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?
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.
Hernandez-Villafuerte, K., Zamora, B. and Towse, A.
This OHE Research paper by Karla Hernandez-Villafuerte, (German Cancer Research Center, DKFZ), Bernarda Zamora (OHE) and Adrian Towse (OHE) sets out a research agenda proposing new approaches in three areas to improve understanding of supply side opportunity costs for the English NHS. A subsequent paper will set out the authors research findings.
Berdud, M., Garau, M., Neri, M., O'Neill, P., Sampson, C. and Towse, A.
Using a multidisciplinary methodological approach combining a theoretical economic framework with uptake/market share analyses by country and interviews, this OHE research concludes that: (i) IP incentives for R&D may have encouraged a high degree of in-class competition of DAAs close to the first entrant launch; (ii) in-class competition had a positive impact on uptake and adoption of DAAs in the top-5 European countries and; (iii) although in-class competition is a necessary condition for early adoption and fast uptake of innovative medicines, it is not sufficient as there are other factors related to the performance of the new technology, characteristics of the healthcare system and political factors which can have an effect.
The high cost of drugs for rare diseases (‘orphan drugs’) has generated considerable debate. While there is debate in the economic literature over whether a premium should be paid for ‘rarity’, these drugs are reimbursed with high prices in many countries. The question then arises as to what should be a reasonable price for an orphan drug?
Cole, A., Towse, A., Lorgelly, P., and Sullivan, R.
The current system of a single price per medicine means that, for multi-indication medicines, the relationship between price and “value” can vary substantially. In this OHE Research paper, we consider: What are the economic implications of an alternative to single-price payments for pharmaceuticals? In particular, what are the implications for: payer budgets, patient access, and the incentives for innovation?