The aim of this paper is to provide an overview of the issues that might limit comparability of PRO data and to highlight some of the evidence that exists on these issues. We note some of the implications for the development and use of PRO instruments, for their application in multi-country clinical trials, and for employing evidence from them in regulatory and reimbursement decisions. Although much progress has been made in this area, there is still scope for further research and improvement. Numerous factors can affect the comparability of PRO data across (and potentially within) countries and cultures. Failure to recognise and account for these differences could lead to incorrect conclusions about the effectiveness and cost effectiveness of new medicines and other health care interventions. We suggest areas where further research and enhanced guidelines for users of PRO instruments and data would be useful.
The WHO Technical Report: Pricing of cancer medicines and its impacts (“the Report”) was published in order to address requests made in a resolution adopted by the 2018 Seventieth World Health Assembly WHA70.12 on Cancer prevention and control in the context of an integrated approach.
Berdud, M., Chalkidou, K., Dean, E., Ferraro, J., Garrison, L., Nemzoff, C. and Towse, A.
This OHE Research Paper focuses on the role that price transparency may play in the efficient and effective procurement of medicines by Middle and Low Income Countries. Will making prices publicly available make procurement more efficient and cost-effective medicines more accessible?
The measurement of quality of life in the context of spinal muscular atrophy (SMA) is challenging. This is because the disease is experienced by children and is rare, which makes data collection difficult. This Briefing reports on a symposium that outlined some lessons that can be learnt from the SMA context that might be more widely applicable.
Hernandez-Villafuerte, K., Zamora, B., Feng, Y., Parkin, D., Devlin, N. and Towse, A.
Estimating a cost-effectiveness threshold reflecting the opportunity cost of adopting a new technology in a health system is not easy. This OHE research paper provides empirical evidence on the relationship between health outcomes and health expenditures in England. Results suggest that setting a cost-effectiveness criterion for NICE may not be capable of being synthesised using scientific methods alone, but involve political judgements.
Cole, A., Towse, A., Segal, C.S., Henshall, C. and Pearson, S.D.
OHE Research Paper 19/02: The combination of rising drug costs in the US and increasing financial stress for individual patients has triggered intense national concern. One target has come under particular scrutiny: rebates. This paper describes how alternatives to the current US rebate system offer potential risks or disadvantages as well as potential benefits. It outlines the evidence presented to date, and the key questions that still need to be answered.
Cole, A., Cubi-Molla, P., Pollard, J., Sim, D., Sullivan, R., Sussex, J. and Lorgelly, P.
OHE Research Paper 19/01: This report explores the feasibility of introducing one type of flexible payment mechanism –outcome-based payment (OBP) –for cancer medicines into the NHS in England. This model links the price the NHS pays for a medicine to the outcomes it achieves in practice for NHS patients. OBP could help to accelerate patient access to some new medicines and ensure close monitoring of real-world patient benefit. It can also promote value for money in NHS spending and support innovation. This is especially valuable against the backdrop of rising overall NHS spending on medicines.
Gene therapies, which can provide cures for diseases, are a new area of research for ICER. Some are within two to three years of becoming available publicly. This is exciting, but also offers serious and critical challenges in valuation. The focus of this seminar briefing is on those challenges.
Whether or not society values curative therapies more highly (or less highly) than the sum of the iterative improvements that might come from conventional therapy has been highlighted as an important area for research. The aim of this research was thus to explore society’s preferences across curative and non-curative therapies and large and small health gains, via a discrete choice experiment.
Debate about funding has highlighted the difficulty of persuading sceptics that the NHS is a good use of public finance. There is a widely held view – particularly in finance ministries and some sections of the media – that health systems such as the NHS are ‘black holes’, constantly demanding increased funding without concomitant returns to society.