MOCA Report a welcome stepping stone, but access to orphan medicinal products remains unfinished
Brussels 08 May 2013, EuropaBio, EFPIA and EBE welcome the final report of the Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA-OMPs) which was endorsed by the Steering Group of the Process on Corporate Responsibility in the field of Pharmaceuticals, and published on 6 May 2013 by the European
The Process was launched by Commission Vice-President Tajani in 2010 with the aim of bringing together Member States, countries of the European Economic Area and relevant stakeholders – including patients and industry – to evaluate a coordinated approach towards Orphan Medicinal Products (OMPs) and to develop a collaborative mechanism to increase access to medicine for patients suffering rare diseases across the EU, irrespective of nationality, economic or social status.
Despite an excellent collaborative report produced by the stakeholders involved under the co-chairmanship of
the Belgian Government and DG Enterprise, it is not yet clear how the report will have impact in practical
terms to address the barriers to access that exist for orphan medicines.
The report concluded that the processes and elements proposed in the MoCA project should take account of
and be linked with existing, on-going projects. In particular, it notes that there should be a coordinated
approach for post-marketing authorisation research activities and further evidence generation. It also firmly
concluded that any such cooperation should be possible within the current legislative framework and that no
changes to the legislative framework were proposed in this context.
Wills Hughes-Wilson, chair of the EuropaBio-EBE Joint Task Force on Rare Diseases and OMPs, stated: “The
EuropaBio-EBE joint task force welcomes the outcome of the Working Group on Mechanism of Coordinated
Access to Orphan Medicinal Products (MoCA-OMPs). In particular, in addition to the organisation of diagnosis
and care, as well as affordability and budget impact, we welcome the report’s focus on a very important aspect for access – which is the identification of the value of a new orphan medicinal product. The Transparent Value Framework adopted as part of the Report will contribute to a structured debate around the ability to assess this. However, the lack of a clear path to implement the Report’s recommendations in such a way that it will have an impact on access, is of concern to industry.
Patients with rare diseases across Europe are still seeing access to therapies denied or delayed, and the diverse data requirements and processes for assessing new orphan medicines in different Member States makes rapid and equitable access across the EU difficult to achieve. We urge the Commission and Member States as part of their plans for rare diseases to develop strategic action plans for implementation – and we are ready to be part of this. We believe that as the purpose of the MoCA Working Group was to develop proposals on a voluntary, non-legislative basis, it is only through continued dialogue and engagement from Member States and other stakeholders, including industry, that access to OMPs can be improved in a coordinated manner across the EU for the benefit of patients. Industry is committed to playing its part in the next steps”.
For more information, please contact:
Communications Manager, EuropaBio
Phone: +32 2 739 1173
GSM: +32 (0) 478 680 301
Or Nicholas Elles
Communications Manager, EFPIA
Phone: +32 2 626 3479
GSM: +32 (0) 470 936 739
To view the final report, please click here
Notes to the editor:
In September 2010 the European Commission launched the Process on Corporate Responsibility in the Field of
Pharmaceuticals focusing on, amongst others areas, non-regulatory conditions for a better access to medicines
following their marketing authorisation.
Under its Platform "Access to Medicines in Europe", EU Member States, countries of the European Economic
Area and relevant stakeholders were invited to participate in a project group to develop the concept of a
coordinated access to orphan medicinal products based on the set up of programmes between companies and
groups of competent authorities, and on a mechanism for the assessment of clinical added value of orphan
medicinal products. The results of the project were intended to be a potential mechanism for approaching this on a collaborative, voluntary basis. The initial idea was to set up a pilot project in a second stage.
Following this call – which was stimulated by the initiative of the Belgian EU Presidency in 2010 “Unmet
medical need and solidarity in Europe: a mechanism for coordinated access to orphan medicinal products
(OMP)” – a number of Member States, experts, patient organisations, industry representatives and other
relevant stake-holders volunteered to participate in the so-called “MoCA” (Mechanism of Coordinated Access
to Orphan Medicinal Products) Working Group.
The purpose of the MoCA Working Group was to develop proposals as to how to create a future voluntary
European collaboration, as well as a pilot project on voluntary basis, to improve access to orphan medicinal
product in Europe.
The MoCA working group is made up of volunteers from: Austria, Belgium, Estonia, Finland, France, Hungary,
Italy, Malta, Netherlands, Portugal, Spain, European Patient Forum (EPF represented by the European
Organisation for Rare Diseases, EURORDIS), Standing Committee of European Doctors (CPME), European Social
Insurance Platform (ESIP), Association Internationale de la Mutualité (AIM), European Federation of
Pharmaceutical Industries and Associations (EFPIA), European Association for Bio-industries (EuropaBio), and
European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), and the European Commission, with
support from the EC-funded project Eminet.
The scope of this project, led by the Belgian National Institute for Health and Disability Insurance (RIZIV/INAMI) and the EC Directorate General Enterprise and Industry, is to identify possible options for the creation of a mechanism of coordinated access to orphan medicinal products, based on a voluntary, non-legislative, non-regulatory and non-binding collaboration among stakeholders who are willing to work together.
About EuropaBio: EuropaBio is the European Association for Bioindustries, bringing together bioscience companies from all fields of research and development, testing, manufacturing and distribution of biotechnology products. It has 56 corporate members, 14 associate members and Bio Regions and 19 National Biotechnology Associations- representing some 1800 small and medium sized enterprises across Europe. Its members are involved in research, development, testing, manufacturing and commercialisation of biotechnology products and processes, and have a wide range of activities: human and animal health care, diagnostics, bio-informatics, chemicals, crop protection, agriculture, food and environmental products and services.
About EFPIA: The European Federation of Pharmaceutical Industries and Associations represents the pharmaceutical industry operating in Europe. Through its direct membership of 33 national associations and 39 leading pharmaceutical companies, EFPIA is the voice on the EU scene of 1900 companies committed to researching, developing and bringing to patients new medicines that will improve health and the quality of life around the world.
About EBE: The European Biopharmaceutical Enterprises (EBE) is the European trade association that represents biopharmaceutical companies of all sizes operating in Europe. It has 53 member companies - of which many are small and medium sized companies - engaged in the research, development, manufacturing and marketing of new medicinal products using biotechnology. EBE also operates as the biotechnology arm of EFPIA, the European pharmaceutical industry federation.