biomedwoRx: Life Sciences Consulting’s Post

In the pharmaceutical development's evolving landscape, medical affairs have never been more critical, especially with complex therapies needing complex clinical trials. By integrating robust data analytics and regulatory expertise, medical affairs professionals are best equipped to address the complexities of clinical trials, ensuring compliance and the integration of valuable patient insights from the outset. Integrating medical affairs from early development stages is crucial, as it optimizes trial design and patient recruitment while ensuring that trials reflect patient needs and real-world conditions more. Medical affairs' early involvement is essential to reshaping the drug development process, increasing efficiency and patient outcomes. https://lnkd.in/evQscsBi

The pharmaceutical industry is undergoing a significant transformation as the demand for complex therapies continues to grow. These therapies require intricately designed clinical trials, with the integration of robust data analytics and regulatory expertise from the early stages of development becoming essential. Early involvement of medical affairs professionals ensures that clinical trials are not only compliant but also optimized for patient recruitment and reflective of real-world conditions. This shift is reshaping the drug development landscape. By prioritizing early-stage integration, the industry is driving greater efficiency in trial design, enhancing patient recruitment strategies, and ultimately improving patient outcomes. These advancements underscore the importance of adapting to new methodologies and leveraging specialized expertise to navigate the complexities of modern clinical trials.

It's fascinating to witness the pharmaceutical industry's shift towards more complex therapies and the crucial role that early integration of medical affairs plays in this transformation. By embedding robust data analytics and regulatory expertise right from the outset, we're not only enhancing compliance but also refining clinical trials for better patient recruitment and real-world applicability. This proactive approach is vital for streamlining drug development and optimizing outcomes, marking a significant evolution in bringing therapies to those needing them.

Role and responsibilities of stakeholders in clinical research In clinical research, stakeholders play crucial roles throughout the process to ensure the safety of participants and the integrity of the data collected. 1)Sponsors: Typically pharmaceutical, biotechnology, or medical device companies, sponsors are responsible for initiating, managing, and financing the clinical trial. They design the study protocol, manage the budget, and ensure compliance with regulatory requirements. They are also responsible for the overall management of the trial data and reporting the findings. 2)Investigators: These are the individuals (usually physicians) responsible for conducting the trial at the study site. Their responsibilities include recruiting patients, ensuring informed consent, overseeing the administration of the study treatments, and collecting data. Investigators ensure that the study is conducted in accordance with the approved protocol and GCP (Good Clinical Practice) guidelines. 3)Clinical Research Coordinators (CRCs): CRCs assist investigators in day-to-day trial operations at the site level. They manage the administrative functions, data collection, patient scheduling, and often serve as the primary point of contact for trial participants. 4)Institutional Review Boards (IRBs)/Ethics Committees: These bodies are responsible for the ethical review of the clinical trial protocols. They ensure that the studies are ethically conducted and that the rights and welfare of participants are protected. They review the study before it begins and conduct periodic reviews throughout the study. 5)Regulatory Authorities: Organizations like the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) are responsible for overseeing the safety and efficacy of medical products. In the context of clinical trials, they review and approve study protocols and are informed of ongoing results, ensuring compliance with legal and regulatory standards. 6)Contract Research Organizations (CROs): CROs are external organizations hired by sponsors to manage one or more aspects of clinical trials. Responsibilities can include patient recruitment, data management, regulatory compliance, and study monitoring. They ensure trials are conducted efficiently and adhere to regulatory standards. 7)Participants: participants are central to clinical trials. They provide informed consent to participate in the study, adhere to study protocols, and communicate with the study team about their experiences and any side effects. 8)Data Monitoring Committees (DMC): Also known as Data Safety Monitoring Boards (DSMB), these independent groups periodically review and evaluate the accumulated study data for participant safety, study conduct and progress, and effectiveness of the intervention. They recommend continuation, modification, or termination of the trial based on safety data.

FDA NEW: Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA-Regulated Medical Products Guidance for Industry The purpose of this guidance is to provide FDA’s expectations for, and recommendations on, use of a standardized approach for collecting and reporting race and ethnicity data in #submissions including information collected and reported from clinical trials and clinical studies for FDA- regulated medical products. FDA’s recommended approach is based on the #OfficeofManagementandBudget (OMB) Statistical Policy Directive No. 15 (#PolicyDirective 15) and was developed in accordance with section 4302 of the #AffordableCareAct; the Health and Human Services (#HHS) Implementation Guidance on Data Collection Standards for Race, Ethnicity, Sex, Primary Language, and Disability Status;and the Food and Drug Administration Safety and Innovation Act (#FDASIA) Section 907 Action Plan. This guidance provides recommendations on: 1. Meeting the requirements set forth in the 1998 final rule10 regarding presentation of demographic data in investigational new drug applications (#INDs) and new drug applications (#NDAs) (known as the Demographic Rule) 2. Collection of race and ethnicity data in biologics license applications (BLAs) and medical device applications 3. Addressing the FDASIA Section 907 Action Plan to improve the completeness and quality of demographic data collection and reporting For drugs, the Demographic Rule requires the sponsor of an IND to tabulate in an IND annual report the number of participants enrolled in the clinical trial by certain demographic subgroups including race and requires NDA submissions to include summaries of effectiveness and #safetydata for demographic subgroups, including racial subgroups. FDA also strongly recommends the collection and reporting of ethnicity data (Hispanic or Latino or not Hispanic or Latino) consistent with OMB standards. This guidance is also intended to help an applicant preparing a BLA or a #devicepremarketsubmission, which should be done in accordance with the OMB standards regarding collection and reporting of race and ethnicity data described herein. This guidance also recommends the use of the OMB race and ethnicity categories in proposed medical product labeling. Sponsors of investigational new drugs and investigational devices should enroll participants who reflect the population that will use the medical product if approved. 15 Sections 505(z) and 520(g) of the Federal Food, Drug, and Cosmetic Act, as amended by section 3601 of the Food and Drug Omnibus Reform Act of 2022 (#FDORA) require that such sponsors submit a diversity action plan outlining (1) the sponsor’s goals for enrollment in the clinical trial, (2) the sponsor’s rationale for such goals, and (3) an explanation of how the sponsor intends to meet such goals. IF YOU LIKE THIS POST FOLLOW ME ON LINKEDIN #FDA #regulatory #clinicaltrials #clinicalstudies #race #ethnicity

Harmonising the EU regulatory framework for medical devices and drugs The EU MDR framework creates different standards for medical devices and drugs, impacting clinical research and HTA. Authors analysed phase II and III clinical trials (interventional studies) funded by the industry, intervention being drugs and MDs. The assumption is that these lead to the most relevant data for submissions to regulatory approval and HTA submissions for pricing and reimbursement. Highlights: ⚠ Bringing a MD to market takes significantly less time. ⚠ EU faces challenges ensuring that only safe and effective devices reach the market and monitoring their real-world utilisation. ⚠HTA that informs the adoption of MDs comes across methodological issues due to scarce evidence on clinical effectiveness, as MDR regulation focuses on safety/performance vs efficacy/effectiveness.  ⚠MDs thus have lower regulatory standards of evidence collection for placing them on the market, ultimately hindering comparison between these health technologies, especially when they have the same goal and are targeted at the same disease and/or population. ⚠MDs are subject to clinical investigations rather than clinical trials. Applications are assessed by demonstrating compliance with safety and performance requirements. An application to conduct a clinical trial is evaluated on its relevance, especially on whether the group of subjects participating in the clinical trial represent the population to be treated. ⚠Manufacturers of MDs may, in certain circumstances, select not to resort to data from clinical investigations, relying on studies reported in the scientific literature showing equivalence to another approved device. Policy changes could be implemented to promote an integrated evidence-based assessment system for a better allocation of resources in healthcare, namely: ✔ Improve the MD classification system taking into account an HTA perspective. ✔ Match regulatory standards between devices and drugs for quality, safety, clinical efficacy and effectiveness: ✔ Improve health and safety through harmonised outcome generation for devices and drugs. ✔ Implement conditional coverage and post-approval evidence development. ✔ Implement high-quality registries for devices.   We add: ⏹ The development and adoption of evidence sandbox facilities and integrative evidence standards adopted by the data industry horizontally for use for both types of devices (s/w and h/w) will significantly improve the MDR implementation in terms of clinical trials and HTA pathways. This includes access, adaptability and efficiency to increase the quality of evidence, of clinical impact results and reduce barriers to market entry for SMEs so that innovative, safe, and effective technologies may be delivered to improve the outcomes that matter to patients in a timely manner.   https://lnkd.in/dh2bUS6j #EU #MDR #RegulatoryPolicy #HTA #MDs #DigitalHealth #AI #SaMD

The impact of the EU MDR on the Quality of Evidence for HTA The EU MDR framework creates different standards for medical devices and drugs impacting clinical research and HTA. Authors analysed phase II and III clinical trials/ studies funded by the industry, where the intervention is drugs and MDs. The assumption is that these lead to the most relevant data for submissions to regulatory approval and HTA submissions for pricing and reimbursement. Highlights: 🔷Bringing MDs to market takes 3–7 years vs an average 12 years for drugs. 🔷 EU faces challenges ensuring that only safe and effective devices reach the market and monitoring their real-world utilisation. 🔷HTA to adopt MDs comes across methodological issues due to scarce evidence on clinical effectiveness when assessing MDs, as the regulation for their access to the market focuses on safety/performance vs efficacy/effectiveness.   🔷MDs have lower regulatory standards of evidence collection for placing them on the market, which ultimately hinders comparison between these health technologies, especially when they have the same goal and are targeted at the same disease and/or population. 🔷MDs are subject to clinical investigations rather than RCTs. They are assessed by demonstrating compliance with safety and performance requirements. An application to conduct a clinical trial is evaluated on its relevance, especially on whether the group of subjects participating in the clinical trial represent the population to be treated. 🔷Manufacturers of MDs may select not to resort to data from clinical investigations, relying on studies reported in the scientific literature showing equivalence to another approved device. Policy changes to promote an integrated evidence-based assessment system for a better allocation of resources in healthcare: ✔ Improve the MD classification system taking into account an HTA perspective. ✔ Match regulatory standards between devices and drugs for quality, safety, clinical efficacy and effectiveness: ✔ Improve health and safety through harmonised outcome generation for devices and drugs. ✔ Implement conditional coverage and post-approval evidence development. ✔ Implement high-quality registries for devices.   We add: 🔷 The development and adoption of evidence sandbox facilities and integrative evidence standards adopted by the data industry horizontally and all types of MD (s/w and h/w) will significantly improve MDR implementation in terms of clinical research and HTA pathways. This includes critical improvements in access to and the quality of evidence, and in the demonstration of impact pre-clinically to reduce market entry barriers for SMEs. This means innovative, safe, and effective technologies may be delivered to improve the outcomes that matter to patients in a timely manner. https://lnkd.in/dh2bUS6j #HTA #MDR #DigitalHealth #RegulatoryPolicy #EvidenceEcosystem #HealthcareInnovation #EU #AI #AIact

Nelson Advisors Partner Lloyd Price shares his thoughts on Clinical Trial diversity with PMLiVe - Pharmaceutical Market Europe It will take a modern alchemy to ensure the recent huge advances in science are translated into better prospects for patients around the globe as, despite life-changing potential, clinical trials struggle to escape their tainted past. Influential voices are now clamouring for cultural, conceptual and mechanical components to be blended in a furnace of intent to create a precious alloy that can deliver new therapies to broader and more relevant audiences. New clinical intelligence and understanding are propelling a marked increase in clinical trials this year – passing the 500,000 mark in April globally – yet many will fall short because they target a narrow band of patients selected, or more critically, available for their clinical trials. A lack of diversity is not just an economic misfire, it has become societally unacceptable and is now laden with the jeopardy of brand alienation that stretches way beyond niche condition areas and minorities. > Price sees a range of digital approaches that could energise trial enrolment among under-represented communities and boost retention. > But he cautions: “It’s important to remember that technology is a tool, and its success hinges on its implementation alongside culturally sensitive practices and a genuine commitment to diversity within the research community. > By making trials more accessible, culturally sensitive and transparent, technology can pave the way for more inclusive research and ultimately, better healthcare outcomes for everyone. ‘Results from a poll of 2,000 people reveal respondents have significant trust in vaccines and prescription drugs but less trust in pharma companies’ “Overall, technology has the potential to revolutionise clinical trial design and recruitment, leading to more diverse participation and ultimately, fairer and more generalisable medical research.” https://lnkd.in/eSgBcGHG

Nelson Advisors Partner Lloyd Price shares his thoughts on Clinical Trial diversity with PMLiVe - Pharmaceutical Market Europe It will take a modern alchemy to ensure the recent huge advances in science are translated into better prospects for patients around the globe as, despite life-changing potential, clinical trials struggle to escape their tainted past. Influential voices are now clamouring for cultural, conceptual and mechanical components to be blended in a furnace of intent to create a precious alloy that can deliver new therapies to broader and more relevant audiences. New clinical intelligence and understanding are propelling a marked increase in clinical trials this year – passing the 500,000 mark in April globally – yet many will fall short because they target a narrow band of patients selected, or more critically, available for their clinical trials. A lack of diversity is not just an economic misfire, it has become societally unacceptable and is now laden with the jeopardy of brand alienation that stretches way beyond niche condition areas and minorities. > Price sees a range of digital approaches that could energise trial enrolment among under-represented communities and boost retention. > But he cautions: “It’s important to remember that technology is a tool, and its success hinges on its implementation alongside culturally sensitive practices and a genuine commitment to diversity within the research community. > By making trials more accessible, culturally sensitive and transparent, technology can pave the way for more inclusive research and ultimately, better healthcare outcomes for everyone. ‘Results from a poll of 2,000 people reveal respondents have significant trust in vaccines and prescription drugs but less trust in pharma companies’ “Overall, technology has the potential to revolutionise clinical trial design and recruitment, leading to more diverse participation and ultimately, fairer and more generalisable medical research.” https://lnkd.in/e_bUfuhN

Clinical trials are impacted by a myriad of variables, making it critical to stay ahead of market dynamics.   We invite you to explore the below article to understand the evolving challenges and opportunities and their influences on clinical trials in 2024.   #ClinicalTrials #CLinicalTrialSupply #Akesa