Health Insurance And Rare Diseases: European Efforts In Ensuring Specialized Care – Has assembled a panel of experts, industry leaders and key players in Brussels to address the issue of how rare disease delivery can be developed in the EU.
Rare diseases pose a major challenge to European health institutions. Because of the common abnormalities with which people suffer from rare diseases, treatments have historically been limited by a lack of knowledge and funding for further research.
Health Insurance And Rare Diseases: European Efforts In Ensuring Specialized Care
Despite this, significant progress has come since 2000 thanks to the European Union’s regulatory framework that encouraged research and development of treatments for rare diseases. Over the past 22 years, the number of treatments for rare diseases has increased dramatically.
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With the regulation coming up for review in 2022, it gathered a panel of experts, industry leaders and key players in Brussels to address the issue of how the provision of rare diseases can be developed in the EU.
Over the past 20 years, innovation around rare diseases in Europe has improved significantly, with a specific framework put in place by the EU called the Orphan Medicinal Products (OMP) Regulation.
This law covers products developed to treat medical conditions that, because they are so rare, would not be profitable to produce without government support.
Initially, only eight orphan drugs were available to patients but this number has increased to more than 200, resulting in the pharmaceutical industry providing treatment to 6.3 million patients.
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In November 2020, the European Commission published the EU Medicines Strategy, which set out a number of proposed measures to increase access to medicines in Europe, as well as address shortages. As part of the strategy, the Commission expects a major review of the OMP regulation in 2022.
Although the availability of orphan drugs has increased, 95% of rare diseases still have no treatment. Yann Le Cam, CEO of EURORDIS believes that the review provides an opportunity to expand the range of treatments for rare diseases significantly over the next two decades.
“Imperial College London analyzed that according to the last twenty years in America and Europe, the trend is that 600 new treatments by 2030. Which looks good but still not enough for unmet needs. But there is a possibility to go to 1,000 treatments in the next ten years if we will improve the research environment, ecosystem and regulatory measures,” he said.
Nathalie Moll, Director General, EFPIA commented that the coordination and effective research seen with the EU’s response to the COVID-19 pandemic should be replicated by future EU rare disease systems.
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“It is important that for the European Commission to review the regulations and the COVID, it makes us as an industry look in detail at the barriers and why it is so difficult to get treatment for rare diseases.”
“The biggest obstacle is science. Because the population is so small, scientific knowledge decreases as it spreads. With COVID-19, we were amazing. We collaborated, we opened up our databases and we did amazing work. So when we review the code we need to be and lunar approach to promote science and accelerate research,” he said.
The European Commission currently offers a number of incentives to the pharmaceutical industry to encourage the development of OMPs. These include fee reductions or exemptions from regulatory assessment procedures, a 10-year post-approval market exclusion period, as well as scientific and protocol support, including early negotiations with the regulator.
Although OMP Regulation is estimated to have resulted in the production of 74 percent of approved OMPs between 2000-2017, 95 percent of rare diseases remain without approved treatments.
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The European Commission published its Staff Working Document on the Evaluation of OMPs in August 2020. According to their evaluation, the Commission believes that the Orphan Regulation has overstimulated innovation, leading to high prices for orphan drugs. The Commission is therefore considering limiting incentives to areas with unmet needs.
In response to potential policy changes, the European Expert Group on Orphan Drug Incentives hopes to be a source of possible solutions for the OMP Regulatory Assessment. The group brings together a range of rare disease experts, including EURORDIS: Voice of Rare Disease Patients in Europe and the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE).
“We have seen 20 positive years in the fight against rare diseases. We have many products on the market that we would not have without the EU system that provides incentives for rare diseases,” Alexander Natz, Secretary General, EUCOPE, said.
“We need to be better and faster. It’s not about the companies themselves. A small company can be very agile in bringing research and products. But we have to be faster.”
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Considering the recommendations that EUCOPE has given to the Commission, Natz adds, “From the company’s point of view, it’s all about predictability. I need to know and tell my investors how much exclusivity I will get for the product, will my offer for the exclusion of the compound be safe.”
In 2000, the European Commission worked with INSERM (French National Institute for Health and Medical Research) to create Orphan.
The online platform provides doctors, researchers and pharmaceutical groups with information about rare diseases, to improve diagnosis and treatment.
This resource provides high-quality information on rare diseases and also includes the Orphanet rare disease nomenclature (ORPHAcode), which improves the visibility of rare diseases in relevant information systems.
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Since its inception, Orphanet has grown rapidly and the consortium now includes 40 countries, in Europe and around the world.
Patient databases can help encourage the development of clinical research in rare diseases, while also improving patient care. Therefore, to facilitate research in rare diseases, the European Commission advocates the creation of a European Platform for the Registry of Rare Diseases.
Currently, there are no uniform standards governing the collection and access of patient registries and databases, and, according to Orphanet, there are 600 rare disease registries in Europe alone. Multiple registries may also exist for the same disease and it is estimated that only 20 percent of rare diseases have registries.
In the European Union, a disease is considered rare if it affects less than five out of 10,000 people.
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This may seem small, but in practice, six to eight percent of the population of the camp is affected by such a rare condition, which means between 27 and 36 million people.
Many of these patients suffer from even rare diseases that affect only one person in 100,000 or more, according to EU data.
About 5,000-8,000 rare diseases in the European Union span all medical areas and include neurological, immune and metabolic diseases, as well as rare cancers.
One of the main topics of discussion was how the objectives of the European Commission can be effectively implemented in each member state.
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“The European Parliament understood the importance of creating a European Health Union. We understood during the pandemic that our national health systems are weak and we need to provide more capacity at the European level,” explained Alessandra Moretti, Member of the European Parliament.
“The priority should be equal rights for everyone in Europe. Everyone across the EU should enjoy some basic rights regarding access to health services. We have asked the European Commission for legal legal measures to access life-saving services.
The importance of sharing knowledge and removing barriers to data from clinical trials being transferred between research centers in the EU was emphasized. Sandra Galina, Director General, DG Santé, European Commission, noted the importance of regulation to create EU registries on rare diseases.
“Once fully populated with clinical data, these will enable clinical research for rare diseases and improve healthcare and patient care planning. Integrating these registries into the European health data space will be a major step. This will create a sea of opportunities for patients to access their data and promote innovation,” Galina said.
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“Member countries have unique powers when it comes to measuring their health systems. But the EU has shared powers in matters of public health and has great power when it comes to drug law,” Moretti said.
“Health care systems are managed at the national level, but the EU can and should create an environment for every patient to have a chance to be cured and to receive accessible and equal treatment,” he concluded.
Alessandra Moretti Member of the European Parliament, representing the northeastern Italian region of Veneto. Alessandra is a member of the Committee on Environment, Public Health and Food Safety, the Committee on Foreign Affairs and the Committee on Women’s Rights and Gender Equality.
Yann Le Cam Executive Director, EURORDIS – Rare Diseases Europe. Yann founded Rare Diseases International (RDI) in 2009. He is an elected member of the RDI Council and Chair of the RDI Advocacy Committee. Yann is also a founding member of the NGO Committee on Rare Diseases and its Vice-Chairman.
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David Nestor Head of Neuromuscular Disease, Europe, Canada and Partner Markets at Biogen. David leads a regional team responsible for the strategic planning and execution of Biogen’s programs in Spinal Muscular Atrophy and Amyotrophic Later Sclerosis.
Philippe Berta Member of the French National Assembly and President of the research group on rare diseases.
Science writer Jeremy Wilks covers everything from climate change to health innovation. He has reported on science research, inventions and
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