To celebrate the Rare Disease Day on Feb 28, 2017, we are pleased to share with our readers the Reflection Paper just published by EURORDIS this month.
http://www.eurordis.org/sites/default/files/reflexion-paper.pdf
This paper is a contribution of the community of rare disease patient advocates. It is a work in-progress by EURORDIS and its members, offering a synthesis of their analysis, reflections and perspectives on the issue of access to orphan medicines.
EURORDIS does not pretend to propose “the solution”, “the new model” or “the new deal”. This paper expresses a set of possibilities, not a position.
In his introduction, Yann le Cam, CEO EURORDIS-Rare Diseases Europe, expresses strong beliefs:
“We are strong about only one thing: when it comes to patients’ access, we do not take “no” for an answer. We cannot passively curb under the weaknesses of the current model. We stand up and speak for patient first. We, rare diseases patients, are not the problem: we are part of the solution.
Our beliefs are that viable new models or strategies can only be:

  • addressed if elevated to a more political level within each stakeholder constituency;
  • developed through a collective conversation involving all stakeholders, so as to be negotiated and co-constructed in a socially responsible manner;
  • shaped into a comprehensive framework with a global outlook in addition to addressing each technical element, each possibility.

This paper is not the “One-Text Paper” for the proposed cooperative process to reach mutually acceptable solutions that respect all stakeholders. If accepted and trusted by the participants to this multi-stakeholder symposium, the One-Text will be elaborated by a multi-stakeholder “group of drafters”, mostly composed of members of the Programme Committee, potentially starting from day one after the EURORDIS symposium, (Feb 22-23, 2017) and will be progressively developed in an iterative process of broad consultations until the next symposium in February 2018.
For more details about EURORDIS, please go to http://www.eurordis.org/