Case Study

    Rare and Orphan Diseases, Gene and Cell Therapy

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    In the 15 years from 2000 to 2015, the number of diseases with known molecular basis increased from ~1,000 to ~5,500, with close to 100 companies developing gene and cell therapies world-wide. Gene therapy now offers the hope of cure or meaningful symptom improvements in many previously untreatable indications; the body of data demonstrating the duration of its effectiveness is accumulating is increasing every year.

    Commercial work in rare diseases and treatments with no established payment models is a special focus of groupH. We recently conducted more than 100 interviews and ad-boards with senior US/Canada and EU payers, many of them in rare diseases, cell and gene therapy. Our goals were to landscape market access environments in different indications or to seek feedback and improve on Target Product Profiles and clinical and payer evidence development plans. groupH research and insights on this topic have been published in publications including In Vivo, Nature Biotech, the Journal of Market Access & Health Policy and Genes. The Alliance for Regenerative Medicines has invited us to deliver an educational webinar.

    To meet the needs of diverse client organisation sizes, stages of asset development and internal resource availability, groupH specialise in creating bespoke solutions. Ultimately, we see ourselves as an extension of your internal teams. This means we are available when you need us and are trusted to deliver because we know not just what you need but also how to present it so you can incorporate it seamlessly into your own work.

    We feel that actions speak louder than words – over 90% of our business is repeat business and our clients come back to us time and again.

    You went beyond the call of duty in your efforts and contributions to supporting us through this. Your incredibly calm and pragmatic approach was welcomed… Just did not want to let the opportunity pass by without more formally recognising your massive contribution to this very important project.”
    Top-5 global Pharma company

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