Voisin Consulting Life Sciences presentation icon
Presentation

Smarter Orphan Drug Development

Post thumbnail Smarter Orphan Drug Development

Rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient suffering from the same disease. Rare diseases can be chronic, progressive, degenerative, and often life-threatening. Given the small patient populations, traditional development pathways may not be relevant or necessary, requiring novel and innovative approaches. From a market access perspective, lack of comparative effectiveness and long-term outcomes data can lead to delays in reimbursement and significant post-launch evidence generation commitments. Treatment costs and patient benefits may not be matched over time giving rise to difficulties with assessing the value and establishing the necessary funding pathways. VCLS is at the forefront of designing new strategies to bring effective and timely treatments to patients in these high unmet need rare disease areas.

This presentation provides an outline for smarter orphan drug development through:

  • Integrating development approach
  • Reducing cost and time
  • Maximizing patient access
Voisin Consulting Life Sciences download icon

Download the full presentation now

Fill in the form to download the content

    Yes, I would like to receive communications regarding VCLS services and events


    Sample of the presentation

    presentation by

    Gopalan Narayanan
    Gopalan Narayanan, M.D.
    Vice President, Disruptive Biologics
    Dr. Narayanan provides leadership within the area of complex and disruptive biologics such as Cell and Gene therapies, called Advanced…
    View profile