Pub Date : 2024-02-20DOI: 10.1101/2024.02.18.24302990
Michael Essman, Tom Bishop, Thomas Burgoine, Andrew Jones, Megan Polden, Eric Robinson, Stephen J Sharp, Richard Smith, Jean Adams, Martin White
Background Mandatory calorie labelling on menus of large out-of-home food outlets was implemented in England on 6 April 2022. Barriers and facilitators that were unforeseen before implementation may modify policy impacts. As part of a process evaluation of the policy, we studied the implementation process, examining business experiences and enforcement by local authorities (LAs) to identify barriers and facilitators in achieving the policy goals. Methods Using purposive sampling, we recruited 11 employees of large food businesses (implementers) and 9 employees of LA environmental health or trading standards departments (enforcers). Post-implementation semi-structured interviews were conducted by video conference. Interviews were audio recorded, transcribed verbatim and analysed using the Framework Method. Results Both groups of participants described a decentralised approach to delivery and enforcement, and resource constraints meant LAs were unable to assist with all business inquiries. Enforcement activity was limited because complaints about labelling from the public were rare, and enforcers prioritized acute food safety issues. Pre-implementation discussions created the presumption among enforcers that most businesses were compliant. Businesses complied to safeguard their reputation and maintain customer trust. While participants supported calorie labelling, potential barriers to policy impact included a presumed lack of customer interest. Financial pressure during implementation strained business resources, and businesses suggested that customers may prioritise financial over health concerns in their decision-making. Conclusions These findings underscore the need for central guidance, verification of adherence, and sufficient enforcement resources. To optimize policy success, future developments should consider economic contexts, customer expectations, and policy refinement, while recognizing common industry arguments against policy implementation.
{"title":"Implementation and enforcement of mandatory calorie labelling regulations for the out-of-home sector in England: qualitative study of the experiences of business implementers and regulatory enforcers","authors":"Michael Essman, Tom Bishop, Thomas Burgoine, Andrew Jones, Megan Polden, Eric Robinson, Stephen J Sharp, Richard Smith, Jean Adams, Martin White","doi":"10.1101/2024.02.18.24302990","DOIUrl":"https://doi.org/10.1101/2024.02.18.24302990","url":null,"abstract":"Background\u0000Mandatory calorie labelling on menus of large out-of-home food outlets was implemented in England on 6 April 2022. Barriers and facilitators that were unforeseen before implementation may modify policy impacts. As part of a process evaluation of the policy, we studied the implementation process, examining business experiences and enforcement by local authorities (LAs) to identify barriers and facilitators in achieving the policy goals. Methods\u0000Using purposive sampling, we recruited 11 employees of large food businesses (implementers) and 9 employees of LA environmental health or trading standards departments (enforcers). Post-implementation semi-structured interviews were conducted by video conference. Interviews were audio recorded, transcribed verbatim and analysed using the Framework Method. Results\u0000Both groups of participants described a decentralised approach to delivery and enforcement, and resource constraints meant LAs were unable to assist with all business inquiries. Enforcement activity was limited because complaints about labelling from the public were rare, and enforcers prioritized acute food safety issues. Pre-implementation discussions created the presumption among enforcers that most businesses were compliant. Businesses complied to safeguard their reputation and maintain customer trust. While participants supported calorie labelling, potential barriers to policy impact included a presumed lack of customer interest. Financial pressure during implementation strained business resources, and businesses suggested that customers may prioritise financial over health concerns in their decision-making. Conclusions\u0000These findings underscore the need for central guidance, verification of adherence, and sufficient enforcement resources. To optimize policy success, future developments should consider economic contexts, customer expectations, and policy refinement, while recognizing common industry arguments against policy implementation.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139910561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-02-14DOI: 10.1101/2024.02.13.24302771
Jonah Popp, Eric Jutkowitz, Thomas Trikalinos
Background: In 2022, the Centers for Medicare & Medicaid Services (CMS) issued its final national coverage policy for aducanumab, a novel FDA-approved treatment for Alzheimers disease, deciding to Cover with Evidence Development (CED). CMS will thus only pay for the treatment of AD patients enrolled in an approved randomized controlled trial (RCT). We sought to understand whether, given current evidence, CED was best from a societal perspective. Methods: We conducted a modeling-based expected value of sample information analysis to estimate the expected net decision-theoretic value of a further RCT to evaluate the clinical efficacy of high-dose (10 mg/kg) aducanumab and to determine what sized trial, if any, is optimal conditional on an initial decision to cover or not. We also evaluated the expected net benefit of the manufacturers proposed RCT (ENVISION). We considered two post-trial decision criteria: cost-effectiveness given updated evidence (efficiency) and does the new trial demonstrate a statistical significant (p<0.05) clinical benefit. Results were used to calculate the expected population net monetary benefit (NMB) of four decision alternatives (including CED) depending on an initial coverage and trial decision. We ranked alternatives and calculated the expected opportunity loss of a suboptimal decision. We used a societal perspective and focused on willingness-to-pay (WTP) values for a quality-adjusted life year (QALY) between $50K-$200K. We conducted scenario analyses using different assumptions about population size, efficacy, and drug cost. Findings: The CMS decision to not cover aducanumab avoids an expected societal loss (NMB) of $15B-$110B. Even an optimally designed RCT would confer no or negative decision-theoretic value for WTP>$100K or with statistical significance as a post-trial decision criterion, respectively, and thus denying coverage without a trial (rather than CED) is clearly preferable. For WTP=$150K (WTP=$200K) and assuming an efficiency criterion, CED with ENVISION or a similar trial is reasonable (decidedly optimal). The case for future research would become less ambiguous if the manufacturer again voluntarily dropped the price >50%. Interpretation: The societal net value of a future trial (and thus CED) depends on how CMS would use the trial results to update its coverage decision and the WTP per QALY. Assuming CMS policymakers can avoid the pitfalls of a legal framework that limits their ability to consider costs in coverage decisions, the CED decision is at least reasonable, if not optimal, if a QALY is valued >$150K.
{"title":"Was CED the Right Choice? A Decision-Theoretic Evaluation of the CMS Cover with Evidence Development Policy for Aducanumab","authors":"Jonah Popp, Eric Jutkowitz, Thomas Trikalinos","doi":"10.1101/2024.02.13.24302771","DOIUrl":"https://doi.org/10.1101/2024.02.13.24302771","url":null,"abstract":"Background: In 2022, the Centers for Medicare & Medicaid Services (CMS) issued its final national coverage policy for aducanumab, a novel FDA-approved treatment for Alzheimers disease, deciding to Cover with Evidence Development (CED). CMS will thus only pay for the treatment of AD patients enrolled in an approved randomized controlled trial (RCT). We sought to understand whether, given current evidence, CED was best from a societal perspective.\u0000Methods: We conducted a modeling-based expected value of sample information analysis to estimate the expected net decision-theoretic value of a further RCT to evaluate the clinical efficacy of high-dose (10 mg/kg) aducanumab and to determine what sized trial, if any, is optimal conditional on an initial decision to cover or not. We also evaluated the expected net benefit of the manufacturers proposed RCT (ENVISION). We considered two post-trial decision criteria: cost-effectiveness given updated evidence (efficiency) and does the new trial demonstrate a statistical significant (p<0.05) clinical benefit. Results were used to calculate the expected population net monetary benefit (NMB) of four decision alternatives (including CED) depending on an initial coverage and trial decision. We ranked alternatives and calculated the expected opportunity loss of a suboptimal decision. We used a societal perspective and focused on willingness-to-pay (WTP) values for a quality-adjusted life year (QALY) between $50K-$200K. We conducted scenario analyses using different assumptions about population size, efficacy, and drug cost. Findings: The CMS decision to not cover aducanumab avoids an expected societal loss (NMB) of $15B-$110B. Even an optimally designed RCT would confer no or negative decision-theoretic value for WTP>$100K or with statistical significance as a post-trial decision criterion, respectively, and thus denying coverage without a trial (rather than CED) is clearly preferable. For WTP=$150K (WTP=$200K) and assuming an efficiency criterion, CED with ENVISION or a similar trial is reasonable (decidedly optimal). The case for future research would become less ambiguous if the manufacturer again voluntarily dropped the price >50%.\u0000Interpretation: The societal net value of a future trial (and thus CED) depends on how CMS would use the trial results to update its coverage decision and the WTP per QALY. Assuming CMS policymakers can avoid the pitfalls of a legal framework that limits their ability to consider costs in coverage decisions, the CED decision is at least reasonable, if not optimal, if a QALY is valued >$150K.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139771117","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-02-13DOI: 10.1101/2024.02.11.24302654
Christian Meagher, Didu Kariyawasam, Joanne Scarfe, Michelle Farrar, Kaustav Bhattcharya, Stacy Carter, Ainsley Newson, Margaret Otlowski, Jo Watson, Nicole Millis, Sarah Norris
Background Newborn bloodspot screening is a well-established population health initiative that detects serious, childhood-onset, treatable conditions to improve health outcomes. With genomic technologies advancing rapidly, many countries are actively discussing the introduction of genomic assays into newborn screening programs. While adding genomic testing to Australia’s newborn screening program could improve outcomes for infants and families, it must be considered against potential harms, ethical, legal, equity and social implications, and economic and health system impacts. We must ask not only ‘can’ we use genomics to screen newborns?’ but ‘should we’?’ and ‘how much should health systems invest in genomic newborn screening?’. Methods This study will use qualitative methods to explore understanding, priorities, concerns and expectations of genomic newborn screening among parents/carers, health professionals/scientists, and health policy makers across Australia. In-depth, semi-structured interviews will be held with 30-40 parents/carers recruited via hospital and community settings, 15-20 health professionals/scientists, and 10-15 health policy makers. Data will be analysed using inductive content analysis. The Sydney Children’s Hospital Network Human Research Ethics Committee approved this study protocol [2023/ETH02371]. The Standards for Reporting Qualitative Research will guide study planning, conduct and reporting. Discussion Few studies have engaged a diverse range of stakeholders to explore the implications of genomics in newborn screening in a culturally and genetically diverse population, nor in a health system underpinned by universal health care. As the first study within a multi-part research program, findings will be used to generate new knowledge on the risks and benefits and importance of ethical, legal, social and equity implications of genomic newborn screening from the perspective of key stakeholders. As such it will be the foundation on which child and family centered criteria can be developed to inform health technology assessments and drive efficient and effective policy decision-making on the implementation of genomics in newborn screening.
{"title":"‘Integrating Ethics and Equity with Economics and Effectiveness for newborn screening in the genomic age: a qualitative study protocol of stakeholder perspectives.","authors":"Christian Meagher, Didu Kariyawasam, Joanne Scarfe, Michelle Farrar, Kaustav Bhattcharya, Stacy Carter, Ainsley Newson, Margaret Otlowski, Jo Watson, Nicole Millis, Sarah Norris","doi":"10.1101/2024.02.11.24302654","DOIUrl":"https://doi.org/10.1101/2024.02.11.24302654","url":null,"abstract":"Background Newborn bloodspot screening is a well-established population health initiative that detects serious, childhood-onset, treatable conditions to improve health outcomes. With genomic technologies advancing rapidly, many countries are actively discussing the introduction of genomic assays into newborn screening programs. While adding genomic testing to Australia’s newborn screening program could improve outcomes for infants and families, it must be considered against potential harms, ethical, legal, equity and social implications, and economic and health system impacts. We must ask not only ‘can’ we use genomics to screen newborns?’ but ‘should we’?’ and ‘how much should health systems invest in genomic newborn screening?’. Methods This study will use qualitative methods to explore understanding, priorities, concerns and expectations of genomic newborn screening among parents/carers, health professionals/scientists, and health policy makers across Australia. In-depth, semi-structured interviews will be held with 30-40 parents/carers recruited via hospital and community settings, 15-20 health professionals/scientists, and 10-15 health policy makers. Data will be analysed using inductive content analysis. The Sydney Children’s Hospital Network Human Research Ethics Committee approved this study protocol [2023/ETH02371]. The Standards for Reporting Qualitative Research will guide study planning, conduct and reporting. Discussion Few studies have engaged a diverse range of stakeholders to explore the implications of genomics in newborn screening in a culturally and genetically diverse population, nor in a health system underpinned by universal health care. As the first study within a multi-part research program, findings will be used to generate new knowledge on the risks and benefits and importance of ethical, legal, social and equity implications of genomic newborn screening from the perspective of key stakeholders. As such it will be the foundation on which child and family centered criteria can be developed to inform health technology assessments and drive efficient and effective policy decision-making on the implementation of genomics in newborn screening.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139771121","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Objective: To develop a machine learning (ML) risk stratification model for predicting all-cause mortality and cardiovascular mortality while estimating the influence of lifestyle behavioral factors on the model's efficacy. Method: A prospective cohort study was conducted using a nationally representative sample of adults aged 40 years or older, drawn from the US National Health and Nutrition Examination Survey from 2007 to 2010. The participants underwent a comprehensive in-person interview and medical laboratory examinations, and subsequently, their records were linked with the National Death Index for further analysis. Result: Within a cohort comprising 7921 participants, spanning an average follow-up duration of 9.75 years, a total of 1911 deaths, including 585 cardiovascular-related deaths, were recorded. The model predicted mortality with an area under the receiver operating characteristic curve (AUC) of 0.848 and 0.829. Stratifying participants into distinct risk groups based on ML scores proved effective. All lifestyle behaviors exhibited an inverse association with all-cause and cardiovascular mortality. As age increases, the discernible impacts of dietary scores and sedentary time become increasingly apparent, whereas an opposite trend was observed for physical activity. Conclusion: We develop a ML model based on lifestyle behaviors to predict all-cause and cardiovascular mortality. The developed model offers valuable insights for the assessment of individual lifestyle-related risks. It applies to individuals, healthcare professionals, and policymakers to make informed decisions.
{"title":"The association of lifestyle with cardiovascular and all-cause mortality based on machine learning: A Prospective Study from the NHANES","authors":"Xinghong Guo, Jian Wu, Mingze Ma, Clifford Silver Tarimo, Yifei Feng, Lipei Zhao, BeiZhu Ye","doi":"10.1101/2024.02.07.24302473","DOIUrl":"https://doi.org/10.1101/2024.02.07.24302473","url":null,"abstract":"Objective: To develop a machine learning (ML) risk stratification model for predicting all-cause mortality and cardiovascular mortality while estimating the influence of lifestyle behavioral factors on the model's efficacy. Method: A prospective cohort study was conducted using a nationally representative sample of adults aged 40 years or older, drawn from the US National Health and Nutrition Examination Survey from 2007 to 2010. The participants underwent a comprehensive in-person interview and medical laboratory examinations, and subsequently, their records were linked with the National Death Index for further analysis. Result: Within a cohort comprising 7921 participants, spanning an average follow-up duration of 9.75 years, a total of 1911 deaths, including 585 cardiovascular-related deaths, were recorded. The model predicted mortality with an area under the receiver operating characteristic curve (AUC) of 0.848 and 0.829. Stratifying participants into distinct risk groups based on ML scores proved effective. All lifestyle behaviors exhibited an inverse association with all-cause and cardiovascular mortality. As age increases, the discernible impacts of dietary scores and sedentary time become increasingly apparent, whereas an opposite trend was observed for physical activity. Conclusion: We develop a ML model based on lifestyle behaviors to predict all-cause and cardiovascular mortality. The developed model offers valuable insights for the assessment of individual lifestyle-related risks. It applies to individuals, healthcare professionals, and policymakers to make informed decisions.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139771364","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-02-07DOI: 10.1101/2024.02.01.24302169
Maria M Martignoni, Julien Arino, Amy M Hurford
Public health responses to the COVID-19 pandemic varied across the world. Some countries (e.g., mainland China, New Zealand, and Taiwan) implemented elimination strategies involving strict travel measures and periods of rigorous non-pharmaceutical interventions (NPIs) in the community, aiming to achieve periods with no disease spread; while others (e.g., many European countries and the United States of America) implemented mitigation strategies involving less strict NPIs for prolonged periods, aiming to limit community spread. Travel measures and community NPIs have high economic and social costs, and there is a need for guidelines that evaluate the appropriateness of an elimination or mitigation strategy in regional contexts. To guide decisions, we identify key criteria and provide indicators and visualizations to help answer each question. Considerations include determining whether disease elimination is: (1) necessary to ensure health care provision; (2) feasible from an epidemiological point of view; and (3) cost effective when considering, in particular, the economic costs of travel measures and treating infections. We discuss our recommendations by considering the regional and economic variability of Canadian provinces and territories, and the epidemiological characteristics of different SARS-CoV-2 variants.
{"title":"Is SARS-CoV-2 elimination or mitigation best? Regional and disease characteristics determine the recommended strategy","authors":"Maria M Martignoni, Julien Arino, Amy M Hurford","doi":"10.1101/2024.02.01.24302169","DOIUrl":"https://doi.org/10.1101/2024.02.01.24302169","url":null,"abstract":"Public health responses to the COVID-19 pandemic varied across the world. Some countries (e.g., mainland China, New Zealand, and Taiwan) implemented elimination strategies involving strict travel measures and periods of rigorous non-pharmaceutical interventions (NPIs) in the community, aiming to achieve periods with no disease spread; while others (e.g., many European countries and the United States of America) implemented mitigation strategies involving less strict NPIs for prolonged periods, aiming to limit community spread. Travel measures and community NPIs have high economic and social costs, and there is a need for guidelines that evaluate the appropriateness of an elimination or mitigation strategy in regional contexts. To guide decisions, we identify key criteria and provide indicators and visualizations to help answer each question. Considerations include determining whether disease elimination is: (1) necessary to ensure health care provision; (2) feasible from an epidemiological point of view; and (3) cost effective when considering, in particular, the economic costs of travel measures and treating infections. We discuss our recommendations by considering the regional and economic variability of Canadian provinces and territories, and the epidemiological characteristics of different SARS-CoV-2 variants.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139771118","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-26DOI: 10.1101/2024.01.26.24301710
Bella M. González-Ponce, José Carmona-Márquez, Angelina Pilatti, Carmen Díaz-Batanero, Fermín Fernández-Calderón
Aims This study aimed to prospectively examine the explanatory value of the Protection Motivation Theory (PMT) for the intention to use MD PBS, and to explore its invariance across genders
{"title":"The Protection Motivation Theory as an explanatory model for Intention to use Alcohol Protective Behavioral Strategies among Young Adults","authors":"Bella M. González-Ponce, José Carmona-Márquez, Angelina Pilatti, Carmen Díaz-Batanero, Fermín Fernández-Calderón","doi":"10.1101/2024.01.26.24301710","DOIUrl":"https://doi.org/10.1101/2024.01.26.24301710","url":null,"abstract":"<strong>Aims</strong> This study aimed to prospectively examine the explanatory value of the Protection Motivation Theory (PMT) for the intention to use MD PBS, and to explore its invariance across genders","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-01-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139588640","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-18DOI: 10.1101/2024.01.18.24301468
Greg M. Hammond, Antonia Needham-Taylor, Nathan Bromham, Elizabeth Gillen, Lydia Searchfield, Ruth Lewis, Alison Cooper, Adrian Edwards, Rhiannon Tudor Edwards, Jacob Davies
The general public are advised to have regular routine eye examinations to check their vision and ocular health; however current UK guidance on how often to have eye examinations is not evidence-based and was issued in 2002.This Rapid Review aims to provide an evidence base that stakeholders can use to form updated guidance for Wales by asking the question ‘What are the prognostic factors for a change in ocular status in the general population attending routine eye examinations?’
{"title":"Prognostic factors for a change in eye health or vision: A rapid review","authors":"Greg M. Hammond, Antonia Needham-Taylor, Nathan Bromham, Elizabeth Gillen, Lydia Searchfield, Ruth Lewis, Alison Cooper, Adrian Edwards, Rhiannon Tudor Edwards, Jacob Davies","doi":"10.1101/2024.01.18.24301468","DOIUrl":"https://doi.org/10.1101/2024.01.18.24301468","url":null,"abstract":"The general public are advised to have regular routine eye examinations to check their vision and ocular health; however current UK guidance on how often to have eye examinations is not evidence-based and was issued in 2002.This Rapid Review aims to provide an evidence base that stakeholders can use to form updated guidance for Wales by asking the question ‘What are the prognostic factors for a change in ocular status in the general population attending routine eye examinations?’","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139516759","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-17DOI: 10.1101/2024.01.16.24301376
Christine Ibilibor, Shannon Armbruster, Rell Parker, Jia-Ray Yu, Andrew Barros
The generalizability of data derived from randomized controlled trials is of paramount importance given their utility in the Food & Drug Administration (FDA) drug approval process. An essential part of this process is the inclusion of reliably reported gender, race and ethnicity data in trials that lead to FDA drug approval. Despite previous mandates by the FDA and Clinicaltrials.gov, gender and race-specific data remains under reported. We reviewed 100 most recently approved FDA medications, and abstracted the clinical trial data from Clinicaltrials.gov that supported their approval. We then compared these FDA approved trials to non-FDA approved trials from the same year and of similar size. We found that 40% of the FDA trials were missing race/ethnicity information, while 24% of these trials did not include gender information. We demonstrate that there remains a significant amount of missing gender and racial/ethnic data in trials that lead to FDA-approved medications.
鉴于随机对照试验数据在食品药品管理局（FDA）药品审批过程中的作用，因此这些数据的通用性至关重要。这一过程中的一个重要环节是在导致 FDA 批准药物的试验中纳入可靠报告的性别、种族和民族数据。尽管 FDA 和 Clinicaltrials.gov 此前已做出规定，但性别和种族特定数据的报告仍然不足。我们回顾了 FDA 最近批准的 100 种药物，并从 Clinicaltrials.gov 中摘录了支持这些药物获得批准的临床试验数据。然后，我们将这些 FDA 批准的试验与同年且规模相似的非 FDA 批准的试验进行了比较。我们发现，40% 的 FDA 试验缺少种族/族裔信息，而其中 24% 的试验未包含性别信息。我们证明，在导致 FDA 批准药物的试验中，仍有大量性别和种族/民族数据缺失。
{"title":"The Dearth of Representation in FDA Approved Drug Trials","authors":"Christine Ibilibor, Shannon Armbruster, Rell Parker, Jia-Ray Yu, Andrew Barros","doi":"10.1101/2024.01.16.24301376","DOIUrl":"https://doi.org/10.1101/2024.01.16.24301376","url":null,"abstract":"The generalizability of data derived from randomized controlled trials is of paramount importance given their utility in the Food & Drug Administration (FDA) drug approval process. An essential part of this process is the inclusion of reliably reported gender, race and ethnicity data in trials that lead to FDA drug approval. Despite previous mandates by the FDA and Clinicaltrials.gov, gender and race-specific data remains under reported. We reviewed 100 most recently approved FDA medications, and abstracted the clinical trial data from Clinicaltrials.gov that supported their approval. We then compared these FDA approved trials to non-FDA approved trials from the same year and of similar size. We found that 40% of the FDA trials were missing race/ethnicity information, while 24% of these trials did not include gender information. We demonstrate that there remains a significant amount of missing gender and racial/ethnic data in trials that lead to FDA-approved medications.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139506774","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-17DOI: 10.1101/2024.01.17.23300197
Rhiannon Tudor Edwards, Llinos Haf Spencer, Bethany Fern Anthony, Jacob Davies, Kalpa Pisavadia, Abraham Makanjuola, Huw Lloyd-Williams, Deborah Fitzsimmons, Brendan Collins, Joanna Mary Charles, Ruth Lewis, Alison Cooper, Sezen Barutcu, Mary-Ann McKibben, Adrian Edwards
Rapid review methodology was used to identify updated economic evidence on programmes or interventions designed to enable employees to remain in and return to the workforce. In Wales, there are currently 1.48 million people in employment and 58,300 people who are unemployed. This equates to an unemployment rate of 3.8% in Wales.
{"title":"Wellness in work - supporting people in work and assisting people to return to the workforce: An economic evidence review","authors":"Rhiannon Tudor Edwards, Llinos Haf Spencer, Bethany Fern Anthony, Jacob Davies, Kalpa Pisavadia, Abraham Makanjuola, Huw Lloyd-Williams, Deborah Fitzsimmons, Brendan Collins, Joanna Mary Charles, Ruth Lewis, Alison Cooper, Sezen Barutcu, Mary-Ann McKibben, Adrian Edwards","doi":"10.1101/2024.01.17.23300197","DOIUrl":"https://doi.org/10.1101/2024.01.17.23300197","url":null,"abstract":"Rapid review methodology was used to identify updated economic evidence on programmes or interventions designed to enable employees to remain in and return to the workforce. In Wales, there are currently 1.48 million people in employment and 58,300 people who are unemployed. This equates to an unemployment rate of 3.8% in Wales.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139506771","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-17DOI: 10.1101/2023.12.15.23300041
Zecharias Fetene Anteneh, Anagaw D. Mebratie, Zemzem Shigute, Getnet Alemu, Arjun S. Bedi
There has been a growing concern about the financial sustainability of community-based health insurance (CBHI) schemes in developing countries recently. However, little empirical studies have been conducted to identify potential contributors including ex-ante and ex-post moral hazards. We respond to this concern by investigating the effects of being insured on household lifestyle -Preventive Care- and the timing of treatment seeking behavior in the context of Ethiopia’s CBHI scheme. Using three rounds of household panel data and a fixed-effects household model, we do not find a significant impact on preventive care activities. However, we find that participation in CBHI increases delay in treatment-seeking behavior for malaria, tetanus, and tuberculosis symptoms. This behavior is costly for the insurer. Therefore, it is essential to identify the primary causes of delays in seeking medical services and implement appropriate interventions aimed at encouraging insured individuals to seek early medical attention when signs of diseases emerge.
{"title":"Does community-based health insurance affect lifestyle and timing of treatment seeking behavior? Evidence from Ethiopia","authors":"Zecharias Fetene Anteneh, Anagaw D. Mebratie, Zemzem Shigute, Getnet Alemu, Arjun S. Bedi","doi":"10.1101/2023.12.15.23300041","DOIUrl":"https://doi.org/10.1101/2023.12.15.23300041","url":null,"abstract":"There has been a growing concern about the financial sustainability of community-based health insurance (CBHI) schemes in developing countries recently. However, little empirical studies have been conducted to identify potential contributors including ex-ante and ex-post moral hazards. We respond to this concern by investigating the effects of being insured on household lifestyle -Preventive Care- and the timing of treatment seeking behavior in the context of Ethiopia’s CBHI scheme. Using three rounds of household panel data and a fixed-effects household model, we do not find a significant impact on preventive care activities. However, we find that participation in CBHI increases delay in treatment-seeking behavior for malaria, tetanus, and tuberculosis symptoms. This behavior is costly for the insurer. Therefore, it is essential to identify the primary causes of delays in seeking medical services and implement appropriate interventions aimed at encouraging insured individuals to seek early medical attention when signs of diseases emerge.","PeriodicalId":501386,"journal":{"name":"medRxiv - Health Policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138818957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
京公网安备 11010802042870号
京ICP备2023020795号-1
扫码关注我们
求助内容：
应助结果提醒方式：