Hi, I’m Melanie Senior. I write about the science and business of drug development, and I’m the author of Evaluate’s 2026 Orphan Drugs Report. This sets out forecasts for orphan drug sales and growth up to 2032. It ranks the top 10 companies, the top 10 products, the top 10 most valuable pipeline products, and looks at a lot more besides. So it’s full of really interesting data.

Hello, I’m Andreas Hadjivasiliou. I am a senior specialist in the content strategy team here at Evaluate. And I was responsible for doing the data analysis for our latest orphan drug report. One of my top findings from the report was the sheer size of the orphan drug market in 2032. We’re expecting a forecasted revenue of over $400 billion, which is significant. To put that into perspective, that is the same size as the entire pharmaceutical industry circa 2003.

My key takeaways from it this year was firstly the strength of some of that data and the numbers, despite the extraordinary FDA turmoil we saw during 2025. Literally FDA saying one thing and doing another. Some really high profile rejections, complete response letters for a couple of gene therapies and cell therapies, some rather frustrated messaging back from companies. Yet there were also some policy wins last year, the tweak to the IRA to allow multi-indication orphans to be exempted as well as single indication orphans. And we also saw that last minute reauthorization of the rare pediatric disease priority review vouchers, which I think people were worried wouldn’t happen and it did.

So, we always like to look at the top 10 companies in this space. And again, big pharma is dominating. Johnson & Johnson are leading the way in number one spot. We see representation from all of the large pharma companies, Bristol-Myers, Merck &Co, but we do have a few interesting entrants, Vertex and Argenx who help make up that top 10. They are more biotech flavored, built around single franchises or single drugs such as Vyvgart, Argenx, and Vertex have a significant cystic fibrosis franchise.

The other really interesting chart for me in the report was the top five most valuable orphan pipeline candidates. Now, of course, that’s what acquirers will be looking at as the next big thing. And already one of them, we’ve already seen a big almost $8 billion deal around. And that of course is Gilead’s Anito-cel came from Arcellx, that’s CAR-T cell therapy for multiple myeloma. Interesting there though, that is also something that Gilead will be looking to in autoimmune disease and inflammatory disease. And I think that’s a big story there also for T-cell engagers. Hint, there’s one of them in the top five too. Yeah. So they’re pivoting towards B-cell mediated autoimmune diseases, and that’s a big deal making space outside of orphans, but also many autoimmune diseases are being sliced into much better-defined categories and some of which are themselves orphans.

Orphan drugs are likely to make up around 21% of forecasted revenues in 2032, really cementing their place as a key component of the industry.

There’s so much in there, lots more to discuss, so I hope you enjoy it.