Do Quality-Adjusted Life Years Discriminate Against the Elderly? An Empirical Analysis of Published Cost-Effectiveness Analyses.

OBJECTIVES: Critics of quality-adjusted life-years argue that it discriminates against older individuals. However, little empirical evidence has been produced to inform this debate. This study aimed to compare published cost-effectiveness analyses (CEAs) on patients aged ≥65 years and those aged <65 years. METHODS: We used the Tufts Cost-Effectiveness Analysis Registry to identify CEAs published in MEDLINE between 1976 and 2021. Eligible CEAs were categorized according to age (≥65 years vs <65 years). The distributions of incremental cost-effectiveness ratios (ICERs) were compared between the age groups. We used logistic regression to assess the association between age groups and the cost-effectiveness conclusion adjusted for confounding factors. We conducted sensitivity analyses to explore the impact of mixed age and age-unknown groups and all ICERs from the same CEAs. Subgroup analyses were also conducted. RESULTS: A total of 4445 CEAs categorized according to age <65 years (n = 3784) and age ≥65 years (n = 661) were included in the primary analysis. The distributions of ICERs and the likelihood of concluding that the intervention was cost-effective were similar between the 2 age groups. Adjusted odds ratios ranged from 1.132 (95% CI 0.930-1.377) to 1.248 (95% CI 0.970-1.606) (odds ratio >1 indicating that CEAs for age ≥65 years were more likely to conclude the intervention was cost-effective than those for age <65 years). Sensitivity and subgroup analyses found similar results. CONCLUSION: Our analysis found no systematic differences in published ICERs using quality-adjusted life-years between CEAs for individuals aged ≥65 years and those for individuals aged <65 years.

Improving Food and Drug Administration-Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval.

Policy Points The increasing number of drugs granted accelerated approval by the Food and Drug Administration (FDA) has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We recommend several administrative and legislative approaches for improving FDA-Centers for Medicare and Medicaid Services (CMS) coordination around accelerated-approval drugs, including promoting earlier discussions among the FDA, the CMS, and drug companies; strengthening Medicare's coverage with evidence development program; linking Medicare payment to evidence generation milestones; and ensuring that the CMS has adequate staffing and resources to evaluate new therapies. These activities can help improve the integrity; transparency; and efficiency of approval, coverage, and payment processes for drugs granted accelerated approval. CONTEXT: The Food and Drug Administration (FDA)'s accelerated-approval pathway expedites patient access to promising treatments. However, increasing use of this pathway has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We examined approaches to improve coordination between the FDA and Centers for Medicare and Medicaid Services (CMS) for drugs granted accelerated approval. METHODS: We argue that policymakers have focused on expedited pathways at the FDA without sufficient attention to complementary policies at the CMS. Although differences between the FDA and CMS decisions are to be expected given the agencies' different missions and statutory obligations, procedural improvements can ensure that Medicare beneficiaries have timely access to novel therapies that are likely to improve health outcomes. To inform policy options and recommendations, we conducted semistructured interviews with stakeholders to capture diverse perspectives on the topic. FINDINGS: We recommend ten areas for consideration: clarifying the FDA's evidentiary standards; strengthening FDA authorities; promoting earlier discussions among the FDA, the CMS, and drug companies; improving Medicare's coverage with evidence development program; tying Medicare payment for accelerated-approval drugs to evidence generation milestones; issuing CMS guidance on real-world evidence; clarifying Medicare's "reasonable and necessary" criteria; adopting lessons from international regulatory-reimbursement harmonization efforts; ensuring that the CMS has adequate staffing and expertise; and emphasizing equity. CONCLUSIONS: Better coordination between the FDA and CMS could improve the transparency and predictability of drug approval and coverage around accelerated-approval drugs, with important implications for patient outcomes, health spending, and evidence generation processes. Improved coordination will require reforms at both the FDA and CMS, with special attention to honoring the agencies' distinct authorities. It will require administrative and legislative actions, new resources, and strong leadership at both agencies.

Developing Criteria for Health Economic Quality Evaluation Tool.

OBJECTIVES: Because existing publication guidelines and checklists have limitations when used to assess the quality of cost-effectiveness analysis, we developed a novel quality assessment tool for cost-effectiveness analyses, differentiating methods and reporting quality and incorporating the relative importance of different quality attributes. METHODS: We defined 15 quality domains from a scoping review and identified 72 methods and reporting quality attributes (36 each). After designing a best-worst scaling survey, we fielded an online survey to researchers and practitioners to estimate the relative importance of the attributes in February 2021. We analyzed the survey data using a sequential conditional logit model. The final tool included 48 quality attributes deemed most important for assessing methods and reporting quality (24 each), accompanied by a free and web-based scoring system. RESULTS: A total of 524 participants completed the methodology section, and 372 completed both methodology and reporting sections. Quality attributes pertaining to the "modeling" and "data inputs and evidence synthesis" domains were deemed most important for methods quality, including "structure of the model reflects the underlying condition and intervention's impact" and "model validation is conducted." Quality attributes pertaining to "modeling" and "Intervention/comparator(s)" domains were considered most important for reporting quality, including "model descriptions are detailed enough for replication." Despite its growing prominence, "equity considerations" were not deemed as important as other quality attributes. CONCLUSIONS: The Criteria for Health Economic Quality Evaluation tool allows users to differentiate methods and reporting as well as quantifies the relative importance of quality attributes. Alongside other considerations, it could help assess and improve the quality of cost-effectiveness evidence to inform value-based decisions.

A review of HTA guidelines on societal and novel value elements.

OBJECTIVES: Health technology assessment (HTA) organizations vary in terms of how they conduct assessments. We assess whether and to what extent HTA bodies have adopted societal and novel elements of value in their economic evaluations. METHODS: After categorizing "societal" and "novel" elements of value, we reviewed fifty-three HTA guidelines. We collected data on whether each guideline mentioned each societal or novel element of value, and if so, whether the guideline recommended the element's inclusion in the base case, sensitivity analysis, or qualitative discussion in the HTA. RESULTS: The HTA guidelines mention on average 5.9 of the twenty-one societal and novel value elements we identified (range 0-16), including 2.3 of the ten societal elements and 3.3 of the eleven novel value elements. Only four value elements (productivity, family spillover, equity, and transportation) appear in over half of the HTA guidelines, whereas thirteen value elements are mentioned in fewer than one-sixth of the guidelines, and two elements receive no mention. Most guidelines do not recommend value element inclusion in the base case, sensitivity analysis, or qualitative discussion in the HTA. CONCLUSIONS: Ideally, more HTA organizations will adopt guidelines for measuring societal and novel value elements, including analytic considerations. Importantly, simply recommending in guidelines that HTA bodies consider novel elements may not lead to their incorporation into assessments or ultimate decision making.

Toward comprehensive value assessment for Alzheimer's disease innovations.

INTRODUCTION: Assessing medical technologies for Alzheimer's disease (AD) creates challenges for current methods of value assessment. New value assessment approaches for AD are also needed. METHODS: We adapted concepts from health economics to help guide decision makers to more informed decisions about AD therapies and diagnostics. RESULTS: We propose a value framework based on five categories: perspective, value elements, analysis, reporting, and decision making. AD value assessments should include the perspective of the patient-caregiver dyad. We propose a broader array of value elements than currently used. Analytics and decision methods can synthesize evidence for all elements of value. Decisions should use a "deliberative appraisal" approach informed by the composite evidence and be transparently reported. DISCUSSION: Using the proposed framework, the value of forthcoming innovations for AD may be more thoroughly assessed for and by all stakeholders. It can guide decision makers to carefully consider all relevant elements of value contributing to more holistic and transparent decision making. RESEARCH HIGHLIGHTS: Alzheimer's disease challenges common methods of evaluating medical technology. Using current methods, new AD innovations might not be appropriately valued. Poor value assessments will adversely affect patient access to AD innovations. A full AD value framework expands perspective, elements, analysis, decision-making, reporting.

Alzheimer's disease medication use and adherence patterns by race and ethnicity.

BACKGROUND: We examined racial and ethnic differences in medication use for a representative US population of patients with Alzheimer's disease and related dementias (ADRD). METHODS: We examined cholinesterase inhibitors and memantine initiation, non-adherence, and discontinuation by race and ethnicity, using data from the 2000-2016 Health and Retirement Study linked with Medicare and Medicaid claims. RESULTS: Among newly diagnosed ADRD patients (n = 1299), 26% filled an ADRD prescription ≤90 days and 36% ≤365 days after diagnosis. Among individuals initiating ADRD-targeted treatment (n = 1343), 44% were non-adherent and 24% discontinued the medication during the year after treatment initiation. Non-Hispanic Blacks were more likely than Whites to not adhere to ADRD medication therapy (odds ratio: 1.50 [95% confidence interval: 1.07-2.09]). DISCUSSION: Initiation of ADRD-targeted medications did not vary by ethnoracial group, but non-Hispanic Blacks had lower adherence than Whites. ADRD medication non-adherence and discontinuation were substantial and may relate to cost and access to care. HIGHLIGHTS: Initiation of anti-dementia medications among newly diagnosed Alzheimer's disease and related dementias (ADRD) patients was low in all ethnoracial groups. ADRD medication non-adherence and discontinuation were substantial and may relate to cost and access to care. Compared to Whites, Blacks and Hispanics had lower use, poorer treatment adherence, and more frequent discontinuation of ADRD medication, but when controlling for disease severity and socioeconomic factors, racial disparities diminish. Our findings demonstrate the importance of adjusting for socioeconomic characteristics and disease severity when studying medication use and adherence in ADRD patients.

Cost-effectiveness of exome and genome sequencing for children with rare and undiagnosed conditions.

PURPOSE: This study aimed to estimate the cost-effectiveness of exome sequencing (ES) and genome sequencing (GS) for children. METHODS: We modeled costs, diagnoses, and quality-adjusted life years (QALYs) for diagnostic strategies for critically ill infants (aged <1 year) and children (aged <18 years) with suspected genetic conditions: (1) standard of care (SOC) testing, (2) ES, (3) GS, (4) SOC followed by ES, (5) SOC followed by GS, (6) ES followed by GS, and (7) SOC followed by ES followed by GS. We calculated the 10-year incremental cost per additional diagnosis, and lifetime incremental cost per QALY gained, from a health care perspective. RESULTS: First-line GS costs $15,048 per diagnosis vs SOC for infants and $27,349 per diagnosis for children. If GS is unavailable, ES represents the next most efficient option compared with SOC ($15,543 per diagnosis for infants and $28,822 per diagnosis for children). Other strategies provided the same or fewer diagnoses at a higher incremental cost per diagnosis. Lifetime results depend on the patient's assumed long-term prognosis after diagnosis. For infants, GS ranged from cost-saving (vs all alternatives) to $18,877 per QALY (vs SOC). For children, GS (vs SOC) ranged from $119,705 to $490,047 per QALY. CONCLUSION: First-line GS may be the most cost-effective strategy for diagnosing infants with suspected genetic conditions. For all children, GS may be cost-effective under certain assumptions. ES is nearly as efficient as GS and hence is a viable option when GS is unavailable.

Effect of the 2012 US Preventive Services Task Force Recommendations on Prostate-Specific Antigen Screening in a Medicare Advantage Population.

BACKGROUND: In 2012, the US Preventive Service Task Force revised its recommendations for prostate-specific antigen (PSA) screening from "insufficient evidence" to "do not recommend" for men aged 70-74 while maintaining "do not recommend" for men aged 75+. METHODS: Using the difference-in-difference approach, we evaluated whether the rate of change in the use of low-value PSA screening differed between the control group (men aged 75+, N=7,856,204 person-years) and the intervention group (men aged 70-74, N=5,329,192 person-years) enrolling in the Medicare Advantage plan without a history of prostate cancer within the OptumLabs Data Warehouse claims data (2009-2019). A generalized estimating equation logistic model was specified with independent variables: an intervention group indicator, a pre- and post-period (after 2012 Q2) indicator, index time, and interaction terms. We assumed a 12-month dissemination period. RESULTS: Before the revised recommendation in 2012, the trends did not significantly differ between the 2 age groups with the odds of receiving PSA screening decreasing by 1.2% (95% confidence interval [1.0, 1.4%]) per quarter. However, the odds of receiving PSA screening increased by 3.0% [2.8, 3.2%] per quarter across both groups since the revision. There was no significant additional change in the trend for those aged 70-74 (0.1% [-0.2, 0.5%]). CONCLUSIONS: Although the 2012 US Preventive Service Task Force's recommendations were expected to only change behaviors among men aged 70-74, our analysis found that men aged 70-74 and aged 75+ exhibited similar trends from 2009 to 2019, including the increased use of low-value PSA screening since 2016. Multifaceted efforts to discourage low-value PSA screening would be important for a sustained impact.

Health technology assessment for gene therapies in haemophilia.

INTRODUCTION: Gene therapies are poised to become a new therapeutic option for persons with haemophilia (PwH), having begun to show durable efficacy and safety in clinical trials to date. However, value assessment of gene therapies faces challenges from small populations that preclude randomized clinical trials (RCT), ethical considerations when treating a serious genetic disorder with lifelong consequences, and appropriate endpoint selection that captures the full scope of the disorder and its lifelong complications. SUMMARY: Health technology assessment (HTA) for new haemophilia therapies may require greater flexibility in evidence requirements and recognition of factors that vary across patient populations and health systems, including current standard-of-care, gains in survival and health-related quality-of-life (HRQoL), and reduced replacement factor utilization. It will be important for HTAs to consider limitations of RCTs for gene therapy and to consider intra-patient data as evidence of clinical effectiveness. Despite long-term clinical trials with up to 8 years of follow-up, ongoing uncertainties about durability of effect may be informed by extrapolating clinical data out ∼10 years, using different projections of durability. Beyond objective endpoints, such as Petersson scores or annualized bleed rates, health utilities that accompany gene therapy (e.g., mental health, freedom of choice, peace of mind) should be considered in HTA evaluations, particularly for comparisons with low dose or no prophylaxis. CONCLUSION: HTAs of gene therapies in haemophilia are encouraged to rise to the challenge of filling evidence gaps and conducting assessments. KEY POINTS OF CONSIDERATION: It is important for HTA bodies to consider the limitations to conduct randomized controlled trials for gene therapy and to consider intrapatient data as evidence of comparative effectiveness. Given the uncertainties around the long-term gene therapy use, clinical trial data should be extrapolated ∼10 years, using scenarios that consider different durations of effect. The major value drivers in a model, in addition to drug pricing itself, will be based on assumptions about duration of effect and savings/cost offsets from reduced use of replacement therapy. Assessment methodologies and modelling configurations need to evolve to fully capture the value of gene therapy, including patient meaningful outcomes, in a validated and quantitative fashion. Regardless of payment system archetype, the intersection between NGOs, the clinical community's voice, HTA willingness to collaborate, and alignment with regulatory acceptance of benefit is critical. [Correction added on 21 February 2022, after first online publication: the 'Key Points of Consideration' have been added in this version.].

The History and Future of the "ISPOR Value Flower": Addressing Limitations of Conventional Cost-Effectiveness Analysis.

OBJECTIVES: Since its publication as part of the 2018 ISPOR Special Task Force (STF) on US Value Assessments, the "ISPOR value flower," with its petals highlighting elements that may be overlooked or underappreciated in conventional drug value assessments, has been discussed and debated. We review the history of the value flower, describe recent developments, and consider implications for future value assessments. METHODS: We discuss various antecedents to the value flower, as well as conceptual and empirical articles published in the past 4 years. RESULTS: Since the publication of the ISPOR STF report, researchers have provided more rigorous theoretical and mathematical foundations for certain novel value elements (eg, severity of illness, value of insurance, value of hope) through "generalized risk-adjusted cost-effectiveness analysis," which incorporates risk aversion in people's preferences and uncertainty in treatment outcomes. Empirical estimates are also emerging to support key elements, such as insurance value, real option value, value of hope, and value of knowing. Although health technology assessment bodies have applied or are considering certain elements (eg, severity modifiers to cost-effectiveness thresholds), other elements have yet to gain traction. CONCLUSIONS: Five years after the STF began its work, the development of novel value measures continues to evolve. Although it is encouraging to see supporting empirical studies emerging, more are needed. Additional efforts are also needed to illustrate how the estimates can be used in the deliberative processes that are integral to health technology assessments.

A Systematic Review of Economic Evaluations of COVID-19 Interventions: Considerations of Non-Health Impacts and Distributional Issues.

OBJECTIVES: This study aims to conduct a systematic review of economic evaluations of COVID-19 interventions and to examine whether and how these studies incorporate non-health impacts and distributional concerns. METHODS: We searched the National Institutes of Health's COVID-19 Portfolio as of May 20, 2021, and supplemented our search with additional sources. We included original articles, including preprints, evaluating both the health and economic effects of a COVID-19-related intervention. Using a pre-specified data collection form, 2 reviewers independently screened, reviewed, and extracted information about the study characteristics, intervention types, and incremental cost-effectiveness ratios (ICERs). We used an Impact Inventory to catalog the types of non-health impacts considered. RESULTS: We included 70 articles, almost half of which were preprints. Most articles (56%) included at least one non-health impact, but fewer (21%) incorporated non-economic consequences. Few articles (17%) examined subgroups of interest. After excluding negative ICERs, the median ICER for the entire sample (n = 243 ratios) was $67,000/quality-adjusted life-year (QALY) (interquartile range [IQR] $9000-$893,000/QALY). Interventions including a pharmaceutical component yielded a median ICER of $93,000/QALY (IQR $4000-$7,809,000/QALY), whereas interventions including a non-pharmaceutical component were slightly more cost-effective overall with a median ICER of $81,000/QALY (IQR $12,000-$1,034,000/QALY). Interventions reported to be highly cost-effective were treatment, public information campaigns, quarantining identified contacts/cases, canceling public events, and social distancing. CONCLUSIONS: Our review highlights the lack of consideration of non-health and distributional impacts among COVID-19-related economic evaluations. Accounting for non-health impacts and distributional effects is essential for comprehensive assessment of interventions' value and imperative for generating cost-effectiveness evidence for both current and future pandemics.

Do Cost-Effectiveness Analyses Account for Drug Genericization? A Literature Review and Assessment of Implications.

OBJECTIVES: We investigated how health technology assessment (HTA) organizations around the world have handled drug genericization (an allowance for future generic drug entry and subsequent drug price declines) in their guidelines for cost-effectiveness analyses (CEAs). We also analyzed a large sample of published CEAs to examine prevailing practices in the field. METHODS: We reviewed 43 HTA guidelines to determine whether and how they addressed drug genericization in their CEAs. We also selected a sample of 270 US-based CEAs from the Tufts Medical Center's CEA Registry, restricting the sample to studies on pharmaceuticals published from 1991 to 2019 and to analyses taking a lifetime time horizon. We determined whether each CEA examined genericization (and if so, whether in base case or sensitivity analyses), and how inclusion of genericization influenced the estimated incremental cost-effectiveness ratios. RESULTS: Fourteen (33%) of the 43 HTA guidelines mention genericization for CEAs and 4 (9%) recommend that base case analyses include assumptions about future drug price changes due to genericization. Most published CEAs (95%) do not include assumptions about future generic prices for intervention drugs. Only 2% include such assumptions about comparator drugs. Most studies (72%) conduct sensitivity analyses on drug prices unrelated to genericization. CONCLUSIONS: The omission of assumptions about genericization means that CEAs may misrepresent the long run opportunity costs for drugs. The field needs clearer guidance for when CEAs should account for genericization, and for the inclusion of other price dynamics that might influence a drug's cost-effectiveness.

The Impact of Broader Value Elements on Cost-Effectiveness Analysis: Two Case Studies.

OBJECTIVES: This study aimed to explore the impact of including broader value elements in cost-effectiveness analyses by presenting 2 case studies, one on human papillomavirus (HPV) infection and one on early-stage Hodgkin's lymphoma (ESHL). METHODS: We identified broader value elements (eg, patient and caregiver time, spillover health effects, productivity) from the Second Panel's Impact Inventory and the ISPOR Special Task Force's value flower. We then evaluated the cost-effectiveness of HPV vaccination versus no vaccination (case 1) and combined modality therapy (CMT) versus chemotherapy alone for treatment of adult ESHL (case 2) using published simulation models. For each case study, we compared incremental cost-effectiveness ratios considering health sector impacts only (the "base-case" scenario) with incremental cost-effectiveness ratios incorporating broader value elements. RESULTS: For vaccination of US girls against HPV before sexual debut versus no vaccination, the base-case result was $38 334 per disability-adjusted life-year averted. Including each broader value element made cost-effectiveness progressively more favorable, with HPV vaccination becoming cost-saving (ie, reducing costs and averting more disability-adjusted life-years) when the analysis incorporated productivity costs. For CMT versus chemotherapy alone in patients with ESHL, the base-case result indicated that CMT was cost-saving. Including all elements made this treatment's net monetary benefits (the sum of its averted resource costs and the net value of its health impacts) less favorable, even as the contribution from CMT's near-term health benefits grew. CONCLUSIONS: Including broader value elements can substantially influence cost-effectiveness ratios, although the direction and the magnitude of their impact can differ across interventions and disease context.

Dementia Diagnosis Disparities by Race and Ethnicity.

BACKGROUND: Dementia is often underdiagnosed and this problem is more common among some ethnoracial groups. OBJECTIVE: The objective of this study was to examine racial and ethnic disparities in the timeliness of receiving a clinical diagnosis of dementia. RESEARCH DESIGN: This was a prospective cohort study. SUBJECTS: A total of 3966 participants age 70 years and above with probable dementia in the Health and Retirement Study, linked with their Medicare and Medicaid claims. MEASURES: We performed logistic regression to compare the likelihood of having a missed or delayed dementia diagnosis in claims by race/ethnicity. We analyzed dementia severity, measured by cognition and daily function, at the time of a dementia diagnosis documented in claims, and estimated average dementia diagnosis delay, by race/ethnicity. RESULTS: A higher proportion of non-Hispanic Blacks and Hispanics had a missed/delayed clinical dementia diagnosis compared with non-Hispanic Whites (46% and 54% vs. 41%, P<0.001). Fully adjusted logistic regression results suggested more frequent missed/delayed dementia diagnoses among non-Hispanic Blacks (odds ratio=1.12; 95% confidence interval: 0.91-1.38) and Hispanics (odds ratio=1.58; 95% confidence interval: 1.20-2.07). Non-Hispanic Blacks and Hispanics had a poorer cognitive function and more functional limitations than non-Hispanic Whites around the time of receiving a claims-based dementia diagnosis. The estimated mean diagnosis delay was 34.6 months for non-Hispanic Blacks and 43.8 months for Hispanics, compared with 31.2 months for non-Hispanic Whites. CONCLUSIONS: Non-Hispanic Blacks and Hispanics may experience a missed or delayed diagnosis of dementia more often and have longer diagnosis delays. When diagnosed, non-Hispanic Blacks and Hispanics may have more advanced dementia. Public health efforts should prioritize racial and ethnic underrepresented communities when promoting early diagnosis of dementia.

An Evidence Review of Low-Value Care Recommendations: Inconsistency and Lack of Economic Evidence Considered.

BACKGROUND: Low-value care, typically defined as health services that provide little or no benefit, has potential to cause harm, incur unnecessary costs, and waste limited resources. Although evidence-based guidelines identifying low-value care have increased, the guidelines differ in the type of evidence they cite to support recommendations against its routine use. OBJECTIVE: We examined the evidentiary rationale underlying recommendations against low-value interventions. DESIGN: We identified 1167 "low-value care" recommendations across five US organizations: the US Preventive Services Task Force (USPSTF), the "Choosing Wisely" Initiative, American College of Physicians (ACP), American College of Cardiology/American Heart Association (ACC/AHA), and American Society of Clinical Oncology (ASCO). For each recommendation, we classified the reported evidentiary rationale into five groups: (1) low economic value; (2) no net clinical benefit; (3) little or no absolute clinical benefit; (4) insufficient evidence; (5) no reason mentioned. We further investigated whether any cited or otherwise available cost-effectiveness evidence was consistent with conventional low economic value benchmarks (e.g., exceeding $100,000 per quality-adjusted life-year). RESULTS: Of the identified low-value care recommendations, Choosing Wisely contributed the most (N=582, 50%), followed by ACC/AHA (N=250, 21%). The services deemed "low value" differed substantially across organizations. "No net clinical benefit" (N=428, 37%) and "little or no clinical benefit" (N=296, 25%) were the most commonly reported reasons for classifying an intervention as low value. Consideration of economic value was less frequently reported (N=171, 15%). When relevant cost-effectiveness studies were available, their results were mostly consistent with low-value care recommendations. CONCLUSIONS: Our study found that evidentiary rationales for low-value care vary substantially, with most recommendations relying on clinical evidence. Broadening the evidence base to incorporate cost-effectiveness evidence can help refine the definition of "low-value" care to reflect whether an intervention's costs are worth the benefits. Developing a consensus grading structure on the strength and evidentiary rationale may help improve de-implementation efforts for low-value care.

Toward Better Data Dashboards for US Drug Value Assessments.

OBJECTIVES: To explore the use of data dashboards to convey information about a drug's value, and reduce the need to collapse dimensions of value to a single measure. METHODS: Review of the literature on US Drug Value Assessment Frameworks, and discussion of the value of data dashboards to improve the manner in which information on value is displayed. RESULTS: The incremental cost per quality-adjusted life-year ratio is a useful starting point for conversation about a drug's value, but it cannot reflect all of the elements of value about which different audiences care deeply. Data dashboards for drug value assessments can draw from other contexts. Decision makers should be presented with well-designed value dashboards containing various metrics, including conventional cost per quality-adjusted life-year ratios as well as measures of a drug's impact on clinical and patient-centric outcomes, and on budgetary and distributional consequences, to convey a drug's value along different dimensions. CONCLUSIONS: The advent of US drug value frameworks in health care has forced a concomitant effort to develop appropriate information displays. Researchers should formally test different formats and elements.

Balancing Value with Affordability: Cell Immunotherapy for Cancer Treatment in the U.S.

Despite the Centers for Medicare and Medicaid Services' recent approval to increase payments for inpatient-delivered chimeric antigen receptor T-cell therapy (CAR-T) for adult lymphoma, reimbursement remains far below the costs of the product and overall treatment of the therapy. We surveyed 92 CAR-T-certified centers in the U.S. to assess the perceived financial viability and related challenges for treating adult patients with lymphoma. Of 92 certified CAR-T centers in the U.S., 20 (22%) directors or chief medical officers responded. More than three quarters of facilities reported treating patients in an inpatient setting, and 60% reported that the majority of their patients were covered under commercial/private insurance. The financial viability rating across centers (median: 62; interquartile range: 48-69; scale 1-100) signals that economic sustainability of institutional programs for adult lymphoma is a concern. These dynamics may limit access to CAR-T for Medicare beneficiaries and lead to greater outpatient use of the therapy, which may limit access for medically complex patients.

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.

Measuring "Fearonomic Effects" in Valuing Therapies: An Application to COVID-19 in China.

OBJECTIVES: To develop a checklist that helps quantify the economic impact associated with fear of contagion and to illustrate how one might use the checklist by presenting a case study featuring China during the coronavirus disease 2019 (COVID-19) outbreak. METHODS: Based on "fearonomic effects," a qualitative framework that conceptualizes the direct and indirect economic effects caused by the fear of contagion, we created a checklist to facilitate empirical estimation. As a case study, we first identified relevant sectors affected by China's lockdown policies implemented just before the Lunar New Year (LNY) week. To quantify the immediate impact, we then estimated the projected spending levels in 2020 in the absence of COVID-19 and compared these projections with actual spending during the LNY week. Data sources used include Chinese and global websites. To characterize uncertainty, we reported upper and lower bound estimates and calculated midpoints for each range. RESULTS: The COVID-19 epidemic is estimated to cost China's economy $283 billion ($196-369 billion), that is, ¥2.0 trillion renminbi (¥1.4-¥2.6 trillion), during the LNY week. Reduced restaurant and movie theater business ($106 [$103-$109] billion, 37.5% [36.4%-38.5%]) and reduced public transportation utilization ($96 [$13-$179] billion dollars, 33.9% [4.6%-63.3%]) explain most of this loss, followed by travel restrictions and the resulting loss of hotel business and tourism ($80.36 billion, 28.4%). CONCLUSION: Our checklist can help quantify the immediate and near-term impact of COVID-19 on a country's economy. It can also help researchers and policy makers consider the broader economic and social consequences when valuing future vaccines and treatments.