Intended for healthcare professionals


When drugs don’t make it to market

BMJ 2015; 350 doi: (Published 09 June 2015) Cite this as: BMJ 2015;350:h2852
  1. Barbara Mintzes, senior lecturer
  1. 1Faculty of Pharmacy and Charles Perkins Centre, University of Sydney, Charles Perkins Centre D17, NSW Australia 2006
  1. Correspondence to: Barbara.mintzes{at}

We need to know why, and regulators should tell us

Drug regulation aims to protect public health by ensuring that only medicines that are effective and safe are marketed. The key regulatory innovation after the thalidomide tragedy was the requirement for systematic evidence of efficacy before marketing approval, with the understanding that no drug exposure is worth risking without evidence of benefit.

Over 50 years later, premarket evaluations remain much needed but imperfect, with efficacy standards not always ensuring clinical benefit for patients,1 and inadequate public communication on the available evidence and how it has been assessed. The United States Food and Drug Administration (FDA) is more open than many regulatory agencies, but, when it comes to non-approvals, the linked study by Lurie and colleagues (doi:10.1136/bmj.h2758) shows that commercial confidentiality continues to trump public health.2

If the FDA rejects an application, the letter to the manufacturer stating the reasons is confidential. Lurie and colleagues are FDA staff members who compared the content of FDA non-approval letters (n=61) with the firms’ press releases.2 This is the first published study to compare what regulators say in confidence to pharmaceutical manufacturers and what those manufacturers then say to the public. The differences are damning: 85% of the regulator’s …

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