29 March 2016 - NICE has today published a report describing a study exploring the assessment and appraisal of regenerative medicines and cell therapies. Part of the study considered a product based on new, as yet unlicensed, cell therapies for treating a type of leukaemia in children and young adults. The exercise, undertaken in collaboration with the University of York and with support from the Cell and Gene Therapy Catapult, was designed to see whether NICE’s health technology appraisal methods are suitable for regenerative medicines and cell therapies.

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).

For more details, go to: https://www.nice.org.uk/News/Press-and-Media/nice-publishes-report-on-approaches-to-assessing-innovative-regenerative-medicines