Intended for healthcare professionals

Editor's Choice

Stop exploiting orphan drugs

BMJ 2010; 341 doi: (Published 18 November 2010) Cite this as: BMJ 2010;341:c6587
  1. Fiona Godlee, editor, BMJ
  1. fgodlee{at}

The most surprising revelation in this week’s BMJ is that there’s a website that lists drugs that can be “orphaned” and exploited for profit. In an open letter to Britain’s prime minister, 21 neurologists and paediatricians call for an urgent review into the pricing of orphan drugs (doi:10.1136/bmj.c6466). Legislation meant to encourage development of new treatments for rare diseases is instead severely limiting availability of existing treatments, they say, costing the taxpayer unnecessary millions and reaping massive profits for drug companies.

As Nigel Hawkes and Deborah Cohen describe (doi:10.1136/bmj.c6459), a company needs only to find an unlicensed drug and license it for use in a rare condition, citing little more than pre-existing evidence of its use in clinical practice. With minor adjustments to the product and new packaging, a modestly priced drug suddenly becomes unaffordable. A 10 year monopoly (seven years in the United States) then protects the drug from competition, banning previous suppliers from producing and distributing the unlicensed version and clinicians from prescribing it.

This is so clearly against the interests of patients and the public that the inaction of governments and drug regulators is astounding. Some countries have acted, say Robin Ferner and Dyfrig Hughes in their editorial (doi:10.1136/bmj.c6456). In France a government agency makes and distributes unlicensed drugs for rare diseases, overcoming claims that a drug’s quality can’t be assured without industry intervention. Our editorialists ask why the NHS doesn’t do this. And they call on the General Medical Council to lift the ban on prescribing an unlicensed drug where a licensed drug exists for the same indication.

Jonathan Roos and colleagues say that the situation warrants investigation under competition law (doi:10.1136/bmj.c6471), while the authors of the open letter invoke the parliamentary health select committee and the Office of Fair Trading. It’s clear that something must be done—and, given the financial state of health systems around the world, the sooner the better.

Less clear are the merits of open peer review, in which authors know the identity of their reviewers. Karim Khan says it won’t work for most journals (doi:10.1136/bmj.c6425). But Trish Groves explains that editors of the BMJ have long been convinced of its ethical superiority (doi:10.1136/bmj.c6424). A randomised trial in 1999 gave us enough evidence to introduce such a system, and 10 years’ experience has confirmed our view that it works and is a good thing.

We were keen to take things further, to post reviewers’ signed comments online with the published article, giving reviewers more credit and accountability for the good work they do and helping readers understand how decisions are made. So we did another randomised trial. The reassuring results were just coming in when I moved to help set up BioMed Central in 2000. So all articles published in BMC’s new open access medical journals were accompanied by their pre-publication history, including signed reviewers’ comments and the submitted version of the article. This system has been running successfully for 10 years.

But the BMJ’s randomised trial had a sad history. Its first author, Sue van Rooyen, fell seriously ill. She died in 2005. This week we finally publish the trial (doi:10.1136/bmj.c5729), and we will start posting reviewers’ comments with all published research papers next spring. In the meantime we look forward to hearing from volunteers who would like their research peer reviewed under the new system.


Cite this as: BMJ 2010;341:c6587