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.
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”.