On March 3, 2014, nearly 50 representatives from the patient and scientific community, the industry as well as health policy specialists gathered in the European Parliament in Brussels to discuss the impact of the Orphan Medicinal Product Regulation (141/2000) on Rare Bleeding Disorders. This was the 21st EHC Round Table and it was chaired by Members of the European Parliament (MEP) Dr Miroslav Mikolášik and Dr Paul Rübig, both from the European People’s Party.
Regulation 141/2000 was implemented by EU Member States in 2000 and was originally written in order to respond to a public health concern, which was the lack of treatment for patients suffering from rare diseases. Through this legislation, regulators encouraged the pharmaceutical industry to invest in the orphan drug sector. With this in mind, the Regulation provides a series of incentives to pharmaceutical companies, such as protocol assistance and ten-year marketing exclusivity, to compensate for the reduced return on investment for this type of pharmaceutical product. To this day, European regulators believe that this legislation has proved highly successful with over 1,000 orphan drug designations since its inception.
Nonetheless, during the Round Table discussions representatives from the medical community, patients and pharmaceutical industry all felt that some aspects of the legislation could be improved through a more pragmatic interpretation by regulators. In particular, both patients and physicians pointed out that there should be a clear distinction between rare conditions that are well-known and characterised and those that are rarer and for which there is a lack of treatment and diagnostics. However, this differentiation should not reduce the extensive efforts by research and development organisations to provide treatments for both types of conditions. This event was also the opportunity for patients’ representatives to voice their concerns with regard to the potential barriers that a ten-year marketing exclusivity period could raise for accessing a wider array of therapies for their conditions.
As for the pharmaceutical industry, speakers at the event gave an overview of the hurdles faced in developing orphan medicinal products and they also provided some suggestions on how to improve the situation in the future: for instance, by having greater harmonisation between the EU and US legislative environments and by having a more pragmatic approach in the interpretation of the regulation for treatments that are better understood versus for completely new molecules.
Finally, during the event the payers’ representative also explained that the EU reimbursement landscape is very diverse and that currently there are a number of EU initiatives to try to harmonise reimbursement assessments.
To conclude, the following recommendations emerged from the discussions:
- A distinction should be made between rarer bleeding disorders and more common bleeding disorders that are well-characterised and better understood and for which there is more treatment and expertise available. However, this differentiation should not reduce research efforts for both types of conditions.
- The EHC believes that the longer-acting products coming to the market have different mechanisms of action and that they would all suit patients differently.
- Pharmaceutical companies should increase their efforts to market older-generation products in a wider array of markets.
- Pharmaceutical companies should consider making novel treatments more affordable; marketing products that cannot be reimbursed due to their high price benefits neither patients nor companies.
- Patients should be included in the reimbursement discussions as they provide a unique expertise on the use of products.
Speakers at the Round Table included:
- Prof Flora Peyvandi, Director of Internal Medicine Department RCCS Maggiore Hospital, University of Milan, member of the EHC Medical Advisory Group.
- Ms Agnès Mathieu, Legal Officer at the European Commission.
- Mr Brian O’Mahony, President, EHC.
- Dr Peter Feldman, Unit Manager, Coagulation Factors Development, Bio Products Laboratory (BPL).
- Dr Claudia Nardini, Product Development Director, Kedrion Biopharma.
- Assoc. Prof. Cristina Solomon, Medical Affairs Director, CSL Behring.
- Dr Stephanie Seremetis, CMO and CVP Haemophilia R&D Portfolio, Novo Nordisk.
- Philippe Van Wilder, Director Studies & Strategy, Independent Health Insurance Funds of Belgium.
To consult documents about this event, please our library section.