How Fast is Europe Filling in the Gaps Identified in Its Rare Disease Networks?

June 7, 2019
Peter O'Donnell
Peter O'Donnell

Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.

Applied Clinical Trials

Peter O'Donnell writes of the European Commission's recently published reports suggesting that some of the gaps were being filled in registries in the field of rare diseases.

Just days after European Reference Networks were criticized over serious gaps in the European Union planning for them, the European Commission published two reports suggesting that some of the gaps were being filled.

The EU's own internal watchdog, known as the Court of Auditors, released in early June an evaluation of the policy framework that the EU has set up to for tackling 24 rare or complex diseases by concentrating expertise via 24 virtual networks.

The watchdog concluded that this "ambitious innovation" suffers from weaknesses in the way the networks operate and in the arrangements for keeping them going.

"The Commission has not provided a clear vision for their future financing and how to develop and integrate them into national healthcare systems," says the report.

In what looks like a deliberately-timed response, the Commission almost immediately published a report of recent moves aimed at accelerating the integration of the networks into national systems.

"Hospital managers committed to strengthen collaboration to support the implementation of the ERNs", it announced. At a meeting in May of 54 hospitals linked to the networks, the managers are recorded as seeing the initiative as "an opportunity to leverage innovation, not only in the field of rare diseases, but also in the way hospitals are managed and interconnected across Europe through networking."

For the next year, they "will continue to cooperate, coordinate their actions and exchange best practices in four main areas: communication, e-training and e-learning actions; data management; human potential and transversal support service", says the Commission report.

Among the specific issues they discussed - alongside invited national health officials and rare disease experts and patient organizations - was the contribution that hospital managers can make to define the role of the ERNs in the national health systems. Another was the controversial issue of how far hospital managers should support daily involvement of their own health professionals in the ERNs – because there has been resistance to allocating working time, providing substitute staff, or providing additional funds or administrative support.

"The commitment and support of the ERNs hospital managers involved in the European Reference Networks is essential for the success of the networks, as well as their close collaboration with the other key players of the ERN community", concluded the Commission report of the meeting.

In parallel the Commission released another report on support for setting up registries of patients affected by rare diseases, which coincided with another ERN meeting in May to discuss their work on in this field.

This report appears to respond to accusations from the watchdog that the EU has been neglecting the coherent development of registries (a gap that was commented on recently in these columns, too.)

The watchdog pointed out that in 2008, the Commission already identified the importance of developing registries to enable epidemiological and clinical research on rare diseases and stressed the need for their long-term sustainability. But it is uncomplimentary about the Commission's approach. The Commission's joint research center started work with funding from the EU health program on a European Platform for Rare Diseases Registries, aiming to tackle fragmentation across Europe with agreed standards. But in parallel, says the watchdog, the Commission also used its research and innovation program to support RD-Connect to link patient registries for rare disease research. "As a result, the Commission is funding two projects with potentially overlapping outputs."

At their May meeting, the ERNs "had an extensive discussion on the registries", reports the Commission. The report also records that the ERNs have conducted "a short survey" among their members "to map the needs of the ERNs in terms of registries". That would seem to confirm rather than to refute the suggestion that on this area too, the ERNs still have a long way to go.

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