Orphan Drugs at a Crossroads: Bigger Numbers, Tougher Calls

Andreas Hadjivasiliou

Andreas Hadjivasiliou

Manager, Content Strategy

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Orphan Drugs at a Crossroads: Bigger Numbers, Tougher Calls

Andreas Hadjivasiliou

Andreas Hadjivasiliou

Manager, Content Strategy

Published

Share:

Drugs for rare diseases have played an increasingly significant role in the biopharma market for years. I’ve been working on Evaluate’s annual orphan drugs report for the past five years and it’s clear from looking at the evolution of the data in that time that orphans offer considerable opportunity.

We’ve just published this year’s report, and the data continue to tell that story. By 2032, orphan drugs are forecast to generate more than $400 billion in annual sales and will account for over 21 percent of the global prescription market. That is a remarkable rise from just 15 percent a decade earlier, and it explains why rare diseases remain firmly on the strategic agenda.

That upward trajectory isn’t straightforward, however, and there are crosscurrents that make the landscape uncertain. But it does make for a fascinating conversation, which is why I am very pleased to be taking part in our upcoming webinar based on the report. I’ll be joining my report author and long-time collaborator, Melanie Senior to try to cover all the big topics.

As always, we’ll start by diving into some of the data and analysis from the report. I’ll highlight Evaluate’s forecasts for the top-selling orphans in 2032, and we’ll look at the changes taking place in the list of the top 10 companies by then too. Plus, it’s always fun to look at the orphan drugs pipeline to see what the potential blockbusters of the future look like.

We won’t dwell too long on the numbers – you can dig into them in the full report which is available here. Our focus will be on discussing the wider market trends and what they mean for the biotechs and pharma companies operating in the rare disease space.

There are four areas we’ll cover:

  1. Policy uncertainty. Changes to the Inflation Reduction Act and the re-authorization of the Rare Pediatric Disease Priority Review Voucher program, among other things.
  2. Deals momentum. What’s driving some sizeable premiums in recent rare disease deals?
  3. The shift towards big drugs for big diseases. With $300bn at risk in the wider market from loss of exclusivity by 2030, how do higher-risk orphans fit into portfolios?
  4. The impact of China. A huge conversation for any part of the market, and orphans are no exception.

There is an awful lot to cover but we’ll aim to do it in 45 minutes (maybe an hour!) and it would be great if you could join us. You can register for the webinar here, and you can submit questions in advance too. I hope to see you there!

Upcoming Webinar

World Preview 2026

Tuesday 16th June 2026 | 3pm BST

By 2032, the global pharmaceutical market is forecast to reach almost $1.9tn. Inside the innovations, dealmaking, and portfolio strategies transforming the global biopharma pipeline.

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Frequently Asked Questions

Evaluate’s annual orphan drugs report forecasts orphan drugs will generate more than $400bn in annual sales by 2032 and make up over 21% of the global prescription market (up from ~15% a decade earlier).

The post highlights policy uncertainty, including potential IRA changes. Shifts in pricing rules, negotiation timelines, or exemptions could alter orphan-drug incentives and revenue expectations—hence the report/webinar focus on scenarios, not a straight-line forecast.

The post calls China “a huge conversation” for orphans, as R&D, regulation, and competition can reshape the space. For detailed forecasts (including projected top-selling orphans in 2032 and the top 10 companies), see the annual orphan drugs report and the companion webinar linked in the post.