Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Introduction: Suboptimal medication adherence is common among patients with cardiovascular diseases. We sought evidence on non-pharmacological interventions used to support adherence for patients with hypertension and/or dyslipidemia.

Methods: We searched MEDLINE, EMBASE, MEDLINE In-Process, ClinicalTrials.gov, EUCTR, and conference proceedings from July 2011 to July 2021 to identify trials evaluating effects of health education, phone reminders, or digital interventions on medication adherence or persistence of adult patients with hypertension and/or dyslipidemia. Risk of bias was assessed using the Cochrane Risk of Bias Assessment Tool v2.

Results: Of 64 studies, 62 used health education approaches (e.g. educational interviews, motivational meetings, advice from physicians, and mobile health content), 16 phone reminders (e.g. text reminders, electronic pill-box linked reminders, bi-directional text messaging), and 10 digital applications as interventions (e.g., various self-management applications). All studies assessed medication adherence; only two persistence. Overall, 30 studies (83%) assessing health education approaches alone and 25 (78%) combined with other strategies, 12 (75%) phone reminders and eight studies (80%) digital applications combined with other strategies reported improved medication adherence. Two studies assessing health education approaches reported improved persistence.

Conclusions: Our findings indicate non-pharmacological interventions may positively impact adherence. Therefore, 'beyond the pill' approaches could play a role in preventing cardiovascular diseases.

Objective: This study compared the survival outcomes of non-traumatic intracerebral hemorrhage (ICH) patients with different famotidine administration routes and explored related risk factors.

Methods: Data from ICH patients between 2008-2019 were extracted from the MIMIC-IV database. Survival differences between patients with intravenous (IV) and non-intravenous (Non-IV) famotidine administration were analyzed using Cox analysis and Kaplan-Meier survival curves.

Results: The study included 351 patients, with 109 in the IV group and 84 in the Non-IV group after PSM. Cox analysis revealed that survival was significantly associated with age (HR = 1.031, 95%CI:1.011-1.050, p = 0.002), chloride ions (HR = 1.061, 95%CI:1.027-1.096, p < 0.001), BUN (HR = 1.034, 95%CI:1.007-1.062, p = 0.012), ICP (HR = 1.059, 95%CI:1.027-1.092, p < 0.001), RDW (HR = 1.156, 95%CI:1.030-1.299, p = 0.014), mechanical ventilation (HR = 2.526, 95%CI:1.341-4.760, p = 0.004), antibiotic use (HR = 0.331, 95%CI:0.144-0.759, p = 0.009), and Non-IV route (HR = 0.518, 95%CI:0.283-0.948, p = 0.033). Kaplan-Meier curves showed a significantly higher 30-day survival rate in the Non-IV group (p = 0.011), particularly in patients with normal ICP (HR = 0.518, 95%CI:0.283-0.948, p = 0.033).

Conclusion: Non-IV famotidine administration significantly improves 30-day survival of ICH patients, especially for those with normal ICP, compared to IV administration.

Objectives: Our study assessed the budget impact and cost per responder of upadacitinib15mg and 30 mg for moderate to severe atopic dermatitis (MS-AD) treatment from social security and private health sector perspective in Argentina.

Methods: A budget impact model was adapted to depict clinical and economic aspects of treatment over a 5-years horizon time. Scenario analyses and deterministic sensitivity analyses were performed. A 16-weeks cost per responder model was adapted based on a network meta-analysis. Primary analyses assessed the cost per Eczema Area and Severity Index 50, 75 and 90 at week 16.

Results: The inclusion of upadacitinib 15 mg and 30 mg in the biological treatment mix for MS-AD was associated with an average budget saving per-member per-month ofU$S0.062 (social security) and U$S0.064 (private sector). Percentage of patients with access to treatment, acquisition cost of upadacitinib 30 mg and prevalence of MS-AD were the most influential parameters in the budget impact results. At week 16, upadacitinib 30 mg was associated with the lowest number needed to treat and the lowest cost per responder for all outcomes.

Conclusion: The introduction of upadacitinib in MS-AD treatment was associated with modest savings for the social security and private payer budget in Argentina.

Objectives: The present study aimed to evaluate the impact of undergoing massive weight loss reconstruction (MWR) on health-related quality of life (HR-QoL) in the Netherlands.

Method: A retrospective study was performed among 131 Dutch bariatric patients, divided into two groups: an intervention group (93 patients who had undergone MWR) and a control group (38 patients who had not undergone MWR). HR-QoL was assessed by the validated BODY-q questionnaire. The sign test was used to measure the difference between the 0 and 12 months' measurements of HR-QoL in both groups, whereas multiple regression analysis was conducted to assess whether undergoing MWR significantly predicted participants' incremental HR-QoL.

Results: Whereas the intervention group showed a significance improvement on all parameters of the BODY-q between 0 and 12 months (all parameters p < .001), the control group did not. The multiple regression analysis showed that having undergone a MWR significantly and positively predicted incremental HR-QoL on all scales on the BODY-q (all parameters p < 0.05).

Conclusion: The present study suggests a positive impact of MWR on the HR-QoL of bariatric patients.

Introduction: Parkinson's Disease (PD) is a progressive, chronic neurodegenerative disease, representing significant economic and social burdens. It is typically defined by motor symptoms (MSs), however, this does not reflect the full patient burden. Non-motor symptoms (NMSs) are increasingly recognized as central characteristics of PD. However, they still lack recognition in research. Therefore, this study aims to identify relevant NMSs, their prevalence, and the effect they have on Quality-of-Life (QoL) and Cost-of-Illness (COI). Secondly, it aims to identify gaps in the current body of knowledge and propose possible ways future research could bridge those gaps.

Methods: The study employed a scoping review, identifying 60 records for inclusion, using PubMed and Web of Science. It included studies from Spain or Italy, including data on People with Parkinson's Disease. A comparative analysis was performed using Microsoft Excel.

Results: It showed that the body of evidence relevant to NMSs, their prevalence, QoL, and COI is limited, or that estimates vary to an extent where interpretation is difficult.

Conclusion: Most studies suffer from generalization, representation, and standardization issues, stemming from their designs and methodological decisions. Although the findings of this study should be interpreted with caution, several recommendations are made for future research.

Background: Estimate the costs of inpatient and outpatient care for people with Cerebral Palsy (CP) in Brazil.

Research design and methods: Health records of people with CP in the Hospital and Outpatient Information Systems of Brazil between 2015 and 2019 were analyzed. Variables analyzed were gender, age, ICD, Intensive Care Unit (ICU) use, total cost, and ICU cost. Costs were adjusted for inflation and converted to dollars. Linear regression analysis was performed to investigate the association between social and clinical variables and direct costs.

Results: A total direct cost of approximately $166 million to the National Health System was identified, with $7.08 million/year and $26.1 million/year of inpatient and outpatient costs, respectively. The healthcare was primarily for children up to 14 years of age. The ICD 'spastic quadriplegic CP' received the most attendance. Rehabilitation was responsible for 75% of the outpatient care, with physiotherapy standing out. Increased age, use of ICU, and the types of CP are related to increased cost.

Conclusions: Healthcare for people with CP produced expressive costs for the Brazilian public health system, mainly with outpatient procedures and rehabilitation, with children being the most attended. Estimating these costs assist in better resource allocation for more effective healthcare provision.

Introduction: In breast cancer clinical trials utilizing digital endpoints, wearable sensors record participants' health information during activities of daily living.  These sensors are worn on the wrist or finger, placed as a skin patch or headband, or embedded on clothing. Data collected from wearable sensors form the basis of a digital endpoint, useful for determining effects of novel treatments on health outcomes, particularly quality-of-life outcomes.

Areas covered: References for this article were selected from a PubMed search spanning from 1 January 2017,to 1 July 2024, using the terms 'wearable sensors,' 'digital endpoints,' 'virtualtrials,' 'breast cancer.'  Additional articles from the authors' personal collection of papers and reviewers suggestions were also used.

Expert opinion: Digital endpoints must be validated as proper surrogate measures for healthcare outcomes, prior to their use in breast cancer trials.  Wearable sensors may introduce biases, such as 'missing not-at-random bias,' and perhaps even exacerbate disparities in healthcare outcomes if patients not comfortable with their use are excluded from clinical trials, or if the accuracy of sensors varies between racial and ethnic groups. Therefore, before embarking on trials with digital endpoints, validation studies are required, and limitations and risks of such trials need to be addressed.

Background: As rheumatoid arthritis (RA) is a chronic and progressive disease that requires lifelong therapeutic intervention, it represents a considerable economic burden on those affected. This study investigated whether tofacitinib is a cost-effective therapeutic alternative to other DMARDs for treating moderate-to-severe RA.

Research design and methods: All economic evaluation studies of tofacitinib compared to other DMARDs were identified. Using random-effects meta-analysis, we pooled incremental net benefit (INB) in (purchasing power parity) adjusted US$ with 95% confidence intervals. The modified economic evaluation bias checklist and Grading of Recommendation, Assessment, Development, and Evaluation (GRADE) instrument for quality appraisal were used. The subgroup analysis was done based on the comparator regimen.

Results: Of the selected 11 studies, the number of studies from high-, upper-middle- and lower-middle-income countries was 7, 3, and 1, respectively. The subgroup analysis showed that tofacitinib with an INB of 19,180 US$ [95% CI, -34520 to -3840; p-value = 0.01] was not statistically cost-effective compared with cDMARDs (p-value > 0.0001). Compared to other DMARDs, the estimated pooled INB of tofacitinib was US$ 7260 [95% CI, 3030 to 11,480; p-value < 0.001], but there was substantial heterogeneity among the included studies, and the observed publication bias.

Conclusion: While tofacitinib shows potential as a cost-effective treatment, tailored economic evaluations are needed to account for the diverse and evolving contexts of RA treatment.

Registration: PROSPERO: CRD42023405970.

Background: This study evaluates the cost-effectiveness of adding ocrelizumab to supportive care for primary progressive multiple sclerosis (PPMS) in Iran.

Research design and methods: Using a lifetime horizon from the payer's perspective, we developed a decision analytic model with Expanded Disability Status Scales (EDSS) as Markov health states while taking transition probabilities and treatment effects into account. Data were sourced from clinical trials and other literature. The target population was PPMS patients receiving either supportive care or ocrelizumab. We assessed cost- effectiveness through total costs, quality-adjusted life-years (QALYs), and the incremental cost- effectiveness ratio (ICER). Sensitivity analyses addressed uncertainties.

Results: The addition of ocrelizumab to supportive care provided an incremental gain of 0.89 QALYs and an additional cost of US$76,771.34, resulting in an ICER of US$86,220.35 compared to supportive care, which is 5.2 times Iran's GDP per capita (US$16,557). Thus, ocrelizumab is not cost-effective at the threshold of one time GDP per capita. However, the probability of cost-effectiveness increases at higher thresholds. Sensitivity analyses confirmed the robustness of the results.

Conclusion: While ocrelizumab is not cost-effective at the threshold of one-time GDP per capita, its clinical benefits are significant. Formulating healthcare policies for high-cost medications with low alternatives like ocrelizumab is essential.