Beyond the Odds: The Rare Disease Winning Formula

Rare diseases represent one of the greatest unmet needs in healthcare—more than 7,000 conditions affect over 400 million patients worldwide, yet only 5% have an approved therapy. The economic and societal burden is immense, but so is the opportunity for innovation.

In this first installment of our three-part series, “Beyond the Odds: The Rare Disease Winning Formula,” we explore how rare diseases evolved from a niche market to a cornerstone of biopharma growth. This paper examines:

• The Turning Point: How policy incentives like the Orphan Drug Act, scientific breakthroughs, and early commercial successes transformed skepticism into momentum.
• The Formula for Success: Why rare disease strategies deliver both patient impact and sustainable business models.
• Market Insights: Trends in orphan drug approvals, cost efficiencies, and the rise of blockbuster therapies in ultra-rare indications.