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British Medical Journal Publishes Critical Review of Orphan Medicines Rules Ahead of Release of European Commission's Own Studies

  • 31/07/2020
  • Articles

As part of the preparatory work for its Pharmaceutical Strategy for Europe which is due in the fourth quarter of 2020, the European Commission (the Commission) is expected to publish shortly an external study and own work assessing the regulatory framework governing orphan medicines and the paediatric rules.
Ostensibly meant as a shot across the bow for the Commission, the British Medical Journal (see, attachment) and the Nederlands Tijdschrift voor Geneeskunde published on 29 July 2020 an article written by two journalists of Amsterdam-based “The Investigative Desk”, Daan Marselis and Lucien Hordijk, which is highly critical of the current orphan medicine rules and, more broadly, the pharmaceutical industry. In their contribution entitled “From blockbuster to “nichebuster”: how a flawed legislation helped create a new profit model for the drug industry” Marselis and Hordijk claim that the orphan medicine rules are overly generous for the sector and turned these products into a “corporate cash machine”.
The authors point to a range of orphan medicines that reached a turnover of EUR 1 billion (there were 20 such products in 2019) and add that the average annual sales of an orphan medicine have multiplied by a factor of five, from EUR 133 million in 2001 to EUR 723 million in 2019. They also explain that a number of orphan medicines managed to benefit from a much longer period of market exclusivity than the 10 years provided for by applicable rules. This extended exclusivity results from other mechanisms such as paediatric incentives and the granting of orphan status to new therapeutic indications of the same active substance.
Much of the criticism voiced by Marselis and Hordijk is not new. As a matter of fact, the Commission had already started a review of the rules and identified a range of possible changes to the regulatory framework (see, attached pharmaceutical committee briefing note of 12 March 2020). For example, the Commission noted unspecified estimates that the number of new orphan medicines attributable to the EU orphan medicine rules falls in a range between 18 and 24 on a total of 146 authorisations during a period of approximatively 15 years. Ironically, part of the explanation for this seemingly limited success stems from the existence of similar orphan medicine rules in the United States which in many cases created the initial impetus for research, development and innovation. The authors also note that 95% of rare diseases still have no treatment but this would appear to be a strong argument in favour of maintaining a set of incentives for innovation.
Clearly, the figures cited by Marselis and Hordijk could also be regarded as indicative of a successful regulatory regime that is now ripe for improvement. Obvious areas for review include the possible attribution of orphan status to multiple therapeutic indications for the same active substance or to off-patent substances that were given a new lease of life following a relatively easy and inexpensive repurposing effort. What seems less helpful is the baseless rhetoric of Marselis and Hordijk when they decry the supposed fact “that companies have reaped billions of profit off the back of orphan drug designations” while stating elsewhere in the article, almost in the same breath, that “it was not possible to estimate profitability, as companies do not specifically disclose investments in developing an orphan medicine”. The authors’ approach, blessed by the British Medical Journal, was clearly that an insignificant technical distinction between the concepts of “turnover” and “profit” should not stand in the way of a good story.

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