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Evaluating the effectiveness and cost-effectiveness of regenerative medicines

Posted on 8 March 2017

A methodological study involving several CRD researchers along with colleagues at the Centre for Health Economics has recently been published as an NIHR Health Technology Assessment report.

The study,1 which investigated the application of current NICE appraisal methods to regenerative medicines, also resulted in a related publication in the journal BMC Medicine in the form of a Debate paper.2

Regenerative medicines, such as cell or gene therapies, sometimes have the potential to yield game-changing benefits to patients - possible breakthroughs often result in headline news. However, these treatments are expensive, are difficult to develop and manufacture to commercial levels, and evaluation of their efficacy, safety and cost-effectiveness is often challenging. Consequently, very few regenerative medicines (also referred to as advanced therapy medicinal products or ATMPs) are licensed in the EU.

There is increasing political interest in this area, demonstrated by recent inquiries into regenerative medicine by both the House of Commons (2016) and the House of Lords (2013) Science and Technology Committees. Recommendations from the latter report resulted in the commissioning of the NICE methods appraisal study which included a ‘mock technology appraisal’ to assess whether changes to NICE methods and processes are needed. The study concluded that although there will be a significant level of uncertainty in determining the clinical effectiveness of regenerative medicines and their long-term costs and benefits, the existing methods available to estimate the implications of this uncertainty are sufficient.

The related Debate article summarises and expands on many key aspects of the NICE project report. It provides both an overview of the history and recent developments of regenerative medicine in the EU, and a consideration of what the future may hold for this fascinating area of medicine.

1. Hettle R, Corbett M, Hinde S, Hodgson R, Jones-Diette J, Woolacott N, et al. The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal. Health Technol Assess 2017;21(7)

2. Corbett M.S, Webster A, Hawkins R, Woolacott N. Innovative regenerative medicines in the EU: a better future in evidence? BMC Medicine 2017, 15:49