OHE Publications

OHE releases a number of publications throughout the year, authored by OHE team members and/or outside experts. All are free for download as pdf files; hard copies of some publications are available upon request.

A description of the OHE publications categories.


 

Towse, A., Hernandez-Villafuerte, K. and Shaw, B.
Consulting Report
May 2018

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.

Towse, A., Cole, A., and Zamora, B.
Consulting Report
May 2018

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 OHE Consulting report, funded by AstraZeneca, provides an overview of the key literature that has contributed to this debate, and of how IBP has been implemented – in its various forms – to date in the U.S., France, Germany, Italy, Spain and the UK.

IBP increases the potential number of patients who can benefit from a medicine and thus most economists regard it as efficient. It also sends the right signals for R&D investment. Differences of opinion remain, however, as to whether IBP is in the interests of payers. IBP may lead to some prices being higher than with a uniform price, as well as some prices being lower. The value at which prices are currently set in a single-price system will impact on the consequences of a move to a multiple-price system. A number of US payers see the potential for IBP to increase price competition in some indications, and the NHSE in England has used contracting by genotype in Hep C drug procurement to increase competition and help it get lower prices.

The literature evaluated as part of this report demonstrates that most of the debate is in the realms of theory, with little evidence in practice. The barriers are numerous. These include legal or regulatory hurdles, data collection problems, as well as contractual or financial flow issues.

Owen. G and Hopkins, M.

Seminar Briefing
April 2018

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.

Research Paper
April 2018

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.

Research Paper
March 2018

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.

It presents a new conceptual framework to address three elements largely missing from these earlier efforts focused on defining “best practices” or “standards” for RWE. These are: 1) how to understand the role that contextual factors play in determining how high the evidentiary standard, or “bar” will be in each situation; 2) how to tailor key process and methodological approaches the height of that evidentiary bar; and 3) how to ensure that broader process principles that support transparency are integrated successfully throughout the course of any RWE initiative.

This paper is a companion piece to a detailed background paper on the use of RWE in coverage decisions developed for the Institute for Clinical and Economic Review (ICER) Policy Summit held in December 2017.

Hampson, G., Towse, A., Dreitlein, B., Henshall, C. and Pearson, S.

Research Paper
March 2018

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.

RWE is already utilised for multiple purposes in the US and globally. The focus of the 2017 ICER Policy Summit was on RWE for HTA assessments and payer coverage decisions - both initial decisions and reassessments – and the majority of the paper focuses on this.

Acceptance of an expanded future role for RWE is not universal, particularly if it is seen as reducing the amount of RCT evidence available. Among the challenges associated with RWE explored in this paper are: bias and confounding; incomplete data; data mining; access to data; and the lack of universally accepted methodological standards.

Current uses of RWE can be strengthened by increasing the quality and credibility of RWE (via national registries, data repositories and strict protocols) and establishment of appropriate governance arrangements.

Key opportunities for the future include use of innovative study designs that combine benefits of collecting data from real world settings while incorporating best practice methods (i.e. randomisation methods from traditional RCTs); real time monitoring of patients; and the development of adaptive regulatory pathways linked to coverage with evidence development.

Towse, A. and Garau, M.

Consulting Report
March 2018

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.

Given the importance of non-QALY elements in the assessment of HSTs, such as treatment impact on the process of care and on the patients’ or their carers’ ability to go to school or to work respectively, and issues in measuring quality of life when the population affected are infants or young children, it is inappropriate to focus the appraisal of treatments for very rare diseases solely on a cost-per-QALY measure. Given the lack of empirical basis, the new £100,000 cost per QALY threshold and its further possible uplift up by a factor of three seem arbitrary.

Cole, A., O'Neill, P., Sampson, C., and Lorgelly, P.

Consulting Report
March 2018

Surgical practice has and continues to develop at a tremendous pace, reflecting the evolving technological landscape as well as the expanding skillset of the surgical workforce. Minimal access surgery (MAS) can offer improved recovery prospects for patients, but uptake in the UK is variable across both procedures and hospitals.

Through in-depth interviews with key stakeholders (surgeons from both the NHS and private sector, clinical directors and finance directors), supported by an evaluation of the literature, we assess the benefits of minimal access surgery, the extent to which these benefits are realised in practice, and the major barriers to wider adoption.

Case, A., and Deaton, A.

Monograph
March 2018

Low levels of mortality are important indicators of societal success. This lecture is about trends in mortality in the white non-Hispanic population in the United States of America (US), a subject which is not only interesting in itself, but also of global significance because we are all wondering whether this could happen to our own societies or to specific groups within them. The lecture was delivered by Professor Case, Sir Angus added some further reflections and then both professors engaged in a question and answer session at the end. The work discussed here, which is part of a much larger research agenda, leads to comparisons between the US and what might be happening in Europe.The authors’ most recent work on the topic of mortality rates is summarised in three papers (Case and Deaton 2015, 2017a, 2017b; there are links to these papers at https://scholar.princeton.edu/accase/publications).

This version of the text is based on a transcript of the lecture and, as such, is often less formal and more colloquial than might be expected in an academic paper.

Mestre-Ferrandiz, J., Berdud, M., and Towse, A.

Consulting Report
January 2018

The CRA Report has an underlying assumption that the EU is as globally competitive in generics and biosimilars as it is in innovative products. There is no evidence to support this. The correct industrial strategy for the EU may well be to focus on the development, manufacture and export of innovative products, rather than on lower value generics where EU global competitiveness appears to be weaker.

The CRA report makes estimates of effect using a number of assumptions, data and calculations that we do not find to be correct or which are not explained. Until these anomalies are addressed, our view is that the CRA analysis is not a fit basis for an impact assessment to guide policy.

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