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Orphan Drug Report 2024: Slowdown a Sign of Success

Hot-off-the-press, Evaluate has just released its latest annual deep dive into the world of orphan drugs and rare diseases. I had the opportunity to preview the content and make the case for why it is essential biopharma reading below. And while I have your attention, you can grab your complimentary copy of Orphan Drug Report 2024 via the link here.

An Orphan Drug Strategy Remains Critical to Success

The two introductory paragraphs can at first glance seem like oxymorons. “Orphan drugs are losing their sparkle” follows into “Orphans are still highly attractive”. But I think this neatly encapsulates the position that rare diseases occupy within pharmaceutical companies’ portfolios: a strategic priority, a mature therapeutic category, and a competitive necessity.

Clearly orphan drugs are now part of pharma’s mainstream business. Sales of $168bn in 2023 are 17% of the industry total. This is not far shy of the entire oncology therapeutic category at $194bn. For such a large cohort of drugs to still be growing at double-digit clip is impressive and shows that continued investment into the unmet need of rare diseases is warranted.

Growth rates may be moderating slightly, but this is an inevitable consequence of the success of orphan drug strategies. It is far easier to achieve high growth from a small base, but to sustain that despite commercial maturity shows that the orphan drug pipeline remains highly productive. More on this can be found within the report.

It is also no surprise that the cohort of leading companies by orphan drug sales shows a high degree of overlap with major biopharmas by total revenues. The only notable absentees from the expected 2028 top ten are Merck and AbbVie, with Vertex rising to fifth spot thanks to its unique portfolio focus on orphan drugs.

The Path Ahead Will be More Turbulent

Some of the major themes that are shaping the wider industry seem to be magnified for orphan drugs. Industry commentators are talking about the renaissance of R&D in previously overlooked therapeutic categories. In the rare disease domain, this shift away from oncology towards novel opportunities in CNS and immunology can already be seen in the latest consensus forecasts.

No doubt legislation has majorly incentivised and benefitted rare disease research over past decades, although the Inflation Reduction Act is poised to have an outsized effect for orphans. From concerns about affordability to the economics of pursuing additional orphan indications, the status quo is being rewritten. Provisions within the IRA to protect the next generation of orphan drugs may end up doing the opposite, and the report has a pragmatic view of lobbying efforts.

Lastly, as the major rare diseases see treatment advances, the shift in focus from orphan to ultra orphans poses new challenges. This may well be the dynamic that dominates the future agenda, as developers wrestle with the practical, ethical, and regulatory considerations of therapies that cater to just a handful of patients. Taken to the extreme, the distinction between a clinical trial for an n-of-1 orphan drug and standard-of-care can be wafer thin. Technological advances will soon bring such theoretical challenges into the mainstream conversation.

Download the full report now.


Daniel Chancellor

Director, Thought Leadership, Consulting & Analytics


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