada (32%) had their initial FDA submissions rejected for safety reasons-including 2 drugs that were ultimately withdrawn from the market in Europe due to safety concerns.

CONCLUSIONS:

If enacted, reciprocal approval legislation would most likely benefit only a small number of US patients receiving treatment for rare diseases, and the benefit may be somewhat mitigated by an increased exposure to harms.

KEYWORDS:

HEALTH SERVICES ADMINISTRATION & MANAGEMENT

PMID:
28179418
PMCID:
PMC5306516
DOI:
10.1136/bmjopen-2016-014582
[Indexed for MEDLINE]
Free PMC Article

Conflict of interest statement

All authors have completed the ICMJE uniform disclosure form at http://www.icmje.org/coi_disclosure.pdf, and declare that (within the past 3 years): ML and FSD are employees of Pharmagellan LLC, a biotechnology advisory firm that provides paid consulting services to companies and investors in the drug, medical device, diagnostics, and healthcare services industries. JSR receives support through Yale University from Medtronic and Johnson and Johnson to develop methods of clinical trial data sharing, from the Centers of Medicare and Medicaid Services (CMS) to develop and maintain performance measures that are used for public reporting, from the Blue Cross Blue Shield Association (BCBSA) to better understand medical technology evidence generation, and from the Food and Drug Administration (FDA) to develop methods for postmarket surveillance of medical devices.

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