Evaluate Releases Orphan Drug Market Outlook Report Towards 2032

Introduction

Evaluate has recently published its latest report, "2026 Orphan Drug Report: Navigating the Rough Seas to Find Safe Passage." This report comprehensively analyzes changes in the regulatory and policy environment, trends in key products and companies, and sales forecasts up to 2032 for the orphan drug market. Even amidst continuing turbulence regarding policies and drug pricing across the pharmaceutical industry, orphan drug sales are steadily growing, supported by significant policy tailwinds.

*This press release summarizes some of the report's content. For data underlying the analysis and detailed market/pipeline evaluations, please refer to the full report.

Full report available here (free)

Excerpted Summary Based on the Full Report

Orphan Drug Market to Exceed 21% of Prescription Drug Sales by 2032

According to Evaluate's sales forecasts, orphan drugs are projected to:

Increase from 15% in 2022 to account for over 21% of total prescription drug sales by 2032.

Rare disease treatment sales will exceed $400 billion in 2032.

This is expected to constitute a significant proportion of the global prescription drug market (approximately $1.9 trillion).

Top Orphan Drugs in 2032: Led by Darzalex and Vyvgart

By 2032, eight leading orphan drugs are each predicted to achieve global sales exceeding $6 billion. Among them, Darzalex (daratumumab) for multiple myeloma is expected to achieve orphan drug sales of approximately $31 billion (40% of its total sales) in 2032, with the introduction of its subcutaneous formulation virtually doubling the franchise's lifecycle. Additionally, Argenx of the Netherlands is projected to achieve $11 billion in orphan drug sales with Vyvgart (efgartigimod alfa) and Vyvgart Hytrulo, and is expected to surpass Pfizer in the orphan drug sales ranking by 2032.

Company-Specific Trends: High-Growth Companies and Diverging Fortunes

Among the top 10 companies, Argenx and Merck & Co. are the only ones projected to achieve a CAGR of over 20% from 2025 to 2032.

Conversely, Bristol Myers Squibb's orphan drug sales are expected to shrink as the patent and orphan exclusivity for its flagship product, Revlimid, begin to face competitive pressure.

Policy Tailwinds: IRA Amendment and Reauthorization of PRV System

The report points out that the 2025 amendment to the Inflation Reduction Act (IRA) is favorable to orphan drugs, and the reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) system until September 2029 is a significant incentive for orphan drug development companies. PRVs can be used for other drug programs or sold to third parties, sometimes trading for over $100 million.

Pipeline Status: Expanding in Scale, Declining in Share

Predicted sales for current orphan pipeline candidates will exceed a total of $100 billion in 2032.

However, the sales ratio of orphan pipeline drugs to total prescription drugs is expected to decline from 30% in 2027 to 22% in 2032. This reflects increased investment in drugs for large-scale diseases such as diabetes and obesity.

Conclusion: Significant Growth Potential Remains

Current orphan drugs cover only a small fraction of all known rare diseases. This report concludes that the fundamental indicators of the orphan drug market remain robust, supported by institutional assistance such as tax credits, application fee waivers, and seven years of market exclusivity.

About the Report

The "2026 Orphan Drug Report: Navigating the Rough Seas to Find Safe Passage" by Evaluate provides a multifaceted analysis of orphan drug market sales, policies, regulations, pipelines, and key players.

The full report further delves into specific examples of FDA decisions on rare disease treatments, the rapid expansion of Chinese biotech companies, and the re-evaluation of small molecule drug discovery in the orphan field, clarifying the market's "next turning points." We encourage you to also refer to the full report.

Download the full report (free)

About the Report Provider

Evaluate has supported the pharmaceutical industry for over 20 years with its proven track record and product portfolio, backed by the biopharmaceutical industry's first fully integrated data assets, serving pharmaceutical companies, biotech ventures, CROs, CDMOs, and investors.