NATIONAL HARBOR, Maryland — Access to therapies for rare disease patients varies greatly from one region of the world to another, with ultra-rare diseases generally requiring ultra-expensive drugs to treat them — if a therapy is available at all.

On August 27, 2021, the final day of the World Orphan Drug Congress USA 2021, a trio of pharmaceutical executives and patient advocates convened to discuss the problem. Moderating the panel was Scott Gray, president and CEO of patient logistics management firm Clincierge.

“The unfortunate reality is that affordability is clearly a challenge, but it’s not the only one,” said Jason Tardio, chief operating officer at Ovid Therapeutics.

Tardio said that Ovid — a New York City-based firm specializing in therapies for Angelman syndrome and other rare neurological disorders — focuses on 5 principles: affordability, accessibility, availability, awareness, and adherence.

“Efficiently getting our products to as many patients as possible requires a proactive strategy,” he said. “You need to think globally, and engage in clinical trial sites beyond just the United States.”

Durhane Wong-Rieger, PhD, president and CEO of the Canadian Organization for Rare Disorders and chair of Rare Diseases International. Credit: Larry Luxner

Maureen Menacher is vice president of market access at Enzyvant, which focuses on tissue-based therapies for rare diseases.

“The biggest challenge is to be able to maintain patient-centricity while staying compliant,” she said. “In rare disease and particularly in ultra-rare disease, you need to maintain the sense of urgency for development and for approval. These are debilitating, life-threatening disorders. There aren’t that many patients out there, and they often feel isolated. We want to make sure there’s an individual approach for patients, caregivers, and all other stakeholders.”

But that feeling of isolation is exactly what’s happening in much of the developing world, said Durhane Wong-Rieger, PhD, president and CEO of the Canadian Organization for Rare Disorders

“Many of our patient communities feel they’re being neglected. Nobody’s really engaging them in an effective way, so we’re working very hard to build capacity,” said Wong-Rieger, who also chairs Rare Diseases International, a global alliance of national and regional rare disease patient organizations. “As we’ve seen with the vaccines, if we don’t recognize this as a global issue and we’re not coming up with global solutions, we’re all going to lose.”


Ensuring access for all rare disease patients. Presented at: World Orphan Drug Congress USA 2021: August 26, 2021; National Harbor, MD.