A patient's perspective: Living with a Rare Disease

Top Quote The continued research of orphan drugs is detrimental to the rare disease community. End Quote
  • (1888PressRelease) February 15, 2017 - In anticipation of Rare Disease Day, now is the ideal time to unite and raise awareness of the impact that rare diseases have on the lives of the patient and those who care for them amongst those who are unaware. Not forgetting the reason why the industry continually strives for optimal drug development - the patient.

    Taking the stage to present the keynote for the first time ever at SMi's Orphan Drugs conference will be, Carol McCudden, Patient Ambassador and Julie Research Officer from Ataxia UK.

    The opening address will introduce the challenges of the ataxias, a set of rare degenerative neurological conditions, covering the following areas:
    Patient experience of being a patient living with ataxia, her strategies for living with the condition, and her engagement as a patient with the NHS.
    An overview of the challenges for Ataxia UK, as a patient group working with a set of rare conditions with no treatments or cures.
    Their research strategy, partnerships with pharmaceutical companies and others, and the progress which has been made to date.

    Alastair Kent, Director, Genetic Alliance UK and Giorgio Iotti, Medical Scientific Manager - Rare Diseases Unit, Chiesi Farmaceutici have also shared their views on the costly yet essential endeavor that is critical to development of treatments for rare diseases.

    "...Current pricing and reimbursement mechanisms are coming under increasing pressure as the number of innovative therapies for rare diseases continues to grow. We will need to look carefully at how to make patient access equitable and sustainable in the medium to long term if progress is to be made and unmet medical needs satisfied..."
    Alastair Kent, Director, Genetic Alliance UK

    "I can understand that price is different in the various countries but for pharma companies the real nightmare is satisfying different requests raised by reimbursement authorities in each of the country in which the product is to be marketed. The second aspect is to design innovative payment schemes (like annualized installments) for reimbursement of advanced therapies in which single interventions are foreseen..."

    Giorgio Iotti, Medical Scientific Manger, Rare Diseases Unit, Chiesi Farmaceutici

    To read the full interviews, visit the download centre on www.orphandrugs-event.com/1888pr

    There is currently a 200 early bird discount for all bookings made before 28th February.
    Orphan Drugs and Rare Diseases Europe 2017

    15th - 16th May 2017
    Berlin, Germany

    Follow the conversation on Twitter and LinkedIn at #smiorphandrugseurope

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