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Multi-year insights. Market-shifting forecasts. A clear view of what is next in rare diseases.
The top eight orphan drugs are projected to each surpass 6 billion dollars in global sales by 2032. Darzalex (daratumumab) leads the group, boosted by its subcutaneous formulation and an orphan‑focused change to the Inflation Reduction Act in 2025.
Darzalex helps secure Johnson & Johnson’s position as the leading orphan drug company, with nearly 31 billion dollars in expected sales in 2032. Argenx is also set to break into the top ten as the Vyvgart franchise grows toward 11 billion dollars in projected worldwide sales, placing the company ahead of Merck & Co. and Bristol Myers Squibb.
Orphan drug developers are facing increased FDA scrutiny, new pricing considerations, and growing competition from large therapeutic categories. Even with these pressures, the fundamentals remain strong.
The 2026 report examines:
Why orphan drugs are expected to represent more than 21 percent of global prescription sales by 2032
How the top eight orphan drugs are projected to each exceed six billion dollars in annual sales
The rise of Argenx, which is forecast to break into the top ten orphan drug companies by 2032
The impact of the renewed Rare Pediatric Disease Priority Review Voucher program and 2025 to 2026 IRA exemptions that strengthen orphan lifecycle strategy
Why the orphan share of the pipeline is projected to fall from 30 percent in 2027 to 22 percent in 2032, driven by significant growth in GLP-1 and CNS or cardiovascular therapies
In this discussion, Evaluate’s experts explore:
See the key shifts shaping this year’s outlook, including therapy area changes, emerging modalities, and important policy developments.
Download the infographic to discover:
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Rare disease strategy is shifting fast. Pricing frameworks, FDA expectations, competitive intensity, and investment patterns are all evolving at the same time. Many teams need more than data. They need a partner who can help them interpret the signals and move with confidence.
Evaluate’s Consulting and Analytics team provides tailored support for organizations that want to go deeper than the core report. Our consultants bring together Evaluate’s forecasting data, secondary research, custom modeling, and direct insight from key opinion leaders, healthcare professionals, and payers. The result is practical, evidence-based guidance that helps you navigate the most complex decisions in rare diseases.
Get in touch with our team to explore the right solutions for your business.