Insights From the 2018 World Orphan Drug Congress

Rare disease zebra

“Peppy” the office zebra

At PEP’s Saratoga Springs office, there’s a story prominently displayed on the wall about horses and zebras. It’s an inversion of the aphorism in medicine, “When you hear hoofbeats, think of horses not zebras.” Because in our work in rare disease marketing and communications, patient relationship management, and clinical trial recruitment, sometimes those hoofbeats are made by zebras.

We were in good company then, at the World Orphan Drug Congress (WODC), held recently in the Washington, DC, area. Not only was the zebra—a symbol of the rare disease community—everywhere, so too was the hope that long-desired medical breakthroughs for patients living with rare diseases are just around the corner.

A conference with a mix of attendees—pharmaceutical industry representatives (including commercial teams, reimbursement and access teams, and clinical trial teams), advocacy group leaders, and patient advocates themselves—WODC featured opportunities for participants to learn about everything from the science of gene editing to the challenges of ultraorphan drug market access.

The conference was truly global in scope and featured a wide range of speakers from across the country and around the world. Networking and pitching opportunities abounded, and the exhibit hall, where PEP had a well-trafficked booth, was vibrant and full of passionate discussion about challenges and opportunities in the rare disease drug space. Lively roundtables allowed participants to trade stories and workshop solutions.

PEP’s booth at WODC




We really have to go after evidence to clearly elucidate the benefit to patients and to stay grounded in the idea of benefit to patients. That’s our obligation and responsibility.




Some of the highlights were a keynote panel discussion about how rare disease companies are ramping up innovation to boost orphan drug development and patient centricity. Horizon’s Chief Commercial Officer Vikram Karnani noted that every person who works in rare disease is a patient themselves of some kind, and we should lean in to our personal experience and insight when working with patients in trials. “We really have to go after evidence to clearly elucidate the benefit to patients and to stay grounded in the idea of benefit to patients. That’s our obligation and responsibility,” he said.

Some of the buzz in sessions was more tactical in nature. Gene editing is a very hot topic, and we heard a lot about resisting the urge to simplify the process when creating patient-facing content about the technology. Patients—who, as we say, are the disease experts—deserve more than one-pagers. Another key insight: quarterly newsletters for patients allow pharma companies to build relationships with disease communities between blockbuster announcements. That one was familiar to us, as PEP knows the value of regular communication: we’ve helped develop effective touchpoints, both on- and offline, for patients throughout their disease journeys.

So what were the main takeaways? As we’ve long said, the value of patient-centric communications for industry is enormous, and patient groups are showing more interest in collaborating with industry. As a whole, patient engagement now touches the whole life cycle of drug development, which is twice as true with rare diseases because understanding the needs of patients and burdens of disease are so important to developing successful treatments.

All in all, WODC was a unique chance to glimpse where the industry is today—and where it’s going. Whatever direction it heads, PEP will be there. Along with the zebras.