Healthcare resource utilization and costs among children with cystic fibrosis in the United States.

BACKGROUND: Adverse health impacts of cystic fibrosis (CF) can be present in children before respiratory complications are observed. Children with CF show progressive health decline, with increasing lung function decline in adolescence. This study aims to quantify the healthcare resource utilization (HCRU) and costs attributable to CF by comparing children with CF with the general pediatric population. METHODS: This retrospective, cross-sectional, observational study compared HCRU and costs among children with CF in the US with demographically similar children without CF (comparison group) over a 12-month period using administrative claims data spanning 2010-2017. Analyses were conducted by insurance type (commercially insured [COM] and Medicaid insured [MED]) and stratified by age (<2 years, 2 to <6 years, 6 to <12 years, and 12-17 years). RESULTS: Children with CF (2831 COM and 1896 MED) were matched to children in the comparison group (8493 COM and 5688 MED). Higher prevalence of comorbidities was seen in children with CF versus the comparison group across all ages. Across all ages, HCRU attributable to CF was substantial (higher hospitalization rates, more outpatient and emergency room visits, and greater use of prescription medications), and there were higher associated costs (all p values < .05), in COM and MED populations. HCRU and costs attributable to CF were highest for children aged 12-17 years. CONCLUSIONS: Substantial HCRU and costs are evident among children with CF across all ages, starting as young as infancy, with highest HCRU and costs among adolescents. Effective treatments from an early age are needed for children with CF.

Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia.

BACKGROUND: Prophylaxis with clotting factor replacement products is recommended by the Medical and Scientific Advisory Council of the National Hemophilia Foundation as the optimal therapy for the prevention of bleeding episodes in individuals with severe hemophilia A or B (< 1 IU per dL endogenous factor VIII or factor IX activity, respectively). Prophylaxis is associated with an improved health-related quality of life and has been shown to be cost-effective compared with on-demand therapy. However, the overall cost of treatment remains high, particularly among patients with a greater propensity to bleed. The overall value of hemophilia treatments and their associated benefits, measured in quality-adjusted life-years (QALYs), and dollar costs compared with other interventions can be evaluated through the use of cost-utility analyses (CUAs). Previous CUA studies in hemophilia have focused primarily on patients with more severe forms of hemophilia and on prophylaxis compared with on-demand treatment. However, to our knowledge, no studies to date have utilized QALYs as a standardized outcome measure to systematically evaluate the relative cost-effectiveness of current hemophilia treatment options. OBJECTIVE: To systematically review the CUA literature of hemophilia treatments and demonstrate the challenges in producing cost-utility evidence compared with other rare diseases. METHODS: We conducted a systematic literature review using the Tufts Medical Center Cost-Effectiveness Analysis Registry and the National Health Service Economic Evaluation Database for English-language CUAs published from 2000 through 2015 with the search terms hemophilia, haemophilia, factor VIII, or factor IX. Two trained reviewers independently reviewed every study to extract relevant data. Incremental cost-effectiveness ratios were converted to 2014 U.S. dollars using exchange rates for currency conversion and the Consumer Price Index to adjust for inflation. RESULTS: Our search yielded 52 studies, 11 of which met our inclusion criteria. The cost-effectiveness of hemophilia treatments varied widely based on variations in the study designs, including differences in time horizon, discount rates, and medical interventions. CONCLUSIONS: We found the cost-effectiveness of hemophilia treatments to be broadly comparable to that of other orphan drugs. Improved standardization of future CUA studies will be important for further evaluation of the cost-effectiveness of hemophilia treatments. DISCLOSURES: This research was funded by Biogen, which provided an unrestricted research grant to the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. Biogen and Sobi reviewed and provided feedback on the manuscript. The authors had full editorial control of the manuscript and provided final approval of all content. The authors report no conflict of interest regarding the material discussed in this article. Neumann and Chambers are employed at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. Thorat was an employee of Center for Evaluation Value and Risk in Health, Tufts Medical Center when the analyses were carried out. Chambers has participated on advisory boards for Sanofi and Astellas Pharma.

Patient Variability Seldom Assessed in Cost-effectiveness Studies.

BACKGROUND: Cost-effectiveness analysis (CEA) estimates can vary substantially across patient subgroups when patient characteristics influence preferences, outcome risks, treatment effectiveness, life expectancy, or associated costs. However, no systematic review has reported the frequency of subgroup analysis in CEA, what type of heterogeneity they address, and how often heterogeneity influences whether cost-effectiveness ratios exceed or fall below conventional thresholds. METHODS: We reviewed the CEA literature cataloged in the Tufts Medical Center CEA Registry, a repository describing cost-utility analyses published through 2016. After randomly selecting 200 of 642 articles published in 2014, we ascertained whether each study reported subgroup results and collected data on the defining characteristics of these subgroups. We identified whether any of the CEA subgroup results crossed conventional cost-effectiveness benchmarks (e.g., $100,000 per QALY) and compared characteristics of studies with and without subgroup-specific findings. RESULTS: Thirty-eight studies (19%) reported patient subgroup results. Articles reporting subgroup analyses were more likely to be US-based, government funded (v. drug industry- or nonprofit foundation-funded) studies, with a focus on primary or secondary (v. tertiary) prevention (P < 0.05 for comparisons). One or more patient characteristics were used to stratify CEA results 68 times within the 38 studies, with most stratifications using one characteristic (n = 47), most commonly age (n = 35). Among the 23 stratifications reported alongside average ratios in US studies, 13 produced subgroup ratios that crossed a conventional CEA ratio benchmark. CONCLUSIONS: Most CEAs do not report any subgroup results, and those that do most often stratify only by patient age. Over half of the subgroup analyses reported could lead to different value-based decision making for at least some patients.

When cost-effective interventions are unaffordable: Integrating cost-effectiveness and budget impact in priority setting for global health programs.

Potential cost-effective barriers in cost-effectiveness studies mean that budgetary impact analyses should also be included in post-2015 Sustainable Development Goal projects says Joshua Salomon and colleagues.

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.

Private payers disagree with Medicare over medical device coverage about half the time.

There are multiple payers in the US health care system, each making its own coverage determinations for medical technologies. For each of the forty-seven medical devices considered in national coverage determinations (NCDs) of the Centers for Medicare and Medicaid Services (CMS) issued between February 1999 and August 2013, we compared CMS's coverage policy with the policies issued by the largest sixteen private payers that made their decisions publicly available. Overall, we found that NCDs were equivalent to the corresponding private payer policies roughly half of the time, more restrictive approximately a quarter of the time, and less restrictive about a quarter of the time. Our findings indicate that patients may have variable access to medical technology across Medicare and private plans. They also suggest that private plans do not necessarily follow CMS's lead in making coverage decisions.

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.

The lag from FDA approval to published cost-utility evidence.

The lag between FDA approval and publication of cost-utility evidence can hamper payers from accounting for value for money in coverage and reimbursement decisions. We examine this gap, and whether it has changed over time. For drugs approved from 2000 to 2010 (n = 274), we searched the Tufts Medical Center Cost-Effectiveness Analysis Registry to identify relevant cost-utility analyses (CUAs). We identified 127 (46%) drugs associated with a CUA, 62 of which had a CUA published in the 3 years following its approval. Compared with drugs approved from 2000 to 2003, a greater proportion of those approved from 2004 to 2006, and from 2007 to 2010, was associated with a CUA published in the 3 years following approval (13 vs 25% [p = 0.06] and 13 vs 32% [p < 0.01], respectively). Study findings indicate that payers now have slightly more rapid access to published CUAs.

The State of Cost-Utility Analyses in Asia: A Systematic Review.

OBJECTIVE: To review and evaluate published cost-utility analyses (CUAs) targeting populations in Asia. METHODS: We examined data from the Tufts Medical Center Cost-Effectiveness Analysis Registry, which contains detailed information on more than 3700 English-language CUAs in peer-reviewed journals through 2012. We focused on CUAs pertaining to Asian countries (Asian CUAs), summarized study features and methodological practices, and compared them with CUAs focusing on non-Asian countries (non-Asian CUAs) from 2000 to 2012. RESULTS: We identified 175 published CUAs pertaining to Asian populations (representing 5.1% of all CUAs) from 2000 to 2012. The number has increased from 19 CUAs in the period 2000 to 2004 to 107 CUAs in the period 2009 to 2012. Roughly one-third focused on Japan (33.1%), followed by Taiwan (15.4%), China (14.9%), and Thailand (8.0%). The diseases targeted in Asian CUAs were cancer (24.6%), infectious diseases (13.7%), cardiovascular diseases (8.6%), and musculoskeletal and rheumatological diseases (5.7%). More Asian CUAs evaluated primary prevention interventions (e.g., vaccinations and screenings) compared with non-Asian CUAs (21.7% vs. 16.5%, P = 0.069). Compared with non-Asian CUAs, significantly more studies in Asia suggest that the health interventions examined provide reasonable value for money. Asian and non-Asian CUAs did not differ in adherence to good methodological practices, including clearly stating the perspective, discounting costs and quality-adjusted life-years, stating a time horizon, and correctly conducting incremental cost-effectiveness analysis. Asian CUAs, however, lagged in reporting sensitivity analyses, disclosing funding status, and currency year. CONCLUSIONS: The number of CUAs in Asia has grown steadily, with more than half focused on pharmaceuticals. The literature reveals that CUAs generally follow good methodological practices though areas for improvement exist.

Despite high costs, specialty drugs may offer value for money comparable to that of traditional drugs.

Specialty drugs are often many times more expensive than traditional drugs, which raises questions of affordability and value. We compared the value of specialty and traditional drugs approved by the Food and Drug Administration (FDA) in the period 1999-2011. To do this, we identified published estimates of additional health gains (measured in quality-adjusted life-years, or QALYs) and increased costs of drug and health care resource use that were associated with fifty-eight specialty drugs and forty-four traditional drugs, compared to preexisting care. We found that specialty drugs offered greater QALY gains (0.183 versus 0.002 QALYs) but were associated with greater additional costs ($12,238 versus $784), compared to traditional drugs. The two types of drugs had comparable cost-effectiveness. However, the distributions across the two types differed, with 26 percent of specialty drugs--but only 9 percent of traditional drugs--associated with incremental cost-effectiveness ratios of greater than $150,000 per QALY. Our study suggests that although specialty drugs often have higher costs than traditional drugs, they also tend to confer greater benefits and hence may still offer reasonable value for money.