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?
Cubi-Molla, P., Mott, D., Shah, K., Herdman, M., Summers, Y. and Devlin, N.
Cancer survival rates have improved dramatically in recent decades due in part to pharmaceutical advances, with a growing range of increasingly effective and targeted medicines being developed, such as immunotherapies. In the economic modelling of such treatments, the question arises of which utilities should be assigned to patients who show a long-term, durable response.
In recent critiques of economic models in this area by the National Institute for Health and Care Excellence (NICE), the idea that long-term cancer survivors (LTCS) who have received such treatments could report quality of life (QoL) scores which are similar to, or higher than, those of equivalent general population samples has not been viewed as credible. This literature review examines whether there is evidence to support the assumption that the QoL of LTCS can be similar to that of age/sex-matched population samples.
This OHE Consulting Report reviews “Estimated costs of production and potential prices of medicines for the World Health Organization Essential Medicines List” (Hill et al., 2018) in which the authors argue for “greater transparency in drug pricing” and propose generating estimates of the cost of manufacturing essential medicines to inform negotiations on drug pricing.
The notion that the price of a medicine should be linked in some way to value it generates for patients and the health system is generally accepted. Yet, how can this be achieved, when increasingly medicines are being developed that derive patient benefit across many different indications? Indication-based pricing (IBP) has been proposed as a way to tackle this issue, permitting price to vary according to indication and – critically – according to value.
This seminar briefing examines (1) why the UK has not produced large biotech firms that develop drugs, similar to those in the United States (US), (2) why the UK biotech firms that do exist have not brought blockbuster drugs to the market and (3) what the implications are for industrial strategy after Brexit. These remarks are based primarily on research we completed for our recent book, Science, the State and the City (Owen and Hopkins, 2016), with some additions and specific observations about the potential effects of Brexit.
Zamora, B., Parkin, D., Feng, Y., Bateman, A., Herdman, M., and Devlin, N.
This paper reports new methods for analysing the distribution of EQ-5D observations. The Health State Density Index and Health State Density Curve are used to summarise the extent to which people’s self-reported health on the EQ-5D is concentrated on a few health states, or distributed more evenly over a wide range of health states. This information can be useful in understanding patients’ treatment needs, as well as providing a way of comparing the nature of data provided by different patients or collected using different patient-reported outcome measures.
Pearson, S., Dreitlein, B., Towse, A., Hampson, G. and Henshall, C.
OHE Research Paper 18/02: Real World Evidence (RWE) – This paper provides a framework and specific steps to help both manufacturers and payers meet the challenge of developing observational RWE through a transparent process that can be considered credible by all stakeholders.
Hampson, G., Towse, A., Dreitlein, B., Henshall, C. and Pearson, S.
OHE Research Paper 18/01: This paper sets out the potential opportunities and important challenges and limitations that must be addressed in considering options for using RWE to inform insurer coverage decisions. The primary purpose of developing the paper was to stimulate discussion at the 2017 ICER Policy Summit meeting. A separate paper is available that summarises the authors reflections and proposed ways forwards based on the discussions that were had at the meeting.
The report addresses the implications of NICE appraising treatments for very rare diseases using a cost-per-QALY gained decision rule of the type used by NICE in its Technology Appraisal Programme to appraise therapies for more common conditions.