Value in Health最新文献

Objectives: To estimate the excess formal social costs or direct non-healthcare costs of dementia-related neuropsychiatric symptoms (NPS).

Methods: The presence of dementia, NPS, antipsychotic and antidepressant use, somatic and psychiatric comorbidities, and formal social benefits were studied in a regionwide cohort of all 60-year-old and older individuals. A random forest-based algorithm identified NPS, and 2-part regression models and entropy balance were used.

Results: Of the 215 859 individuals, 7553 (3.50%) had dementia, 74 845 (34.7%) had some NPS, and 20 787 (9.63%) received long-term care benefits. Notably, nearly two-thirds (63.9%) of people with dementia received benefits. The probability of having social costs varied markedly with age (odds ratio [OR] 12.28 [10.17-14.82] for >90-year-olds category), and the presence of dementia (OR 7.36 [6.13-8.84]) or NPS (OR 3.23 [2.69-3.88]). NPS (relative change [RC] 1.39 [1.31-1.49]) and dementia (RC 1.32 [1.24-1.41]) were associated with higher average benefit costs. Low socioeconomic status was significantly associated with both a higher probability of receiving benefits (OR 1.52 [1.38-1.68]) and higher costs of their provision (RC 1.18 [1.15-1.21]).

Conclusions: The burden of caring for NPS is greater than that indicated by the literature as these symptoms multiply the social costs of dementia by more than 3, owing to the greater use of residential care and formal coverage reaching more patients than that indicated by the literature. The greater presence of dementia and NPS in the population of lower socioeconomic status indicates an inequality in health attenuated by greater use of social benefits.

Objectives: For individuals living with rare neurodevelopmental disorders, particularly those who are at the most severe end of the spectrum, standardized outcome measures may lack the sensitivity to capture small but meaningful changes. Personalized endpoints such as goal attainment scaling (GAS) allow the assessment of treatment response across variable baseline states and disease manifestations and, thus, provide a highly sensitive measure of efficacy. The current study tested the feasibility of using GAS in rare SCN2A-associated developmental and epileptic encephalopathy (SCN2A-DEE).

Methods: The caregivers of 10 individuals with SCN2A-DEE (M_age = 8.2 years, SD = 5.62, range 3.4-20.4; N_male = 8) took part in in-person goal setting and remote follow-up interviews facilitated by 4 clinical researchers. Implementation was standardized using clinician training, patient orientation, and an electronic data capture platform, GoalNav®; surveys were used to evaluate implementation.

Results: All 10 caregivers completed the goal-setting interviews and were able to set scale 3 goals, and assess attainment levels at follow-up interviews. The mean (SD) times to conduct the goal setting and follow-up interviews were 59.4 (14.5) and 18.4 (10.5) minutes, respectively. Participants set a variety of goals relating to communication (n = 10), feeding (n = 4), gross and fine motor abilities (n = 6), behavior (n = 5), gastrointestinal function (n = 3), sleep (n = 1), and seizures (n = 1). Data completeness, interview time, and the presence of high-quality goals (29/30) indicated the feasibility of using GAS in this population, whereas survey responses indicated its acceptability.

Conclusions: This pilot project provided evidence supporting the feasibility of GAS as a method for assessing treatment outcomes for patients with rare neurodevelopmental disorders.

Objectives: Psychological distress is a state of emotional suffering and discomfort that often manifests as anxiety, depression, or other mental health symptoms, impairing daily functioning and hindering concentration, relationships, and work or school performance. We aimed to examine the disutility associated with psychological distress.

Methods: We used longitudinal data obtained from the Household, Income, and Labour Dynamics in Australia Survey. We measured health state utility values (HSUVs) using the Short-Form 6-Dimension (SF-6D) Utility Index and psychological distress using the Kessler Psychological Distress Scale. We used longitudinal fixed-effects regression model to examine the effects of psychological distress on HSUVs.

Results: The results from fixed-effects panel regression models indicate a negative effect of psychological distress on HSUVs. We found that moderate psychological distress (β = -0.057, 95% CI -0.059 to -0.055) and high psychological distress (β = -0.123, 95% CI -0.126 to -0.121) led to a significant reduction in HSUVs. These findings hold across different subsamples, such as age, gender, and race.

Conclusions: By quantifying the reduction in HSUVs due to psychological distress, our study provides valuable data for future economic evaluations of healthcare interventions. The evidence generated from future economic evaluations will assist policymakers in making informed decisions about the cost-effective interventions for treating psychological distress.

Objectives: Between 2015 and 2017, 41% of National Institute for Health and Care Excellence (NICE) cancer single-technology appraisal (STA) decisions relied upon immature survival data. This occurs when clinical trials that form the evidence base in support of new or existing technologies suffer from limited follow-up. During this period, NICE did not negatively recommend any cancer technologies that used immature data. This suggests a potential incentive to submit to NICE with immature data to avoid rejection. Using immature survival data in cost-effectiveness evaluations has resulted in significantly different conclusions compared with cost-effectiveness reestimations using matured data. We assessed the reliance on immature survival data in NICE decision making of cancer treatments, appraised after 2017.

Methods: A structured literature review of NICE cancer STAs published between 2018 and 2022 was conducted. The relationship between data maturity and NICE recommendations was assessed, and the extent to which past decisions were later reviewed was explored.

Results: 56% (n = 57) of NICE's cancer recommendations relied upon immature survival data. Fifty-four percent (n = 31) of these received a positive recommendation, 39% (n = 22) were placed into the Cancer Drugs Fund (CDF), and 7% (n = 4) received a negative recommendation. STAs with mature data received a similar proportion of negative recommendations. Only 1 non-CDF recommendation based on immature data was reappraised using updated survival data.

Conclusion: The majority of NICE cancer technology decisions are based on immature survival data and receive positive recommendations. Non-CDF decisions are unlikely to be reappraised. Consequently, many technologies could receive an inappropriate recommendation based on immature data and not be subsequently rectified.

Objectives: We aimed to provide a comprehensive summary, synthesis, and appraisal of minimally important difference (MID) estimates for EQ-5D instruments.

Methods: We conducted a systematic search using relevant terms related to "minimally/clinically, meaningful/ important difference/change" and "EQ-5D" in 6 major databases, including MEDLINE, Embase, PsycINFO, CINAHL, Scopus, and Cochrane Library (up to January 2023). We included studies that provided at least 1 original MID estimate for the EQ-5D.

Results: A total of 90 studies reporting 840 MID estimates were included. MID estimates for the EQ-5D-3L index score ranged from 0.075 to 0.8 using distribution-based approaches (239 estimates; 20 studies), from 0.003 to 0.72 using anchor-based approaches (189 estimates; 43 studies), and from 0.038 to 0.082 using instrument-defined approaches (4 estimates; 1 study). For the EQ-5D-5L, MID estimates ranged from 0.023 to 0.115 using distribution-based approaches (17 estimates; 12 studies), from 0.01 to 0.41 using anchor-based approaches (97 estimates; 15 studies), and from 0.037 to 0.101 using instrument-defined approaches (62 estimates; 8 studies). For the EQ visual analog scale, MID estimates ranged from 0.96 to 16.6 using distribution-based approaches (87 estimates; 14 studies) and from 0.42 to 51.0 using anchor-based approaches (84 estimates; 24 studies). MID estimates varied by underlying clinical conditions, baseline scores, and direction of change.

Conclusions: A wide range of MID estimates for EQ-5D instruments were identified, highlighting the variability of MID across populations, estimation methods, direction of change, baseline scores, and EQ-5D versions. These factors should be carefully considered when selecting an appropriate MID for interpreting EQ-5D scores.

Objectives: To assess the impact of a parallel regulatory and reimbursement process on (1) direction of funding decisions, (2) time lag until funding recommendation, and (3) type of evidence submitted, for cancer medicines.

Methods: Public regulatory and reimbursement decision documents were reviewed for cancer medicines considered by the Pharmaceutical Benefits Advisory Committee since the introduction of parallel processing. Medicine-indication pairs were identified from these documents and data extracted on the type and quality of evidence submitted, funding decisions, and timelines, by type of review process. Associations were explored using univariate and multivariable logistic regression analysis.

Results: A total of 182 cases were selected from the 1590 screened. Compared with sequential evaluation, a higher proportion of parallel review submissions presented a cost-effectiveness/cost-utility analysis (67.0% vs 79.7% P < .01) and used the same pivotal evidence as used for market authorization (77.5% vs 90.0%, P = .08). There was no difference in the quality of supporting evidence and other decision-relevant predictors used for either process. Submissions undergoing parallel review were not more likely to receive a negative public funding decision (adjusted odds ratio 0.46; 95% CI 0.14-1.39; P = .17). Under parallel processing, the lag between drug registration and Pharmaceutical Benefits Advisory Committee funding recommendation was drastically reduced (67 weeks) when compared with the sequential process.

Conclusions: The type of evidence supplied to regulators and health technology assessment bodies and the funding decisions that ensue have not been affected by the introduction of parallel processing in Australia. The parallel process has, however, drastically reduced the time taken to the initial funding decision.

Objectives: Bundled payments (BPs) are increasingly being adopted to enable the delivery of high-value care. For BPs to reach their goals, accounting for differences in patient risk profiles (PRPs) predictive of spending is crucial. However, insight is lacking into how this is done in practice. This study aims to fill this gap.

Methods: We conducted a systematic review of literature published until February 2024, focusing on BP initiatives in the Organization for Economic Cooperation and Development countries. We collected data on initiatives' general characteristics, details on the (stated reasons for) approaches used to account for PRP, and suggested improvements. Patterns within and across initiatives were analyzed using extraction tables and thematic analysis.

Results: We included 95 documents about 17 initiatives covering various conditions and procedures. Across these initiatives, patient exclusion (n = 14) and risk adjustment (n = 12) of bundle prices were the most applied methods, whereas risk stratification was less common (n = 3). Most authors stated mitigating perverse incentives as the primary reason for PRP accounting. Commonly used risk factors included comorbidities and sociodemographic and condition/procedure-specific characteristics. Our findings show that, despite increasingly sophisticated approaches over time, key areas for improvement included better alignment with value and equity goals, and enhanced data availability for more comprehensive corrections for relevant risk factors.

Conclusions: BP initiatives use various approaches to account for PRP differences. Despite a trend toward more sophisticated approaches, most remain basic with room for improvement. To enable cross-initiative comparisons and learning, it is important that stakeholders involved in BPs be transparent about the (reasons for) design choices made.