2026 Orphan Drugs Report: A safe(r) passage amid stormy waters?

An article from Sponsored 2026 Orphan Drugs Report: A safe(r) passage amid stormy waters? Published June 1, 2026 Share Copy link Email LinkedIn X/Twitter Facebook Print Sponsored content By Evaluate SPONSORED CONTENT BY Orphan drug developers face a temperamental FDA and must battle for attention with blockbusters in obesity and other common diseases. But orphan drug sales trajectories remain strong, buoyed by important policy wins, according Evaluate’s Orphan Drugs Report, “A Safe(r) passage amid story waters?”.

Despite policy and pricing turmoil across the pharma industry, sales of orphan drugs continue to rise steadily. They will account for over 21% of all prescription pharmaceutical sales by 2032, according to Evaluate forecasts, up from 15% in 2022. Drugs for rare diseases will generate over $400 billion of the $2 trillion in projected global prescription drug sales in 2032 – about the same as the entire prescription medicines market twenty years ago.

Worldwide Orphan Drug Sales and Share of the Prescription Drug Market, 2022–2032 The top eight orphans will each sell over $6 billion worldwide in 2032. Category leader Darzalex (daratumumab), for multiple myeloma, will reach almost twice that thanks in part to a sub-cutaneous formulation, which doubles the franchise’s effective lifespan. An orphan-friendly tweak to the Inflation Reduction Act (IRA) legislation in 2025 also helps.

Top 10 Selling Orphan Drugs in 2032 by Worldwide Sales At the company level, orphan drug revenues remain concentrated among a small group of global pharmaceutical companies, but the competitive order is evolving. Johnson & Johnson is forecast to lead the market in 2032 with nearly $31 billion in orphan drug sales, representing around 40% of its total pharmaceutical revenues. Focused biopharma players are also reshaping the landscape.

Gate-crashing the top ten companies in 2032 is Dutch-incorporated Argenx, forecast to displace Pfizer in the orphan drug sales rankings, thanks to its Vyvgart franchise. Looking Ahead The orphan drug pipeline is healthy. Sales of today’s orphan pipeline candidates are collectively forecast to top $100 billion in 2032.

The most valuable late-stage orphan programs are expected to sell over $1 billion each that year; in net present value terms, the top five exceed $7 billion. Yet orphan pipeline drugs’ share of overall prescription drugs sales is expected to fall over the next seven years, from a projected 30% peak in 2027 to 22% in 2032. This likely reflects growing interest and investment in big drugs for big diseases – including most prominently GLP-1 based therapies, fast expanding beyond diabetes/obesity into cardiovascular, autoimmune and other conditions.

Policy and Regulation Offer a Complex Backdrop Recent US policy developments have been broadly supportive of orphan drug development. Amendments to the Inflation Reduction Act in 2025 expanded exemptions for multi‑indication orphan drugs, reducing the risk that successful franchises face early exposure to Medicare price negotiations. 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.

At the same time, the regulatory environment has become more complex. While the FDA has reduced administrative requirements in some areas, it has also raised expectations around evidentiary standards, particularly for cell and gene therapies. Recent regulatory decisions reinforce the importance of robust trial design, appropriate endpoints and clear regulatory strategy in rare disease development.

Like almost every corner of the market, China’s rise is impacting rare diseases, a fact that is reflected in the share of FDA orphan designations assigned to China-based sponsors, which rose from less than 5% in 2020 to 15% in 2025. China’s rise could add further pricing pressure on orphans sold in the West, if China-licensed molecules result in more crowding and competition. Despite these pressures, the fundamentals of orphan drug development remain strong.

Regulatory incentives, market exclusivity and persistent unmet need continue to support innovation. More than 95% of rare diseases still lack an approved treatment, leaving substantial room for growth. Advances in data science, genomics and AI‑enabled drug discovery are also improving diagnosis, patient identification and trial design, helping reduce development risk and inform better decision‑making.

You can download the full Evaluate Orphan Drugs report here. You’ll find top companies by orphan sales, late-stage assets ranked by NPV and the reasons behind the shrinking of orphans’ share of the market. Share Copy link Email LinkedIn X/Twitter Facebook Print Filed Under: Drug Pricing