Anne Mason

Topical preparations for the treatment of psoriasis: a systematic review

Summary Background There is clinical uncertainty about the appropriate use of first‐line topical treatments for psoriasis.

European Perspective on the Costs and Cost-Effectiveness of Cancer Therapies

In Europe, the vast majority of the costs of cancer therapy fall on third-party payers, normally the government, or sickness funds. Therefore, the main focus of cost-effectiveness studies is to assist payers in deciding whether new therapies are worthwhile, despite their high cost. Drug budgets are regulated in most European countries. The main form of central control is price setting, with some form of reference pricing being the most common approach. This sets the price of drugs, either to an international standard, or to a common price for drugs in the same group or cluster. At the hospital level, the main control over cancer drugs is the hospital formulary. Studies have shown a wide variation among European countries in access to cancer drugs. Explanations for these variations include differences in research funding, the drug approval process, the role of health economics in decision making, and budgetary issues. Several countries in Europe now require economic data in making decisions about the reimbursement of new drugs. An examination of decisions made by the National Institute for Health and Clinical Excellence in the United Kingdom suggests that cancer drugs have fared quite well, with most recommendations being positive. This could be because of the seriousness of the health condition and the lack of alternative therapies for some cancer patients. If the policy of requesting cost-effectiveness evidence for pricing and reimbursement decisions becomes more popular, a major implication for the pharmaceutical industry is that studies should be conducted during phase III of clinical development to generate the required data.

Comparison of Anticancer Drug Coverage Decisions in the United States and United Kingdom: Does the Evidence Support the Rhetoric?

PURPOSE In contrast to the United States, several European countries have health technology assessment programs for drugs, many of which assess cost effectiveness. Coverage decisions that consider cost effectiveness may lead to restrictions in access. METHODS For a purposive sample of five decision-making bodies, we analyzed US and United Kingdom coverage decisions on all anticancer drugs approved by the US Food and Drug Administration (FDA) from 2004 to 2008. Data sources for the timing and outcome of licensing and coverage decisions included published and unpublished documentation, Web sites, and personal communication. RESULTS The FDA approved 59 anticancer drugs over the study period, of which 46 were also approved by the European Medicines Agency. In the United States, 100% of drugs were covered, mostly without restriction. However, the United Kingdom bodies made positive coverage decisions for less than half of licensed drugs (National Institute for Health and Clinical Excellence [NICE]: 39%; Scottish Medicines Consortium [SMC]: 43%). Whereas the Centers for Medicare and Medicaid Services (CMS) and the Department of Veterans Affairs (VA) covered all 59 drugs from the FDA license date, delays were evident for some Regence Group decisions that were informed by cost effectiveness (median, 0 days; semi-interquartile range [SIQR], 122 days; n = 22). Relative to the European Medicines Agency license date, median time to coverage was 783 days (SIQR, 170 days) for NICE and 231 days (SIQR, 129 days) for the SMC. CONCLUSION Anticancer drug coverage decisions that consider cost effectiveness are associated with greater restrictions and slower time to coverage. However, this approach may represent an explicit alternative to rationing achieved through the use of patient copayments.

The Generalisability of Pharmacoeconomic Studies Issues and Challenges Ahead

Developing from a previous review, this article revisits the generalisability theme to summarise recent advances in methodology and provide an update of challenges faced by producers and users of pharmacoeconomic data. Our original evaluative criteria encompassed technical issues, applicability and transferability.The technical elements of best practice are comparatively uncontroversial: choosing relevant alternatives; transparent reporting of methods and findings; accessing and applying the best-quality evidence; using best methods to synthesise data; and using deterministic sensitivity analysis to explore potential systematic bias whilst employing probabilistic sensitivity analysis to explore the influence of random error at the whole model level. The applicability of economic findings within their original policy context (e.g. national analyses based on generalisable within-country data) can be determined, provided that best practice guidelines for economic modelling are adhered to. The transferability of economic findings (from one policy setting to another, e.g. country, region, clinical setting or patient population) requires careful exploration of changes in resource implications, unit prices and outcomes, a process facilitated again by transparent reporting of methods, adjustment for baseline risk and potentially by recent statistical developments intended to deal with hierarchically structured data.Although there is considerable consensus in the published literature about these key issues, limitations remain for economic analysis as implemented because of its opaqueness of method, failure to reflect the opportunity cost of decisions and lack of societal mandate. If the primary purpose of health economic evaluation is to help society to obtain the best value from limited resources, then, at a time when most technologically advanced societies need to engage with the realities of limited healthcare funding, technocratic solutions alone appear insufficient. Making health economic findings accessible to patients, clinicians and society, in the form of relevant narratives, will help this essential debate and expose assumptions underpinning economic analysis to broader critical inspection.

Value for money and the Quality and Outcomes Framework in primary care in the UK NHS.

BACKGROUND The Quality and Outcomes Framework (QOF) is a pioneering attempt to improve the quality of primary care in the UK through the use of financial rewards. Despite its achievements, there are concerns that the QOF may offer poor value for money. AIM To assess the cost-effectiveness of QOF payments. DESIGN OF STUDY Economic analysis. SETTING England, UK. METHOD Cost-effectiveness evidence was identified for a subset of nine QOF indicators with a direct therapeutic impact. These data were then applied to an analytic framework to determine the conditions under which QOF payments would be cost-effective. This framework was constructed to assess the cost-effectiveness of QOF payments by modelling the incentive structure using cost-effectiveness thresholds of 20 000 and 30 000 UK pounds per quality-adjusted life year (QALY) gained, to represent good value to the NHS. It used 2004/2005 data on the QOF performance of all English primary care practices. RESULTS Average indicator payments ranged from 0.63 to 40.61 UK pounds per patient, and the percentage of eligible patients treated ranged from 63% to 90%. The proportional changes required for QOF payments to be cost-effective varied widely between the indicators. Although most indicators required only a fraction of a 1% change to be cost-effective, for some indicators improvements in performance of around 20% were needed. CONCLUSION For most indicators that can be assessed, QOF incentive payments are likely to be a cost-effective use of resources for a high proportion of primary care practices, even if the QOF achieves only modest improvements in care. However, only a small subset of the indicators has been considered, and no account has been taken of the costs of administering the QOF scheme.

The Effectiveness and Cost‐Effectiveness of Respite for Caregivers of Frail Older People

The proportion of frail elderly people in the industrialized world is increasing. Respite care is a potentially important way of maintaining the quality of life for these people and their caregivers. The objective of this systematic review was to determine the effectiveness and cost‐effectiveness of different models of community‐based respite care for frail older people and their caregivers. To identify relevant studies, 37 databases were searched, and reference checking and citation searches were undertaken. Well‐controlled effectiveness studies were eligible for inclusion, with uncontrolled studies admissible only in the absence of higher‐quality evidence. Studies assessed the effect of community‐based respite on caregivers of frail elderly people relative to usual care or to another support intervention. Eligible economic evaluations also addressed costs. Where appropriate, data were synthesized using standard meta‐analytic techniques. Ten randomized, controlled trials, seven quasi‐experimental studies and five uncontrolled studies were included in the review. For all types of respite, the effects upon caregivers were generally small, with better‐controlled studies finding modest benefits only for certain subgroups, although many studies reported high levels of caregiver satisfaction. No reliable evidence was found that respite care delays entry to residential care or adversely affects frail older people. The economic evaluations all assessed day care, which tended to be associated with similar or higher costs than usual care. Given the increasing numbers of frail elderly people and the lack of up‐to‐date, good‐quality evidence for all types of respite care, better‐quality evidence is urgently needed to inform current policy and practice.

Using QALYs in Cancer: A Review of the Methodological Limitations

The objective of this paper is to examine how well the QALY captures the health gains generated by cancer treatments, with particular focus on the methods for constructing QALYs preferred by the UK National Institute for Health and Clinical Excellence (NICE). Data were obtained using a keyword search of the MEDLINE database and a hand search of articles written by leading researchers in the subject area (with follow up of the references in these articles). Key arguments were discussed and developed at an oncology workshop in September 2009 at the Office of Health Economics. Three key issues emerged. First, the EQ-5D, NICEs preferred measure of health-related quality of life (QOL) in adults, has been found to be relatively insensitive to changes in health status of cancer patients. Second, the time trade-off, NICEs preferred technique for estimating the values of health states, involves making assumptions that are likely to be violated in end-of-life scenarios. Third, the practice of using valuations of members of the general population, as recommended by NICE, is problematic because such individuals typically display a misunderstanding of what it is really like for patients to live with cancer. Because of the way in which it is constructed, the QALY shows important limitations in terms of its ability to accurately capture the value of the health gains deemed important by cancer patients. A research agenda for addressing these limitations is proposed.

New medicines in primary care: a review of influences on general practitioner prescribing

Background and objective:  The uptake of new medicines is slower in the UK than in many other countries. Previous research found that cost and price have little influence on general practitioner (GP) behaviour, but recent UK government policy may have heightened cost‐consciousness. Focussing on new medicines, this review aimed to explore the determinants of uptake, the causes of geographical variations, and the influence of price, cost and financial incentives on prescribing behaviour.

Using QALYs in Cancer

The objective of this paper is to examine how well the QALY captures the health gains generated by cancer treatments, with particular focus on the methods for constructing QALYs preferred by the UK National Institute for Health and Clinical Excellence (NICE). Data were obtained using a keyword search of the MEDLINE database and a hand search of articles written by leading researchers in the subject area (with follow up of the references in these articles). Key arguments were discussed and developed at an oncology workshop in September 2009 at the Office of Health Economics.Three key issues emerged. First, the EQ-5D, NICE’s preferred measure of health-related quality of life (QOL) in adults, has been found to be relatively insensitive to changes in health status of cancer patients. Second, the time trade-off, NICEs preferred technique for estimating the values of health states, involves making assumptions that are likely to be violated in end-of-life scenarios. Third, the practice of using valuations of members of the general population, as recommended by NICE, is problematic because such individuals typically display a misunderstanding of what it is really like for patients to live with cancer.Because of the way in which it is constructed, the QALY shows important limitations in terms of its ability to accurately capture the value of the health gains deemed important by cancer patients. A research agenda for addressing these limitations is proposed.

The hospital costs of care for stroke in nine European countries

Stroke is a major cause of mortality and morbidity, but the reasons for differences in costs of care within and between countries are not well understood. The HealthBASKET project used a vignette methodology to compare the mean costs and prices of hospital care across providers in nine European Union countries. Data on resource use, unit costs and prices of care for female stroke patients without co-morbidity were collected from a sample of 50 hospitals. Mean costs for each provider were analysed using multiple regression. Sensitivity analysis explored the effects on cost of using official exchange rates, purchasing power parity (PPP) and proportion of national income per capita. The mean cost of a hospital episode per patient for stroke at PPP was 3813 euros (standard error 227) with an additional day in hospital typically associated with 6.9% (95% CI: 4-9%) higher costs and thrombolysis associated with 41% higher costs (10-73%). After adjusting for explanatory factors, about 76% of the variation in cost could be attributed to between-country differences, and the extent of this variation was sensitive to the method of currency conversion. There was considerable variation in the care pathways within and between countries, including differences in the availability of stroke units and access to rehabilitative services, but only the length of stay and use of thrombolytic therapy were significantly associated with higher cost. The vignette methodology appears feasible, but further research needs to consider access to healthcare over a longer follow up and to include both costs and outcomes.