Family Spillover Effects: Are Economic Evaluations Misrepresenting the Value of Healthcare Interventions to Society?

The societal impacts of health interventions are seldom incorporated into health economic evaluations, including the impact that illness can have on informal or unpaid caregivers and other family members (i.e., "family spillover effects"). Previous research has demonstrated that by excluding family spillover effects, the value of health interventions may be underestimated on average. In this commentary, we discuss how the inclusion of spillover effects influences how we value interventions and, given the extent to which caregiver/family effects are largely not captured or known, propose ways in which these data could be more systematically collected or estimated and used by researchers. These recommendations include prioritizing data collection alongside clinical trials and patient registries, engaging expert opinion panels, and developing mapping algorithms for estimating caregiver/family utility values from non-preference-based caregiver health-related quality-of-life measures and/or from patient preference-based measures.

Limited role of patient input in specialty drug coverage policies.

BACKGROUND: Despite increased financial contributions towards care, consumers' role in shaping their insurance benefits is unclear. OBJECTIVE: To examine the role played by patient input when US commercial health plans formulate specialty drug coverage policies, along with the benefits and challenges of considering this input. METHODS: We employed a parallel, mixed-methods approach. First, we reviewed health plans' policy development processes as reported on their websites. Second, we reviewed a data set of private health plan coverage decisions for specialty drugs and examined whether the evidence cited in policies included patient-reported outcomes (eg, health-related quality of life endpoints) and patient-based methodological designs (eg, interviews or surveys of patients). Third, we performed a survey (N = 21 respondents) and interviews (N = 5 interviewees) with plan decision-makers to determine the current role of patient input in plan decision-making, and the benefits and challenges of incorporating this data when formulating specialty drug coverage policies. RESULTS: We found that plans do not commonly solicit patient input when developing coverage policies, with only two instances of limited interaction between plans and patients or members. 1,316 (9%) of the studies plans cited in their specialty drug coverage policies included at least one patient-reported endpoint, and 0.4% (N = 62) used a patient-based methodological design. Of studies with patient-based designs, 40 used interviews, 26 included surveys/questionnaires, and one concerned shared decision-making (design categories not mutually exclusive). Almost half of the survey respondents reported having never engaged with patients or members when developing coverage policies. Among respondents who had engaged with patients or members, most reported doing so only rarely. The survey and interviews highlighted various benefits of soliciting patient input, including the value of obtaining a humanistic perspective, and several challenges, including resource requirements and the quality of obtained information. CONCLUSIONS: We found a notable lack of patient and member engagement by commercial health plans when formulating drug coverage policies. Survey respondents and interviewees identified benefits of accounting for patients' and plan members' values and preferences in specialty drug coverage policies, but also reported a number of important challenges to doing so. DISCLOSURES: National Pharmaceutical Council provided funding for this research.

Growth and capacity for cost-effectiveness analysis in Africa.

As economic evaluation becomes increasingly essential to support universal health coverage (UHC), we aim to understand the growth, characteristics, and quality of cost-effectiveness analyses (CEA) conducted for Africa and to assess institutional capacity and relationship patterns among authors. We searched the Tufts Medical Center CEA Registries and four databases to identify CEAs for Africa. After extracting relevant information, we examined study characteristics, cost-effectiveness ratios, individual and institutional contribution to the literature, and network dyads at the author, institution, and country levels. The 358 identified CEAs for Africa primarily focused on sub-Saharan Africa (96%) and interventions for communicable diseases (77%). Of 2,121 intervention-specific ratios, 8% were deemed cost-saving, and most evaluated immunizations strategies. As 64% of studies included at least one African author, we observed widespread collaboration among international researchers and institutions. However, only 23% of first authors were affiliated with African institutions. The top producers of CEAs among African institutions are more adherent to methodological and reporting guidelines. Although economic evidence in Africa has grown substantially, the capacity for generating such evidence remains limited. Increasing the ability of regional institutions to produce high-quality evidence and facilitate knowledge transfer among African institutions has the potential to inform prioritization decisions for designing UHC.

Family and Caregiver Spillover Effects in Cost-Utility Analyses of Alzheimer's Disease Interventions.

BACKGROUND AND OBJECTIVE: Alzheimer's disease or dementia can impose a significant burden on family and other informal caregivers. This study investigated how the inclusion of family/informal caregiver spillover effects in a cost-utility analysis may influence the reported value of Alzheimer's disease/dementia interventions. METHODS: We used PubMed to identify Alzheimer's disease or dementia cost-utility analyses published from 1 January, 2000 to 31 March, 2018. We reviewed and abstracted information from each study using a two-reader consensus process. We investigated the frequency and methods in which family/caregiver spillover costs and health effects were incorporated into cost-utility analyses, and examined how their inclusion may influence the reported incremental cost-effectiveness ratios. RESULTS: Of 63 Alzheimer's disease/dementia cost-utility analyses meeting inclusion criteria, 44 (70%) considered at least some family/caregiver spillover costs or health effects. Thirty-two studies incorporated spillover costs only, two incorporated spillover health effects only, and ten incorporated both. The most common approach for accounting for spillover was adding informal caregiving time costs to patient costs (n = 36) and adding informal caregiver quality-adjusted life-years to patient values (n = 7). In a subset of 33 incremental cost-effectiveness ratio pairs from 19 studies, incorporating spillover outcomes made incremental cost-effectiveness ratios more favorable (n = 15; 45%) or kept the intervention cost saving (n = 13; 39%) in most cases. In fewer cases, including spillover increased incremental cost-effectiveness ratios (n = 2; 6%), kept the intervention dominated [more costs/less quality-adjusted life-years] (n = 2; 6%), or changed incremental cost-effectiveness ratio from dominated to less cost/less quality-adjusted life-years (n = 1; 3%). In 11 cases (33%), adding spillover effects into analyses resulted in a lower incremental cost-effectiveness ratio that crossed a common cost-effectiveness threshold, which could have downstream implications for programs or policies that are adopted based on cost-effectiveness analysis results. DISCUSSION: Most Alzheimer's disease/dementia cost-utility analyses incorporated spillover costs, often as caregiver time costs, but considered spillover health impacts less often. In about 85% of the analyses, including Alzheimer's disease/dementia spillover cost or health effects decreased incremental cost-effectiveness ratios or kept the intervention cost saving. The broader value of an Alzheimer's disease/dementia intervention to society may in some cases be underestimated without considering these spillover effects on family and informal caregivers.

Health utilities and parental quality of life effects for three rare conditions tested in newborns.

BACKGROUND: Measurement of health utilities is required for economic evaluations. Few studies have evaluated health utilities for rare conditions; even fewer have incorporated disutility that may be experienced by caregivers. This study aimed to (1) estimate health utilities for three rare conditions currently recommended for newborn screening at the state or federal level, and (2) estimate the disutility, or spillover, experienced by parents of patients diagnosed with a rare, heritable disorder. METHODS: A stated-preference survey using a time trade-off approach elicited health utilities for Krabbe disease, phenylketonuria, and Pompe disease at varying stages (mild, moderate, severe) and onset of disease symptoms (infancy, childhood, and adulthood). We recruited respondents from a nationally representative community sample (n = 862). Respondents valued disease specific health states in three consecutive question frames: (1) adult health state (> = 18 years of age), (2) child health state (< 18 years of age), and (3) as a parent of a child with a condition (parent spillover state). Corresponding mean utilities were calculated for plausible disease states in adulthood and childhood. Mean disutility was estimated for parental spillover. Predictors of utilities were evaluated using a negative binomial regression model. RESULTS: More severe conditions and infant health states received lower estimated utility and greater estimated disutility among parents. Conditions with the lowest estimated health utilities were severe infantile Pompe disease (0.40, CI: 0.34-0.46) and infantile Krabbe disease (0.37, CI: 0.32-0.43). Disutility was evident for all conditions evaluated (range: 0.07-0.19). CONCLUSIONS: Rare childhood conditions are associated with substantial estimated losses in quality of life. Evidence of disutility among parents further warrants the inclusion of spillover effects in cost-effectiveness analyses. Continued research is needed to assess and measure the effects of childhood disease from a family perspective.

Family Spillover Effects in Pediatric Cost-Utility Analyses.

BACKGROUND: Childhood illness can impose significant costs and health strains on family members, but these are not routinely captured by pediatric economic evaluations. This review investigated how family "spillover effects" related to costs and health outcomes are considered in pediatric cost-utility analyses (CUAs). METHODS: We reviewed pediatric CUAs published between 2000 and 2015 using the Tufts Medical Center Cost-effectiveness Analysis (CEA) Registry and the Pediatric Economic Database Evaluation (PEDE) Registry. We selected studies conducted from the societal perspective and included in both registries. We investigated how frequently family spillover was incorporated into analyses, and how the inclusion of spillover health effects and costs changed CUA results. RESULTS: We found 142 pediatric CUAs meeting inclusion criteria. Of those, 105 (72%) considered either family spillover costs (n = 98 time costs, n = 33 out-of-pocket costs, n = 2 caregiver healthcare costs) or health outcomes (n = 15). Twenty-four studies included 43 pairs of incremental cost-effectiveness ratios (ICERs) with and without spillover. In 19 pairs of ICERs, adding spillover changed the ICER enough to cross a common cost-effectiveness threshold (i.e., $50,000/QALY, $100,000/QALY, $150,000/QALY; values are in 2016 US$). Incorporating spillover generally made interventions more cost-effective (n = 18; 42%), or did not change CUA results enough to cross a threshold (n = 24; 56%). Including family spillover reduced ICERs by 31% ($40,000/QALY) on average. CONCLUSION: Most pediatric CUAs conducted from a societal perspective include family costs but fewer include family health effects. Inclusion of family spillover effects tends to make CUA results more favorable. Future pediatric CUAs should aim to more fully incorporate the family burden of illness.

Palivizumab Prophylaxis for Respiratory Syncytial Virus: Examining the Evidence Around Value.

Respiratory syncytial virus (RSV) infection is the most common cause of lower respiratory tract infection and the leading cause of hospitalization among young children, incurring high annual costs among US children under the age of 5 years. Palivizumab has been found to be effective in reducing hospitalization and preventing serious lower respiratory tract infections in high-risk infants. This paper presents a systematic review of the cost-effectiveness studies of palivizumab and describes the main highlights of a round table discussion with clinical, payer, economic, research method, and other experts. The objectives of the discussion were to (1) review the current state of clinical, epidemiology, and economic data related to severe RSV disease; (2) review new cost-effectiveness estimates of RSV immunoprophylaxis in US preterm infants, including a review of the field's areas of agreement and disagreement; and (3) identify needs for further research.

Comparing the cost-per-QALYs gained and cost-per-DALYs averted literatures.

Background: We examined the similarities and differences between studies using two common metrics used in cost-effectiveness analyses (CEAs): cost per quality-adjusted life year (QALY) gained and cost per disability-adjusted life year (DALY) averted. Methods: We used the Tufts Medical Center CEA Registry, which contains English-language cost-per-QALY gained studies, and the Global Cost-Effectiveness Analysis (GHCEA) Registry, which contains cost-per-DALY averted studies. We examined study characteristics, including intervention type, sponsor, country, and primary disease, and also compared the number of published CEAs to disease burden for major diseases and conditions across geographic regions. Results: We identified 6,438 cost-per-QALY and 543 cost-per-DALY studies published through 2016 and observed rapid growth for both literatures. Cost-per-QALY studies most often examined pharmaceuticals and interventions in high-income countries. Cost-per-DALY studies predominantly focused on infectious disease interventions and interventions in low and lower-middle income countries. We found that while diseases imposing a larger burden tend to receive more attention in the cost-effectiveness analysis literature, the number of publications for some diseases and conditions deviates from this pattern, suggesting "under-studied" conditions (e.g., neonatal disorders) and "over-studied" conditions (e.g., HIV and TB). Conclusions: The CEA literature has grown rapidly, with applications to diverse interventions and diseases.  The publication of fewer studies than expected for some diseases given their imposed burden suggests funding opportunities for future cost-effectiveness research.

A Review of Empirical Analyses of Disinvestment Initiatives.

BACKGROUND: Disinvesting in low-value health care services provides opportunities for investment in higher value care and thus an increase in health care efficiency. OBJECTIVES: To identify international experience with disinvestment initiatives and to review empirical analyses of disinvestment initiatives. METHODS: We performed a literature search using the PubMed database to identify international experience with disinvestment initiatives. We also reviewed empirical analyses of disinvestment initiatives. RESULTS: We identified 26 unique disinvestment initiatives implemented across 11 countries. Nineteen addressed multiple intervention types, six addressed only drugs, and one addressed only devices. We reviewed 18 empirical analyses of disinvestment initiatives: 7 reported that the initiative was successful, 8 reported that the initiative was unsuccessful, and 3 reported that findings were mixed; that is, the study considered multiple services and reported a decrease in the use of some but not others. Thirty-seven low-value services were evaluated across the 18 empirical analyses, for 14 (38%) of which the disinvestment initiative led to a decline in use. Six of the seven studies that reported the disinvestment initiative to be successful included an attempt to promote the disinvestment initiative among participating clinicians. CONCLUSIONS: The success of disinvestment initiatives has been mixed, with fewer than half the identified empirical studies reporting that use of the low-value service was reduced. Our findings suggest that promotion of the disinvestment initiative among clinicians is a key component to the success of the disinvestment initiative.