NATIONAL HARBOR, Maryland—Challenges surrounding access and policy may interfere with rare disease patients’ ability to receive select therapies in the United States and Europe, panelists here at the 2023 World Orphan Drug Congress (WODC) suggested.

Jayson Slotnik, JD, a partner of Health Policy Strategies, said Europe is not going in the right direction. “It’s not promoting access, it’s not providing access, a lot of mixed messages.”

He referred to the Inflation Reduction Act (IRA) passed in the US last year. Heidi Ross, MPH, vice president of policy and regulatory affairs at the National Organization for Rare Disorders, said the IRA includes provisions that could result in fewer nations having fewer therapies. “We know what it’s like when the incentives aren’t properly aligned,” she added.

“In the pre-[Orphan Drug Act] space, there were very few drugs available; now we have about 140 FDA-improved indications. That’s a huge move for our community. At the same time, we have to be honest that a lot of our patients can’t afford these therapies,” Ross said.

Mark Battaglini, JD, chief strategy officer of the Alliance for Regenerative Medicine,  pointed out the community needs in the rare disease space, highlighting the issue of delivery.

“The challenge of infrastructure on diagnosis is really bad. The diagnostic odyssey, a term that is often used in this rare disease space, takes 5 to 8 years. We have to find better ways, better diagnosis, better ways of newborn screenings, or effective ways of newborn screening,” he said.

Battaglini added that considerations should be made for how to make moving from state to state or country to country in Europe for care more seamless. 

“Our fractured healthcare system makes it very challenging for these patients to go between providers, to go between payers, that are traveling across state lines for their care to access those therapies. The procedural and the administrative burdens that exist are really significant,” Ross said.

She noted that data show the average rare disease patient travels more than 60 miles to receive medical care and that 17% of patients have considered moving to be closer to their medical provider.

“I think telehealth will break down those barriers,” Ross concluded.


Keynote panel on policy and access: a changing world-how can we increase access for rare disease patients in the US and beyond? Presented at: World Orphan Drug Congress USA 2023: May 24, 2023; National Harbor, MD