VASCERN will hold a webinar on Tuesday November 23rd, 2021 from 5pm to 6pm (CET) entitled: Diagnostic and Management Pathway for Severe and/or Rare Infantile Hemangiomas with Dr. Andrea DIOCIAIUTI, VASCA WG member and dermatologist from Bambino Gesù Children’s Hospital, I.R.C.C.S, in Rome, Italy. This will be the the first of a series of four webinars exploring the Patient (Diagnostic and…
The European Medicines Agency (EMA) has announced the publication of a guideline on registry-based studies following its adoption by the EMA’s cross-Committees Task Force on registries and the Committee for Medicinal Products for Human Use (CHMP). The guideline provides pharmaceutical organisations with key methods and good regulatory practices on the planning and conduct of studies…
The Funders Constituent Committee of the International Rare Diseases Research Consortium (IRDiRC) has set up a Task Force to identify barriers and opportunities for the use of telehealth to improve diagnosis, care, and research experiences for rare disease patients – including technological, legal, cultural, linguistic, healthcare system, and patient/provider factors. This will be accomplished through survey and…
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) are launching a pilot project to support the repurposing of medicines. The aim of this initiative is to support not-for-profit organisations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication with the view to have this new use formally…
The Chan Zuckerberg Initiative (CZI) has launched a Request for Information (RFI): Patient Registry Data Interoperability as part of the Rare As One project, aimed at mapping data interoperability challenges and support ideas that will make it easier for patient communities to share data with researchers, and for researchers to fully leverage patient voices in…
The BioData World Congress 2021 organised by Terrapinn is Europe’s largest congress covering big data in pharmaceutical development and healthcare aimed to showcase innovation, demonstrate success and break through the obstacles and barriers to ensure that innovations in genomics and big data enter the clinic with speed and efficiency. This in-person event will take place in Basel, Switzerland over three days from November 2nd–4th.…
The World Orphan Drug Congress organised by Terrapinn is a global, multi-stakeholder orphan drugs & rare diseases meeting that aims to provide attendees with a one-stop progressive scientific and strategic solution to the orphan drugs industry. The in-person event will take place in Sitges (Barcelona), Spain over four days from November 15th – 18th. Patient groups can ask…
Yesterday marked the official launch of Screen4Care which is a new EU Research Project focussed on accelerating diagnosis for Rare Disease patients through genetic newborn screening and advanced analysis methods such as machine learning and Artificial Intelligence. The project will run for a period of five years with a total budget of EUR 25 million provided by the Innovative Medicines Initiative (IMI 2 JU), a joint undertaking…
The European Commission (EC) is inviting interested stakeholders to participate in the open consultation of the Strategic Research and Innovation Agenda (SRIA) of the European Partnership « Fostering an ERA for Health » (ERA4Health) by providing feedback through this online survey. The deadline for providing feedback to the Draft SRIA is October 29th, 2021. The draft SRIA…
The Duchenne Data Foundation, in collaboration with the World Duchenne Organization, is launching the 2021 edition of the Duchenne Patient Academy (DPA). In this intensive training session, patient advocates receive training and updates to build a strong base for current and future global advocacy. Applications are now open here. Duchenne Patient Academy works in partnership…