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Characterizing and evaluating the holistic value of innovative healthcare technologies (e.g. treatments, services) constitutes a crucial goal to maximize limited resources. However, the characteristics of innovation have not been well identified. This review aims to describe the characteristics of healthcare innovation. We performed a comprehensive systematic search using PubMed, Embase, PsycINFO, and Econlit from inception to July 2022. Articles were included if they described innovation or the characteristics of innovation of the technologies in healthcare. Characteristics or definitions of innovation directly or indirectly described as innovation were extracted from the included articles. Two independent reviewers then conceptualized the identified characteristics of innovation to generate innovation attributes in healthcare. In total, 103 articles were included in this review. Eight attributes describing innovation, i.e. novelty, step change, substantial benefits, an improvement over existing technologies, convenience and/or adherence, added value, acceptable cost, and uncounted benefits, were conceptualized. Most of the identified innovation attributes were based on the researchers’ perspective. This study conceptualized innovation attributes in healthcare based on the characteristics of healthcare innovation as defined in the literature. Further research is warranted to obtain a complete understanding of the perspectives of researchers and other stakeholders, including patients, healthcare providers, healthcare payers, and the pharmaceutical industry, on recognizing innovation in healthcare.KEY POINTSThis is the first systematic review to conceptualize attributes of healthcare innovation.We conceptualized eight attributes describing innovation, i.e. novelty, step change, substantial benefits, an improvement over existing technologies, convenience and/or adherence, added value, acceptable cost, and uncounted benefits based on the similar concept.In existing literature, patients’ and caregivers’ perspectives were less frequently found to describe the innovation attributes.Future research is needed to identify, measure, and value various stakeholders, including patients’ and caregivers’ perspectives on healthcare innovation. This is the first systematic review to conceptualize attributes of healthcare innovation. We conceptualized eight attributes describing innovation, i.e. novelty, step change, substantial benefits, an improvement over existing technologies, convenience and/or adherence, added value, acceptable cost, and uncounted benefits based on the similar concept. In existing literature, patients’ and caregivers’ perspectives were less frequently found to describe the innovation attributes. Future research is needed to identify, measure, and value various stakeholders, including patients’ and caregivers’ perspectives on healthcare innovation.

Background and ObjectivesA recent report estimated that approximately 1 million adults were living with multiple sclerosis (MS) in the United States. Although MS is rarely the direct cause of death, its debilitating effects on normal body functions can result in considerable disruption to daily living and life roles including work, physical independence, mobility, social interaction, and participation in leisure activities. This study estimated the total economic burden of MS in the United States in 2019.MethodsThis study used a prevalence-based approach to estimate the national economic burden of MS. Claims from 3 sources (Medicare Current Beneficiary Survey, Medicare Standard Analytical File, and Optum de-identified Normative Health Information System) were used to obtain direct costs and a survey was developed to collect indirect costs (e.g., labor market productivity losses, costs of paid and unpaid caregivers, home modification) from 946 patients with MS (PwMS). Direct medical costs reflected the difference in the total average annual amount paid for PwMS vs matched controls without MS. Future earnings loss due to premature death attributable to MS was calculated using Centers for Disease Control and Prevention mortality data and Medicare claims data.ResultsThe estimated total economic burden was $85.4 billion, with a direct medical cost of $63.3 billion and indirect and nonmedical costs of $22.1 billion. Retail prescription medication (54%); clinic-administered drugs, medication, and administration (12%); and outpatient care (9%) were the 3 largest components of the direct costs. The average excess per-person annual medical costs for PwMS was $65,612; at $35,154 per person, disease-modifying therapies (DMTs) accounted for the largest proportion of this cost. The cost per DMT user ranged from $57,202 to $92,719, depending on sex–age strata. The average indirect and nonmedical costs were $18,542 per PwMS and $22,875 per PwMS if caregivers' costs were included. Lost earnings due to premature death, presenteeism, and absenteeism losses were the largest indirect cost components.DiscussionMS is a costly chronic disease, with direct costs of prescription drugs and indirect productivity loss being important cost drivers. Our findings suggested that the burden of MS in the United States has been underestimated.

Cardiotoxicity is a severe side effect for colorectal cancer (CRC) patients undergoing fluoropyrimidine-based chemotherapy. To develop and compare machine learning algorithms to predict cardiotoxicity risk among nationally representative CRC patients receiving fluoropyrimidine, CRC Patients with at least one claim of fluoropyrimidine after their cancer diagnosis were included. The outcome was the 30-day cardiotoxicity from the first day of starting fluoropyrimidine. The machine learning models including extreme gradient boosting (XGBoost), random forest (RF), and logistic regression (LR) were developed using 2006-2011 SEER-Medicare data, and model performances were evaluated using 2012-2014 data. Precision, F1 score, and area under the receiver operating characteristics curve (AUC) were measured to evaluate model performances. Feature importance plots were obtained to quantify the predictor importance. Among 36,030 CRC patients, 18.74% of them developed cardiotoxicity within 30 days since the first fluoropyrimidine. The XGBoost approach had better prediction performance with higher precision (0.619) and F1 score (0.406) in predicting the 30-day cardiotoxicity, compared to the RF (precision, 0.607 and F1 score, 0.395) and LR (precision, 0.610 and F1 score, 0.398). According to the DeLong's test for AUC difference, the XGBoost significantly outperformed the RF and LR (XGBoost, 0.816 vs. RF, 0.804, P < 0.001; XGBoost vs. LR, 0.812, P = 0.003, respectively). Feature importance plots identified pre-existing cardiac conditions, surgery, older age as top significant risk factors for cardiotoxicity events among CRC patients after receiving fluoropyrimidine. In summary, the developed machine learning models can accurately predict the occurrence of 30-day cardiotoxicity among CRC patients receiving fluoropyrimidine-based chemotherapy.

Bidur Banjara,1,2 Nabin Poudel,1 Kimberly B Garza,1 Salisa Westrick,1 Heather P Whitley,3 David Redden,4 Surachat Ngorsuraches1 1Department of Health Outcomes Research and Policy, Auburn University, Harrison College of Pharmacy, Auburn, AL, USA; 2Cytel Inc, Waltham, MA, USA; 3Department of Pharmacy Practice, Auburn University, Harrison College of Pharmacy, Auburn, AL, USA; 4Department of Biomedical Affairs and Research, Auburn University, Edward via College of Osteopathic Medicine, Auburn, AL, USACorrespondence: Surachat Ngorsuraches, Department of Health Outcomes Research and Policy, Auburn University, Harrison College of Pharmacy, 4306A Walker Building, Auburn, AL, 36849, USA, Tel +1 334 844 8357, Fax +1 334 844 8307, Email surachat@auburn.eduPurpose: To determine patients’ preferences for sodium-glucose cotransporter 2 inhibitors (SGLT-2is) and glucagon-like peptide-1 receptor agonists (GLP-1RAs).Patients and Methods: A cross-sectional, web-based discrete choice experiment was conducted among US adults with type 2 diabetes mellitus (T2DM) in May 2021. Six attributes—the route and frequency of administration, the chance of reaching target HbA1c in six months, the percentage reduction in the risk of major adverse cardiovascular events (MACE), the chance of gastrointestinal side effects, the chance of genital infection, and out-of-pocket cost per month—were identified from literature review and consultation with patients and clinicians. A Bayesian efficient design was used to generate choice sets. Each choice set contained two hypothetical SGLT-2i and GLP-1 RA alternatives described by the attributes and an opt-out alternative. A total of 176 patients were asked to select the most preferred option from each choice set. Mixed logit (ML) and latent class (LC) models were developed. The conditional relative importance of each attribute was determined.Results: The ML model showed the out-of-pocket cost had the highest conditional relative importance, followed by the chance of reaching the target HbA1c. The best LC model revealed two patient classes. All attributes were significantly important to the patients in both classes, except the chance of genital infection in class 2. Compared to the patients in class 2, the patients in class 1 were older (approximately 65 vs 56 years) and had a higher number of comorbidities (approximately three vs two).Conclusion: T2DM patients placed different preference weights or importance across SGLT-2i and GLP-1 RA attributes. Preference heterogeneity was found among patients with different ages and numbers of comorbidities.Keywords: diabetes, discrete choice experiment, patient preference, second-line antihyperglycemic agents

Innovative technologies (e.g. treatments) play a pivotal role in improving patient’s well-being and in consequence population health outcomes. However, there is lack of consensus and comprehensive summary what constitutes innovation. Additionally, valuing them using traditional cost-effectiveness analysis is unlikely to capture the full range of benefits of these innovative technologies. This review aims to understand how innovation attributes were measured and/or valued in healthcare. We systemically searched four databases, PubMed, Embase, PsycINFO, and Econlit, from inception to April 2021. Studies were included if they measured and/or valued the attributes of innovation for healthcare identified in our previous systematic review. Any other potential recommended methods to measure and/or value the innovation attributes were also extracted. Of 546 articles, a total of 17 articles were finally included in this review. If attributes were measured and traded-off relative to costs, then it was considered as valuation of those attributes. Two specific attributes of innovation, i.e. substantial benefits and convenience and/or adherence were measured using adherence rate and life year or QALY gain. When innovation attribute was non-specific it was described as “overall innovation” and measured using overall innovativeness scale (e.g. point/binary scale). QALY-based cost-effectiveness analysis (CEA) was commonly used to assess and value substantial benefit attribute. Other valuation approaches were (i) rating, (ii) the economic value of life year gain, (iii) multiple criteria decision analysis (MCDA), (iv) incremental net health benefit (INHB), and (v) quality-adjusted cost of care (QACC). ICER threshold adjustment and multiple-criteria decision analysis (MCDA) are two common recommended approaches to capture the innovation comprehensively. We found that MCDA approaches often promoted and discussed but were sub-optimally used to incorporate different value attributes into decision-making. Existing methods used by payers to measure and value the innovation component of a new product do not reflect the full range of health and cost impacts. They generally do not consider the alternative perspectives of patients, providers, caregivers, and society. Key challenges remain to appropriately measure and value innovation attributes and incorporate them into HTA decision making.

Cost-effectiveness analysis (CEA) with quality-adjusted life-year (QALY) was introduced to address health equity concerns in value assessment. However, QALY fails to capture patient preference. Stated preference methods (eg, discrete choice experiment [DCE]) have been increasingly used to incorporate patient preference into the value assessment framework in health care. Still, ones with a moral dimension such as health equity do not exist. The objective of this paper was to describe 2 stated preference approaches that can empirically value health equity. First, the decision-maker perceptions of the prevalence of equity dimensions in DCE choice tasks are identified. A latent class model based on random utility theory is proposed to derive the value of equity from the decision makers with different perceptions of the prevalence of equity dimensions. Second, equity attributes are incorporated in DCE choice tasks, and a quantum choice model, which can capture stochasticity during the decision process in the mind of the decision makers, is used to value the equity. These approaches will improve existing value assessment methods to address health equity adequately. DISCLOSURES: This study received no outside funding. Ngorsuraches has received research grants from Bristol Myers Squibb and through the University of Utah and PhRMA Foundation.

Objectives Globally, nutraceuticals have been increasingly used. Nevertheless, the consumer preferences for nutraceuticals have not been quantitatively investigated. This study used discrete choice experiment (DCE) to examine consumer preferences and willingness to pay for nutraceuticals. Methods Four attributes (ie, the scientific proof of effectiveness, the scientific proof of safety, the source of recommendation, and cost) were identified from a systematic review and focus group interviews. They were used to develop a DCE questionnaire. Consumers at community pharmacies in Malaysia were asked to respond to 8 DCE choice sets. A conditional logit model was employed to obtain the relative importance of each attribute and to estimate respondents’ WTP for nutraceuticals. Results A total of 111 valid responses were analyzed. A negative constant term in the developed model indicated that generally the respondents preferred not to use nutraceuticals before they considered the study attributes. The respondents preferred nutraceuticals with no side effect, clear evidence of effectiveness, and recommendation of a healthcare professional. The respondents were willing to pay $252/month more for nutraceuticals proven with no side effect than for those without proof of safety, and $102/month more for nutraceuticals proven with clear effectiveness than for those without proof of effectiveness. Conclusions Consumers weighed relatively high on the availability of safety and effectiveness proofs when they chose nutraceuticals. The study highlights on the crucial need to inform consumers using clinical evidences of nutraceuticals as the information is highly preferred by consumers.

Introduction: Despite the increasing role of patients in the US healthcare system, patients have yet been engaged in the value assessment of their treatments, including disease-modifying therapies (DMTs) for multiple sclerosis (MS). The objectives of this review were therefore to summarize existing studies on cost-effectiveness analysis (CEA) with quality-adjusted life years (QALYs) and patients' preferences of DMTs for MS, and to discuss how to incorporate patients' preferences into the value assessment of DMTs.Area covered: We reviewed previous systematic reviews and conducted further search until November 2020 for studies on CEA with QALYs and patients' preferences of DMTs for MS. We identified the outcomes that were assessed or valued in the CEA studies and the DMT attributes that were important to patients with MS.Expert opinion: Our literature review showed that the studies using CEA with QALYs failed to capture some important DMT attributes, e.g., route and frequency of administration, identified in the studies on the patients' preferences. Various approaches were available for incorporating the patients' preferences in the value assessment of DMTs for MS. We supported this incorporation, which subsequently would increase patient access to preferred DMTs.

Objective To evaluate the clinical impact of mental health collaborative teams that include pharmacists. Data sources PubMed, PsychInfo, Clinialtrials.gov , International Pharmaceutical Abstracts. Study selections Studies in which pharmacists were part of a mental health collaborative team (defined as 2 or more health care providers working together to provide enhanced mental health care services to patients), mental health clinical outcomes were measured with a validated tool, and the articles were written in English were included. Articles were searched from database inception to July 2019 and were excluded if a quantifiable comparison of mental health clinical outcomes was not included or collaboration was not described. Data extraction Two authors independently screened titles and abstracts for relevance. Full-text articles that potentially met inclusion criteria were retrieved, read, and evaluated for inclusion using the eligibility criteria. Results All 9 included studies reported improvements in mental health clinical outcomes when using collaborative teams that included pharmacists. Depression (n = 8) and post-traumatic stress disorder (PTSD) (n = 1) were the mental health conditions included in the studies. Overall, 5 of 7 of the randomized controlled trials (RCTs) had statistically significant improvement in mental health clinical outcomes between the intervention groups, which included the collaborative teams and a “usual care” groups, which did not. Four of the 5 studies were set in Veterans Affairs (VA) clinics. The 2 non-RCT pre-post studies showed improvements in clinical outcomes but did not achieve statistical significance. Conclusion Evidence shows that collaborative teams that include pharmacists are effective at improving mental health outcomes in patients with depression and PTSD. Future studies should include non-VA settings and other mental health conditions to understand pharmacists’ impact more broadly in mental health collaborative teams. Clarifying and understanding the overlapping roles and responsibilities of members of the team may be the next step to continue improving mental health clinical outcomes.

Multiple sclerosis (MS) is multifactorial in nature, with both genetic and environmental factors contributing to disease risk. In this umbrella review, we aimed to summarize various non-HLA genes that have been investigated for their association with MS risk in meta-analyses (MAs) of observational studies.Medline, Embase, Epistemonikos, and Cochrane Database of Systematic Reviews were searched for MAs up to July 2021. Evidence of association was ranked according to established criteria as follows: convincing, highly suggestive, suggestive, weak, or not significant.From 1,533 publications, 85 full-text articles were evaluated for eligibility, and 15 articles were selected that described 114 genetic associations. No associations were supported by convincing evidence. One association between the rs2104286 (A vs G contrast) polymorphism of the interleukin 2RA (IL2RA) gene and increased MS susceptibility was initially supported by highly suggestive evidence (OR 1.17; 95% CI 1.10-1.25) but it was downgraded to suggestive in the sensitivity and subgroup analyses. We also found six associations supported by suggestive evidence in the main analysis.The findings of this study imply that the cytokines genes interleukin IL2RA and IL7R may play a role in the development of MS. Data from more and better-designed studies are needed to establish robust evidence.

IntroductionPrior to the COVID-19 pandemic, substance use health services for treatment of alcohol use disorder and problematic alcohol use (AUD/PAU) were fragmented and challenging to access. The pandemic magnified system weaknesses, often resulting in disruptions of treatment as alcohol use during the pandemic rose. When treatment services were available, utilisation was often low for various reasons. Virtual care was implemented to offset the drop in in-person care, however accessibility was not universal. Identification of the characteristics of treatment services for AUD/PAU that impact accessibility, as perceived by the individuals accessing or providing the services, will provide insights to enable improved access. We will perform a scoping review that will identify characteristics of services for treatment of AUD/PAU that have been identified as barriers to or facilitators of service access from the perspectives of these groups.Methods and analysisWe will follow scoping review methodological guidance from the Joanna Briggs Institute. Using the OVID platform, we will search Ovid MEDLINE including Epub Ahead of Print and In-Process and Other Non-Indexed Citations, Embase Classic+Embase, APA PsychInfo, Cochrane Register of Controlled Trials, the Cochrane Database of Systematic Reviews and CINAHL (Ebsco Platform). Multiple reviewers will screen citations. We will seek studies reporting data collected from individuals with AUD/PAU or providers of treatment for AUD/PAU on service-level factors affecting access to care. We will map barriers to and facilitators of access to AUD/PAU treatment services identified in the relevant studies, stratified by service type and key measures of inequity across service users.Ethics and disseminationThis research will enhance awareness of existing evidence regarding barriers to and facilitators of access to services for the treatment of alcohol use disorder and problematic alcohol use. Findings will be disseminated through publications, conference presentations and a stakeholder meeting. As this is a scoping review of published literature, no ethics approval was required.

Discrete choice experiments (DCEs) are increasingly used to elicit preferences for health and healthcare. Although many applications assume preferences are homogenous, there is a growing portfolio of methods to understand both explained (because of observed factors) and unexplained (latent) heterogeneity. Nevertheless, the selection of analytical methods can be challenging and little guidance is available. This study aimed to determine the state of practice in accounting for preference heterogeneity in the analysis of health-related DCEs, including the views and experiences of health preference researchers and an overview of the tools that are commonly used to elicit preferences.An online survey was developed and distributed among health preference researchers and nonhealth method experts, and a systematic review of the DCE literature in health was undertaken to explore the analytical methods used and summarize trends.Most respondents (n = 59 of 70, 84%) agreed that accounting for preference heterogeneity provides a richer understanding of the data. Nevertheless, there was disagreement on how to account for heterogeneity; most (n = 60, 85%) stated that more guidance was needed. Notably, the majority (n = 41, 58%) raised concern about the increasing complexity of analytical methods. Of the 342 studies included in the review, half (n = 175, 51%) used a mixed logit with continuous distributions for the parameters, and a third (n = 110, 32%) used a latent class model.Although there is agreement about the importance of accounting for preference heterogeneity, there are noticeable disagreements and concerns about best practices, resulting in a clear need for further analytical guidance.

Cardiotoxicity is a severe side effect for colorectal cancer (CRC) patients undergoing fluoropyrimidine-based chemotherapy. To develop and compare machine learning algorithms to predict cardiotoxicity risk among nationally representative CRC patients receiving fluoropyrimidine, CRC Patients with at least one claim of fluoropyrimidine after their cancer diagnosis were included. The outcome was the 30-day cardiotoxicity from the first day of starting fluoropyrimidine. The machine learning models including extreme gradient boosting (XGBoost), random forest (RF), and logistic regression (LR) were developed using 2006-2011 SEER-Medicare data, and model performances were evaluated using 2012-2014 data. Precision, F1 score, and area under the receiver operating characteristics curve (AUC) were measured to evaluate model performances. Feature importance plots were obtained to quantify the predictor importance. Among 36,030 CRC patients, 18.74% of them developed cardiotoxicity within 30 days since the first fluoropyrimidine. The XGBoost approach had better prediction performance with higher precision (0.619) and F1 score (0.406) in predicting the 30-day cardiotoxicity, compared to the RF (precision, 0.607 and F1 score, 0.395) and LR (precision, 0.610 and F1 score, 0.398). According to the DeLong's test for AUC difference, the XGBoost significantly outperformed the RF and LR (XGBoost, 0.816 vs. RF, 0.804, P 0.001; XGBoost vs. LR, 0.812, P = 0.003, respectively). Feature importance plots identified pre-existing cardiac conditions, surgery, older age as top significant risk factors for cardiotoxicity events among CRC patients after receiving fluoropyrimidine. In summary, the developed machine learning models can accurately predict the occurrence of 30-day cardiotoxicity among CRC patients receiving fluoropyrimidine-based chemotherapy.

Objective: To identify and describe the methodological approaches for assessing the certainty of the evidence in umbrella reviews (URs) of meta-analyses (MAs).Study Design and Setting: We included URs that included SR-MAs of interventions and non-interventions. We searched from 3 databases including PubMed, Embase, and The Cochrane Library from 2010 to 2020.Results: 138 URs have been included consisting of 96 and 42 URs of interventions and non-interventions, respectively. Only 31 (32.3%) of URs of interventions assessed certainty of evidence, in which the GRADE approach was the most frequently used method (N=20, 64.5%) followed by creditability assessments (N=6, 12.9%). Conversely, thirty (71.4%) of URs of non-interventions assessed certainty of evidence, in which the criteria for credibility assessment were mainly used (N=28; 93%). URs published in journals with high journal impact factor (JIF) are more likely to assess certainty of evidence than URs published in low JIFs. Conclusions: Only one-third of URs that included MAs of experimental designs have assessed the certainty of the evidence in contrast to the majority of the URs of observational studies. Therefore, guidance and standards are required to ensure the methodological rigor and consistency of certainty of evidence assessment for URs.

Objectives To compare the numbers of approval days of the generic drugs with and without citizen petitions. Methods Data from a previous study were obtained to create a data set containing Food and Drug Administrations (FDA) abbreviated new drug applications (ANDAs) with and without citizen petitions, and their filing and approval dates. Descriptive analyses were conducted. An independent t-test was used to analyze the difference of the numbers of approval days of the generic drugs with and without citizen petitions. Results A total of 289 ANDAs of 81 drugs submitted between the first quarter of 2008 and the third quarter of 2014 were included. Citizen petitions were submitted for 184 (63.7%) ANDAs during this period. More than a third (36.3%) of all ANDAs had a single petition. Among the ANDAs with the citizen petition, the average number of petitions per ANDA was approximately two. The numbers of approval days of the ANDAs with and without citizen petitions were 1221.7 and 978.6 days, respectively, and they were statistically significantly different (p-value Conclusion The number of approval days of the generic drugs with citizen petitions was significantly higher than the number of approval days of the generic drugs without citizen petitions.

Multiple sclerosis (MS) is one of the most common, nontraumatic, disabling diseases diagnosed in adults. Self-empowered patients and families are valued members of the MS research team. The objective of this study was to explore patient and family perceptions of the influence of psychosocial state on their willingness to be research partners. Researchers conducted 5 focus groups with MS patients and family from the Upper Midwest Chapter of the National Multiple Sclerosis Society. The researchers asked questions addressing psychosocial factors influencing ability and willingness to work with MS researchers as partners. Relevant themes were identified including comfort level of individuals in formulating research questions, comfort level engaging in research, understanding of the meaning of research and self-perception about skills, research training, and knowledge needs. The findings of this study support the role of MS patients’ perspectives about MS, their understanding of the science of MS, and role of their psychosocial states as all these factors were patient identified as being key to their ability to be active, engaged and willing research participants.

Objective: Evaluating Student Performance and Perception of a Workshop Integrating Pharmacy Practice and a Pharmaceutics Lab Innovation: Common methods for curricular integration are often time and faculty-intensive. An innovative approach to integration was developed and utilized in an introductory compounding workshop. Faculty members collaborated with a compounding pharmacist to design and facilitate a pharmaceutics workshop for first-year pharmacy students. The workshop was composed of four major sections, an introduction to pharmaceutical compounding and the regulations surrounding manufacturing and sterility, a case discussion involving a pediatric patient and the need to develop an appropriate drug delivery system, a short review of pharmaceutical calculations and labeling requirements, and then an introduction to logistics and active learning in a lab setting. Critical Analysis: After taking part in the workshop, students indicated a significantly higher comfort level going into the pharmaceutics lab (3.48±0.83 to 4.04±0.70) and in the compounding process (3.06±0.83 to 3.71±0.80). Their views of the clinical application of the lab and the need to use knowledge gained from other courses in the lab were also significantly improved (4.36±0.68 to 4.61±0.49 and 3.71±0.77 to 4.26±0.74, respectively). In addition, their perceptions of how they will utilize the skills developed as a practicing pharmacist, and their feelings towards the safety procedures involved in compounding, were also positively affected (3.96±0.87 to 4.45±0.59 and 3.28±0.92 to 3.91±0.72, respectively). Finally, students’ average quiz score in Spring 2016, when the workshop was instituted, significantly increased from Spring 2015 (90.154±4.98 versus 85.89±10.87, respectively). Article Type: Note

Background Medication adherence for chronic conditions continues to be a challenge for patients. Patient incentives for medication adherence may help. Financial incentives delivered at the point of care may act as cues for medication-taking behavior. Objectives The purpose of this study was to investigate patient preferences for specific structures of financial medication adherence incentives that could feasibly be delivered at the point of care. Methods A discrete choice experiment (DCE) was performed using a national online survey. Study participants were adults who self-reported taking at least one prescription medication for one or more chronic conditions. Following an orthogonal design generated in SAS, the DCE included 32 paired-choice tasks. Data were analyzed using mixed logit models and stratified on participants’ income level. Results In the full cohort (n = 933), form of financial reward (such as gift-card or cash) was 1.02 times as important to participants as the probability of incentive receipt, 1.58 times as important as monetary value, and 1.93 times as important as timing of receipt. Participants were willing to give up $31.04 of an incentive's monetary value (95% CI = $27.11-$34.98) to receive the incentive 5 months sooner (1-month vs. 6-month time-lag); $60.79 (95% CI = $53.19-$68.39) for probability of receipt to increase from a 1 out of 100 chance to a 1 out of 20 chance; and $10.52 (95% CI = $6.46-$14.58) to receive an incentive in the form of a Visa® gift-card instead of grocery store voucher. These patterns of trade-offs between attributes were generally consistent among participants with lower and higher income. Conclusions Regardless of socioeconomic status, patient preferences for financial medication adherence incentives delivered at the point of care may be most heavily influenced by incentive form and probability of receipt. This has implications for designing medication adherence programs in terms of incentive sustainability, patient engagement, plan star ratings, and patient outcomes.

Colorectal cancer (CRC) is one of the public health burdens that can be lowered by early detection. This study aims to examine the preferences and willingness-to-pay of a population at risk for CRC screening in Thailand. Understanding the preferences for these individuals at risk would help Thailand, as an example of LMICs, to design effective population-based CRC screening programs. A discrete choice experiment (DCE) was conducted among screening-naïve adults aged 50–75 years, who were at risk of CRC, in the out-patient department of a tertiary care hospital in Thailand. A DCE questionnaire was developed from six CRC screening attributes. Each questionnaire was composed of six choice sets and each contained two alternatives described by the different levels of attributes and an opt-out alternative. Participants were asked to choose one alternative from each choice set. A multinomial logit model was developed to determine the relative preference of each attribute. The willingness-to-pays for all attributes and screening modalities and the estimated preferred choices of the annual fecal immunochemical test (FIT), 10-yearly colonoscopy, 5-yearly double-contrast barium enema (DCBE), 5-yearly computed tomographic colonography (CTC), 5-yearly flexible sigmoidoscopy (FS), and no screening was calculated and compared. Four hundred participants were included. All attributes, except pain and less bowel preparation, were statistically associated with the participants’ preference (p The risk reduction of CRC-related mortality, complication, screening interval, and cost influenced the CRC screening preferences of Thai adults. FIT was the most preferred. Policymakers can develop a successful CRC screening campaign using these findings, incorporating the perspective of the population at risk in policy formulation to accomplish their goals.

The study objective was to (1) develop a statistical model that creates a novel patient engagement score (PES) from electronic medical records (EMR) and health claim data, and (2) validate this developed score using health-related outcomes and charges of patients with multiple chronic conditions (MCCs). This study used 2014-16 EMR and health claim data of patients with MCCs from Sanford Health. Patient engagement score was created based on selected patients’ engagement behaviors using Gaussian finite mixture model. The PES was validated using multiple logistic and linear regression analyses to examine the associations between the PES and health-related outcomes, and hospital charges, respectively. Patient engagement score was generated from 5095 patient records and included low, medium, and high levels of patient engagement. The PES was a significant predictor for low-density lipoprotein, emergency department visit, hemoglobin A1c, estimated glomerular filtration rate, hospitalization, and hospital charge. The PES derived from patient behaviors recorded in EMR and health claim data can potentially serve as a patient engagement measure. Further study is needed to refine and validate the newly developed score.

Introduction: Cancer patients are at increased risk of experiencing cardiotoxicity during their fluoropyrimidine-based chemotherapy. However, risk factors for fluoropyrimidine-induced cardiotoxicity (FIC) are not entirely understood. Methods: We searched PubMed, PsycINFO, IPA, CINAHL, Web of Science and ClinicalTrials.gov for studies published between January 1, 1990 and December 31, 2019, examining risk factors for cardiotoxicity induced by 5-fluorouracil, capecitabine or floxuridine. Two reviewers independently assessed publication quality and extracted study-level data into standardized evidence tables. Review Manager 5 software was used to convert data to risk ratios (RRs) and calculate pooled RRs for meta-analyses using a random-effects method. We conducted a Cochran’s Q test and obtained I 2 index to quantify study heterogeneity in each meta-analysis, with prediction interval (PI) to show the expected range of true effects in future similar studies. Results: Of 690 publications identified for abstract and title screening, 22 unique studies were included in the final review, and 20 of them had sufficient data for meta-analyses. Results indicated that patients undergoing capecitabine-based combination therapy had a higher risk of FIC than those with capecitabine monotherapy (pooled RR=1.61, 95% CI=1.01-2.55, I 2 =0%, 95% PI=0.08-31.71). Also, patients with pre-existing cardiac disease (pooled RR=3.01, 95% CI=2.02-4.49, I 2 =42%, 95% PI=1.03-8.78), hypertension (pooled RR=1.52, 95% CI=1.20-1.93, I 2 =0%, 95% PI=1.08-2.13) or smoking habit (pooled RR=2.22, 95% CI=1.03-4.78, I 2 =39%, 95% PI=0.15-32.46) had a significantly higher risk of FIC than their counterparts, while gender and comorbidities including diabetes, hyperlipidemia, and obesity were not significant risk factors of FIC. Conclusions: Cancer patients with pre-existing cardiac disease, hypertension, and smoking behavior had a higher risk of FIC when they are undergoing fluoropyrimidine-based treatments. Further research is needed to develop risk assessment tools for a risk prediction of FIC among cancer patients, which could advance risk stratification strategies and improve patient outcomes during the application of fluoropyrimidine-based treatments.

Background High and ever-increasing costs of Multiple Sclerosis (MS) disease-modifying therapies (DMTs) have impaired patient access to DMTs in the US. Patients’ willingness-to-pay (WTPs) for DMTs were recently examined, but their influencers were not determined. Thus, the objective of this study was to examine factors influencing patients’ WTPs for DMTs for multiple sclerosis (MS). Methods Data were obtained from a previous survey of 1,200 US patients with MS on their preferences and WTPs for DMTs. Patients’ characteristics (i.e., age, gender, race, marital status, education, employment status, comorbidity, health status, and health insurance) and their MS experiences (i.e., number of years with MS, MS type, number of relapses, fatigue, mood-change, MS symptom, and DMTs experience) were investigated as influencing factors. Patient's WTP for a DMT was obtained from a direct question in the survey. A two-part model was estimated using logistic regression and generalized linear regression. Results Responses from 480 patients were analyzed. Their average age was 53 years old. Most of them were female (79%), white (97%), and married (71%). Approximately 61% of them had a four-year college degree or lower, 54% were either unemployed, retired, or students, 59% were enrolled in private insurance, 81% had at least one comorbid condition, and 73% considered themselves having good or better health status. Approximately 44% had at least one relapse in two years, 89% experienced fatigue, 37% experienced mood-change, and on average had MS for more than 13 years. The majority of them had relapsing-remitting MS (66%), considered themselves to have some levels of disability for MS (78%), and had used or were currently using DMTs (97%). The average WTP for a DMT was $579 per month. Patients with professional degrees, or with one or more comorbid conditions were more likely willing to pay for a DMT. Patients who were white, had a professional degree, or were in fair or better health status were willing to pay a significantly higher amount for a DMT. Patients, who were female, were employed, did not have private insurance, had a higher number of MS experience years, or who experienced mood change were willing to pay significantly less amount for a DMT. Conclusion Various patients’ characteristics and MS experiences, including gender, race, education, employment, health insurance, comorbidity, health status, DMT experience, and mood change influenced patients’ WTPs for a DMT.

Background This study examined the feasibility of an interprofessional high-fidelity pharmacology simulation and its impact on pharmacy and nursing students’ perceptions of interprofessionalism and pharmacology knowledge. Interprofessional education activity Pharmacy and nursing students participated in a pharmacology simulation using a high-fidelity patient simulator. Faculty-facilitated debriefing included discussion of the case and collaboration. To determine the impact of the activity on students’ perceptions of interprofessionalism and their ability to apply pharmacology knowledge, surveys were administered to students before and after the simulation. Attitudes Toward Health Care Teams scale (ATHCT) scores improved from 4.55 to 4.72 on a scale of 1–6 (p = 0.005). Almost all (over 90%) of the students stated their pharmacology knowledge and their ability to apply that knowledge improved following the simulation. Discussion A simulation in pharmacology is feasible and favorably affected students’ interprofessionalism and pharmacology knowledge perceptions. Implications Pharmacology is a core science course required by multiple health professions in early program curricula, making it favorable for incorporation of interprofessional learning experiences. However, reports of high-fidelity interprofessional simulation in pharmacology courses are limited. This manuscript contributes to the literature in the field of interprofessional education by demonstrating that an interprofessional simulation in pharmacology is feasible and can favorably affect students’ perceptions of interprofessionalism. This manuscript provides an example of a pharmacology interprofessional simulation that faculty in other programs can use to build similar educational activities.

Background While disease-modifying therapies (DMTs) for multiple sclerosis (MS) treatments are costly, patient valuation of DMTs has not been examined. The objective of this study was to examine patients' preferences and willingness-to-pay (WTP) for DMTs. Methods Six attributes (i.e., number of relapses, percentage of disability progression, percentage of severe adverse events, route of administration, frequency of administration, and out-of-pocket cost) and their levels were used to develop a discrete choice experiment questionnaire. Each questionnaire comprised seven choice sets and each choice set contained two hypothetical DMTs and an opt-out alternative. A total of 1,200 U.S. patients with MS were asked to choose a DMT option or opt-out in each choice set. Multinomial logit model was used to determine relative preferences of each attribute. WTPs for all attributes and DMTs were calculated. Results A total of 508 patients were analyzed. Patients preferred DMTs with lower relapse rate, lower disability progression, lower severe adverse event, lower frequency of administration, and lower cost. In addition, they preferred oral DMTs. They were willing to pay $2,768, $289, $292, and $76 a month in exchange for every 1-time decrease in the number of relapses in two years, every 1% decrease in disability progression in two years, every 1% decrease in severe adverse events, and every 1-time decrease in the frequency of administration per month, respectively. The patients were willing to pay, in relation to market prices, between $7,020 and $134,934 per year for all DMTs, but interferon beta-1a SC. Conclusions Patients with MS considered relapse rate, disability progression, severe adverse events, route of administration, frequency of administration, and out-of-pocket cost, when they chose DMTs. Their WTPs for DMTs varied widely.

Introduction Filgrastim, a granulocyte colony-stimulating factor, is commonly used in autologous hematopoietic stem cell transplants (HSCTs) to assist with peripheral blood progenitor cell (PBPC) collection and to support stem cell engraftment. In the United States, tbo-filgrastim is approved under its own Biologic License Application and is limited to a single indication excluding the HSCT population. Methods Approximately one year after a system-wide formulary change to tbo-filgrastim for all on- and off-label indications, our institution conducted an IRB-approved retrospective comparison of tbo-filgrastim to filgrastim in the autologous HSCT setting. The study included 71 patients who received an autologous HSCT from 1 January 2013 to 31 December 2016 with a documented administration of tbo-filgrastim or filgrastim. Results There were no statistically significant differences noted on CD34 + counts during stem cell mobilization, neutrophil engraftment, infection rates during the engraftment phase, nor duration of hospitalization during the engraftment phase. More patients in the tbo-filgrastim group received plerixafor per protocol resulting in more patients meeting their PBPC collection goal in one day with fewer collection days overall, a result potentially confounded by institutional protocol changes. Utilizing tbo-filgrastim offered an average cost savings per patient of $2664.26 ($1907.33 for PBPC mobilization and $756.93 for stem cell engraftment) when comparing dollars spent on granulocyte colony-stimulating factor products only. Conclusion Tbo-filgrastim demonstrates comparable efficacy with a cost savings benefit compared to filgrastim for autologous PBPC mobilization and stem cell engraftment.

Supplemental Digital Content is available in the text., New melanoma therapies have shifted the expectations of patients and providers. Evaluating the impact of treatment characteristics may enhance shared decision making. A survey, including a discrete choice experiment, was utilized to evaluate perceived trade-offs of different melanoma treatments and to estimate out-of-pocket (OOP) willingness-to-pay (WTP) thresholds (January 2016 to March 2016). Participants included patients with melanoma at Huntsman Cancer Institute and their cancer care providers. Stakeholder focus groups were conducted to identify treatment attributes. Descriptive and comparative statistics and multinomial logit model were used to evaluate responses. Response rates were 41.9% (N = 220) for patients and 37.7% (N = 20) for providers. Immunotherapy and targeted therapy attributes considered important by participants were overall survival, immunotherapy-related side effects, and skin toxicities. Patients and providers had significantly different views of quality-of-life expectations, anxiety toward melanoma, trust to make treatment decisions, sharing concerns about treatment, time to discuss treatment, understanding OOP costs, and willingness to undergo/recommend treatment (half of the patients would undergo treatment if it was effective for > 24 months). Among patients, the average monthly OOP WTP for combination immunotherapy with nivolumab + ipilimumab was $ 2357 and for BRAF/MEK inhibitor was $1648. Among providers, these estimates were $ 2484 and $1350, respectively. Discordance existed between patients’ and providers’ perceptions about quality of life expectations, degree of anxiety, sharing of opinions, and progression-free survival. Our study suggests that patients and providers exhibit a higher OOP WTP for combination immunotherapy treatment compared with BRAF/MEK inhibitors, influenced predominately by overall survival expectations.

There is a high and increasing global prevalence of nutraceuticals use. This study aims to systematically review and critically appraise all available evidence to identify the factors affecting consumers' decisions in taking nutraceuticals. Questionnaire, interview or focus group studies which directly reported factors affecting consumers' decisions in using nutraceuticals were included. A thematic synthesis method was employed to synthesis the findings from the included studies. Out of the 76 studies included, the key factors identified as the most important factors motivating consumers to take nutraceuticals were the perceived health benefits and safety of nutraceuticals, as well as the advice from healthcare professionals, friends and family. The identified barriers to take nutraceuticals were a lack of belief in the health benefit of nutraceuticals, the high cost of nutraceuticals and consumers' lack of knowledge about nutraceuticals. As a chief course of recommendation for the use of nutraceuticals, healthcare professionals should strive to utilise reliable information from clinical evidence to help consumers in making an informed decision in using nutraceuticals. Future studies should explore the possible ways to improve channelling clinical evidence information of nutraceuticals to the public.

Cancer patients experienced an increased risk of cardiotoxicity during fluoropyrimidine-based chemotherapy (5-fluorouracil or capecitabine). We searched PubMed, PsycINFO, IPA, CINAHL, Web of Science, and ClinicalTrials.gov for studies published between January 1, 1990 and December 31, 2019, in English, examining risk factors for cardiotoxicity induced by fluoropyrimidine. Included study-level data were converted to risk ratios (RRs) and pooled RRs were calculated for meta-analyses using a random-effects method. Among 690 publications identified for abstract and title screening, 22 unique studies were included in the review, and 20 had sufficient data for meta-analyses. Results indicated that patients undergoing capecitabine-based combination therapy had a higher risk than those with monotherapy (pooled RR = 1.61). Patients with pre-existing cardiac disease (pooled RR = 3.26), hypertension (pooled RR = 1.52) or smoking (pooled RR = 2.22) also had higher risks than their counterparts. Developing risk assessment tools to mitigate the risk could be a viable strategy to improve outcomes for cancer patients undergoing fluoropyrimidine-based treatments.

The purpose of this study is to assess the benefit of bedside alteplase preparation as a component of the acute stroke process.A retrospective, single center study, designed to evaluate the impact of a bedside alteplase preparation protocol. Stroke patients receiving intravenous (IV) alteplase prepared at bedside were compared to pre-bedside alteplase preparation patients. The primary outcome was to compare door-to-needle (DTN) times between the groups. The secondary outcomes included comparison of pre-bedside alteplase preparation to post-bedside alteplase preparation on the following variables: imaging-to-drug times, order entry to drug administration times, percentage of patients achieving the 60 minute DTN goal, rate of intracranial hemorrhage (ICH), and patient discharge disposition.Patients in the pre-bedside preparation group included those who received IV alteplase between Jan. 1, 2012 and Jan. 31, 2015 and post-bedside preparation patients between Feb. 1, 2015 and March 31, 2016. Thirty-one patients were enrolled in the study, 16 in the pre-bedside preparation group and 15 in the post-beside preparation group. The mean DTN time in the post-bedside alteplase preparation group was significantly reduced, as compared to the pre-bedside preparation group (66.6 minutes vs. 95.9 minutes, p=0.024). Percent of patients meeting the 60 minute DTN time goal was significantly improved when alteplase was prepared at bedside (53.3 percent vs. 18.8 percent) (p=0.044). Rates of ICH were not significantly different between the two populations.Bedside alteplase preparation significantly reduced DTN times in an academic hospital emergency department.

Background Lenalidomide and pomalidomide are two immunomodulatory medications with the potential to improve outcomes for patients with multiple myeloma; however, a black box warning for venous thromboembolism exists. Purpose The purpose of this study was to assess overall adherence to guideline recommendations for anticoagulation therapy with lenalidomide and pomalidomide in multiple myeloma patients. Methods This retrospective study at an ambulatory oncology clinic utilized chart reviews from the calendar years 2013–2016. The primary endpoint was prescription of appropriate anticoagulation upon initiation of therapy based on a list of predetermined risk factors. Secondary endpoints included incidence of deep venous thromboembolism, pulmonary embolism, myocardial infarction, stroke, and major bleed; initial anticoagulant prescribed; and whether or not anticoagulation was prescribed for another disease state. Results A total of 130 patients met inclusion criteria: 70.8% (n = 92) and 29.2% (n = 38) were prescribed lenalidomide and pomalidomide, respectively. A total risk score of two was most common (n = 54, 41.5%). Aspirin 81 mg oral tablet was prescribed most often (n = 53, 40.8%), followed by no anticoagulation (n = 30, 23.1%). Overall, 27 patients (20.8%) were prescribed anticoagulation in accordance with National Comprehensive Cancer Network guidelines. Incidence of deep venous thromboembolism was the most common adverse event (n = 4, 3.1%), followed by major bleed (n = 1, 0.8%). No reports of pulmonary embolism, myocardial infarction, or stroke were documented. Conclusions Overall, a disparity exists between appropriate prescribing of prophylactic anticoagulation and current practice guidelines. However, documentation of thromboembolic events was lower than recorded in previously published literature.

Objectives To describe the process, challenges, and future direction of health technology assessment (HTA), focusing on the drug selection of the National List of Essential Medicines (NLEM) in Thailand. Methods Literature and government documents were reviewed and analyzed by authors with experiences in HTA and drug policy in the country. Results The structure of HTA and its process in the drug selection of the NLEM were described, followed by the outcomes of the use of HTA. Examples of lowering drug prices, as a result of price negotiation using HTA, were presented. A few examples were also provided to demonstrate how decisions were made from considering factors beyond cost-effectiveness findings. Finally, challenges on various issues including improvement of HTA structure and process were discussed for the future direction of HTA in Thailand. Conclusions HTA has been adopted as a tool for the drug selection of the NLEM to help Thailand achieve universal health coverage. Nevertheless, various challenges exist and need to be addressed.

OBJECTIVES The selection of sedative medications for mechanically ventilated pediatric patients remains an ongoing clinical challenge. Although continuous ketamine infusion has been used in this population, support for its use remains largely anecdotal. This study describes a single institution's use of ketamine infusions as part of a sedation protocol in the pediatric intensive care unit (PICU). METHODS This was a retrospective study of children who received ketamine infusions as part of a multidrug sedation protocol in a 12-bed PICU at a tertiary children's hospital. Outcomes included effectiveness of ketamine infusion in providing adequate sedation as determined by State Behavioral Scale (SBS) scores and incidence of adverse events. RESULTS A total of 22 children receiving ketamine continuous infusion as part of a multidrug sedation protocol from February 2014 through October 2015 were eligible and enrolled in the study. Ketamine continuous infusion was administered in addition to at least 2 other sedation infusions at an average rate of 1.02 ± 0.50 mg/kg/hr, with a range of 0.07 to 2.0 mg/kg/hr. The duration of ketamine was 65.7 ± 41.01 hours, with a range of 19 to 153 hours. There was no significant change in SBS scores before and after initiation of ketamine infusion. Although not statistically significant, patients with inadequate sedation prior to starting ketamine required fewer bolus sedation doses and had improved sedation after ketamine was started. There were no reported adverse events. CONCLUSIONS The addition of a ketamine infusion as part of a multidrug sedation regimen was at least as effective as patients' regimen prior to ketamine addition in this population of intubated pediatric patients, with no adverse events.