Brogaard, N., Abdul-Ghani, R., Bayle, A., Henderson, N., Bréant, A, and Steuten, L.
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.
In recent years, US policymakers have been considering reforms to reduce drug spending, including allowing the government to directly limit prices and price growth for branded medicines. By reducing prices, such policies will negatively impact incentives for innovation, but the magnitudes and timings of these impacts are unclear.
Cole, A., Cubi-Molla, P., Elliott, R., Feast, A., Hocking, L., Lorgelly, P., Payne, K., Peek, N., Sim, D., Sussex, J., Zhang, K and Steuten, L.
Outcome based payment (OBP) is a flexible payment mechanism that links the price the NHS pays for a medicine to the outcomes it achieves in practice for NHS patients. In 2019 we published the results of a research study which described that OBP could help to accelerate patient access to some new medicines, ensure close monitoring of real-world patient benefit, promote value for money in NHS spending and support innovation.
For the growing number of multi-indication medicines, access may be delayed or even denied due to challenges in linking payment with a medicine’s value across those indications. We assembled a broad range of stakeholders to work toward consensus on the challenges and solutions which promote better patient access and sustainable health care and innovation.
Up to 5,000 people in the UK require a kidney transplant due to renal failure each year, but only about 3,000 transplants can be performed each year and around 250 people die each year while waiting for a transplant. To close this gap, it is important to find ways to make better use of the organs currently available. This includes improving post-transplant care to increase the likelihood of a successful transplant and extending the duration of graft survival, with the ultimate goal of “one transplant for life”.
Brassel S., Neri M., Schirrmacher H., and Steuten L.
The NHS is facing unprecedented health system pressure, with a record number of patients waiting for care, while their underlying condition is potentially worsening due to exceptionally long waits.
When there is excess demand for health care, treating one patient means losing the opportunity to treat another. These opportunity costs demonstrate the need to consider health system capacity value, one of the so-called "broader value elements" that is often not fully captured in traditional value assessments.
Henderson, N., Firth, I., Errea, M., Skedgel, C. and Jofre-Bonet, M.
Medicines for rare and ultra-rare conditions face distinct economic and ethical challenges compared to medicines for more common conditions. These challenges are implicitly acknowledged by the National Institute of Health and Care Excellence (NICE) by providing an auxiliary appraisal route for highly specialized technologies. However, concerns have been raised regarding the appraisals of medicines that don’t meet the strict criteria and are, therefore, evaluated via the standard appraisal route (STA).
Hitch, J., Firth, I., Hampson, G., Jofre-Bonet, M., Garau, M., Garrison, L. and Cookson, G.
The US spends significantly more on healthcare per person than other wealthy countries. H.R. 3 is a recent policy proposal aimed at reducing national spending on prescription drugs, one component of overall healthcare spending. Drug manufacturers would be required to negotiate prices with the US government, and prices would be capped at a level based on the prices of the drug in a set of foreign countries. Evaluation of a policy with such large potential impact on pharmaceutical revenues must consider potential negative effects on incentives to innovate.