Economics of Health Technology Assessment

A paradigm shift is occurring in cancer care with the introduction of tumour-agnostic therapies, for which the indication is defined by the molecular signature of the tumour rather than by its location. Several agents have already gained regulatory approval, including pembrolizumab for solid tumours with high microsatellite instability (MSI-H) or high tumour mutational burden (TMB-H), and larotrectinib and entrectinib for neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumours, and many other emerging molecules are set to enter the market over the next decade.

Using medicines in combination can deliver better outcomes for patients across different tumour types and disease stages. Yet many HTA agencies do not find that the expected additional benefits from adding a new medicine to a currently reimbursed medicine represents value for money to the health system. In markets that utilise cost-per-QALY approaches for assessing value, a clinically effective medicine might even be found to be “not cost-effective at zero price” when used as part of a regimen that increases treatment duration.

Most value assessments of vaccination programs are carried out by taking the perspective of the health system. To stimulate the debate concerning the broader value of vaccination beyond this perspective, this report quantifies the related return on investment to the UK government.

OHE presents an overview on the use of cost-effectiveness thresholds (CETs) in a number of selected countries in their decision-making process for health technology assessments. In addition to the different levels of CETs in these countries, this review examines whether an explicit or implicit CET is used, and the additional considerations (here termed ‘modifiers’) that are incorporated when funding and reimbursement decisions are made.

Although the science underlying drug development has evolved, there has been little change in how we pay for them. As more and more medicines come to market with multiple indications (or even more importantly the unrealised potentialto treat multiple indications), the way we pay for those medicines becomes critical in making sure we can benefit from them. “Indication-based pricing” (IBP) permits price to vary according to indication and has been proposed to tackle this issue.

In light of concerns that not all medicines for ultra-rare (also known as ultra-orphan) conditions are appraised under the same NICE process, a new OHE Consulting Report discusses the distinct ethical and economic challenges faced by medicines for ultra-rare conditions, with particular reference to the challenges of HTA in the UK. A failure to consistently consider all ultra-rare disease medicines under the HST process could lead to inequalities in access and health outcomes for patients with ultra-rare conditions.

Cancer survival rates have improved dramatically in recent decades due in part to pharmaceutical advances, with a growing range of increasingly effective and targeted medicines being developed, such as immunotherapies. In the economic modelling of such treatments, the question arises of which utilities should be assigned to patients who show a long-term, durable response.

In recent critiques of economic models in this area by the National Institute for Health and Care Excellence (NICE), the idea that long-term cancer survivors (LTCS) who have received such treatments could report quality of life (QoL) scores which are similar to, or higher than, those of equivalent general population samples has not been viewed as credible. This literature review examines whether there is evidence to support the assumption that the QoL of LTCS can be similar to that of age/sex-matched population samples.

This study was funded by Roche Products Ltd.