Notes

Ferulic Acid-Loaded Polymeric Nanoparticles for Potential Ocular Shipping.
Population-adjusted comparisons of progression-free survival (PFS) from single-arm trials of cancer treatments can be derived using matching-adjusted indirect comparisons (MAICs); however, results are still susceptible to bias, particularly if the trials had different tumor assessment schedules. This study aims to assess the effects of assessment-schedule matching (ASM) on the relative effectiveness on the PFS of avelumab versus approved comparator immunotherapies or chemotherapy after population matching in the second-line (2L) setting for metastatic urothelial carcinoma.

The MAIC used patient-level data for avelumab from the JAVELIN Solid Tumor trial (NCT01772004). PFS was compared with published curves for other treatments to obtain population-adjusted hazard ratios (HRs). The MAIC was repeated after conducting ASM for differences in tumor assessment scheduled first at 6 weeks for avelumab and durvalumab and at 8 or 9 weeks for other treatments.

MAIC adjustment alone altered the HR estimates up to 23econd-line urothelial carcinoma treatment options, numerically favoring avelumab versus immunotherapies and chemotherapy agents. Correcting this bias is especially important when HRs are applied in cost-effectiveness models to transition patients between states.
Value of information (VOI) analysis can support health technology assessment decision making, but it is a long way from being standard use. The objective of this study was to understand barriers to the implementation of VOI analysis and propose actions to overcome these.

We performed a process evaluation of VOI analysis use within decision making on tomosynthesis versus digital mammography for use in the Dutch breast cancer population screening. Based on steering committee meeting attendance and regular meetings with analysts, we developed a list of barriers to VOI use, which were analyzed using an established diffusion model. We proposed actions to address these barriers. Barriers and actions were discussed and validated in a workshop with stakeholders representing patients, clinicians, regulators, policy advisors, researchers, and the industry.

Consensus was reached on groups of barriers, which included characteristics of VOI analysis itself, stakeholder's attitudes, analysts' and policy makers' skilld focus on application and evaluation of the proposed actions in real-world assessment processes.
Single-arm trial (SAT) data is increasingly reviewed for drug approvals by regulators and Health Technology Assessment (HTA) bodies. Supplementary data in the form of external comparators (ECs) can be used to provide clinical context to support these drug evaluations. In this study we characterized HTAs for SAT-based submissions, the use of supplementary EC data and outcomes from HTA review.

HTA Accelerator database was used to describe SAT-based HTA submissions with decisions (2011-2019).

A total of 433 SAT-based HTA submissions were identified between 2011 and 2019 with a 13-fold increase during this period. Around 65%(283/433) were in oncology or hem-oncology. Around 52%(226/433) of submissions contained some type of EC data, including prior clinical trials (24%, 104) and real-world data (RWD) (20%, 87), but 40%(175) contained no EC data. The overall acceptance rate for SAT-based submissions was 48% and with RWD EC data acceptance was 59%. In the latest 5-year period (2015-2019), use of RWD ECs increased 22% as a proportion of submissions per year, whereas, prior trial ECs decreased (-14%) and use of no EC remained stable (-2%). Between 2015 to 2017 and 2018 to 2019, acceptance rate for RWD ECs increased by 20% (41% in 2015-2017 to 61% in 2018-2019) whereas prior trial EC use decreased by 10% and no EC submissions decreased 16%. Of 226 submissions using ECs, only 29%(66) used an adjusted indirect treatment comparison method.

SAT-based submissions to HTA bodies are rapidly evolving in terms of composition and acceptance. Types of EC and methodological approach used are important determinants of positive outcomes.
SAT-based submissions to HTA bodies are rapidly evolving in terms of composition and acceptance. Types of EC and methodological approach used are important determinants of positive outcomes.
For men with intermediate prostate-specific antigen (PSA) levels (4-10 ng/mL), urine-based biomarkers and multiparametric magnetic resonance imaging (MRI) are increasingly used as reflex tests before prostate biopsy. We assessed the cost effectiveness of these reflex tests in the United States.

We used an existing microsimulation model of prostate cancer (PCa) progression and survival to predict lifetime outcomes for a hypothetical cohort of 55-year-old men with intermediate PSA levels. Urine-based biomarkers-PCa antigen (PCA3), TMPRSS2ERG gene fusion (T2ERG), and the MyProstateScore (MPS) for any PCa and for high-grade (Gleason score ≥7) PCa (MPShg)-were generated using biomarker data from 1112 men presenting for biopsy at 10 United States institutions. MRI results were based on published sensitivity and specificity for high-grade PCa. Costs and utilities were sourced from literature and Medicare reimbursement schedules. Outcome measures included life years, quality-adjusted life years (QALYs), and lifetime medical costs per patient. Incremental cost-effectiveness ratios were empirically calculated on the basis of simulated life histories under different reflex testing strategies.

Biopsying all men provided the most life years and QALYs, followed by reflex testing using MPShg, MPS, MRI, T2ERG, PCA3, and biopsying no men (QALY range across strategies 15.98-16.09). www.selleckchem.com/TGF-beta.html Accounting for costs, MRI and MPShg were dominated by other strategies. PCA3, T2ERG, and MPS were likely to be the most cost-effective strategy at willingness-to-pay thresholds of $100 000/QALY, $125 000/QALY, and $150 000/QALY, respectively.

Using PCA3, T2ERG, or MPS as reflex tests has greater economic value than MRI, biopsying all men, or biopsying no men with intermediate PSA levels.
Using PCA3, T2ERG, or MPS as reflex tests has greater economic value than MRI, biopsying all men, or biopsying no men with intermediate PSA levels.
Nonattendance of appointments in outpatient clinics results in many adverse effects including inefficient use of valuable resources, wasted capacity, increased delays, and gaps in patient care. This research presents a modeling framework for designing positive incentives aimed at decreasing patient nonattendance.

We develop a partially observable Markov decision process (POMDP) model to identify optimal adaptive reinforcement schedules with which financial incentives are disbursed. The POMDP model is conceptually motivated based on contingency management evidence and practices. We compare the expected net profit and trade-offs for a clinic using data from the literature for a base case and the optimal positive incentive design resulting from the POMDP model. To accommodate a less technical audience, we summarize guidelines for reinforcement schedules from a simplified Markov decision process model.

The results of the POMDP model show that a clinic can increase its net profit per recurrent patient while simultaneously increasing patient attendance. An increase in net profit of 6.10% was observed compared with a policy with no positive incentive implemented. Underlying this net profit increase is a favorable trade-off for a clinic in investing in a targeted contingency management-based positive incentive structure and an increase in patient attendance rates.

Through a strategic positive incentive design, the POMDP model results show that principles from contingency management can support decreasing nonattendance rates and improving outpatient clinic efficiency of its appointment capacity, and improved clinic efficiency can offset the costs of contingency management.
Through a strategic positive incentive design, the POMDP model results show that principles from contingency management can support decreasing nonattendance rates and improving outpatient clinic efficiency of its appointment capacity, and improved clinic efficiency can offset the costs of contingency management.
This study aimed to quantify the indirect costs of sickle cell disease in the United States.

Adult patients from a sickle cell disease clinic at an urban academic healthcare system completed an adapted Institute for Medical Technology Assessment Productivity Cost Questionnaire related to the impact of their disorder on absenteeism, presenteeism, ability to contribute through unpaid work outside of employment, and other aspects of life. Additional data were collected from patient records about each participant's genotype, total hemoglobin level, and pain level.

Of the 192 individuals, 187 who completed the survey reported experiencing vaso-occlusive crisis pain events during the last year that negatively affected their productivity at work and in daily roles. Three-fourths of respondents reported impairment in their ability to complete everyday tasks, such as caring for children, running errands, doing housework, shopping for groceries, and volunteer (unpaid) work. Only 30% of respondents reported being employed or self-employed. Of those employed, estimated costs of absenteeism and presenteeism attributable to pain events averaged $15 103 per person annually. Estimated total annual losses in unpaid work productivity averaged $3 145 862 for the study respondents and another $2 870 652 for their caregivers.

Sickle cell disease affected the work productivity, nonwork productivity, and the daily lives of adults seen with the disorder in an academic medical center.
Sickle cell disease affected the work productivity, nonwork productivity, and the daily lives of adults seen with the disorder in an academic medical center.
This study aimed to map the World Health Organization Quality of Life-BREF (WHOQOL-BREF) onto the 5-level EQ-5D (EQ-5D-5L) using a real Thai valuation set of the EQ-5D-5L. The second objective was to explore the impacts of the differences between observed and predicted EQ-5D-5L index scores on the incremental cost-utility ratio (ICUR) using 5 hypothetical scenarios.

This is a secondary data analysis. A total of 800 outpatients with chronic diseases were recruited from 2 university hospitals in Bangkok, Thailand, between July 2014 and March 2015. The 800 patients were randomly divided into 2 samples estimation and validation samples. The estimation sample was used to assess the relationships between the EQ-5D-5L index score and 4 WHOQOL-BREF dimension scores and to find the best-fit model and its equation. For the validation sample, the equation of the best-fit model from the estimation sample was used to calculate predicted EQ-5D-5L index scores.

A multiple linear regression showed that only the physical domain of the WHOQOL-BREF was significantly associated with the EQ-5D-5L. Among 11 regression models, the curve estimation found that the inverse model was the best-fit model. The prediction equation of EQ-5D-5L was equal to 1.385 minus 7.572/physical domain of the WHOQOL-BREF. The impacts of the differences between the observed and predicted EQ-5D-5L index scores on ICUR were only 0.4% to 1.8% from the base case.

A nonlinear relationship between the physical domain of the WHOQOL-BREF and EQ-5D-5L utility was shown. The impacts of the differences between the observed and predicted EQ-5D-5L index scores on ICUR were minimal.
A nonlinear relationship between the physical domain of the WHOQOL-BREF and EQ-5D-5L utility was shown. The impacts of the differences between the observed and predicted EQ-5D-5L index scores on ICUR were minimal.
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