The orphan drug industry has matured considerably in the past decade. With that, more orphan drugs get approved, while pharma and biotechs get closer to launch new and innovative therapies for rare disease patients. Their focus has shifted from early stage challenges to strategies on commercialization, marketing, pricing and reimbursement.
As these companies move from pre-revenue to commercial and start recouping the investments from years of R&D, more capital starts to flood into promising new biotechs, and consolidation starts to rise. VCs investment in orphan drugs biotechs has grown from $101 million in 2005 to over $346 million in 2014, according to Pitch Book.
From VCs to pharma investors, funding is a much more optimistic reality for orphan drug biotechs. However there are still major challenges and uncertainties in terms of how investors assess these investment opportunities. Evolving regulatory guidelines, increased scrutiny over orphan drugs high price, policies risk, and the common R&D failures, are some of the key areas impacting investment decisions in this market.
The lack of a platform for orphan drug biotechs to meet and present their development programs to investors contributed to the launch of Pitch & Partner at the World Orphan Drug Congress USA.
Pitch & Partner 2017 featured:
VC Panel: Investing in the orphan drug space – targeting big business by treating a small number of patients
J.P. Morgan Healthcare Investment Banking