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LONDON — Evaluate, a leading provider of market insights for the pharmaceutical industry, has released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030.
The 2025 report, based on Evaluate Pharma forecasts, projects that orphan drugs will make up a fifth of the forecasted $1.6 trillion in worldwide prescription drug sales by 2030. The segment will continue to outpace the wider pharmaceutical market for the next few years but its growth advantage over non-orphans is narrowing and is forecast to be just 1% by the end of the decade.
The report highlights that the regulatory and exclusivity advantages designed to support development of drugs for rare diseases means that many orphan drugs now resemble mainstream pharmaceutical blockbusters. These mega-orphans have caused the lines between niche and non-orphan drugs to blur, and the lifecycle management techniques used by leading pharmaceutical companies are becoming increasingly evident.
The growing awareness of less-prevalent diseases has also inflated overall orphan sales. In addition to 20% of global sales, orphan drug candidates will make up 20% of global pipeline forecast sales over the rest of this decade.
“Drugs for rare diseases now occupy a significant share of the overall drugs market” said Andreas Hadjivasiliou, Manager, Content Strategy at Evaluate. “Their increasing similarities to mainstream drugs may mean that they are just as heavily impacted by shifting policy and regulatory changes as the rest of the industry”.
The full report highlights forecasts of the top companies and drugs poised to impact this industry, as well as the biggest therapy areas. Key findings include:
● Orphans’ growth will continue to outpace mainstream drugs for the next few years, with the category’s 2025-2030 forecasted compound annual growth rate (CAGR) topping 10%, versus 7.5% for non-orphan prescription drugs
● The top-selling orphan drugs in 2030 is forecast to be J&J's Darzalex (multiple myeloma), which will exceed $16bn in annual sales, followed by Alyftrek (Vertex - $8.3bn) and Winrevair (Merck & Co - $6.6bn)
● Darzalex also places its owner, J&J, in the number one spot in the list of top-selling companies of orphans, with a predicted $31.7bn in sales
