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Evaluate’s 2024 Orphan Drug Report Signals Slow Down in Growth

New report shows moderating growth rates but demonstrate strategic importance of a competitive sector

LONDON – 24 April 2024 – Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2024 Orphan Drug Report signalling growth rates are expected to moderate over the next ten years. For more than a decade, the orphan drug market’s growth has outpaced that of non-orphan prescription drugs; sales growth averaged almost 11% in the ten years leading up until 2023. However, Evaluate anticipates that the market will barely make double digits for the rest of the decade, resulting in a narrowing growth gap. You can find Evaluate’s 2024 Orphan Drug in full here.

Despite the anticipated slowdown in growth, orphan drugs remain a strategic priority and competitive necessity for pharmaceutical companies for good reason. The top 10 orphan drugs will collectively generate more than $57 billion in 2028, with the top three rounding out as Johnson & Johnson’s blood cancer drug Darzalex, Vertex’s Trikafta for cystic fibrosis and Roche’s Hemlibra for haemophilia A treatment. The top 10 list of drugs – including a shake-up further down in the rankings – can be found in the full report.

“Even with the anticipated moderation in growth, orphan drugs continue to punch above their weight class in terms of value,” said Daniel Chancellor, Director, Thought Leadership at Evaluate. “It’s not surprising that we’d see this moderation as orphan drugs become a mainstream business; sales within the category were $168bn in 2023. The entire oncology therapeutic category, for context, was $194bn. It’s a highly productive pipeline dealing with the same mainstream challenges as the rest of the industry,” Chancellor added.

Orphan drug strategies remain critical to the pharmaceutical industry’s success, though the path ahead may be becoming increasingly turbulent. The Inflation Reduction Act could have an outsized effect on orphan drugs, while many companies are prioritising more widespread health challenges driving a shift in focus back to big diseases, such as obesity and Alzheimer’s.

“It’s important to understand that the slowdown of orphan drugs is relative. As the overall prescription drug market continues to grow, so will that for rare disease drugs,” said Chancellor. “High prices, small trials and well-defined populations and development incentives will continue to buoy this important sector,” Chancellor added.

The report: Orphan Drugs are Losing Their Sparkle is available here.

About Evaluate Ltd
Since 1996, Evaluate has provided the life sciences industry with the data, insight and intelligence to facilitate confident decision making on high-value investments in treatments and markets. We enable our clients to embed our proprietary & industry data into their workflows, tools and processes so they can work more effectively and efficiently. For more information about Evaluate, visit and follow us on LinkedIn and twitter.

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