Evaluate Releases 2026 Orphan Drug Report

Report reveals that FDA temperament and blockbuster drugs fall short of disrupting the ever-increasing orphan drug market

LONDON – 13 March 2026 – Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2026 Orphan Drug Report, highlighting the resilience of orphan drugs in the current pharma industry.  

The report, shaped by forecasts from Evaluate Pharma, states that despite policy changes and pricing turmoil, the sales of orphan drugs will continue to rise steadily. By 2032, orphan drugs will account for over 21% of all prescription pharmaceutical drugs, up from 15% in 2022. Drugs for rare diseases will generate over $400 billion of the almost $1.9 trillion in projected 2032 prescription drug sales – a number close to the entire prescription market twenty years ago. 

The report also discusses policy changes that are positively influencing the market. Until 2025, only single-indication orphan drugs were exempt from Inflation Reduction Act (IRA) Price negotiations. Tweaks to the IRA through the current administration’s One Big Beautiful Bill Act expanded orphan exemptions to include multi-indication drugs. The Rare Pediatric Disease Priority Review Voucher program was re-authorized in early 2026, promising faster FDA review times for products addressing rare childhood conditions. While these wins have helped to maintain momentum in the industry, orphans’ share of overall prescription drugs sales is expected to fall over the next seven years. This is likely a result of a focus on big drugs for big diseases, most notably obesity drugs – a trend in part related to Big Pharma’s need to fill a $300bn loss-of-exclusivity gap.

“We continue to see drugs for rare diseases cement their place as major players in the overall drug market,” said Andreas Hadjivasiliou, Manager, Content Strategy at Evaluate. “Their capacity to navigate a changing industry, policy and regulatory changes while maintaining momentum is something to be noted by developers in this space and in the mainstream drug markets as well.”

The full report highlights forecasts for the top companies and drugs poised to impact this industry, as well as the biggest R&D and therapy areas. Key findings include:

• The top eight orphan drugs will each sell over $6 billion in 2032, with the sales of today’s orphan drug pipeline candidates collectively forecasted to top $100 billion in 2032

• The top selling orphan drug by 2032 is forecasted to be J&J’s Darzalex (multiple myeloma), partially due to Darzalex’s subcutaneous formulation, which doubles the franchise’s effective lifespan

• Darzalex once again places its sponsor J&J, in the number one spot in the list of top-selling companies of orphan drugs, with almost $31 billion in projected sales

Evaluate will host a webinar on March 19, 2026 to discuss the latest market outlook for rare disease drugs alongside the Orphan Drug Report’s findings and forecasts. More information and registration for the webinar can be found here.