Medicines and Healthcare products Regulatory Agency’s Post

In a forthcoming paper, members of ECNP, including EMA's CMO, discuss the knowledge gaps they believe must be resolved in order to enable the successful implementation of psychedelic drugs as medicines. https://lnkd.in/eMSq2Ni7

FDA/CDER’s Quantitative Medicine Center of Excellence Announces the Availability of New Resources The U.S. Food and Drug Administration’s (FDA’s) Center for Drug Evaluation and Research (CDER) Quantitative Medicine Center of Excellence (QM CoE) was founded on March 25, 2024 to foster integration of quantitative methods across CDER and help advance therapeutic medical product development and inform regulatory decision-making. To fulfill, in part, the mission of the QM CoE to: 1) spearhead QM-related policy development and best practices; 2) facilitate systematic outreach; and 3) coordinate QM education and training, the QM CoE is pleased to announce the availability of the following new resources: A quick reference Fact Sheet, providing a high-level overview of the QM CoE The recording and slides for the April 25, 2024 FDA public workshop entitled Streamlining Drug Development and Improving Public Health through Quantitative Medicine: An Introduction to the CDER Quantitative Medicine Center of Excellence A list of Frequently Asked Questions regarding the purpose, scope and upcoming activities of the QM CoE, as well as information on how to get involved An Educational Resources hub that features an assortment of educational content including free, web-based training modules on model-informed drug development (MIDD) developed by the FDA Office of Translational Sciences in collaboration with the Critical Path Institute (C-Path) and experts in the community. The series includes eight modules with 29 presentations and more than 10 hours of content on a variety of MIDD topics. #fda #clinicalpharmacology #centerofexcellence #resources #quantitativemedicine https://lnkd.in/giN6NxZh

How will the EU Joint Clinical Assessment (JCA) scoping process work? For each JCA, there will be a scoping process whereby the 27 member states have the opportunity to put forward their requirements by filling out a PICO (population, intervention, comparator, outcome) survey. Following the survey, the JCA assessor and co-assessor will consolidate all PICOs. These will then be validated by EU patients, clinical experts, and the Committee for Scientific Consistency and Quality (CSCQ). The finalised PICOs will be communicated to manufacturers approximately 3 months prior to dossier submission, which should be 45 days before expected Committee for Medical Products for Human Use (CHMP) opinion. Manufacturers must then complete their JCA dossier using all available clinical evidence to address the consolidated PICOs. It is expected that extensive evidence synthesis will be required to accommodate the potentially diverse PICOs. To address questions like this and other common queries about the EU JCA, our HTA experts have written this article: https://lnkd.in/e4dZTXZ4 #Europe #JCA #marketaccess #HTA #Pharma #Medtech

How will the EU Joint Clinical Assessment (JCA) scoping process work? For each JCA, there will be a scoping process whereby the 27 member states have the opportunity to put forward their requirements by filling out a PICO (population, intervention, comparator, outcome) survey. Following the survey, the JCA assessor and co-assessor will consolidate all PICOs. These will then be validated by EU patients, clinical experts, and the Committee for Scientific Consistency and Quality (CSCQ). The finalised PICOs will be communicated to manufacturers approximately 3 months prior to dossier submission, which should be 45 days before expected Committee for Medical Products for Human Use (CHMP) opinion. Manufacturers must then complete their JCA dossier using all available clinical evidence to address the consolidated PICOs. It is expected that extensive evidence synthesis will be required to accommodate the potentially diverse PICOs. To address questions like this and other common queries about the EU JCA, our HTA experts have written this article: https://lnkd.in/eKQnwGCw #Europe #JCA #marketaccess #HTA #Pharma #Medtech

The US Food and Drug Administration (FDA) has issued new draft guidance that highlights key considerations for researchers conducting clinical trials of psychedelic drugs for the treatment of medical conditions. The move, which was announced by the agency on June 23, marks the first time the FDA has issued guidance for investigating psychedelics, including psilocybin, LSD and MDMA, which have shown potential as possible treatments for serious mental health conditions.   Growing interest in the therapeutic potential of psychedelics has led to research evaluating the drugs as possible treatments for medical conditions, including depression, anxiety, substance misuse disorders and post-traumatic stress disorder (PTSD). Early studies have been promising, and the FDA has granted psilocybin and MDMA status as “breakthrough therapies,” a designation indicating that clinical evidence suggests a therapy shows a substantial improvement over standard treatments for a particular medical condition.   The FDA published a notice about the new draft guidance for psychedelic research in the Federal Register on June 26. The proposal is now in the midst of a 60-day public comment period for interested parties to provide input on the draft and submit suggestions for the agency to consider before adopting a final version of the guidelines.   Read the full article here at: https://lnkd.in/d63pwUVh

Regulation Plus+... Embedding a research culture: the evidence Encouraging a research-positive culture in health and care organisations is important to give patients wider access to clinical research, improving patient care and treatment options Lucy Chappell #OCTUK23 Will Prof Alejandro et al. #LoveWhatYouDo! #ABPIConf2024 Reflections on the Review – James O'Shaughnessy 1. Government immediately accepted all the recommendations and pledged significant funding for the ‘big 5’ • Progress is being made on MHRA/HRA + national contracting + data visibility 2. Focus on clinical trials and research in the NHS Workforce Plan • Should I have gone harder on protected time? 3. Research Reset and Recovery Group performance data is moving in positive direction but not on track to reach the “double-doubling” 4. Enthusiasm among all parties plus DAs but is there enough funding available to deliver UK-wide? 5. Design of CTANs will be critical so they truly become the transformational vehicle intended, not just more of the same 6. How to keep this topic front of mind for Ministers, given all the other issues they’re grappling with? #BeInspired #PassionAndPurpose #MakingADifferenceTogether

A concept paper on the creation of a reflection paper on a customized clinical approach in the development of biosimilars has been released by EMA. The necessity of reviewing the present specifications for specific clinical efficacy and safety studies for biosimilars is covered. The upcoming reflection paper will assess whether a similarity conclusion could be reached by combining clinical PK/PD trials with an analytical comparability exercise, thereby eliminating or modifying the need for extensive patient Phase III studies. A three-month consultation period is in place. https://lnkd.in/dcSt8ssB

How will the EU Joint Clinical Assessment (JCA) scoping process work? For each JCA, there will be a scoping process whereby the 27 member states have the opportunity to put forward their requirements by filling out a PICO (population, intervention, comparator, outcome) survey. Following the survey, the JCA assessor and co-assessor will consolidate all PICOs. These will then be validated by EU patients, clinical experts, and the Committee for Scientific Consistency and Quality (CSCQ). The finalised PICOs will be communicated to manufacturers approximately 3 months prior to dossier submission, which should be 45 days before expected Committee for Medical Products for Human Use (CHMP) opinion. Manufacturers must then complete their JCA dossier using all available clinical evidence to address the consolidated PICOs. It is expected that extensive evidence synthesis will be required to accommodate the potentially diverse PICOs. To address questions like this and other common queries about the EU JCA, our HTA experts have written this article: https://lnkd.in/epgK4qDe #Europe #JCA #marketaccess #HTA #Pharma #Medtech

Meeting highlights from the Committee for Medicinal Products for Human Use 27-30 May 2024 EMA's human medicines committee recommended 14 medicines for approval at its May 2024 meeting. The European Commission has now asked the Committee to further consider whether the data available on Translarna are sufficiently comprehensive to conclude on the medicine's benefit-risk balance, and whether additional real-world data brought to the attention of the Commission during its decision-making process may change the negative outcome previously reached by the CHMP. In addition, following the appellate judgment of the Court of Justice of the European Union of 14 March 2024 in Case C-291/22 P, EMA has decided to convene a new scientific advisory group on neurology for Translarna. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products at the time of approval to determine whether the information available to date allows maintaining the medicine's orphan status and granting the medicine ten years of market exclusivity. https://lnkd.in/dmcZvciE #PublicHealth #Europe #StayInformed #StayEmpowered

#Restlesslearners #Healthcareconsultant 🚨 Australia's clinical trials sector is on the rise, positioning the country as a prime location for such research endeavours. Following the recent Federal Budget announcement, there has been a surge in the utilisation of marketing and commercial strategies to highlight Australia's strengths in this area. The country boasts fast regulatory approvals and easy patient recruitment, supported by medical affairs strategies like direct clinician engagement with MSL on the field and promotional material that presents preliminary evidence without definitive outcomes or patient benefits. In today's age of misinformation, it is crucial to address the false ideas perpetuated by marketing efforts, especially in educating clinicians and patients about clinical trials. The risks associated with misinformation are significant, underscoring the importance of the *Eligibility criteria for trials, which should not be viewed merely as a template; rather, it requires a comprehensive understanding of the patient journey and the drug's mechanism of action to ensure patient suitability. Stay abreast of the dynamic clinical trial landscape by delving into the nuances of risk-benefit assessment for medical monitoring. Healthcareconsultant #Clinicaltrials #Oncology #PrecisionOncology #newstrategies #evidencedrivendecisions #Clinicaltrialmatching #rightpatient #righttime leveraging evidence to identify the right patients at the right time. Stay informed and engaged in the evolving landscape of clinical trials by checking out this insightful article: https://lnkd.in/g_UZiTBd