CAVOD: Advocating to improve orphan drug access
The creation of a Working Party for European collaboration on the scientific assessment of the Clinical Added Value of Orphan Drugs
Many rare disease patients in Europe are not accessing orphan drugs that have life-saving potential. This is often because national authorities take too long deciding on the pricing and reimbursement of these drugs, thus delaying their market provision; or because companies are facing highly fragmented national procedures.
In order to eliminate this bottleneck, EURORDIS, industry and academic leaders in the field of orphan drugs have developed a proposal to the European Commission and the European Medicines Agency (EMA) for the establishment of a Working Party for European collaboration toward common scientific assessment of the clinical added value of orphan drugs. The proposed Working Party would facilitate collaboration amongst EU level authorities and Member States in order to make the most of already existing information at the EU level, to help national health authorities make their pricing and reimbursement decisions, thus improving effective access to orphan drugs by rare disease patients. This approach has been recommended by consensus by all 27 Member States, industry and patient groups in the framework of the EU Pharmaceutical Forum.
Currently, EMA's scientific committees rigorously assess orphan drugs during the review process for marketing authorisation, for paediatric studies, and to maintain their orphan status. They are already required to show that there is no existing satisfactory treatment or that the new treatment offers a significant benefit over existing therapeutic interventions. In themselves, these reviews help identify the value of the orphan medicine. But, currently, these scientific reviews are not gathered in a simple document nor made transparently available in a usable way to Member States. A Working Party - situated at the EMA, where the scientific review takes place - would be able to bring together all the scientific evaluations into one useable document. Additionally, by working together, Member States would be able to coordinate their respective requests to the sponsor, so as to define the minimum data set required to understand the place of the product in the therapeutic strategy in real life settings. This is important to avoid the current situation where different Member States ask drug companies for different development programmes to demonstrate the in-use effectiveness of the product.
“Unnecessary delays in national decisions on pricing and reimbursement are a major obstacle impeding patient access to orphan drugs. An orphan drug is not a medicine if it doesn't reach the patients,” says EURORDIS Chief Executive Officer Yann Le Cam. “The Common Assessment of the Clinical Added Value of Orphan Drugs is the way forward to address this issue. It has a broad consensus among stakeholders and it is well validated politically. We are asking that, in the spirit of the EU Orphan Drug Regulation, a Working Party be created immediately and that it be located at the EMA, where the expertise and data are to be found.”
In order to encourage Member States to use the Common Assessment Reports, the proposal also includes a set of recommendations to Member States to ensure they will use the Reports and promote innovative pricing policy to speed up access, within their National Plans for Rare Diseases.
The Proposal for the Practical Implementation of Policy Principles to Improve Access to Orphan Drugs in the EU is the result of an intense two-year long process of consultation amongst patient representatives, industry representatives, and EU decision-makers at the Commission and EMA.
“Although we have been fighting for this idea for many years, it has particular relevance today, as three of the most important pieces of EU strategy on rare diseases (the EU High Level Pharmaceutical Forum conclusions and recommendations, the Commission Communication and the Council Recommendation on a European Action in the Field of Rare Diseases) have recently called for increased cooperation between EU level authorities and Member States in order to improve access to orphan drugs for people living with rare diseases,” argues Flaminia Macchia, Director for European Public Affairs at EURORDIS.
Patient organisations, and industry and academic leaders in the field of orphan drugs have been asked to co-sign the paper, in order to give it the necessary political weight for its implementation. Already pharmaceutical industry federations such as EBE (EFPIA) and EuropaBio have voiced their support for this initiative.
“We are supporting this initiative as a way to turn the political commitments made by Member States and stakeholders, during e.g. the High Level Pharmaceutical Forum and the EU Council Recommendation, to increase access to treatments for rare and serious diseases,” explains Wills Hughes-Wilson, Senior Director, Health Policy Europe at Genzyme and member of the joint EBE-EuropaBio industry Task Force on Orphan Drugs and Rare Diseases. “If all interested stakeholders work together, we stand a better chance of creating a functioning, collaborative system that successfully achieves the goal of increasing patient access. We are looking forward to continuing the dialogue on an ongoing basis with all the stakeholders to achieve this.”
What's next? Discussions are currently underway with pharmaceutical industry federations, high officials at the EMA and the European Commission. The proposal will be presented shortly to the Committee for Orphan Medicinal Products (COMP) at the EMA; and discussions will take place with Member States. EURORDIS will continue to work to build understanding of the need for this process amongst all the stakeholders, who will need to play a role in creating this collaborative system - and who will make the most of all opportunities. The dialogue continued at the EPPOSI workshop in Brussels on 26-27 October 2009 and will be furthered at the ERTC on 11 December 2009.
Sign up to this initiative by sending a message of support to: firstname.lastname@example.org
This article was previously published in the December 2009 issue of our newsletter.
Author: Paloma Tejada
Photo credits: © Europa, 2009, EURORDIS & EMA