New Publication: Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value

Just published is a new report entitled: Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value. The report was developed by OHE’s Grace Marsden and Adrian Towse, in collaboration with Steve Pearson and Bill Dreitlein from the Institute for Clinical and Economic Review (ICER), and Chris Henshall (Independent Consultant).

Gene therapies repair, deactivate or replace dysfunctional genes that cause disease, with the aim of (re)establishing normal function. Two gene therapies have been granted marketing authorisation in the EU to date, and gene therapies are likely to enter use in the US health care system within the next 12-24 months.

Many of these products will offer the potential of a short “one-off” treatment regimen promising lifelong benefits.

However, evaluating the effectiveness and value of gene therapies at launch will be difficult, and their high cost is likely to present severe affordability challenges for payers and society. These challenges raise the question of whether new models of pricing and payment and new or modified health technology assessment (HTA) methodologies are needed.

This new report presents an analysis of the significant clinical potential of gene therapy and the unique challenges in developing and evaluating evidence on their effectiveness and value. Special attention is given to pricing and payment mechanisms, including new approaches to payment based on long-term amortisation of initial costs.

ICER held a Policy Summit in December 2016 to discuss gene therapies and to explore the opportunities and challenges of adopting these technologies in the US health care system. Based on the discussions at the Policy Summit, this report concludes with a set of recommendations for future consideration by payers, manufacturers, and policymakers.

Download the full report here.

For more information please contact Grace Marsden at OHE.

Posted in Drug Development/R&D, Health Technology Assessment, Innovation, Pricing and Reimbursement | Tagged Research Papers