Access Evidence Framework for innovative technologies for HMs

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.

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Project Partnership

• BfArM, UoY, AEMPS, UULM, LeukaNET 

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Project Leadership

• NICE

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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

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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. 
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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.

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