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In Case You Missed It: Three Things we Learned in our Orphan Drugs Webinar

If you missed our recent webinar in which we dived in the orphan drugs landscape, you skipped a fascinating discussion between biopharma journalist Melanie Senior, author of our recent report “Have Orphans Lost Their Sparkle?”, Evaluate analyst Puru Gaur and special guest Stephanie Léouzon, Managing Director, Biopharma Healthcare at Stifel. The good news is that the webinar is available on demand so you can catch up whenever suits you.

In the meantime, here are three quick takeaways from the conversation.

  • The sparkle may be fading – but we needn’t weep for orphan drug developers. Our report showed that the growth gap between orphans and the wider market is narrowing, particularly with the shift towards “big drugs for big diseases” and the challenges raised by the Inflation Reduction Act (IRA). However, the top selling orphan drug in 2028, Darzalex, is expected to hit revenues of around $17bn in 2028, with the top 10 in that year totalling over $57bn. Orphans now account for over half of FDA approvals. Talk of fading sparkles is all relative – there’s still plenty of shine in this space.
  • Payer innovation may be on the cards. Reimbursement of drugs for rare diseases is hugely varied, and Stephanie pointed to an increasing trend in outcomes-based pricing, particularly for some gene therapies. The high costs of many orphan therapies means that payers need to understand and see proof of value. This is hard to track, with a significant administrative load, but real-world evidence can be helpful in demonstrating the benefit of costly treatments. This isn’t about restricting access to these therapies, but to ensure they’re being used properly.
  • Everyone still wants to know about the IRA. We had several questions from the audience looking for our experts’ views on the impact of the IRA on orphan drugs, particularly around R&D. Melanie noted that a few companies claim to have halted development of some orphan drug indications as a result of the IRA, but cautioned there is – as yet- little evidence of a wider pullback. Stephanie highlighted how companies may seek to test their drug candidates across multiple indications all at once, to limit the potential impact of a shorter exclusivity period downstream. But such ‘parallel development’ strategies, Melanie noted, would be challenging for smaller organisations – and won’t always make sense scientifically, either.

There’s much more in the full webinar, including plenty of excellent geeking out about the report’s data and more about regulatory, legislative and access challenges that I’ve skimmed over above.

A huge thanks to our excellent panel, and we hope to see you at our next webinar, in which we’ll be geeking out once more – this time over our upcoming World Preview report! Hope to see you there.



Carolyn Hall

Director, Content and Thought Leadership Marketing


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