OSMR - The Romanian Medicines Serialisation Organisation’s Post

HOT OFF THE PRESS! What can FDA learn about #raredisease #drugdevelopment from the European Medicines Agency and vice versa? How can we as a rare disease community speed the time to market, better incorporate #patientperspectives, and more effectively leverage #patientregistries, #naturalhistory studies, and other #realworlddata #RWD and #realworldevidence? I was so honored to serve as a reviewer for this important study - a BIG THANK YOU to the many experts and dedicated staff who made this study possible! The new report from the NASEM Health and Medicine Division offers recommendations for enhancing rare disease drug development by improving engagement with people affected by rare diseases, advancing regulatory science, fostering collaboration between FDA and EMA. Access the report and supporting materials here:https://lnkd.in/e4pHJaxR

Rare diseases, such as #sicklecell disease and #thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Congress called on the U.S. Food and Drug Administration (FDA) to sponsor a National Academies study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the U.S. and the European Union. The newly released report and supporting materials can be accessed here: https://lnkd.in/ekHdYeKS #DrugDevelopment #RareDisease

Drug repurposing offers new hope for Rare Disease treatment. Our "Cracking the EMA Code" series finale explores this innovative approach, guiding you through the European landscape. Industry veteran Keith Williams, shares invaluable insights gleaned from his journey, from initial market appraisal and asset development to a successful company exit. This concise yet informative article is a fitting conclusion to our 7-part series. If you're developing a Rare Disease asset, Partner Rare's experienced team is ready to support your journey. Contact us today to discuss your specific needs and discover how we can help you achieve success. #rarediseases #drugrepurposing #EMA #marketaccess

Cracking the EMA Code: The Final Chapter on Drug Repurposing for Rare Diseases in Europe The final instalment of our insightful 7-part series, "Cracking the EMA Code," is now available. In this concluding piece, industry expert Keith Williams explores the strategic advantages of drug repurposing for Rare Disease treatments, offering a roadmap for navigating the complexities of #regulatory approval and #marketaccess. Discover how repurposing can: 💚 Accelerate the development timeline, bringing critical therapies to patients faster. 💚 Optimize resource allocation by significantly reducing development costs. 💚 Expand treatment options, improving the quality of life for individuals with Rare Diseases. Gain valuable insights from a seasoned expert who has consistently navigated the intricacies of drug development and commercialization in Europe. Elevate your understanding of the European Rare Disease landscape. Visit the Partner Rare website now to access the complete "Cracking the EMA Code" series and delve into the final article on drug repurposing. https://lnkd.in/e2PZnDJ9 Contact us for a complimentary discussion with Keith and our team of Rare Disease experts to discuss your specific needs. https://lnkd.in/gRJHVmnZ #rarediseases #drugrepurposing #EMA #patientcare #biotech

PN Update: Human medicines European public assessment report (EPAR): Nyxthracis (previously Obiltoxaximab SFL), obiltoxaximab, Date of authorisation: 18/11/2020, Revision: 3, Status: Withdrawn (authorisation)

Did you know almost 10 years ago the European Medicines Agency established the Patient Registries Initiative to enhance the use of registries in regulatory processes? At Cisiv Ltd, we are continually advocating for the use of patient registries. There is an untapped potential of patient registries in regulatory decision-making for medicinal products. Setting up a registry means you can systematically collect critical data on patients with specific diseases or conditions over time, offering valuable insights for regulatory decision-making, especially for rare diseases. A super interesting paper we recommend, that can be found below, written by Patricia McGettigan, Carla Alonso, Kelly Brown-Plueschke, Mireia Castillón Melero, Daniel Nogueras Zondag, Priya Bahri and Xavier Kurz discusses and highlights the potential and current underutilisation of patient registries in regulatory assessments of medicinal products. If you want to learn more about how to set up a registry or discuss your own registry, get in touch through https://lnkd.in/eH-gkw4w or at email us at info@cisiv.com #realworldevidence #patientregistries #EMA #healthcare

At a Rare Access Action Project (RAAP) recent event, Amy Comstock Rick explained the chilling effect that the Medicare Drug Price Negotiation Program’s narrow exemption for orphan drugs is having on #raredisease research. The orphan drug exclusion disincentivizes further research into additional diseases and indications. This disincentive is dampening investment in rare diseases, which will result in fewer #treatments for individuals with #rarediseases. This policy is contrary to the spirit of the 1983 bipartisan Orphan Drug Act, which created incentives for research and innovation in the development of new treatments for rare disease. Thankfully, U.S. Representatives Joyce and Nickel and U.S. Senators Carper and Barrasso have introduced the #ORPHANCuresAct. This bipartisan, bicameral legislation that would restore the research incentives of the #OrphanDrugAct and protect the innovation paramount to developing future treatments for people with rare diseases. Learn more about the #ORPHANCuresAct at: https://lnkd.in/g7j2YEeT

Great DIA session discussing the potential impact of the pharma review on orphan drugs development with Virginie HIVERT, Mariska Mulder, Matthias Heck and Victor Maertens and enhanced by the pertinent questions of the attendees who expressed concerns on the uncertainties surrounding some concepts such as ‘high unmet medical needs’(HUMN), and the qualifying criteria of ‘exceptional therapeutical advancement’ and ‘meaningful reduction in disease morbidity and mortality’; as well as querying on the economic consequences for an orphan drug in development that fails to qualify for the ‘HUMN’ label. Indeed the impact goes beyond a reduction of IP regulatory protection, it is likely to affect pricing decisions and HTA decisions as well as the level of investments in the EU. This will ultimately impact on the availability of new treatments for patients with rare disease in the EU. The latest opinions expressed by the ITRE Committee shows that the EP has heard the voice of the industry and understood the concerns. The position of the European Parliament as a whole should be available shortly ( after the plenary session scheduled on 10 April). However, this will not be the end but rather the beginning as the two proposals with EP amendments will then go to the Council ( and back again…). It is crucial that the legislator gets it right as we are all likely to have to live with the new package, once adopted, for quite a while. #DIA2024. #rarediseases #pharmalaw

The FDA's final guidance on drug development programs for rare diseases marks a significant moment in advancing treatments for rare diseases. It recognizes the unique challenges in developing new therapies and allows for more flexibility when developing drugs for severely debilitating or life-threatening rare diseases.   The guidelines also highlight the importance of including patients, caregivers, and advocates in the drug development process. This ensures that the experiences, perspectives, and needs of those affected by rare diseases are considered. The emphasis on patient involvement echoes our long-standing commitment to a patient-centric approach. We look forward to a future where patients will have a voice at every step of their therapeutic journey. Learn more via Regulatory Affairs Professionals Society (RAPS): https://lnkd.in/ggHpCvJd #FDAGuidance #PatientCentricCare #AnovoRxInnovation

PN Update: Human medicines European public assessment report (EPAR): Viekirax, ombitasvir,paritaprevir,ritonavir, Date of authorisation: 14/01/2015, Revision: 28, Status: Withdrawn (authorisation)