Rare Diseases Day: How the French Presidency can change the rare disease landscape

The Parliament Magazine spoke to French and European policymakers to find out.

By Rajnish Singh

Rajnish Singh is Political Engagement Manager at Dods

28 Feb 2022

Seizing Opportunities

Philippe Berta, President of the rare diseases study group in the French Assembly, says the French Presidency of the EU is an opportunity to take advantage of future innovations

With the development of personalised medicine, in the future, the challenges facing rare diseases today will be no more acute than those of the majority of diseases. Although rare diseases affect three million people in France, and 30 million people in Europe, they are divided into 7,000 pathologies. The low number of patients per country for each syndrome — sometimes as low as one, two or three for ultra-rare diseases — makes working at European level relevant.

Today we can pave the way, clear the path, and invent what will become the norm for the future. The French Presidency of the EU is our chance to seize this opportunity

For example, in the future the treatment of cancers by precision techniques will only target restricted populations and will require a change of scale. Today we can pave the way, clear the path, and invent what will become the norm for the future. The French Presidency of the EU is our chance to seize this opportunity. The health crisis has demonstrated that, even without its own marked competence in the field of health, the European Union is an asset in this area for its members. Rare 2030 has formulated well-founded, documented and realistic proposals. Patients, the medical profession and industry are mobilised to advance research, the marketing of drugs and the care pathway. In short, all the ingredients are there, all we need now is the political will.


A Rare Chance

With the French Presidency at the helm, this year is an opportunity to help people with rare diseases connect, support and inspire each other, says David Lega (SE, EPP), a member of the European Parliament living with the rare disease AMC

Growing up with a rare disease can be hard. Many children with rare diseases grow up feeling excluded. This year, with the French Presidency of the Council of the European Union, we have  an opportunity to improve the conditions for the people who are affected by rare diseases. One issue I want addressed this year is the lack of networks connecting people who are affected by rare diseases. It is vital that children with rare diseases also feel hopeful and excited about their future, like all children deserve to do. But people with rare diseases are rarely in the spotlight, and these diseases are indeed “rare”. For this reason, I believe EU Member States should support networks across the Union, giving everyone affected by rare diseases a place where they can connect. I think we should offer better platforms for people to learn for themselves that rare diseases don’t have to define their lives and that we can live a full life. Many people with rare diseases have inspiring lives, beautiful families, and prosperous careers; let’s highlight that. Let’s create platforms for support and inspiration.


Stars are aligned

A new European action plan to unify various polices is needed says Frédérique Ries, vice-president of the Renew Group in the European Parliament

Rare diseases represent an area where European action brings true added value, and the most effective strategies are cross-border and EU-wide. Great progress has been made thanks to scientific advances and efforts at EU and national levels. However, too many people living with a rare disease face unmet needs and inequities in accessing diagnosis, treatments, and care. The priority for patients and their families today must be to move away from silos. The Foresight Study Rare 2030, a project I initiated in 2017 to project future policies for rare diseases, highlighted the need for greater coherence between European policies. The assessment was clear: we need a new European action plan to unify the policies that touch the lives of the 30 million people in Europe living with a rare disease.  The stars are aligned: the European Parliament recently showed its support during a debate on Europe’s Action Plan for Rare Diseases. A position I will have the honour to defend at the Ministerial Conference organised on the issue by the French Presidency of the Council this 28th February, on Rare Disease Day.


Building An EU Health Union

By bringing health policy together across Europe, we can bring real benefit to rare disease patients everywhere, argues MEP Cindy Franssen (BE, EPP)

Around 30 million Europeans suffer from a rare disease, however, because they affect fewer than five in 10,000 people, good treatment is often hard to find and expensive. By building a true EU Health Union, we can drastically improve treatment and access.

I am happy to see that the topic of health is high on the agenda of the French presidency, starting with the Conference on Rare Diseases in February

In the European Parliament’s Special Committee on Beating Cancer, we have looked at how to improve treatment of rare cancers. Often, the best possible treatment is not found in your own country. Therefore, we want to enable patients to move across borders quickly and easily. This is not possible without a European system of collecting and sharing data. The same goes for research. The EU also has to develop a system that supports the development of medicines and treatments with real added value. The upcoming revision of EU pharmaceutical legislation will not only be crucial but will ensure treatments are affordable. We also advocate for better joint procurement procedures for rare disease medicines. I believe all these actions, together with identifying even more rare diseases, will really benefit patients everywhere. I am happy to see that the topic of health is high on the agenda of the French presidency, starting with the Conference on Rare Diseases in February. We have considered health as a strictly national competence for too long. I believe the last few months have shown the enormous added value the EU can bring. It is time to fully capitalise on this potential and build an EU Health Union.


A Long Term Plan

We need EU-level coordination if we are to address the unmet needs of Europe’s 30 million rare disease patients, believes MEP Kateřina Konečná (CZ, GUE/NGL)

We need a plan for the 30 million EU citizens living with a rare disease for the next decade. Since the last overarching EU policy was introduced in 2009, our technology and understanding have improved dramatically. These diseases can only be effectively tackled at European level. We must also use the lessons learned over the last two years in our fight with the pandemic. The needs of rare disease patients require a greater level of cross-border healthcare cooperation. We need to transform the experience of joint procurements of vaccines and Covid-19 drugs into a permanent orphan drug purchasing system that will allow more patients to access treatment. We need to introduce massive screening of new-borns to give them a chance to live a normal life. These are all steps that we cannot manage without coordination through a common a plan that would set out objectives and policy recommendations for Member States. For this reason, my colleagues and I pressured the French Presidency — as a champion of EU cooperation in this area — to address the issue of the new plan through a debate in plenary, because they should open the discussion for a proposal.

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