Evidence generation is key
Evidence generation in real world settings will be needed to overcome uncertainty around clinical outcomes. Registries and electronic health records all need to be adapted for this.
All three speakers focussed on the clinical uncertainty in safety and efficacy associated with gene therapies. This poses a problem particularly for payers and HTA agencies for whom value assessment is difficult with high uncertainty.
Electronic health records have not been set up to capture data to inform continued assessment of efficacy, safety and value after approval. Some countries and health systems are better equipped than others to handle this evidence generation requirement.
EU level guidelines on data standards would help health systems to gather data that is standardised and can be used to make decisions about efficacy and safety at a national level.
Real world data can be leveraged for the implementation of innovative payment mechanisms through outcomes-based payments. Outcome-based payments allow payers to pay for outcomes, thereby reducing the risk of making the wrong reimbursement decision and reducing the upfront cost for payers.
Annie Hubert- Industry perspective
Regulators are adapting to manage different evidence packages for gene therapies and are motivated to approve gene therapies quickly because of the potentially transformative health gain. However this doesn't solve the problem of uncertainty as HTA agencies and payers need different evidence, on long-term value, to make decisions.
In addition, the way that evidence is collected is important for payers looking to use that data to form the basis of outcomes-based payment models.
A recognition that evidence generation requirements may be different across stakeholders is important for designing systems and processes to gather real world evidence. More on this in takeaway 5.
