Annual Lecture 2014: Is Universal Health Coverage the Holy Grail?

Anne Mills Annual Lecture on Universal Heath Care

Towse, Mills, Drummond

Each year, the OHE sponsors a lecture that explores a timely issue in medicine or health economics. At the 22nd Annual Lecture, held in June 2014, the issues and challenges of universal health care coverage in low- and middle-income countries were presented by Professor Anne Mills of the London School of Hygiene and Tropical Medicine. What follows is a brief summary of her remarks.

Universal health coverage[1] (UHC) in developing countries currently is the topic of intense debate in the global health community. With the Millennium Development Goals set to expire in 2015, momentum is growing towards making universal coverage a key global goal. At the World Health Assembly in 2014, the Director General of the WHO suggested a target of 80% effective coverage for key health services for all population groups within each country by 2030.

In part, the focus on UHC is a response to the concerns about the unintended consequences of the large increase in development assistance for health since the early 1990s. Most such assistance focused on specific diseases or disease areas, but neglected development of national health care infrastructures and sustainable financing systems. Access to care continues to be a serious challenge in low- and middle-income countries (LMICs), with comparatively high out-of-pocket payments still posing a serious problem.

To achieve UHC, countries must solve the puzzles created by three core challenges: sufficient and stable financing, reliable and effective financial intermediaries, and a sufficient supply of competent service providers.

 Financing

The greatest challenge is coverage for low income workers and the self-employed — usually a large portion of the working-age population — and those outside the workforce, i.e. the young, elderly, disabled and unemployed. A mix of financing approaches and sources currently are used in the LMICs, including: social health insurance, general taxation, voluntary contributions and out-of-pocket payments. The particular mix varies by country; evidence is not yet available to suggest which specific mix may be most effective in a particular setting. However, experience does suggest that a core mandatory financing mechanism is essential – either social health insurance or general tax revenues.

Financial intermediaries

Issues include whether systems should be single payer or allow more than one fund, and what the role of the private sector should be. Multiple funds can produce inequities, but many countries still use a mix. Although the public sector is the traditional intermediary, more countries are experimenting with a mix of public and private, contracting with private companies to manage arrangements. India’s RSBY, for hospital coverage, is an example.

Service providers

To achieve UHC, the first concern should be care for those most vulnerable, i.e. the poorest sectors of the population. This requires, of course, that sufficient services be available in the appropriate locales, not just in the larger cities. Discussion of financing issues, especially insurance options, too often tends to eclipse the essential focus on extending service provision. Ensuring access to both primary and hospital care, and integrating the two, remains problematic. Providers may be either public or private, although the private sector is expanding and, with greater resources, often has superior infrastructure.

Research most often focuses on the evaluation of performance in relation to the design features of UHC, but just as important are the institutional underpinnings of UHC. More attention needs to be paid to what political, economic, social and cultural institutions might allow government to pursue and make progress towards UHC. This includes an effective civil service with recruitment and tenure based on merit; ensuring that the voice of those less well off are heard and factored into policy debates; incentives to encourage those better off to remain in the system, along with evidence of the impact of this; and sufficient social solidarity to support cross-subsidisation of income groups.

It is critical to put the necessary elements of UHC in place early on because it can be politically difficult to make changes later. Four elements are particularly important:

  1. A combination of financing sources
  2. Strong purchasing arrangements that make use of both public and private providers and include an emphasis on health promotion and disease prevention
  3. A payment system that includes incentives for both cost containment and quality of care
  4. Strong primary care and local-level infrastructure with appropriate geographic spread.

The evolution of health care systems towards UHC is, and will continue to be, very country-specific; what works in one simply may not work in another. Whatever the system or ideology, however, a strong government role is necessary, even if the government is not the only actor.

Although discussions — and the movement towards adopting UHC as a global goal — may suggest the opposite, it is crucial to understand that UHC is a process over time, not a fixed point that can be achieved once and for all.

[1] WHO defines universal health coverage as ‘ensuring that all people can use the promotive, preventive, curative, rehabilitative and palliative health services they need, of sufficient quality to be effective, while also ensuring that the use of these services does not expose the user to financial hardship.’

About Professor Mills

Professor Anne Mills CBE MA DHSA PhD FMedSci FRS is a world renowned expert in health economics, health care financing and policy in low- and middle-income countries.

She has a distinguished academic career at the London School of Hygiene and Tropical Medicine, where she is Vice Director of the School and Professor of Health Economics and Policy in the Department of Global Health and Development. She previously served as Director of the Health Economics and Financing Programme, which was supported by a variety of research grants from funders such as DFID, the Wellcome Trust, EU and WHO, and as the Head of the Faculty of Public Health and Policy. She has advised many multilateral, bilateral and government agencies; served on the WHO’s 2001 Commission on Macroeconomics and Health chaired by Jeffrey Sachs; and co-chaired one of the two Working Groups for the 2009 High Level Taskforce on Innovative International Finance for Health Systems, co-chaired by Prime Minister Gordon Brown.

In 2006 she was awarded a CBE for services to medicine and elected a Foreign Associate of the US Institute of Medicine. In 2009 she was elected Fellow of the UK Academy of Medical Sciences and received the Prince Mahidol Award in the field of medicine. She was President of the International Health Economics Association (iHEA) for 2012/13. She was elected a Fellow of the Royal Society in 2013.

This Annual Lecture will be available as a publication and, as for previous years, will be available for download from the publications section of this website. To view blog posts about earlier OHE Annual Lectures, click on the Annual Lecture tag in the right column of this blog.

 

 

EmailLinkedInShare

What Determines the Shape of an EQ-5D Index Distribution?

What Determines the Shape of an EQ-5D Index Distribution? Parkin, Devlin and FengIn a new OHE Research Paper, OHE’s Nancy Devlin and Yan Feng join David Parkin from King’s College London in analysing characteristics of the EQ-5D indexing process that may obscure useful, and important, information about health states.

Because the EQ-5D is one of the most used health-related quality of life instruments worldwide, it is crucial that the characteristics of the data be well understood. The EQ-5D is used to ask patients or others to self-report their health in terms of five dimensions and three levels of problems (‘no’, ‘some’ or ‘extreme’). That information from patients is then summarised by a single number, anchored at 0 (dead) and 1 (full health) to show how ‘good’ or ‘bad’ each state is. The authors of this paper note that distributions of EQ-5D index values in patient and general populations have an interesting characteristic: they typically divide into two distinct groups. This might reflect the actual distribution of ill health, but it also might be an artefact of how the EQ-5D index is constructed.

In this OHE Research paper, the authors examine the determinants of the shape of EQ-5D distributions, particularly the origins of the ‘two groups’ distribution. They analyse data from elective English NHS surgery patients (hip and knee replacements, and varicose vein and hernia repairs) and a study of primary care patients with chronic disease (asthma and angina). The distributions of EQ-5D index values are compared to distributions based on data that have not been weighted; with the distributions that arise when different countries’ weights are used to summarise patients’ data; and with condition-specific indexes for the same patients.

The research shows that a very small number of profiles account for most of the observed data: just twelve EQ-5D health state profiles account for 86% of all the health states reported by patients. The explanation for the ‘two groups’ characteristic seems to lie both in the grouping of profiles and in the nature of the weights applied to them. Examining the group with higher index values, the most commonly observed health states all had ‘some’ problems with mobility, usual activities and pain, and had either ‘no’ or ‘some’ problems with self care and anxiety/depression. In the group with lower index values, the most commonly observed health states all had ‘extreme’ problems with pain/discomfort and some also had the worst level of usual activities. The weights commonly used to calculate the index place more weight on level 3 health problems, creating a noticeable gap in index values across the two groups.

The authors also show that the ‘two-groups’ characteristic of EQ-5D index distributions is not uniquely associated with the use of the UK value set. Applying different value sets produces different distributions, but the two-group distribution arises in most cases. However, the difference between the distributions in each case serves as a reminder that, for any given set of patients’ EQ-5D data, which value set is used to summarise them will have an important bearing on the results.

The authors point out that concentrating on the EQ-5D index may obscure useful information about health states and possibly produce misleading information. They emphasise the importance of exploratory analysis of EQ-5D data — both to improve analyses of EQ-5D data for comparison and inference purposes, and to help develop more accurate mapping across different health measures. Any health status index based on weighted profiles, the authors note, will benefit from this analytical approach.

Download Parkin, D., Devlin, N. and Feng, Y., 2014. What determines the shape of an EQ-5D distribution? Research Paper 14/04. London: Office of Health Economics.

For further information, please contact Professor Nancy Devlin. For an overview of OHE’s extensive activities in patient-reported outcomes measures, please click here.

EmailLinkedInShare

Bengt Jönsson Honoured for Contribution to Health Economics

Portrait of a Health Economist -- Bengt JonssonLaunched at a seminar in his honour May 2014, Portrait of a Health Economist: Essays by Colleagues and Friends of Bengt Jönsson is an impressive collection of essays commemorating Bengt’s lifetime contribution to health economics. He was one of the true pioneers in the field – his 1976 doctoral thesis was on cost-benefit analysis in public health and medical care. He has since published hundreds of papers, reports and book chapters worldwide. In 1979, Bengt founded the Swedish Institute for Health Economics (IHE), using the OHE as a model, and in 1982 became professor of health economics at the University of Linköping, where he founded the Center for Medical Technology (CMT). IHE and CMT today are the focal institutions for health economics in Sweden. In 1991, Bengt moved to the Stockholm School of Economics, where he is now Professor Emeritus and remains as active as ever. He joined the OHE Policy Board[1] in 2007.

Edited by Tony Culyer and Gisela Kobelt, the book’s list of contributors reads like a worldwide who’s who of experts in the field, including OHE Policy and Editorial Board members Martin Buxton, Tony Culyer, Mike Drummond, and Peter Zweifel, as well as past OHE Annual Lecturers Bob Evans, Uwe Reinhardt and Milt Weinstein. The thirty chapters address a range of topics including: health economics and politics; the theory underlying the design of health economic evaluation and its use in decision making; specific analytical techniques and approaches, e.g. QALYs; appropriate use of cost effectiveness analyses in health care decision making in general, in specific countries or to meet specific goals such as designing a benefits package; the health economics of particular diseases, e.g. diabetes and cancer; and chapters specifically on aspects of Bengt’s contributions to the field.

The chapter contributed by OHE’s Adrian Towse[2] discusses Bengt’s observations in a 2011 publication[3] about the role of relative effectiveness research (RE) in the European medicines market. To ensure value for money, understanding actual use of medicines in the health care system is an essential supplement to information from clinical trials. To move in this direction, Towse defines a deceptively simple challenge: creating an environment where choice of therapy is based on an optimal amount of evidence from relative effectiveness research that is generated and used efficiently across health care systems. He identifies three sets of changes that must occur to meet the challenge:

  1. A new drug development paradigm supported by regulatory authorities, HTA agencies and pharmaceutical companies that encourages the efficient collection of RE data before and after a medicine reaches the market
  2. Concerted efforts to eliminate the currently costly duplication across countries in infrastructure and RE-based assessments
  3. Understanding how and how well individual health systems use and benefit from new medicines, both to identify when expected differences mean country-specific RE may be needed and help identify instances where health systems use treatments inefficiently.

Towse notes that ‘appraisal of RE evidence should edge both parties towards efficient pricing and use of a new drug’, (p. 185), which he believes EU reforms can encourage in various ways. He is somewhat less optimistic about trans-Atlantic convergence.

[1] OHE’s Policy Board provides advice and guidance on OHE’s research programmes. A full list of current Policy and Editorial Board members is available here on our website.

[2] Towse, A., 2014. Comparative and relative effectiveness: a challenge for health systems, regulators, or the pharmaceutical industry? In Culyer, A.J. and Kobelt, G. eds. Portrait of a health economist: essays by colleagues and friends of Bengt Jönsson. Lund: Swedish Institute for Health Economics, pp.179-188.

[3] Jönsson, B., 2011. Relative effectiveness and the European pharmaceutical market. European Journal of Health Economics. 12(2), pp.97-102.

The book may be downloaded as a pdf from the website of the Swedish Institute of Health Economics.

 

 

 

EmailLinkedInShare

Spotlight on OHE: Competition in the NHS, HESG, Publications

OHE was active in several forums in June. This blog post summarises the highlights.

Competition in the NHS

Jon SussexJon Sussex was a keynote speaker at the Capita conference on competition in health care held in June, where the audience was primarily from the NHS. Jon’s presentation summarised the state of provider competition in the NHS to date as well as national and international evidence about the impact of competition on quality. Competition would be important for some services, Jon noted, but unimportant for others. Assessing the feasibility of competition is essential using, for example, the framework developed by the OHE Commission on NHS Competition.

OHE at HESG: Health Care Decision Making, Hospital Market Concentration

The Health Economists’ Study Group (HESG) was founded in 1972 to support and promote the work of health economists. Its members are from academic, commercial, and government settings. The purpose of HESG is to transmit knowledge and ideas, ranging from the theoretical to very practical policy issues. HESG meets twice a year, most recently in June 2014.

Two OHE papers were discussed at the most recent HESG meeting.

  1. A paper by Karla Hernandez-Villafuerte and Jon Sussex that sets out a preliminary model intended to characterise the behaviour of decision makers in the health sector who must make choices based on budget constraints set by government or another health system authority. Specifically, the model focuses on the options available when use of new health technology is mandated.

See also the recent OHE Research Paper Opportunity costs of implementing NICE decisions in NHS Wales.

  1. A paper co-authored by Yan Feng, Michele Pistollato, Anita Charlesworth, Nance Devlin, Carol Propper and Jon Sussex that assesses the relationship between market concentration in hospital care and health gains as reported by patients following primary hip replacement surgery. This research is a joint project of OHE and the Nuffield Trust.

June Publications

In June, OHE welcomed two new members to its team, Dr Amanda Chapman and Grace Marsden. Shortly after beginning her position at OHE, a paper on which Amanda is lead author appeared online in Applied Health Economics and Health Policy. The article is based on Amanda’s work on medical devices while at the University of Birmingham and assesses the effect of NICE’s Medical Technologies Evaluation Programme over its first three years of operation[1].

Also just published online is an article by Karla Hernandez-Villafuerte on age and inequalities in access to care[2].

To inquire further, please contact the relevant OHE team member by visiting the About Us section of the website.

 

[1] Chapman, A., Taylor, C.A. and Girling, A.J., 2014. Are the UK systems of innovation and evaluation of medical devices compatible? The role of NICE’s Medical Technology Evaluation Programme (MTEP). Applied Health Economics and Health Policy. DOI: 10.1007/s40258-014-0104-y.

[2] Hernandez-Villafuerte, K., 2014. El adulto mayor y la universalidad de la salud: Análisis de desigualdad basado en una comparación entre los diferentes grupos de edad. Estudios de Economia Aplicada. [Available in Spanish only.]

 

EmailLinkedInShare

Interdependencies in Funding Medical Research in the UK

Exploring the Interdependencies of Research Funders in the UKCancer Research UK has recently released a report completed for it by OHE and the Science Policy Research Unit (SPRU) at the University of Sussex that focuses on the strength and nature of interdependence in the funding of cancer research.

As earlier OHE research has demonstrated, sources of funding for medical research—public, charity and private sector—are complementary in effect, not duplicative. The three sectors also differ in their approaches; reduced funding from one would not only decrease the overall financing available, but also change the nature of the research effort overall. This is a concern given that austerity in government spending in the UK is likely to continue for some time.

OHE and SPRU produced a report for Cancer Research UK to explore two sets of questions:

  1. What are the differences in the research activities supported by various funders in the UK? How complementary and interdependent are they?
  2. If government funding of life sciences research was cut, what would be the effect on life sciences research in the UK? Could other funders compensate for the loss of funds?

The research for the report included the following.

1.  Economies of scale in charity- and publicly-funded medical research in the UK were explored via a literature review, interviews with major UK medical research funders and analysis of cost data provided by those interviewed.

The study found that economies of scale are relatively modest except when research requires particularly specialized and costly equipment and infrastructure.

2. Analysis of the interdependencies and the differences across the research activities supported by the diverse funders of cancer research in the UK. Research publications were examined to gain an understanding of the importance of funders named or acknowledged, including those from outside the UK.

The study found that funding for two-thirds of projects reported in publications routinely came from more than one funder, just under half had funding from outside the UK, and a fifth received funding from industry. The study’s findings reflect the highly collaborative nature of cancer research, with the majority of papers being produced by teams working across organisations and many relying on international co-authorship ties. These ties leverage thousands of national and international funding sources, large and small, to support the 7% of global cancer publications that the UK produces.

3. To explore the connections between government and charity funding of cancer research in the UK, the study included an Internet-based survey of how changes in government funding might affect the general public’s willingness to donate to cancer research charities.

The study found that the general public would not want public funding for cancer research to be reduced, but would also not donate more to compensate for a reduction.

The report concludes that:

Exploring the interdependencies between different medical research funders, particularly in cancer, provides us with a striking picture of the extent to which research funders contribute together to produce world class research. The findings provide a compelling case for why investment—by all sectors—is needed to allow the UK to maximize its research outputs.

Download Shah, K., Sussex, J., Hernandez-Villafuerte, K., Garau, M., Rotolo, D., Hopkins, M., Grassano, N., Crane, P., Lang, F., Hutton, J., Pateman, C., Mawer, A., Farrell, C. and Sharp, T., 2014. Exploring the interdependencies of research funders in the UK. Research Paper 14/03. London: Office of Health Economics.

For additional information, please contact Jon Sussex at OHE. For earlier OHE research on these issues, click here.

The report is also available on Cancer Research UK’s website.

 

 

EmailLinkedInShare

OHE Consulting Report on the Trajectory of Dementia in the UK

The trajectory of dementia in the UK – making a differenceAlzheimer’s Research UK commissioned OHE Consulting to model the growing prevalence and costs of dementia in the UK and the impact that new treatments could have were they to be introduced from 2020.

Today over 800,000 people aged 60 and over are living with dementia in the UK. The risk of developing dementia increases greatly with age, from around 1% of men and 1.5% of women in their sixties to more than a quarter of men and a third of women aged 90 and over. The ageing of the UK population, then, will mean an increasing number of people living with dementia.

Based on the latest official UK population projections and the most authoritative estimates of the prevalence of dementia, OHE Consulting built a model to project the number of people with dementia in the UK to 2050. The model then was used to project the health and social care costs of dementia. The results show that unless ways are found to prevent or cure dementia, the number or people in the UK aged 60 and over who will be living with dementia is likely to double over the next 25 years and reach two million by 2050.

The economic cost to the UK of caring for people with dementia is estimated to grow correspondingly from £24 billion in 2014 to £59 billion (at constant prices) by 2050. A large part of the cost of care is the time given up by informal carers to look after loved ones with dementia; the UK will need around 1.7 million informal carers by 2050 compared to the around 700,000 currently giving care.

OHE’s model shows how new interventions to prevent and treat dementia could radically change this picture. Delaying the onset of dementia by five years in the UK from 2020 could reduce the projected number of people with dementia in 2050 by one third (666,000) and reduce the need for informal carers by 566,000; costs of care would be £21 billion lower in 2050 (£38 billion rather than £59 billion).

Even if it were not possible to delay the onset of dementia, slowing the rate at which it progresses – from mild to moderate to severe – would bring major improvements in quality of life and less premature mortality from dementia.

Download Lewis, F., Schaffer, S.K., Sussex, J., O’Neill, P. and Cockcroft, L., 2014. The trajectory of dementia in the UK – making a difference. Consulting Report. London: Office of Health Economics.

For additional information, please contact Jon Sussex at OHE.

 For an associated policy report by Alzheimer’s Research UK click here.

EmailLinkedInShare

Spotlight on OHE: NICE, Biosimilars, HTA

OHE’s active involvement in a number of recent conferences and meetings has contributed to discussions on a range of important issue in health economics.

 Changes in NICE decision making

The consultation process for changes in how NICE assesses the value of a health technology is scheduled to be completed in June 2014. In a presentation at the PharmaAccess Leader’s Forum, Martin Garau summarised the proposed changes that are part of the value based assessment consultation. She illustrated how two suggested decision-making factors, burden of illness and wider societal impact, might be measured and calculated for specific interventions.

For a comprehensive reviews of OHE’s extensive publications and research on NICE, click here.

For additional information about this presentation, please contact Martina Garau.

HTA and Biosimilars

At the 34th Spanish Health Economics Association annual meeting, Jorge Mestre-Ferrandiz reviewed the results of the discussions of an expert panel about the role of HTA for biosimilars. Representatives from the three UK HTA agencies, the MHRA and academia were involved in debating which HTA methods are most appropriate for biosimilars in specific situations.

The results of the roundtable also are available on this website as an OHE Briefing.

For additional information, please contact Jorge Mestre-Ferrandiz.

HTA in Latin America

In a poster presentation at the 34th Spanish Health Economics Association annual meeting, Karla Hernandez-Villafuerte presented the first stage of research into the pharmaceutical market in Latin America. The poster presentation demonstrated the variety of approaches across countries and the importance of HTA.

For additional information, please contact Karla Hernández-Villafuerte.

 

 

EmailLinkedInShare

Implementing NICE Decisions in Wales: Opportunity Costs

Opportunity Costs of Implementing NICE Decisions in NHS WalesJust out is a new Research Paper from OHE that examines how local NHS organisations in Wales adjust spending to accommodate ‘shocks’ such as new NICE mandates for covering specific technologies.

By law, technologies recommended by NICE are to be funded within three months of publication of NICE’s technology appraisal (TA) decision. Explicit in NICE’s approach to health technology assessment (HTA) is the assumption that new, cost-increasing technology will displace spending on existing, less cost-effective health care. To date, however, no research has explored whether this happens in practice, which is important in understanding the actual effects of NICE’s use of HTA. In this Research Paper, the authors seek to document experience in NHS Wales and review how budget holders set priorities for spending.

Semi-structured interviews were completed with finance directors and/or medical directors from all seven Local Health Boards (LHBs) in NHS Wales. These interviews covered processes for setting priorities and approaches to dealing with sudden financial demands, or ‘shocks’, such as new NICE TAs, for the period October 2010 – March 2013.

The study found that the financial impact of NICE TAs is generally anticipated and planned for in advance and that the majority of LHBs have contingency funds available to cope with these and other ‘shocks’. Efficiency savings, i.e. reductions in costs with no expected reductions in quality, were a major source of funds for dealing with cost pressures of all kinds. Additionally, the Welsh Government has acted as the funder of last resort on occasion. Service displacements, moreover, could not be linked to particular NICE TAs.

The authors conclude that: ‘Services may be displaced as part of a response to the cumulative impact of all types of cost pressures, including cost-increasing health technologies newly recommended by NICE, but such displacements were not direct responses to the publication of individual NICE TAs.’ Cost pressures from new NICE TAs were likely to be accommodated at least in part by greater efficiency and increased expenditure.

Download Schaffer, S.K., Sussex, J., Hughes, D. and Devlin, N., 2014. Opportunity costs of implementing NICE decisions in NHS Wales. Research Paper 14/02. London: Office of Health Economics.

For additional information, please contact Sarah Karlsberg Schaffer at OHE.

EmailLinkedInShare

OHE’s Adrian Towse to Take Office as ISPOR President

Towse speaking LI 2-24-14

Adrian Towse

OHE’s Director, Adrian Towse, is President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) for the 2014–15 term, beginning 1 July. His 3 June incoming president’s address to the plenary session at the 19th Annual International Meeting is reproduced below. 

ISPOR Incoming President’s Remarks

When I stood for election as President of ISPOR, I set out my view of the challenges ISPOR must address:

1. Continuing to globalise, by which I mean responding to the reality that most of the growth of interest in outcomes research and economic analysis is taking place outside North America and Europe

2. Working together with payers, who are demanding greater evidence of value, to establish what is feasible, given data, costs and the science and, over time, improve methods, the evidence base, and decision making

3. Continuing to raise scientific standards for health economics and outcomes research and

4. Supporting the next generation of researchers, by providing forums and opportunities both for learning and for exchanging important new ideas and approaches.

This meeting sets a record for the number of attendees at a North America ISPOR meeting and includes participants from a record number of different countries, a record number of student chapters, and regional chapters; the Asia Consortium is celebrating its first decade, the Latin American Consortium is celebrating ten years next year, and the Arabic and African chapter is looking to achieve Consortium status. Clearly, then, ISPOR is addressing important challenges, but I want to say a little more about the first challenge, globalisation.

  • Globalisation of ISPOR must be linked to an understanding of decision making in middle- and low-income country (MLIC) health systems.
  • MLICs increasingly wish to achieve universal health coverage (UHC) for their populations.
  • Using HTA, economic analysis and outcomes research to support priority setting as part of UHC will be important.
  • A key challenge in HTA in these, and indeed all regional settings, will be understanding the potential transferrability of evidence (its public good characteristics) to avoid reinventing the wheel. In Europe, we finally are trying to do this through the EUnetHTA Joint Action. Let us try to avoid building in duplication from the beginning elsewhere in the world.
  • Successful implementation of UHC also will require creating incentives in health care systems to achieve better outcomes, and creating the tools and systems to measure performance.

In other words, diverse health systems have diverse needs for information, economic analysis and outcomes research.

ISPOR must lead the scientific developments necessary to respond to these needs. We must break out of the narrow perspective of focussing on very detailed analysis of new drugs. Undertaking such analyses is a key challenge for many health systems. And ISPOR members are very good at meeting this challenge in any setting. But issues around health policy, the architecture of the health system, and how high quality outcomes can be incentivised and monitored are also pressing – particularly, I would argue, in countries seeking to increase access to health care services.

To this end, we should remind ourselves that we already recognise this wider role for our methods and skills in ISPOR. Here are some definitions from the ISPOR Book of Terms:

  • Outcomes research is “the scientific discipline that evaluates the effect of health care interventions on patient related if not patient specific, clinical, humanistic and economic outcomes” (my emphasis)
  • A health care intervention is in turn “a program, policy, measure, or activity designed to have an impact on an illness or disease in an individual or a population” (my emphasis). This is not necessarily a single product
  • Health care technology (for HTA) consists of:

– Drugs, biologics, devices, procedures

Support systems, organisational, delivery and managerial systems (e.g. disease management programme, health care payment system) (my emphasis)

So I repeat again: diverse health systems have diverse needs for information, economic analysis and outcomes research. ISPOR can lead the scientific developments essential to responding to these needs. At stake is the ability of people around the world to gain access to universal health coverage — for the first time.

Of course, ISPOR already is global in its reach. An important part of ISPOR’s growing global footprint are the biennial conferences in Asia and in Latin America. I urge you to join me in Beijing in September for the next ISPOR conference, my first as ISPOR President, and remind you of the third ISPOR conference of 2014, in Amsterdam in November.

ISPOR’s publications are global and now include not only our flagship journal Value in Health, but also three regional issues of Value in Health – Asia, CEEWAA (Central and Eastern Europe, Western Asia, and Africa), and Latin America. The regional issues have moved to article-based electronic publishing with one full volume each year. The rising quality of these publications is reflected by (1) a projected 30% increase in the impact factor of Value in Health and (2) plans to get the regional issues included in Medline/PubMed.

Raising the global impact of ISPOR’s drive for good science is also reflected in the expanding webinar series and the current activities of the ISPOR Good Practice Task Forces.

I look forward to working with the new Incoming President Dan Malone, his newly elected colleagues on the board, and the continuing board members. Together, we will work to ensure that ISPOR continues to develop, with the support of a new executive director.

In closing, I emphasise once more that providing efficient and effective health care is truly a global challenge. ISPOR is a global organisation that exists to help meet this challenge. Diverse health systems face different challenges. ISPOR must anticipate and respond to this broad range of needs. We can do this.

 

Founded in 1995, ISPOR promotes the science of pharmacoeconomics (health economics) and outcomes research and facilitates the translation of this research into useful information for health care decision makers to increase the efficiency, effectiveness, and fairness of health care to improve health. It has more than 7300 members from 105 countries; the 71 ISPOR Regional Chapters have more than 6000 members, extending ISPOR’s outreach to over 13,300 members worldwide.
EmailLinkedInShare

Orphan Drugs Policies: A Suitable Case for Treatment

OHE's Prof Adrian TowseAn editorial just published by OHE’s Adrian Towse and Michael Drummond of the University of York argues that current orphan drug policies are ‘not fit for purpose’ and discusses the issues that need to be clarified as the basis for policy revisions. The editorial is intended ‘not to provide complete solutions to all the policy problems, but rather to set out a roadmap whereby they can be resolved’. Four groups of issues are identified.

  1. Recent surveys appear to suggest that the general public would not necessarily allocate more resources to those with orphan diseases than those with common diseases. The authors suggest that those surveyed may have been influenced by how the questions were framed and that other methods of eliciting preferences should be tested. These include asking directly whether special funding for those suffering from orphan disease should exist and, if so, how much. Clarifying society’s preferences should be the first priority in working towards better public policy.
  1. How orphan drugs may be best assessed to determine pricing and reimbursement continues to present challenges. Towse and Drummond identify the primary challenges this has presented, and suggest potential responses. But they also raise an important, and controversial, issue:

If it is known in advance that most of these drugs will not be cost-effective based on standard criteria, why undertake the assessments in the first place, unless there is the possibility that better value for money could be obtained if the drugs were better targeted? It might be better to acknowledge at the outset that high-priced orphan drugs will not be reimbursed, thereby avoiding wasting resources on their assessment.  

  1. New approaches to treatment, including advances in targeting treatment to the individual patient, may produce the temptation for companies to make more ordinary diseases appear as ‘orphans’, according to Towse and Drummond. The problem of ensuring that incentives produce the intended consequences may require redefining ‘orphan disease’ or removing orphan status when a product achieves other, non-orphan, indications.
  1. ‘Probably the biggest concern about current policies for orphan drugs’, according to the authors, ‘is that the policies for stimulating research and providing reimbursement are at odds with one another, leading to inefficiencies if scarce resources are devoted to the research and development of drugs that are not going to be used’. These policies, they argue, must be ‘joined up’. This might include explicit statements of priority, signaling to drug developers which new products are most likely to receive reimbursement. Setting appropriate priorities, moreover, could be supported by product development partnerships that involve the public and charity sectors.

Towse and Drummond conclude that: ‘Without changes in the current policies, pharmaceutical companies will eventually cease responding to the incentives to develop orphan drugs because they will increasingly be uncertain whether the drugs, if developed, will be reimbursed’.

Open access: Drummond, M. and Towse, A., 2014. Orphan drugs policies: a suitable case for treatment. The European Journal of Health Economics. 15(4), pp.335-340.

For additional information, please contact Adrian Towse.

EmailLinkedInShare