Beyond the Odds: The Rare Disease Winning Formula

Session details:

Rare diseases affect 400 million people globally, yet 95% of conditions still lack an approved therapy. Against the odds, rare disease R&D has gone from a niche experiment to one of the fastest-growing engines of biotech value creation. How did this happen and what does it mean for the next wave of small and mid-cap innovators? How might big-pharma capitalize on rare disease value creation?

Join us as we unpack the rare disease success formula, policy incentives, scientific breakthroughs, and commercial proof points, that transformed skepticism into billion-dollar blockbusters. Drawing on analysis of all approved and pipeline assets and insights from senior industry experts across R&D, regulatory, commercial, and BD&L, we’ll also challenge the big questions shaping the future:

• What is driving success in rare disease and what are the key hurdles that remain?  
• What new approaches will shape the next wave of rare disease development?
• How should small and mid-cap biotechs position themselves in this shifting landscape?