Symptomatology and knowledge regarding pelvic floor dysfunctions and influence of gender stereotypes in female athletes.

Pelvic floor dysfunctions (PFD) are highly prevalent among females who do athletics, a sport requiring jumping, strength, and running. Although educational approaches are useful options, the educational need for this particular population remains unknown. The objective of the present study was to describe the level of knowledge regarding PFD and its relationship with symptomatology and gender stereotypes in female athletes in Spain. A total of 255 female athletes completed an anonymous online survey to explore their knowledge regarding urinary incontinence (UI), pelvic organ prolapse (POP), anal incontinence (AI), and sexual dysfunction (SexD), as well as their PFD symptoms and gender stereotyped beliefs related to sport. Educational level and sports characteristics (training volume, experience, and athletic modality) were also explored. Participants demonstrated a low level of knowledge in terms of POP (52.5%), AI (64.0%), and SexD (40%), but not for UI (70.8%). The proportion of PFD complaints was 63.5% for dyspareunia, 51.8% for urine leakage, 42.4% for pelvic pain, 17.3% for AI, and 9.0% for POP, with no associations with knowledge (p > 0.05). Lower knowledge about UI and SexD was related to greater gender stereotypes (p < 0.05) and rejection of professional healthcare (p = 0.010). As a conclusion, the level of knowledge about PFD was low in female athletes who train and compete in athletics in Spain, mainly with regard to sexual dysfunction. Although 63.5% of athletes had dyspareunia and 51.8% urinary leakages, symptomatology was not associated with level of knowledge. However, a lower level of knowledge was associated with more stereotyped beliefs and rejection of professional healthcare for PFD. These findings confirm the need to design appropriate educational interventions to disseminate information on all the types of PFD, particularly sexual contents. The potential influence of gender stereotypes makes it appropriate to include the gender perspective in these interventions.

The Peptidoglycan Recognition Protein 1 confers immune evasive properties on pancreatic cancer stem cells.

OBJECTIVE: Pancreatic ductal adenocarcinoma (PDAC) has limited therapeutic options, particularly with immune checkpoint inhibitors. Highly chemoresistant 'stem-like' cells, known as cancer stem cells (CSCs), are implicated in PDAC aggressiveness. Thus, comprehending how this subset of cells evades the immune system is crucial for advancing novel therapies. DESIGN: We used the KPC mouse model (LSL-KrasG12D/+; LSL-Trp53R172H/+; Pdx-1-Cre) and primary tumour cell lines to investigate putative CSC populations. Transcriptomic analyses were conducted to pinpoint new genes involved in immune evasion. Overexpressing and knockout cell lines were established with lentiviral vectors. Subsequent in vitro coculture assays, in vivo mouse and zebrafish tumorigenesis studies, and in silico database approaches were performed. RESULTS: Using the KPC mouse model, we functionally confirmed a population of cells marked by EpCAM, Sca-1 and CD133 as authentic CSCs and investigated their transcriptional profile. Immune evasion signatures/genes, notably the gene peptidoglycan recognition protein 1 (PGLYRP1), were significantly overexpressed in these CSCs. Modulating PGLYRP1 impacted CSC immune evasion, affecting their resistance to macrophage-mediated and T-cell-mediated killing and their tumourigenesis in immunocompetent mice. Mechanistically, tumour necrosis factor alpha (TNFα)-regulated PGLYRP1 expression interferes with the immune tumour microenvironment (TME) landscape, promoting myeloid cell-derived immunosuppression and activated T-cell death. Importantly, these findings were not only replicated in human models, but clinically, secreted PGLYRP1 levels were significantly elevated in patients with PDAC. CONCLUSIONS: This study establishes PGLYRP1 as a novel CSC-associated marker crucial for immune evasion, particularly against macrophage phagocytosis and T-cell killing, presenting it as a promising target for PDAC immunotherapy.

Suggesting global insights to local challenges: expanding financing of rehabilitation services in low and middle-income countries.

Purpose: Following the rapid transition to non-communicable diseases, increases in injury, and subsequent disability, the world-especially low and middle-income countries (LMICs)-remains ill-equipped for increased demand for rehabilitative services and assistive technology. This scoping review explores rehabilitation financing models used throughout the world and identifies "state of the art" rehabilitation financing strategies to identify opportunities and challenges to expand financing of rehabilitation. Material and methods: We searched peer-reviewed and grey literature for articles containing information on rehabilitation financing in both LMICs and high-income countries. Results: Forty-two articles were included, highlighting various rehabilitation financing mechanism which involves user fees and other innovative payment as bundled or pooled schemes. Few studies explore policy options to increase investment in the supply of services. Conclusion: this paper highlights opportunities to expand rehabilitation services, namely through promotion of private investment, improvement in provider reimbursement mechanism as well as expanding educational grants to bolster labor supply incentive, and the investment in public and private insurance schemes. Mechanisms of reimbursement are frequently based on global budget and salary which are helpful to control cost escalation but represent important barriers to expand supply and quality of services.

Is economic growth enough to propel rehabilitation expenditures? An empirical analysis of country panel data and policy implications.

PURPOSE: Rehabilitation is a set of services designed to increase functioning and improve wellbeing across the life course. Despite being a core part of Universal Health Coverage, rehabilitation services often receive limited public expenditure, especially in lower income countries. This leads to limited service availability and high out of pocket payments for populations in need of care. The purpose of this research was to assess the association between macroeconomic conditions and rehabilitation expenditures across low-, middle-, and high-income countries and to understand its implications for overall rehabilitation expenditure trajectory across countries. MATERIALS AND METHODS: We utilized a panel data set from the World Health Organization's Global Health Expenditure Database comprising the total rehabilitation expenditure for 88 countries from 2016 to 2018. Basic macroeconomic and population data served as control variables. Multiple regression models were implemented to measure the relationship between macroeconomic conditions and rehabilitation expenditures. We used four different model specifications to check the robustness of our estimates: pooled data models (or naïve model) without control, pooled data models with controls (or expanded naïve model), fixed effect models with all controls, and lag models with all controls. Log-log specifications using fixed effects and lag-dependent variable models were deemed the most appropriate and controlled for time-invariant differences. RESULTS: Our regression models indicate that, with a 1% increase in economic growth, rehabilitation expenditure would be associated with a 0.9% and 1.3% increase in expenditure. Given low baseline levels of existing rehabilitation expenditure, we anticipate that predicted increases in rehabilitation expenditure due to economic growth may be insufficient to meet the growing demand for rehabilitation services. Existing expenditures may also be vulnerable during periods of economic recession. CONCLUSION: This is the first known estimation of the association between rehabilitation expenditure and macroeconomic conditions. Our findings demonstrate that rehabilitation is sensitive to macroeconomic fluctuations and the path dependency of past expenditures. This would suggest the importance of increased financial prioritization of rehabilitation services and improved institutional strengthening to expand access to rehabilitation services for populations.

Design of an innovative digital application to facilitate access to healthy foods in low-income urban settings.

Background: Under-resourced urban minority communities in the United States are characterized by food environments with low access to healthy foods, high food insecurity, and high rates of diet-related chronic disease. In Baltimore, Maryland, low access to healthy food largely results from a distribution gap between small food sources (retailers) and their suppliers. Digital interventions have the potential to address this gap, while keeping costs low. Methods: In this paper, we describe the technical (I) front-end design and (II) back-end development process of the Baltimore Urban food Distribution (BUD) application (app). We identify and detail four main phases of the process: (I) information architecture; (II) low and high-fidelity wireframes; (III) prototype; and (IV) back-end components, while considering formative research and a pre-pilot test of a preliminary version of the BUD app. Results: Our lessons learned provide valuable insight into developing a stable app with a user-friendly experience and interface, and accessible cloud computing services for advanced technical features. Conclusions: Next steps will involve a pilot trial of the app in Baltimore, and eventually, other urban and rural settings nationwide. Once iterative feedback is incorporated into the app, all code will be made publicly available via an open source repository to encourage adaptation for desired communities. Trial Registration: ClinicalTrials.gov NCT05010018.

Exploring the Influence of Health Insurance Plans on Biosimilar Adoption Rates.

BACKGROUND: We conducted a study to investigate the role of health insurance plans on biosimilar adoption among commercially insured patients in the USA. Flexible and rigid health plans may exhibit differing biosimilar coverage due to variations in cost considerations, formulary design, and provider networks. OBJECTIVE: To identify the characteristics of switchers and biosimilar initiators for six biologic-biosimilar pairs. METHODS: Using claims data from 2015 to 2019, we implement sequential regression models to assess the role of health plans on biosimilars adoption. FINDINGS: We found that low-flexibility plans, such as Health Maintenance Organization (HMOs) and Exclusive Provider Organization (EPOs), are more likely to have patients who are switchers and/or biosimilar initiators. CONCLUSION: Our findings highlight the importance of health insurance plan design in promoting biosimilar uptake.

Determinants of Communication Failure in Intubated Critically Ill Patients: A Qualitative Phenomenological Study from the Perspective of Critical Care Nurses.

AIM: To explore what factors determine communication with awake intubated critically ill patients from the point of view of critical care nursing professionals. BACKGROUND: Impaired communication frequently affects mechanically ventilated patients with artificial airways in the intensive care unit. Consequences of communication breaches comprise emotional and ethical aspects as well as clinical safety, affecting both patients and their conversation partners. Identification of determining factors in communication with awake intubated patients is needed to design effective action strategies. DESIGN: A qualitative phenomenological approach was used. METHODS: Semi-structured interviews were used as the data collection method. A total of 11 participants from three intensive care units of three Majorcan public hospitals, selected by purposive sampling, were interviewed. FINDINGS: Three major themes regarding the communication determinants of the awake intubated critically ill patients were identified from the interviewees' statements: factors related to the patient (physical and cognitive functionality to communicate, their relational and communicative style and their personal circumstances), to the context (family presence, ICU characteristics, workload, availability/adequacy of communication aids, features of the messages and communication situations) and, finally, those related to the professionals themselves (professional experience and person-centredness). CONCLUSIONS: The present study reveals determinants that influence communication with the awake intubated patient, as there are attitudes and professional beliefs. RELEVANCE TO CLINICAL PRACTICE: The discovery of relations between different kinds of determinants (of patient, context or professionals) provides a multi-factor perspective on the communicative problem which should be considered in the design of new approaches to improve communicative effectiveness. This study is reported according to the COREQ checklist.

Improving COVID-19 vaccine uptake: a message co-design process for a national mHealth intervention in Colombia.

BACKGROUND: COVID-19 vaccination is a global priority. Latin American countries have some of the highest COVID-19 death rates worldwide with vaccination hampered by a variety of reasons, including mis- and disinformation, vaccine hesitancy, and vaccine supply constraints. Addressing vaccine hesitancy through effective messages has been found to help increase vaccine uptake. Participatory processes could be used to co-design health messages for this purpose. OBJECTIVE: This article describes the methodology used to co-design evidence-based audio messages to be deployed in a cohort of individuals through an interactive voice response (IVR) mobile phone survey intervention, aimed towards increasing vaccination uptake in an adult population in Colombia. METHODS: Participants of the COVID-19 vaccination message co-design process included a sample of the general population of the country, representatives of the funder organisation, and research team members. The co-design process consisted of four phases: (1) formative quantitative and qualitative research, (2) message drafting based on the results of the formative research, (3) message content evaluation, and (4) evaluation of the voices to deliver the audio messages; and was informed by reflexive meetings. RESULTS: Three categories of evidence-based audio messages were co-designed, each corresponding to an arm of the mHealth intervention: (1) factual messages, (2) narrative messages, and (3) mixed messages. An additional fourth arm with no message was proposed for control. The iterative co-design process ended with a total of 14 audio messages recorded to be deployed via the intervention. CONCLUSIONS: Co-developing health messages in response to health emergencies is possible. Adopting more context-relevant, participatory, people-centred, and reflexive multidisciplinary approaches could help develop solutions that are more responsive to the needs of populations and public health priorities. Investing resources in message co-design is deemed to have a greater potential for influencing behaviours and improving health outcomes.

The associations of continuity of care with inpatient, outpatient, and total medical care costs among older adults with urinary incontinence.

INTRODUCTION: Urinary incontinence is a significant health problem with considerable social and economic consequences among older adults. The objective of this study was to investigate the financial impact of continuity of care (CoC) among older urinary incontinence patients in South Korea. METHODS: We used the NHIS-Senior cohort patient data between January 1, 2010, and December 31, 2010. Patients who were diagnosed with urinary incontinence in 2010 were included. Operational definition of CoC included referrals, number of providers, and number of visits. A generalized linear model (GLM) with γ-distributed errors and the log link function was used to examine the relationship between health cost and explanatory variables. Additionally, we conducted a two-part model analysis for inpatient cost. Marginal effect was calculated. RESULTS: Higher CoC was associated with a decrease in total medical cost (-0.63, P < .0001) and in outpatient costs (-0.28, P < .001). Higher Charlson Comorbidity Index (CCI) score was a significant predictor for increasing total medical cost (0.59, P < .0001) and outpatient cost (0.22, P < .0001). Higher CoC predict a reduced medical cost of $360.93 for inpatient cost (P = 0.044) and $23.91 for outpatient cost (P = 0.008) per patient. CONCLUSION: Higher CoC was associated with decrease in total medical costs among older UI patients. Policy initiatives to promote CoC of older UI patients in the community setting could lead to greater financial sustainability of public health insurance in South Korea.

Strengthening HIV and HIV co-morbidity care in low- and middle-income countries: insights from behavioural economics to improve healthcare worker behaviour.

INTRODUCTION: Despite advances in HIV and HIV co-morbidity service delivery, substantial challenges remain in translating evidence-based interventions into routine practice to bring optimal care and prevention to all populations. While barriers to successful implementation are often multifactorial, healthcare worker behaviour is critical for on-the-ground and in-clinic service delivery. Implementation science offers a systematic approach to understanding service delivery, including approaches to overcoming delivery gaps. Behavioural economics is a field that seeks to understand when and how behaviour deviates from traditional models of decision-making, deviations which are described as biases. Clinical policies and implementation strategies that incorporate an understanding of behavioural economics can add to implementation science approaches and play an important role in bridging the gap between healthcare worker knowledge and service delivery. DISCUSSION: In HIV care in low- and middle-income countries (LMICs), potential behavioural economic strategies that may be utilized alone or in conjunction with more traditional approaches include using choice architecture to exploit status quo bias and reduce the effects of cognitive load, overcoming the impact of anchoring and availability bias through tailored clinical training and clinical mentoring, reducing the effects of present bias by changing the cost-benefit calculus of interventions with few short-term benefits and leveraging social norms through peer comparison. As with any implementation strategy, understanding the local context and catalysts of behaviour is crucial for success. CONCLUSIONS: As the focus of HIV care shifts beyond the goal of initiating patients on antiretroviral therapy to a more general retention in high-quality care to support longevity and quality of life, there is an increasing need for innovation to achieve improved care delivery and management. Clinical policies and implementation strategies that incorporate elements of behavioural economic theory, alongside local testing and adaptation, may increase the delivery of evidence-based interventions and improve health outcomes for people living with HIV in LMIC settings.

Examining Opportunities to Increase Savings From Medicare Price Negotiations.

Importance: Allowing the US Centers for Medicare & Medicaid Services to negotiate prescription drug prices for Medicare may improve drug affordability. Objective: To estimate savings from Medicare price negotiation under the Inflation Reduction Act (IRA) and examine opportunities to increase savings. Design, Setting, and Participants: This cross-sectional, population-based study used data from 2020 Medicare prescription drug claims. The study was conducted and data were analyzed in 2022. Exposures: Eligibility for Medicare price negotiation under the IRA and alternative criteria. Main Outcomes and Measures: Minimum savings under the IRA's eligibility criteria were estimated and compared with savings within alternative scenarios, including (1) selecting drugs for negotiation based on net spending after rebates rather than gross spending; (2) extending eligibility to drugs with biosimilar or generic competitors; (3) reducing the minimum years since US Food and Drug Administration approval for eligibility; and (4) changing 2 or 3 of these factors. Estimated savings were calculated at different levels of scale-up of price negotiation under the IRA, from 10 Part D drugs in 2026 to 60 Part B and D drugs in 2029. Gross spending was calculated using the US Centers for Medicare & Medicaid Services 2020 Medicare drug spending dashboard. Rebates were estimated using SSR Health data. Information on FDA approvals, generics, and biosimilars was obtained from FDA websites. Results: Under IRA rules, estimated minimum savings from price negotiation in 2026 for 10 Part D drugs would be $3.2 billion. For 2029 for 60 Part D and B drugs, estimated savings were $16.0 billion. Selecting drugs for negotiation based on net rather than gross spending would be associated with estimated savings of $4.6 billion (a 45% increase) in 2026 and $18.9 billion (an 18% increase) in 2029. Including drugs with generic competitors or biosimilars would be associated with an estimated savings of $6.6 billion (a 109% increase) in 2026 and $24.9 billion (a 56% increase) in 2029. Making both changes would be associated with savings of $9.5 billion (a 200% increase) in 2026 and $28.3 billion (a 77% increase) in 2029. A sensitivity analysis suggested that reducing the required number of years since marketing approval by 2 years would be associated with increased estimated savings of 4% when 10 Part D drugs are negotiated and 12% when 60 Part D and B drugs are negotiated. Changing all 3 criteria would be associated with the greatest increase in estimated savings in 2029 (119% increase when 10 Part D drugs are negotiated and 93% increase for 60 Part D and B drugs). Conclusions and Relevance: The results of this cross-sectional study suggest that adjusting the eligibility criteria for Medicare prescription drug price negotiation to permit inclusion of drugs with biosimilar or generic competitors and selecting drugs based on net rather than gross spending may be a promising approach to substantially increase estimated savings.

Accelerated approval drug labels often lack information for clinical decision-making.

STUDY OBJECTIVE: We evaluated US Food and Drug Administration labels for drugs approved under the accelerated approval pathway and whether these labels contained in sufficient information regarding their accelerated approval. DESIGN: Retrospective, observational, cohort study. DATA SOURCE: Label information for drugs with an accelerated approved indication were ascertained from two online platforms: Drugs@FDA and FDA Drug Label Repository. INTERVENTION: Drugs with indications receiving accelerated approval after January 1, 1992, but had not received full approval by December 31, 2020. MEASUREMENTS: Outcomes include whether the drug label indicated the use of the accelerated approval pathway, identified the specific surrogate marker(s) that supported it, or described the clinical outcomes being evaluated in post-approval commitment trials. RESULTS: 253 clinical indications corresponding to 146 drugs received accelerated approval. We identified a total of 110 accelerated approval indications across 62 drugs that had not received full approval by December 31, 2020. A total of 13% of labels for accelerated approved indications lacked sufficient information that approval was via the accelerated approval or based on surrogate outcome measures: 7% did not mention accelerated approval but described surrogate markers, 4% did not mention accelerated approval nor describe surrogate markers, and 2% mentioned accelerated approval but did not describe surrogate markers. No label described the clinical outcomes being evaluated in post-approval commitment trials. CONCLUSION: Labels for accelerated approved clinical indications that do not yet have full approval should be revised to include the information required in the FDA guidance to help guide clinical decision-making.

Effect of Ultra-High Pressure Homogenization (UHPH) and Conventional Thermal Pasteurization on the Volatile Composition of Tiger Nut Beverage.

Tiger nut beverages are non-alcoholic products that are characterized by their pale color and soft flavor. Conventional heat treatments are widely used in the food industry, although heated products are often damaging to their overall quality. Ultra-high pressure homogenization UHPH) is an emerging technology that extends the shelf-life of foods while maintaining most of their fresh characteristics. The present work deals with the comparison of the effect of conventional thermal homogenization-pasteurization (H-P, 18 + 4 MPa at 65 °C, 80 °C for 15 s.) and UHPH (at 200 and 300 MPa, and inlet temperature of 40 °C), on the volatile composition of tiger nut beverage. Headspace-solid phase microextraction (HS-SPME) was used for detecting volatile compounds of beverages, which were then identified by gas chromatography-mass spectrometry (GC-MS). A total of 37 different volatile substances were identified in tiger nut beverages, which were primarily grouped into the aromatic hydrocarbons, alcohols, aldehydes and terpenes chemical families. Stabilizing treatments increased the total amount of volatile compounds (H-P > UHPH > R-P). H-P was the treatment that produced the most changes in the volatile composition of RP, while treatment at 200 MPa had a minor impact. At the end of their storage, these products were also characterized by the same chemical families. This study evidenced the UHPH technology as an alternative processing of tiger nut beverages production that minimally modifies their volatile composition.

The price paradox of biosimilar-like long-acting insulin.

OBJECTIVES: To describe the uptake and out-of-pocket (OOP) costs of Basaglar, the first long-acting insulin biosimilar, in a commercially insured population in the United States. STUDY DESIGN: Retrospective analysis of commercial pharmacy claims and pharmacy co-payment offsets. METHODS: We assessed Basaglar uptake by examining trends in the composition of the long-acting insulin market in the United States from 2014 to 2018. As patient demographics and plan type may be important determinants of biosimilar uptake, we also assessed characteristics of all long-acting insulin users by drug. We examined Basaglar OOP costs by assessing mean OOP costs per claim for users of Basaglar and other long-acting insulins, overall and by plan type, and the number and source of co-payment offsets for Basaglar and other insulin glargine products from Basaglar market entry through 2018. We used multivariate linear models to examine the relationship between Basaglar OOP expenditures and insurer-negotiated amounts, overall and by plan type. RESULTS: Basaglar experienced a rapid uptake. However, there was no evidence that Basaglar users had lower OOP costs than reference product (Lantus) users. CONCLUSIONS: Given our results and the approval of the first interchangeable biosimilar, we recommend the empirical evaluation of biosimilar cost savings to patients and insurers prior to promoting their automatic substitution.

Acid and Rennet Coagulation Properties of A2 Milk.

This study investigated the acid and rennet milk coagulation properties of A2 milk (ß-casein (CN) A2A2 genotype), in comparison to a control milk (blend of A2A1/A1A1/A2A2 genotypes). Acid and rennet coagulation were evaluated using the Optigraph® system, measuring the coagulation time, aggregation rate, and gel density or curd firmness. The acidification kinetics were monitored using a CINAC® system, evaluating the time to reach pH 4.6, the acidification rate, the maximum acidification rate, the time required to reach it, and the latency time. The water-holding capacity of acid milk gels and the potential yield, total solids, and syneresis of enzymatic gels were also evaluated. Some variables were highly influenced by the farm factor, showing the importance of the effect of extrinsic parameters. Acid and enzymatic coagulation times were not affected in either milk. The A2 milk presented higher acid gel density and latency time than the control milk. Although the differences in water-holding capacity were not statistically significant, the A2 milk presented lower values, related with the higher gel density. The A2 milk also showed higher rennet aggregation rate and curd firmness than the control milk. Potential yield and syneresis were higher in the A2 milk, which is in accordance with the higher firmness of curd. Coagulation results and gel and curd properties indicate that it is possible to manufacture acid and rennet coagulation dairy products from A2 milk with no major differences when compared with a control milk.

The Baltimore Urban Food Distribution (BUD) App: Study Protocol to Assess the Feasibility of a Food Systems Intervention.

Low-income urban communities in the United States commonly lack ready access to healthy foods. This is due in part to a food distribution system that favors the provision of high-fat, high-sugar, high-sodium processed foods to small retail food stores, and impedes their healthier alternatives, such as fresh produce. The Baltimore Urban food Distribution (BUD) study is a multilevel, multicomponent systems intervention that aims to improve healthy food access in low-income neighborhoods of Baltimore, Maryland. The primary intervention is the BUD application (app), which uses the power of collective purchasing and delivery to affordably move foods from local producers and wholesalers to the city's many corner stores. We will implement the BUD app in a sample of 38 corner stores, randomized to intervention and comparison. Extensive evaluation will be conducted at each level of the intervention to assess overall feasibility and effectiveness via mixed methods, including app usage data, and process and impact measures on suppliers, corner stores, and consumers. BUD represents one of the first attempts to implement an intervention that engages multiple levels of a local food system. We anticipate that the app will provide a financially viable alternative for Baltimore corner stores to increase their stocking and sales of healthier foods, subsequently increasing healthy food access and improving diet-related health outcomes for under-resourced consumers. The design of the intervention and the evaluation plan of the BUD project are documented here, including future steps for scale-up. Trial registration #: NCT05010018.

Trajectories of prices in generic drug markets: what can we infer from looking at trajectories rather than average prices?

BACKGROUND: Well-functioning competitive markets are key to controlling generic drug prices. This is important since over 90% of all drugs sold in the US are generics. Recently, there have been examples of large price increases in the generic market. METHODS: This paper examines price trajectories for generic drugs using a group-based trajectory modelling approach (GBTM). We fit the model using quarterly price information in the IBM MarketScan claims database for the past decade. RESULTS: We identify three dominant price trajectories for this period: rapid increase trajectories, slow decline and rapid decline. Most generic drugs show a slow or a rapid decline in price trajectories. However, around 17% of all generic drugs show rapid price increase trajectories. CONCLUSIONS: As Congress is exploring an excise tax on drugs whose list price increases faster than the rate of inflation, we discuss what drugs would be most likely to be affected by this law.

Medicare Spending on Drugs With Accelerated Approval.

BACKGROUND: The U.S. Food and Drug Administration provides accelerated approval to drugs on the basis of surrogate end points deemed to be "reasonably likely" to predict clinical benefit. To receive full approval, drugs must complete a confirmatory trial. Although most accelerated approved drugs ultimately receive full approval, others remain on the market without full approval for many years, and some are withdrawn before full approval is granted. Until confirmatory trials are completed and full approval is granted, there is uncertainty surrounding each drug's clinical benefits. OBJECTIVE: To estimate fee-for-service Medicare payments on accelerated approved drugs without full approvals. DESIGN: Cross-sectional analysis. SETTING: Fee-for-service Medicare Part B and Part D drug claims in 2019. PARTICIPANTS: Beneficiaries enrolled in Medicare Part B and Part D plans. MEASUREMENTS: Medicare spending for drugs treating accelerated approved indications without full approval, beneficiary spending, and drug characteristics. RESULTS: In 2019, 45 drugs associated with 69 accelerated approved indications lacked full approval. Of those, the fee-for-service Medicare program spent $1.2 billion on 36 drugs across 55 indications. Medicare beneficiaries had $209 million in out-of-pocket spending on these drugs. Oncology drugs represented 82% of these indications and 72% of the Medicare spending. Extrapolating to Medicare Advantage, total Medicare spending on these drugs in 2019 was $1.8 billion. LIMITATIONS: The study drugs may have clinical benefit and may come to receive full approval after this analysis. The algorithm used to identify accelerated approved indications is novel. Generalizability to other years is unclear. CONCLUSION: In 2019, fee-for-service Medicare spent $1.2 billion on accelerated approved drugs without full approval. Medicare should adjust incentives to encourage sponsors to complete confirmatory trials as soon as possible. PRIMARY FUNDING SOURCE: Laura and John Arnold Foundation.