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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.

PMEA 2020: continuing to recognise excellence in improving patient outcomes

PMEA 2020: continuing to recognise excellence in improving patient outcomes

Excellence in Rare Diseases and Orphan Drugs categories. ... with support from Learner Adams Bones Ltd. 8. Excellence in Rare Diseases and Orphan Drugs.

Latest news

  • Building a brighter future for rare disease patients Building a brighter future for rare disease patients

    Before 1983 only thirty-eight orphan drugs gained regulatory approval while by 2014 that figure had reached 468 designations covering 373 drugs. ... The calibre of the shortlist in the Excellence in Rare Diseases, and Orphan Drugs category at the PMEA

  • AstraZeneca buys FDA priority review voucher from Sobi AstraZeneca buys FDA priority review voucher from Sobi

    The FDA’s PRV is used as an incentive for companies to invest in orphan drugs, by promising a cost reduction for the application fee for future products and a ... hundreds of millions for the most promising drugs.

  • The most annoying ‘F’ word in healthcare The most annoying ‘F’ word in healthcare

    We know that some of the largest employers in the US participate in these plans and we also know that fail first policies are being applied to the coverage of drugs ... In other words, these policies are not reserved for the orphan disease drugs or the

  • Europe urged to adapt faster to gene therapy challenges Europe urged to adapt faster to gene therapy challenges

    Existing models include conditional reimbursement, such as England’s Cancer Drugs Fund and Scotland’s fund for ultra-orphan drugs. ... Pay-for-performance deals for traditional drugs are also becoming established in Europe and the US, linking price

  • Sobi takes ownership of rare disease drug Gamifant for $518m Sobi takes ownership of rare disease drug Gamifant for $518m

    This gives companies investing in orphan drugs a cost reduction for the application fee for future products and shortens the review period, and can be used or sold on to other

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Approximately 81 fully matching, plus 92 partially matching documents found.

Latest Intelligence

  • Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England

    Comparison of orphan drug assessments from NICE versus the NHS. The question of how to adequately capture the holistic value of orphan drugs (ODs) for rare diseases is the subject of ... to drugs targeting ‘very rare’ diseases (typically ultra-orphan

  • Successful product commercialisation: what can we learn from the rare disease setting? Successful product commercialisation: what can we learn from the rare disease setting?

    Furthermore, orphan drugs are anticipated to account for 55% of the EU pipeline’s cumulative value in 2022. ... specialty or orphan drug status – all of which are typically associated with drugs targeting rare diseases.

  • Biopharma and orphan drugs Biopharma and orphan drugs

    Price concerns. Another argument in favour of the support given to the orphan drug industry is that rare disease drugs generate lower revenues than mass market medicines. ... According to a 2019 study, for example, the mean orphan drug cost per patient

  • Cortellis Drugs to Watch in 2020 report Cortellis Drugs to Watch in 2020 report

    The 11 drugs are almost universally accelerated in their development by orphan drug status or other designations intended to spur closer cooperation between regulators and drug developers with the intention of ... The 11 drugs identified collectively

  • Pharma funding and M&A in 2020 Pharma funding and M&A in 2020

    sectors of gene therapy, immune-oncology and orphan drugs therapeutic categories. ... By contrast, the larger pharmaceutical companies often have both the time and resources to see these drugs over the line.

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Approximately 14 fully matching, plus 43 partially matching documents found.

Latest appointments

  • New hires at Crescendo, Healx and Galecto New hires at Crescendo, Healx and Galecto

    Prior to joining Healx, he worked as CMO at Aparito and as therapeutic area head of orphan drugs at Mereo BioPharma. ... Before that, he lead global R&D at Almirall, which involved 500 staff and the development and approval of several new drugs in a

  • New hires at FibroGen, Saniona and Turbine New hires at FibroGen, Saniona and Turbine

    He has extensive experience of the regulatory, access, commercial and financial environment for developing orphan drugs in the US – a region which is directly relevant to Saniona’s next phase of ... The first members of the SAB bring 70 years of

  • Orchard Therapeutics appoints Mark Rothera as CEO Orchard Therapeutics appoints Mark Rothera as CEO

    market, and launching several orphan drugs.

  • XenoGesis appoints business development consultant XenoGesis appoints business development consultant

    business.”. Currently, Mogle serves as chief executive officer of RareMoon Consulting, a non-clinical development and regulatory affairs consulting firm specialising in orphan drugs she co-founded earlier this year. ... She has previously held

  • Hansa Medical strengthens management team Hansa Medical strengthens management team

    In his new role, van Troostwijk will be responsible for overseeing the firm's commercial assessments of its lead project IdeS, an orphan drug in development for organ rejection. ... IdeS.”. Van Troostwijk brings over 20 years of sales and marketing

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Approximately 0 fully matching, plus 12 partially matching documents found.

Latest from PMHub

More from PMHub
Approximately 5 fully matching, plus 18 partially matching documents found.

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