Evaluating the full costs and effects of high-price, disease-modifying therapies

When a new therapy is developed, healthcare decision‑makers must compare the benefits of funding new therapies with other potential uses of funding. The timely reimbursement of new, cost‑effective therapies by health systems is essential to ensuring patients can access the medicines they need without undue delay.

As the pipeline of new drug candidates is shifting increasingly towards high‑price, disease‑modifying therapies that promise curative or partially curative effects, it is important to assess whether existing health economic methods are fit for purpose to evaluate their costs and effects. Currently, little research has been done on whether and how the economic value of these therapies is affected by their unique cost and benefit profile: namely, that they often come with a high sticker price (£1 million plus) but promise significant cost savings in other parts of the health system, as well as transformative, lifelong improvements in the health of treated patients.

This project aims to analyse the extent to which existing methods for evaluating these therapies in health technology assessment processes capture their full costs and benefits. Where existing methods are lacking, it aims to develop new approaches to economic evaluation and ultimately inform the methods applied by academic health economists and health technology assessment bodies such as the National Institute for Health and Care Excellence (NICE). These improved methods could avert unnecessary rejections of medicines — in particular, high‑price disease‑modifying gene therapies — when they are submitted for reimbursement on the NHS, and lead to improved resource allocation across health systems in the UK and globally.

Funder: Medical Research Council, UKRI 

See project website for more.