The global orphan drug market has reached a significant milestone, with sales totaling $168 billion worldwide in 2023, but growth is expected to moderate in the coming years according to Evaluate's 2024 Orphan Drug Report. After a decade of robust expansion averaging nearly 11% annually, growth rates are projected to slow to just under 10% through 2028.
Despite this relative deceleration, the orphan drug sector remains a strategic priority for pharmaceutical companies, with the market expected to reach approximately $270 billion by 2028.
Shifting Market Dynamics
The slowdown in orphan drug growth comes as pharmaceutical companies renew their focus on widespread conditions like obesity and common immunological and neurological disorders. Additionally, payers have begun to scrutinize the premium pricing of orphan drugs more closely, potentially prioritizing treatments that address broader population health challenges.
"Even with the anticipated moderation in growth, orphan drugs continue to punch above their weight class in terms of value," said Daniel Chancellor, director of thought leadership at Evaluate. "It's not surprising that we'd see this moderation as orphan drugs become a mainstream business."
Chancellor noted that the orphan drug market is now approaching the value of the oncology market, which was worth $194 billion last year.
Regulatory Support Remains Strong
Regulatory flexibility continues to bolster the orphan drug sector. Nearly 60% of the FDA's new drug approvals in 2023 were for orphan drugs, highlighting the continued regulatory support for treatments addressing rare diseases with significant unmet needs.
"High prices, small trials, well-defined populations and development incentives continue to buoy this important sector," the report states, emphasizing the ongoing advantages of orphan drug development despite the moderating growth.
Market Leaders and Future Prospects
The top three orphan drugs are expected to maintain their market leadership through 2028, according to Evaluate's projections:
- Johnson & Johnson's Darzalex (daratumumab) for blood cancer
- Vertex's Trikafta (elexacaftor/tezacaftor/ivacaftor) for cystic fibrosis
- Roche's Hemlibra (emicizumab) for hemophilia A
New entrants are expected to join the top 10 list, including Incyte and Novartis' Jakafi (ruxolitinib) for myelofibrosis, Johnson & Johnson's Carvykti (ciltacabtagene autoleucel) for bone marrow cancers, and BeiGene's Brukinsa (zanubrutinib), which is supplanting AbbVie's Imbruvica after demonstrating superior efficacy in comparative trials.
Collectively, the top 10 orphan drugs are projected to generate more than $57 billion in 2028.
Pipeline Innovation Continues
Despite the moderating market growth, innovation in the orphan drug pipeline remains robust. Evaluate identifies several promising candidates, including:
- Vertex's VX-121 triple therapy for cystic fibrosis
- Johnson & Johnson's nipocalimab for inflammatory and autoimmune diseases
- Intellia Therapeutics' NTLA-2002 for hereditary angioedema
- Cancer treatments paltusotine from Crinetics Pharma and bemarituzumab from Amgen
Legislative Considerations
The orphan drug landscape continues to evolve against a backdrop of potential legislative changes. Both the U.S. Orphan Drug Act, now 40 years old, and the European Union's 1999 Orphan Drug Regulation have faced calls for reform.
Paul Verdin, VP of consulting and analytics at Evaluate, notes that these calls for legislative revision stem partly from the changing nature of orphan drugs. "Some things highlighted in the Report are the growth in the actual budgetary impact of drugs. Some could say that when you think of a rare disease that conjures up images of super-rare genetic conditions, rather than things like oncology, which are areas that attract a lot of interest, and often designations."
The challenge for payers and regulators is that orphan incentives are now being applied to drugs with much larger market potential than originally envisaged when the legislation was created.
Scientific Progress Driving Market Evolution
The scientific understanding of rare diseases has advanced significantly since orphan drug legislation was first implemented. Andreas Hadjivasiliou, managing analyst at Evaluate, points out that "Science has progressed considerably since the legislation was implemented in the 80s. There's been a move towards oncology as a more detailed set of almost sub-diseases with genetic markers within those."
This evolution in scientific understanding has transformed the orphan drug market from a niche sector to a mainstream pharmaceutical business. As Verdin states, "The key headline is that rare diseases are not a niche. It's mainstream pharma and a big part of the pharma market. Developing drugs for rare diseases is also a breeding ground for next-generation therapeutics."
Future Outlook
While growth may be moderating, the orphan drug sector continues to represent a significant opportunity for pharmaceutical companies and potential benefit for patients with rare diseases. The sector's continued innovation, particularly in emerging therapeutic modalities, suggests that orphan drugs will remain an important component of pharmaceutical pipelines and portfolios.
"New sciences are coming through in this area. That's quite an exciting space, and I think that's something we're going to see a lot of over the next few years," Hadjivasiliou concludes, highlighting the ongoing potential for scientific breakthroughs in rare disease treatment.
