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Orphan drugs and rare diseases

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

Malthus’ orphans

Malthus’ orphans

By and large, rare diseases and orphan drugs don’t lend themselves to the former or the latter. ... Failure to apply some cold-hearted cost-benefit analysis to orphan drugs, rare diseases and other niches would inevitably lead to the ‘lower

Latest news

  • Lucid picks up rare disease and orphan drugs trophy at PMEA 2017 Lucid picks up rare disease and orphan drugs trophy at PMEA 2017

    Lucid picks up rare disease and orphan drugs trophy at PMEA 2017. ... Judges praised the AbbVie project for its impressive reach. Lucid picked up the award for Excellence in Rare Diseases and Orphan Drugs at this year’s PMEA Awards, which were hosted

  • Celgene scoops Socially Responsible Initiative prize at PMEA 2016 Celgene scoops Socially Responsible Initiative prize at PMEA 2016

    The objectives were laudable and the implementation made a big difference to many children's lives.”. ... Celgene was also a finalist with '75 Not Out' in the Rare Diseases and Orphan Drugs group.

  • Shire completes $32bn Baxalta merger Shire completes $32bn Baxalta merger

    Shire has finalised its $32bn merger with Baxalta, creating one of the world's leading firms specialising in rare diseases and conditions. ... Shire now has over 50 rare diseases programmes in various stages of clinical development and, together with its

  • Novel drugs and rare diseases drive US prescription drug spend Novel drugs and rare diseases drive US prescription drug spend

    Novel drugs and rare diseases drive US prescription drug spend. But increased competition and cost-cutting measures forced price concessions. ... It notes that there were 43 new active substances launched in the US last year, with a high proportion of

  • Wakix backed for European approval in narcolepsy Wakix backed for European approval in narcolepsy

    Wakix backed for European approval in narcolepsy. Bioprojet Pharma’ s orphan drug given a positive opinion by CHMP. ... Narcolepsy is a rare, long-term sleep disorder, which affects the brain's ability to regulate the normal sleep-wake cycle.

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Latest Intelligence

  • A rare talent A rare talent

    Change has also been the order of the day at Shire, with the Dublin-headquartered company taking strides towards future growth over the last couple of years, with rare diseases and ... A rare challenge. That knowledge is currently focused on bringing new

  • Reaching new heights Reaching new heights

    for rare diseases and technology-enabled changes in care that can harness big data to better inform decisions, help drive patient behaviour changes and improve outcomes. ... biologics. Patients will have greater access to breakthrough therapies and

  • Applying real-world evidence to orphan drugs Applying real-world evidence to orphan drugs

    This is particularly challenging in the area of rare diseases, in which there have been substantial advances, largely due to our increased knowledge of the genomic and biological makeup of the ... In Europe, the EU Committee of Experts on Rare Diseases

  • Another lost decade for research? Another lost decade for research?

    Another lost decade for research? What does the patent landscape look like for antibiotics, vaccines and orphan drugs? ... The annual number of patent families filed in the field of rare diseases is far higher than for vaccines or antibiotics.

  • The distressing impact of rare diseases The distressing impact of rare diseases

    What role can pharma play? The pharmaceutical industry is undergoing a transformative approach to rare diseases, as orphan drugs address a high unmet medical need and have been proven to be ... This article was originally published in the PME supplement

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