Budget Impact Analysis—Principles of Good Practice: Report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force

BackgroundBudget impact analyses (BIAs) are an essential part of a comprehensive economic assessment of a health care intervention and are increasingly required by reimbursement authorities as part of a listing or reimbursement submission.ObjectivesThe objective of this report was to present updated guidance on methods for those undertaking such analyses or for those reviewing the results of such analyses. This update was needed, in part, because of developments in BIA methods as well as a growing interest, particularly in emerging markets, in matters related to affordability and population health impacts of health care interventions.MethodsThe Task Force was approved by the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council and appointed by its Board of Directors. Members were experienced developers or users of BIAs; worked in academia and industry and as advisors to governments; and came from several countries in North America and South America, Oceania, Asia, and Europe. The Task Force solicited comments on the drafts from a core group of external reviewers and, more broadly, from the membership of the International Society for Pharmacoeconomics and Outcomes Research.ResultsThe Task Force recommends that the design of a BIA for a new health care intervention should take into account relevant features of the health care system, possible access restrictions, the anticipated uptake of the new intervention, and the use and effects of the current and new interventions. The key elements of a BIA include estimating the size of the eligible population, the current mix of treatments and the expected mix after the introduction of the new intervention, the cost of the treatment mixes, and any changes expected in condition-related costs. Where possible, the BIA calculations should be performed by using a simple cost calculator approach because of its ease of use for budget holders. In instances, however, in which the changes in eligible population size, disease severity mix, or treatment patterns cannot be credibly captured by using the cost calculator approach, a cohort or patient-level condition-specific model may be used to estimate the budget impact of the new intervention, accounting appropriately for those entering and leaving the eligible population over time. In either case, the BIA should use data that reflect values specific to a particular decision maker’s population. Sensitivity analysis should be of alternative scenarios chosen from the perspective of the decision maker. The validation of the model should include at least face validity with decision makers and verification of the calculations. Data sources for the BIA should include published clinical trial estimates and comparator studies for the efficacy and safety of the current and new interventions as well as the decision maker’s own population for the other parameter estimates, where possible. Other data sources include the use of published data, well-recognized local or national statistical information, and, in special circumstances, expert opinion. Reporting of the BIA should provide detailed information about the input parameter values and calculations at a level of detail that would allow another modeler to replicate the analysis. The outcomes of the BIA should be presented in the format of interest to health care decision makers. In a computer program, options should be provided for different categories of costs to be included or excluded from the analysis.ConclusionsWe recommend a framework for the BIA, provide guidance on the acquisition and use of data, and offer a common reporting format that will promote standardization and transparency. Adherence to these good research practice principles would not necessarily supersede jurisdiction-specific BIA guidelines but may support and enhance local recommendations or serve as a starting point for payers wishing to promulgate methodology guidelines.     

Using “Big Data” in the Cost-Effectiveness Analysis of Next-Generation Sequencing Technologies: Challenges and Potential Solutions

Next-generation sequencing (NGS) is considered to be a prominent example of “big data” because of the quantity and complexity of data it produces and because it presents an opportunity to use powerful information sources that could reduce clinical and health economic uncertainty at a patient level. One obstacle to translating NGS into routine health care has been a lack of clinical trials evaluating NGS technologies, which could be used to populate cost-effectiveness analyses (CEAs). A key question is whether big data can be used to partially support CEAs of NGS. This question has been brought into sharp focus with the creation of large national sequencing initiatives. In this article we summarize the main methodological and practical challenges of using big data as an input into CEAs of NGS. Our focus is on the challenges of using large observational datasets and cohort studies and linking these data to the genomic information obtained from NGS, as is being pursued in the conduct of large genomic sequencing initiatives. We propose potential solutions to these key challenges. We conclude that the use of genomic big data to support and inform CEAs of NGS technologies holds great promise. Nevertheless, health economists face substantial challenges when using these data and must be cognizant of them before big data can be confidently used to produce evidence on the cost-effectiveness of NGS.     

Reproductive Factors and Risk of Thyroid Cancer in Women: An Analysis in the Nurses’ Health Study II

BackgroundThe incidence of thyroid cancer in women is increasing at an alarming rate, with greatest risk in the reproductive years. Establishing relationships of hormonally related reproductive factors with thyroid cancer has been difficult. We aimed to elucidate potential risk factors for thyroid cancer in a large cohort of women.MethodsAmong 116,228 women in the Nurses’ Health Study II followed from 1989 to 2013, 620 cases of thyroid cancer were identified. We examined reproductive and hormone-related factors, including age at menarche, age at menopause, parity, oral contraceptive use, and postmenopausal hormone therapy use. Pregnancy, reproductive years, and months of breastfeeding were used as surrogate markers for exposure to endogenous reproductive hormones. We used multivariable Cox models to calculate relative risks and 95% confidence intervals for the associations between these factors and risk of thyroid cancer.ResultsNumber of reproductive years of 41 years or more was associated with more than double the risk of thyroid cancer compared with 30 years or fewer (relative risk, 2.20; 95% confidence interval, 1.19–4.06). The other variables analyzed (parity number, months of breastfeeding, age at menarche, menopausal status, and postmenopausal hormone therapy) were not associated with the risk of thyroid cancer. Women who entered menopause at age 45 years or older had a higher risk of thyroid cancer compared with women who entered menopause at a younger age. This result did not reach statistical significance; however, there was a linear trend between later age at menopause and increased risk of thyroid cancer (p_trend = .009).ConclusionsThis study used a unique large, longitudinal dataset to assess thyroid cancer risk factors and potential confounders over an extended time frame. Our key finding suggests increased risk of thyroid cancer may be associated with a variety of indicators of longer reproductive years. The Nurses’ Health Study II has provided new insights into the hormonal risks associated with thyroid cancer.     

How Do Insured Perceive Their Financial Security in the Event of Illness?—A Panel Data Analysis for Germany

ObjectiveThere is a lack of research regarding the subjective perception of financial security in the event of illness of insured persons. Therefore, the aim of our study was to analyze the subjective perception of financial security in the event of illness in the German setting over time and to identify major determinants of that perception.MethodsWe applied a probit-adapted ordinary least squares estimation procedure including fixed effects to a balanced data set from the German Socio-Economic Panel. After correcting our data set, we included approximately 23,500 observations in our analyses.ResultsWe show that higher income and the existence of private health insurance have a positive and significant impact on the perception of financial security. Furthermore, private supplementary health insurance has a positive and significant effect on this perception; however, this is solely true for policies that cover special features during hospital stays. Experience with the health care system is also positively related to the individual's perception. Finally, our regression results illustrate that the overall perception is declining over time.ConclusionsThe results indicate that political decision makers are facing challenges regarding the declining subjective perception in the German health care system. Because of the positive correlation between experience and subjective perception, it can be assumed that the health care system and especially statutory health insurance are better than their presentation in the media. Hence, there is a problem of communication and information, and political decision makers face challenges in presenting the system objectively and handling the media in a proper way.     

Ciguatera Fish Poisoning and Climate Change: Analysis of National Poison Center Data in the United States, 2001–2011

Background: Warm sea surface temperatures (SSTs) are positively related to incidence of ciguatera fish poisoning (CFP). Increased severe storm frequency may create more habitat for ciguatoxic organisms. Although climate change could expand the endemic range of CFP, the relationship between CFP incidence and specific environmental conditions is unknown.Objectives: We estimated associations between monthly CFP incidence in the contiguous United States and SST and storm frequency in the Caribbean basin.Methods: We obtained information on 1,102 CFP-related calls to U.S. poison control centers during 2001–2011 from the National Poison Data System. We performed a time-series analysis using Poisson regression to relate monthly CFP call incidence to SST and tropical storms. We investigated associations across a range of plausible lag structures.Results: Results showed associations between monthly CFP calls and both warmer SSTs and increased tropical storm frequency. The SST variable with the strongest association linked current monthly CFP calls to the peak August SST of the previous year. The lag period with the strongest association for storms was 18 months. If climate change increases SST in the Caribbean 2.5–3.5°C over the coming century as projected, this model implies that CFP incidence in the United States is likely to increase 200–400%.Conclusions: Using CFP calls as a marker of CFP incidence, these results clarify associations between climate variability and CFP incidence and suggest that, all other things equal, climate change could increase the burden of CFP. These findings have implications for disease prediction, surveillance, and public health preparedness for climate change.Citation: Gingold DB, Strickland MJ, Hess JJ. 2014. Ciguatera fish poisoning and climate change: analysis of National Poison Center data in the United States, 2001–2011. Environ Health Perspect 122:580–586; http://dx.doi.org/10.1289/ehp.1307196     

Effect of Incorporating Genetic Testing Results into Nutrition Counseling and Care on Health Outcomes: An Evidence Analysis Center Systematic Review—Part II

In recent years, literature examining implementation of nutritional genomics into clinical practice has increased, including publication of several randomized controlled trials (RCTs). This systematic review addressed the following question: In children and adults, what is the effect of incorporating results of genetic testing into nutrition counseling and care compared with an alternative intervention or control group, on nutrition-related health outcomes? A literature search of MEDLINE, Embase, PsycINFO, CINAHL, and other databases was conducted for peer-reviewed RCTs published from January 2008 until December 2018. An international workgroup consisting of registered dietitian nutritionists, systematic review methodologists, and evidence analysts screened and reviewed articles, summarized data, conducted meta-analyses, and graded conclusion statements. The second in a two-part series, this article specifically summarizes evidence from RCTs that examined health outcomes (ie, quality of life, disease incidence and prevention of disease progression, or mortality), intermediate health outcomes (ie, anthropometric measures, body composition, or relevant laboratory measures routinely collected in practice), and adverse events as reported by study authors. Analysis of 11 articles from nine RCTs resulted in 16 graded conclusion statements. Among participants with nonalcoholic fatty liver disease, a diet tailored to genotype resulted in a greater reduction of percent body fat compared with a customary diet for nonalcoholic fatty liver disease. However, meta-analyses for the outcomes of total cholesterol, low-density lipoprotein cholesterol, body mass index, and weight yielded null results. Heterogeneity between studies and low certainty of evidence precluded development of strong conclusions about the incorporation of genetic information into nutrition practice. Although there are still relatively few well-designed RCTs to inform integration of genetic information into the Nutrition Care Process, the field of nutritional genomics is evolving rapidly, and gaps in the literature identified by this systematic review can inform future studies.     

Contaminants in Stream Sediments From Seven United States Metropolitan Areas: Part II—Sediment Toxicity to the Amphipod Hyalella azteca and the Midge Chironomus dilutus

Relationships between sediment toxicity and sediment chemistry were evaluated for 98 samples collected from seven metropolitan study areas across the United States. Sediment-toxicity tests were conducted with the amphipod Hyalella azteca (28 day exposures) and with the midge Chironomus dilutus (10 day exposures). Overall, 33 % of the samples were toxic to amphipods and 12 % of the samples were toxic to midge based on comparisons with reference conditions within each study area. Significant correlations were observed between toxicity end points and sediment concentrations of trace elements, polycyclic aromatic hydrocarbons (PAHs), polychlorinated biphenyls (PCBs), or organochlorine (OC) pesticides; however, these correlations were typically weak, and contaminant concentrations were usually below sediment-toxicity thresholds. Concentrations of the pyrethroid bifenthrin exceeded an estimated threshold of 0.49 ng/g (at 1 % total organic carbon) in 14 % of the samples. Of the samples that exceeded this bifenthrin toxicity threshold, 79 % were toxic to amphipods compared with 25 % toxicity for the samples below this threshold. Application of mean probable effect concentration quotients (PECQs) based on measures of groups of contaminants (trace elements, total PAHs, total PCBs, OC pesticides, and pyrethroid pesticides [bifenthrin in particular]) improved the correct classification of samples as toxic or not toxic to amphipods compared with measures of individual groups of contaminants.     

Stearidonic acid: is there a role in the prevention and management of type 2 diabetes mellitus?

Obesity and its related comorbidities are major public health concerns in the United States with over two-thirds of adults and one-third of children classified as overweight or obese. The prevalence of type 2 diabetes mellitus (T2DM) has similarly risen to an estimated 25.8 million, which accounts for a staggering $174 billion in annual healthcare costs. Identification of dietary interventions that protect against the development of T2DM would markedly reduce the medical and economic consequences of the disease. Hence, we review current evidence supporting a role of (n-3) PUFA in T2DM and explore potential therapeutic implications of stearidonic acid (SDA). The low consumption of fish in the US along with a reduced efficiency to interconvert most plant (n-3) PUFA highlights a need to find alternative sources of (n-3) PUFA. The efficient biological conversion of SDA to EPA underscores the potential implications of SDA as a source of (n-3) PUFA. The full therapeutic efficacy of SDA remains to be further determined. However, recent data have suggested a protective role of SDA consumption on markers of dyslipidemia and inflammation. The AHA recommends that healthy individuals consume oily fish at least twice per week and individuals with a history of cardiovascular disease consume 1 g of EPA+DHA/d. These goals will likely not be met by the typical American diet. Therefore, SDA may represent a sustainable alternative to marine-based (n-3) PUFA and may have novel therapeutic efficacy regarding the development of T2DM.     

Analysis of some variables concerning guidelines adopted in a hospital in Milan: quantity,quality and physicians' attitude; which is the reciprocal influence of these factors?

BACKGROUND: Starting in 1998 the Italian NHS promoted the adoption of practice guidelines without, however, launching directions on how they should be produced and adopted. The aim of the current work was to explore the relationship between number and quality of guidelines and physicians' attitudes towards them. METHODS: All documents named 'guidelines' adopted after 1998 by each unit at the San Paolo Hospital (SPH), a 600-bed university hospital were collected. The quality of these guidelines was assessed according to a seven point scale looking at methodological quality and completeness of reporting. A 16-item questionnaire was delivered to all physicians of the SPH investigating barriers affecting their attitude towards adopting guidelines. The correlation between quantity and quality of guidelines and physicians' attitude was explored. RESULTS: In total, 319 documents from 19 units were examined. The overall quality varied across units and was generally poor (range: 0.5-3 items satisfied per guideline). Of the 392 physicians sampled, 301 filled in the questionnaire, and a positive attitude to guidelines was found. None of the relationships analysed--quantity, quality and attitude--turned out to be significantly associated. CONCLUSIONS: This study highlights contrasting results. The number of recorded documents and physicians' attitude indicate a vivacious interest in guidelines even if the quantitative production was extremely variable among units and the quality was unsatisfactory. This scenario may suggest a transition period in which the lack of a solid methodological background in the production and adoption of guidelines runs into the need to be compliant to a new rule.     

WHO Co-operative studies on a simple culture technique for the isolation of mycobacteria: 2. Comparison of the efficacy of lyophilized liquid medium with that of L?wenstein-Jensen (L-J) medium*

This is the second report on a series of co-operative studies undertaken with the object of devising a simplified technique, suitable for use in developing countries, for the isolation of mycobacteria from pathological specimens.