To some, rare diseases represent the epitome of what the biopharma industry is all about: devoted researchers, entrepreneurs and leaders fighting for patients with few treatment options and solving the most difficult medical puzzles.
The space is growing at a rapid pace with a wide spectrum of industry players digging in, from Big Pharma to mid-sized biotechs to small research-centric outfits. Rare diseases are also one of the big drivers of M&A in biopharma as demonstrated by the largest deal of 2022 — Amgen’s purchase of rare disease biotech Horizon Therapeutics for almost $28 billion.
The rare disease leaders on this year’s PharmaVoice 100 list are warriors for the patients they serve. Many are working against the odds to treat diseases that have been overlooked for years or decades. Some have close family members with a rare disease, motivating them even further to fight for progress. They also devote time and energy to educating the public and the wider medical community about these uncommon conditions so no patient is left behind.
What these warriors have in common is the excitement, the diligence and the perseverance to bring their expertise and know-how to the patients who need them most, driving the necessary research, funding and regulatory gumption it takes to succeed for the rare disease community.
These are the rare disease warriors of the PharmaVoice 100.
Dr. Aoife Brennan
“There is so much beyond our control in biotech and life generally. We cannot take ourselves too seriously.” Read more ➔
Dr. Sarah Gheuens
“Conversations with patient advocates anchor us to why we do what we do.” Read more ➔
“It is helpful to unify around a common mission that everyone can buy into and be self-motivated and inspired by.” Read more ➔
“Within rare diseases, where over 90% lack therapies, every decision we make has the potential to drive meaningful change.” Read more ➔
“I get tremendous energy and inspiration from people across the organization — listening to diverse perspectives helps me make better-informed decisions.” Read more ➔
“There is so much work to be done to build equity for the rare disease community, so we stay inspired by celebrating each of the small wins.” Read more ➔
Jinky Ang Rosselli
“Inspiration comes when teams know they have the space to dream, fail and dream again, without consequence.” Read more ➔
Dr. Shehnaaz Suliman
“We are inspired creators, not bound by convention, carving out elegant solutions to complex health challenges and expanding the scope of what’s possible.” Read more ➔
“When tough times come — and they always do, R&D is a tough business — having a solid footing as a close-knit team goes a long way toward getting us through to the other side.” Read more ➔