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European Policy Reforms Noteworthy to Follow in the Near Future

Updated: Apr 10, 2022

There is currently a policy reform movement in Europe in which patient representatives are actively involved. This reform is noteworthy to follow, both in content and form in the study of social innovations for rare diseases in Europe, and primarily as these have significant implications for the pharmaceutical industry.

A review process of the European Orphan Medicines Regulation is underway. Adopted more than 20 years ago, this regulation specific to medicines for rare diseases, which was promoted by patient organizations including Eurordis (a patient-driven alliance of rare disease patient organizations) in the wake of the mobilization that led to the passage of the Orphan Drug Act in 1983 in the United States, had the primary objective of allowing the greatest number of affected patients to have access to treatment. The revision of the European Orphan Medicines Regulation is a significant part of the revision of the EU general pharmaceuticals legislation. Health economists (Kyle & Perrot, 2021) and associations led by the French Muscular Dystrophy Association (AFM) (Braun, Cottet, Duguet, 2016), emphasize that major changes have taken place in the pharmaceutical industry in recent years, which are linked to the transition from technologies derived from the so-called chemical industry to new technologies based on biotechnologies and genomics. The main effect, they write, is the shift from a "standard" blockbuster model - a very large number of patients, for a relatively inexpensive drug - to smaller "niche-buster" markets, which are more complex, technological drugs, whose development today relies essentially on university research and university spin-offs. These niche products are aimed at a small number of patients, and are composed of very expensive treatments. For more than thirty years the AFM has financed several research projects for the discovery of treatments for neuromuscular diseases and, beyond that, gene therapies. Currently the AFM is campaigning in France and within Eurordis for the implementation of an alternative economic model that is fairer and more likely to improve access to innovative medicines.

The current review process, which should end with the adoption of a new text by the European Commission in the fourth quarter of 2022, is an opportunity to discuss in depth the implementation of the European Orphan Medicines Regulation and to put forward proposals for reform. The review program has already given rise to two Feedback and Public consultation periods during 2021, in which a number of stakeholders have participated, ranging from industry unions, biopharma representatives and patient organizations (Eurordis urged "patient organizations and other interested stakeholders to provide their feedback"). The project also generated reports, for example the report "Orphan Medicine Incentives: How to address the unmet needs of rare disease patients by transforming the European OMP landscape" by the European Expert Group on Orphan Drug Incentives (2021). The European Expert Group on Orphan Drug Incentives is composed of "representatives of the broad rare disease community, including researchers, academia, patient representatives, members of the investor community, rare disease companies and trade associations", and at the national level includes the work of the French parliamentary information mission on medicines (Dufeu & Touraine, 2021).

Some of the main issues discussed include:

- Expanding the scope of the financial incentives and rewards at the various stages needed to develop innovative medicines, from basic research to marketing. This is meant to empower change in the current clustering pattern of orphan medicinal products development in certain conditions, leaving other areas unexplored (for instance rare diseases affecting sensory organs, the respiratory system, or children).

- Enhance the role of real-world evidence (as opposed to evidence collected in standard clinical trial settings) at the R&D stage, as well as at the regulatory approval stage and the market access stage.

- Increasing the flexibility and predictability of the orphan medicinal products regulatory pathway.

At the same time, some 15 other EU member states, including Belgium, Italy, the Netherlands, Hungary, Spain, Greece, and France have signed a Manifesto for an Important Projects of Common European Interest (IPCEI) on Health on March 3, 2022. Pleading for an "independent, competitive and innovative" Europe of health, this manifesto focuses on “three strategic areas”, and an important part of which concerns the treatment of rare diseases. These areas include: “(i) developing innovative and greener technologies and production processes for manufacturing medicines; (ii) innovating with regard to strategic challenges such as fighting antibiotic resistance, developing treatments for rare diseases, and in conjunction with the European Health Emergency Preparedness and Response Authority (HERA), responding to future pandemics; and (iii) developing gene and cell therapies” (French Presidency of the Council of the European Union, Press release, March 3, 2022).

Through consultations and changing regulations, it becomes evident that there is an urgent need to restructure the ways in which drugs for rare diseases are brought to market, the timelines and pathways they take to become accessible to patients, and the need for social pharmaceutical innovation to address the unmet needs of patients with rare diseases.



Braun Serge (Scientific Director of the AFM, Pharmacist, President of GenoSafe[1]), Cottet Christian (CEO of AFM-Telethon) & Duguet Christophe (Director of Public Affairs at AFM-Telethon) (2016). Les maladies rares, modèle de santé publique [Rare diseases as a public health model]. In François Bourdillon, Gilles Brücker, Didier Tabuteau (Eds). Traité de santé publique. Paris, Lavoisier, 549-556.

Dufeu Audrey & Touraine Jean-Louis (2021). Rapport d’information déposé en application de l’article 145 du Règlement par la Commission des affaires sociales en conclusion des travaux de la mission d’information sur les médicaments [Information report submitted pursuant to Article 145 of the Rules of Procedure by the Social Affairs Committee at the conclusion of the work of the information mission on medicines]. (Rapport d’information N° 4275 ; p. 251). Assemblée Nationale.

European Expert Group on Orphan Drug Incentives (2021)."Orphan Medicine Incentives. How to address the unmet needs of rare disease patients by transforming the European OMP landscape", Copenhague Economics, 50 pages.

Kyle Margaret & Perrot Anne (2021). Innovation pharmaceutique : comment combler le retard français ? [Pharmaceutical innovation: how to bridge the French gap?]. Les notes du conseil d’analyse économique, n° 62, Janvier.


Written by: Florence Paterson

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