The report concludes that NICE’s current appraisal methods and decision framework are applicable to regenerative medicines and cell therapies.

Regenerative medicine is a relatively new field of medicine and involves replacing or regenerating human cells, tissues or organs in order to restore normal function. This includes cell therapies, tissue engineering, gene therapy and biomedical engineering techniques, as well as more traditional treatments involving pharmaceuticals, biologics and devices.

To test the application of the NICE appraisal methodology to regenerative medicines and cell therapies, a special NICE study and Expert Panel was set up to consider CAR (chimeric antigen receptor) T-cell therapy in relapsed or refractory B-cell acute lymphoblastic leukaemia in children and young adults. CAR T-cell therapy is a real but as yet unlicensed therapy at the early stages of development with sparse data and an unknown price. The exercise was undertaken in response to a recommendation by the Department of Health Regenerative Medicine Expert Group (RMEG)ⁱ

Commenting on the exercise, Professor Andrew Stevens, NICE Technology Appraisals Committee Chair, said: “The relatively new field of regenerative medicine is potentially important to both the UK economy and to patients. It is also thought to present special difficulties for NICE’s appraisal process because, although regenerative medicines and cell therapies have the potential to confer substantial health gains, they can be expensive and there is unlikely to be much evidence to support their use at the time of initial marketing authorisation.”

“Our exercise was able to demonstrate the versatility and adaptability of NICE’s approach to appraising health technologies to enable appropriate consideration of these innovative treatments.”

The exercise demonstrated that quantifying and presenting uncertainty over the long- term balance of benefits and possible harms was key to the consideration of the hypothetical example products. The report therefore recommends that NICE continues to further develop the ways in which uncertainty can be quantified and presented to its decision-making committees.

Where there is a combination of great uncertainty with potentially very substantial patient benefits, innovative payment methods may have a key role to play in managing financial risk to facilitate timely patient access. The report advises NICE to continue to collaborate with other stakeholders to develop practical methods of how innovative payment methods could be implemented to benefit patients in the NHS.

Dr Nick Crabb, Programme Director – Scientific Affairs at NICE commented: “Products with these, albeit hypothetical, characteristics would represent major advances in therapy, offering profound benefits to eligible patients compared with the current NHS standard of care. An additional 10 quality adjusted life years (QALYs), as in the example used in our exercise, represents a 10 year life extension at full health. Step-change improvements in patient outcomes from cancer treatments of this magnitude are rarely seen with conventional therapies. But the introduction of these products may also present financial challenges. Methods will need to be developed that allow healthcare systems to find ways of making them available to patients in a financially sustainable way.”

Keith Thompson, CEO of the Cell and Gene Therapy Catapult said: “This report is progress in delivering a better environment for the adoption of Advanced Therapies in the UK and we are pleased to have supported it with our expertise. It’s part of a series of initiatives that are helping to ensure we have the right frameworks in place for advanced medicines, such as cell and gene therapies, that are showing such promise in clinical trials but haven’t yet showed that the benefits can last over long periods of time.”

Ends

For more information call the NICE press office on 0845 003 7782 or out of hours on 07775 583 813.

Notes to Editors

References

            ^i.            Following the House of Lords Regenerative Medicine Inquiry, the Department of Health established the Regenerative Medicine Expert Group (RMEG). Throughout 2014, RMEG worked on an NHS regenerative medicine readiness strategy and assessed the effect of regulation on the development of regenerative medicines in the UK. NICE was represented on RMEG and also on a sub-group on Evaluation and Commissioning. This subgroup recommended that an exploratory study of the appraisal of example regenerative medicine products be commissioned and published by NICE to highlight key issues in the evaluation of regenerative medicines and explore the suitability (or otherwise) of current methods. The subgroup recommended that the exercise should be designed to add to the learning emerging from actual appraisals of regenerative medicines. A study outline was subsequently developed and agreed with subgroup members and the RMEG.  The Centre for Reviews and Dissemination/Centre for Health Economics, University of York was assigned to this project through a commission by the NIHR HTA Programme.

About the NICE study

1. The NICE study report: Exploring the assessment and appraisal of regenerative medicines and cell therapy products is available on the NICE website.
2. The study objectives were:

• To test the application of NICE appraisal methodology to regenerative medicines and cell therapies, identifying challenges and any areas where methods research and/or adaptation of methodology is appropriate
• To identify specific issues related to the appraisal of regenerative medicines and cell therapies using the current NICE appraisal process and decision framework
• To develop a framework for those developing regenerative medicines and cell therapies to facilitate understanding of how NICE evaluates clinical and cost effectiveness and to identify the most important evidence areas to develop before cost-effectiveness can be reasonably estimated.

1. The study protocol was published on the NICE website (https://www.nice.org.uk/about/what-we-do/science-policy-research/nice-research).
2. The study concerned a hypothetical example product with characteristics based on early clinical data for related real products supplemented with hypothetical evidence (CAR (chimeric antigen receptor) T-cell therapy specific to antigen CD19, for treating relapsed or refractory B-cell acute lymphoblastic leukaemia (B-ALL) in children and young adults).