A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies.

Cell and gene therapy (CGT) innovations have provided several significant breakthroughs in recent years. However, CGTs often come with a high upfront cost, raising questions about patient access, affordability, and long-term value. This study reviewed cost-effectiveness analysis (CEA) studies that have attempted to assess the long-term value of Food and Drug Administration (FDA)-approved CGTs. Two reviewers independently searched the Tufts Medical Center CEA Registry to identify all studies for FDA-approved CGTs, per January 2023. A data extraction template was used to summarize the evidence in terms of the incremental cost-effectiveness ratio expressed as the cost per quality-adjusted life year (QALY) and essential modeling assumptions, combined with a template to extract the adherence to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. The review identified 26 CEA studies for seven CGTs. Around half of the base-case cost-effectiveness results indicated that the cost per QALY was below $100,000-$150,000, often used as a threshold for reasonable cost-effectiveness in the United States. However, the results varied substantially across studies for the same treatment, ranging from being considered very cost-effective to far from cost-effective. Most models were based on data from single-arm trials with relatively short follow-ups, and different long-term extrapolations between studies caused large differences in the modeled cost-effectiveness results. In sum, this review showed that, despite the high upfront costs, many CGTs have cost-effectiveness evidence that can support long-term value. Nonetheless, substantial uncertainty regarding long-term value exists because so much of the modeling results are driven by uncertain extrapolations beyond the clinical trial data.

Racial/Ethnic Disparities in Use of Angiotensin II Receptor Type 2/4 Stimulatory Vs. Inhibitory Antihypertensive Among Hypertensive Adults in the USA.

OBJECTIVES: Studies showed angiotensin II type 2 receptor/angiotensin II type 4 receptor (AT2R/AT4R) stimulatory antihypertensive was associated with a lower risk of dementia and cognitive impairment compared to the inhibitory one. This study aimed to identify the racial and ethnic differences in using these agents among the USA adults with hypertension. METHODS: A cross-sectional study was conducted using data from the Medical Expenditure Panel Survey (MEPS, 2016-2019). Individuals with a diagnosis of hypertension or self-reported hypertension and without dementia or Alzheimer's disease diagnosis were included in the analysis. We applied two multivariable logistic regressions to compare racial/ethnic differences in AT2R/AT4R stimulatory antihypertensive use and AT2R/AT4R inhibitory antihypertensive use, adjusting for covariates. RESULTS: Twenty-four thousand five hundred eighty-one individuals with hypertension and without dementia or Alzheimer's disease were identified. Among non-Hispanic Whites, 72.39% were using AT2R/AT4R inhibitory antihypertensive agents, vs. 66.97% using AT2R/AT4R stimulatory antihypertensive agents. In contrast, both non-Hispanic Black and Asian Americans were using more AT2R/AT4R stimulatory agents than inhibitory ones (16.40% vs. 12.16% and 4.79% vs. 3.43%, respectively). Compared to non-Hispanic White, non-Hispanic Black (OR 1.980, 95% CI 1.839-2.132) and non-Hispanic Asian Americans (OR 1.545, 95% CI 1.356-1.761) were significantly associated with higher odds of prescribing AT2R/AT4R stimulatory agents, while Hispanics (OR 0.744, 95% CI 0.685-0.808) were associated with lower odds of prescribing AT2R/AT4R inhibitory agents compared to non-Hispanic Whites. CONCLUSIONS: The results showed that the high-dementia risk populations like non-Hispanic Black and Asian American races are proportionally prescribed with higher use of low-dementia risk antihypertensive agents, compared to non-Hispanic Whites.

Complementary and alternative medicine for urinary tract illnesses: A cross-sectional survey in Jordan.

The aim of this study was to assess the extent of Complementary and Alternative Medicine (CAM) usage, especially herbal preparations, in treating urinary tract illnesses, and their perceived efficacy. This was a cross-sectional survey that used a convenience sample of 278 adults who reported having any form of urinary tract illness. During the last 3 months, 105 (37.8%) of participants had used CAM for various urinary tract conditions, of which, 87 (82.9%) used herbal remedies. Urinary tract infections (UTIs) were the most reported urinary condition (n = 77, 73.3%) among CAM users. The most commonly used herbs were, parsley (n = 54, 19.2%), followed by chamomile (n = 29, 10.4%), barley (n = 20, 7.4%) and ginger (n = 18, 6.7%).The study provides an overview of various CAM remedies used to treat urinary tract illnesses in the Jordanian society, which would draw attention to the necessity of conducting interventional studies to evaluate the efficacy and safety of CAMs in treating urinary tract illnesses, either as stand-alone or adjuvant treatment.

Promotion of appropriate knowledge and attitude towards medicines among schoolchildren in Jordan: the role of teachers.

OBJECTIVES: The current study aimed to examine the knowledge, attitude and practice of schoolteachers in Jordan towards medication. Furthermore, it assessed teachers' views regarding the importance of school education in the promotion of appropriate and safe use of medicines. METHODS: All Facebook groups of teachers who were members of the Jordan Teachers Syndicate and/or teachers employed by Jordan Ministry of Education were invited to participate. A cross-sectional online survey using a validated pre-piloted anonymous Arabic questionnaire was created using Google documents. The questionnaire was automatically hosted via a unique URL. Researchers downloaded real-time questionnaire responses. Data were coded and entered into SPSS database for windows version 22 for statistical analysis. KEY FINDINGS: A total of 902 teachers filled in the questionnaire. The mean knowledge score achieved in multiple-choice questions was 2.43 ± 0.51 out of 4.00. The top three factors affecting all of teachers' knowledge questions were as follows: age, the presence of chronic disease, and the school grade that they taught. Teachers showed variation in their attitude towards education about medicines in children. The majority (80.7%) of teachers were unfamiliar with education about medication and had never taught schoolchildren about any issues related to proper use of medications. CONCLUSIONS: The study highlighted that teachers' knowledge about medicines was not satisfactory enough for them to become active health educators. At the same time, they were willing to educate children about proper use of medicines, and such education should be started at the first grade (i.e. 6 years old).