Letter to AMEQUIS, the Evaluation Body appointed to evaluate the EU’s ERN system

From: Philippe Pakter
Date: 22 March 2023
Subject: ERN-Cranio – feedback

Dear AMEQUIS Evaluation Body,

Last Friday 17 March you and your colleagues from AMEQUIS, the Evaluation Body appointed by the European Commission, were kind enough to gather feedback from the patient representatives of ERN-Cranio.

During the meeting you asked about patient involvement in the development of Clinical Practice Guidelines.

ERN-Cranio recently produced EU Clinical Practice Guidelines for the rare disease, Pierre Robin Sequence. Concerning this EU-funded ERN-Cranio project:

1. ERN-Cranio’s working group to create EU Guidelines for Pierre Robin Sequence included zero Pierre Robin Sequence patients.
2. ERN-Cranio’s consensus vote on these EU Guidelines for Pierre Robin Sequence included zero Pierre Robin Sequence patients.

Zero Pierre Robin Sequence patients in the working group and zero Pierre Robin Sequence patients in the consensus vote for ERN-Cranio’s EU-funded Clinical Practice Guidelines for the rare disease, Pierre Robin Sequence.

This EU-funded document which ERN-Cranio produced fails to meet the minimum standards of the EU’s ERN system, which legally requires that such EU-funded materials be “patient-centered”. Zero and zero does not equal patient-centered.

I am writing on behalf of Pierre Robin Europe, as the organization’s chairman. We represent patients with the rare disease, Pierre Robin Sequence. In ERN-Cranio, we are the only patient representatives who represent patients with Pierre Robin Sequence. We, Pierre Robin Europe, have been a member of ERN-Cranio since 2018; thus we were officially admitted to ERN-Cranio several years before this Pierre Robin Sequence Guideline development project began. ERN-Cranio’s Coordinator, Dr. Irene Mathijssen, intentionally excluded us from this EU-funded Guideline Development Project. This raises several questions, including the following:

• why did ERN-Cranio Coordinator Dr. Irene Mathijssen exclude us from this guideline development project when we are the only patient representatives in ERN-Cranio who represent Pierre Robin Sequence patients?
• why did the highly respected Coordinator of another ERN who won the EURORDIS “Black Pearl” award for his outstanding contributions to the rare disease movement send an official letter of complaint to the German Health Minister concerning this particular ERN-Cranio Pierre Robin Sequence guideline development project?
• how can the European Commission, which provided ERN-Cranio with 50,000 euros of direct funding for this Pierre Robin Sequence guideline development project, claim that it is “patient-centered”, when the working group had zero Pierre Robin Sequence patients, and the consensus vote had zero Pierre Robin Sequence patients?
• what mechanism is in place in the EU’s ERN system to ensure accountability when an EU-funded project demonstrates this level of dysfunction?

[…]

We look forward to speaking with you. Thank you.

Kind regards,

Philippe Pakter
Chairman, Pierre Robin Europe: ERN-Cranio’s official patient organization for Pierre Robin Sequence
Member, EURORDIS, The European Organisation for Rare Diseases
Member, VSOP, Vereniging Samenwerkende Ouder-en Patiëntenorganisaties
PhD candidate, law: “Access to healthcare in Europe: the effectiveness of EU legislation in the context of rare disease patients”