Since 2000, the European Union has authorised nearly 260 orphan medicinal products, offering significant benefits for people living with rare conditions that are life-threatening or chronically debilitating in terms of clinical impact or care. However, access to these OMPs still varies greatly across EU Member States, and many people living with a rare disease in the EU either do not have, or face significant delays in access to, the treatments they need.
Current access policies fail to address the multifaceted barriers that prevent or delay access. Policymakers focus on supply-side measures and incentivise pharmaceutical companies in the revision of the EU General Pharmaceutical Legislation, such as “launch conditionality”, “obligatory pricing and reimbursement filing”. This approach, however, neglects the fact that the barriers preventing and delaying patient access to medicines are multifaceted. They stem not only from the supply side, but also from the demand side, involving policies, processes, and decisions that fall under the exclusive competence of the respective EU Member States.
Solutions put forward at the EU level alone are not capable of improving access. Enabling early and equitable access requires parallel action at the Member State level who have competencies over their healthcare policies.
Against this background, the European Expert Group on Orphan Drug Incentives has come together to
The group’s work builds on the recognition that only an ambitious policy agenda developed in a multi-stakeholder setting involving both the EU and the Member States can bring about the quantum leap needed to address the unmet needs of people living with a rare disease today.
The OD Expert Group identified eight best practices addressing three key access challenges for OMPs and issued 15 calls to action for their swift adoption. We encourage you to download the infographic for a comprehensive overview.
