Intended for healthcare professionals

Feature Investigation

From blockbuster to “nichebuster”: how a flawed legislation helped create a new profit model for the drug industry

BMJ 2020; 370 doi: https://doi.org/10.1136/bmj.m2983 (Published 29 July 2020) Cite this as: BMJ 2020;370:m2983
  1. Daan Marselis, journalist,
  2. Lucien Hordijk, journalist
  1. The Investigative Desk, Amsterdam, Netherlands
  1. Correspondence to: marselis{at}investigativedesk.com

Twenty years ago, the EU passed a law to motivate the drug industry to develop medicines for rare diseases. But a system intended to help patients with neglected maladies primarily turned into a corporate cash machine. Daan Marselis and Lucien Hordijk report

After reaping 12 years of orphan exclusivity rewards in Europe, company executives at Celgene (now Bristol-Myers Squibb) were on the verge of celebrating yet another monopoly extension. If the European Medicines Agency (EMA) authorised Celgene’s latest orphan application for lenalidomide (Revlimid), the company would obtain a fourth orphan designation for its crown jewel—prolonging “market exclusivity” of this famous oncology drug.

Lenalidomide is not a typical rare disease treatment. It is derived from thalidomide, the notorious molecule that was pulled from the European market in the 1960s for causing birth defects. After a few molecular tweaks lenalidomide was launched and became one of the most profitable orphan medicines ever marketed. Our data show that, up until 2019, lenalidomide made €55bn (£50bn; $64bn) in worldwide sales.

Why would the EMA consider rewarding this already very lucrative product with yet another 10 years of market exclusivity—an incentive package meant only for drugs that would otherwise not be profitable?

We have analysed two decades of sales data and found that companies have reaped billions of profit off the back of orphan drug designations (box 1). The European Commission is set to publish a review that suggests most of these drugs would have been launched anyway without favourable monopoly rights. The findings could lead to reform in Europe.

Box 1

How the Investigative Desk conducted its investigation

Our list of orphan products is based on the datasets published by the EMA and FDA. We limited the scope to the 2000-19 period. We included all medicines that were given orphan status by the EMA. If the orphan status expired (or was withdrawn) by …

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