Real-world evidence to support Payer/HTA decisions about highly innovative technologies in the EU-actions for stakeholders

Karen M. Facey, Piia Rannanheimo, Laura Batchelor, Marine Borchardt, Jo de Cock

Research output: Contribution to journalArticlepeer-review


OBJECTIVES: There are divergent views on the potential of real-world data (RWD) to inform decisions made by regulators, health technology assessment (HTA) bodies, payers, clinicians, and patients. This RWE4Decisions initiative explored the particularly challenging setting of highly innovative technologies, which require Payers/HTAs to make decisions on a small evidence base with major uncertainties. The aim was to go beyond strategic intent to consider actions that each stakeholder could take to improve use of RWD in this setting.

RESULTS: Case studies of recent Payer/HTA decisions about highly innovative technologies were considered in light of recent international initiatives about RWD. This showed a lack of clarity about the Payer/HTA questions that could be answered by RWD and how the quality of real-world evidence (RWE) could be assessed. All stakeholders worked together to create a vision whereby stakeholders agree what RWD can be collected for highly innovative technologies based on principles of collaboration and transparency. For each stakeholder group, recommended actions to support the generation, analysis, and interpretation of RWD to inform decision making were developed. For HTA bodies, this includes cross border HTA/regulatory collaboration to agree RWD requirements over the technology life cycle to inform initial recommendations and reassessment, data analytics methods development for HTA, and promotion of transparency in RWE studies.

RECOMMENDATIONS: Stakeholders need to collaborate on demonstration projects to consider how RWE can be developed to inform healthcare decisions and contribute to a learning network that can develop systems to support a learning health system and improve patient outcomes through best use of RWD.

Original languageEnglish
Pages (from-to)459 - 468
Number of pages10
JournalInternational journal of technology assessment in health care
Issue number4
Early online date3 Sep 2020
Publication statusE-pub ahead of print - 3 Sep 2020


  • Decision making
  • Reimbursement mechanisms
  • Stakeholder participation
  • Real-world data
  • Real-world evidence

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