Ensuring patient access to cell and gene therapies: The case for an innovative payment model

The BioIndustry Association (BIA) has today launched Ensuring patient access to cell and gene therapies: The case for an innovative payment model. The report, which has been produced with the support of the BIA’s Cell and Gene Therapy Advisory Committee, explores the potential benefits of cell and gene therapies for patients, the challenges they face in the current system and some of the solutions being explored in other countries. 

Cell and gene therapies are among the most exciting areas of modern medicine and are already delivering life-changing and life-extending outcomes for patients in the UK. Over the next few years, the number of cell and gene therapies is set to grow, and more patients will soon be able to benefit from these innovations.

In order to prevent these treatments outpacing our capacity to adopt them, action is needed to develop a reimbursement process that works for industry and the NHS, balancing affordability and risk.

Innovative payment models enable fair reimbursement for higher cost single administration treatments and their adoption would further incentivise manufacturers to develop life changing therapies, while ensuring that patients are able to benefit from them now and in the future.

The report calls for:

  • HM Treasury to consider amending its accounting rules to allow multi-year payments for cell and gene therapies to secure patient access.
  • The Department for Health and Social Care to work with NHS England, industry, patient groups and other partners to develop an innovative payment model that balances affordability and risk to ensure patients are able to benefit to innovative cell and gene therapies now and in the future.
  • The Department for Health and Social Care to work with NHS England, industry, patient groups and other partners to ensure that the data infrastructure is in place to collect outcome measures to support any new payment model.
  • NICE to work with the Department for Health and Social Care to ensure that changes are made to its discount rates for health outcomes and costs to bring it in line with HM Treasury’s Green Book in the near future.

Steve Bates OBE, Chief Executive of the BioIndustry Association said:

“If the UK is to retain its leading role in the development of innovative treatments like cell and gene therapies, action is needed urgently to secure a route to patient access that effectively balances affordability with incentivising R&D investment for new therapies.”

“Despite the long-term benefits that cell and gene therapies can provide, the high upfront costs of these treatments pose significant challenges to NHS reimbursement.”

“Innovative payment models are already being used elsewhere to enable fair reimbursement for higher cost single administration treatments. Adoption of an innovative payment model that balances risk and benefits between the NHS and industry will ensure that patients are able to benefit from cell and gene therapies now and in the future.”

Sven Kili, Chair of the Cell and Gene Therapy Advisory Committee said: “The UK is recognised as a world-leader in the discovery, development and manufacture of cell and gene therapies. However, the current system of evaluation and reimbursement fails to ensure that patients can access these transformative treatments. The introduction of an innovative payment model would help to address this issue by ensuring fair reimbursement for higher cost single administration treatments, incentivising further investment in these life-changing therapies.”

Notes to Editors

  1. For more information, please contact Jack Fellows, Strategic Communications and Media Manager at the BIA, jfellows@bioindustry.org
  2. To read the report, please follow this link.
  3. The BIA would like to thank the following member companies which have financially supported this report: Astellas, Autolus, Gilead Sciences, Novartis and Sarepta Therapeutics