industry pipeline. Home; Agenda Menu Toggle. Now in its 11th annual edition, the World Orphan Drug Congress … Co-hosted by: Ray Huml, Vice President, Medical & Scientific Strategy, Rare Diseases Consortium . > As the EU Regulation on Orphan Medicinal Products (OMPs) reaches its 20th Anniversary the Office of Health Economics has conducted an study (commissioned by EUCOPE) aimed at assessing how far it has incentivised investment in the R&D of OMPs and what would be the impact of changing core legislative elements. Bringing together a global gathering of 1,500 leaders in orphan drugs from 50 countries, World Orphan Drug Congress … OMPs generate lower and more volatile returns than medicinal products for common diseases. November 2, 2020 - November 5, 2020 « Krabbe Disease: Externally-Led Patient-Focused Drug Development Meeting; Live Webinar on 2020 World Cosmetic and Dermatology Conference » The World Orphan Drug Congress is coming to you, wherever you are. Important. > Our first asset is volaserib, a drug candidate in-licensed from Boehringer Ingelhelm, to be developed for paediatric rhabdomyosarcoma. 3:20 pm - Preparing for access beyond clinical data, 4:30 pm - Identifying and Accommodating for the Spectrum of Evidence in Evaluations of Orphan Drugs & Clinical Services for Rare Diseases, 5:00 pm – SPONSORED SPOTLIGHT PRESENTATION, 5:30 pm - Breaking down barriers to Patient Access: Bringing the stakeholders together, 2:20 pm - How patients can lead drug development: the case of the AKU Society, 2:50 pm - Accelerating Scientific development for rare disease from breakthrough technologies, 3:20 pm - Think of the Children- considerations working with children in clinical trials at home. This is "World Orphan Drug Congress 2020 - Chris Cameron's Presentation" by EVERSANA on Vimeo, the home for high quality videos and the people who… 12:30 pm - Plenary Keynote: What were the outcomes of the Paediatric and Orphan drug Regulations and what can be done to further stimulate the development of orphan medicines for paediatric use? Confirmed Speakers; … Due to the pandemic, we are looking to host a HYBRID Event, still the same congress but with a different set-up. The World Orphan Drug Congress is an award-winning event with an exhibition that has grown to become the largest and most established orphan drugs & rare diseases meeting of its kind across the globe. > How the SGC approach accelerates progress for all stakeholders in the drug discovery pipeline. Please, … Strategy, advocacy and partnering for the Orphan Drug industry We have a strong focus on re-purposing for paediatric indications either de-risked developmental compounds or already approved drugs. World Orphan Drug Congress Europe. El World Orphan Drug Congress 2020, l'esdeveniment de referència per a la comunitat científica i la indústria biofarmacèutica en malalties rares, se celebrarà del 2 al 5 de novembre a Barcelona. 4:30 pm - Precision Medicines and the challenges of Personalised Medicine. View Larger Image. EVERSANA was a proud sponsor of the 2020 virtual edition of World Orphan Drug Congress USA. - Opportunities to help optimise orphan paediatric medicines development, improve PIP efficiency, and avoid duplication of studies and unnecessary trials in children, will be discussed. Dr. Femida Gwadry-Sridhar, Chief Executive Officer & Founder, Pulse Infoframe, Moderator: Ian Hodgson, Head of Clinical Operations, Mereo BioPharma Group plc, Dr Rohit Batta, Chief Medical Officer, Vicore Pharma Holding AB, - How NICE evaluates drugs for rare diseases, - What are the challenges for the system and how can they be overcome, Sheela Upadhyaya, Associate Director - Highly Specialised Technologies, NICE. World Orphan Drug Congress Europe. 6:30 pm - Chairperson’s closing remarks, 7:30 – 9:30 pm - Networking Gala Dinner. Events - World Orphan Drug Congress USA 2020 - - - - - - World Orphan Drug Congress USA focusses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. Article. Monday, November 2nd | 10 – 13:00 CET. Agenda; Pre Conference Workshop; Day 1; Day 2; 2019 Recap; Sponsor / Exhibit Menu Toggle. Michael.hodge@terrapinn.com, To speak: The World Orphan Drug Congress is an award-winning event with an exhibition that has grown to become the largest and most established orphan drugs & rare diseases meeting of its kind across the globe. Orphan Drugs is a unique conference for the orphan drugs industry including discussions and networking with top tier individuals & organisations. It is a congress for strategy, advocacy, and partnering for the orphan drug industry. Skip to content . > Utilising mobile research nursing in paediatric trials. >Conclusion: policymakers would need to take into account the broader challenges related to development and access in the OMPs field, including high failure rates in the R&D process, challenges in the generation of real word evidence and the higher financial vulnerability of small and medium OMP-focused companies. - How are policymakers and regulators approach gene therapy products? Sponsors / Exhibitors; Media Partners; Become a Sponsor / Exhibitor; Speakers Menu Toggle. More than a third (35%) of granted MAs are held by OMP-focused developers. Over the last 11 years, the World Orphan Drug Congress has grown to become the largest and most establisehd orphan drugs & rare diseases meeting in Europe and this year will be … World Orphan Drug Congress USA focusses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. Main Menu. > Study approach combined different pieces of evidence collection and analysis to assess financial challenges of developing OMPs. -  Improving clinical development by new-age clinical trial design and recruitment. 2 novembre 2020 - 5 novembre 2020 Cet évènement est passé Stratégie, plaidoyer et partenariat pour l’industrie des médicaments orphelins World Orphan Drug Congress 2020 Panel : Optimiser les stratégies de développement clinique pour la thérapie génique. Bringing together a global gathering of 1,200 leaders in orphan drugs from 50 countries, World Orphan Drug Congress … Updates. About demomultisub This author has not yet filled in any details. > Pediatric oncology is an area of high unmet medical need. Rare Diseases Congress 2021 provides a head interdisciplinary stage for analysts to introduce the most recent research discoveries and portray developing advances, and … Add. Developing strong economic models in demonstrating the value of subsidizing patient healthcare costs, The need for accurate forecasting to ensure a successful market access, Optimizing a supply chain management system, Establishing a well-informed sales and marketing plan. Add. The evidence collected and results from data analyses were combined to produce a general assessment of the EU OMP Regulation and a set of messages for policy makers for future developments of the current Regulation. -  An effective framework for developing the delivery system for the next generation of medicine. Now in its 12th annual edition, the World Orphan Drug Congress Europe, together with the co-located Cell & Gene Therapy Congress, will continue to bring experts … This year’s event featured over 260 presentations covering all aspects of orphan drug development and rare disease research. 5:00 pm - Inside Out: Organelles Pathobiology and Computational Modelling Discovery for Rare and Ultrarare Diseases, 5:30 pm - SPONSORED SPOTLIGHT PRESENTATION. Experiences when communicating with healthcare professionals, Social and economic implications of living with rare disease. 2:50 pm - Getting Ready: initiatives for gaining better access to ATMPs in Europe. > Oncoheroes was set up to fill this gap. Review . August 24 - August 26 2020 - Maryland, USA. What impact does rare disease have to the patient and caregiver? Strategy, advocacy and partnering for the Orphan Drug industry -  How to find the right patient and design a collaborative clinical trial? The financial performance of OMP-focused companies is characterised by financial instability. > Giving the child control over their participation in the trial. > To study the financial challenges OMP innovators face, we followed a twofold approach: analysis of financial indicators and Net Present Value (NPV) modelling. Michael Hodge Dr. Femida Gwadry-Sridhar, Chief Executive Officer & Founder, Pulse Infoframe. > Partnering with patient groups drives focus and innovation, whilst Open Science outputs drive maximum impact globally. The World Orphan Drug Congress is an award-winning event with an exhibition that has grown to become the largest and most established orphan drugs & rare diseases meeting of its kind across the globe. Dr Cesare Spadoni, Co-founder & Chief Operating Officer, Oncoheroes Biosciences Inc. -How are this advance therapy fundamentally changing the way rare diseases patients are treated? Fox Foundation in GBA1 Parkinson) support those research avenues, > Use of antisense oligonucleotide-induced exon skipping to restore the transcript reading frame, > Exploitation of AAV-mediated gene addition therapy, > Therapeutic potential of each and update on stage of development. World Orphan Drug Congress USA 2020 August 24, 2020 - August 26, 2020 What is the World Orphan Drug Congress USA? The congress opens doors to fantastic opportunities to form lucrative partnerships and source new ground-breaking solutions. It’s the 13th in the series of our Flagship tri-annual Orphan Drugs & Rare Diseases event. 4:30 pm - The Importance of Patient Networks and Advocacy Groups in Designing of Clinical Trials and Patient Recruitment and the importance of patient reported outcomes of clinical trials and their perception. -  Developing strong economic models in demonstrating the value of subsidizing patient healthcare costs, -  Establishing a well-informed sales and marketing plan, -  The need for accurate forecasting to ensure successful market access, -  Optimizing a supply chain management system, Moderator: Dr Rohit Batta, Chief Medical Officer, Vicore Pharma Holding AB, Panellist: Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross, Joanne M. Hackett, Chief Commercial Officer, Genomics England, Robert Donnell, Head of Business Development, Durbin PLC, part of uniphar group. -  Making manufacturing and commercialization viable, -  Reimbursement models for gene therapies, Moderator: Jonathan Appleby, Chief Scientific Officer, Cell and Gene Therapy Catapult, Panellist: Paolo Morgese, EU Director Market Access, Alliance for Regenerative Medicines, Josie Godfrey, Director, JG Zebra Consulting, Project Director, Duchenne UK's Project Hercules, Karen Matthews, Business Development Manager, NIHR, Dr. Didier Caizergues, Head, Regulatory Affairs Department, GENETHON, Steven Zimmer, CEO, EPICOMBI THERAPEUTICS LTD. - More can be done to address the demand for therapeutic options and cures to treat rare childhood conditions. Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases 2020 Europe. > Volv Global’s inTrigue AI algorithms identify diagnostic signals from patient medical record data and help patients receive treatment earlier by enabling earlier diagnosis, particularly of rare and difficult to detect or diagnose diseases. On behalf of the operating committee, we are pleased to announce that the 2nd Global Congress on Rare Diseases and Orphan Drugs will be held during September 20-21, 2021. World Orphan Drug Congress USA focuses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. Orphan Drugs is a unique conference for the orphan drugs industry including discussions and networking with top tier individuals & organisations. How can governments and orphan drug companies help support each other’s initiative to provide access to patients? To sponsor or exhibit: >   Understanding the patients’ needs on a “Rare Clinical Trial. The incentives of the EU OMP Regulation are crucial to maintain a healthy R&D pipeline of and investment on new OMPs. About: The World Orphan Drug Congress Europe 2020 is an award-winning event with an exhibition that has grown to become the largest and most established orphan drugs & rare diseases meeting of its kind across the globe. Schedule a Meeting. Now in its 11th annual edition, the World Orphan Drug Congress Europe, together with the co-located Cell & Gene Therapy Congress, will continue to bring experts from the whole value chain under one roof, where you get to meet decision makers in the areas that are most applicable for your business offering. ECRD attendees spent the course of the conference laying the foundation of these preparations and all of you are invited to continue your involvement in Rare 2030 throughout the year! Orphan Drugs is a unique conference for the orphan drugs industry including discussions and networking with top tier individuals & organisations. Bringing together a global gathering of 1,500 leaders in orphan drugs from 50 countries, World Orphan Drug Congress USA will feature over 260 presentations covering all aspects of orphan drug development and rare disease research. Orphan Drugs America Attend Orphan Drugs 2020 Orphan Drugs & Rare Diseases Global Congress 2020 Americas will provide a unique platform for the convergence of stakeholders in the orphan drugs industry to discuss and network with top tier government, hospitals, pharmaceuticals, biopharmaceuticals, non-profit organizations, orphan drugs developers as … This congress will provide you with a … - The implementation of both the Paediatric and the Orphan Regulations can be further improved through pragmatic changes and collaborative research programmes. RARE DISEASES 2021. Skip to content . > Case studies of SGC partnerships with patient groups and medical charities have advanced drug discovery. Value also comes from, cost-benefit to the healthcare system and community, > However, with high value and high prices, we can easily tip the balance to become unaffordable & unsustainable - for patient, payors and insurance/ government reimbursement, to work together towards a sustainable balance between patient needs, payor, >   The challenges in getting reimbursement decisions in rare diseasesÂ, >   A case study in Duchenne Muscular DystrophyÂ, >   How collaboration can help drive better data, and better outcomes for patients, >   Understanding evidentiary needs & considerations, >   Recognizing the need for variety in and working effectively with different levels of evidence in these evaluations, >   Introducing seminal frameworks for evaluations, >   Establishing the differences between clinical investigations based on incidences of disease, >   Taking cues & guidance from regulatory bodies, >   Level setting a few of the main alternative & emerging options within the hierarchy of evidence, >   International comparison of appraisal processes Â, >   Optimizing deliberative processes to determine valueÂ, >   Post appraisal determination of value, Innovations, Discovery & Clinical Development, >   Setting up an ultra rare disease clinical trial with a patient group, >   Recruiting patients for clinical trials, >   Positive results from an off label study of nitisinone for AKU and from an international phase 3 clinical trial. The World Orphan Drug Congress is an award-winning event with an exhibition that has grown to become the largest and most established orphan drugs & rare diseases meeting of its kind across the globe. 14:15 pm - Attend any of the 4 Topic Focused Sessions. So far demomultisub has created 0 blog entries. 08:00 am - Conference Registration and Continental Breakfast. > The EU OMP Regulation has successfully incentivised companies to invest in the development of OMPs, >The EU OMP Regulation is crucial to manage the financial unpredictability of OMPs development. In parallel, Oncoheroes is in-licensing promising clinic-ready candidates from academia and the. Agenda; Pre Conference Workshop; Day 1; Day 2; 2019 Recap; Sponsor / Exhibit Menu Toggle. > Which are the best practices and challenges for accessing ATMPs in major European countries? Full content is still available on … nadia.konneh@terrapinn.com. WODC 2020. > ATMPs and payer landscape, what should we expect in the years to come? August 24th-26th, 2020 Oxon Hill, Maryland . 2:50 pm - Overcoming the fourth hurdle: The story of the award-winning Project Hercules and how a tiny patient organisation brought together nine pharmaceutical companies to address the challenge of reimbursement in rare diseases. World Orphan Drug Congress USA 2020. World Orphan Drug Congress 2020. World Orphan Drugs Congress, New York, New York. How to find the right patient and design a collaborative clinical trial? ECRD 2020 provided the opportunity to co-design policy options today that can lead to better conditions for people living with rare diseases for the years ahead. 6:00 pm - Development of gene therapies for Duchenne muscular dystrophies: From lab bench to bedside. Skip to content . El congrés ofereix l'oportunitat de trobar contactes per fer front als reptes comercials, científics, assajos clínics, regulació, market access i preus. Use of Novel AI/ML Tools to Support Patient Identification; Perspectives From the Syneos Health Rare Disease Consortium. Now in its 11th annual edition, the World Orphan Drug Congress … The World Orphan Drug Congress USA is a digital media and events platform for the rare disease community. It’s the 12th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs. 2:50 pm - INPDR – A disease based registry driven by “Patients, Partnership and Progress", 3:20 pm - Sponsored Spotlight Presentation, 3:50 pm Afternoon Networking & Refreshment Break, 4:30 pm - Changing the world - one genome at a time, 5:00 pm - Meaningful Engagement with Patients and their Representatives, 5:30 pm - Developing new treatments for rare childhood cancers: Oncoheroes Biosciences' strategy, 6:00 pm - Open Science Approaches to Accelerating Drug Discovery for Rare Diseases. Sponsors / Exhibitors; Media Partners; Become a Sponsor / Exhibitor; Speakers … Agenda; Pre Conference Workshop; Day 1; Day 2; 2019 Recap; Sponsor / Exhibit Menu Toggle. World Orphan Drug Congress EU 2020. The World Orphan Drug Congress provides you with a one stop progressive scientific and strategic solution to the orphan drugs industry. >   Genotype-specific medicines have been developed that are disease-modifying for an increasing variety of mutations.  Initially 5% of the population (2012) and by 2020 we anticipate will be for 90%. Nearly half (46%) are held by broader portfolio companies. Pluristem Therapeutics Inc. MATAM Advanced Technology Park Building #5 Haifa 3508409, Israel Israel +972-74-710-8600 U.S. +1-347-973-2098 Germany +49-30-2215-0786 Main Menu. 2:20 pm - Is value helpful, and not, to patients and advocates in fighting for broad sustainable access to medicines? 249 likes. It’s the 12th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs. Home; Agenda Menu Toggle. Main Menu. Sponsors / Exhibitors; Media Partners; Become a Sponsor / Exhibitor; Speakers … 08:00 am - Conference Registration and Continental Breakfast, 08:20 am - Chairperson’s Welcome and Opening Remarks, 08:30 am - Opening Keynote Panel Discussion: Patient and Caregiver Perspective: Living with Rare, 09:15 am - Keynote Panel Discussion: Commercialization of Orphan Drugs: Overcoming unique challenges, 09:15 am - Plenary Keynote: Challenges in Evaluating Drugs for Rare Diseases, 09:45 AM - Keynote Panel Discussion: Commercialization of Orphan Drugs: Overcoming unique challenges, 10:30 am - Morning Networking & Refreshment Break, 11:15 am - Keynote Panel Discussion: Cell and gene therapy for rare disease: Solutions for realizing the potential of the next generation of medicine, 12:00 pm - Plenary Keynote: SPONSORED SPOTLIGHT. We are a biotech 100% focused on paediatric oncology drug development, > We are building our strategy on a strong connection with the key opinion leaders in the field and with the major international clinical networks.Â, > The company's activities spans the entire drug discovery and development spectrum. Changes in the EU OMP regulation would impact financial returns generated by OMP investment, could significantly decrease the number of developed OMPs and in some cases could threaten the financial sustainability of OMP-focused companies (35% of the marketed products). 249 likes. >   There are a number of different mutations in the gene that cause cystic fibrosis. Home; Agenda Menu Toggle. Wing-Yun Cheung > The development of these products is worldwide or at least European, we will highlight some of the problems met regarding missing of harmonization or the design of clinical trials. World Orphan Drug Congress Europe. Skip to content . Paradigm Global Events (PGE) is an Independent Business Information provider, specializing in Business Intelligent Reports and analysis in Solution Based Networking Events to decision makers. Does the European Regulation tackle them? > High quality open science protein, molecule and assay tools as a critical requirement in the post-genome era. 2020 World Orphan Drug Congress. World Orphan Drug Congress USA focuses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. World Orphan Drug Congress USA focusses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. wing-yun.cheung@terrapinn.com, Nadia Konneh Sponsors / Exhibitors; Media Partners; Become a Sponsor / Exhibitor; Speakers … 5:00 pm – AI in rare diseases and personalised medicines, 5:30 pm - Engaging Early and Often: How to Design Patient Centric Trials, 6:00 pm - The role of patient organisations in developing innovative precision medicine strategies. >   Do the clinical endpoints capture the real benefits? > ARM recommendations for removing structural barriers. Featured Presentations . value to life and even small real-world benefits. Thomas Kühler, Head Regulatory Science & Policy, EU/AMEE, Sanofi, Commercialization, Current Trends & Collaborations. >   This talk would touch upon the need for enhanced genomic understanding to tackle some of the rare and orphan diseases; >   how Genomics England is well placed to assist with this (a data base of 120,000+ whole genomes with linked clinical data);Â, >   how we are moving to precision clinical trials in the UK to push this agenda forward, > Personal perspective of supporting a child with a rare disease, > Developing a patient organisation and working in partnership with clinicians and pharmaceutical companies – the journey of Alström Syndrome UK, > Patient involvement throughout the life cycle of drug development – CIOMS XI, Patient Involvement in the Development & Safe Use of Medicines, > Understanding what is important to patients and their families - ‘People not subjects’. We are enabling pharma, biotechs, governments, payers, patient advocates and more to discuss new ideas and developments, with exclusive content across both our digital and live platforms in 2021. 250 likes. Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2020 Americas. Main Menu. > A worldwide, purpose built, anonymised, rare disease, web-based registry, > Developed by the NP Community, run by professionals, > Together: Advocacy Groups, Clinicians, Researchers and Industry. World Orphan Drug Congress 2020 November 2 - 5, 2020 - ES Terrapinn. The conference will provide … Day 1: Tuesday 20th October 2020. > Learn about the most effective ways to include patients and advocates in designing trials, > Discover optimal timing and important milestones, > Understand the need to continue engagement even after the trial is complete, > A new strategy for research into rare diseases and the roles that patient organisations play, > The unmet need of precision medicine for scleroderma and other rare orphan conditions, > Working together with industry to enable development and access to therapies. World Orphan Drug Congress USA 2020 - 29 Apr - 01 May, 2020, Gaylord National Resort & Convention Center, National Harbor, MD, United States (12669) World Orphan Drug Congress USA 2019 - 10-12 Apr 2019, Gaylord National Resort & Convention Center, National Harbor, MD, United States (23743) Reminders. 08:20 am - Chairperson’s Welcome and Opening Remarks. >   Challenges remain in terms of understanding the variation in individual responses and managing expectations of the CF community, > Taking the example of lysosomal enzyme related diseases, I will present evidences on how a genetic, molecular and pathobiological understanding of rare and ultrarare diseases discovery and product development, > Evidences of how an organelles centric discovery can identify and lead to development of novel approaches for genetic defined neurodegenerative diseases and why patient centric associations and foundations (such as the Michael J. Home; Agenda Menu Toggle. 2:20 pm - Orphan Drugs Gene and Cell therapies product: The difficulties encountered and the supports proposed by the EC, EMA and National Agencies for the development and registration. What is it like living with a rare disease? Get your brand, your message and your sales people in front of your customers. Compound annual growth rate of 15% off granted marketing authorisations (MA) for the period 2000-2018. > What mobile research nursing is all about. New Virtual Agenda We are bringing you The World Orphan Drug Congress to you virtually. 2:20 pm – Economic and Financial Challenges of Developing OMPs. The regulators have developed some supports and expedited programs to  promote the development of these orphan drugs .We will describe this during our presentation. 899 likes. Bringing together a global gathering of 1,200 leaders in orphan drugs from 50 countries, World Orphan Drug Congress USA will feature over 135 presentations covering all aspects of orphan drug … >   Communication with the patient community, >   Design with reimbursement in mind not just approval, >   Patient Reported Outcomes and the need to move to a validated model, >   Design for the failure as well as the success. Agenda; 2019 Recap; Sponsor / Exhibit Menu Toggle. riette.dejager@terrapinn.com Phone:+44 207 092 1061 . As dedicated advocates of the rare disease community, EVERSANA is on a mission to advance the commercialization of orphan drugs … Strategy, advocacy and partnering for the Orphan Drug industry Orphan Drugs is a unique conference for the orphan drugs industry including discussions and networking with top tier individuals & organisations. Orphan Drugs & Rare Diseases Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases 2020 Europe. 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