Position papers

Our 2020 Vision

EuropaBio-EFPIA Joint Task Force on Rare Diseases and Orphan Medicinal Products

A call to Europe

Member States, European Commission and European Parliament

  1. It is crucial that the focus on rare diseases is maintained as a health policy priority and that decision-makers work with all stakeholders, to develop smooth pathways at EU and national level that bring innovative therapies to patients.
  2. Member States are encouraged to fulfill the commitments made in the Council Recommendation on an action in the field of rare diseases to adopt and implement a national plan for rare diseases, with sufficient funding.
  3. We support increased cooperation between Member States to improve accurate early diagnosis of rare diseases and develop/harmonise the criteria to decide which conditions countries should add to their newborn screening panels.
  4. We support a more holistic, joined-up approach that addresses the challenges of patient access to treatments. The orphan medicinal products industry is fully committed to making this approach a reality. We also support the adaptation of Health Technology Assessment processes, methodologies and decision-making frameworks to the specificities of orphan medicinal products, so that rare disease patients have equitable access to treatments.
  5. We encourage policy makers to maintain their commitment on what EU health systems can and should deliver in terms of preventing and treating rare diseases. Maintaining such an ambition will ensure that new products continue to be developed, contributing to a healthier and more productive society in the future, while creating high-quality jobs and adding value to the European economy.

About the Joint EFPIA-EuropaBio Task Force on Rare Diseases and Orphan Medicines

The Joint Task Force between EFPIA (the European Federation of Pharmaceutical Industries and Associations) and EuropaBio (the European Association for Bioindustries) on Orphan Medicinal Products and Rare Diseases is a European alliance of over 45 member companies committed to the development of orphan medicinal products (OMPs) and improving the lives of patients with rare diseases.

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