Use Of Cost-effectiveness Studies Research Articles

The Complex Cancer Care Coverage Environment - What is the Role of Legislation? A Case Study from Massachusetts.

Over the past decades, anti-cancer treatments have evolved rapidly from cytotoxic chemotherapies to targeted therapies including oral targeted medications and injectable immuno-oncology and cell therapies. New anti-cancer medications come to markets at increasingly high prices, and health insurance coverage is crucial for patient access to these therapies. State laws are intended to facilitate insurance coverage of anti-cancer therapies.Using Massachusetts as a case study, we identified five current cancer coverage state laws and interviewed experts on their perceptions of the relevance of the laws and how well they meet the current needs of cancer care given rapid changes in therapies. Interviewees emphasized that cancer therapies, as compared to many other therapeutic areas, are unique because insurance legislation targets their coverage. They identified the oral chemotherapy parity law as contributing to increasing treatment costs in commercial insurance. For commercial insurers, coverage mandates combined with the realities of new cancer medications - including high prices and often limited evidence of efficacy at approval - compound a difficult situation. Respondents recommended policy approaches to address this challenging coverage environment, including the implementation of closed formularies, the use of cost-effectiveness studies to guide coverage decisions, and the application of value-based pricing concepts. Given the evolution of cancer therapeutics, it may be time to evaluate the benefits and challenges of cancer coverage mandates.

Mapping PROMIS Global Health Items to EuroQol (EQ-5D) Utility Scores Using Linear and Equipercentile Equating.

Mapping Patient-Reported Outcomes Measurement Information System-Global Health (PROMIS-GH) to EuroQol 5-dimension, three-level version (EQ-5D-3L) provides a utility score for use in quality-of-life and cost-effectiveness analyses. In 2009, Revicki et al. mapped the PROMIS-GH items to EQ-5D-3L utilities using linear regression (REVReg). More recently, regression was shown to be ill-suited for mapping to preference-based measures due to regression to the mean. Linear and equipercentile equating are alternative mapping methods that avoid the issue of regression to the mean. Another limitation of the prior models is that ordinal predictors were treated as continuous. Using data collected from the PROMIS Wave 1 sample, we refit REVReg, treating the PROMIS-GH items as categorical variables (CATReg). We applied linear and equipercentile equating to the REVReg model (REVLE, REVequip) and the CATReg model (CATLE, CATequip). We validated and compared the predictive accuracy of these models in a large sample of neurological patients at a single tertiary-care hospital. In the neurological disease patient sample, CATLE produced the strongest correlations between estimated and observed EQ-5D-3L scores and had the lowest mean squared error. The CATequip model had the lowest mean absolute error and had estimated scores that best matched the overall distribution of observed scores. Using linear and equipercentile equating, we created new models mapping PROMIS-GH items to EQ-5D-3L utility scores. EQ-5D-3L utility scores can be more accurately estimated using our models for use in cost-effectiveness studies or studies examining overall health-related quality of life.

What Are the Strength of Recommendations and Methodologic Reporting in Health Economic Studies in Orthopaedic Surgery?

Cost-effectiveness research is an increasingly used tool in evaluating treatments in orthopaedic surgery. Without high-quality primary-source data, the results of a cost-effectiveness study are either unreliable or heavily dependent on sensitivity analyses of the findings from the source studies. However, to our knowledge, the strength of recommendations provided by these studies in orthopaedics has not been studied. We asked: (1) What are the strengths of recommendations in recent orthopaedic cost-effectiveness studies? (2) What are the reasons authors cite for weak recommendations? (3) What are the methodologic reporting practices used by these studies? The titles of all articles published in six different orthopaedic journals from January 1, 2004, through April 1, 2014, were scanned for original health economics studies comparing two different types of treatment or intervention. The full texts of included studies were reviewed to determine the strength of recommendations determined subjectively by our study team, with studies providing equivocal conclusions stemming from a lack or uncertainty surrounding key primary data classified as weak and those with definitive conclusions not lacking in high-quality primary data classified as strong. The reasons underlying a weak designation were noted, and methodologic practices reported in each of the studies were examined using a validated instrument. A total of 79 articles met our prespecified inclusion criteria and were evaluated in depth. Of the articles included, 50 (63%) provided strong recommendations, whereas 29 (37%) provided weak recommendations. Of the 29 studies, clinical outcomes data were cited in 26 references as being insufficient to provide definitive conclusions, whereas cost and utility data were cited in 13 and seven articles, respectively. Methodologic reporting practices varied greatly, with mixed adherence to framing, costs, and results reporting. The framing variables included clearly defined intervention, adequate description of a comparator, study perspective clearly stated, and reported discount rate for future costs and quality-adjusted life years. Reporting costs variables included economic data collected alongside a clinical trial or another primary source and clear statement of the year of monetary units. Finally, results reporting included whether a sensitivity analysis was performed. Given that a considerable portion of orthopaedic cost-effectiveness studies provide weak recommendations and that methodologic reporting practices varied greatly among strong and weak studies, we believe that clinicians should exercise great caution when considering the conclusions of cost-effectiveness studies. Future research could assess the effect of such cost-effectiveness studies in clinical practice, and whether the strength of recommendations of a study's conclusions has any effect on practice patterns. Given the increasing use of cost-effectiveness studies in orthopaedic surgery, understanding the quality of these studies and the reasons that limit the ability of studies to provide more definitive recommendations is critical. Highlighting the heterogeneity of methodologic reporting practices will aid clinicians in interpreting the conclusions of cost-effectiveness studies and improve future research efforts.

Estimating EQ-5D Weights from other Trial Outcome Measures for Use in Cost-Effectiveness Studies: An Example in Patients with Frozen Shoulder

Background Health-related quality of life (HRQoL) weights, such as EQ-5D, needed for conducting economic evaluations in frozen shoulder populations are currently lacking. The present study examines the feasibility of using mapping methodology to estimate HRQoL, obtain quality-adjusted life-years (QALYs) and undertake a cost-effectiveness analysis to help inform decision-making. Methods Patient-level data from a randomized trial in a similar population were used to undertake a mapping exercise evaluating a variety of regression modelling techniques. The Short-Form-36 component summary scores and pain visual analogue scale scores were mapped to EQ-5D. The best fit model was selected and the predicted weights were used for estimating QALYs and cost-effectiveness results. Results The selected model explained 41% ( R2) of the variation in EQ-5D weights and the estimates for interventions ranged from 0.5025 to 0.6619 at baseline and from 0.6903 to 0.7600 at 3 months. The QALYs (gained) ranged from 0.1518 to 0.1715 and the estimates of cost-effectiveness were up to £400 per QALY for steroid injection compared to physical therapy with steroid injection. Discussion Few studies in frozen shoulder populations include HRQoL weights. We have shown that mapping can potentially take advantage of datasets in similar populations and estimate HRQoL outcomes and subsequently cost-effectiveness. The results may be uncertain but can be helpful in informing the discussion around cost-effectiveness and future areas of research.

Comment on: Ethical dilemmas in antibiotic treatment

Sir, Leibovici et al. are addressing very important issues regarding the ethical dilemma in antimicrobial stewardship, such as the central question: whether we could and should value benefits in the distant future for unidentified patients as more important than benefits in the near future for an identified patient. However, some aspects in the argumentation by Leibovici et al. are problematic: (i) overlapping use of two ethical frameworks, i.e. utilitarianism and principlism; (ii) justifying the use of cost-effectiveness studies by only one ethical framework; and (iii) other ethical solutions to deal with this dilemma exist. (i) Generally, the use of different theoretical frameworks in approaching an ethical dilemma is to be encouraged because different frameworks may result in different solutions. But, importantly, one has to resolve inconsistencies that arise from using different frameworks. The two ethical approaches used by Leibovici et al. are principlism and utilitarianism. Principlism is based on the four moral principles of autonomy, beneficence, non-maleficence and justice. In contrast, the single goal of utilitarianism is to maximize ‘total utility’. The weakness in this article lies in emphasizing cost-effectiveness studies as a tool justified solely by utilitarianism while simultaneously relying on a principlistic framework. However, these two frameworks are mutually exclusive, because utilitarianism only allows one moral principle: maximizing total utility. On the other hand, principlism does not allow one of its four principles to become absolute. Thus, if cost-effectiveness studies are used as a normative instrument justified by utilitarianism, this conflicts with this tenet of principlism. When using cost-effectiveness as an operationalization of utilitarianism, respect for patient autonomy and justice is no longer a concern, as long as violations of patient autonomy and justice do not lead to a heavy loss of overall utility. The reason for this is that, in contrast to principlism, respect for autonomy and justice have no intrinsic value in utilitarianism. There are multiple theories of utilitarianism, however, and some modern versions of preference utilitarianism, e.g. the theory of Gesang, take secondary preferences, such as moral preferences, into account. If, for example, many persons wish to live in a healthcare system that always respects patient autonomy and demands informed consent, the current practice of antimicrobial stewardship would lead to a severe loss of utility, rendering the practice immoral from a utilitarian point of view. Thus, the concept of utility in utilitarianism is broader than just medical benefit. (ii) Leibovici et al. note that ‘The inclusion of costeffectiveness together with the most quoted principles of bioethics looks strange.’ While it is easier to argue for costeffectiveness within a utilitarian framework, cost-effectiveness as a means to solve an ethical dilemma can be supported by principlism as well. In fact, Beauchamp and Childress discuss cost-effectiveness as part of the principle of beneficence. What they point out, though, is that such quantitative methods may only function as auxiliary means in ethical decision-making and must not become a surrogate for decision-making. The important difference is that all four principles have to be specified and balanced. Moreover, if using principlism, the exception to the rule that Leibovici et al. introduced in the case of lifethreatening scenarios would not have to be an exception. It would be just a specification of the principle of non-maleficence, which very likely would have gained enough weight in principlistic balancing to be morally preferred to all other courses of action. So, the same result might have been possible in the argumentation without mixing conflicting ethical approaches. (iii) One could further ask if there is no possibility of reconciling the duties of non-maleficence or beneficence for unidentified patients with the duty of respect for autonomy for the actual patient. We suggest three considerations. First, analogously to tacit consent when being treated in a teaching hospital (possibly by junior doctors and residents) someone treated with antibiotics tacitly consents to non-maximum empirical treatment, as long as there is no life-threatening situation. Secondly, informed patients might accept non-maximal empirical coverage because of ethical reasoning. Thirdly, one could consider that nowadays, respect for patient autonomy already does not include the duty to do everything that a patient wishes. Specific broad-spectrum antibiotics might, as Leibovici et al. suggest, be regarded as a ‘scarce’ resource when being used for empirical treatment, and thus, these antibiotics would represent a treatment restricted to those needing it most, e.g. in transplantation. In summary, restricting universal coverage in empirical treatment can be justified within various ethical frameworks. Cost-effectiveness analysis as a measure to solve the ethical dilemma can be used in a utilitarianistic or principlistic ethical framework. In order to assess the dilemma in the patient– doctor relationship, empirical studies should be conducted addressing what patients would accept in terms of non-maximal Letters to the Editor

Development of the EXACT-U: A Preference-Based Measure to Report COPD Exacerbation Utilities

Background The exacerbations of chronic obstructive pulmonary disease tool (EXACT) is a condition-specific daily diary recently developed to evaluate the frequency, severity, and duration of chronic obstructive pulmonary disease (COPD) exacerbations. A preference-based algorithm for the EXACT would allow utilities to be reported from patients during an exacerbation when EQ-5D data are not available. Objective To develop the exacerbations of chronic obstructive pulmonary disease tool-utility (EXACT-U), a condition-specific preference-based measure to report utilities from the EXACT for use in cost-effectiveness studies. Methods Five items with three to five levels comprise the EXACT-U. Two groups of health states and respondents were constructed to allow for model development (Development group) and predictive validity testing (Validation group) using independent samples. Members of the UK general public each valued 11 randomized health states using time trade-offs (TTOs) scaled from full health/dead with 10-year durations. Regression models estimated from the Development group using individual data, mean data, and panel designs. Models assessed by number of inconsistent coefficients estimated and R 2 and tested against observed utilities from the Validation group using mean absolute error (MAE) and root mean squared error (RMSE). Results A total of 55 health states, including the best and worst states, were valued in TTO interviews conducted with 400 respondents. Ten models were developed. The final preferred model contained no logical inconsistencies and found MAE = 0.04 and RMSE = 0.05 with a predicted utility range from 0.09 to 0.95. Conclusions The EXACT-U is a condition-specific preference-based measure with strong predictive validity to report daily utilities during an exacerbation.

Evaluation of the Health Utilities Index Mark-3 in Heart Failure

Background The purpose of this study was to evaluate the reliability, validity, and responsiveness to change of the Health Utilities Index Mark-3 (HUI-3) in heart failure (HF) for use in cost-effectiveness studies. Methods and Results Two hundred eleven patients with HF recruited from outpatient clinics were enrolled; 165 completed the 26-week study. Patients completed 4 health-related quality of life questionnaires (baseline and 4, 8, and 26 weeks), including the HUI-3, the Medical Outcomes Study Short-form 12 (SF-12), the Minnesota Living with Heart Failure Questionnaire (LHFQ), and the Chronic Heart Failure Questionnaire (CHQ). The HUI-3 indicated moderate or fair health-related quality of life overall; the attributes most impaired were pain, ambulation, cognition, and emotion. Internal consistency reliability (Cronbach’s alpha = 0.51) was low and test-retest reliability (intraclass correlation coefficient = 0.68) was adequate. The HUI-3 total score was significantly associated with the SF-12, LHFQ, and CHQ total scores. It discriminated among patients with varying New York Heart Association class ( P < .001) and varying perceived health ( P < .001). The HUI-3 was less responsive to perceived change in health condition than the LHFQ or the CHQ. Conclusions The HUI-3 demonstrated satisfactory reliability and validity in this sample supporting its use in cost-effectiveness studies.

Hospital pharmacy decisions, cost containment, and the use of cost-effectiveness analysis

The key hypothesis of the study was that hospital pharmacies under the pressure of managed care would be more likely to adopt process innovations to assure less costly and more cost-effective provision of care. We conducted a survey of 103 hospitals and analyzed secondary data on cost and staffing. Compared to the size of the reduction in length of stay, changes in the way that a day of care is delivered appear to be minor, even in areas with substantial managed care share. The vast majority of hospitals surveyed had implemented some form of therapeutic interchange and generic substitution. Most hospitals used some drug utilization guidelines, but as of mid 1995 these were not yet important management tools for hospital pharmacies. To our knowledge, ours was the first survey to investigate the link between hospital formularies and use of cost-effectiveness analysis. At most cost-effectiveness was a minor tool in pharmaceutical decision making in hospitals at present. We could determine no differences in use of such analyses by managed care market share in the hospital's market share. One impediment to the use of cost-effectiveness studies was the lack of timeliness of studies. Other stated reasons for not using cost-effectiveness analysis more often were: lack of information on hospitalized patients and hence on the potential cost offsets accruing to the hospital; lack of independent sponsorship, and inadequate expertise in economic evaluation.