Building a company focused on orphan drug development is not an easy task. Investors hesitate to invest due to the complexity of the technologies. Diseases are often not widely known. Preclinical models do not exist and patients are difficult to recruit for clinical trials. Drug development costs are not much lower than established indications, and pricing and reimbursement is an ongoing challenge. But pharma has recently shown interest in orphan drug companies, and many success stories have emerged. In this panel, established professionals discuss how normal rules in building a company do not apply to orphan drug companies. They will discuss innovative ways to secure investment and continued financial support; how to forge relationships with research centers and patient advocacy groups; how to secure pharma partners; and how to work with regulators to expedite approval of and access to their drugs.

Learning Objectives:

  • Explore how orphan drug companies differ from companies focused on established drug indications
  • Evaluate the challenges and opportunities of building an orphan drug company
  • Discuss solutions to overcome difficulties orphan drug development

Ability Level: All

Session ID: 1969

myBIO Chatter

Speakers (3)

contact_2286 Mark Boulding EVP and CLO PTC Therapeutics, Inc
contact_8259 Kevin Lee VP, CSO Orphan and Genetic Diseases R & D Unit Pfizer Inc.
contact_9900 Hans Preusting Chief Business Officer Uniqure NV

Moderators (1)

contact_4370 Luc Dochez Chief Business Officer Prosensa Holding NV