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Ashfield has a breadth of experience with orphan drugs and we are passionate about helping to improve the lives of people living with rare diseases. Last week we were delighted to attend the ‘Orphan Drugs for Rare Diseases Summit’ 22-23 March in Berlin.

The event was well attended by healthcare experts and industry leaders, all keen to share learnings and experiences in the field of rare diseases. Through a series of presentations, the event explored the challenges of rare diseases from different perspectives including:

  • High science and clinical development
  • Commericalisation
  • Patient support and communication
  • Regulatory

Some of the presentations focused on specific rare diseases whilst others explored broader themes. Highlights included:

  • ‚Developing therapies for monogenic neurodegenerative disorders‘ presented by Dr. Rajeev Sivasankaran
    Head – Rare Diseases, Neuroscience Division, Novartis Institution for BioMedical Research
  • ‚From rare to common: targeting Gaucher defects for the treatment of Parkinson’s Disease‘ presented by Dr. Pablo Sardi, Neuroscience Therapeutic Area, Sanofi
  • ‚Precision medicine in chronic kidney diseases: zooming in and out rare diseases‘ presented by Dr. Maria Chiara Magnone, Sr Director, Head of Translational Sciences CVMD iMed, AstraZeneca

As sponsors of the conference, Ashfield was delighted to attend, meet with our pharma clients and deliver a presentation. Matt Brierley, VP Medical and Scientific Services, and Nagore Fernandez, Head of Patient Services for EUCAN, presented ‚From infancy to adulthood: a new generation of challenges for rare disease communities‘ covering:

  1. Enabling timely, successful patient diagnosis
  2. Addressing evolving informational and emotional needs
  3. Delivering practical, patient-centric, multichannel support
  4. Nurturing collaborative care from joined-up HCPs
  5. Demonstrating value to payers

You can see an extract of the Ashfield presentation below. Please get in touch if you would like to arrange a presentation of the full deck, including patient videos.