Gene therapies represent a new era of medicine, offering the potential for truly transformational health gains, and further benefits for society and health systems. Gene therapy is particularly relevant to rare disease patients, as more than 80 per cent of rare diseases have a known monogenic (single gene) cause. In contrast to traditional small molecule medicines, gene therapies have the potential to correct underlying genetic defects, offering the potential for transformational health gains rather than simply managing symptoms.
In recent years, U.S. policymakers have been considering reforms to tackle high and rising prescription drug spending, including unprecedented direct limits on prices and price growth for top-selling medicines. By affecting expected prices and revenues, this type of reform will impact biopharmaceutical companies’ incentives to innovate. However, the magnitudes and timings of impacts on the numbers of new drugs coming to market are unclear.
Generic preference-based measures are often used for capturing patients’ health-related quality of life (QOL) to assess quality-adjusted life years (QALYs) in health technology assessment (HTA). Whilst this type of measure, which includes commonly used EQ-5D instruments, are useful for enabling comparability between assessments, they might not always capture all the dimensions of QOL that are important to patients.
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.
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.
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.
Rodes Sanchez, M., Rachev, B., Spencer, J., Sharma, I., Tantri, A., Towse, A., Mitrovich, R. and Steuten, L.
By placing a strain on health care systems and the global economy, the COVID-19 pandemic clearly shows the need to more comprehensively understand both supply- and demand-side aspects of a “healthy” vaccines market that can meet public health demand over time and across dynamic events. The goal of a healthy vaccines market, as defined for this study, is to support sustainable innovation and equitable access to address public health needs.
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”.