Interpreting Life Impact Burn Recovery Evaluation Profile Scores for Use by Clinicians, Burn Survivors, and Researchers.

The Life Impact Burn Recovery Evaluation (LIBRE) Profile is a patient-reported outcome measure developed to assess social participation in adult burn survivors. This study identified numeric score cut-points that define different levels of social participation ability and described each level. An expert panel identified numeric score cut-points that distinguish different levels of social participation for the six LIBRE Profile domains. Methods employed an iterative, modified-Delphi approach, and bookmarking to review calibrated item banks. Analyses (using calibration sample data and repeated in a validation sample) examined means, SDs, and sample distributions for each level. Analyses of variance examined score differences between levels. The panel developed descriptions for each established level. Initial cut-points resulted in four levels for five domains (Social Activities, Social Interactions, Romantic Relationships, Sexual Relationships, and Work & Employment) and five levels for the sixth domain (Relationships with Family & Friends). Comparisons demonstrated significant differences between level mean scores for all domains (P < .05) except Relationships with Family & Friends. Based on follow-up surveys, Relationships with Family & Friends score cut-points were adjusted to identify four levels with significant score differences between all levels. Panelists reached consensus for level descriptions. Score cut-points and descriptions identify different levels of social participation, providing a relevant context for interpreting LIBRE Profile numeric scores. LIBRE Profile Social Participation levels will help clinicians and persons with burn injury interpret LIBRE Profile numeric scores and promote use of this important new assessment.

Burn survivors injured as children exhibit resilience in long-term community integration outcomes: A life impact burn recovery evaluation (LIBRE) study.

INTRODUCTION: There is limited understanding of how burn injuries at different ages are associated with normal growth and development as well as the burn recovery process. This study provides new useful insights by comparing social participation outcomes among burn survivors injured in childhood compared with injuries sustained in middle age, and older adulthood. METHODS: Items from the development of the LIBRE profile were administered to 601 adult burn survivors with ≥5% TBSA burned or burns to critical areas (hands, feet, face, or genitals). Each item was answered on a 5-point Likert scale with higher scores denoting better outcomes. Mean scores for the 6 LIBRE profile scales (sexual relationships, family and friends, social interactions, social activities, work and employment, and romantic relationships) were compared between those burned as children (<18years) and those burned as adults (≥18years). Regression analyses were used to assess differences between groups with adjustment for demographic and clinical characteristics. RESULTS: Of the 597 burn survivors having complete data on age at injury, 165 (27.6%) sustained burn injuries as a child. Those burned as children were more frequently female than those burned as adults (57% vs 47%) and were also more frequently white non-Hispanic (89% vs 77%). Marital status and education level were similar in the two groups. Those who were burned as children had slightly higher scores on the social activities, work and employment and romantic relationships scales. However, these differences did not persist in adjusted regression analyses. CONCLUSIONS: Burn survivors who sustained injuries as a child fared at least as well as those burned as adults in a broad range of long-term social participation outcomes. The impact on long-term social participation outcomes of burn survivors was not significantly different between individuals with burns sustained during important developmental stages at young ages and those injured later in life.

EXAMINING EVIDENCE IN U.S. PAYER COVERAGE POLICIES FOR MULTI-GENE PANELS AND SEQUENCING TESTS.

OBJECTIVES: The aim of this study was to examine the evidence payers cited in their coverage policies for multi-gene panels and sequencing tests (panels), and to compare these findings with the evidence payers cited in their coverage policies for other types of medical interventions. METHODS: We used the University of California at San Francisco TRANSPERS Payer Coverage Registry to identify coverage policies for panels issued by five of the largest US private payers. We reviewed each policy and categorized the evidence cited within as: clinical studies, systematic reviews, technology assessments, cost-effectiveness analyses (CEAs), budget impact studies, and clinical guidelines. We compared the evidence cited in these coverage policies for panels with the evidence cited in policies for other intervention types (pharmaceuticals, medical devices, diagnostic tests and imaging, and surgical interventions) as reported in a previous study. RESULTS: Fifty-five coverage policies for panels were included. On average, payers cited clinical guidelines in 84 percent of their coverage policies (range, 73-100 percent), clinical studies in 69 percent (50-87 percent), technology assessments 47 percent (33-86 percent), systematic reviews or meta-analyses 31 percent (7-71 percent), and CEAs 5 percent (0-7 percent). No payers cited budget impact studies in their policies. Payers less often cited clinical studies, systematic reviews, technology assessments, and CEAs in their coverage policies for panels than in their policies for other intervention types. Payers cited clinical guidelines in a comparable proportion of policies for panels and other technology types. CONCLUSIONS: Payers in our sample less often cited clinical studies and other evidence types in their coverage policies for panels than they did in their coverage policies for other types of medical interventions.

A Systematic Review of Cost-Effectiveness Studies Reporting Cost-per-DALY Averted.

INTRODUCTION: Calculating the cost per disability-adjusted life years (DALYs) averted associated with interventions is an increasing popular means of assessing the cost-effectiveness of strategies to improve population health. However, there has been no systematic attempt to characterize the literature and its evolution. METHODS: We conducted a systematic review of cost-effectiveness studies reporting cost-per-DALY averted from 2000 through 2015. We developed the Global Health Cost-Effectiveness Analysis (GHCEA) Registry, a repository of English-language cost-per-DALY averted studies indexed in PubMed. To identify candidate studies, we searched PubMed for articles with titles or abstracts containing the phrases "disability-adjusted" or "DALY". Two reviewers with training in health economics independently reviewed each article selected in our abstract review, gathering information using a standardized data collection form. We summarized descriptive characteristics on study methodology: e.g., intervention type, country of study, study funder, study perspective, along with methodological and reporting practices over two time periods: 2000-2009 and 2010-2015. We analyzed the types of costs included in analyses, the study quality on a scale from 1 (low) to 7 (high), and examined the correlation between diseases researched and the burden of disease in different world regions. RESULTS: We identified 479 cost-per-DALY averted studies published from 2000 through 2015. Studies from Sub-Saharan Africa comprised the largest portion of published studies. The disease areas most commonly studied were communicable, maternal, neonatal, and nutritional disorders (67%), followed by non-communicable diseases (28%). A high proportion of studies evaluated primary prevention strategies (59%). Pharmaceutical interventions were commonly assessed (32%) followed by immunizations (28%). Adherence to good practices for conducting and reporting cost-effectiveness analysis varied considerably. Studies mainly included formal healthcare sector costs. A large number of the studies in Sub-Saharan Africa addressed high-burden conditions such as HIV/AIDS, tuberculosis, neglected tropical diseases and malaria, and diarrhea, lower respiratory infections, meningitis, and other common infectious diseases. CONCLUSION: The Global Health Cost-Effectiveness Analysis Registry reveals a growing and diverse field of cost-per-DALY averted studies. However, study methods and reporting practices have varied substantially.

Assessing the Value of Treatment to Address Various Symptoms Associated with Multiple Sclerosis: Results from a Contingent Valuation Study.

BACKGROUND: Although it is well recognized that people with multiple sclerosis (MS) may experience impairments in addition to limited mobility, there has been little effort to study their relative importance to patients with the condition. The objective of this study was to assess patient preferences for addressing various MS symptoms. METHODS: This study was conducted at Tufts Medical Center, Boston, Massachusetts. We developed a national online survey of MS patients and neurologists to estimate the value each group places on treating specific MS symptoms. Each respondent was presented with two randomly selected scenarios with different symptoms and treatments. MS patients were asked about their own preferences, whereas neurologists were asked to consider what a patient of theirs would do or think in each scenario. We used a bidding game approach to elicit respondents' willingness to pay (WTP) for the treatments. RESULTS: To treat mobility alone, WTP for MS patients averaged US$410-US$520 per month, depending on the scenario. For paired symptoms, MS patients would pay most to treat mobility and upper limb function (US$525/month) or mobility and cognition (US$514/month), somewhat less to treat mobility and eyesight (US$445/month), and least to treat mobility and fatigue (US$371/month). Patient WTP values increased with income and education. Neurologists believed their patients would be willing to pay US$216-US$249 per month to treat mobility alone, depending on the scenario. For paired symptoms, neurologists believed patients would pay most to treat mobility and fatigue (US$263/month) and least to treat mobility and upper limb function (US$177/month). CONCLUSION: Our findings suggest MS patients may value one outcome (e.g., improved arm and hand coordination) over another (e.g., less fatigue). Further, MS patients and neurologists may rank the importance of treating various symptoms differently. Given this potential mismatch, it is crucial for MS patients and their clinicians to discuss treatment priorities that take into account patient preferences.

The peculiar economics of life-extending therapies: a review of costing methods in health economic evaluations in oncology.

Published literature lacks consensus, and most guidelines lack definitive recommendations as to whether cost-effectiveness analyses (CEAs) should include all "future" costs or distinguish between related and unrelated medical costs. This systematic review of oncology CEAs evaluated cost methods used and the impact on the cost-effectiveness of incorporating different cost categories, including costs due to study intervention, related medical costs of the treated condition, and unrelated medical costs. Of the 59 studies reviewed, none included medical costs unrelated to the treated condition and 14 studies (32%) excluded direct medical costs related to the condition but not the evaluated intervention. Recomputing ICERs using different cost categories altered overall cost-effectiveness conclusions. The authors propose conventional CEA methods may implicitly penalize therapies that add "expensive" life years for chronically ill patients. Presenting ICERs computed with and without disease-attributable costs can help better convey how much the treatment itself contributes to overall costs.

When Does FDAMA Section 114 Apply? Ten Case Studies.

BACKGROUND: Section 114 of the Food and Drug Administration Modernization Act of 1997 regulates the promotion of health economic information by pharmaceutical companies to US health plans. Greater clarity is important given demands by payers and other stakeholders for evidence of value. OBJECTIVES: To develop hypothetical case studies of health economic promotions to examine legal and policy implications. METHODS: We constructed for pedagogical purposes 10 categories of potential health economic promotions. We generated hypothetical case studies for each category, including questions about whether each might be allowable under Section 114. The case studies were developed around the following categories: 1) costing out on-label clinical end points; 2) promotion of a costing exercise to physicians working in an accountable care organization setting; 3) burden-of-illness claims; 4) economic analysis of a formulary restriction policy; 5) extrapolations to doses, populations, or settings not covered in trials; 6) adherence claims; 7) "utilization of care" as a secondary end point in randomized clinical trials; 8) costing out a competitor drug's adverse event; 9) economic analysis of comparative effectiveness claims using an indirect treatment comparison; and 10) extrapolating from surrogate to long-term outcomes in an economic model. DISCUSSION: Most cases seem to fall into a gray zone given haziness around what constitutes "competent and reliable evidence" and "directly relate[d]" to an approved indication. In practice, it is difficult to know what the section allows given the imprecision of the statute and lack of guidance about its scope. CONCLUSION: Ideally, future guidance will provide clarity and flexibility.

The changing face of the cost-utility literature, 1990-2012.

OBJECTIVES: Cost-utility analyses (CUAs) have been published widely over the years to measure the value of health care interventions. We investigated the growth and characteristics of CUAs in the peer-reviewed English-language literature through 2012. METHODS: We analyzed data from the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry, a database containing more than 3700 English-language CUAs published through 2012. We summarized various study characteristics (e.g., intervention type, funding source, and journal of publication) and methodological practices (e.g., use of probabilistic sensitivity analysis) over three time periods: 1990 to 1999, 2000 to 2009, and 2010 to 2012. We also examined CUAs by country, region, and the degree to which diseases studied correlate with disease burden. RESULTS: The number of published CUAs rose from 34 per year from 1990 to 1999 to 431 per year from 2010 to 2012. The proportion of studies focused on the United States declined from 61% during 1990 to 1999 to 35% during 2010 to 2012 (P < 0.0001). Although still small compared with CUAs in higher income countries, the number of CUAs focused on lower and middle-income countries has risen sharply. A large fraction of studies pertain to pharmaceuticals (46% during 2010-2012). In recent years, most studies included probabilistic sensitivity analysis (67% during 2010-2012). Journals publishing CUAs vary widely in the percentage of their studies funded by drug companies. Some conditions, such as injuries, have high burden but few CUAs. CONCLUSIONS: Our review reveals considerable growth and some change in the cost-utility literature in recent years. The data suggest growing interest in cost-utility methodology, particularly in non-Western countries.

Value of innovation in hematologic malignancies: a systematic review of published cost-effectiveness analyses.

We analyzed cost-effectiveness studies related to hematologic malignancies from the Tufts Medical Center Cost-Effectiveness Analysis Registry (www.cearegistry.org), focusing on studies of innovative therapies. Studies that met inclusion criteria were categorized by 4 cancer types (chronic myeloid leukemia, chronic lymphocytic leukemia, non-Hodgkin lymphoma, and multiple myeloma) and 9 treatment agents (interferon-α, alemtuzumab, bendamustine, bortezomib, dasatinib, imatinib, lenalidomide, rituximab alone or in combination, and thalidomide). We examined study characteristics and stratified cost-effectiveness ratios by type of cancer, treatment, funder, and year of study publication. Twenty-nine studies published in the years 1996-2012 (including 44 cost-effectiveness ratios) met inclusion criteria, 22 (76%) of which were industry funded. Most ratios fell below $50,000 per quality-adjusted life-years (QALY) (73%) and $100,000/QALY (86%). Industry-funded studies (n = 22) reported a lower median ratio ($26,000/QALY) than others (n = 7; $33,000/QALY), although the difference was not statistically significant. Published data suggest that innovative treatments for hematologic malignancies may provide reasonable value for money.

A survey of individuals in US-based pharmaceutical industry HEOR departments: attitudes on policy topics.

We surveyed US-based leaders in health economics and outcomes research (HEOR) departments in drug and device companies to examine their views on the state of the field. We created a questionnaire that was emailed to 123 US-based senior HEOR professionals at 54 companies. Of the 123 recipients, 74 (60%) completed the survey. Most respondents (92%) expected their company's HEOR use to increase, and 80% reported that their organization's senior management viewed HEOR work as critical. Approximately 62% agreed that Academy of Managed Care Pharmacy (AMCP) dossiers are useful to US health plans, and 55% stated that Food and Drug Administration Modernization Act (FDAMA) Section 114 is useful. Approximately 49% believed the US government should use cost-effectiveness analysis in coverage and reimbursement decisions, but only 31% expected this to occur within 3 years. The findings suggest strong support for the function at senior management levels and optimism about the field.