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orphan drugs

This page shows the latest orphan drugs news and features for those working in and with pharma, biotech and healthcare.

‘Inflexible’ NICE blocking access to rare disease drugs

‘Inflexible’ NICE blocking access to rare disease drugs

NICE has a Highly Specialised Technology (HST) appraisal, which has seen a number of innovative pricing and reimbursement deals done with companies to bring orphan and ultra-orphan drugs to NHS

Latest news

  • IQVIA sees market slowdown looming for pharma IQVIA sees market slowdown looming for pharma

    All told, 18 of the current top 20 drugs facing generic or biosimilar competition by 2023, says IQVIA. ... That also applies to new specialty, niche and orphan drugs which can also have significant costs.

  • Orphan drugs dominate FDA's record-breaking year Orphan drugs dominate FDA's record-breaking year

    drugs, driven on by new pharma and biotech science, and the FDA's determination to accelerate its review times. ... are treated.” Ultomiris need only be dosed every eight weeks, while current drugs for PNH have to be given every two weeks.

  • Better together when buying medicines? Taking stock of the BeNeLuxA initiative Better together when buying medicines? Taking stock of the BeNeLuxA initiative

    Many more documents have been produced on HTA. While there have not been joint HTAs for all six of the drugs looked at, there has been a joint HTA (and it ... It’s not just price that was subject to negotiation. In a way that has become almost business

  • PMEA 2018: celebrating excellence in improving patient outcomes PMEA 2018: celebrating excellence in improving patient outcomes

    AbbVie also took home two other awards, Excellence in Healthcare Professional Education Support Programmes (with support from Sciterion) and Excellence in Rare Diseases and Orphan Drugs (with support from Lucid). ... Meanwhile, for AbbVie’s and

  • Orphan drugs from Biogen and Amicus win UK Prix Galien Orphan drugs from Biogen and Amicus win UK Prix Galien

    There was a much warmer welcome at the awards, hosted by ValueBase, for the pharma and biotech companies with products on the shortlist, which was dominated by orphan medicines. ... All bar one of the finalists were medicines for orphan conditions, and

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Latest Intelligence

  • Launch trends 2019 Launch trends 2019

    A key trend is increasing evidence needs, ie, having greater evidence to ensure reimbursement of high prices, even for orphan drugs. ... 2018 saw the FDA approve a record number of drugs, as well as an increasing number in orphan disease areas.

  • Drug R&D

  • The sound of silence puts the squeeze on generics and biosimilars The sound of silence puts the squeeze on generics and biosimilars

    drugs - from Adalimumab to Rituximab - go off-patent and boost savings across eight European nations to 33.4bn over the next three years. ... What is surprising is that the whole of Europe is deeply concerned about the price of new medicines and how it

  • Europe vs the US: New drug product approvals Europe vs the US: New drug product approvals

    Altogether 39 orphan drugs were approved in the US during 2016 and 14 in the EU (a mix of NME, BLA, NAS or old substances with a new use for an ... We expect the strong overall trend for more orphan drugs submitted to and approved by the FDA and the EMA

  • Shoring up the framework Shoring up the framework

    This first document, set out in a Q&A format, covers information on company location, with respect to centralised procedures and certain other activities, including the location of orphan designation holders ... Marketing authorisation holders for

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