OHE’s Towse on Challenges in Valuing Genomic Medicines

Posted on 23 May 2013 by OHE News Editor

In this video, Adrian Towse discusses the challenges and importance of appropriately valuing genomic medicine. He addresses both gene therapies and “pharmacogenomics” — those medicines that will allow targeted, precision treatment. He points out the importance, with respect to the latter, of the importance of appropriately valuing the diagnostics that are essential to realising the full value of treatment.

To view other videos from the Translation Genomics Event presented by the NHS’s National Genetics Education and Development Centre click here.

Adrian Towse from Office of Health Economics

Related OHE publications and materials include the following.
Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2013) Can and should value-based pricing be applied to molecular diagnostics? Personalized Medicine. 10(1), 61-72. An earlier version of this article is available as a Research Paper on the OHE website.

Garau, M. (2013) Assessing the value of co-dependent technologies: How can current methods and processes be improved? Presentation (available here). ScHARR Seminar. Sheffield. 16 April.

For additional information, contact Adrian Towse.

Contracting for Quality in the NHS Is Topic of OHE’s 20th Annual Lecture

Posted on 20 May 2013 by OHE News Editor

Last year’s annual lecture

On the evening of 16 July 2013, OHE will welcome Professor Alan Maynard of the University of York, who will speak on Contracting for Quality in the NHS: Putting the Francis Report in Perspective. His summary of the focus of the lecture follows.

The report of the Independent Inquiry into Care provided by the Mid-Staffordshire NHS Foundation Trust offers a depressing insight into the quality of care in parts of the NHS. Robert Francis QC recommended a number of unevidenced interventions, which focus on increased regulation.

What insight can we gain into NHS quality from an economic perspective? Since the 1991 NHS reforms, there has been potential for an active contracting process, but this opportunity has largely been ignored. Commissioners of health care in the NHS, like those in the private sector, have tended to be passive, accepting the quality of care provided in primary care and hospitals with little challenge.

Incentives for quality health care provision in today’s NHS are muted. Traditionally, NHS managers have been rewarded for controlling expenditure rather than ensuring quality. This focus on cost has led at times to clashes between managers and clinical professionals with managers taking the blame when NHS care has been found inadequate. Policy efforts to improve quality have tended to involve considerable expenditure increases. ‘Quality’ care of course has to take into account both costs and outcomes; in current austere financial times, the chances of increased expenditure are slim.

In this lecture, the roles of incentives and regulation in NHS contracts will be explored. In a context of severely constrained budgets, how can we design and enforce contracts that ensure good quality NHS care?

Professor Alan Maynard began his career at the University of Exeter (1968–71) as an assistant lecturer and lecturer in Economics. Between 1971 and 1977, he lectured in Economics in the Department of Economics and Related Studies, University of York, where he later was Founding Director of the Graduate Course in Health Economics and Founding Director of the Centre for Health Economics. Prof Maynard currently is Professor of Health Economics in the Department of Health Sciences at the University of York.

Prof Maynard has been involved in NHS management since 1983, initially as a non-executive director and subsequently as Chair of the York Hospitals NHS Foundation Trust (1997-2010). Currently, he is Lay Chair of the Vale of York Clinical Commissioning Group.

Prof Maynard’s work extends well beyond the UK: he has worked as a consultant for the World Bank, the World Health Organisation, the European Union and the UK Department for International Development in two dozen countries.

A Fellow of the Academy of Medical Sciences, Prof Maynard also has honorary doctorates from the Universities of Aberdeen and Northumbria. He was awarded the OBE for services to the NHS in 2009.

To register, please click here. Please note that the deadline for registration is Thursday, 11 July.

Exploring the Link Between Health and Wealth in Government Decision Making

Posted on 16 May 2013 by OHE News Editor

Traditionally, decision makers both within and outside the health sector have thought of the value of health interventions primarily in terms of reduced morbidity or mortality. Health care, however, produces wealth effects outside the health care sector—for example, improvement in labour productivity for both patients and caregivers, cost savings in health and social care and other sectors, and an increase in national income. These wealth effects are considered infrequently, at best, in government decisions outside health agencies.

The extent to which these wealth effects have had an impact on the advice given by health technology assessment (HTA) bodies or on budget setting in other government departments has not been explored fully. With that in mind, this research was intended to examine whether the wealth effects of one type of health care—prescription medicine interventions—are considered in resource allocations and, if not, why not.

To gather information, we targeted decision makers in three types of government agencies—finance, health, and HTA—in eight countries: Australia, France, Germany, Italy, Poland, South Korea, Sweden and the UK. We found that only in Sweden does decision making take into account that the investment in health care, including medicines interventions, can improve economic output. Barriers to a broader perspective included system fragmentation that precludes taking advantage of spillovers across sectors, methodological issues in HTA, and a short-term focus by governments exacerbated by the effects of the economic recession. The paper concludes with a discussion how to address the barriers identified in order to ensure efficient priority setting within health and across public sectors.

Download: Garau, M., Shah, K., Sharma, P. and Towse, A. (2013) Is the link between health and wealth considered in decision makikng? Results from a qualitative study. Research Paper 04/13. London: Office of Health Economics.

OHE’s Adrian Towse is ISPOR President-Elect for 2013-14

Posted on 13 May 2013 by OHE News Editor

Prof Adrian Towse OHE’s Director, Professor Adrian Towse, has been elected President of the International Society for Outcomes Research (ISPOR) for the 2014–15 term and will serve on the ISPOR Board of Directors as President-elect during 2013-14. Adrian earlier has served on the Board and on the Management Advisory Board of ISPOR’s journal, Value in Health. He has been active in the scientific life of ISPOR as well, presenting at plenary sessions in the US and Europe and participating in many issue panels and workshops.

A member of the Task Force on Prospective Observational Studies which published its report earlier this year, Adrian currently is co-chair of the Task Force on Performance Based Risk Sharing Agreements (PBRSAs), which is due to report later this month. On the latter topic, Adrian and colleagues Lou Garrison and Josh Carlson of the University of Washington, Seattle, have conducted educational workshops at the ISPOR annual meetings for the past five years.

Adrian set out his vision for ISPOR as follows.

Three challenges face ISPOR: (1) continuing to globalise, (2) responding to growing payers’ demands for evidence while addressing their healthy scepticism about the value of the evidence they receive, and (3) continuing to raise scientific standards for health economics and outcomes research (HEOR), thus supporting the next generation of researchers.

These challenges are intertwined.

Globalisation, which involves understanding the realities of decision making in health systems, particularly in emerging economies, must not compromise on the standards of the science.

Decision maker scepticism about HEOR evidence can only be tackled by maintaining high scientific standards and by better understanding how and why decisions are made.

Improving the science involves innovation in methods and making clear their practical value in delivering important evidence for decision makers. This, in turn, takes us back to understanding new groups of decision makers worldwide and the changing needs of established payers.

ISPOR has made great strides on all three of these challenges since I served on the Board a decade ago. At that time, the European meeting was a gamble; we were renegotiating our first Value in Health contract with Blackwell publishing and ensuring listing on PubMed; and we were just beginning to create groups of thought leaders around scientific methods, which evolved into the current successful Task Forces and Special Interest Groups. Links with the FDA, EMA, and payers were tenuous.

Successive Presidents, Boards and the full time staff, led by Marilyn Dix Smith, deserve the credit for all that has happened since then, along with the members whose participation in meetings and scientific work ultimately have ensured the success of the organisation. It is an honour for me to have the opportunity to join the Board again, as President-elect and then to serve as President. I will use this opportunity to help ISPOR realise its 2020 Vision to be recognized globally as a leader in raising the quality of health economics and outcomes research and increasing their use in health care decisions.

Valuing Orphan Medicines Using Multi-Criteria Decision Analysis

Posted on 7 May 2013 by OHE News Editor

OHE has just released a research paper that describes an experimental study intended to test whether and how a multi-criteria decision analysis (MCDA) approach[1] can be applied to orphan medicinal products (OMPs). To be successful, the project had to both establish an explicit framework for defining the value of OMPs and provide an explicit understanding of the trade-offs that must be made in coverage decisions.

In all health care systems, HTA and reimbursement decisions begin with an assessment of value. Many systems take a distinct approach where orphan medicines are concerned. Policy makers and payers continue in seek improvements in approaches to determining the value of medicines. The move towards “value based pricing” in the UK is just one example. As yet, however, no HTA agency explicitly uses MCDA. This method applies more than one criterion to establish value and explicitly identifies the impact and relative importance of each criterion on a decision. Trades offs across attributes is characteristic of this approach; more of one attribute can compensate for less of another.

The purpose of this research was to identify which attributes to include in an OMP value framework, determine their relative importance using an MCDA process, and test whether an MCDA approach can provide a practical aid to decision making.

The project included both literature reviews and workshops. Literature searches were completed for the natural history and burden of 40 rare diseases and for how payers assess treatment value. The three workshops included, respectively (1) GlaxoSmithKline managers working on OMPs, (2) EU clinical and health economics experts, and (3) rare disease patients’ groups based in the EU. Participants refined the attributes, weighted them, scored two case study OMPs on those attributes, and tested the sensitivity of the overall ratings to changes in weights and scores.

Eight non-monetary attributes were agreed: four focussed on the disease being treated and four addressing the treatment itself. Workshop participants reached consensus on the weights for the attributes. The patients’ group representatives and the clinical and health economics experts both assigned about half of the weight to attributes reflecting the disease being treated and half to attributes of the treatment. Patients’ group representatives, however, gave greater weight than the experts to the patient’s quality of daily life and less weight to clinical factors. The weighted attributes proved usable in practice; they readily were applied by workshop participants to two example OMPs and yielded distinct value ratings.

From this project, we conclude that an OMP value framework with agreed attributes and weights is a viable proposition using an MCDA approach. Moreover, it could improve clarity and transparency in decision making about the value of OMPs.

[1] For a thorough discussion of MCDA and its uses, see Devlin, N.J. and Sussex, J. (2011) Incorporating multiple criteria in HTA: Methods and processes. London: Office of Health Economics. The publication may be downloaded from this website by registered users.

Download: Sussex, J., Rollet, P., Garau, M., Schmitt, C., Kent, A. and Hutchings, A. Multi-criteria decision analysis to value orphan medicines. Research Paper 13/03. London: Office of Health Economics.

For additional information, please contact Jon Sussex.

OHE Welcomes Professors Rawlins and Smith to Its Policy Board

Posted on 6 May 2013 by OHE News Editor

Composed of academics and thought leaders from within and outside of the UK, OHE’s Policy Board has provided advice and guidance to the OHE on its research programmes since the 1960s. In April, Professor Sir Michael Rawlins and Professor Peter C Smith joined the group.

Professor Sir Michael Rawlins, President of the Royal Society of Medicine, was chairman of the National Institute of Health and Clinical Excellence (NICE) from its formation in 1999 to April 2013. A physician and pharmacologist, Sir Michael has provided insight in numerous roles, including as head of both the Committee on Safety of Medicines and the Advisory Council on the Misuse of Drugs. He currently is an honorary professor at the London School of Hygiene and Tropical Medicine and the University of London, as well as an emeritus professor at the University of Newcastle upon Tyne.

Professor Peter C. Smith is co-director of the Centre for Health Policy in the Institute of Global Health Innovation at Imperial College London and previoulsy was Director of the Centre for Health Economics at the University or York. His primary focus is on the economics of health and the broader public services. Prof Smith has acted in numerous governmental advisory capacities, including as a board member of the Audit Commission, and, currently, on the Monitor’s Cooperation and Competition Panel. He also has advised several other governments and international agencies.

A full list of members of the Policy Board are listed here.

NICE Decisions: Exploring the Influence of Cost-Effectiveness and Other Factors

Posted on 30 April 2013 by OHE News Editor

OHE presents a series of lunchtime seminars throughout the year. The most recent seminar, held in late April, considered the influence of cost-effectiveness and other factors on NICE decisions. Led by OHE’s Prof Nancy Devlin and Helen Dakin of the University of Oxford, the seminar discussed research recently completed by them in collaboration with OHE’s Yan Feng and Phill O’Neill, Nigel Rice of the University of York, and David Parkin, senior visiting researcher at OHE.

Established in 1999, the National Institute for Health and Clinical Excellence (NICE) undertakes appraisals of selected technologies and issues guidance intended to ensure quality and value for money. Its decisions are binding within the NHS and potentially also affect decisions by health technology assessment bodies and payers in other countries. Understanding what factors actually affect NICE decisions, then, is important for life sciences companies, other stakeholders and patients worldwide.

The research discussed at this OHE lunchtime seminar has three objectives: estimating the cost-effectiveness threshold apparent from NICE’s observed decisions, identifying which factors other than cost effectiveness affect or explain NICE’s decisions, and evaluating whether either of these has changed over time. The seminar’s discussion focused on the first two; the third is in progress.

included in the basic model for this research were the factors deemed most likely to affect NICE’s decisions : (1) incremental cost-effectiveness ratios (ICERs); (2) clinical evidence, hypothesising that NICE will reject a technology with insufficient clinical evidence; (3) insights provided by stakeholders, hypothesising that involvement increases the chance of approval; (4) when the technology is the only treatment available for a given disease, hypothesising that NICE is more likely to recommend it; (5) whether the technology is for the treatment of children, on the grounds that NICE says it gives ‘the benefit of the doubt’, given methodological challenges in measuring health outcomes in children; (6) publication date, to capture whether NICE decisions have changed through time; and (7) severity of the underlying illness, as NICE states that it accepts higher ICERs for treatments for serious conditions.

A wide range of other variables also was included in extended modelling. These included exploring the effect on decisions of the introduction of the single technology appraisal (STA) process and the use of end-of-life criteria and whether decisions differ systematically across disease area and appraisal committees.

Most of the data for the research were derived from HTAinSite, a commercial database of NICE decisions developed by the OHE, Abacus and City University. An overview of the analytic approach is included in the presentation (above).

The research found that ICERs are by far the strongest predictor, alone explaining 81% of NICE’s decisions. While some variables other than cost effectiveness are significant, their contribution to the ability of the model to predict “yes” and “no” decisions is relatively weak. The “best” model suggests that the average decision with an ICER of £42,000 has a 50% chance of being approved/rejected.

Next steps in the research include sensitivity and subgroup analysis to refine the model specification.

A paper reporting these results will be released as an OHE Research Paper in the coming months and also will be submitted to be a journal.

For further information about this research, please contact – Nancy Devlin or Helen Dakin.

OHE’s Towse on Quality Standards in the ‘New’ English NHS

Posted on 29 April 2013 by OHE News Editor

The 2013 conference of the Association of the British Pharmaceutical Industry (ABPI) addressed the challenges and opportunities presented by the recent changes in the NHS. Understanding quality, and setting and meeting standards for quality, are essential going forwards. At the conference, OHE’s Adrian Towse reviewed the experiences and concerns that have shaped the approach to quality and identified critical components.

Quality and Standards in the ‘New’ English NHS from Office of Health Economics

Valuing Co-Dependent Medical Technologies: Improving Methods and Processes

Posted on 23 April 2013 by OHE News Editor

Advances in science and technology are producing more and better means for diagnosing disease, matching patients to the best therapies, and tracking the progress of treatment. The potential impact of using diagnostic and therapeutic tests in tandem with treatment—“co-dependent technologies”—potentially can maximise both treatment effectiveness and economic efficiency. Everyone benefits—patients and payers. Approaches for valuing co-dependent technologies, however, are in their infancy. In this presentation, OHE’s Martina Garau reviews the current situation and offers suggestions for improving methods and processes for assessing the value of co-dependent technologies.

Improving Methods and Processes for Assessing Codependent Technologies from Office of Health Economics

For further information, please contact Martina Garau.

See also: Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2012) Can and should value based pricing be applied to molecular diagnostics? Personalized Medicine. 10(1), 61-72. An earlier version of this paper is available as an OHE Research Paper.

Performance-Based Risk Sharing in Health Care: Current Status

Posted on 17 April 2013 by OHE News Editor

Prof Towse on performance based risk sharing Performance-based risk sharing arrangements for prescription medicines and other treatments are attractive to payers because they promise to base what is paid for a treatment on whether/how well it actually works. Programmes typically include data collection and either implicitly or explicitly link pricing, reimbursement and/or revenue to what the data show.

At the mid-year symposium of the International Society of Pharmacoepidemiology (ISPE), OHE’s Adrian Towse reviewed the challenges such approaches present and experience to date. Adrian and Lou Garrison (University of Washington) co-chair the current ISPOR Performance-Based Risk-Sharing Arrangements Good Practices Task Force which will publish its report in Value in Health this summer. They will be presenting a half-day short course on risk-sharing/performance-based arrangements for drugs and other medical products at the ISPOR annual meeting in May 2013.

Performance based risk sharing arrangements for prescription medicines from Office of Health Economics

Examples of other OHE research on this topic

Puig-Peiró, R., Mestre-Ferrandiz, J., Sussex, J. and Towse, A. (2011) Literature review on Patient Access Schemes, flexible pricing schemes and risk sharing agreements for medicines. Poster presented at the PPRI Conference 2011. Vienna. 29-30 September.

Towse, A. and Garrison, L.P. (2010) Can’t get no satisfaction? Will pay for performance help? Toward an economic framework for understanding performance-based risk-sharing agreements for innovative medical products. Pharmacoeconomics. 28(2), 93-102.

Towse, A., Garrison, L. and Puig-Peiró, R. (2012) The use of pay-for-performance for drugs: Can it improve incentives for innovation? Occasional Paper. 12/01. London: Office of Health Economics.