Therapeutic innovation & regulatory science最新文献

{"title":"Toward an Extensible Regulatory Framework for N-of-1 to N-of-Few Personalized RNA Therapy Design.","authors":"Mélissa Bou-Jaoudeh, Gabriele Piaton-Breda, Florian Pereme, Stephen Gilbert","doi":"10.1007/s43441-025-00752-8","DOIUrl":"https://doi.org/10.1007/s43441-025-00752-8","url":null,"abstract":"<p><p>The emergence of personalized RNA therapeutics, tailored to individual patients' genetic profiles, offers new hope for treating both common and rare diseases. This review explores regulatory aspects of N-of-1 and N-of-few approaches, providing promising treatments for ultra- or nano-rare diseases that lack established therapies. These diseases present unique challenges, as patients may represent the sole individual or a small group worldwide with a specific mutation, necessitating personalized approaches to treatment development, validation, and approval. While progress is promising, the regulatory landscape remains nascent, raising challenges in ensuring safety and industry sustainability. Artificial intelligence (AI) and automated systems, coupled with real-world evidence (RWE) monitoring, offer significant potential to address these challenges by optimizing development, manufacturing, and regulatory compliance. Drawing parallels from other regulatory domains, this review presents a design envelope framework, integrated with AI tools, to streamline the approval process and enhance the adaptability of RNA-based treatments. Case studies of individualized RNA-based treatments highlight successes and setbacks, underscoring the need for regulatory alignment. Collaborative efforts from stakeholders and regulatory authorities are essential to refine this framework for real-world application. Overall, this review emphasizes the transformative potential of personalized RNA therapeutics in advancing precision medicine.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143516784","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":"Promoting Diversity through an Understanding of Barriers and Drivers for Inclusive Clinical Trials.","authors":"Annick de Bruin, Jasmine Masullo, Shalome Sine, Kenneth Getz","doi":"10.1007/s43441-025-00751-9","DOIUrl":"https://doi.org/10.1007/s43441-025-00751-9","url":null,"abstract":"<p><strong>Importance: </strong>Racially and ethnically diverse, equitable representation among clinical trial participants is important for enhancing the drug development process and promoting equitable healthcare outcomes.</p><p><strong>Objective: </strong>To understand the barriers and drivers for inclusive clinical trials, focusing on the attitudes, perceptions, experiences, and challenges faced by underrepresented populations.</p><p><strong>Design: </strong>An online questionnaire was administered online from April to June 2023 and involved 12,017 respondents from 54 countries. This survey utilized a convenience sampling strategy. Statistical analysis was performed to compare responses among racial and ethnic groups.</p><p><strong>Setting: </strong>The study was conducted globally. Survey respondents were recruited through various patient recruitment organizations, patient advocacy groups, and contract research organizations.</p><p><strong>Respondents: </strong>Adults 18 years or older who received an email or had online access were eligible to participate. Racial and ethnic composition included White (81%), Hispanic/Latino (15%), Black/African American (6%), Asian (6%), and other ethnicities.</p><p><strong>Exposure(s): </strong>Respondents were asked about their perceptions, concerns and experiences related to clinical research access and participation.</p><p><strong>Main outcome(s) and measure(s): </strong>Key outcomes included barriers to clinical research participation, factors influencing trust in pharmaceutical companies and past experiences.</p><p><strong>Results: </strong>Barriers to clinical research participation varied among ethnic groups. Asian respondents cited concerns about time off work (22%) and time required to participate (19%) more frequently as compared to White respondents (7% and 7%, respectively; p < 0.05). Hispanics expressed higher concerns about time off work (15%) and receiving placebo (10%) as compared to Non-Hispanics (8% and 5%, respectively, p < 0.05). Black and Hispanic respondents placed higher importance on diversity in staff compared to White and non-Hispanic respondents (B: 32%; W: 12%; Hispanic: 22%; Non-Hispanic: 13% p < 0.05). Black, Asian, and Hispanic respondents reported higher levels of disruption in participation related to technology use (Black: 31%; Hispanic: 30%; Asian: 29%) and completing study requirements at home (Black: 32%; Hispanic: 30%; Asian: 26%) as compared to White (13%, 15%; p < 0.05%) and non-Hispanic respondents (14%, 17%; p < 0.05).</p><p><strong>Conclusions: </strong>The findings highlight the need to address barriers to diversity in clinical trials and improve trial experiences of underrepresented communities, facilitating design of more inclusive and patient-centered trials.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143504340","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":"Designing a Patient Preference Study on Subcutaneous Medical Devices: Incorporating Health Authority Scientific Advice and Patient Perspectives.","authors":"Marie Picci, Nigel S Cook, Byron Jones, Mo Zhou, Conny Berlin, Christine Sturchler, Clemence Martinez, Irene Garcia Baena, Lauren Ziegler, Harriet Gaunt, Brad Mason, Dominique Hamerlijnck, Yoshiyuki Majima","doi":"10.1007/s43441-024-00725-3","DOIUrl":"https://doi.org/10.1007/s43441-024-00725-3","url":null,"abstract":"<p><p>This paper describes the planning of a patient preference study for evaluating device features for the subcutaneous (SC) delivery of high dose/large volume (e.g., > 2 ml) of drugs. Multiple sources, including qualitative patient interviews, the involvement of patient partners, and solicitation of advice from the US Food and Drugs Administration (FDA), were used to refine the attributes and levels in the development of a preference study protocol to investigate what Multiple Sclerosis (MS) patients consider important regarding medical device features for high dose SC administration.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143516840","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 Data Monitoring Experience in Empagliflozin Randomized Clinical Trials Between 2011 and 2024.","authors":"Francine K Welty, Klaus G Parhofer, Marvin Konstam, Michael K Palmer, Barry Greenberg, Ralph Daher, Tim Clayton","doi":"10.1007/s43441-025-00749-3","DOIUrl":"https://doi.org/10.1007/s43441-025-00749-3","url":null,"abstract":"<p><p>In November 2007, a black box warning was mandated for rosiglitazone in type 2 diabetes mellitus (T2DM) based on an increased risk of ischemic cardiovascular (CV) events. The Food and Drug Administration (FDA) issued a directive that a CV outcomes trial must be done for any new diabetes drug to demonstrate no CV harm. Therefore, the Empagliflozin Cardiovascular Outcome Event Trial in Type 2 Diabetes Mellitus Patients (EMPA-REG OUTCOME) trial was started in 2011 alongside 13 additional randomized clinical trials (RCTs) of empagliflozin in T2DM. The results of EMPA-REG OUTCOME set the stage for later RCTs in heart failure. Results from these clinical trials have changed the outlook for patients both with and without T2DM and with and without heart failure. A Program Data Monitoring Committee (DMC) with the same core members was utilized for these trials between 2011 and 2024. This committee is likely to be one of the longest serving DMCs since it served 28 trials with empagliflozin between 2011 and 2024. The committee encountered several important challenges which are discussed in this article. Moreover, the committee provides several important take-home messages which we hope will be of value in discussing issues in creating, developing and running DMCs in the future. These include: 1. Whether and when to be blinded and unblinded; 2. How to proceed when the primary endpoint shows no evidence of benefit, but there is evidence for a mortality benefit; 3. Development of presentation of data using figures and boxplots for rapid review of adverse events and laboratory data to assess clinical challenges; 4. How to manage a catastrophic serious adverse event; 5. Suggestions for an ideal structure of the report for the DMC closed session; and 6. The relation between the DMC, sponsor and Contract Research Organization. Our experience emphasizes the value of continuity with the same members serving over a 13-year period.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143516780","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":"Optimizing Quality Tolerance Limits Monitoring in Clinical Trials Through Machine Learning Methods.","authors":"Lei Yan, Ziji Yu, Liwen Wu, Rachael Liu, Jianchang Lin","doi":"10.1007/s43441-025-00754-6","DOIUrl":"https://doi.org/10.1007/s43441-025-00754-6","url":null,"abstract":"<p><p>The traditional clinical trial monitoring process, which relies heavily on site visits and manual review of accumulative patient data reported through Electronic Data Capture system, is time-consuming and resource-intensive. The recently emerged risk-based monitoring (RBM) and quality tolerance limit (QTL) framework offers a more efficient alternative solution to traditional SDV (source data verification) based quality assurance. These frameworks aim at proactively identifying systematic issues that impact patient safety and data integrity. In this paper, we proposed a machine learning enabled approach to facilitate real-time, automated monitoring of clinical trial QTL risk assessment. Unlike the traditional quality assurance process, where QTLs are evaluated based on single-source data and arbitrary defined fixed threshold, we utilize the QTL-ML framework to integrate information from multiple clinical domains to predict the QTL of variety types at clinical program, study, site and patient level. Moreover, our approach is assumption-free, relying not on historical expectations but on dynamically accumulating trial data to predict quality tolerance limit risks in an automated manner. Embedded within ICH-E6 recommended RBM principles, this innovative machine learning solution for QTL monitoring has the potential to transform sponsors' ability to protect patient safety, reduce trial duration, and lower trial costs.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143504338","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":"Sponsor- vs. FDA-Initiated Changes to Pediatric Clinical Trial Protocols: A Comparison of Associated Participant Burden.","authors":"Susan Abdel-Rahman, Zoe Sund, Cheryl Alderman, Karylle Abella, Phyllis Kennel","doi":"10.1007/s43441-025-00760-8","DOIUrl":"https://doi.org/10.1007/s43441-025-00760-8","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Risks associated with clinical trial participation are a foremost consideration during protocol development whereas trial-associated burdens receive less focus despite their measurable impact on enrollment and retention. Of late, the U.S. Food and Drug Administration (FDA) has elevated discussions on barriers to research participation resulting from overly burdensome trials. Given the agency's role in shaping clinical protocol design, this study examined the perceived burden associated with FDA-proposed study changes in the context of pediatric, off-patent, labeling studies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Materials and methods: &lt;/strong&gt;Pediatric Trials Network (PTN) studies conducted between 2013 and 2023 for which there existed a record of formal communication between the PTN and FDA were evaluated. All protocol versions and regulatory communications were reviewed, and every protocol change with the potential to alter participant burden was extracted and attributed to the PTN Sponsor or FDA. Changes were grouped into 11 themes (e.g. change in the number of visits, change in invasive procedures) and each change assigned a perceived burden score on an 11-point Likert scale by pediatric clinical trialists who were blinded to attribution. An abbreviated list of protocol changes were reviewed and scored by children to examine their concordance with adult scores. Quantitative and qualitative differences between changes introduced by Sponsors and the FDA were compared.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Twenty-one studies (94 protocol versions) met the criteria for inclusion (18 drug, 3 device). Half of the protocol versions incorporated changes (n = 123) that could perceivably affect participant burden (77 initiated by the sponsor, 46 proposed by the FDA). Changes classified as introducing, increasing, or extending protocol features occurred almost twice as often (64%) as changes that reduced, removed, or restricted features of the protocol (36%), the latter also occurring later in the life of the protocol (1.2 vs. 2.0 year, p &lt; 0.01). Changes recommended by the FDA were primarily related to ensuring safety (77%), optimizing trial design (16%) and adequately capturing effectiveness (7%) and, on average, were associated with statistically higher burden scores. Modifications driven by sponsors reflected trial design refinements (34%), safety assessment (32%), expansion of primary/secondary scientific questions (22%), and effectiveness evaluation (12%). Burden scores demonstrated strong concordance between trialists and children (r = 0.7).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Half of all protocol amendments are associated with changes that conceivably alter the burden of clinical trial participation, and these changes are twice as likely to add to (vs. reduce) participant burden. Given the time and effort involved with protocol amendments, we suggest that modifications to trial protocols be accompanied by a reassessment of the role and util","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143484012","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":"Points to Consider on the Use of Medicines in Pregnancy Throughout the Product Lifecycle Based on Global Regulatory Guidance.","authors":"Amalia Alexe, Keele Wurst, Leesha Balramsingh-Harry, Olatayo Apara, Nadezda Abramova, Osa Eisele, Maria Fernanda Scantamburlo Fernandes, Anju Garg, Birgit Kovacs, David Lewis","doi":"10.1007/s43441-024-00736-0","DOIUrl":"https://doi.org/10.1007/s43441-024-00736-0","url":null,"abstract":"<p><p>The thalidomide tragedy of the 1960s led to restrictions and limitations in the participation of pregnant women in clinical trials. Despite the paucity of information on the safe and effective use of medicines in this population, most pregnant women are prescribed medications. A landscape assessment review of guidelines and legislation governing the use of medicines in pregnancy and during breastfeeding was conducted by the TransCelerate Pharmacovigilance Pregnancy and Breastfeeding Team. Insights from the landscape assessment review were compiled to identify important points to consider concerning the use of medicines in pregnancy throughout a product lifecycle. Four main areas were identified for consideration for use of medicines in pregnancy: (1) Product development considerations: Key points on the disease itself, the medicine characteristics, non-clinical and clinical development. (2) Interventional study considerations: Key aspects in enrollment of pregnant women in clinical trials and the follow-up requirements for such women. (3) Post-marketing considerations: Key elements in spontaneous case reporting of medicines exposure during pregnancy, implementation of appropriate risk management plans for medicines likely to be used in pregnancy. (4) Full lifecycle considerations: Activities required by regulators to ensure safety surveillance and maintenance throughout product lifecycle. There is a need for harmonized guidance on how to study the use of medicine in pregnancy. This paper addresses regulatory considerations, to aid in the planning and execution of research programs focused on developing medicines for use in pregnancy when permissible under established regulatory framework.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143477037","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":"Catering the Need of Drug Manufacturing Standard in India: An Update to Indian Pharmacopoeia.","authors":"Bikash Kumar Sah, Faiz Ahmad, Ankit Kumar, Sachin Kumar Singh, Rajesh Kumar Sachdeva","doi":"10.1007/s43441-025-00759-1","DOIUrl":"https://doi.org/10.1007/s43441-025-00759-1","url":null,"abstract":"<p><p>India is currently among the top 10 pharmaceutical markets by value and is the third largest by volume in 2024; it manufactures more than 65,000 generic drugs across 60 therapeutic segments. Holding a 20% market share of generics, the industry is a key supplier to Africa and the USA and other markets. Following the Drugs and Cosmetics Act of 1940, for the manufacture of drugs in India, there is always legal supervision on the quality of the products being manufactured in the country. The IP, published every ten years by the IPC, which is an autonomous body of the Department of Pharmaceuticals, Ministry of Chemicals and Fertilizers, Government of India, is the official reference for drug standards in India for the purpose of guaranteeing drug quality and effectiveness. The IPC, set up in 1948 under the Ministry of Health and Family Welfare, monitors the publication and changes of the IP in consultation with other agencies. The IPC consists of four major divisions: Governing, Scientific, General, and Executive, which deal with the policy, scientific and academic standards, performance, organizational, and administrative work, respectively. The Indian Pharmacopoeia Laboratory has also proactively adapted or added to its roles in areas of research, writing monographs, and providing the public with drug standards. The monograph development in the IP undergoes an evaluation by the specialist, public comment, and the need to update and align with the current scientific research and internationally recognized guidelines. There are significant changes on average every 4 to 5 years, with supplements and addenda between revisions. This review article focuses on India's position as the world's largest supplier of generic medicines, the mechanisms of drug safety and quality control and the IPC's current and further endeavours to harmonize Indian standards with those of the global world. It also provides information about the position of India in the global market of pharmaceuticals, the large production and export of the Indian generics and vaccines.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143473074","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":"Adoption Maturity Model for Risk-based Quality Management (RBQM) in Clinical Trials.","authors":"Maria Florez, Abigail Dirks, Linda Sullivan, Steve Young, Kenneth Getz","doi":"10.1007/s43441-025-00746-6","DOIUrl":"https://doi.org/10.1007/s43441-025-00746-6","url":null,"abstract":"<p><strong>Background: </strong>The global drug development regulatory community, through the International Council for Harmonisation (ICH) guidelines for the development of pharmaceuticals for human use, has long encouraged the use of quality by design (QBD) principles in clinical trials. Risk-based Quality Management (RBQM) offers an approach to improve clinical research quality and performance by identifying and mitigating risks related to critical safety and efficacy data based on Quality by Design (QbD) principles. Several studies have been published quantifying current levels of RBQM adoption, but a systematic study mapping the stages of adoption maturity has not been conducted. This lens is needed to inform organizations on adoption paths that have been followed and to facilitate more rapid RBQM adoption considering recent regulatory guidance (ICH E6 R3).</p><p><strong>Methods: </strong>The Tufts Center for the Study of Drug Development conducted a global online survey and collected responses from 119 individual pharmaceutical and biotechnology companies. Responses related to the use of 32 distinct RBQM practices in ongoing clinical trials in late 2022 and early 2023, and were analyzed and used to (1) develop an adoption maturity model, (2) map levels of adoption by company size, and to (3) isolate the characteristics and barriers experienced by five RBQM maturity cohorts: Innovators, Early Adopters, Early Majority, Late Majority, and Resisters.</p><p><strong>Results: </strong>RBQM components are used primarily by larger companies-those conducting more than 25 clinical trials annually. These companies tend to fall in the Innovators, Early Adopters, and Early Majority RBQM maturity cohorts. These companies have implemented nearly all of 32 RBQM components in at least one trial. The most common areas of reported use are in the documentation and resolution of risk areas. The areas with lowest rates of use are the detection of duplicate patients, statistical data monitoring, reduced/targeted SDR, identification of critical to quality factors, and documentation of updates to definitions.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426230","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":"Changes in Clinical Trials of Dermatological Drugs in Mainland China Between 2016 and 2022: A Narrative Review.","authors":"Beibei Zhu, Jing Li, Qi Ni, Shuo Yang, Zheng Yin, Xueyuan Yang","doi":"10.1007/s43441-025-00743-9","DOIUrl":"https://doi.org/10.1007/s43441-025-00743-9","url":null,"abstract":"<p><strong>Introduction: </strong>Policy reforms in drug regulation and reimbursement have encouraged drug research and development in China. However, there is a lack of insights on industry trends for dermatology research. We aim to describe trends and features of clinical trials for dermatology drugs in China, understand recent achievements, and forecast development trends.</p><p><strong>Methods: </strong>Clinical trial records posted on the Registration and Information Disclosure Platform of Center for Drug Evaluation (CDE) were screened. All trials for dermatological drugs, posted on platform between 2016 and 2022 were included.</p><p><strong>Results: </strong>A total of 1172 trial records were identified, among which studies for skin infection, immune-mediated and inflammatory skin disease (IMIDs) and skin malignancy accounted for 48.9% (n = 573), 42.0% (n = 492) and 9.1% (n = 107), respectively. Most trials focused on generic drugs (n = 728, 62.1%). Multi-regional clinical trials (MRCTs) accounted for less than 6% of all trials. The number of trials on dermatology drugs increased sharply from 2016 to 2018 followed by a decline, which is mainly driven by the drop in bioequivalence evaluation (BE) studies for generic drugs indicated for skin infection. A growing trend in the number of trials for innovative drugs was observed. After removing duplicated drugs based on generic name, a total of 607 tested dermatology drugs were identified among which 51.9% were indicated for IMIDs. The number of innovative drugs exceeded generic drugs from 2020 to 2022. The geographic distribution of lead sites (the site where the principal investigator being employed) was uneven, with most of them located in east China. Of 1,068 trials sponsored by Chinese firms, most were BE (n = 692, 64.8%) and generic drugs (n = 722, 67.6%), while among 104 trials sponsored by multinational corporations (MNC), a majority were phase III (n = 53, 51.0%) trials and focused on innovative drugs (n = 94, 90.4%).</p><p><strong>Conclusion: </strong>Findings demonstrated positive consequences of reforms in the healthcare industry in China. Nevertheless, long-term policies are expected to enhance the innovative capabilities of Chinese pharmaceutical companies while ensuring accessible and affordable drug supply with generics, encourage early participation in global drug R&D activities to shorten \"drug lag\", and promote investment in innovative drugs.</p>","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143415308","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}