Just published is a new OHE Consulting Report entitled: Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products: Is the NICE Approach Fit for Purpose? The purpose of the new OHE report is to explore a review exercise undertaken by NICE and the University of York and to assess whether or not the resulting conclusions are appropriate.

Just published is a new OHE Consulting Report entitled: Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products: Is the NICE Approach Fit for Purpose?

Regenerative medicine is an umbrella term which covers a range of medical treatments including tissue engineering, cell therapies and gene therapies. There is considerable excitement around the development of these medicines, with the expectation that that they may bring substantial clinical gains and offer cures for previous debilitating and fatal diseases.

However, securing reimbursement is a significant hurdle for manufacturers for two main reasons:

• data is often insufficient for calculating robust estimates of clinical and cost-effectiveness;
• list prices are generally high.

In March 2016, the Centre for Reviews and Dissemination and the Centre for Health Economics at the University of York, published the results of a review exercise which was undertaken to determine whether NICE ’s existing methods and processes are appropriate for assessment of regenerative medicines. A summary of the results and NICE’s conclusions were also published separately.

The purpose of the new OHE report is to explore this review exercise and to assess whether or not the resulting conclusions are appropriate.

In our view 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. We 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.

Further, we question 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 misleading. We also suggest 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.

Finally, any significant departures from the usual HTA process must be based on solid economic rationale if we are to ensure efficient allocation of NHS resources. Further research will help shed light on whether or not there exist solid economic grounds for valuing regenerative medicines differently to conventional medicines. 

Access the full report here.

This work was commissioned by Pfizer.

For more information contact Grace Marsden at OHE.