An Alternative Measure of Health for Value Assessment: The Equal Value Life-Year.

The quality-adjusted life-year (QALY) is an international standard in cost-effectiveness analysis. A known concern arises from the relatively lower QALY gains attributed to treatments that extend the life of individuals with chronic disability. We analyze here the advantages and disadvantages of the equal value life-year (evLY) as an alternative or a complementary measure to the QALY, and share learned experiences from using this measure in health technology assessments. We present the conceptual rationale for the evLY, describe how it is estimated, and assess the differences in results between analyses based on the evLY and the QALY. We share a how-to guide in estimating the evLY using a downloadable tool and summarize our empirical experience using this measure. Incremental evLYs are feasible and address concerns regarding the risk for a cost-effectiveness analysis to undervalue treatments for people with chronic disabilities. Based on our set of analyses using the evLY, a threshold of $84,000 per evLY gained would be needed to maintain alignment with a threshold of $100,000 per added QALY. The evLY is a measure of health gain that can be used as an alternative or a complement to the QALY to address concerns related to undervaluing treatments that extend the life of individuals with serious illness or chronic disability. We recommend that it be reported within all cost-effectiveness analyses but may have special relevance in the current political environment in the USA, where use of the QALY is often challenged or prohibited.

Oral and monoclonal antibody treatments for relapsing forms of multiple sclerosis: Effectiveness and value.

DISCLOSURES: Ms McKenna, Dr Lin, Dr Whittington, Mr Nikitin, Ms Herron-Smith, Dr Campbell, and Dr Peterson report grants from Arnold Ventures, grants from Blue Cross Blue Shield of MA, grants from California Healthcare Foundation, grants from The Commonwealth Fund, and grants from The Peterson Center on Healthcare, during the conduct of the study; other from America's Health Insurance Plans, other from Anthem, other from AbbVie, other from Alnylam, other from AstraZeneca, other from Biogen, other from Blue Shield of CA, other from CVS, other from Editas, other from Express Scripts, other from Genentech/Roche, other from GlaxoSmithKline, other from Harvard Pilgrim, other from Health Care Service Corporation, other from Kaiser Permanente, other from LEO Pharma, other from Mallinckrodt, other from Merck, other from Novartis, other from National Pharmaceutical Council, other from Premera, other from Prime Therapeutics, other from Regeneron, other from Sanofi, other from United Healthcare, other from HealthFirst, other from Pfizer, other from Boehringer-Ingelheim, other from uniQure, other from Envolve Pharmacy Solutions, other from Humana, and other from Sun Life, outside the submitted work.

Cost-effectiveness of Lifestyle Africa: an adaptation of the diabetes prevention programme for delivery by community health workers in urban South Africa.

BACKGROUND: Lifestyle Africa is an adapted version of the Diabetes Prevention Program designed for delivery by community health workers to socioeconomically disadvantaged populations in low- and middle-income countries (LMICs). Results from the Lifestyle Africa trial conducted in an under-resourced community in South Africa indicated that the programme had a significant effect on reducing haemoglobin A1c (HbA1c). OBJECTIVE: To estimate the cost of implementation and the cost-effectiveness (in cost per point reduction in HbA1c) of the Lifestyle Africa programme to inform decision-makers of the resources required and the value of this intervention. METHODS: Interviews were held with project administrators to identify the activities and resources required to implement the intervention. A direct-measure micro-costing approach was used to determine the number of units and unit cost for each resource. The incremental cost per one point improvement in HbA1c was calculated. RESULTS: The intervention equated to 71 United States dollars (USD) in implementation costs per participant and a 0.26 improvement in HbA1c per participant. CONCLUSIONS: Lifestyle Africa reduced HbA1c for relatively little cost and holds promise for addressing chronic disease in LMIC. Decision-makers should consider the comparative clinical effectiveness and cost-effectiveness of this intervention when making resource allocation decisions. TRIAL REGISTRATION: Trial registration is at ClinicalTrials.gov (NCT03342274).

Living Health Technology Assessment: Issues, Challenges and Opportunities.

Health technology assessments (HTAs) are typically performed as one-off evaluations and can potentially become out-of-date due to the availability of new data, new comparators, or other factors. Recently, living approaches have been applied to systematic reviews and network meta-analyses to enable evidence syntheses to be updated more easily. In this paper, we provide a definition for 'Living HTA' where such a living approach could be applied to the entire HTA process. Living HTA could involve performing regular or scheduled updates using a traditional manual approach, or indeed in a semi-automated manner leveraging recent technological innovations that automate parts of the HTA process. The practical implementation of living HTA using both approaches (i.e., manual approach and using semi-automation) is described along with the likely issues and challenges with planning and implementing a living HTA process. The time, resources and additional considerations outlined may prohibit living HTA from becoming the norm for every evaluation; however, scenarios where living HTA would be particularly beneficial are discussed.

Differences in cardiac testing resource utilization using two different risk stratification schemes.

OBJECTIVE: Assess whether changing an emergency department (ED) chest pain pathway from utilizing the Thrombolysis in Myocardial Infarction (TIMI) score for risk stratification to an approach utilizing the History, EKG, Age, Risk, Troponin (HEART) score was associated with reductions in healthcare resource utilization. METHODS: A retrospective, quasi-experimental study using difference-in-differences and interrupted time series specifications evaluated all ED patients with a chest pain encounter from 8/2015 to 7/2019 at a large academic medical center. We included patients age ≥ 18 with negative troponin testing discharged from the ED. Our standardized care pathway utilized TIMI for risk stratification until 09/2017 and HEART thereafter. We evaluated patients undergoing hospital-based cardiac diagnostic testing (CDT), length of stay (LOS), and 30-day Major Adverse Cardiovascular Events (MACE) at the intervention site before and after the pathway change and compared these outcomes to a similar control site within the health system for the difference-in-differences specification. RESULTS: During the study period, 6.3% (450 of 7117) of patients in the TIMI cohort and 7.2% (546 of 7623) in the HEART cohort among 400,965 total ED visits underwent CDT. In a multivariable analysis, transition to the HEART pathway was associated with greater odds of receiving CDT (odds ratio 2.88 [95% CI 1.21 to 6.86]), a reduction in LOS of 34 min (95% CI 2.2 to 67.6), and no significant difference in 30-day MACE. CONCLUSION: The transition from TIMI to HEART was associated with mixed consequences for healthcare resource utilization, including increased CDT but reduced length of stay.

The Influence of the Rural Transitions Nurse Program for Veterans on Healthcare Utilization Costs.

BACKGROUND: The Veterans Affairs (VA) Healthcare System Rural Transitions Nurse Program (TNP) addresses barriers veterans face when transitioning from urban tertiary VA hospitals to home. Previous clinical evaluations of TNP have shown that enrolled veterans were more likely to follow up with their primary care provider within 14 days of discharge and experience a significant reduction in mortality within 30 days compared to propensity-score matched controls. OBJECTIVE: Examine changes from pre- to post-hospitalization in total, inpatient, and outpatient 30-day healthcare utilization costs for TNP enrollees compared to controls. DESIGN: Quantitative analyses modeling the changes in cost via multivariable linear mixed-effects models to determine the association between TNP enrollment and changes in these costs. PARTICIPANTS: Veterans meeting TNP eligibility criteria who were discharged home following an inpatient hospitalization at one of the 11 implementation sites from April 2017 to September 2019. INTERVENTION: The four-step TNP transitional care intervention. MAIN MEASURES: Changes in 30-day total, inpatient, and outpatient healthcare utilization costs were calculated for TNP enrollees and controls. KEY RESULTS: Among 3001 TNP enrollees and 6002 controls, no statistically significant difference in the change in total costs (p = 0.65, 95% CI: (- $675, $350)) was identified. However, on average, the increase in inpatient costs from pre- to post-hospitalization was approximately $549 less for TNP enrollees (p = 0.02, 95% CI: (- $856, - $246)). The average increase in outpatient costs from pre- to post-hospitalization was approximately $421 more for TNP enrollees compared to controls (p = 0.003, 95% CI: ($109, $671)). CONCLUSIONS: Although we found no difference in change in total costs between veterans enrolled in TNP and controls, TNP was associated with a smaller increase in direct inpatient medical costs and a larger increase in direct outpatient medical costs. This suggests a shifting of costs from the inpatient to outpatient setting.

Oral treatments for outpatient COVID-19: Effectiveness and value.

DISCLOSURES: Ms Beinfeld and Nahn and Drs Whittington, Mohammed, and Pearson report grants from Arnold Ventures, Kaiser Foundation Health Plan Inc., The Patrick and Catherine Weldon Donaghue Medical Research Foundation, Blue Cross Blue Shield of Massachusetts, and The Commonwealth Foundation, during the conduct of the study; and other from America's Health Insurance Plans, Anthem, AbbVie, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Premera, Prime Therapeutics, Regeneron, Sanofi, United Healthcare, HealthFirst, Pfizer, Boehringer-Ingelheim, uniQure, Humana, Sun Life, and Envolve Pharmacy Solutions, outside the submitted work. Dr Yeung received a contract from ICER to be an evidence author for COVID-19 outpatient treatments.

Cost-Effectiveness Analysis: Personal Systems Approach in Improving Medication Adherence in Adult Kidney Transplant Patients.

ABSTRACT: Interventions to improve medication nonadherence in transplantation have recently moved from a focus on motivation and intention, to a focus on person-level quality improvement strategies. These strategies link adherence to established daily routines, environmental cues, and supportive people. The objective of this evaluation was to estimate the cost of implementation and the cost-effectiveness of a person-level intervention shown to increase medication adherence. To estimate the intervention costs, a direct measure microcosting approach was used after key informant interviews with project champions and a review of implementation expenditures. Cost-effectiveness was calculated by comparing the incremental implementation costs and healthcare costs associated with nonadherence to the incremental percent adherent, defined as the percent of patients who took greater or equal to 85% of their medication doses, for each pairwise comparison. The intervention was low-resource to implement, costing approximately $520 to implement per patient, and was associated with significant improvements in medication adherence. These implementation costs were more than outweighed by the expected healthcare savings associated with improvements in adherence. This person-level intervention is a low-cost, efficacious intervention associated with significant statistical and clinical improvements in medication adherence in adult kidney transplant recipients.

Common Ground: Primary Care and Specialty Clinicians' Perceptions of E-Consults in the Veterans Health Administration.

OBJECTIVE: The US Department of Veterans Affairs (VA) introduced electronic consultation (e-consult) to increase access to specialty care. The objective of this study was to understand perceptions of e-consults that may be relevant to increasing adoption in the VA. METHODS: Deductive and inductive content analysis of semistructured qualitative telephone interviews with VA primary care practitioners (PCPs), specialists, and specialty division chiefs was performed. Participants were identified based on rates of e-consult in 2016 at the individual and facility level within primary care, hematology, cardiology, gastroenterology, and endocrinology. Interview guide development was informed by the Practical, Robust, Implementation, and Sustainability (PRISM) framework. RESULTS: We interviewed 35 PCPs and 25 specialists working in 36 facilities. Four themes emerged across both PCPs and specialists: (1) e-consults are best suited for certain types of clinical questions; (2) high-quality e-consults include complete background information from the requesting clinician and clear diagnostic or treatment recommendations from the responding clinician; (3) PCPs and specialists perceive e-consults as a novel opportunity to provide efficient, transparent care; and (4) lack of awareness of e-consults hinders adoption despite obvious benefits. CONCLUSIONS: We identified themes that are informative for further adoption of high-quality e-consults in the VA. Educating PCPs and specialty practitioners about the benefits of e-consults, and providing support, such as lists of specialties available for e-consults at the facility are 2 such practices.

The Cost-Effectiveness of Remdesivir for Hospitalized Patients With COVID-19.

OBJECTIVES: This study aimed to estimate the cost-effectiveness of remdesivir, the first novel therapeutic to receive Emergency Use Authorization for the treatment of hospitalized patients with COVID-19, and identify key drivers of value to guide future pricing and reimbursement efforts. METHODS: A Markov model evaluated the cost-effectiveness of remdesivir in patients hospitalized with COVID-19 from a US healthcare sector perspective. A lifetime time horizon captured potential long-term costs and outcomes. Model outcomes included discounted total costs, life-years, and quality-adjusted life-years (QALYs). Remdesivir was modeled as an addition to standard of care and compared with standard of care alone, including dexamethasone for patients requiring respiratory support. COVID-19 hospitalizations were assumed to be reimbursed through a single payment based on the respiratory support received alongside a remdesivir carveout payment in the base case. Sensitivity and scenario analyses identified key drivers. RESULTS: At a unit price of $520 per vial and assuming no survival benefit with remdesivir, the incremental cost-effectiveness was $298 200/QALY for patients with moderate to severe COVID-19 and $1 847 000/QALY for patients with mild COVID-19. Although current data do not support a survival benefit, if one was assumed, the cost-effectiveness estimate was $50 100/QALY for the moderate to severe population and $103 400/QALY for the mild population. Another key driver included the hospitalization payment structure (per diem vs bundled payment). CONCLUSIONS: With the current evidence available, remdesivir's price is too high to align with its expected health gains for hospitalized patients with COVID-19. Results from this study provide a rationale for iterative health technology assessment.

Cost-Effectiveness and Value-Based Pricing of Aducanumab for Patients With Early Alzheimer Disease.

BACKGROUND AND OBJECTIVES: Aducanumab was granted accelerated approval with a conflicting evidence base, near-unanimous Food and Drug Administration Advisory Committee vote to reject approval, and a widely criticized launch price of $56,000 per year. The objective of this analysis was to estimate its cost-effectiveness. METHODS: We developed a Markov model to compare aducanumab in addition to supportive care to supportive care alone over a lifetime horizon. Results were presented from both the health system and modified societal perspective. The model tracked the severity of disease and the care setting. Incremental cost-effectiveness ratios were calculated and a threshold analysis was conducted to estimate at what price aducanumab would meet commonly used cost-effectiveness thresholds. RESULTS: Using estimates of effectiveness based on pooling of data from both pivotal trials, patients treated with aducanumab spent 4 more months in earlier stages of Alzheimer disease. Over the lifetime time horizon, treating a patient with aducanumab results in 0.154 more quality-adjusted life-years (QALYs) gained per patient and 0.201 equal value of life-years gained (evLYG) per patient from the health care system perspective, with additional costs of approximately $204,000 per patient. The incremental outcomes were similar for the modified societal perspective. At the launch price of $56,000 per year, the cost-effectiveness ranged from $1.02 million per evLYG to $1.33 million per QALY gained from the health care system perspective and from $938,000 per evLYG to $1.27 million per QALY gained in the modified societal perspective. The annual price to meet commonly used cost-effectiveness thresholds ranged from $2,950 to $8,360, which represents a discount of 85%-95% off from the annual launch price set by the manufacturer. Using estimates of effectiveness based only on the trial that suggested a benefit, the mean incremental cost was greater than $400,000 per QALY gained. CONCLUSION: Patients treated with aducanumab received minimal improvements in health outcomes at considerable cost. This resulted in incremental cost-effectiveness ratios that far exceeded commonly used value thresholds, even under optimistic treatment effectiveness assumptions. These findings are subject to the substantial uncertainty regarding whether aducanumab provides any true net health benefit, but evidence available currently suggests that an annual price of aducanumab of $56,000 is not in reasonable alignment with its clinical benefits.

Performing Cost-Effectiveness Analyses to Support Policy Making: Key Lessons From the Assessment of Aducanumab.

The purpose of this article is to describe the process and the methods of cost-effectiveness analysis for clinicians interested in joining or leading aspects of this branch of evidence-based research. Cost-effectiveness is a useful tool for policymakers and is considered a starting point for discussions of fair pricing. Clinicians are important members of teams conducting cost-effectiveness analyses, particularly as it relates to integrating their clinical expertise into the decisions around the design and conduct of the analysis. Their input is essential in assuring that models adequately reflect clinical practice and are informed by expert judgments of how existing data can best be interpreted to build a comprehensive analysis of the clinical and economic outcomes of different treatment options. We illustrate specific contributions that clinicians are well positioned to make in these teams using a recent cost-effectiveness analysis of aducanumab that was conducted to support fair drug pricing. While discussing these contributions, we explain key components of a cost-effectiveness analysis, such as time horizon, health states, and perspective, to support understanding of the methods of cost-effectiveness by the clinical researchers and to promote a common dialogue among these multidisciplinary teams.

A Systems-Based Morbidity and Mortality Conference Was Associated With a Transient Reduction in ECG Completion Times.

BACKGROUND AND OBJECTIVES: During its monthly morbidity and mortality conference (MMC), the University of Colorado Division of Cardiology reviewed a "near-miss" patient safety event involving the delayed completion of a Stat-priority (ie, statim, meaning high priority) electrocardiogram (ECG). Because critical and interprofessional stakeholders participated in the conference, we hypothesized that the MMC would be associated with reduced ECG completion times. METHODS: Data were collected for in-hospital ECGs performed at the University of Colorado Hospital between January 1, 2017, and June 30, 2018. An interrupted time series analysis was used to estimate the immediate and ongoing impact of the MMC (held on February 28, 2018) on ECG completion times, stratified by order priority (Stat, Now, or Routine). The percentage of delayed Stat-priority ECGs was analyzed as a secondary outcome. RESULTS: Before the MMC, ECG completion times were stable for all order priorities ( P > .2), but the proportion of delayed Stat-priority ECGs increased from 5% in January 2017 to 20% in February 2018 ( P < .01). The MMC was associated with an immediate reduction in average daily ECG completion times for Routine (-18.4 minutes, P = .03) and Now (-8 minutes, P = .024) priority ECGs. No reduction was seen for Stat ECGs ( P = .97), though the percentage of delayed Stat ECGs stopped increasing ( P = .63). In the post-MMC period, completion times for Routine-priority ECGs increased and approached pre-MMC levels. CONCLUSIONS: The MMC was associated with an immediate, but temporary, improvement in ECG completion times. Although the observed clinical benefit of the MMC is novel, these data support the need for more durable reforms to sustain initial improvements.

The effectiveness and value of aducanumab for Alzheimer's disease.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Sun Life Financial, uniQure, and United Healthcare. Whittington, Rind, and Pearson are employed by ICER. Through their affiliated institutions, Synnott and Lin received funding from ICER for the work described in this summary. In addition, Synnott reports support from Biogen for the Tufts Medical Center Cost-Effectiveness Analysis Registry, which is maintained by the Center for the Evaluation of Value and Risk in Health.

Health costs of women with chronic overlapping pain conditions by opioid and complementary and integrative health use.

OBJECTIVE: To estimate differences in average annual health care expenditures of adult women with chronic overlapping pain conditions (COPCs) by pain treatment modality as follows: (1) no long-term opioid or complementary and integrative health (CIH) use; (2) CIH only use; (3) long-term opioid only use; and (4) long-term opioid and CIH use. DATA SOURCE: Cross-sectional Medical Expenditure Panel Survey data (2012-2016). STUDY DESIGN: We estimated differences between average annual expenditures of adult women with COPCs by their use of long-term opioids and CIH modalities. Generalized linear regression with a log link function was used to estimate adjusted marginal effects in annual expenditures. The distribution family was chosen based on Modified Park Tests. We controlled for pain severity, patient demographic characteristics, physical limitations, comorbidities, mental health, insurance status, physical therapy use, and census region. We also employed propensity-score based marginal mean weighting through stratification to balance our treatment groups on observed covariates. DATA COLLECTION/EXTRACTION METHODS: We identified adult women (>17 years) with one or more self-reported COPC using 3-digit International Classification of Diseases (ICD)-9/10-Clinical Modification (CM) codes (N = 9169) and categorized their use of CIH and long-term opioids. PRINCIPAL FINDINGS: Compared to women without long-term opioid or CIH use, CIH only use was significantly associated with lower inpatient expenditures (-$947 [-$1699, -$196]; p-value < 0.01), higher office-based expenditures ($1345 [$944, $1746]; p-value < 0.001), and higher patient out-of-pocket expenditures ($628 [$409, $848]; p-value < 0.001). Long-term opioid use, alone or in combination with CIH, was significantly associated with higher expenditures (p-value < 0.05) in total and across all utilization categories compared to women without any long-term opioid or CIH use. CONCLUSIONS: Our results indicate that CIH treatment approaches for chronic pain have the potential to be utilized without increasing overall costs. Future research should further examine the role of CIH modalities in achieving cost-effective pain management that reduces avoidable opioid use.

The influence of a Sprint optimization and training intervention on time spent in the electronic health record (EHR).

OBJECTIVE: We report the influence of Sprint electronic health record (EHR) training and optimization on clinician time spent in the EHR. MATERIALS AND METHODS: We studied the Sprint process in one academic internal medicine practice with 26 providers. Program offerings included individualized training sessions, and the ability to clean up, fix, or build new EHR tools during the 2-week intervention. EHR usage log data were available for 24 clinicians, and the average clinical full-time equivalent was 0.44. We used a quasi-experimental study design with an interrupted time series specification, with 8 months of pre- and 12 months of post-intervention data to evaluate clinician time spent in the EHR. RESULTS: We discovered a greater than 6 h per day reduction in clinician time spent in the EHR at the clinic level. At the individual clinician level, we demonstrated a time savings of 20 min per clinician per day among those who attended at least 2 training sessions. DISCUSSION: We can promote EHR time savings for clinicians who engage in robust EHR training and optimization programs. To date, programs have shown a positive correlation between participation and subjective EHR satisfaction, efficiency, or time saved. The impact of EHR training and optimization on objective time savings remains elusive. By measuring time in the EHR, this study contributes to an ongoing conversation about the resources and programs needed to decrease clinician EHR time. CONCLUSIONS: We have demonstrated that Sprint is associated with time savings for clinicians for up to 6 months. We suggest that an investment in EHR optimization and training can pay dividends in clinician time saved.

Patient and Payer Preferences for Additional Value Criteria.

Background: Defining the value of healthcare is an elusive target, and depends heavily on the decision context and stakeholders involved. Cost-utility analysis and the quality-adjusted life year (QALY) have become the method and value definition of choice for traditional value judgements in coverage and pricing decisions. Other criteria that may influence value are often not measured and therefore omitted from value assessments, or are only used to qualitatively contextualize assessments. The objective of this study was to engage two key stakeholders; patients and payers to elicit and rank the importance of additional value criteria, potentially assessed in Multiple Criteria Decision Analysis (MCDA). Methods: This study consisted of a focus group with cancer patients (n = 7), including follow-up questions through an electronic survey, and in-depth phone interviews with payers (n = 5). Results: For payers, value equated either with criteria that provided tangible benefits (from their perspective) such as new treatment options that respond to serious unmet need. For patients, population-level value equated to options that would potentially benefit them in the future and the value of hope. However, these criteria were seen by payers as difficult to measure and incorporate into objective decision making. Limitations: The findings from this study are primarily limited due to generalizability. Due to the small sample size, it was outside the scope of this study to calculate a weight for each criterion that could be used as part of a quantitative MCDA. Conclusion: MCDA, with particular attention to qualitative aspects, is an avenue to incorporate these additional criteria into value assessments, as well as provide an opportunity for reflecting the patient's preferences in assessing the value of a treatment.

Economic and clinical burden of comorbidities among patients with acromegaly.

OBJECTIVE: Acromegaly is a rare, pituitary hormonal disorder that requires improved awareness worldwide. The objective of this analysis was to quantify the clinical and economic burden of comorbidities for patients with acromegaly and examine the influence of biochemical control on these outcomes. STUDY DESIGN: Markov cohort decision analytic model consisting of two states, including alive (with and without comorbidity) and dead. METHODS: A cohort of patients with acromegaly who had achieved biochemical control, a cohort of patients with acromegaly who had not achieved biochemical control, and a cohort of individuals from the general US population were tracked over a lifetime time horizon. The model tracked the proportion of the alive population that had each comorbidity based on age, sex, presence of acromegaly, and biochemical control status. The proportion of patients with each acromegaly-associated comorbidity were assigned comorbidity-associated costs, disutilities, and increased risk of mortality. RESULTS: Compared with the general population, controlled acromegaly resulted in $192,000 additional comorbidity-related costs, 0.7 fewer years of life, 2.9 fewer quality-adjusted life years, and 1.1 more comorbidities across the remaining lifespan. Compared with the general population, uncontrolled acromegaly resulted in $285,000 additional comorbidity-related costs, 0.9 fewer years of life, 4.2 fewer quality-adjusted life years, and 1.6 more comorbidities across the remaining lifespan. CONCLUSIONS: Achieving biochemical control is associated with improvements in cost, quality of life, and mortality, albeit not to the level of the general population. A multimodal treatment strategy including biochemical control and management of comorbidities is necessary to improve patient outcomes.

Does Cost-Effectiveness Analysis Overvalue Potential Cures? Exploring Alternative Methods for Applying a "Shared Savings" Approach to Cost Offsets.

OBJECTIVES: To evaluate alternative methods to calculate and/or attribute economic surplus in the cost-effectiveness analysis of single or short-term therapies. METHODS: We performed a systematic literature review of articles describing alternative methods for cost-effectiveness analysis of potentially curative therapies whose assessment using traditional methods may suggest unaffordable valuations owing to the magnitude of estimated long-term quality-adjusted life-year (QALY) gains or cost offsets. Through internal deliberation and discussion with staff at the Health Technology Assessment bodies in England and Canada, we developed the following 3 alternative methods for further evaluation: (1) capping annual costs in the comparator arm at $150 000 per year; (2) "sharing" the economic surplus with the health sector by apportioning only 50% of cost offsets or 50% of cost offsets and QALY gains to the value of the therapy; and (3) crediting the therapy with only 12 years of the average annual cost offsets or cost offsets and QALY gains over the lifetime horizon. The impact of each alternative method was evaluated by applying it in an economic model of 3 hypothetical condition-treatment scenarios meant to reflect a diversity of chronicity and background healthcare costs. RESULTS: The alternative with greatest impact on threshold price for the fatal pediatric condition spinal muscular atrophy type 1 was the 12-year cutoff scenario. For a hypothetical one-time treatment for hemophilia A, capping cost offsets at $150 000 per year had the greatest impact. For chimeric antigen receptor T-cell treatment of non-Hodgkin's lymphoma, capping cost offsets or using 12-year threshold had little impact, whereas 50% sharing of surplus including QALY gains and cost offsets greatly reduced threshold pricing. CONCLUSIONS: Health Technology Assessment bodies and policy makers will wrestle with how to evaluate single or short-term potentially curative therapies and establish pricing and payment mechanisms to ensure sustainability. Scenario analyses using alternative methods for calculating and apportioning economic surplus can provide starkly different assessment results. These methods may stimulate important societal dialogue on fair pricing for these novel treatments.