Innovation in rare diseases: How can Europe be a global leader? | Euronews Debates

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Euronews Debates – Innovation in rare diseases: How can Europe be a global leader?


Euronews has gathered a panel of experts, industry leaders and key players in Brussels to address the issue of how rare disease provision can be advanced in the EU.

Debate host and Euronews science journalist Jeremy Wilks will accompany the panel as they consider rare diseases and the battle for progress. They will tackle questions including how the private and public sectors can work efficiently together and what the future formula for better healthcare should look like.

The event is organised on the sidelines of Rare Disease Awareness month, with Rare Disease Day taking place on 28 February.

You can watch the debate live in this article on February 15 at 15:00 CET.

You can register to watch our debate here:

In the European Union, a disease is considered rare if it affects fewer than five people in 10,000.

This might sound small, but in practice, six to eight per cent of the bloc’s population is affected by a distinct rare condition like this, which translates to between 27 and 36 million people.

Most of these patients suffer from even rarer diseases that affect just one person in 100,000 or more, according to EU data.

Approximately 5,000-8,000 distinct rare diseases in the EU span all medical areas and include neurological, immunological and metabolic diseases, as well as rare cancers.

Our four panellists joining the debate around such conditions are:

© euronewsAlessandra Moretti© euronews

Alessandra Moretti Member of the European Parliament, representing the northeast Italian region of Veneto. Alessandra is a member of the Committee on the Environment, Public Health and Food Safety, the Committee on Foreign Affairs and the Committee on Women’s Rights and Gender Equality.

© euronewsYann Le Cam© euronews

Yann Le Cam CEO, EURORDIS – Rare Diseases Europe. Yann initiated 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 for Rare Diseases and its Vice-Chair.

© euronewsDavid Nestor© euronews

David Nestor Head of Neuromuscular Disease, Europe, Canada and Partner Markets at Biogen. David leads the regional team accountable for the strategic planning and execution of Biogen’s programmes in Spinal Muscular Atrophy and Amyotrophic Later Sclerosis.

© euronewsPhilippe Berta© euronews

Philippe Berta Member of the National Assembly of France & President of the study group on rare diseases.

EuronewsJeremy WilksEuronews

Jeremy Wilks Euronews reporter and debate moderator

Euronews science reporter Jeremy Wilks covers everything from climate change to healthcare innovation. He has reported on science research, innovation and digital technology across Europe for over a decade. He regularly hosts live debates both on Euronews digital platforms and at large conference events.

Jeremy is the presenter of the monthly Climate Now series on Euronews.

The debate will also feature special guests including Sandra Gallina, Director General, DG Santé, European Commission, Doddie Weir OBE, Rugby legend in Scotland and ALS/MND advocate, Dolors Montserrat, Member of the European Parliament, Alexander Natz, Secretary General, EUCOPE, and Nathalie Moll, Director General, EFPIA.

What will the panel be discussing?

Our panel will look at the challenges facing policymakers in devising a framework that improves the development of orphan medicinal products. They’ll also be discussing how innovation in the medicines and treatments that are essential for those with rare diseases, is notoriously challenging, due to a lack of knowledge and small patient populations.

The group of experts will also consider the work and recommendations of the European Expert Group on Orphan Drug Incentives, an expert unit that brings together representatives of the broad rare disease community.

After coming together in 2020, the group’s goal is to develop policy proposals to facilitate EU policymakers and its members include researchers, academics, patient representatives, members of the investor community, individuals from rare disease companies and also trade associations.


What is the EU’s current strategy on rare diseases?

Over the last 20 years, innovation surrounding rare diseases in Europe has improved dramatically, with a specific framework put in place by the EU called the Orphan Medicinal Products (OMP) Regulation.

This rule concerns products developed to treat medical conditions which, because they are so rare, would not be profitable to produce without supporting governmental measures.

Previously only eight orphan medicines were available for patients but this number has risen to more than 200, resulting in the pharmaceutical sector delivering treatments for up to 6.3 million patients.

In November 2020, the European Commission published the EU Pharma 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 OMP regulation in 2022.

How is the EU encouraging rare disease innovation?

Incentives for pharmaceuticals

The European Commission currently provides a number of incentives to the pharmaceutical sector to encourage the development of OMPs. These include fee reductions or exemptions from regulatory assessment procedures, a 10-year market exclusivity period post-authorisation, as well as scientific and protocol assistance, including an early dialogue with the regulator.

While OMP Regulation is estimated to have led to the production of 74 per cent of authorised OMPs between 2000-2017, 95 per cent of rare diseases remain without authorised treatment.

The European Commission published its Staff Working Document on the Evaluation of OMPs in August 2020. According to their evaluation, the Commission believes that Orphan Regulation has over-incentivised innovation, resulting in high prices for orphan medications. The Commission is therefore considering limiting incentives to areas of unmet medical need.

In response to potential policy changes, the European Expert Group of Orphan Drug Incentives is hoping to become a source of potential solutions for the OMP Regulation Evaluation. The group brings together a diverse range of rare disease experts, including EURORDIS: The Voice of Rare Disease Patients in Europe and the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE).

How else is the EU leading on rare disease innovation?

In 2000, the European Commission worked with INSERM (the French National Institute for Health and Medical Research) to develop Orphanet.

The online platform provides doctors, researchers and pharmaceutical groups with information about rare diseases, in order to improve diagnosis and treatment.

The 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.

Since its inception, Orphanet has grown dramatically and the consortium now includes 40 countries, both in Europe and the wider world.

Is a European platform for rare disease registration in the pipeline?

Patient databases can help to encourage the development of clinical research into rare diseases, while also improving patient care too. So in order to streamline research into rare diseases, the European Commission is advocating for the creation of a European Platform on Rare Diseases Registration.

Currently, there are no uniform standards governing the collection and availability of patient registries and databases, and, according to Orphanet, there are 600 rare disease registers in Europe alone. Multiple registers may also exist for the same disease and there are estimated to be registries for only 20 per cent of rare diseases.


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