Challenge
Currently, there is a lack of a consistent set of clinical and economic evidence that can guide decision-makers in relation to HM. This is primarily about a framework that considers both clinical and patient perspectives, the challenges facing new classes of HM drugs, and jurisdictional differences across Europe.
HARMONY will identify how decision-makers – regulatory, HTA, and payers – decide on requirements for specific HM outcome measures in their assessments. The framework will take into account factors such as efficacy/safety, comparative effectiveness, and the clinical aspects that influence cost-effectiveness, clinical transition points, patient-reported outcomes, and health-related quality of life.
Project Partnership
- BfArM, UoY, AEMPS, UULM, LeukaNET
Project Leadership
- NICE
Impact
- Develop an HM-specific clinical and economic evidence framework that addresses regulator, HTA, and payer requirements
- Include the patient’s clinical perspective on HM therapies in the framework
- Elaborate on the summary of evidence and analytical approaches that are commonly required to reduce HM-specific HTA/payer decision uncertainty post-marketing authorization
Project Summary
To achieve the project’s goals, HARMONY will undertake a review of relevant information from public agencies. This was divided into 3 phases:
- Conducting an analysis to determine how decision-makers decide on requirements for specific HM performance measures in their evaluations.
- Reviewing previous reimbursement decisions and supporting materials to understand how decision-makers use outcome measures.
- Reviewing the uncertainties identified in the decision-making phase and explore how real-world evidence and data synthesis can be used to resolve or mitigate these uncertainties. The outcome will be a summary of the evidence and analytical approaches that are commonly required to reduce uncertainty in post-authorization HTA/payer-specific HM decisions.
HARMONY aims to provide a consistent approach to coordinating regulatory / HTA and payer data across projects and streamlining applications to individual agencies. All identified collaborative partners will contribute to the tasks and outcomes.