Rare Disease Drugs Poised to Capture Over 21% of Global Pharma Sales by 2032, Report Finds

Evaluate released its 2026 Orphan Drug Report, outlining a strong long-term outlook for rare disease therapies, even as developers navigate mounting regulatory and commercial pressures. According to the report, orphan drugs are projected to account for more than 21% of global prescription pharmaceutical sales by 2032, up from 15% in 2022.¹ Despite ongoing “pricing turmoil” and policy uncertainty, the sector continues to expand as innovation in rare disease treatment accelerates.¹

The report notes that orphan drug developers in 2026 face a more complex operating environment. Regulatory signals from the FDA have become less consistent, while competition for investment capital has intensified. At the same time, many large pharmaceutical companies are shifting resources toward high-volume blockbuster markets—most notably obesity treatments—as they seek to offset a looming $300 billion loss-of-exclusivity gap created by expiring patents on major non-orphan products.²

Despite these headwinds, orphan drugs are expected to remain a significant and growing share of the pharmaceutical market, generating more than $400 billion of the nearly $1.9 trillion projected global prescription market by 2032.² Andreas Hadjivasiliou, manager of Content Strategy at Evaluate, emphasized the sector’s resilience in a company release. “We continue to see drugs for rare diseases cement their place as major players in the overall drug market. 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,” he said.¹

What Is the Commercial Outlook for Orphan Drugs?

The commercial outlook for orphan drugs is characterized by high concentration among a few top-performing franchises. The top eight orphan drugs are each forecast to exceed $6 billion in sales in 2032.¹ Furthermore, the collective revenues from today’s orphan drug pipeline candidates are projected to surpass $100 billion by that same year.¹

Johnson & Johnson is expected to maintain its position as the top-selling company in the orphan space, with nearly $31 billion in projected sales in 2032.¹ The top-selling individual orphan drug is forecast to be J&J’s Darzalex (indicated for multiple myeloma). The report attributes Darzalex's continued dominance in part to its subcutaneous formulation, which has effectively doubled the franchise's effective commercial lifespan. Other key drugs expected to lead the market by 2032 include Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), Winrevair (sotatercept), and Vyvgart (efgartigimod alfa).²

Which Factors Have Influenced the Orphan Drug Outlook?

The orphan drug market outlook is influenced by recent legislative updates that provide a more predictable commercial runway. Previously, the Inflation Reduction Act only exempted single-indication orphan drugs from price negotiations. However, the One Big Beautiful Bill Act expanded these exemptions to include multi-indication drugs, a change that benefits developers pursuing subsequent approvals in non-orphan indications.²

Briefly augmenting these market signals are the provisions of the Consolidated Appropriations Act (CAA) of 2026. This legislation restored the Rare Pediatric Disease Priority Review Voucher program through September 2029, providing a transferable incentive often valued at over $100 million for developers of childhood rare disease therapies.³ The CAA also clarified the scope of orphan drug exclusivity, codifying the FDA’s interpretation that exclusivity applies to the "same approved use or indication within such rare disease or condition" rather than the entire disease.³ This resolution of past legal ambiguities allows for the approval of multiple versions of a drug for different sub-indications, such as separate adult and pediatric uses.³

While the orphan drug sector continues to face regulatory uncertainty, evolving pricing pressures, and competition for capital from high-volume therapeutic categories, its long-term growth trajectory remains strong.²

References

1. Evaluate Ltd. Evaluate releases 2026 orphan drug report. Evaluate. March 12, 2026. https://www.evaluate.com/press-release/2026-orphan-drug-report/
2. Evaluate. Orphan Drugs Outlook 2032: Sales, Pipeline & Policy for Rare Diseases. Evaluate Ltd. Accessed March 13, 2026. https://www.evaluate.com/thought-leadership/orphan-drugs-outlook-2032/
3. Wilson Sonsini Goodrich & Rosati. Congress reauthorizes the Rare Pediatric Disease Priority Review Voucher Program and clarifies orphan drug exclusivity. February 9, 2026. Accessed March 13, 2026. https://www.wsgr.com/en/insights/congress-reauthorizes-the-rare-pediatric-disease-priority-review-voucher-program-and-clarifies-orphan-drug-exclusivity.html