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<?xml version="1.0" encoding="UTF-8"?><rss version="2.0" xmlns:content="http://purl.org/rss/1.0/modules/content/" xmlns:wfw="http://wellformedweb.org/CommentAPI/" xmlns:dc="http://purl.org/dc/elements/1.1/" xmlns:atom="http://www.w3.org/2005/Atom" xmlns:sy="http://purl.org/rss/1.0/modules/syndication/" xmlns:slash="http://purl.org/rss/1.0/modules/slash/" xmlns:media="http://search.yahoo.com/mrss/" > <channel> <title>Journal for Clinical Studies</title> <atom:link href="https://journalforclinicalstudies.com/feed/" rel="self" type="application/rss+xml" /> <link>https://journalforclinicalstudies.com</link> <description>Your resource for Multisite Studies</description> <lastBuildDate>Fri, 02 May 2025 09:01:50 +0000</lastBuildDate> <language>en-GB</language> <sy:updatePeriod> hourly </sy:updatePeriod> <sy:updateFrequency> 1 </sy:updateFrequency> <generator>https://wordpress.org/?v=6.8.1</generator> <image> <url>https://journalforclinicalstudies.com/wp-content/uploads/2023/10/Favicon-JCS-150x150.png</url> <title>Journal for Clinical Studies</title> <link>https://journalforclinicalstudies.com</link> <width>32</width> <height>32</height></image> <item> <title>A CRO Should be the Partner that Gives Full Visibility on a Gene Therapy Trials Path</title> <link>https://journalforclinicalstudies.com/a-cro-should-be-the-partner-that-gives-full-visibility-on-a-gene-therapy-trials-path/</link> <dc:creator><![CDATA[chloe euripides]]></dc:creator> <pubDate>Fri, 02 May 2025 09:01:48 +0000</pubDate> <category><![CDATA[Current features]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24904</guid> <description><![CDATA[<p>Ahead of ARVO 2025 next week where Emmes is poised to launch the first dedicated ophthalmology clinical trial technology platform, we spoke with Saqib Parkar, Managing Director at Emmes’ OptymEdge, about how CROs can reshape gene therapy trials in ophthalmology. . With only one FDA-approved ocular gene therapy on the market, the stakes are high […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/a-cro-should-be-the-partner-that-gives-full-visibility-on-a-gene-therapy-trials-path/">A CRO Should be the Partner that Gives Full Visibility on a Gene Therapy Trials Path</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>Ahead of ARVO 2025 next week where Emmes is poised to launch the first dedicated ophthalmology clinical trial technology platform, we spoke with Saqib Parkar, Managing Director at Emmes’ OptymEdge, about how CROs can reshape gene therapy trials in ophthalmology. . With only one FDA-approved ocular gene therapy on the market, the stakes are high and timelines are critical. Ophthalmologists working with Saqib emphasise the growing importance of CROs as strategic partners—valued for their transparency, therapeutic expertise, and collaboration.</p> <p>As cell and gene therapies redefine treatment possibilities, clinicians play a key role in shaping trials that are not only scientifically sound but also tailored to the unique needs of rare disease patients. In this article we explore the pivotal role they play in helping deliver bespoke clinical trials for gene therapies in eye diseases and the profound benefits these trials bring to both practitioners and their patients.</p> <p><strong>The Evolving Landscape of Clinical Trials</strong></p> <p>The field of ophthalmology has witnessed extraordinary progress, thanks to breakthroughs in cell and gene therapies. These innovations offer a lifeline to patients with debilitating eye diseases. Dr. Paul A. Sieving, Director, Center for Ocular Regenerative Therapy at UC Davis School of Medicine, underscores the critical need for these trials, particularly in addressing rare inherited ocular disorders such as the Inherited Retinal Dystrophies (IRDs). He states, ‘these conditions, although individually rare, collectively impact a substantial number of individuals within communities”. The advent of gene therapies has rekindled hope for these patients, providing treatment options where none previously existed.</p> <p>While it’s important to note that results from ocular gene therapy trials have exhibited variability, and only one therapy has received FDA approval thus far, Dr. Ian M MacDonald, a distinguished ophthalmologist from the Department of Ophthalmology & Visual Sciences at the University of Alberta, reminds us that these trials are inherently experimental. “They serve as invaluable learning opportunities for the ophthalmic community, allowing for the refinement of protocols, redirection of research efforts, and, most significantly, unwavering support for patients as they embark on a journey towards potential breakthroughs,” added MacDonald.</p> <p><strong>The Significance of Gene Therapy Trials</strong></p> <p>Dr Sieving has been involved with a gene therapy trial for X-linked retinoschisis (XLRS). XLRS represents just one of many genetic conditions causing progressive vision loss, ranking among the top 10 most frequent conditions within the realm of Inherited Retinal Dystrophies (IRDs). When I spoke with him recently he agreed that the impact of such eye diseases on communities is substantial and stressed on the urgency of starting and hopefully completing successful trials. He highlighted why working more closely with CROs, and the relationship between sponsor trial manager and clinician has never been more important. A breakdown here will inevitably lead to delays and, in the worst of cases, can be a highly complicit factor in a trial’s failure – so we owe to patients to ensure this is avoided whenever possible.</p> <p>But first let’s look at a couple of successes, and Dr. Sieving is hopeful that the approval of Luxturna will herald in encouragement from clinicians to search out more trials and help these patients through what can be a dauting trial journey. While Luxturna – a Leber congenital amaurosis (LCA) treatment – was approved back in 2017, it remains the only one available and is a historic milestone as the first in vivo gene therapy. Infants and children treated with Luxturna experience vision restoration, offering tangible evidence of gene therapy’s potential to address genetic eye diseases.</p> <p>However, progress in the field since 2017 has been slower than anyone would have liked, and there is a clear need for more Human Gene Therapy ocular trials. What is encouraging as a CRO is the clinicians we work with strongly believe that these trials should be more collaborative. If we can share more expertise – maybe the practical learnings across competing trials that now we are positioned to appropriately leverage this potential. The challenge of course is that gene therapy trials for the eyes come with their unique set of difficulties and requirements, including skilled drug delivery, patient recruitment challenges, lengthy patient follow-up, and intricate trial data analysis. And, while it might be understandable people say ‘you would say that’, I would encourage clinicians to bring in one of the handful of experienced CROs in the space because trials design here are fundamental not only to the patients, but in how to engage with the regulators as we start to see successes.</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/a-cro-should-be-the-partner-that-gives-full-visibility-on-a-gene-therapy-trials-path/">A CRO Should be the Partner that Gives Full Visibility on a Gene Therapy Trials Path</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>UK Company, Stablepharma Ltd, Enters Phase 1 Clinical Trials with World-First Fridge-Free Tetanus & Diphtheria Vaccine</title> <link>https://journalforclinicalstudies.com/uk-company-stablepharma-ltd-enters-phase-1-clinical-trials-with-world-first-fridge-free-tetanus-diphtheria-vaccine/</link> <dc:creator><![CDATA[theArchitect]]></dc:creator> <pubDate>Thu, 01 May 2025 10:03:17 +0000</pubDate> <category><![CDATA[Regional News]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24897</guid> <description><![CDATA[<p>Following MHRA approval, Stablepharma Ltd has commenced a Phase 1 clinical trial with its lead candidate, fridge-free tetanus and diphtheria vaccine, SPVX02. The Phase 1, first-in-human clinical trial led by Professor Saul Faust, is being conducted at the National Institute for Health and Care Research (NIHR) at Southampton Clinical Research Facility. Stablepharma’s pioneering science has progressed into […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/uk-company-stablepharma-ltd-enters-phase-1-clinical-trials-with-world-first-fridge-free-tetanus-diphtheria-vaccine/">UK Company, Stablepharma Ltd, Enters Phase 1 Clinical Trials with World-First Fridge-Free Tetanus & Diphtheria Vaccine</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>Following MHRA approval, Stablepharma Ltd has commenced a Phase 1 clinical trial with its lead candidate, fridge-free tetanus and diphtheria vaccine, SPVX02. The Phase 1, first-in-human clinical trial led by Professor Saul Faust, is being conducted at the National Institute for Health and Care Research (NIHR) at Southampton Clinical Research Facility.</p> <p>Stablepharma’s pioneering science has progressed into the clinical trial stage thanks to funding from shareholders and the UK Government, including Innovate UK and the NIHR. The first participant was dosed in the trial on the 15<sup>th</sup> April 2025 and is expected to be completed in Q3 2025.</p> <p>Stablepharma’s next-generation technology platform StablevaX<img src="https://s.w.org/images/core/emoji/15.1.0/72x72/2122.png" alt="™" class="wp-smiley" style="height: 1em; max-height: 1em;" /> reformulates existing and new vaccines and biologicals into thermostable products that do not require cold storage such as refrigeration or freezing, thus eliminating the need for cold chain.</p> <p>This award-winning innovation tackles major global issues which include the distribution, storage, wastage, and CO2 emissions linked to the transportation of temperature-sensitive products. The World Health Organization reports that over 50% of vaccines are wasted each year due to cold chain failures, highlighting the significant impact StablevaX<img src="https://s.w.org/images/core/emoji/15.1.0/72x72/2122.png" alt="™" class="wp-smiley" style="height: 1em; max-height: 1em;" /> will have in improving vaccine access and reducing waste worldwide.</p> <p>Dr Karen O’Hanlon, CDO at Stablepharma who is leading the phase 1 trial with Professor Faust, said, “Our team have worked tirelessly to reach the start of this phase 1 trial with SPVX02 – it marks a pivotal moment for Stablepharma, as we advance into the clinical development phase with the world’s first fridge-free Td vaccine, a ground-breaking innovation in vaccine technology.”</p> <p>The team has demonstrated that SPVX02 remains stable and fully potent after three cycles of extreme temperature fluctuations, ranging from -20°C to +40°C. These findings underscore the resilience of StablevaX<img src="https://s.w.org/images/core/emoji/15.1.0/72x72/2122.png" alt="™" class="wp-smiley" style="height: 1em; max-height: 1em;" /> technology and its ability to effectively thermostabilise vaccines while maintaining vaccine efficacy. While most vaccines need constant refrigeration between 2-8°C, some biologics require storage at -20°C, making StablevaX<img src="https://s.w.org/images/core/emoji/15.1.0/72x72/2122.png" alt="™" class="wp-smiley" style="height: 1em; max-height: 1em;" /> a game-changer in addressing the challenges of maintaining product potency without any reliance on strict temperature controls. “We are very pleased that a room temperature (up to 30°C) shelf life of 18 months has been approved for the current clinical batch of SPVX02, manufactured in collaboration with our partner CDMO Thermo Fisher Scientific, for the phase 1 study,” added Dr O’Hanlon.</p> <p>Özgür Tuncer, CEO & Executive Director commented, “This phase 1 clinical trial is an important step towards launching the world’s first fridge-free Tetanus & Diphtheria vaccine (SPVX02) by 2027. We have shown that our StablevaX<img src="https://s.w.org/images/core/emoji/15.1.0/72x72/2122.png" alt="™" class="wp-smiley" style="height: 1em; max-height: 1em;" /> technology can be manufactured under commercially scalable GMP conditions, enabling us to scale-up the manufacturing process to provide millions of doses per year without the need for the global cold chain. Our ambition is to create a ‘new era’ in vaccines by reducing reliance on the cold chain.”</p> <p>Stablepharma was one of five UK companies awarded the €2.5 million grant through the European Innovation Council (EIC) Accelerator program. The grant recognises the company’s cutting-edge fridge-free pharmaceutical technology, which meets EIC’s rigorous criteria for excellence, impact, and risk management.</p> <p>Stablepharma’s R&D team have identified 60 temperature-sensitive vaccines that could benefit from the company’s StablevaX<img src="https://s.w.org/images/core/emoji/15.1.0/72x72/2122.png" alt="™" class="wp-smiley" style="height: 1em; max-height: 1em;" /> technology. The company is actively partnering with global vaccine manufacturers, academic institutions, and NGOs to accelerate the development of its pipeline of thermostable products, aiming to improve vaccine accessibility and reduce storage-related challenges worldwide.</p> <p>Stablepharma’s projects align with their commitment to positively impact global health, reduce wastage and ensure equitable access of vaccines.</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/uk-company-stablepharma-ltd-enters-phase-1-clinical-trials-with-world-first-fridge-free-tetanus-diphtheria-vaccine/">UK Company, Stablepharma Ltd, Enters Phase 1 Clinical Trials with World-First Fridge-Free Tetanus & Diphtheria Vaccine</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>Spotlight on Hepatitis B: New Report Examines the Disease’s Clinical Trial Landscape in 2025</title> <link>https://journalforclinicalstudies.com/spotlight-on-hepatitis-b-new-report-examines-the-diseases-clinical-trial-landscape-in-2025/</link> <dc:creator><![CDATA[theArchitect]]></dc:creator> <pubDate>Thu, 01 May 2025 09:51:53 +0000</pubDate> <category><![CDATA[Regional News]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24895</guid> <description><![CDATA[<p>Gain valuable insights into novel therapies, ongoing clinical trials, and future innovations shaping the treatment of Hepatitis B with a new, in-depth report. Hepatitis B is a highly infectious liver disease caused by the Hepatitis B virus (HBV) and is primarily transmitted through infected blood, bodily fluids, or childbirth. Although many cases are acute and […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/spotlight-on-hepatitis-b-new-report-examines-the-diseases-clinical-trial-landscape-in-2025/">Spotlight on Hepatitis B: New Report Examines the Disease’s Clinical Trial Landscape in 2025</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>Gain valuable insights into novel therapies, ongoing clinical trials, and future innovations shaping the treatment of Hepatitis B with a new, in-depth report.</p> <p>Hepatitis B is a highly infectious liver disease caused by the Hepatitis B virus (HBV) and is primarily transmitted through infected blood, bodily fluids, or childbirth. Although many cases are acute and asymptomatic, Hepatitis B can become chronic, posing a serious risk of cirrhosis and liver cancer. It remains a major global health concern, accounting for 65% of the worldwide hepatitis burden, with significant disparities in prevalence and clinical outcomes across regions and ethnicities.</p> <p>A new report from Novotech, a leading global contract research organization, examines the global clinical trial landscape for Hepatitis B in 2025, offering valuable insights into novel therapies, ongoing clinical trials, and future innovations shaping the treatment.</p> <p>According to the report, the disease burden varies significantly by region, with the Western Pacific and African regions carrying the highest prevalence. In the Asia-Pacific region, China and India have the highest affected populations, while Europe and the Americas maintain lower but steady infection rates. Racial and ethnic disparities affect Hepatitis B outcomes, with Asian populations having the highest chronic infection rates. Variations in HBV mutations influence disease progression and treatment, highlighting the need for preventive measures, early detection, and targeted therapies that address regional needs.</p> <h3 class="wp-block-heading" id="h-treatment-landscape">Treatment landscape</h3> <p>The report outlines how the treatment landscape for Hepatitis B focuses on antiviral therapies to suppress viral replication and reduce liver damage. Guidelines established by organizations like the American Association for the Study of Liver Diseases (AASLD) and the European Association for the Study of the Liver (EASL) provide personalized treatment options, recommending nucleos(t)ide analogs like tenofovir and entecavir for managing chronic Hepatitis B. While these treatments do not cure the infection, they slow disease progression, reduce the risk of complications like cirrhosis and liver cancer, and improve long-term outcomes. Ongoing research aims to develop next-generation therapies targeting HBV replication and enhancing immune control.</p> <p>Building on these established treatment strategies, the global Hepatitis B clinical trial landscape is expanding, with the Asia-Pacific leading 65% of trials. Research focuses on achieving a functional cure through RNA-based treatments, monoclonal antibodies, and gene therapies. Most trials are in the early and mid-stages, with over 55% in Phase II. Emerging therapies, including capsid assembly modulators, translation inhibitors, and cccDNA-targeting treatments, aim to improve viral control and immune response. Immunotherapy options like checkpoint inhibitors and therapeutic vaccines are also being explored, with combination approaches showing the most promise for a long-term cure.</p> <p>Advancements in Hepatitis B research have led to a better understanding of clinical features influencing disease progression, particularly through emerging biomarkers such as HBV RNA and HBcrAg. These biomarkers provide deeper insights into viral activity, immune response, and liver health, improving disease monitoring and treatment strategies. While traditional biomarkers such as HBsAg and HBV DNA are essential for staging and monitoring, they have limitations in predicting treatment outcomes. The latest Hepatitis B treatment data highlights advancements in antiviral therapies, with emerging biomarkers improving the refinement of treatment endpoints. As research continues, these new diagnostic tools are expected to enhance personalized treatment approaches and improve long-term patient outcomes.</p> <h3 class="wp-block-heading" id="h-clinical-trial-investment">Clinical trial investment</h3> <p>Shifting focus to the current investment landscape for Hepatitis B, the report highlights how growing efforts to develop a functional cure for Hepatitis B has seen investment in clinical trials surge, driven by public funding and venture capital. Between 2020 and 2024, the U.S. and China led investments, while the UK, Singapore, and France contributed moderate amounts. Building on this momentum, key players leading Hepatitis B cure trials include Bluejay Therapeutics, Precision Biosciences, and Tune Therapeutics. These advancements, backed by strong financial support, highlight a growing commitment to achieving a functional cure.</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/spotlight-on-hepatitis-b-new-report-examines-the-diseases-clinical-trial-landscape-in-2025/">Spotlight on Hepatitis B: New Report Examines the Disease’s Clinical Trial Landscape in 2025</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>Modern Clinical Trials — Apply a Mix of Co-Innovation and Best Practices to Improve Productivity and Drive ROI </title> <link>https://journalforclinicalstudies.com/modern-clinical-trials-apply-a-mix-of-co-innovation-and-best-practices-to-improve-productivity-and-drive-roi/</link> <dc:creator><![CDATA[theArchitect]]></dc:creator> <pubDate>Thu, 01 May 2025 09:48:12 +0000</pubDate> <category><![CDATA[Regional News]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24893</guid> <description><![CDATA[<p>Maximizing productivity and ROI in modern clinical trials  The development of new medications is a long, complex, and costly process. Productivity—how efficiently a drug developer creates value from their investment—is key to success, yet it often remains elusive. For pharma and life sciences stakeholders, improving R&D productivity entails balancing clinical outcomes, commercial success, and the […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/modern-clinical-trials-apply-a-mix-of-co-innovation-and-best-practices-to-improve-productivity-and-drive-roi/">Modern Clinical Trials — Apply a Mix of Co-Innovation and Best Practices to Improve Productivity and Drive ROI </a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>Maximizing productivity and ROI in modern clinical trials </p> <p>The development of new medications is a long, complex, and costly process. Productivity—how efficiently a drug developer creates value from their investment—is key to success, yet it often remains elusive. For pharma and life sciences stakeholders, improving R&D productivity entails balancing clinical outcomes, commercial success, and the resources required to bring a drug to market. </p> <p>The first article in this productivity series explores strategies to optimize every stage of clinical trials. It highlights the importance of rethinking workflows and leveraging innovative tools and techniques. As trial costs rise and productivity stagnates, stakeholders must shift away from traditional methods and embrace a structured approach. </p> <p><strong>Defining productivity in clinical trials</strong> </p> <p>Productivity in clinical trials can be measured in multiple ways: </p> <ul class="wp-block-list"><li>The number of assets in a portfolio multiplied by average lifetime revenue, divided by development costs per year. </li> <li>Average lifetime revenue per asset divided by annual costs to bring the asset to market. </li></ul> <p>Some stakeholders use effectiveness (achieving intended outcomes) and efficiency (resource utilization) as proxies for productivity. While related, they are distinct concepts. </p> <p><strong>The productivity framework</strong> </p> <p>Improving productivity starts with identifying and assessing opportunities while prioritizing initiatives. Sponsors and clinical research organizations (CROs) often rely on pilot programs to test new strategies. However, when time is constrained, rolling out parallel initiatives—treated as rigorous experiments—can yield faster, impactful results. Analyzing relevant metrics can help refine strategies over time. CROs should also adopt best practices developed from diverse trials to maximize their efforts and strategically implement productivity initiatives at optimal points in the clinical trial process. This systematic approach enables trial sponsors to compare initiatives, generate insights, and continuously enhance processes. </p> <p><strong>Harnessing technology and data</strong> </p> <p>Closing productivity gaps often requires innovative solutions. Advanced modeling and data-analytics technologies—including artificial intelligence (AI), machine learning (ML), and natural language processing (NLP)—can significantly reduce timelines and costs while improving outcomes. Regulatory-grade real-world data (RWD) and real-world evidence (RWE) are increasingly essential tools, offering valuable insights and supporting clinical and business decisions. </p> <p><strong>Final thoughts</strong> </p> <p>Achieving measurable ROI gains demands a departure from the status quo. Drug developers must critically evaluate each step of the clinical trial process and take bold steps toward implementing innovative practices. Deploying multiple initiatives simultaneously, optimizing resource allocation, and continuously analyzing outcomes will drive meaningful improvements. </p> <p>By embracing a mix of co-innovation and best practices, stakeholders can boost productivity, streamline clinical trials, and improve ROI, ultimately accelerating the journey from drug development to patient care. </p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/modern-clinical-trials-apply-a-mix-of-co-innovation-and-best-practices-to-improve-productivity-and-drive-roi/">Modern Clinical Trials — Apply a Mix of Co-Innovation and Best Practices to Improve Productivity and Drive ROI </a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>Roche App Relieves Diabetes Distress in Clinical Trial</title> <link>https://journalforclinicalstudies.com/roche-app-relieves-diabetes-distress-in-clinical-trial/</link> <dc:creator><![CDATA[theArchitect]]></dc:creator> <pubDate>Thu, 01 May 2025 09:46:04 +0000</pubDate> <category><![CDATA[Regional News]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24891</guid> <description><![CDATA[<p>A digital health application (DiGA) has been shown to relieve the distress that patients can experience when managing diabetes, which can compromise their care, in a randomised clinical trial. Diabetes distress is an emotional reaction specific to the demands of diabetes and diabetes therapy – leading to feelings of hopelessness, discouragement and exhaustion – which is not […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/roche-app-relieves-diabetes-distress-in-clinical-trial/">Roche App Relieves Diabetes Distress in Clinical Trial</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p><strong>A digital health application (DiGA) has been shown to relieve the distress that patients can experience when managing diabetes, which can compromise their care, in a randomised clinical trial.</strong></p> <p>Diabetes distress is an emotional reaction specific to the demands of diabetes and diabetes therapy – leading to feelings of hopelessness, discouragement and exhaustion – which is not necessarily pathological, but can become so if present at an elevated level, according to study investigator Dominic Ehrmann of the FIDAM diabetes research institute in Germany.</p> <p>He told the EASD meeting this week that it is clinically relevant to monitor diabetes distress, as high levels have been linked to a risk of depression, poorer quality-of-life, and suboptimal management of diabetes that can worsen blood glucose control, as measured by higher haemoglobin A1c levels. It is estimated that around 35% of patients with type 1 and type 2 diabetes have elevated distress levels.</p> <p>DiGAs can relieve distress in several ways. For example, by providing a simple overview of glucose control, making data easier to interpret, and providing motivational feedback and positive reinforcement when goals are achieved, said Ehrmann.</p> <p>The new trial investigated the role of the Pro version of the mySugr app, a digital diabetes logbook which Roche acquired along with its developer in 2017 and – in a defining moment for digital health within pharma – positioned it at the heart of its diabetes management ecosystem alongside products like its Accu-Chek blood glucose testing range.</p> <p>The study was specifically designed to test the effects of the app on diabetes distress, the primary endpoint, with the app compared to no digital intervention and patients followed up after 12 weeks. It included nearly 400 evaluable patients with type 1 and 2, as well as gestational diabetes, who were asked to carry out daily blood glucose measurements and input the results into the app.</p> <p>Diabetes distress was assessed using a standard patient-reported outcome (PRO) questionnaire called PAID, with various secondary measures, including Hba1C to give an indication of blood glucose control.</p> <p>There was an improvement in diabetes distress with mySugr Pro – a reduction of around 3.6 on the PAID scale versus a slight increase in the control group – which met the threshold for statistical significance with a p-value of 0.0182.</p> <p>There were no significant improvements on secondary endpoints in the study, including HbA1c, although Ehrmann noted there were 80 women with gestational diabetes who might not be expected to see a significant change in the biomarker over the course of the study.</p> <p>If only type 1 and type 2 patients were assessed, there was a 2.2 higher chance of achieving a target Hba1c level of 6.5% or lower, along with evidence for lower rates of very high or very low blood sugar, which provided some evidence of improved glycaemic control.</p> <p>There was also a high degree of patient satisfaction with the app, with a very low drop-out rate of 6.4 – unusual for studies of digital interventions – backed up by user data that showed that it was used as directed on 93% of days overall.</p> <p>“We hope that we can now go all the way to making the mySugr Pro app a prescribable digital health application in Germany,” said Ehrmann.</p> <p>There is a well-defined framework for approval and reimbursement of DiGAs in Germany, where there are 55 prescribable products on the market including five in the metabolic disease area, including apps to support lifestyle changes, manage obesity, and help patients deal with depression caused by diabetes.</p> <p>In the first nine months of 2022, the latest period for which data is available, there were more than 200,000 prescriptions for DiGA products, out of which around 80% (164,000) were redeemed.</p> <p>Around 31,000 of those were for diabetes-related apps at a cost of roughly €15 million to the German healthcare system, out of total spending on DiGAs of €55 million since the law was enacted.</p> <p>mySugr Pro is aiming to plug a gap in the category with its focus on diabetes self-management and glucose control, said Ehrmann.</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/roche-app-relieves-diabetes-distress-in-clinical-trial/">Roche App Relieves Diabetes Distress in Clinical Trial</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>Expert Predictions for 2025: Spotlight on Efficiency, AI, Regulatory, Risk and Better Communications</title> <link>https://journalforclinicalstudies.com/expert-predictions-for-2025-spotlight-on-efficiency-ai-regulatory-risk-and-better-communications/</link> <dc:creator><![CDATA[chloe euripides]]></dc:creator> <pubDate>Thu, 17 Apr 2025 10:20:04 +0000</pubDate> <category><![CDATA[Current features]]></category> <category><![CDATA[Volume 17 Issue 1]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24879</guid> <description><![CDATA[<p>In 2024 the life sciences sector experienced high levels of cost pressure that drove process transformation in regulatory functions and smarter use of data and human resources. At the same time, the potential of artificial intelligence (AI) began to be widely realised, with new use cases emerging to address the multiple challenges facing the sector. […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/expert-predictions-for-2025-spotlight-on-efficiency-ai-regulatory-risk-and-better-communications/">Expert Predictions for 2025: Spotlight on Efficiency, AI, Regulatory, Risk and Better Communications</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>In 2024 the life sciences sector experienced high levels of cost pressure that drove process transformation in regulatory functions and smarter use of data and human resources. At the same time, the potential of artificial intelligence (AI) began to be widely realised, with new use cases emerging to address the multiple challenges facing the sector. In this article, expert commentators review the developments of the past year and look forward to 2025.</p> <p>The life sciences sector has seen the beginning of a major transformation in 2024, driven by AI and data science technology that has underpinned process change and efficiencies in areas ranging from regulatory through to communications strategies.</p> <p>Cost reductions and attempts to leverage AI to save cost or become more efficient were overriding themes for 2024, according to Peter Muller, Director, Americas at Schlafender Hase: “This looks set to continue into 2025. For many organisations looking at the horizon right now there is a lot of uncertainty.”</p> <p>He adds, “Also a lot of companies are facing patent cliffs. Their portfolios have drugs that have lost or are losing patent protection, and they are not generating, or expected to generate the kind of revenue the company needs. Companies anticipate a period of financial struggle, and so they want to improve their operating profitability.”</p> <p><strong>AI Transforming Regulatory and Safety Functions</strong></p> <p>“The themes which have dominated the life sciences regulatory and quality landscape in 2024 are the integration of advanced data analytics and AI in regulatory compliance. This shift is driven by the need for more efficient and accurate compliance processes from the molecule to the product,” says Jens Marburg, Principal Consultant at MAIN5. He points out that evidence of this can be seen in the increased adoption of AI-driven tools for data validation, risk assessment, and regulatory reporting, adding: “2025 is likely to be the year of digital transformation and adoption in regulatory compliance.”</p> <p>ArisGlobal’s CEO Aman Wasan cautions: “As one of the most safety- and risk-conscious industries there is, it’s important that pharma/biopharma gets AI right. Where patient safety is concerned, there can be no tolerance for ‘black box’ mystery; nor data security/ privacy breaches. Results must be reliable, robust, explainable, consistent, and trust-inspiring. It’s why the sector is pushing hard not only to harness trailblazing applications but also to test the boundaries of AI explainability, optimised human sampling, and transparency (e.g. through the combination of LLMs and retrieval augmented generation or RAG), so that regulators can see and assess outcomes for their reliability and consistency.”</p> <p>Marburg at MAIN5 has seen clients increasingly embracing risk based approaches to computerised system validation (CSV), moving away from exhaustive validation processes to more targeted, risk focused strategies that allows for more efficient use of resources and faster project timelines. However, he says, “Companies are still not fully appreciating the importance of data governance and ownership. Many are at risk of non-compliance due to fragmented data management practices. The urgency around this issue is high, as regulatory bodies are increasingly scrutinising data integrity. Companies should establish clear data governance frameworks and assign ownership to ensure compliance and data quality.”</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/expert-predictions-for-2025-spotlight-on-efficiency-ai-regulatory-risk-and-better-communications/">Expert Predictions for 2025: Spotlight on Efficiency, AI, Regulatory, Risk and Better Communications</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>Visualisation Approaches Coming to Light: Advances in Optical Imaging</title> <link>https://journalforclinicalstudies.com/visualisation-approaches-coming-to-light-advances-in-optical-imaging/</link> <dc:creator><![CDATA[chloe euripides]]></dc:creator> <pubDate>Thu, 17 Apr 2025 10:17:21 +0000</pubDate> <category><![CDATA[Current features]]></category> <category><![CDATA[Volume 17 Issue 1]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24876</guid> <description><![CDATA[<p>Amidst the “burgeoning” interest in developing novel optical imaging drugs and imaging devices to assist various standard surgical procedures, the US Food and Drug Administration (FDA) recently issued guidance regarding clinical trial design features supporting the development and approval of optical imaging agents. According to the agency, a contributing factor propelling the advancement of these […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/visualisation-approaches-coming-to-light-advances-in-optical-imaging/">Visualisation Approaches Coming to Light: Advances in Optical Imaging</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>Amidst the “burgeoning” interest in developing novel optical imaging drugs and imaging devices to assist various standard surgical procedures, the US Food and Drug Administration (FDA) recently issued guidance regarding clinical trial design features supporting the development and approval of optical imaging agents. According to the agency, a contributing factor propelling the advancement of these drugs is the use of minimally invasive surgical procedures, which are associated with a loss of tactile perception and a more narrowed field of view.</p> <p>As explained in the FDA’s new draft guidance for industry Developing Drugs for Optical Imaging published in January 2025, optical imaging is the use of light in conjunction with imaging drugs and devices during medical procedures to assist in the detection of tumours or other pathology and delineation of normal anatomical structures. Surgeons employ these drug/device pairings to help with the direct visual inspection and palpation of tissue in the surgical field. Because imaging drugs enhance the surgeon’s ability to discern tumours from normal tissue, they can augment the likelihood of safe and complete removal of cancers and minimise the risk of unintended injury to the normal structures.</p> <p>Optical imaging is advantageous as an imaging modality because it significantly reduces patient exposure to harmful radiation by using non-ionising radiation, which includes visible, ultraviolet, and infrared light, the National Institute of Biomedical Imaging and Bioengineering, National Institutes of Health (NIH), explains. Since it is much safer than techniques that require ionising radiation such as X-rays, optical imaging can be used for repeated procedures to monitor disease progression or the results of treatment.</p> <p>The new guidance focuses on the use of optical imaging for tumour detection (surgery, endoscopic resection of neoplasm), lymph node staging (lymphatic mapping, Sentinel lymph node identification), and the enhanced delineation of normal anatomy to decrease risk of injury (e.g., nerve structures in head and neck surgery). The FDA notes that optical imaging drugs are generally governed by the same regulations as other drugs.</p> <p>Moreover, its recommendations to developers of other medical imaging drugs – published in three separate guidance documents more than 20 years ago– remain applicable to optical imaging drugs.</p> <p><strong>Strengthening Surgical Success</strong></p> <p>In 2024, the FDA approved 50 “novel” drugs, either as new molecular entities (NMEs) under new drug applications (NDAs) or as new therapeutic biologics under biologics license applications (BLAs), as summarised in the agency’s 14th annual report, Advancing Health Through Innovation: New Drug Therapy Approvals 2024 issued in January 2025. In novel drugs, the active ingredients have not been previously approved in the US. Patrizia Cavazzoni, MD, then directorof the Center for Drug Evaluation and Research (CDER), FDA, stated in the report that it features “notable” drug approvals that CDER considers “likely to have a significant impact on patient care and public health.”</p> <p>Among the 50 agents, CDER highlighted the optical imaging prodrug pegulicianine as one of the notable first-in-class approvals. Granted marketing authorisation under the trade name Lumisight from Lumicell, Inc, pegulicianine is optically inactive when intact but produces a fluorescent signal after its peptide chain is cleaved by cathepsins and matrix metalloproteases. The levels of these enzymes are higher in and around tumour and tumour-associated cells than normal cells. This enzymatic cleavage generates two optically active metabolites that fluoresce and another fragment that contains the fluorescence quencher that maintains the intact molecule optically inactive.</p> <p>Pegulicianine is administered as an intravenous injection for fluorescence imaging in adults with breast cancer as an adjunct for the intraoperative detection of cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery. It was approved for use with the Lumicell Direct Visualisation System (DVS) or another fluorescence imaging device that is FDA approved for use with pegulicianine in the indicated population.</p> <p></p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/visualisation-approaches-coming-to-light-advances-in-optical-imaging/">Visualisation Approaches Coming to Light: Advances in Optical Imaging</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>How Technology will Shape Pharma in 2025: Predictions from Veeva</title> <link>https://journalforclinicalstudies.com/how-technology-will-shape-pharma-in-2025-predictions-from-veeva/</link> <dc:creator><![CDATA[chloe euripides]]></dc:creator> <pubDate>Thu, 17 Apr 2025 10:10:56 +0000</pubDate> <category><![CDATA[Current features]]></category> <category><![CDATA[Volume 17 Issue 1]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24871</guid> <description><![CDATA[<p>As we advance through 2025, biopharma companies are increasingly using artificial intelligence (AI) and new data tools to improve commercial performance and make research and development (R&D) more efficient. AI is changing the way the industry engages with healthcare professionals (HCPs), allowing for personalised interactions, better content strategies, and useful insights for field teams. In […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/how-technology-will-shape-pharma-in-2025-predictions-from-veeva/">How Technology will Shape Pharma in 2025: Predictions from Veeva</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>As we advance through 2025, biopharma companies are increasingly using artificial intelligence (AI) and new data tools to improve commercial performance and make research and development (R&D) more efficient. AI is changing the way the industry engages with healthcare professionals (HCPs), allowing for personalised interactions, better content strategies, and useful insights for field teams. In R&D, new methods for handling data and navigating regulations are speeding up clinical trials, shortening approval times, and improving transparency at research sites.</p> <p>On the commercial side, making the most of AI and data-driven solutions depends on having clean, organised data and investing in systems that are scalable and compliant. Companies focusing on data quality, following regulations, and using AI-driven insights will be better prepared for long-term success. Key improvements include AI powered tools for creating and reviewing content, advanced analytics for smarter business decisions, and streamlined operations through integrated quality and regulatory systems.</p> <p>In R&D, the spotlight is on speeding up approvals through simultaneous submissions, sharing data more openly with contract research organisations (CROs), and automating safety monitoring. Simultaneous submissions can cut approval times significantly, while better data sharing with CROs leads to faster, more informed decisions. Advanced automation, supported by reliable safety data, will simplify pharmacovigilance and reduce operational challenges.</p> <p><strong>Prediction 1: A Focus on Clean Data Will Fuel Compliant AI Innovation in the EU</strong></p> <p>The recent wave of AI innovation has fallen short of transforming commercial life sciences. In 2025, European biopharma’s that unlock harmonised internal and external data will start to reap commercial rewards.</p> <p>Biopharma organisations will combine off-the-shelf AI engines with more harmonised, clean data. The integration of these AI solutions with well-structured, high-quality datasets will enable organisations to achieve deeper insights, enhance operational efficiency, and drive evidence-based decision-making. Acquiring data from trusted, internally verified sources will lead to greater confidence in AI-generated outcomes. By ensuring data integrity and consistency across different functions, companies can minimise biases and inaccuracies, ultimately boosting stakeholder trust. This will make it easier to scale pilots from single-market, single-brand solutions across the enterprise. As these scaled solutions prove their value, biopharma organisations will be better positioned to optimise their supply chains, improve patient outcomes, and streamline regulatory reporting.</p> <p>The EU recently introduced the Artificial Intelligence Act — the first comprehensive AI regulation by any regulator, designed to ensure that AI is developed and used safely. This landmark legislation establishes clear guidelines on risk categorisation, transparency, and accountability, compelling companies to align their AI strategies with ethical and legal standards. Along with existing European data privacy rules, European biopharma’s will have clear principles to support future investment and innovation. These combined regulations will foster an environment where AI-driven projects are not only innovative but also aligned with societal expectations and legal mandates. Commercial success will come to those that clean up their data, secure new sources, and interrogate them within this regulatory framework. In the long run, organisations that proactively embrace compliance while leveraging AI will gain a competitive advantage, positioning themselves as industry leaders in ethical AI adoption.</p> <p></p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/how-technology-will-shape-pharma-in-2025-predictions-from-veeva/">How Technology will Shape Pharma in 2025: Predictions from Veeva</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>Addressing Disparities in Clinical Research Participation in Genetically Based Rare Paediatric and Neurodevelopmental Diseases</title> <link>https://journalforclinicalstudies.com/addressing-disparities-in-clinical-research-participation-in-genetically-based-rare-paediatric-and-neurodevelopmental-diseases/</link> <dc:creator><![CDATA[chloe euripides]]></dc:creator> <pubDate>Thu, 17 Apr 2025 10:06:12 +0000</pubDate> <category><![CDATA[Current features]]></category> <category><![CDATA[Volume 17 Issue 1]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24865</guid> <description><![CDATA[<p>We are at an important inflection point in our identification and understanding of genetically based diseases, particularly rarer paediatric-onset disorders that impact neurodevelopment. Concurrent with this movement forward in our knowledge and capacity for diagnosis is a demand for increased treatment, ideally at earlier stages of development. While early diagnosis of some conditions (e.g., Autism […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/addressing-disparities-in-clinical-research-participation-in-genetically-based-rare-paediatric-and-neurodevelopmental-diseases/">Addressing Disparities in Clinical Research Participation in Genetically Based Rare Paediatric and Neurodevelopmental Diseases</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>We are at an important inflection point in our identification and understanding of genetically based diseases, particularly rarer paediatric-onset disorders that impact neurodevelopment. Concurrent with this movement forward in our knowledge and capacity for diagnosis is a demand for increased treatment, ideally at earlier stages of development. While early diagnosis of some conditions (e.g., Autism Spectrum Disorders) has led to enhanced referral, subsequent intervention at younger ages, and better outcomes, many rarer genetically based paediatric diseases remain without such early treatment. This is particularly the case with medications and genetic interventions, which require significant time for development within the laboratory and then multiphase clinical trials to assess efficacy. The ability to engage disparately affected communities has also proven challenging. A shared commitment by both the disease support communities and drug regulatory agencies has encouraged greater efforts for increased diverse participation in clinical trials. This article reviews where we stand regarding the involvement of diverse affected communities and identifying where specific emphases can be placed, to better allow for enhanced recruitment, assessment of change, and improved outcomes.</p> <p>We are in the midst of a significant revolution in our identification and understanding of genetically based diseases, particularly regarding the range of paediatric-onset disorders that impact neurodevelopment and contribute to significant morbidity and mortality. Concurrent with this substantial leap in our knowledge and resulting capacity for diagnosis is a demand for increased treatment, ideally at an earlier stage of development, to maximise outcomes and opportunities for affected individuals. Early diagnosis in such conditions as Autism Spectrum Disorders has led to enhanced referral and engagement in intervention at a younger age, and hence, better outcomes given this earlier opportunity for significant support and change. However, many of the rarer genetically based paediatric diseases, including several lysosomal storage disorders, remain elusive to early treatment. This is particularly the case regarding medications and genetically based interventions, which require significant time for development within the laboratory, followed by multiphase clinical trials, to assess their validity and efficacy. There have, however, been important improvements in identifying and developing pharmacological interventions over the past decade, specifically for diseases like the mucopolysaccharidoses (MPS), Gaucher, and Rett. And these interventions have opened promising avenues for developmental improvement.</p> <p>One significant challenge has been the difficulty with more effectively connecting and engaging the diverse communities that are affected by these diseases into clinical trials, particularly at phase II and III levels. This has been identified most recently as a significant need – in recognition that many of these diseases are more readily present in diverse groups and settings where opportunity for intervention is limited. The emphasis on this particularly important challenge has led to a shared commitment by both the disease support communities, often through patient advocacy efforts, the United States Food and Drug Administration (FDA), and the European Medicines Agency (EMA), to engage greater efforts for such increased participation. With a clear emphasis on expanding treatment development in tandem with enhanced community involvement and breadth of patient engagement, it is hoped that better options for effective interventions and stronger outcomes will alter the current landscape dramatically.</p> <p>This article reviews where we presently stand with regard to the involvement of the broad diversity of communities, both in the Western countries where much research takes place, but also more directly worldwide. Furthermore, we will identify and discuss where specific emphases can be placed to better allow for enhanced recruitment, assessment of change, and for improved outcomes that are seen as efficacious and valid across the range of communities these diseases affect.</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/addressing-disparities-in-clinical-research-participation-in-genetically-based-rare-paediatric-and-neurodevelopmental-diseases/">Addressing Disparities in Clinical Research Participation in Genetically Based Rare Paediatric and Neurodevelopmental Diseases</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> <item> <title>First-ever MHRA Analysis of UK Clinical Trial Applications Finds New Opportunities to Drive Medical Breakthroughs for Patients</title> <link>https://journalforclinicalstudies.com/first-ever-mhra-analysis-of-uk-clinical-trial-applications-finds-new-opportunities-to-drive-medical-breakthroughs-for-patients/</link> <dc:creator><![CDATA[theArchitect]]></dc:creator> <pubDate>Thu, 17 Apr 2025 10:06:07 +0000</pubDate> <category><![CDATA[Regional News]]></category> <guid isPermaLink="false">https://journalforclinicalstudies.com/?p=24867</guid> <description><![CDATA[<p>New analysis of the current clinical trial landscape in the UK shows clear opportunities to shape the future of medical research and patient care. The first-ever analysis of the UK clinical trial landscape by the Medicines and Healthcare products Regulatory Agency (MHRA) and the University of Liverpool reveals the UK is a global leader in […]</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/first-ever-mhra-analysis-of-uk-clinical-trial-applications-finds-new-opportunities-to-drive-medical-breakthroughs-for-patients/">First-ever MHRA Analysis of UK Clinical Trial Applications Finds New Opportunities to Drive Medical Breakthroughs for Patients</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></description> <content:encoded><![CDATA[<p>New analysis of the current clinical trial landscape in the UK shows clear opportunities to shape the future of medical research and patient care.</p> <p>The first-ever analysis of the UK clinical trial landscape by the Medicines and Healthcare products Regulatory Agency (MHRA) and the University of Liverpool reveals the UK is a global leader in clinical research – and sets out key opportunities to deliver even more life-changing treatments for patients.</p> <p>Published today in the British Journal of Clinical Pharmacology, the report offers the most detailed picture yet of the UK’s clinical trials landscape. It finds strong innovation – but also a concentration of research in certain disease areas, and opportunities for increased representation of certain patient groups.</p> <h2 class="wp-block-heading" id="a-roadmap-for-stronger-more-inclusive-research">A roadmap for stronger, more inclusive research</h2> <p>The MHRA is using the insights to build upon the country’s world-leading clinical research and deliver its new clinical trials regulations to create a more efficient, streamlined and adaptable regulatory framework. Working in partnership with patients, the NHS, industry and academia, the MHRA will support increased research into underrepresented conditions, improve diversity in trial participation, and attract further global investment in innovation.</p> <h3 class="wp-block-heading" id="professor-andrea-manfrin-lead-author-of-the-study-and-mhra-deputy-director-clinical-investigations-and-trials-said">Professor Andrea Manfrin, lead author of the study and MHRA Deputy Director, Clinical Investigations and Trials, said:</h3> <p>“Clinical trials are the backbone of medical progress, essential for developing new medicines and advancing our understanding of diseases. This analysis shows clearly where the UK is leading – and where we need to work with our stakeholders to go further. By working together with patients, the NHS, industry, and researchers across the life sciences ecosystem to identify and maximise these opportunities, we can ensure clinical trials are faster, fairer, and more inclusive. Better trials mean better, more effective treatments, reaching NHS patients as quickly and as safely as possible.”</p> <h3 class="wp-block-heading" id="professor-sir-munir-pirmohamed-co-author-of-the-study-at-the-university-of-liverpool-said">Professor Sir Munir Pirmohamed, co-author of the study at the University of Liverpool, said:</h3> <p>“The analysis from the MHRA clinical trials database shows the richness of UK clinical trial activity involving medicines. Importantly it also provides a baseline which can be used to increase future UK clinical trial activity, which is important for improving both patient outcomes and economic investment.”</p> <p>With the global clinical trials market expected to nearly double to over £80 billion by 2032, insights from the analysis will help shape policies that can bring innovative, new medicines to patients, attract investment, accelerate medical innovation, and expand trial access for UK patients. </p> <p>Key findings from the MHRA and University of Liverpool’s analysis of all 4,616 clinical trials submitted between 2019 and 2023:</p> <ul class="wp-block-list"><li>The UK is a hub for pioneering research, with one in eight trials testing treatments in humans for the first time. There is strong commercial investment in UK trials, with 85% industry sponsored. A smaller share (15%) comes from universities, hospitals, and charities.</li> <li>Cancer trials dominate, making up nearly a third of all studies, but other major diseases lag behind. Heart disease – the world’s biggest killer – receives just 5.2% of research focus. Trials for conditions such as chronic pain, respiratory conditions and mental health disorders were among the least common, despite their major impact on public health.</li> <li>Both sexes were included in most trials (90%), however male-only trials (6.1%) were nearly twice as common as female-only studies (3.7%). Pregnant and breastfeeding women were represented in 1.1% and 0.6% of trials, respectively, which could impact treatment suitability for these groups.</li> <li>Cutting-edge treatments, such as gene and cell therapies, represent a growing clinical area but make up only 3.4% of trials, despite their potential to transform care for patients with limited treatment options.</li></ul> <h3 class="wp-block-heading" id="partnership-working-to-strengthen-uk-clinical-research">Partnership working to strengthen UK clinical research</h3> <p>The report sets a baseline to track progress and inform future funding, policy and regulation. The MHRA is already working with partners across the life sciences sector to increase research and streamline approvals in areas of unmet need through the Innovative Licensing and Access Pathway (ILAP); improve diversity in trial participation through the development of joint guidance with the Health Research Authority (HRA) so trials reflect the populations they aim to serve; and support more advanced therapy trials through collaboration with researchers via the Centres of Excellence for Regulatory Science and Innovation (CERSIs).</p> <p>These initiatives form part of wider clinical trials reform, including new legislation we are committed to implementing that will streamline how clinical trials are run in the UK. Backed by the MHRA and healthcare system partners, the changes aim to protect patient safety, boost global investment, and cut unnecessary red tape – helping bring new treatments to patients faster.</p> <p>As the government pushes forward the development of the Life Sciences Sector Plan and the 10 Year Health Plan, these findings come at a crucial time. They can be used to shape policies that ensure clinical trials deliver maximum benefit for patients, the NHS and the wider economy.</p> <h3 class="wp-block-heading" id="health-minister-karin-smyth-said">Health Minister Karin Smyth said:</h3> <p>“The government is determined to make Britain a world leader in life sciences, developing groundbreaking treatments focused on the conditions that matter most to patients.</p> <p>“As part of our Plan for Change, we’re laying the foundations for a modern, resilient health system that delivers, which is why the Prime Minister announced £520 million investment this week to turbocharge medical research.</p> <p>“By driving forward research and expanding access to clinical trials, we can ensure patients benefit from cutting-edge treatments quicker, while creating high-quality jobs and attracting global investment.</p> <p>“Strengthening the trial environment will help ensure we have an NHS fit for the future – one that harnesses innovation to improve outcomes for patients.”</p> <h3 class="wp-block-heading" id="science-minister-lord-vallance-said">Science Minister Lord Vallance said:</h3> <p>“As home to a thriving life sciences sector and the NHS, the UK is uniquely placed to host the trials and research that are taking the fight to a host of devastating health conditions. But as this data shows, we can go further and move faster through targeted investment, and smart regulation.</p> <p>“We are committed to doing precisely that – through this year’s record £13.9 billion funding for R&D in life sciences and beyond, as well as the efforts of our new Regulatory Innovation Office. We must make sure that trials of new medicines are available to everyone to take part.”</p> <h3 class="wp-block-heading" id="lawrence-tallon-mhra-chief-executive-said">Lawrence Tallon, MHRA Chief Executive, said:</h3> <p>“This first-of-its-kind analysis builds on our important work to strengthen clinical research in the UK. We are committed to implementing a flexible and risk-proportionate regulatory approach for clinical trials, which accelerates patient access to potentially life-saving medicines without compromising safety.</p> <p>“We’re making the UK one of the best places in the world to run clinical trials, with combined review approval times with the Health Research Authority now at 60 days or less for all trials. These changes not only benefit patients today but are laying the groundwork to accelerate innovation and deliver life-changing treatments to patients faster.”</p> <h3 class="wp-block-heading" id="matt-westmore-health-research-authority-chief-executive">Matt Westmore, Health Research Authority Chief Executive:</h3> <p>“Health and social care research should be done with, and for, everyone.</p> <p>“We know that trials that involve a diverse group of participants help provide a better understanding of how effective a treatment is for different groups of people. In turn this helps us support efforts to address health inequalities.</p> <p>“We are pleased to be working alongside the MHRA to develop new guidance designed to make it easier for researchers to ensure they are designing trials that are more representative of the people it is for and about.”</p> <p>The MHRA will continue tracking progress and working with its partners to ensure the UK remains a world leader in medical research, keeping patient safety at the heart of clinical trials.</p><p>The post <a rel="nofollow" href="https://journalforclinicalstudies.com/first-ever-mhra-analysis-of-uk-clinical-trial-applications-finds-new-opportunities-to-drive-medical-breakthroughs-for-patients/">First-ever MHRA Analysis of UK Clinical Trial Applications Finds New Opportunities to Drive Medical Breakthroughs for Patients</a> appeared first on <a rel="nofollow" href="https://journalforclinicalstudies.com">Journal for Clinical Studies</a>.</p>]]></content:encoded> </item> </channel></rss> If you would like to create a banner that links to this page (i.e. this validation result), do the following:
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