An increasingly common strategy for treating many cancers is to utilise several medicines with distinct but complementary mechanisms of action in combination or in close sequence (IQVIA, 2019; Bashraheel et al, 2020). Valuing and pricing the components of a combination therapy gives rise to several challenges for companies, payers and HTA bodies particularly when two or more of the products in combination are owned by different companies, each of which seeking a value-based price for their product (Davis, 2014; Greber et al. 2014; Person et al. 2018; Danko et al. 2019).
Tackling the climate crisis is an international priority. Healthcare has a high carbon footprint, accounting for 5% of the UK's carbon footprint (Lenzen et al., 2020) and NHS England has estimated that the manufacture, supply, and use of pharmaceuticals account for 25% of the NHS's total carbon footprint (NHS England, 2020).
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.