News - March 7, 2019

Pricing & Reimbursement processes of Orphan Medicinal Products diverge from the ORPH-VAL principles

A report, commissioned by EUCOPE points out the uneven alignment of Pricing & Reimbursement (P&R) systems in five European countries with the 2015 ORPH-VAL Principles.

Back in 2015, in an effort to improve consistency in P&R processes for Orphan Medicinal Products (OMPs) and avoid delays in treating patients, the ORPH-VAL Working Group, which comprised multiple stakeholders from patient representatives to academics, politicians, regulators, payers and industry including EUCOPE, developed recommendations on patient improved access to orphan medicines. In particular, the nine principles relate to OMP assessment decision criteria, OMP P&R decision processes, sustainable funding of OMPs and increased European collaboration.

Following the publication of the principles, EUCOPE recently commissioned a report which providing an assessment of the extent to which the big five EU Member States are aligned with, or diverging from, these principles, and point at differences among the national healthcare systems with regards to patient access to OMPs.

From the results of the assessment of individual country, the report identified potential areas of focus at the EU level to improve the alignment of rare diseases P&R processes, inter alia:

  • Societal values underpinning value assessment should be explicit;
  • Patients and experts should be consistently involved;
  • Collection/analysis of real-world data (registries & databases) should be coordinated at a EU or international level;
  • Uncertainty as a key factor relating to OMPs should be a high priority for consideration.

The report conclusions aim to stimulate further discussions and recommendations on possible next steps. Acknowledging the national authorities’ efforts to improve access pathways for orphan drugs,  the report can shed light on the areas which should prioritised in order to further refine pricing & reimbursement processes and establish effective systems at both national and international levels.

Should you have any question on the topic, please contact Delphine Roulland (