Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

Abstract

I.

Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy

“…Most reference cases for economic evaluation recommend or require that the same discount rate should be applied to both costs and benefits. Jönsson et al 9 question this in the case of advanced therapy medicinal products, where often there is a large upfront cost if the therapy is delivered in a single dose, with benefits stretching far into the future. They also point out that there is a minority of national HTA bodies (eg, in Belgium, the Netherlands, and Poland) that allow benefits to be discounted at a lower rate than costs.…”

“…Marsden et al 8 question this argument and suggest that patients with rare genetic diseases, along with the gene replacement therapies they use, present a unique set of conditions that warrant equally unique analytic approaches to estimating value for money. Furthermore, Jönsson et al 9 suggest that in the case of advanced therapy medicinal products, which include gene therapy approaches, 3 issues pose challenges for current methods: the uncertainty about future costs and benefits, aspects of value that are not captured in the quality-adjusted life-year (QALY), and the social and ethical aspects of the implications of discounting. Finally, Garrison et al 10 argue that the novel aspects of value provided by gene therapies justify a higher (cost-effectiveness) decision-making threshold.…”

Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment

“…The frameworks used by EU HTA bodies are typically based on comparative clinical benefit assessment and economic evaluation (cost utility analysis or cost-effectiveness analysis) as the key methods for defining the value of new technologies. This may involve reflecting health gain as quality-adjusted life years (QALY), or an alternative patient-relevant outcome, such as life years gained (LYG) [2].…”

“…Advanced therapy medicinal products (ATMPs), are a heterogeneous class of medicinal products that includes products based on genes (gene therapy medical products), cells (somatic cell therapy medical products) and tissues (tissue engineering medical products) [1]. ATMPs may offer the potential of a one-time cure and are considered disruptive technologies [2].…”

Keeping Pace with Pharmaceutical Innovation: The Importance of the NICE Methods Review

“…Advances in science mean we have new medicines for previously untreatable diseases, often very specific to a small sub-set of patients. Medicines are being developed to treat patients earlier on in the disease pathway, with novel mechanisms of action that in some cases are potentially curative [4][5][6][7]. There are significant developments in areas like gene-editing technology, stem cell treatments, exosomes and chimeric antigen receptor T-cell therapies [8].…”