Our recent orphan drugs webinar provided a wealth of insights into the current state and future of the orphan drug market. It was based on our annual report (which is available here) and if you missed it, I recommend taking the time to watch the on-demand version. Our panel included the report’s author, Melanie Senior and key report contributor, Andreas Hadjivasiliou. We were also delighted to be joined by CEO and Chair of Insmed, Will Lewis.

If you’d like a speedy rundown of what you missed, there are three things we learning from the discussion.

1. Orphans are at an inflection point
   Evaluate has been watching the orphan drug space for several years and as Andreas highlighted some of the data from the report, he discussed the evolution of the industry in that time. These “niche” drugs are forecast to hit total sales of $333bn in 2030, which will make up a fifth of the global prescription drugs market. This segment’s growth has significantly outpaced the wider market but Evaluate’s forecasts suggest that will slow down by the end of the decade. Orphans will continue to be a significant sector in the market, but their big run may be coming to an end.
2. The Orphan Drug Act has made an impact
   Will Lewis shared his thoughts on the benefits of orphan drug designation, particularly for smaller biotech companies. Orphan designation can offer several advantages, including market exclusivity, waived registration fees, and R&D tax credits. These benefits can be crucial for smaller companies looking to bring innovative treatments to market. Will discussed the loss of orphan designation for one of Insmed’s pipeline therapies, as a potential “identity crisis” for the company which led to a decision to be an “orphans plus” business. He noted that having taken a therapy to market before, they had the infrastructure in place to do the same without orphan designation. A useful lesson for other smaller players looking to commercialize.
3. Orphans have not been greatly impacted by China….yet
   One topic that’s come up in many of our recent reports and webinars is China, particularly the increasing role of the country’s biotechs in deals with Western players looking to boost their portfolios. That impact isn’t yet translating into a large number of Chinese players in the rare disease space but our panel felt that may change. In recent years, China took very deliberate steps to enable its biotech industry to thrive and it was incredibly successful. Will Lewis commented that if and when they choose to turn their attention to rare conditions, it will not take them long to catch up with the rest of the world.

These highlights only scratch the surface of the discussion which touched on a huge range of topics and could have covered many more. To catch up on the detail, you can watch the orphan drugs webinar on demand here.

You can also find our full webinar back catalogue as well as register for upcoming events here.