FINAL PROGRAMME

Workshop co-chaired by:

Existing orphan medicines policies have public support, as evidenced by several wide-ranging surveys. However, increasing cost pressures risk to result in a rationing system to control healthcare costs in general, creating additional access barriers for novel treatments, which may in particular hamper access for orphan medicines. At this time, patients’ access to orphan medicines seems variable while they also are not available to patients within the legal time limit after approval across the EU. Where available, they reach patients more slowly and sometimes with tighter conditions of access than other medicines.

The use of health economics for orphan medicines is still evolving and only limited attention has been paid to how it fits with the societal values expressed in the OMP regulation. This session will focus on these issues with the aim of:

1. Promoting dialogue with multiple stakeholders on the role of health economics in creating a stable and sustainable access to and marketplace for orphan medicines.
2. Evaluating the current impact of applying health economics to the field of orphan medicine policy and research.
3. Discussing the basis for differentiating health technology and economic assessments of orphan medicines from the assessments of common disease medicines with specific consideration to rarity, innovation, and social values.
4. Identifying areas of further research to help identify and address shortcomings of current health economic methodology and develop criteria for an informed application of health economic evaluation to inform resource allocation decisions.
5. Involving all stakeholders to advance health policy and funding for orphan medicines.

1. Presenting the data currently available
2. Discussing the predictive model based on FDA and EMEA data
3. Agreeing on the conclusions
4. Involving all stakeholders in a constructive dialogue with health authorities and third-party payers on this basis