Orphan drugs are forecast to make up 21% of all prescription drug sales by 2032, according to Evaluate forecasts. So it’s hardly surprising that we had a lot of interest in our latest orphan drugs market webinar. If you missed it, you can find the on-demand version here. If you’re saving that treat for later, here are my three key takeaways from our experts’ discussion.
- Orphan drugs are central to dealmaking
Dealmaking is at the heard of the wider pharma market and orphans are no exception. With fewer late stage assets available and competition for quality pipelines intensifying, orphan drugs are playing a key role in licensing and M&A decisions. In fact, all the drugs in the 2032 top ten were either acquired or in-licensed. Smaller biotechs are using rare disease assets to attract partners earlier, while larger companies are looking to invest in orphan franchises that can scale over time. - The regulatory environment is complex but broadly positive
There was no glossing over the uncertainty, particularly in the US. Regulatory decision making feels less predictable, and recent setbacks have had a real impact on confidence. However, the incentive framework that underpins orphan drug development remains intact, and the recent change to the Inflation Reduction Act is good news for rare disease players. Policy tweaks and renewed programs continue to support investment, even as the bar for approval rises. Our speakers agreed that the number of sizeable deals in the orphan space in the last year suggests that companies aren’t too worried about the volatility from the FDA. At least for now. - Innovation is becoming more global
Naturally China appeared in the discussion. Melanie and Andreas agreed it’s no longer an emerging theme, but a central part of any discussion about the orphan drugs pipelines. Not only do China-originated drugs appear in the 2032 top ten, but China is now developing its own gene therapies – and they are incredibly efficient and quick at development. Competition is fierce in China too, with tens of companies working on the same thing and typically the top two surviving. How Western developers – and governments – deal with this evolution remains to be seen.
In the years I have worked on Evaluate’s orphan drugs report, we’ve documented the shift of orphans from niche to mainstream. It does look like therapies for rare diseases will peak in terms of their share of the wider market, but they influence strategy for many key players. And companies’ strategies for these not-that-niche drugs now mirror mainstream blockbusters. Lifecycle management, portfolio strategy and savvy dealmaking are all vital in this high-risk, high-reward segment of the market.
Those were my highlights but there is a lot more in the conversation so I recommend watching the full webinar. And then check out the report so you can dig into the numbers in more detail.
