Czech presidency commits to follow up on EU rare diseases action plan –

Czech presidency commits to follow up on EU rare diseases action plan –

The Czech Presidency of the EU Council said it will cooperate with forthcoming Council presidencies to create an EU action plan for rare diseases.

Read the original Czech article here.

In the EU, about 30 million citizens in Europe are affected by more than 6,000 various rare diseases, of which only a fraction have available treatments.

During its time at the helm of the rotating presidency of the EU Council, the Czechs sought to push for the creation of an EU action plan for rare diseases, seeking additional financing from other countries to ensure that there are no ‘second-class patients’.

“A European action plan on rare diseases is a big issue for the Czech presidency,” Czech Deputy Health Minister Jakub Dvořáček said in Brussels in October.

However, there is still a way to go before significant progress is made. A proposal to revise the orphan medicinal products legislation is scheduled for 2023, and the final meeting of health ministers for this Council rotation, slated for 9 December, is set to instead focus on the health data space and cancer screening.

“If we will not manage to have this launch during our time, I can assure you we will work on that with our Swedish colleagues and incoming presidencies and I believe it will happen,” said Dvořáček.

“We all have to make sure that in the upcoming years we do not abandon a goal we have been working on for a long time,” he added.

Regulation as a cornerstone

Before the start of the millennium, rare diseases were not systematically tackled in Europe, which was reflected in the number of drugs developed – close to zero. But regulations introduced in 2000 and 2007 on orphan drugs and paediatric medicines shifted the picture: To date, over 160 therapies for rare diseases have received approval.

The incentive system should remain predictable, while stimulating innovation and making Europe more competitive globally, stakeholders urged.

“We must not resort to the dogmatism of increasing or decreasing incentives. It is about doing what will entice investment where it is needed. That is the role of regulation,” said Yann Le Cam, executive director of EURORDIS, the patient umbrella organisation for rare diseases.

Some have proposed making incentives conditional on the availability of drugs in all European markets. However, the industry says such an “experiment” cannot work, as many countries do not have the infrastructure in place to use the new drugs properly and efficiently, and such an obligation will only deter investors.

“No investors will come, it will wipe Europe off the map. Please let’s not make that mistake,” urged Nathalie Moll, Director of the European Federation of Pharmaceutical Industries and Associations (EFPIA).

Inspiration and cooperation

One major step forward was the creation of European Reference Networks (ERNs) for rare diseases in 2017, where 1,500 European centres of expertise now share the necessary knowledge and experience.

However, Milan Macek of Prague’s Charles University and Motol University Hospital highlighted that the networks face a number of challenges, particularly regarding financing for operations. As a result, doctors and other professionals work for free and “mainly out of enthusiasm”, Macek said.

“We are working very hard to integrate ERNs into national health systems. We are still a virtual institution without buildings, which is not such a problem, but we are not officially recognised at the European level,” Macek explained.

According to Macek, networks lack European guidelines for diagnosis, as well as common standards of care across the EU. The lack of attractiveness to younger generations or the challenge of meeting GDPR data protection rules is also a problem.

Victoria Hedley, who researches rare diseases at Newcastle University, sees great potential for ERNs.

“ERNs are in a perfect position. They link care and research, which is key in rare diseases. But to make meaningful progress forward, they need collaboration with the pharmaceutical industry, which has a number of barriers so far,” Hedley said.

Communication as a key

Macek also highlighted that communication between stakeholders and patients is crucial.

In the Czech Republic, this has been successful, as they have included patients in the advisory committee of the Ministry of Health, which issues recommendations on the basis of which decisions on the reimbursement of medicines are made.

“It took a long time, but it has been successful,” said Deputy Minister Dvořáček.

At the European level, progress is slower. Although the European Commission is talking to patient organisations, Le Cam said it often does not base its decisions on the data that patients provide.

The director added that “the debate is not sufficiently informed”, so other relevant players are not invited. In this regard, EURORDIS proposes, for example, strengthening the role of the Committee for Orphan Medicinal Products (COMP), which recommends medicines for approval within the European Medicines Agency (EMA).

Despite the Commission’s assertion that it places patients first in its revisions of regulations, Le Cam remains critical, saying that it often proposes innovations not requested by patients at all.

[Edited by Nathalie Weatherald]

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