Are Medical Devices Cost-Effective?

OBJECTIVE: Medical devices can offer important therapeutic advances but, as for any medical interventions, there are questions about their costs and benefits. We examined health benefits and costs for pre-market approved (PMA) devices approved by the US Food and Drug Administration (FDA) (1999-2015), grouping them by generic category (e.g., drug-eluting stents) and indication. METHODS: We searched PubMed for incremental health gain estimates [measured in quality-adjusted life-years (QALYs)] and incremental costs for each device category compared to previously available treatments. We calculated incremental cost-effectiveness ratios by dividing the average incremental costs by the average incremental QALY gains. In sensitivity analysis, we repeated the analysis when excluding industry-funded studies. RESULTS: We identified at least one relevant cost-utility or comparative-effectiveness study for 88 devices (15.9% of non-cosmetic devices approved from 1999 to 2015), and at least one device across 53 (26.2%) generic categories. The median (mean) incremental cost across generic device categories was $1701 ($13,320). The median (mean) incremental health gain across generic device categories was 0.13 (0.46) QALYs. We found that cost-effectiveness ratios for 36 of 53 (68%) and 43 of 53 (81%) device categories fell below (were more favorable than) $50,000 and $150,000 per QALY, respectively. Results were roughly similar when we excluded industry-funded studies. CONCLUSIONS: We found that roughly one-quarter of the major PMA medical device categories have published cost-effectiveness evidence accessible through a large, publicly available database. Available evidence suggests that devices generally offer good value, as judged relative to established cost-effectiveness benchmarks.

Frequency and impact of the inclusion of broader measures of value in economic evaluations of vaccines.

BACKGROUND: The health and economic benefits of immunization may extend beyond the elements traditionally included in cost-effectiveness analyses (CEAs). This review investigated how broader impacts are considered in economic evaluations of vaccines and whether their inclusion would substantially change CEA findings. METHODS: We reviewed CEAs of vaccines associated with the largest global health burden, published from 2014 to 2019 using the Tufts CEA Registry and Tufts Global Health CEA Registry. We supplemented this with a systematic review of published and grey literature. We conducted descriptive analyses to examine the frequency of inclusion of specific social factors and study characteristics associated with their inclusion. We also conducted a case study of the human papilloma virus (HPV) vaccine to illustrate the potential change in CEA findings from selected social impacts. RESULTS: We identified 475 relevant health economic assessments. Overall, 40% of studies included at least one category of social impact. The most commonly included non-healthcare cost among cost-per-QALY studies was productivity (25%), while cost-per-DALY studies reported transportation costs most frequently (24%). Few studies examined the impact of vaccination on other sectors such as education and housing (<3%). Middle-income and North American settings were positively associated with social impact inclusion, while sub-Saharan African location was negatively associated. In the HPV case study, the addition of nonhealth costs improved cost-effectiveness by up to 90% or made the vaccine cost-saving, depending on geographic setting. The cost-saving scenario saved up to $30,000 in costs per case of cervical cancer averted. CONCLUSIONS: A minority of vaccine CEAs include social impacts, particularly for nonhealth sectors. The omission of these impacts may result in a systematic undervaluation of vaccines from a societal perspective. Further efforts are required to document the full benefits of vaccination for policymaker consideration.

Correction to: Perspective and Costing in Cost­Effectiveness Analysis, 1974-2018.

The article Perspective and Costing in Cost-Effectiveness Analysis.

Perspective and Costing in Cost-Effectiveness Analysis, 1974-2018.

OBJECTIVE: Our objective was to examine perspective and costing approaches used in cost-effectiveness analyses (CEAs) and the distribution of reported incremental cost-effectiveness ratios (ICERs). METHODS: We analyzed the Tufts Medical Center's CEA and Global Health CEA registries, containing 6907 cost-per-quality-adjusted-life-year (QALY) and 698 cost-per-disability-adjusted-life-year (DALY) studies published through 2018. We examined how often published CEAs included non-health consequences and their impact on ICERs. We also reviewed 45 country-specific guidelines to examine recommended analytic perspectives. RESULTS: Study authors often mis-specified or did not clearly state the perspective used. After re-classification by registry reviewers, a healthcare sector or payer perspective was most prevalent (74%). CEAs rarely included unrelated medical costs and impacts on non-healthcare sectors. The most common non-health consequence included was productivity loss in the cost-per-QALY studies (12%) and patient transportation in the cost-per-DALY studies (21%). Of 19,946 cost-per-QALY ratios, the median ICER was $US26,000/QALY (interquartile range [IQR] 2900-110,000), and 18% were cost saving and QALY increasing. Of 5572 cost-per-DALY ratios, the median ICER was $US430/DALY (IQR 67-3400), and 8% were cost saving and DALY averting. Based on 16 cost-per-QALY studies (2017-2018) reporting 68 ICERs from both the healthcare sector and societal perspectives, the median ICER from a societal perspective ($US22,710/QALY [IQR 11,991-49,603]) was more favorable than from a healthcare sector perspective ($US30,402/QALY [IQR 10,486-77,179]). Most governmental guidelines (67%) recommended either a healthcare sector or a payer perspective. CONCLUSION: Researchers should justify and be transparent about their choice of perspective and costing approaches. The use of the impact inventory and reporting of disaggregate outcomes can reduce inconsistencies and confusion.

Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs.

BACKGROUND: Orphan drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan drugs, leading to questions about their cost-effectiveness. OBJECTIVE: To compare the value of orphan and non-orphan drugs approved by the FDA from 1999 through 2015. DESIGN: We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a drug was approved for multiple indications, we considered each drug-indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). RESULTS: We identified estimates for 49 orphan drug-indication pairs, and for 169 non-orphan drug-indication pairs. We found that orphan drug-indication pairs offered larger median incremental health gains than non-orphan drug-indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p < 0.001, KS p < 0.001) and less favorable cost-effectiveness ($276,288 vs. $100,360 per QALY gained; MWU p = 0.0068, KS p = 0.009). CONCLUSIONS: Our study suggests that orphan drugs often offer larger health gains than non-orphan drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan drugs while achieving value from drug spending.

Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?

BACKGROUND: The Institute for Clinical and Economic Review (ICER) has gained prominance through its work conducting health technology assessments of pharmaceuticals in the United States. OBJECTIVE: To understand the influence of industry comments on pharmaceutical value assessments conducted by ICER. METHODS: We reviewed 15 ICER reports issued from 2017 through 2019. We quantified ICER's revisions to its cost-effectiveness analysis (CEA) estimates between release of its draft and revised evidence reports and whether ratios shifted across ICER-specified categories of high, intermediate, or low value. We also reviewed industry-submitted comments recommending revision to ICER's CEAs, noting ICER's response as no change, text revised, assumption(s) revised, or conclusion revised. We evaluated each comment in terms of clarity, whether it offered an alternative to ICER's approach, and whether it characterized the expected impact of revision on ICER's analysis. RESULTS: We identified 53 ICER-reported ratios. Of these, 45 (84.9%) changed between the draft and revised report, but 26 changes (57.8%) were small (<10%). Six ratios shifted across value categories. We identified 256 industry comments recommending that ICER revise its CEA. Of these, 159 (62%) lacked clarity, 145 (57%) offered no alternative, and 243 (95%) did not characterize their impact on ICER's estimated ratio. Ninety-one comments (35.5%) caused ICER to revise its assumptions, but only 5 (2.0%) caused ICER to revise its conclusions. Four of these 5 comments characterized their impact on ICER's findings. CONCLUSIONS: Changes in ICER's estimates of cost-effectiveness between its draft and revised evidence reports are generally modest. Greater precision in industry comments could increase the influence of industry critiques, thus enhancing the dialogue around pharmaceutical value.