Cost–effectiveness of colorectal cancer screening strategies: A systematic review.
Clinical Gastroenterology and Hepatology, (2019) 17(10): 1969-1981.
(available on request)
Widespread screening for colorectal cancer (CRC) has reduced its incidence and mortality. Previous studies investigated the economic effects of CRC screening. We performed a systematic review to provide up-to-date evidence of the cost effectiveness of CRC screening strategies by answering 3 research questions.
We searched PubMed, National Institute for Health Research Economic Evaluation Database, Social Sciences Citation Index (via the Web of Science), EconLit (American Economic Association) and 3 supplemental databases for original articles published in English from January 2010 through December 2017. All monetary values were converted to US dollars (year 2016). For all research questions, we extracted, or calculated (if necessary), per-person costs and life years (LYs) and/or quality-adjusted LYs, as well as the incremental costs per LY gained or quality-adjusted LY gained compared with the baseline strategy. A cost-saving strategy was defined as one that was less costly and equally or more effective than the baseline strategy. The net monetary benefit approach was used to answer research question 2.
Our review comprised 33 studies (17 from Europe, 11 from North America, 4 from Asia, and 1 from Australia). Annual and biennial guaiac-based fecal occult blood tests, annual and biennial fecal immunochemical tests, colonoscopy every 10 years, and flexible sigmoidoscopy every 5 years were cost effective (even cost saving in most US models) compared to no screening. In addition, colonoscopy every 10 years was less costly and/or more effective than other common strategies in the United States. Newer strategies such as computed tomographic colonography, every 5 or 10 years, was cost effective compared with no screening.
In an updated review, we found that common CRC screening strategies and computed tomographic colonography continued to be cost effective compared to no screening. There were discrepancies among studies from different regions, which could be associated with the model types or model assumptions.
Moving towards accountability for reasonableness: A systematic exploration of the features of legitimate healthcare coverage decision-making processes using rare diseases and regenerative therapies as a case study.
International Journal of Health Policy and Management, (2019) 8(7): 424-443.
The accountability for reasonableness (A4R) framework defines 4 conditions for legitimate healthcare coverage decision processes: Relevance, Publicity, Appeals, and Enforcement. The aim of this study was to reflect on how the diverse features of decision-making processes can be aligned with A4R conditions to guide decision-making towards legitimacy. Rare disease and regenerative therapies (RDRTs) pose special decision-making challenges and offer therefore a useful case study.
Features operationalizing each A4R condition as well as three different approaches to address these features (cost-per-QALY-focused and multicriteria-based) were defined and organized into a matrix. Seven experts explored these features during a panel run under the Chatham House Rule and provided general and RDRT-specific recommendations. Responses were analyzed to identify converging and diverging recommendations.
Regarding Relevance, recommendations included supporting deliberation, stakeholder participation and grounding coverage decision criteria in normative and societal objectives. Thirteen of 17 proposed decision criteria were recommended by a majority of panelists. The usefulness of universal cost-effectiveness thresholds to inform allocative efficiency was challenged, particularly in the RDRT context. RDRTs raise specific issues that need to be considered; however, rarity should be viewed in relation to other aspects, such as disease severity and budget impact. Regarding Publicity, panelists recommended transparency about the values underlying a decision and value judgements used in selecting evidence. For Appeals, recommendations included a life-cycle approach with clear provisions for re-evaluations. For Enforcement, external quality reviews of decisions were recommended.
Moving coverage decision-making processes towards enhanced legitimacy in general and in the RDRT context involves designing and refining approaches to support participation and deliberation, enhancing transparency, and allowing explicit consideration of multiple decision criteria that reflect normative and societal objectives.
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Health Technology Assessment (HTA) and economic evaluation: Efficiency or fairness first.
Journal of Market Access & Health Policy, (2019) 7(1): 1-12.
The economic evaluation which supports Health Technology Assessment (HTA) should inform policy makers of the value to society conferred by a given allocation of resources. However, neither the theory nor practise of economic evaluation satisfactorily reflect social values. Both are primarily concerned with efficiency, commonly conceptualised as the maximisation of utility or quality adjusted life years (QALYs). The focus is upon the service and the benefits obtained from it. This has resulted in an evaluation methodology which discriminates against groups and treatments which the population would like to prioritise. This includes high cost treatments for patients with rare diseases. In contrast with prevailing methods, there is increasing evidence that the public would prefer a fairness-focused framework in which the service was removed from centre stage and replaced by the patient. However methods for achieving fairness are ad hoc and under-developed.
The article initially reviews the theory of economic evaluation and argues that its focus upon individual utility and efficiency as defined by the theory omits potentially important social values. Some empirical evidence relating to population values is presented and four studies by the first author are reviewed. These indicate that when people adopt the social perspective of a citizen they have a preference for sharing the health budget in a way which does not exclude patients who require services that are not cost effective, such as orphan medicinal products (OMP’s) and treatments for patients with ultra-rare diseases (URD’s).
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Is the National Institute for Health and Care Excellence (NICE) in England more ‘innovation-friendly’ than the Federal Joint Committee (G-BA) in Germany?
Expert Rev Pharmacoecon Outcomes Res (2018). DOI: [Epub ahead of print].
Our study explores whether, and how, different methodological choices are associated with different health technology assessment (HTA) outcomes. We focus on the Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) in Germany and the National Institute for Health and Care Excellence (NICE) in England. Both agencies may be considered as exemplars for the application of the principles of evidence-based medicine and the logic of cost-effectiveness, respectively. We extracted data from all publically available G-BA appraisals until April 2015, as well as all NICE single technology appraisals completed during this period. We compared HTA results for matched condition-intervention pairs by G-BA and NICE, and explored other factors including therapeutic area, clinical effectiveness and cost-effectiveness. NICE issued guidance for 88 technologies (125 subgroups) and recommended 67/88 technologies (99/125 subgroups). G-BA completed 105 appraisals (226 subgroups) and determined additional benefit for 64/105 appraisals (90/226 subgroups). We identified 37 matched pairs; for 24/37 drugs, evaluations diverged. NICE recommended 78% (29/37) of technologies appraised, whereas G-BA confirmed additional benefit for 57% (21/37) only (p < 0.05). NICE evaluates new drugs more favorably than G-BA. However, our analysis suggests differences by therapeutic area. Results indicate that different methods are associated with systematic differences in HTA outcomes.
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Comparing the German Translation of the ICECAP-A Capability Wellbeing Measure to the Original English Version: Psychometric Properties across Healthy Samples and Seven Health Condition Groups?
Applied Res Qual Life (2018). DOI: 10.1007/s11482-018-9681-5 [Epub ahead of print].
As the cross-cultural use of outcome measures grows, it is important to determine whether these instruments are: appropriate for use in other settings, translated accurately, and perform in a similar manner to their original tools. This research aimed to compare the validity of the German translation of the ICECAP-A to the original English version of the instrument, across healthy adults and seven health condition groups (arthritis, asthma, cancer, depression, diabetes, hearing loss and heart disease). Data were analysed from a cross-cultural study, which recruited participants through online panels in 2012. Data were analysed on capability wellbeing (ICECAP-A), health-related quality of life (EQ-5D-5L and SF-6D), satisfaction with life (SWLS), and a series of other condition-specific outcome measures. The ICECAP-A was assessed for internal consistency, convergent validity and construct validity. 2501 individuals were included in the analysis. The ICECAP-A demonstrated good internal consistency within Germany and the UK population, and across all seven health condition sub-groups (α = .74–.86). In both countries, ICECAP-A scores were significantly correlated with SWLS, SF-6D and EQ-5D-5L scores for healthy participants and health condition groups (r = .35–.77). Finally, experiencing one of the seven health conditions (compared to being healthy) was significantly associated with lower levels of capability wellbeing in the German and UK samples (construct validity). The German translation of the ICECAP-A yielded valid and reliable data, in both healthy respondents and the seven health condition groups. Further work could be undertaken to develop a German specific value-set for the ICECAP-A.
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Determining Value in Health Technology Assessment: Stay the Course or Tack Away?
PharmacoEconomics (2018). DOI: 10.1007/s40273-018-0742-2 [Epub ahead of print].
The economic evaluation of new health technologies to assess whether the value of the expected health benefits warrants the proposed additional costs has become an essential step in making novel interventions available to patients. This assessment of value is problematic because there exists no natural means to measure it. One approach is to assume that society wishes to maximize aggregate health, measured in terms of quality-adjusted life-years (QALYs). Commonly, a single ‘costeffectiveness’ threshold is used to gauge whether the intervention is sufficiently efficient in doing so. This approach has come under fire for failing to account for societal values that favor treating more severe illness and ensuring equal access to resources, regardless of pre-existing conditions or capacity to benefit. Alternatives involving expansion of the measure of benefit or adjusting the threshold have been proposed and some have advocated tacking away from the cost per QALY entirely to implement therapeutic area-specific efficiency frontiers, multicriteria decision analysis or other approaches that keep the dimensions of benefit distinct and value them separately. In this paper, each of these alternative courses is considered, based on the experiences of the authors, with a view to clarifying their implications.
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The health economics of cancer screening in Germany: Which population-based interventions are cost-effective?
Bundesgesundheitsbl (2018); 61:1559–1568 [German Article].
Only a small proportion of German health expenditure is spent on prevention and early detection (screening). The rationale for screening is to identify persons with diseaseprecursors or at the early stage of diseases when they are still asymptomatic, in order to decrease disease-specificmorbidity and mortality. In Germany, the economic evidence is one of the evaluation criteria for screening measures, which, among other things, takes into account the additional cost per additional case detected or per case-related event avoided, as well as a cost-benefit balance. For this purpose, cost-effectiveness analyses, which report marginal or incremental cost effectiveness ratios, comparing a measure with its appropriate alternatives, may be a useful tool. Their application requires a defensible benchmark (threshold) for cost effectiveness and a supplementary analysis of the necessary infrastructure and the budgetary impact associated with program implementation. Also (albeit not only) because of the usually long time required to observe the clinical outcomes of a screening measure, the economic evaluation of such programs regularly involves the application of decision analytic simulationmodels. With regard to cancer screening programs, the available models indicate an excellent costbenefit ratio for the fecal occult blood test and colonoscopy for colorectal cancer screening and, similarly, for the use of mammography for breast cancer screening. On the other hand, the economic evidence in favor of lowdose computed tomography for lung cancer screening does not yet appear sufficiently strong, and the currently available health economic evidence does not support the use of PSA testing for prostate screening.
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The cost incurred by victims of bullying from a societal perspective: estimates based on a German online survey of adolescents.
Eur Child Adolesc Psychiatry (2018). DOI: 10.1007/s00787-018-1224-y [Epub ahead of print].
Being a victim of bullying is linked to various social, emotional and behavioral problems potentially leading to a reduced quality of life. Furthermore, victims of bullying may cause extensive costs for society, for example by an above-average need for healthcare services. The present study was designed to quantify the costs and the loss of quality of life attributable to bullying by comparing victims with a control group of non-bullied students. A cross-sectional sample of 1293 adolescents (mean age 14.07, SD = 1.36) and their parents reported on bullying victimization, quality of life (adolescents’ self-report), and annual direct (medical and non-medical) as well as indirect costs (parents’ self-report) from a societal perspective (all expressed in €, year 2014 and 2015). For frequent (20.6% of our sample; costs: €8461.80 p.a.) but not occasional (13.3%; costs: €2850.06) bullying, victimization was associated with significantly higher costs compared to non-bullied adolescents (costs: €3138.00; annual difference between frequently bullied students and controls: €5323.01 p.a.; p = 0.008). Cost drivers included increased direct medical costs, but mostly indirect costs caused by productivity losses of the parents. Self-reported quality of life of frequent victims was considerably reduced (T = − 10.96; p < 0.001); also occasional bullying showed significantly reduced values in global quality of life (T = − 5.73; p < 0.001). The present findings demonstrate that frequent bullying is associated with substantial cost to society and reduced quality of life of victims. This observation underscores the need for effective school-based bullying prevention and suggests a high potential of effective programs to be cost effective as well.
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Kosten der Onkologie in Deutschland [The cost of cancer in Germany].
Forum (2018). https://doi.org/10.1007/s12312-018-0481-5 [Epub ahead of print].
Cancer is the second leading cause of mortality in Germany and believed to be associated with high costs. Health economic analyses indicate that cancer indeed accounts for almost one fifth of the total burden of disease in Germany, as measured by means of disability-adjusted life years (DALYs). This implies extraordinarily high intangible costs due to cancer. As to direct medical costs, German cost of illness data were last compiled by the Federal Statistical Office for calendar year 2015. The official statistics suggest that cancer accounts for 19.9 billion Euro or about six percent of total German health care expenditures (343 billion Euro in 2015), a finding corresponding well to the situation in other European states. The share of total spending dedicated to cancer medicine remained largely stable since 2004; the observation does not lend support to the hypothesis of recently escalating health care costs due to cancer care. With regard to indirect costs, recent estimates suggest that the productivity loss attributable to cancer in Germany amounted to 18.5 billion Euro (2015) – a dimension which is comparable to the direct cost. In the future, health economists should pay attention to the – to date relatively moderate – extra cost of highly specialized comprehensive cancer care centers in Germany and to the significant rise of cancer drug costs. Furthermore, there is a need to better understand the cost of cancer from the perspective of patients and their families.
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Projecting Pharmaceutical Expenditure in EU5 to 2021: Adjusting for the Impact of Discounts and Rebates.
Appl Health Econ Health Policy (2018): DOI: 10.1007/s40258-018-0419-1 [Epub ahead of print].
Within (European) healthcare systems, the predominant goal for pharmaceutical expenditure is cost containment. This is due to a general belief among healthcare policy makers that pharmaceutical expenditure—driven by high prices—will be unsustainable unless further reforms are enacted.
The aim of this paper is to provide more realistic expectations of pharmaceutical expenditure for all key stakeholder groups by estimating pharmaceutical expenditure at ‘net’ prices. We also aim to estimate any gaps developing between list and net pharmaceutical expenditure for the EU5 countries (i.e. France, Germany, Italy, Spain, and the UK).
We adjusted an established forecast of pharmaceutical expenditure for the EU5 countries, from 2017 to 2021, by reflecting discounts and rebates not previously considered, i.e. we moved from ‘list’ to ‘net’ prices, as far as data were available.
We found an increasing divergence between expenditure measured at list and net prices. When the forecasts for the five countries were aggregated, the EU5 (unweighted) average historical growth (2010–2016) rate fell from 3.4% compound annual growth rate at list to 2.5% at net. For the forecast, the net growth rate was estimated at 1.5 versus 2.9% at list.
Our results suggest that future growth in pharmaceutical expenditure in Europe is likely to be (1) lower than previously understood from forecasts based on list prices and (2) below predicted healthcare expenditure growth in Europe and in line with long-term economic growth rates. For policy makers concerned about the sustainability of pharmaceutical expenditure, this study may provide some comfort, in that the perceived problem is not as large as expected.
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Budgetary Impact and Cost Drivers for Rare and Ultrarare Diseases.
Value in Health (2017): DOI 10.1016/j.jval.2017.10.015.
To review recent studies reporting health care expenditures (budgetary impact) for orphan medicinal products (OMPs) in Europe and to contribute to our understanding of the cost drivers of nononcological OMPs by means of an empirical analysis in Germany. A systematic search for relevant studies on rare diseases was conducted in PubMed and Embase (until December 2016). In addition, annual treatment costs of nononcological OMPs in Germany were analyzed with respect to five explanatory variables: total prevalence of disease, prevalence with added benefit, availability of alternative treatments for the same indication, extent/probability of treatment benefit, and evidence for a treatment effect on mortality. A total of nine studies with specific estimates of the budget impact of OMPs for a total of 11 countries were identified; one study addressed specifically ultrarare diseases. Annual per-capita spending for OMPs ranges from €1.32 in Latvia to €16 in France. Per-patient annual treatment costs vary between €27,811 and €1,647,627 in Germany. On the basis of the German data set, the regression analysis shows that log prevalence has a significant inverse relationship with log annual treatment cost. In this model, doubling the prevalence leads to a 43% decrease in annual treatment cost. Despite per-patient annual treatment costs ranging up to several hundreds of thousands of euros for some OMPs, per-capita spending for OMPs is relatively small. In this study an inverse relationship between prevalence and annual treatment costs was found.
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Woran bemisst sich Effizienz im Gesundheitswesen? Zur Klärung fachwissenschaftlicher Begriffe und Kriterien.
Amos International 11 (2017) 1: 22-31.
The word efficiency has a positive connotation. Those who are not familiar with economic theory often do not realize that the technical meaning of the term differs markedly from common usage. This may cause misunderstandings and problematic consequences when economists make influential statements on efficiency and inefficiency in the health care system. An instructive case in point is the debate on efficient conduct and the so-called “rule of rescue”. To understand the underlying issues it is necessary to know about the expectations towards and objectives of health care systems. For “efficiency” can by definition be an instrumental objective only, secondary to the (primary) effectiveness criterion.
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Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases.
Orphanet Journal of Rare Diseases 12 (50), 2017: DOI 10.1186/s13023-017-0601-9.
The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field.
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Conventional Health Economic Evaluation Fails to Capture Social Value of Interventions for Rare and Ultra-Rare Disorders.
Drug Information Association (DIA) Global Forum, October 2016: 13-14.
Orphan drug legislation provided for a broad range of incentives for research and development (R&D) into interventions for the prevention and treatment of rare and ultra-rare disorders. These measures have contributed to a stream of new medications, some of which rank among “the most expensive drugs in the world”. In times of economic austerity, health care policy makers need to address whether these interventions offer “value for money”. Decision makers struggle with the absence of accepted validated tools how to determine – and how to quantify – the social value of such interventions.
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Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement.
Journal of Market Access & Health Policy 4 (2016): 33039; 1-9.
In order to critically appraise the problems posed by the systematic valuation of interventions for ultra-rare disorders (URDs), an international group of clinical and health economic experts was convened in conjunction with the Annual European ISPOR Congress in Berlin, Germany, in November 2012. Following this meeting and during subsequent deliberations, the group achieved a consensus on the specific challenges and potential ways forward. The group concluded that the complexities of research and development for new treatments for URDs may require conditional approval and reimbursement policies, such as managed entry schemes and coverage with evidence development agreements, but should not use as justification surrogate end point improvement only. As a prerequisite for value assessment, the demonstration of a minimum significant clinical benefit should be expected within a reasonable time frame. As to the health economic evaluation of interventions for URDs, the currently prevailing logic of cost-effectiveness (using benchmarks for the maximum allowable incremental cost per quality-adjusted life year gained) was considered deficient as it does not capture well-established social preferences regarding health care resource allocation.
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Comparison of Methylphenidate and Psychotherapy in Adult ADHD Study (COMPAS) Consortium: Effects of Group Psychotherapy, Individual Counseling, Methylphenidate, and Placebo in the Treatment of Adult Attention-Deficit/Hyperactivity Disorder: A Randomized Clinical Trial.
JAMA Psychiatry, 72 (12), 2015: 1199-1210.
Attention-deficit/hyperactivity disorder (ADHD) is a neurodevelopmental disorder with high prevalence in adulthood. A prospective, multicenter, randomized clinical trial of 18- to 58-year-old outpatients with ADHD assessed the effects group psychotherapy, individual counseling, methylphenidate, and placebo in the treatment of adult ADHD. Patients received either methylphenidate or placebo for 1 year. The primary outcome was the change in the ADHD Index of the Conners Adult ADHD Rating Scale from baseline to the end of the 3-month intensive treatment (blinded observer ratings). Secondary outcomes included ADHD ratings after 1 year, blinded observer ratings using the Clinical Global Impression Scale, and self-ratings of depression. 433 patients were centrally randomized, and 419 were analyzed as randomized. After 3 months, the ADHD Index all-group baseline mean of 20.6 improved to adjusted means of 17.6 for GPT and 16.5 for CM, with no significant difference between groups. Methylphenidate (adjusted mean, 16.2) was superior to placebo (adjusted mean, 17.9) (difference, −1.7; 97.5%CI, −3.0 to −0.4; P = .003). After 1 year, treatment effects remained essentially stable. Descriptive analyses showed that methylphenidate was superior to placebo in patients assigned to GPT (difference, −1.7; 95%CI, −3.2 to −0.1; P = .04) or CM (difference, −1.7; 95%CI, −3.3 to −0.2; P = .03). Thus, a highly structured group intervention did not outperform individual CM with regard to the primary outcome. Psychological interventions resulted in better outcomes during a 1-year period when combined with methylphenidate as compared with placebo.
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Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe.
Expert Review of Pharmacoeconomics & Outcomes Research, 15 (1), 2015: 171-179.
Ultra-orphan diseases (UODs) have been defined by a prevalence of less than 1 per 50,000 persons. However, little is known about budget impact of ultra-orphan drugs. For analysis, the budget impact analysis (BIA) had a time horizon of 10 years (2012–2021) and a pan-European payer’s perspective, based on prevalence data for UODs for which patented drugs are available and/or for which drugs are in clinical development. A total of 18 drugs under patent protection or orphan drug designation for non-oncological UODs were identified. Furthermore, 29 ultra-orphan drugs for non-oncological diseases under development that have the potential of reaching the market by 2021 were found. Total budget impact over 10 years was estimated to be e15,660 and e4965 million for approved and pipeline ultra-orphan drugs, respectively (total: e20,625 million). Conclusion: The analysis does not support concerns regarding an uncontrolled growth in expenditures for drugs for UODs.
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