Abstract
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. 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. 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.
Highlights
Innovative technologies aim to deliver transformative patient benefit
A mixed-methods approach was used that explored recent policy proposals about use of real-world data (RWD) applied to the specific context of Payer/health technology assessment (HTA) decisions about highly innovative technologies and developed feasible actions that were implementable by each stakeholder group
Some have excellent electronic health records that can be linked via a unique patient identifier, but analyses can be cumbersome and key information such as diagnosis may not be recorded on prescriptions
Summary
Innovative technologies (such as immuno-, cell, and gene therapies that use novel molecular biology to target the underlying cause of a disease) aim to deliver transformative patient benefit. Regulatory approval based on interim analyses of short-term end points and early data cut-offs is common, as is the use of uncontrolled trials This means that some health technologies enter the market with a limited evidence base to demonstrate clinical effectiveness. Uncertainties exist about the population to be treated, natural history of disease, size, and durability of clinical effects compared to treatment alternatives, safety, cost effectiveness, and budget impact. As, these highly innovative technologies often have a high price due to the complexity of development and purported high patient benefit, decisions about their value are challenging
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