Orphan Drugs Access & Economics Masterclass 2015

Rare disease therapies would be unattractive from an investor perspective. The small number of patients cannot financially justify expending resources into orphan therapy research and development, which is a expensive process.

As the population of patients with a specific rare disease is very small, the investments must be recouped by increasing the price of the product. It has been acknowledged that, while regulatory incentives have stimulated research and development of orphan therapies on a global level, equitable and timely access to approved orphan medicinal products for rare disease patients remains an issue. Decisions on Pricing and Reimbursement are the exclusive competence of the Member States of the European Union. Nevertheless, these Member States foster the same undisputed principles of equity and solidarity, face common challenges when providing urgently needed medicines for their patients and suffer similar burdens when organizing this access.

-EU regulation, programmes and mechanisms
-Adaptive licensing
-Methods for economic evaluation
-Alternative ways to raise funds and finance orphan drugs
-Pricing and reimbursement models

Speakers:
Carlos Camozzi, Guenter Harms, Luis Cruz, Paula Albuquerque, Camille Metais, Deborah O'Neil, Mark Nuijten