BOSTON, Massachusetts—The COVID-19 pandemic and its ripple effect on rare disease patients worldwide offers valuable lessons for the future, said panelists at the World Orphan Drug Congress (WODC) that took place here July 11-13, 2022.

The conference attracted 850 people including top pharmaceutical executives and the heads of several global patient advocacy coalitions.

“We need to bring the concept of a pandemic to address rare diseases with urgency and a common purpose,” said Shannon Resetich, global franchise head for rare diseases at French drug giant Sanofi. “Rare disease has been a pandemic from the beginning. The question is, how can we translate that into greater care for rare disease patients?”

Continue Reading

Durhane Wong-Rieger, PhD, president and CEO of the Canadian Organization for Rare Disorders, said that as the coronavirus spread rapidly around the world, many patients got left behind—and not only those with rare conditions.

“Cancers and cardiovascular diseases also progressed because patients couldn’t get in for care,” she said. “The challenge is what do we do with that information going forward? Can we build back smarter? We don’t want to just do the same thing and just do it better. We need to make a pivot here.”

A sign at the 2022 World Orphan Drug Congress (WODC). Credit: Larry Luxner

For example, she said, “you invest in prevention upfront. You vaccinate people so they don’t get COVID. You want to prevent the disease, not just wait until they’re infected . . . We’re paying a whole lot in terms of fixing problems after the fact.”

One of the biggest problems in the aftermath of the COVID outbreak was an abundance of vaccines in wealthy countries and crippling shortages in poor ones.

To that end, Wong-Rieger said, companies—including those in the rare disease space—should consider a global marketing strategy that focuses on equity rather than strictly profits.

“We understand how it works; you need to get the most money back where you invested,” she said. “But what if we said don’t release a drug at least until you have a global strategy that you can sell back to your shareholders? Because that would maximize the good.”

Wong-Rieger stressed the importance of a United Nations resolution, adopted on December 16, 2021, on persons living with a rare disease.

“This recognizes rare diseases not just as a medical issue, but also as a human rights issue, an equity issue, and a social issue,” she said. “If we go back to COVID, some of the failures of the vaccines had to do with people not wanting to take it. Politics got in the way. Our failure is that we truly didn’t address those people, who had real concerns and still do.”

Fumie Griego, deputy director-general of the International Federation of Pharmaceutical Manufacturers and Associations (IPPMA) in Geneva, Switzerland, said the UN declaration also highlights the need for innovation.

“Whether it’s real-world evidence or any other components we can use to continue to incentivize R&D, a lot of policies are quite short-sighted and really undermine long-term investment in what we need,” she said.


Keynote fireside chat: State of global rare ecosystem—leveraging public-private partnerships to further shape the future of rare diseases around the world. Presented at: World Orphan Drug Congress; July 12, 2022; Boston, MA.