The EU’s European Reference Network / ERN system is composed of 24 separate healthcare networks, which were established in order to improve the diagnosis and care of patients suffering from rare and complex diseases and conditions. The ERN system was designed to represent the “best of the best” of Europe’s rare disease experts, across the entire EU. ERN-Cranio, which is run by Erasmus Rotterdam Medical Center in the Netherlands, is one of the ERNs. This year they launched a project to create official EU “Clinical Practice Guidelines” for the treatment of babies suffering from a life-threatening rare disease called Pierre Robin Sequence. These EU Clinical Practice Guidelines will be the first international medical guidelines ever produced for this complex rare disease. They will therefore have a significant impact on Pierre Robin Sequence babies for many years to come, and on the medical care these babies receive.
Pierre Robin Europe is an official rare disease patient advocacy organization, an ePAG representative, in ERN-Cranio. Our duty in ERN-Cranio is to promote the interests of patients suffering from the life threatening rare disease, Pierre Robin Sequence. We have watched with increasing concern as this EU-funded project has been managed in a way which we believe violates EU rules, scientific rigor – a view shared by two leading international experts on this rare disease – and once again in an opinion held strongly by respected outside observers, common sense – based on ERN-Cranio’s arbitrary decision to exclude from the drafting group Europe’s most respected Orphanet Centers of Expertise for this rare disease.
We attempted to resolve the issues internally, but our efforts were unsuccessful. We then requested mediation from EURORDIS, which did not resolve the obstacles. We then reached out to the responsible authorities, including the ERN Board of Member States, the European Commission DG SANTE, and the Dutch Ministry of Health; none of these authorities wanted to get involved.
We finally filed official complaints with the EU Ombudsman, concerning inaction by the European Commission, and the Dutch Ombudsman, concerning inaction by the Dutch Ministry of Health. We finally decided to speak out publicly, by publishing our actual correspondence with the authorities in question – the very authorities which are legally mandated to resolve such problems.
Please read these letters; then after reading the letters, consider sharing this page with others, as requested on the bottom of this page. The next time around, this could be an EU ERN Clinical Practice Guideline development project for the rare disease which affects you, your children, your patients, or people you care about. The EU’s ERN system can do and should do much better than this for Europe’s rare disease patients.
Letter to the ERN Board of Member States, 30 June 2021
The ERN Board of Member States, which is responsible for overseeing the ERN system, responded with silence. We decided to approach the European Commission, which funds the ERN system.
Letter to the European Commission DG SANTE, 7 September 2021
The European Commission DG SANTE sent Pierre Robin Europe an official response to the two letters published above. The European Commission wrote that:
“the project for development of ERN Clinical Practice Guidelines (CPG) and Clinical Decision Support Tools (CDST), supported by the Commission under the 3rd Health Programme, is not yet providing any direct support to ERN-Cranio for development of any new guidelines or updates of existing ones. The issues that you raise therefore do not seem to be related to this particular project.”
Then, in the same letter, lower down on the same page, the European Commission wrote:
“Regarding the third question, an amendment of the grant for the coordination of ERN-Cranio under the 3rd Health Programme was accepted in 2020 by the Consumers, Health, Agriculture and Food Executive Agency (CHAFEA) for the transfer of 50.000 € from the international meetings budget, saved due to the COVID-19 restrictions, to subcontracting budget for launching the ERN Clinical Practice Guideline for Pierre Robin Sequence in line with the justifications provided by the beneficiary.”
Thus, the European Commission will not get involved in this matter because they are not providing ERN-Cranio any support to develop these EU Clinical Practice Guidelines for Pierre Robin Sequence; also, the European Commission is providing ERN-Cranio 50.000 € of support to develop these EU Clinical Practice Guidelines for Pierre Robin Sequence.
Is there an intelligent way of responding to a letter like this from the European Commission?
The European Commission did not just give ERN-Cranio 50.000 € to develop these particular EU Clinical Practice Guidelines for Pierre Robin Sequence. The European Commission also spent money, a much larger sum of money, we estimate hundreds of thousands of Euros, to create the EU Methodology which ERNs are supposed to follow when creating EU Clinical Practice Guidelines – the EU’s “Methodological Handbooks & Toolkit for Clinical Practice Guidelines and Clinical Decision Support Tools for Rare Diseases“, as well as a set of templates (“Taxonomy and templates for the European Reference Networks”) which, once again, the ERNs are supposed to use when creating EU Clinical Practice Guidelines. We asked the European Commission DG SANTE how much money these EU ERN documents cost; we have not yet received an answer. However, if the European Commission DG SANTE refuses to qualify 50.000 € as “support”, then the European Commission DG SANTE will not qualify 250.000 € as support either. The European Commission DG SANTE’s overarching goal seems to be to avoid taking responsibility for EU funds spent, and misspent, once those funds have been transferred out of the European Commission’s accounts.
These are public funds, not the European Commission DG SANTE’s private money, and what is at stake here is a crucially important set of EU Clinical Practice Guidelines for babies suffering from a life threatening rare disease. The EU’s ERN “Methodological Handbooks & Toolkit for Clinical Practice Guidelines and Clinical Decision Support Tools for Rare Diseases”, the EU Methodology, which EU citizens bought and paid for, and which is an absolutely outstanding tool, should be used and respected by the ERNs. If not, then the European Commission should take action. So far they have refused to do so. Who pays the price? Babies suffering from a life threatening rare disease, who are being cheated out the top quality EU Clinical Practice Guidelines which they desperately need.
Erasmus Rotterdam Medical Center, which runs ERN-Cranio, and which received the EU funds to create these EU Guidelines, is based in the Netherlands. Pierre Robin Europe therefore contacted the Dutch Ministry of Health, and asked them to intervene. The response we received from the Dutch Ministry of Health, and the 50.000 € letter we received from the European Commission, were printed on different letterhead, but both letters sent the same message: “we are not responsible for this.” Nobody is responsible. This demonstrates an important principle of European Union programs and activities, as described by a noted scholar of EU law: “The actual situation, therefore, is one where there are multiple remedies which are theoretically capable of providing effective redress, but not a single one which effectively does so in practice.”
Pierre Robin Europe did not believe it would make a meaningful difference, but for the sake of truth, and to maintain a full documentary record, we decided to respond to the Dutch Health Minister.
Letter to the Dutch Ministry of Health, 30 September 2021
What to do
Rare disease patients desperately need and are entitled to the very best Clinical Practice Guidelines possible. When drafting EU ERN Clinical Practice Guidelines for a complex rare disease, the best available EU Centers of Expertise should be brought in as members of the drafting group – this is self-evident. In our opinion and in the opinion of actual rare disease healthcare experts, that did not happen here.
Furthermore the medical evidence which these top EU experts bring to the table should be duly incorporated into the project. That did not happen here.
Also, rare disease patients and rare disease patient groups should be brought in as members of the drafting group and should be treated as actual participants and stakeholders, from day one; the EU Methodology which was created for the ERNs, and which the ERNs are supposed to respect, absolutely requires this. That did not happen here.
If you are a rare disease patient, the family member of a rare disease patient, a rare disease expert or healthcare provider, if you play any role in the international rare disease movement, if you believe that “evidence-based medicine” and “patient-centered care” should be taken seriously – then please share this page with others. This situation is not acceptable, and those responsible for correcting it, including the ERN Board of Member States, the European Commission DG SANTE, and the Dutch Ministry of Health – they need to know that people are watching. You don’t need to write hopeless letters to the European Commission or to the Dutch Health Minister; instead, please simply share this page with others.
Rare disease patients and their families, regardless of the rare disease in question, deserve much better than this from the EU’s ERN system.