Asembia 2025: Navigating the Product Commercialization Process

Image Credit: Nicholas Saraceno

Here at Asembia’s AXS25 Summit in Las Vegas, attendees were treated to behind-the-scenes insight regarding “Commercialization Considerations for Orphan/Rare, Cell & Gene, and Precision Medicine.”

Led by Brett Casper, a senior principal at Blue Fin Group, his agenda revolved around outlining the evolving orthodoxies for commercialization of ORD, CGT, and precision medicine; commercialization considerations for orphan/rare; and commercialization considerations for cell, gene, and precision medicine.

For one, he notes, this aforementioned orthodoxy has formed over the past seven to 10 years in regard to commercialization rules. For example, pertaining to orphan/rare, a small patient population and need for manufacturer control lends itself to limited distribution and dispensing networks; high-touch patient support services; and data visibility into the treatment journey.

In his eyes, we are arguably living in in the orphan and rare prime.

“It's been argued for a while now, and we completely agree at Blue Fin Group that we are solidly in the era of orphan and rare,” Casper pointed out. “Typically, this argument rests on data where if you look back at all the novel assets that are approved by the FDA in any given year—2017, 2018 give or take a few year—about 50% or more of those assets are indicated for some type of orphan, orphan rare condition.”

Another misconception pertaining to orphan and rare is related to competition. When hearing orphan-rare, many people think of products approved for the treatment of a high-need disease state with having little to no competition, but this perception is found to be incorrect in various disease states.

Overall, when it comes to these orphan/rare considerations, it’s important to consider that:

• A manufacturer’s strategy for finding patients can be a vital determinant of success or failure for these types of products
• ORD manufacturers should be constantly engaging with patients/caregivers even prior to the prescribing decision in order to educate and establish relationships
• Manufacturers commercializing ORD products should have a comprehensive data strategy that account for data gaps or blind spots

With CGT and precision medicine on the other hand, an extreme complexity and need for precision lends itself to shortened supply chain and distribution networks; the use of qualified treatment centers; individualized support for patients, providers, and sites of administration; and complete data visibility.

Along this avenue, he says, distribution and dispensing providers are scaling at a fast pace, and are offering more solutions for manufacturers. For instance, in terms of specialty distribution, distributors are aligning more of their solutions to meet growing needs by having verticals becoming aligned to expand the ability to provide support across multiple areas. Meanwhile, for specialty pharmacy, those that are PBM-aligned are dominating the market for support CGT products, which results in payers and PBMs being able to insert themselves into product solutions more directly.

At the end of the day, engagement with patients and caregivers for this line of medicine is key—the phrase “if you build it, they will come” does not necessarily apply anymore. With distribution and dispensing providers quickly developing their services to support manufacturers, these stakeholders must also be willing to assess and evaluate potential partners for their organization.

Reference

Casper B. Commercialization Considerations for Orphan/Rare, Cell & Gene, and Precision Medicine. April 28, 2025. Asembia AXS25 Summit. Las Vegas.