The Committee for Orphan Medicinal Products (COMP) organized their Plenary Meeting last week (13-15 February), that was chaired by Regulatory Scientific Committee (RSC) Vice Chair, Violeta Stoyanova-Beninska, and Armando Magrelli. Among the topics discussed were the new applications for orphan medicinal product designation (including amendments of existing designations), requests for protocol assistance with significant benefit […]
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Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. Since its creation in 2008, Rare Disease Day has played a critical part in building an international rare disease community that is multi-disease, global, and […]
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A roundtable of EU and national policymakers, industry experts and patients will gather together to discuss the best approach to define unmet medical needs and Belgium’s multi-criteria approach. Around 36 million Europeans suffer from rare diseases and 95% of these diseases lack a specific treatment. Moreover, medicine development is lagging due to a risky and […]
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The Canadian Organization for Rare Disorders (CORD) is very excited to be celebrating this once-in-four- years February 29th Rare Disease Day in Ottawa (Canada) on February 28-29, 2024. A panel, “Canadian Pathway to Rare Disease Drug Access”, is also scheduled on February 28 at 10:45 AM – 12:00 PM (local Ottawa time). This year, there […]
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Health Technology Assessment International (HTAi) in late 2023 approved the set up of the HTAi Rare Diseases Interest Group (RDIG), the role of the group is a service to members of HTAi who have interest in HTA of health technologies for rare diseases. This Interest Group is dedicated to sharing good practices in evidence generation […]
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The European Joint Programme on Rare Diseases, together with the Companies Constituent Committee (CCC) member EFPIA, is organizing the training webinar: “Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?” that will be held online on 26 January 2024 […]
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Project CASK, in collaboration with Orphan Disease Center, is seeking proposals to advance research that supports therapeutic development for CASK gene disorders. The project will consider applications that will make a significant impact on the CASK community and the prospects for accelerating therapeutic development in one of two categories: 1) Grants to expand the study […]
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The European Conference on Rare Diseases & Orphan products (ECRD) is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place. This 12th edition will […]
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The Innovative Health Initiative (IHI) has now launched IHI – call 6 and IHI – call 7. IHI – call 6 is a two-stage call for proposals with the following topics: Topic 1: Support healthcare system resilience through a focus on persistency in the treatment of chronic diseases Topic 2: Development of evidence based practical […]
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Join us for this 2-day multi-stakeholder event on 29-30 January covering the day’s most pressing ethical issues in non-trial preapproval access at CUPA Con 2024! In-person attendees will be able to take advantage of interactive workshops, various networking opportunities, a cocktail reception, and other perks. For those unable to attend in-person, tickets providing access to […]
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