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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The Orphan Disease Center (ODC) at the University of Pennsylvania, in collaboration with the Loulou Foundation, is pleased to announce the 2024 CDKL5 Program of Excellence Pilot Grant Program. The deadline for the Letters of Interest (LOIs) is Friday, February 16, 2024, 5pm EST. CDKL5 Deficiency Disorder (CDD) is a monogenic, neurodevelopmental disorder characterized […]
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