Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies
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Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies Aris Angelis1 · Huseyin Naci1 · Allan Hackshaw2
© Springer Nature Switzerland AG 2020
Abstract Recently licensed cell and gene therapies have promising but highly uncertain clinical benefits. They are entering the market at very high prices, with the latest entrants costing hundreds of thousands of dollars. The significant long-term uncertainty posed by these therapies has already complicated the use of conventional economic evaluation approaches such as costeffectiveness and cost-utility analyses, which are widely used for assessing the value of new health interventions. Cell and gene therapies also risk jeopardising healthcare systems’ financial sustainability. As a result, there is a need to recalibrate the current health technology assessment methods used to measure and compensate their value. In this paper, we outline a set of technical adaptations and methodological refinements to address key challenges in the appraisal of cell and gene therapies’ value, including the assessment of efficiency and affordability. We also discuss the potential role of alternative financing mechanisms. Ultimately, uncertainties associated with cell and gene therapies can only be meaningfully addressed by improving the evidence base supporting their approval and adoption in healthcare systems. Key Points for Decision Makers There are significant uncertainties associated with both the clinical benefits and economic costs of cell and gene therapies that complicate their appraisal using standard health technology assessment methods. We outline a set of methodological refinements to improve the assessment of efficiency (costs, benefits and cost effectiveness) and affordability (budget impact) of cell and gene therapies, together with options for their financing. Recommendations range from relatively small adjustments such as adopting different time horizons and costeffectiveness thresholds, to developing more advanced statistical techniques for survival modelling and applying multi-criteria decision analysis.
* Aris Angelis [email protected] 1
Department of Health Policy, London School of Economics and Political Science, Cowdray House, Portugal Street, London, UK
Cancer Research UK and UCL Cancer Trials Centre, UCL Cancer Institute, University College London, London, UK
2
1 Introduction Cell therapies, such as cellular immunotherapies and gene therapies, long discussed since the beginning of the century, have recently become a reality with a number of products entering the market over the last few years. Despite their breakthrough nature, which is accompanied by escalating patient expectations and hype, their extremely high prices risk overwhelming the already-stretched financial resources of healthcare systems. A debate is emerging about how these new therapies should be assessed and compensated by payers. Central to the pricing discussion is the value that these therapies offer, mainly to patients and possibly pa
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