The Rare Trials Summit Wants To Produce More Than Conversation

By Dan Schell, Chief Editor, Clinical Leader

Trust me, I know it’s easy to become cynical about industry conferences. After all, there are plenty of opportunities to listen to keynote presentations, network in hotel hallways, and collect enough business cards to last until next year’s event.

That’s why the organizers of the Rare Trials Summit knew they had to create something more than just a standard conference. And after a conversation I recently had with Miro Pastrnak, PhD, who leads speaker recruitment and agenda development for the event, I’m thinking those organizers are onto something.

Returning to Boston for its second year this September 14-16, the summit was created around a simple observation. “We noticed that when we would go to rare disease conferences, clinical research was a very small portion of the agenda,” recalled Pastrnak. “Or we’d go to clinical research conferences where rare disease is a very small portion of the agenda. “So, we thought, why not create an event that fills the gap and focuses primarily on both of those elements?”

That first year the audience showed up, with roughly 200 attendees, including about 70 speakers, representatives from nearly 50 rare disease biotech and pharmaceutical companies, and leaders from 35 patient advocacy organizations. Two-thirds of attendees held VP-level positions or higher, creating a deliberately senior audience focused on solving the practical challenges of rare disease drug development rather than simply discussing them. That type of audience also can be a differentiator.

Turning Discussion Into Deliverables

Perhaps the most distinctive aspect of the summit isn’t found in the keynote presentations or breakout sessions; it’s the “Accelerate Rare” working groups that meet before the conference officially begins. (These reminded me of the Dive Teams at the Innovation Network Gathering [ING] conference.) Rather than ending with applause, these sessions are designed to produce documents that can influence policy and regulatory thinking.

Last year’s working group focused on rare disease priorities for the next PDUFA negotiations. The participants developed a briefing document that was later presented to members of the Congressional Rare Disease Caucus.

This year, the organizers are tackling two new issues. One working group, chaired by former FDA Acting Commissioner Janet Woodcock, will focus on helping industry interpret the FDA’s emerging regulatory pathway for therapies targeting ultra-rare diseases. Pastrnak believes that guidance could eventually affect the overwhelming majority of rare diseases.

“The FDA guidance is just going to be a philosophical framework,” he explained. “It’s not going to tell the companies how to actually submit drugs under this regulatory pathway.”

A second working group will develop recommendations for pediatric rare disease trials, an area where sponsors continue to operate without comprehensive regulatory guidance despite the fact that most rare disease trial participants are children.

Participation in these working groups is intentionally limited. “It’s invitation only,” Pastrnak said. “We can only fit 40 or 50 people in the room, and writing a document with 40 people is already a challenge.”

Built Around Participation

Even outside the working groups, the conference is structured differently than many traditional events. The program opens and closes with general sessions, but most of the conference consists of two concurrent tracks featuring brief presentations, fireside discussions, and interactive workshops. Rather than relying exclusively on lecture-style sessions, attendees are encouraged to work through realistic scenarios alongside experienced moderators. “We’ve always received the feedback: ‘Keep it small, make it interactive,’” Pastrnak said.

The agenda covers four major areas spanning the rare disease development lifecycle: patient identification and enrollment, clinical operations, regulatory and endpoint strategy, and commercial planning. Many of the workshops use mock exercises designed to let professionals from different therapeutic areas learn from one another.

That approach also reflects the changing makeup of the rare disease workforce. As companies expand beyond oncology and other traditional therapeutic areas, more clinical development professionals are finding themselves working in rare disease programs for the first time.

A Growing Focus On Global Collaboration

While the overall structure remains similar to last year’s event, organizers have made several changes based on attendee feedback. Workshops have been extended to allow for more interaction, and the agenda places greater emphasis on the global nature of rare disease research. “The feedback from the steering committee was that it needs to be a little more global because most rare disease trials are global,” Pastrnak said. That includes sessions addressing international endpoint harmonization and a keynote examining China’s growing role in rare disease development. The summit is also expanding its support for patient advocacy organizations by providing complimentary exhibit space and travel support for several advocacy groups, giving patient leaders a larger presence throughout the event.

Why The Timing Matters

One reason Pastrnak is particularly excited about this year’s summit is the rapid evolution occurring in ultra-rare disease research. He points to new FDA thinking around plausible mechanism evidence, increasing investment in gene therapies, and a growing number of patient-led efforts to develop treatments for diseases that previously attracted little commercial attention. “If we can find a way to industrialize ultra-rare gene therapy development, we can really make a difference and move the entire field of rare disease forward,” Pastrnak said.

Whether the summit ultimately achieves that goal remains to be seen. But its emphasis on producing tangible guidance, encouraging genuine collaboration, and bringing regulators, sponsors, sites, and patient advocates into the same conversations gives it an identity that extends beyond simply being another stop on the conference calendar.

Use this link to get a 50% off code (CLNLDR) to attend.