Takeaway 4

There are practical and legislative barriers to progress

Quick summary

Science has delivered transformative therapies, but there are practical barriers to delivering them to patients. Some of the barriers discussed by speakers were:

Lack of strong data systems for monitoring outcomes
Accounting standards that make annuity payments difficult
HTA processes that are not fit to assess the full value of gene therapies

Data systems aren't readyThere was significant discussion during the debate about the limitations in data systems to collect data to address the uncertainty surrounding the long-term efficacy and safety of gene therapies.Electronic health records are not designed to capture outcomes that can be used to make reimbursement decisions. Registries, that are set up to capture data on individual diseases or disease areas, are not flexible for new disease areas.Speakers agreed that real world evidence generation was a necessity for the adoption of gene therapies and that there could be a role for cross-border alignment on standards and data platforms to overcome this barrier.Accounting standards could limit annuity paymentsEuropean system of accounts, for both companies and for governments, may restrict the use of annuity payments which could be used to spread the upfront cost of gene therapies.

What is an annuity payment?

The overall cost is paid in instalments, spreading the cost of a product or service over multiple budget cycles. Annuity payments can be conditional on a patient meeting certain predefined clinical outcomes. These are called outcomes-based payments.

There are other barriers, such as accounting systems in Europe which really are a barrier for the adoption of spread payments over time.

Annie Hubert- Industry perspective

Health Technology Assessment may need to be adapted for gene therapiesHealth Technology Assessment (HTA) processes and other processes such as budget impact evaluation may not be fit for purpose for gene therapies. Speakers agreed that HTA should take account of societal benefits of gene therapies. This would require HTA processes to be adapted to incorporate other forms of value in addition to the health gains and costs that are traditionally considered. For example, for childhood diseases which require parents to become full-time carers, a treatment that removes that need has a much greater value than just the health gain to the child, there are also benefits for the parents and to wider society as a result.

How can HTA be adapted?

If you are interested in learning more about adapting HTA to incorporate other elements of value, take a look at these OHE publications.

I think at the moment we are in an incredible window of opportunity to translate the scientific advances, but we need to accompany them with innovation in processes not only in the way we pay but also the way we assess the value.