Final Europlan conference takes stock of the progress of Member State national strategies for rare diseases
On 25 February in Rome, Italy, the final conference of the Europlan project was held. Europlan, a three-year DG Sanco-funded project coordinated by the Italian National Centre for Rare Diseases (Instituto Superiore di Sanita) launched in April 2008, as an instrument to help the European Union Member States (MS) define a strategic plan for rare diseases following the adoption of a Council Recommendation on an Action in the Field of Rare Diseases that calls on the MS to elaborate and adopt a rare disease plan or strategy by the end of 2013. Europlan is an inclusive project, with 57 associated and collaborating partners - including clinicians, scientists, health authorities, and patient groups from 34 countries. The Europlan project created a “toolbox” designed to aid countries determine their priority areas and actions to include in a national plan. Some 15 individual EU countries hosted national conferences via the project. These conferences, designed to move forward the process of developing a national strategy for rare diseases, followed a format based on Europlan guidance documents. The final reports of these national conferences are available on the Eurordis website (consult the national conference final reports).
The European dimension
At the final Europlan meeting, it was observed that one of the strongest elements of the Europlan project was its role in adding the European dimension to individual national strategies. This point is critical to the field of rare diseases, which relies on coordination and collaboration at the European and international levels. The recent Eurobarometer survey results demonstrate that there is support for European cooperation (learn more). Catherine Berens (DG Research) adduced the E-Rare project as an effective strategy for the funding of collaborative research.
A Round Table meeting reviewed some of the overall results of the Europlan project such as its role in harmonising concepts and terminology between the MS and in helping to raise awareness at the MS level for key EU documents in the field of rare diseases and orphan drugs. During this session, the need for integrating the elements of the national rare disease plans into the national health care systems was discussed. The importance of mapping existing resources - which Orphanet, the pan-European information portal for rare diseases and orphan drugs, is doing – was also evoked. Other elements identified include the need for inclusivity – ie, involving all the various stakeholders in the development of a national plan; the need for the national protocols for diagnostics and care of a disease to include the provisions for patient coverage for testing and care; and the healthcare pathways – the multidisciplinary algorithms of care structured to support the implementation of clinical guidelines and protocols.
At the national level…
Discussion of the status of particular countries was also raised. While Bulgaria has a concrete plan, accessing funding remains problematic, especially in the area of diagnostics. Croatia hopes to put forward a plan in 2012. Denmark is in a period of regression, illustrated by the country’s information centre exclusively for rare diseases that has been extended to encompass all diseases. Greece has a plan on paper, but it is not yet legally recognised and the country has no national committee to implement it. There are also significant problems with access to orphan drugs in Greece. Starting a process to develop a plan in a country with 21 autonomous regions is a priority Italy – but the country’s organisation presents a daunting challenge. Italy also reports a long time for orphan drug approvals to be processed. The Netherlands is a country with a solid general health plan, which could explain why the Minister of Health is not in favour of developing a plan specifically for rare diseases. Furthermore, the country’s Steering Committee for Orphan Drugs is to be shelved at the end of 2011. In Poland, the process of elaborating a plan has not yet began, but awareness is increasing. Poland needs to focus on all elements of rare disease strategising – not just the orphan drugs. Spain does have a plan, but it has neither a budget nor a time-table. The UK seems to be moving forward, thanks in large part to the steam of the Rare Disease UK and similar patient-driven efforts.
A challenging dynamic
With several plans existing only on paper, other countries such as Denmark and the Netherlands reporting a regression, and other MS lacking resources, the dream that each MS will have a specific strategy to care for its rare disease patients, and which includes cooperation between the EU countries to share resources, is a fragile one. This is a critical time for each stakeholder to continue acting as a catalyst to push change forward. The recent adoption of the Cross Border Health Care Directive increases the need for concerted effort, with each EU country identifying its pockets of expertise and making them known, within the context of acknowledging and respecting the individual dynamic of each country, particularly its size and resources. Analysis of the results of this first Europlan project can help refine the second leg of the plan, which is being funded via the upcoming DG Sanco three-year joint action, Support to the implementation of national plans/strategies on rare diseases and related measures to implement Council Recommendation and Commission Communication on rare diseases. The second Europlan will continue to offer support and guidance to countries that have delineated a strategy and will aid countries that have not developed a plan to move forward, taking into account the specifics of each country in terms of size, prioritisation of measures and health care systems. There will also be an emphasis on the exchange of expertise between countries, as well as identifying outcome indicators that can be monitored. Twenty national conferences are being planned for the second Europlan.