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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