Appraisal methods for regenerative medicines

NICE have undertaken an exercise to assess whether changes to its methods and processes will be needed for regenerative medicines. Part of this work was to undertake a mock technology appraisal of CAR T-cell therapy for treating acute lymphoblastic leukaemia. Professor Allan Wailoo from HEDS was a member of the mock appraisal committee used in the project. 

NICE’s report of the exercise is available here, but a summary of their summary is:

“The overall findings of the exercise were that:

• The NICE appraisal methods and decision framework are applicable to regenerative medicines and cell therapies.
• Quantifying and presenting clinical outcome and decision uncertainty was key to the Expert Panel consideration of the hypothetical example products.
• Where there is a combination of great uncertainty but potentially very substantial patient benefits, innovative payment methodologies need to be developed to manage and share risk to facilitate timely patient access while the evidence is immature.”