Table 1 Overview of principles
From: Principles for consistent value assessment and sustainable funding of orphan drugs in Europe
Sections | Principles |
|---|---|
Value Assessment | 1: National pricing and reimbursement processes should acknowledge the EMA’s assessment of therapeutic benefit |
2: National Authorities should incorporate rare disease expertise within their local assessment processes | |
3: OMP assessment should consider all relevant elements of value | |
4: Value assessment methods for OMPs should incorporate multiple criteria | |
5: Value mechanisms should be flexible to accommodate evidential uncertainty at time of OMP approval | |
Innovation and Price | 6: Adequate funding should be provided to ensure optimal patient access to OMPs and to incentivise research |
7: OMP reimbursement decisions should be determined by benchmarking value and price against treatments with similar characteristics | |
8: If used, ICER thresholds should be modulated to reflect the specificities of rare diseases and OMPs | |
Sustainability of OMP Model | 9: National authorities should develop adaptive and efficient processes to optimise use of real world data collected before and after value assessment |
10: Rational and evidence-based funding mechanisms should be developed to guarantee long-term sustainability |