Gain Insights & Stay Ahead – RAPS 2025 Global Regulatory Strategy Conference March 10-12, 2025
Mastering Global Regulatory Strategy for Success
Regulatory landscapes are evolving rapidly, making it crucial to stay ahead of emerging trends and challenges. The RAPS 2025 Global Regulatory Strategy Conference is the premier platform to gain the latest insights, practical tools, and networking opportunities to refine your global regulatory strategies.
Why Attend?
✔ Engage in Real-World Regulatory Solutions – Gain practical insights into complex regulatory challenges.
✔ Discover Cutting-Edge Tools & Software – Optimize regulatory processes and improve outcomes.
✔ Make Informed Strategic Decisions – Align your approach with global best practices.
✔ Network with Industry Experts – Exchange ideas, reinvigorate your passion, and drive innovation.
Join with Reguclin founder Dr. Siddharth Chachad at this prestigious event as he speaks about "Solutions Circle: Designing Clinical Trials for Complex and Branded Generics - Global Development Challenges and Mitigation Strategy". Talk to him about exploring innovative approaches, streamline processes, and enhance regulatory decision-making.
📅 Event Date: March 10–12, 2025
📍 Location: Hilton Baltimore, Maryland, USA
📢 Connect with ReguClin to discuss pre-clinical and clinical requirements. Our Smart Clinical Solutions leverage global product development strategies, patient-focused study designs, and execution quality oversight to ensure efficient, cost-effective drug development—helping bring life-changing medicines to patients faster and at an affordable price. Join us and take your regulatory expertise to the next level!
NEWS
FDA raises concerns over global cancer clinical trials, as outlined in the new draft guidance.
September 25, 2024
To assist clinical trial sponsors planning international cancer studies in securing FDA approval, the agency has issued draft guidance on 16th September 2024 on conducting multiregional clinical trials in oncology. This draft outlines factors to consider when choosing trial sites, analyzing study data, and integrating regional information regarding the disease and available treatments.
The FDA urges sponsors to undertake multiregional clinical trials but emphasizes that these trials must be conducted in the right context. It is crucial that the data generated from these trials is relevant to U.S. patients who may use the drug and aligns with current standards of cancer care.
The FDA noted that a key concern is the "known differences in the prevalence, presentation, causes, or severity of cancer" across different countries. These variations can impact the relevance of clinical trial data for U.S. patients and medical practices. When companies fail to account for these differences, the submitted data is likely unsuitable for supporting the regulatory decision.
Accordingly, in case of planning a single oncology multiregional clinical trial to support approval, the agency recommends conducting such trials with sites across continents rather than in a single country or geographical area. Sponsors should consider following factors while designing the clinical development programme.
- patient-related factors (e.g. genetic background)
- disease-related factors (e.g. prevalence of disease subtypes)
- health care system factors (e.g. access to health care, including specialized oncology care, cancer screening practices, availability and affordability of cancer treatments)
- socio-cultural factors (e.g. diet, cultural beliefs regarding alternative cancer therapies)
The agency also recommends the sponsors to include an estimation of regional treatment effects in their analysis plan, pre-specify the approach for this evaluation and explain the reasoning behind their chosen approach.
This draft guidance is open to public comments and suggestions until 18th November 2024.
To find out more, please refer to the link.
NEWS
FDA releases “Diversity Action Plans” to ensure the real world reflection in the clinical study populations.
June 29, 2024
On the 26th of June 2024, the U.S. Food and Drug Administration issued a draft guidance, “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies,” to assist medical product sponsors in submitting Diversity Action Plans to support certain clinical studies.
“Participants in clinical trials should be representative of the patients who will use the medical products,” said FDA Commissioner Robert M. Califf, M.D. “The agency’s draft guidance is an important step—and one of many ongoing efforts—to address the participation of underrepresented populations in clinical trials to help improve the data we have about patients who will use the medical products if approved.”
This guidance not only provides the template and the procedure for the submission of Diversity Action Plan to the FDA but also defines the evaluation criteria in cases where sponsors would want to apply for submission waiver. The scope for plan submission includes all the pivotal clinical studies for drugs, biologics; and even relevant device studies for determining safety, effectiveness and risk-benefit assessment.
Clearly, this guidance sets the foundation of equitable clinical trial practices through inclusion of diversity and designing of global studies with adequate representation of underrepresented groups; thereby ensuring the true reflection of the populations who will be treated in the real world.
The draft guidance is open to public comments and suggestions until 25th September 2024.
To find out more, please refer to the link.
Indian drug regulators strengthen safety norms further for vaccines
June 06, 2024
India’s Central Drugs Standard Control Organization recently revised the guidance document on the pharmacovigilance requirements for human vaccines. The draft Guidance for Industry on Pharmacovigilance requirements for Human Vaccines, Version 2.0 has been prepared in consultation with the Adverse Events Following Immunization (AEFI) Secretariat and Indian Pharmacopoeia Commission's (IPC) Pharmacovigilance Programme of India (PvPI) to align with the New Drugs and Clinical Trial Rules (NDCTR), 2019.
The revised document has been developed to provide the guidance to all the stakeholders including the Marketing Authorization Holders about Vaccine Safety Monitoring, Audits and Inspection; Post-marketing Surveillance, Post-marketing trials, Signal Detection and Management, Risk Management Plan (RMP) wherever applicable and Periodic submission of Risk Benefit Evaluation Report i.e., PSUR to the Licensing Authority.
The main focus of this guideline is to identify the risks, formulate the risk profile of a vaccine and its administration programme, design an appropriate pharmacovigilance plan to mitigate such risks and to explore the missing critical information which did not emerge during premarket Phase 1/2/3 trials and therefore safety profile had not been established.
The Marketing Authorisation Holders must have a robust Pharmacovigilance system in place that enhances the overall quality of the receipt, processing and reporting of AE/ AEFI while ensuring that accurate and complete information with respect to patient safety is provided to CDSCO throughout the life cycle of the product. Decisive causality assessment needs to be conducted after collation and thorough investigation of the AEFI cases. Comprehensive Periodic Safety Update Report (PSUR) must be prepared and submitted on timely basis. Based on the recommendation of the PSUR committee, the licensing authority DCGI will take appropriate regulatory action in accordance with Drugs & Cosmetics Act and Rules made thereunder, so as to monitor the safety and effectiveness of human vaccine in the market so as to safeguard the public health.
To find out more, please refer to the link.
EMA releases revised guidance for drug-device combinations
May 22, 2024
Recently EMA published a new guidance document for medical devices to be used in combination with the medicines. The document provides guidance to the marketing authorisation holders, applicants and notified bodies through some of the changes introduced by the medical devices and in-vitro diagnostics regulations.
These include:
- insights on integral drug-device combinations and their lifecycle management
- labelling requirements for medical devices co-packaged with medicinal products
- information on the consultation procedures for medical devices with ancillary medicinal substances and companion diagnostics
To find out more, please refer to the link below.
https://www.ema.europa.eu/en/news/medical-devices-new-guidance-industry-notified-bodies
EMA's safety review follows the FDA investigation for ATMPs
February 14, 2024
EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) review follows the FDA investigation on long-term safety monitoring of the Advanced therapy medicinal products (ATMPs).
ATMPs are the medicinal products containing recombinant genes, modified cells or tissues and these potentially offer new opportunities in the treatment of disease. While EMA has been the frontrunner in approving 26 cell and gene therapies and tissue-based products till date, the FDA has given green light only to 6 such therapies.
In November 2023, FDA announced the investigation of the reports of Serious Risk of T-cell Malignancy Following BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies. This was prompted by 20 reports of serious adverse events, including hospitalizations and deaths associated with these therapies since the approval of the first CAR-T therapy, Novartis’s Kymriah (tisagenlecleucel), in 2017. The safety concerns were reported in the clinical trials as well as in the FDA’s Adverse Event Reporting System (FAERS).
The US prescribing information for BCMA-directed and CD19-directed genetically modified autologous T cell immunotherapies includes the risk of secondary malignancies as a class warning. Further, the initial approvals of these products included postmarketing requirements (PMRs) to conduct 15-year long term follow-up observational safety studies to assess the long-term safety and the risk of secondary malignancies occurring after treatment.
FDA stated that although the overall benefits of these products continue to outweigh their potential risks for their approved uses, there is a clear need for regulatory oversight and life-long monitoring of the patients and clinical trial participants receiving treatment with these products. In case of a new malignancy being reported, the manufacturer should not only report the event to the FDA but also conduct thorough investigation by collecting and testing the patient samples for the presence of the Chimeric Antigen Receptor (CAR) transgene.
Recently, at the DIA's 2024 Global Pharmacovigilance and Risk Management Strategies Conference in Baltimore, Rob Sokolic, chief of the malignant hematology branch at FDA’s Center for Biologics Evaluation and Research (CBER), and Emil Cochino, scientific senior officer specializing in advanced therapies and haematological diseases at EMA, discussed potential pharmacovigilance strategies for monitoring the safety of these products.
Sokolic said “there are lot moving parts with ATMP”; and gene therapies can cause biochemical toxicities while cell therapies can cause biological toxicities. Hence, these products should be monitored for safety as long as they are active on the market.
Cochino spoke about the impact of manufacturing processes on the malignant potential of such products and the disease transmission risk with allogenic cells. He also stressed upon the need for 15 years follow-up safety reporting of ATMPs, similar to the FDA requirements.
ACT-EU - Developing the EU environment for better & faster clinical trials
June 25, 2023
“For the clinical trial environment to evolve with advances in regulation, methodologies, technology and science, there is a need for multi-stakeholder discussions to drive and support change”, reflected the concept paper released by the European Commission, European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) and adopted this year. Multistakeholder engagement and nine other priority actions were set forth as part of the ACT-EU (Accelerating Clinical Trials in the EU) workplan 2022 – 2026 with the key objective of making European Union as the attractive location for innovative clinical research.
Recent pandemic has clearly called for the need to change or rather innovate the way clinical trials are designed, regulated and executed to bring novel treatments on expedited basis to the patients. With this as the thought process, and in line with the recommendations of the European Medicines Regulatory Network (EMRN) strategy to 2025 and the European Commission’s Pharmaceutical Strategy for Europe, the ACT-EU workplan complements and supports the Clinical Trial Regulation (CTR) and the Clinical Trials Information System (CTIS) that became applicable on 31st January 2022.
On 22nd and 23rd June 2023, two-day event was held in Amsterdam to discuss different stakeholder perspectives and the priority areas on how to develop the EU environment for better and faster clinical trials. The discussion focussed on the priorities for the year 2023: CTR, non-commercial clinical trials and the multi-stakeholder platform. The multistakeholder platform will serve as a common platform between all the key stakeholders in clinical trials including patients, healthcare professionals (HCPs), sponsors, regulators, ethics committee members, health technology assessment (HTA) bodies, payers and policy makers to discuss all the aspects of clinical trials, understand different perspectives in plenary and ad hoc topic group meetings, enhance trust amongst different CT stakeholders and enable innovation through appropriate change in regulation(s), transparency of data and patient engagement. Additionally, other priority areas that were debated in the workshop included harmonisation of CT requirements across member states, innovative CT designs & methodologies and the need for consolidated scientific advice.
In case of in vitro diagnostics, current lack of defined processes for the conduct of performance studies, their submission and assessment was highly argued in the meeting indicating an urgent need for the implementation of IVDR & CTR interface.
To create an invigorated clinical trials environment, patient organisations strongly insisted on adequate representation of the patient groups in the platform including an ask for inclusiveness of special populations such as HIV patients. Better collaboration during clinical trials between patients, researchers and HCPs would also make the clinical trials less bureaucratic and more patient-centric and efficient. Especially, where the patient reported outcome measures are critical, the patients should be fully empowered with necessary tools required to capture the clinical trial data comprehensively. Also important is the publication of clinical trials data in CTIS with an access to the information of interests to the patients yet achieving right balance between required transparency and confidentiality requirements.
Ethics in clinical research is of paramount importance; and the rights, safety, dignity and well-being of the subjects have to be protected and must prevail at all times while carrying out clinical studies. Though safeguarding this is primarily governed by the national medical research ethic committees(MRECs), there has to be good alignment between the MRECs of the different member states in the EU. Establishing a common EU ethics review platform with the representatives from each member state and harmonisation across Europe will certainly help overcoming current ethical challenges associated with novel technologies, and public health emergency situations; and enable the trials to run much faster and yet deliver quality data. Whether there is a need for randomised clinical trial always, whether the study can be done in smaller sample sizes and whether preclinical programme is good enough especially in cases of unmet medical needs were raised as some of the questions that need to be addressed while implementing ethical considerations for the newer study designs and expediting the drug development process for faster reach to the patients.
With regards to the non-commercial clinical trials, academic sponsors expect ACT-EU to facilitate appropriate funding schemes, multinational infrastructure, harmonisation of national legislations and site agreements across EU, and clear methodological guidance. EMA has planned methodology workshop in Nov 2023 to discuss the existing guidance, gaps and the need for additional guidance.
In order to facilitate consistency between the scientific advice received from different member states, EU Innovation Network (EU IN) has launched phase 2 of the Simultaneous National Scientific Advice (SNSA) pilot. This is intended to be used in situations where an applicant wishes to obtain national scientific advice from more than one national competent authority (NCA) at the same time. Benefits include a simplified procedure with a single application point, a common meeting request and a briefing book, predictable procedural timelines agreed upon at the start and clearly documented position of each member state. Europe intends to encourage the medicinal product manufacturers to seek this consolidated advice bridging the pure national scientific advice and centralised European scientific advice procedures from EMA. Although, this advice is not legally binding, having preliminary opinion from the competent authorities is certainly beneficial. In order to prepare for the public health emergencies, the Emergency Task Force (ETF) has been established that provides expedited scientific advice support to the potential applicants, thereby ensuring faster access of medicines to the patients.
USFDA issues draft guidance on decentralized clinical trials
May 19, 2023
The popularity of decentralized clinical trials has clearly increased since the Covid pandemic, and more so because of increasing cost, complexity and time required for conducting conventional clinical trials to match regulatory expectations.
On the 3rd of May 2023, the FDA released its draft guidance providing recommendations for sponsors, investigators, and other stakeholders regarding the implementation of decentralized clinical trials (DCTs) for drugs, biological products, and devices. This guidance fulfils the requirement set forth in section 3606(a)(1) of the Food and Drug Omnibus Reform Act (FDORA). In this guidance, a DCT refers to a clinical trial where some or all of the trial-related activities occur at locations other than traditional study sites. So, the study activities may be carried out either at the home of study subject or at a local health care facility convenient for the study subjects.
DCTs allow for more diversity and inclusion of patient populations in clinical trial by leveraging digital health technologies, fewer in-person visits to the study sites and enhanced convenience to the patients and caregivers through remote trial outcome assessments via telehealth or in-home visits. While fully decentralized approach may be used for the investigational products that easy to administer, require simple outcome assessments, and have well-established safety profiles; hybrid DCT may be the way to go forward in case of drugs requiring complex medical assessments or administrations at the hospital sites. There may be a requirement of local health care facilities to provide trial-related services such as clinical laboratory services under the oversight of participating study investigators.
Given that some of the trial related activities would be conducted by the local health care providers (HCPs), there is a likelihood of variability in the approach followed by different HCPs, for example in the documentation of study outcomes. In order to address this aspect of variability, robust quality control must be in place with regular reviews for consistency and completeness of the study data. Sponsors should implement a comprehensive safety monitoring plan considering decentralized nature of the trial thereby ensuring that the safety data is adequately captured and addressed, and the well-being of the patient is maintained.
Software programs that are used in the DCTs are subject to 21 CFR part 11, and must ensure data reliability, security, privacy, and confidentiality.
This draft guidance is open for comments and suggestions until 1st of August 2023.
References
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FDA, Decentralized Clinical Trials for Drugs, Biological Products, and Devices, Draft Guidance, May 2023, https://www.fda.gov/media/167696/download.
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H.R. 2617; Food and Drug Omnibus Reform Act (FDORA), https://www.congress.gov/117/bills/hr2617/BILLS-117hr2617enr.pdf.