Abstract
Background The European orphan legislative framework has been successful in delivering approved new treatments since its establishment in 2000. But the data needed to reach a positive risk-benefit analysis for regulatory approval often falls short of that needed by Member States to justify reimbursed availability to patients. This gap leads to differences in availability of new orphan medicines. The CAVOD proposals build on more than 12 years of formal collaborative approaches to European orphan drug policy, which have consistently agreed on the need to gather and share information at a European level. They would establish a process for the exchange of knowledge from the earliest stage in a drug’s development, through to inlife outcomes after a treatment is available to patients, with the objective of bundling fragmented know-how to allow the timely production of well-informed decisions on national pricing and reimbursement, while respecting existing roles, responsibilities and competences.
Highlights
The European orphan legislative framework has been successful in delivering approved new treatments since its establishment in 2000
The CAVOD proposals build on more than 12 years of formal collaborative approaches to European orphan drug policy, which have consistently agreed on the need to gather and share information at a European level
All activities should be based on existing and planned roles, responsibilities and legislative frameworks, and in collaboration between all parties involved at Member State and EU level, including regulators, HTA bodies, payers, patients and the sponsor
Summary
The European orphan legislative framework has been successful in delivering approved new treatments since its establishment in 2000. A coordinated EU approach to informed access decisions: CAVOD process proposals – the possibility to turn concept into reality? From 6th European Conference on Rare Diseases and Orphan Products Brussels, Belgium.
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