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orphan medicines
This page shows the latest orphan medicines news and features for those working in and with pharma, biotech and healthcare.
Shionogi and F2G partner to develop antifungal treatment olorofim
It also covers azole-resistant strains, coccidioidomycosis and scedosporiosis (including lomentosporiosis). The treatment has already been granted orphan drug status by the European Medicines Agency (EMA) and orphan drug status, Qualified
Latest news
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Roche’s Evrysdi improves survival rates for infants with SMA
The drug was granted prime designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the US Food and Drug Administration in 2017.
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Building a brighter future for rare disease patients
Historically it was commercially challenging for sponsors to develop medicines to treat these so-called 'orphan' conditions. ... However, the Orphan Drug Act, passed in 1983, ushered in a new era for rare disease development.
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Who's calling the shots in European healthcare?
This has been driven by the sector’s shift towards developing more and more orphan medicines – these are often groundbreaking advances for patients, but also carry very high price tags which ... Natsis believes this is a pointer to the future
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Europe responding to gene therapy challenge, but picture remains fragmented
Among its provisions are plans for compulsory patient registries for all orphan products and medicines approved via conditional approval or exceptional circumstances. ... However Mr Iotti indicated that the administration of this was more complicated
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NICE and Canadian counterpart to offer joint advice service
Just last month NICE, CADTH and The US-based Institute for Clinical and Economic Review (ICER) announced they will team up on a medicines cost evaluation project. ... orphan medicines.
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Approximately 2 fully matching, plus 45 partially matching documents found.
Latest Intelligence
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Biopharma and orphan drugs
The success is clear: before the Regulation was introduced, only eight orphan-like medicines were available for patients. ... diseases. “In the area of orphan medicines, as the patient population is very small, the opportunities for the companies
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Shoring up the framework
Consequently pharmaceutical companies that hold marketing authorisations for centrally authorised medicines will be required to take certain steps. ... Marketing authorisation holders for medicines, including orphan drugs, must be established in the EU
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Brexit: Implications for the pharma industry
Similarly, orphan drug designations currently need to be held by an EU-based entity. ... If the UK ends up outside the EEA, it may, like other non-EEA countries, put in place reduced assessment processes for products categorised as orphan drugs by the
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A rare talent
Shire’s Sebastian Stachowiak on dealing with change and working for a fit-for-purpose orphan drug environment. ... The latter, says Sebastian, is “a very good first step, in terms of recognising that there is a difference when it comes to
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Market access improvements in Turkey
Almost the entire pharmaceutical market is made up of prescription medicines (93.8%) with 87% reimbursed. ... While welcoming the promise of a slight acceleration of review time with high priority medicines, industry is disappointed that orphan drugs and
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Approximately 1 fully matching, plus 11 partially matching documents found.
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Improving healthcare for rare disease patients
Accessed March 1, 2022. European Medicines Agency. Orphan designation: Overview. ... Accessed March 1, 2022. NHS England. NHS England announces new Innovative Medicines Fund to fast-track promising new drugs.
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Rare Disease Day – why it’s important to show your stripes
A standardised definition for rare diseases is currently lacking, with incidences typically defined by regional legislations developed to incentivise drug development (termed ‘orphan medicines/drugs’ by the FDA and EMA).
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Approximately 0 fully matching, plus 2 partially matching documents found.
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