Therapeutic innovation & regulatory science最新文献

{"title":"Impact of Rule 11 on the European Medical Software Landscape: Analysis of EUDAMED and Further Databases Three Years After MDR Implementation.","authors":"Arndt A Schmitz, Miriam Font-Nieves, Toumani Doucouré, Hans-Peter Podhaisky","doi":"10.1007/s43441-025-00747-5","DOIUrl":"10.1007/s43441-025-00747-5","url":null,"abstract":"<p><p>Medicine is increasingly supported by software, with digital health technologies offering innovative ways to capture insights and drive therapies. Globally, medical device software must follow regulatory processes based on risk classification. The introduction of MDR represents a significant shift in risk-based classification for Medical Devices in Europe, including classification Rule 11 for software, which has caused significant discussions among European regulators. Three years after implementation, we conducted a systematic impact assessment of MDR classification Rule 11 for MDSW through a qualitative and quantitative analysis of over 2000 software entries from the European Medical Device database, complemented by data from other public databases such as the German DiGA directory and mHealthBELGIUM. Our results indicate that classification Rule 11 of the MDR results in a narrow bandwidth for class I software, whereas this used to be the most frequent classification for software under the MDD: while most of legacy software in EUDAMED falls in the lowest risk category as MDD Class I (53%), the situation reverses after the implementation of MDR with the most entries in Class IIa (55%). Analyzing the legacy MDD patient apps in Germany implies that three quarters will have to re-classify as MDR Class IIa at the end of the transition period in 2028. A comparison of the European and US regulatory landscapes, along with a systematic review of software features for Class I vs. Class IIa products, explains our findings and enables us to recommend a regulatory strategy for developing MDSW compliant with MDR Class I rules, ensuring fast access to the European market.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"365-378"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143060821","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Risk-Based Quality Management: A Case for Centralized Monitoring.","authors":"Nicole Stansbury, Danilo Branco, Cris McDavid, Jennifer Stewart, Kristin Surdam, Nycole Olson, Joanne Perry, Jeremy Liska, Linda Phillips, Amanda Coogan, Anina Adelfio, Lauren Garson","doi":"10.1007/s43441-024-00719-1","DOIUrl":"10.1007/s43441-024-00719-1","url":null,"abstract":"<p><p>Since 2019, the Association of Clinical Research Organizations has conducted a landscape survey of risk based quality management (RBQM) adoption in clinical trials. Here, we present data from four years of surveys, with an emphasis on the most recent: the 2022 survey included data from 4958 trials across seven contract research organizations, of which 1004 were new studies started in 2022. Results indicate that while overall risk assessment adoption is strong, it is lagging in other risk-based components which suggests companies are not deriving the full expected benefits of performing a risk assessment and mitigation process to their trials. The 2022 study also suggests new study starts showing promising traction, with adoption hovering near 50% for most RBQM elements. At the same time, the survey suggests industry has mixed views on the potential value of quality tolerance limits (QTLs). Ultimately, centralized monitoring is being underutilized despite the potential of increased patient safety oversight and improved data quality. The authors of this paper developed a case study based on a trial in clinicaltrials.gov to demonstrate how RBQM adoption could include the key RBQM elements such as centralized monitoring, reduced source data review and source data verification as well as implementation of QTLs in a real-world scenario. The authors believe the clinical trial industry has an obligation to utilize centralized monitoring to produce more efficient and effective clinical trials and will make a case to do so in this paper.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"199-210"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142814222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction: Consumer Understanding of Prescription Drug Indications in Direct-to-Consumer Television Advertisements.","authors":"Helen W Sullivan, Kathryn J Aikin, Mihaela Johnson, Kate Ferriola-Bruckenstein","doi":"10.1007/s43441-024-00738-y","DOIUrl":"10.1007/s43441-024-00738-y","url":null,"abstract":"","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"398"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955520","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Interim Clinical Trial Data: Who Can See What, and When?","authors":"Susan S Ellenberg, Yimei Li","doi":"10.1007/s43441-024-00728-0","DOIUrl":"10.1007/s43441-024-00728-0","url":null,"abstract":"<p><p>It has long been a basic principle of randomized clinical trials addressing serious outcomes and/or major public health issues that interim data should be inaccessible to investigators and to industry sponsors, with interim data reviewed on a regular basis by an independent data monitoring committee (DMC). Challenges to this principle may arise when sponsors and/or regulators perceive a need to review interim data while the trial remains ongoing-for example, when a trial is being considered for accelerated approval. In this paper we propose approaches that could minimize the extent of interim data that is made available to others while the trial continues.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"211-214"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Ethics of the \"Right-to-Try\" Movement in an Era of Regulatory Flux.","authors":"Neil Jain, David Ralston, Cheryl Erwin","doi":"10.1007/s43441-025-00758-2","DOIUrl":"10.1007/s43441-025-00758-2","url":null,"abstract":"<p><p>The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 signed on May 30, 2018 under President Trump has been championed by patient advocacy groups as a victory for individuals with life threatening illnesses willing to undergo experimental treatment. The act is not a novel idea, but rather a nuanced result of the previous attempts to challenge the US Food and Drug Administration's (FDA) authority on drug approval and distribution. Currently, right-to-try programs coexist with an already existing expanded access program run by the FDA. The difference is that right-to-try requests eliminate FDA guidance and authorization. The objectives of this study are to review prior historical challenges to the FDA and how they eventually influenced right-to-try movements, examine the law itself and its arguments written by advocates and critics, discuss how its implications fit into the current climate of regulatory flux, and propose the impacts it has on influencing patient care and the scientific process.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"256-263"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256735","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Five-Year Analysis of Market Share and Sales Growth for Original Drugs after Patent Expiration in Korea.","authors":"Minyoung Bae, Sung Ryul Shim, Dong-Wook Yang, Kyung-Bok Son, Sang-Won Lee","doi":"10.1007/s43441-025-00741-x","DOIUrl":"10.1007/s43441-025-00741-x","url":null,"abstract":"<p><strong>Introduction: </strong>The sales patterns of original drugs after patent expiration in Korea show a relatively high market share and continuous sales growth differently from those in the U.S. and European countries. This study aims to investigates a five-year sales pattern of original drugs after patent expiration in Korea using empirical data.</p><p><strong>Methods: </strong>Using data from the Ministry of Food and Drug Safety, original drugs whose patents expired in 2012-2018 were extracted. And we used IQVIA data to determine the market share and sales growth rate of 48 original drugs, whose generic drug launched for the first time in the same molecule market, and whose sales data over five years after first generic entry were available. We analyzed the differences by the attribute of variables.</p><p><strong>Results: </strong>The sales volume of original drugs in the fifth year (Q 20) had an average growth rate of 150.6% compared with that before the first generic drug launched, indicating a continuous growth. The average market share of original drugs in the fifth year (Q 20) decreased to 70.6%, but it was higher than previously reported research results in Korea and other countries. Differences were observed across the category of attribute.</p><p><strong>Conclusion: </strong>This study demonstrated that while market share of original drugs is decreasing, the sales volume increased continuously until the fifth year, differently from those of other countries. Variations in sales patterns by attributes reflect unique dynamics in Korea.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"349-358"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880101/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955501","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessment of Local Pharmaceutical Manufacturing Sector in a Low-income Country: A Descriptive Study.","authors":"Tesfa Marew, Anteneh Belete, Frances J Richmond, Tsige Gebre-Mariam","doi":"10.1007/s43441-025-00756-4","DOIUrl":"10.1007/s43441-025-00756-4","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;In high-income countries and emerging economies, pharmaceutical manufacturing is a driver for socioeconomic development. In sub-Saharan Africa, local pharmaceutical production is still fledgling largely because of historical economic and technological asymmetry. In Ethiopia alike, this environment has changed little despite six decades long operations and several support initiatives; access to essential medicines remained a persistent challenge. Despite a few fragmented reports on trends and profiles of the sector, in-depth investigations into underlying challenges remain limited. This study explores the perspectives and insights of upfront executives and technical personnel in the Ethiopian pharmaceutical industry on its current state, challenges and opportunities for enhancing local production capacity. The findings are triangulated with literature reports and provide valuable insights for developing intervention strategies and policy updates.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A validated structured survey questionnaire was disseminated to professionals working in local pharmaceutical companies. Multiple regression analysis was conducted to study the effects of different factors on the performance of the local manufacturing sector.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Over half of the professionals (55.1%) rated the current production technology as advanced while 68% described it as semi-automated. Close to one-third (31.6%) reported that the companies are operating at acceptable level of performance, and 51.5% reported low-capacity utilization in their companies. Most professionals (67.8%) viewed export market activities as poor and many identified weak research and development activities. Unplanned operations downtimes, limited physical infrastructure, inadequate access to foreign currency, shortage and highstaff turnover of qualified experts, lack of commitment from top management, absence of merit-based support system and limitations in compliance with regulatory and quality requirements were reported as major challenges. Multiple linear regression analysis demonstrated that capacity utilization (α = 0.008), research and development capacity (α = 0.014) and export market activities (α = 0.027) have significant impact on the sector performance.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;Despite commendable efforts by the industry and government, limitations in financing, infrastructure, qualified workforce, and quality system implementation are affecting capacity utilization and performance. Absence of reliable staff attraction and retention system, lack of effective leadership and non-favourable working environment were identified as missing elements. Enhancing economies of scale and capacity utilization, addressing policy and infrastructure bottlenecks, providing merit-based support for R&D and quality management, and implementing staff attraction and retention strategies are key steps towards developing competent local ph","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"379-396"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143371220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Consumer Understanding of Prescription Drug Indications in Direct-to-Consumer Television Advertisements.","authors":"Helen W Sullivan, Kathryn J Aikin, Mihaela Johnson, Kate Ferriola-Bruckenstein","doi":"10.1007/s43441-024-00732-4","DOIUrl":"10.1007/s43441-024-00732-4","url":null,"abstract":"<p><strong>Background: </strong>Prescription drugs may be indicated to treat more than one medical condition, and companies may promote more than one indication in the same direct-to-consumer (DTC) ad. This study examined how presenting multiple prescription drug indications in one DTC television ad affects consumers' processing of drug information.</p><p><strong>Methods: </strong>We conducted two studies with adults diagnosed with diabetes (Study 1, N = 408) or rheumatoid arthritis (Study 2, N = 411). We randomly assigned participants to view one of three television ads: primary indication only (Study 1: diabetic peripheral neuropathy; Study 2: rheumatoid arthritis), primary plus a similar secondary indication (Study 1: fibromyalgia; Study 2: psoriatic arthritis), or primary plus a dissimilar secondary indication (Study 1: generalized anxiety disorder; Study 2: ulcerative colitis).</p><p><strong>Results: </strong>Remembering and understanding the primary indication was not significantly affected by the presence of a secondary indication (similar or dissimilar). Higher health literacy participants remembered and understood secondary indications.</p><p><strong>Conclusions: </strong>Including a second indication in DTC television ads does not appear to have detrimental effects and can increase awareness of the second indication for some participants. Industry and regulators should continue to ensure DTC promotion is truthful and non-misleading, irrespective of the number of indications presented.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"278-287"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11879756/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865540","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global Harmonization of Biosimilar Development by Overcoming Existing Differences in Regional Regulatory Requirements - Outcomes of a Descriptive Review.","authors":"Thomas M Kirchlechner, Hillel P Cohen","doi":"10.1007/s43441-024-00740-4","DOIUrl":"10.1007/s43441-024-00740-4","url":null,"abstract":"<p><p>Global harmonization of biosimilar developmental requirements will facilitate development leading to increased patient and societal benefits. However, there are several technical and regulatory hurdles that must be addressed to harmonize the regulatory requirements in different countries and regions. At times, there is a requirement for use of locally sourced reference product, forcing biosimilar developers to repeat analytical or clinical comparability studies against reference product batches sourced from within a given country. While most health authorities no longer require comparative animal toxicology studies of the proposed biosimilar and reference product, these are still required in several countries, forcing biosimilar companies to conduct such studies or risk non-approval of their product. At times, different health authorities request different clinical study designs. In some jurisdictions there is a requirement to generate clinical data in local ethnic populations. Some health authorities require a hybrid label that combines clinical data from the reference biologic and the biosimilar, in the patient leaflet. Recommendations are provided to address each of these hurdles to facilitate global regulatory harmonization of biosimilars. Overcoming these barriers will ultimately increase patient access to these medicines in all regions while providing financial relief to healthcare systems.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"245-255"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880096/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143011664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Data Monitoring Committee Reports: Telling the Data's Story.","authors":"Lijuan Zeng, Toshimitsu Hamasaki, Scott R Evans","doi":"10.1007/s43441-024-00727-1","DOIUrl":"10.1007/s43441-024-00727-1","url":null,"abstract":"<p><p>A Data Monitoring Committee (DMC) plays a pivotal role in monitoring participant safety and efficacy and overseeing the integrity of clinical trials. DMCs accomplish this mission by periodically reviewing accumulating data to assess benefits and harms of interventions in ongoing studies and making subsequent recommendations regarding future clinical trial conduct to the trial sponsor. Reports summarizing data from the clinical trial are prepared for the DMC by statistical and data analysis centers to inform DMC decision-making. In practice however, these reports are often disorganized, complex, and provide overwhelming detail yet insufficient information, that hinders accurate and efficient interpretation of interim data. This review paper delves into the nuances of preparing effective DMC reports, highlighting the importance of simplicity, clarity, and thoughtful relevance in data presentation. We discuss structured approaches for preparing closed reports, which deal with sensitive and sometimes messy interim data, and underscore the use of visual summaries and narrative elements that enhance comprehension and facilitate efficient assessments of trial data. The paper outlines key principles for preparing DMC reports and provides practical guidance on their structure. Ultimately, this guidance seeks to ensure that the data's story is clearly and efficiently conveyed to facilitate the DMC decision-making process.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"222-233"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142802263","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}