Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products: Is the NICE Approach Fit for Purpose?

In 2016 the University of York undertook a review exercise to determine whether NICE’s existing methods and processes are appropriate for assessment of regenerative medicines. The purpose of this OHE report is to explore this review exercise and to assess whether or not the resulting conclusions are appropriate. The Report concludes that the York and NICE exercise provided a thorough mock appraisal of CAR T cell therapy. However, it did not seek to identify the most suitable approach for assessing regenerative medicines, but rather to test whether regenerative medicines could fit into the existing pathway developed for conventional medicines. The authors suggest a more interesting question would have been to look at whether or not use of the existing pathway is the most suitable approach, rather than whether or not it is possible. The report also questions the relevance of some additional parameters that were presented to the expert panel as part of the mock appraisal, arguing that the presentation of uncertainty is potentially misleading. It also suggests that NICE’s end of life criteria and criteria for allowing use of a 1.5% discount rate should be amended in the context of regenerative medicines.