Journal of Genetic Syndromes & Gene Therapy
Open Access

Value creation and transformative opportunities in the rare diseases space: Lessons learned & what lies ahead

Annual Congress on Rare Diseases & Orphan Drugs

October 26-27, 2016 Chicago, USA

Francois Nader

Acceleron Pharma, USA

Posters & Accepted Abstracts: J Genet Syndr Gene Ther

Abstract :

Rare diseases have been a source of value creation for the Biopharma industry in the last 10 years. The transformational journey of NPS Pharma from a failing company to a $5.2 billion exit is a vivid example of a success story with significant value delivered to patients, shareholders and employees. The presentation will share the lessons learned along the way as they are continue to be applicable to public and private companies that are in the space. Yet, the winds could be turning when it comes to pricing and reimbursement of orphan drugs, especially within the context of the US presidential elections and the shift in the public opinion regarding drug costs. If not addressed, they could put significant damp on this vertical. However, innovation and patients can still win the battle through understanding cost-containment and cost-cutting approaches that are currently in play and proactively developing and executing the necessary strategies to address them. Part two of the presentation will details these challenges and offer practical options to dealing with them.

Biography :

Email: Francois.Nader@jesra.com