EURORDIS: Rare diseases need more centralised processes within the EU

EURORDIS: "The speed of research and quality of designation and applications should be improved." [Shutterstock/RUCHUDA BOONPLIEN]

This article is part of our special report Reviewing orphan drugs law: A tough equation.

Future improvement of the EU Orphan Medicines Regulation (OMP) could involve more centralised processes for all rare diseases at the EU level, the European Organisation for Rare Diseases  (EURORDIS) told EURACTIV in a written interview.

“If there is already a lesson to be learnt by the current crisis we are all experiencing, is that a more functioning integrated Europe in health is needed when facing a common enemy,” EURORDIS said.


How do you evaluate the application of the EU Orphan Regulation so far? 

The Regulation on Orphan Medicinal Products enshrined since 2000 the right for people affected by rare conditions to be entitled to the same quality of treatment as other patients. It has spearheaded common European work of great added value in rare diseases at large – in pooling of knowledge, in directing research funding, and in bringing into being innovative policies and systems.

The Regulation has been a success in fulfilling one of its primary purposes – to attract investment to the development of therapies for life-threatening or debilitating diseases for millions of people who today have either no treatment at all or no satisfactory treatment. For example, each year, 15% to 18% of new designations represent a first orphan product for its indication (rare diseases with no designated product ever before). 82% of authorised OMPs are new active substances, indicating a very innovation-oriented sector. 44% of all orphan products target a disease that has a prevalence of less than 1 in 10,000.

Unfortunately, this is not yet enough. The speed of research and quality of designation and applications should be improved. According to estimates by the Gianni Benzi Foundation, 23% of designated orphan products are later abandoned. Furthermore, we are witnessing a steady decline in the orphan designation since 2016. We cannot afford this: the vast majority of the 6,000 recognised rare diseases do not have a centrally authorised therapy.

We are also worried about some cases we have seen when existing, well established, and inexpensive hospital preparations for which there is no safety concern are repurposed into a product subject to marketing authorization, and that new product should then come at a price hundred times higher than that of the original preparation even though the approval remains based on a limited clinical data set, good manufacturing processes and toxicology studies. These practices are detrimental to both the accessibility of products to patients and the overall reputation of the industry working on making therapies available.

Commission’s ‘risky’ move to re-visit orphan drugs regulation

The European Commission is expected to present by the end of July an evaluation study on the pros and cons of the application of orphan and paediatric regulations, which deal with a niche segment of rare diseases that affect fewer than five in 10,000 people.

Do you have specific statistics/data on how many people managed to get treated/get therapy all this period of time?

As the number of orphan medicinal products approved centrally at the EU level has increased, so do the numbers of OMPs available in individual countries. Whilst authorisation is for the whole of the European Union, coverage is managed at the individual country, region, or even local hospital level. The accessibility for patients grows at an unequal pace across European countries. EURORDIS has carried out a number of surveys in the past, highlighting some of the issues that our community has experienced.

Results on patient experience of treatments from our latest Rare Barometer survey (a quantitative survey, with over 7.500 respondents across the EU and world, presented at our European Conference on Rare Diseases and Orphan Drugs in May 2020) indicate that only 5% have already experienced a curative treatment, whilst 31% of respondents have never experienced any treatment for various reason – because there is no treatment, or they could not take part in the clinical trial, or the treatment is not affordable. We also observed a great variation of the number of treatments the patient could access depending on the disease area.

What could be improved in the EU legislation considering its upcoming review?

At EURORDIS we are maturing our vision on what could be better legislation, better addressing the needs of people living with a rare disease and doctors, the interests of industry and the interest of payers.

Our ideas for future improvements to the OMP Regulation focuses on a reinforcement of the EU centralised processes in a structured and seamless way for all rare disease therapies. In particular, rare disease therapies – because of their very specific characteristics – do require a continuum of evidence generation, all through their life cycle, from scientific guidance and assessments until well after the moment of marketing authorisation.

Patients should be granted access to address the unmet needs of conditions which are frequently life-threatening and debilitating. We welcome the recent decision of the European Medicines Agency to waive fees for scientific advice to academics when investigating orphan products.

Our key concern prior to the pandemic was that the political environment didn’t feel ready to maintain or expand incentives: some Member States’ concerns on the sustainability of their healthcare budget are currently superior to their common objective of an EU agenda for knowledge, innovation, competitiveness and attractiveness in support of the needs of people with rare diseases. However, if there is already a lesson to be learnt by the current crisis we are all experiencing, is that a more functioning integrated Europe in health is needed when facing a common enemy.

Drug pricing has been a sensitive topic especially for rare diseases: What’s the best solution according to you, to ensure affordability and enable pharma innovation at the same time?

Our daily experience provides countless examples of situations when the price of a new orphan medicine proves to be the stumbling block on which manufacturers and payers fail to agree, with potentially dramatic consequences for the patients in need of access to therapies. We understand the multiple challenges policymakers and payers face, in an environment that is systematically failing patients. We call out the high price by default that we are seeing with little or next to no justification other than what the market is perceived to be able to bear, but there is also mistrust between payers and companies on the current economic model for orphan medicines.

EURORDIS has constantly tried to propose solutions to improve access to therapies for rare diseases, as well as increasing the number of therapies developed. In our Breaking the Access Deadlock to Leave No one Behind (2018) paper we expressed the ambition to have 3 to 5 times more new rare disease therapies approved per year by 2025, 3 to 5 times cheaper than today.  How? Amongst the four pillars we set out in that paper, we believe Europe has a great role to play to reduce the uncertainties that member states individually have when appraising therapies for rare diseases – pricing is one of the essential components.

Building on existing experiences and tools already available, such as cross-country (e.g. BeneluxAI) or European (on HTA for example) cooperation, we must link any discussion on the price with the level of evidence provided at the time of the negotiation and with clear post-marketing research activities towards the reduction of uncertainties.

Europe has the opportunity, with the forthcoming Pharmaceutical Strategy, to consider that a new ecosystem is possible, a framework based on a global approach to innovation for unmet medical needs and on sustainability for healthcare systems as well as financial attractiveness to industry and investors.

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