Insights & news

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.

Key contacts

Related practice areas

Related insights

Sign up for updates
    • 18/01/2021
    • Articles

    Belgium – Publication of Law on Medical Devices

    The Belgian Official Journal publishes today (i.e., 18 January 2021) the Law of 22 December 2020 on medical devices (Wet van 22 december 2020 betreffende medische hulpmiddelen / Loi du 22 décembre 2020 relative aux dispositifs médicaux – the Law). The Law implements in Belgium (i) Regulation (EU) 2017/745 of 5 April 2017 on medical devices (the MDR); and (ii) chapter IV of Regulation (EU) 2017/746 of 5 April 2017 on in vitro diagnostic medical devices, i.e., the chapter on notified bodies. Following its adoption by the federal Chamber of Representatives’ Committee for Public Health and Equal Opportunities on 8 December 2020, the Law was adopted in plenary session on 17 December 2020. The adopted text is identical to that of the Bill which the federal Government submitted to the Chamber of Representatives on 29 September 2020. For a discussion of the Law, we refer to our news alert of 3 December 2020 (see, Van Bael & Bellis Life Sciences News Alert of 3 December 2020). Subject to exceptions, the Law will enter into force on 26 May 2021, i.e., on the currently scheduled date of entry into force of the MDR.

    Read more
    • 06/01/2021
    • Articles

    Covid-19 Vaccine Moderna® Is Second Covid-19 Vaccine Recommended by European Medicines Agency for Use in European Union

    The European Medicines Agency (EMA) issued today its recommendation that the European Commission (the Commission) should grant a conditional marketing authorisation for the vaccine Covid-19 Vaccine Moderna®, a medicine developed by Moderna which will be indicated for active immunisation to prevent COVID-19 caused by SARS-CoV-2 in individuals 18 years of age and older (see, attached press release). The Commission is expected to give its approval shortly, possibly still today, paving the way for an expansion of existing vaccination programmes in the European Union. The recommendation follows a similar recommendation given on 21 December 2020 for Comirnaty®, a vaccine developed by BioNTech and Pfizer (see, Van Bael & Bellis Life Sciences News Alert of 21 December 2020). As is the case for Comirnaty®, Covid-19 Vaccine Moderna® contains a molecule called messenger ribonucleic acid (mRNA) which harbours instructions for making the spike protein which is characteristic of the SARS-CoV-2 virus and allows the virus to enter the human body’s cells. The vaccine thus causes the recipient’s body to recognise and produce the spike protein temporarily which, in turn, will activate the person’s immune system and cause it to produce antibodies and activate white blood cells to attack it. The person’s immune system is thus readied for an attack with the genuine SARS-CoV-2 virus and defend the body against it. The EMA made its recommendation on the basis of placebo-controlled clinical trial data involving 30,000 people. Efficacy was calculated in around 28,000 persons and showed a significant 94.1% reduction in the number of symptomatic Covid-19 cases in persons who were given the vaccine compared with persons from the control group who received a dummy injection. These strong results applied irrespective of pre-existing risk-enhancing conditions such as chronic lung disease, heart disease or diabetes and regardless of age (18 and up), gender, race or ethnicity. The marketing authorisation awarded to Covid-19 Vaccine Moderna® will be conditional in that the marketing authorisation holder will be required to continue providing results from the ongoing main trial which is scheduled to last two years. Additional trials will seek to gain fresh information regarding the length of protection afforded, the level of protection created against severe Covid-19 infections and the degree of protection established for the benefit of specific groups, including immunocompromised patients, children, and pregnant women. Further research will also focus on the vaccine’s ability to prevent asymptomatic cases. Other checks will result from the application of the EU’s safety monitoring plan for Covid-19 vaccines and from the risk management plan for Covid-19 Vaccine Moderna®.

    Read more
    • 04/01/2021
    • News

    Medicinal Products under the EU-UK Trade and Cooperation Agreement

    The attached note discusses the status of medicinal products under the recent EU-UK Trade and Cooperation Agreement

    Read more

Subscribe to our updates

Please select the practice areas you are interested in: *