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🇪🇺🔬 A significant milestone for rare disease patients in the EU is on the trail as the European Parliament’s Committee on the Environment, Public Health, and Food Safety (ENVI) votes on crucial amendments to reform pharmaceutical legislation. Achieving broad consensus across European political groups, these amendments aim to enhance the development and accessibility of treatments for rare diseases within the EU. Key highlights of the approved amendments include: ✅ Maintaining market exclusivity for all orphan medicinal products at nine years, with an extension to eleven years for treatments addressing critical diseases. ✅ Refining the 'significant benefit' definition and clarifying the 'High Unmet Medical Needs' classification. ✅ Expanding the PRIME scheme to cover all orphan medicinal products, accelerating the development of medicines for unmet medical needs. ✅ Increasing incentives for academic research and mandating patient involvement in European Medicines Agency (EMA) consultations, particularly in defining guidelines for identifying unmet medical needs. ✅ Advocating for a comprehensive policy framework for rare diseases, regulatory data protection adjustments, and streamlined processes for advanced therapies. These amendments represent a significant step forward in addressing the unique challenges faced by rare disease patients, ensuring greater access to innovative treatments and improving their quality of life. Referrals https://lnkd.in/eU7sBi8X #SfRD #RareDiseases #EUHealth #PharmaceuticalReform #ENVICommittee

'The consultation follows the publication of the draft guideline for review in other parts of the world. The European Medicines Agency (EMA) began a consultation in June, and the US Food and Drug Administration requested feedback on the text earlier in July. Consultations are also underway in Brazil, China, Korea and Japan.   Swissmedic is accepting feedback on the draft until 30 August. Once the consultation phase is complete, the ICH working group will discuss responses received around the world and update the text as needed. ICH anticipates finalization of the document for implementation in local regulatory systems in May 2025.' There are a few consultations going on regarding real world evidence studies. I hope we can have some harmonised approach which will not make manufacturers jumping through loopholes all the time. There is a great need of guidance how to improve data collection in RWE studies and also how to deal with missing data especially considering that standard of care can vary between different regions. #Realworldevidence #clinicaltrials #ICH

From today's FDA Daily Digest Bulletin email: "CDER has added three examples of Bayesian statistical analysis plans to the CDER Center for Clinical Trial Innovation (C3TI)’s Bayesian Supplemental Analysis Demonstration (BSA) Project webpage. "The BSA demonstration project seeks to advance the understanding of Bayesian statistical methods and establish processes for integrating these approaches into clinical trial design in the future. "The BSA demonstration project is one of three projects in the C3TI Demonstration Program, which provides the opportunity to test, implement, and scale the integration of innovation into clinical trials through increased communication and interaction between CDER and sponsors of innovative clinical trials in certain project areas. Visit this webpage to view more information about submitting proposals for the demonstration program. "C3TI aims to enable and amplify innovative approaches to clinical trials that are designed to improve the efficiency and effectiveness of drug development." https://lnkd.in/eXVp7rni? Readers: Would you like to receive FDA updates in your inbox? Subscribe to FDA email updates here: https://lnkd.in/eu4v_wBr See also the FDA Industry page, which offers many helpful links, including Basics for Industry, a good jumping off place when starting your search for information: https://lnkd.in/eFeh75xt #FDA #CDER #C3TI #BayesianStatistics #ClinicalTrialDesign

In this Breaking Down Health Care conversation, John Hennessy, MBA, and Michael Kolodziej, MD, discuss the process of drug approvals and the role of the US Food and Drug Administration (FDA), including how and why the process takes so much time—and can add to the cost of treatments once they hit the market. "Until fairly recently, the FDA wasn't very tough on companies to complete their follow-up studies. But the political climate has changed, and now it's acknowledged that those follow-up studies have got to be done. And if the data doesn't confirm that efficacy that we thought was present, those drugs should go. We need to see more of that at the FDA. That, in fact, is the primary mechanism by which we safeguard the efficacy part of the FDA mandate," said Dr Kolodziej. Check out the full conversation here 👉 https://lnkd.in/eNkzTp45 #JournalOfClinicalPathways #DrugApprovals #HealthCareCosts #FDA #Medication

The South African Health Products Regulatory Authority (SAHPRA) Signs MoU With Rwanda Food And Drug Authority Areas of cooperation 1.SAHPRA and Rwanda FDA will cooperate in joint products reviews and inspections to enable efficient access to health products. 2. Both Health Agencies will collaborate in the area of mRNA vaccines regulatory oversight. We congratulate @SAHPRA CEO, Dr Boitumelo Semete-Makokotlela and Rwanda FDA Director-General, Professor Emile Bienvenu for cooperative partnership towards ensuring access to safe, quality, and effective health products in the respective countries #regulatoryaffairs #regulatorysubmissions #regulatorysubmissions #dossier #CMC

Africa CDC celebrates Rwanda Food and Drugs Authority and Agence sénégalaise de Réglementation pharmaceutique (ARP) regulatory milestones. https://lnkd.in/dru3Fyy9  #africacdc #rwandafda #senegal #arp #regulatory #milestones

For #medicaldevice manufacturers in the #EU. 🇪🇺 European Medicines Agency #EMA and #HMA published the latest developments from their ongoing efforts to advance the use of Real-World Data #RWD in supporting EU regulatory decisions. Building on the groundwork laid from September 2021 to February 2023, the report covers the period from 8 February 2023 to 7 February 2024, marking the second year of DARWIN EU. The report highlights the strides towards integrating RWE alongside traditional randomized controlled trials in regulatory decisions, in alignment with the European Medicines Regulatory Network (EMRN) and the EMA’s Regulatory Science Strategy to 2025. Key updates include: ✅ Inclusion of all studies conducted by EMA, including those related to vaccine safety, effectiveness, and public health emergencies.  ✅ Detailed review of the three RWE generation pathways: DARWIN EU network, in-house EMA studies, and commissioned research organizations.  ✅ Insights into the use of RWD by national competent authorities (NCA). EMA is committed to establishing a robust framework that will enable better integration of RWD/RWE into the development, authorization, and supervision of medicines by 2025, following the Big Data Task Force's priority recommendations. Contact us for qualified support in ensuring compliance to the regulatory requirements and if you need support around your clinical study design, benchmarking it or do the statistic for its evaluation?☞ meetus@taoexcellence.ch #HealthcareInnovation #RegulatoryScience #RealWorldEvidence #RWE #EMA #BigData #Pharmacovigilance #DARWINEU #DataDrivenHealthcare #EURegulations #HealthData #HealthTech #Clinicalaffairs #Publichealth #ClinicalTrials #Patientsafety 

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Did you know that pharmacovigilance helps ensure the safety of medications in vulnerable populations, such as children, the elderly, and individuals with certain medical conditions? https://Vigilarebp.com We are #Pharmacovigilance and Global #Regulatoryservices Providers From Case Studies to Aggregate reports and #Regulatory #Intelligence we are the right partner #Vigilarebp #vigilarebio #pharma #sevices #global #usfda #eu