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The rare disease patient community is the beating heart of the Biotechnology Innovation Organization’s (BIO) mission. Whether it is driving policy that supports innovation, improving coordination with regulatory bodies, or facilitating partnerships, BIO is committed to helping rare disease patients live longer, healthier lives.
Rare disease has been a major theme at the 2025 BIO International Convention. As many of the discussions from the week have shown, rare disease patients can’t wait.
Here are a few takeaways.
‘Why we do what we do’
Speaking at the Patient Advocacy Pavilion in the center of the exhibit hall, BIO President & CEO John F. Crowley explained the importance of patients.
“When we think about why we do what we do, the purpose and meaning to everything in biotechnology, it goes back to that moment when a patient and their family, their loved ones, receive a diagnosis, and they’re scared and they’re confused and they need hope and they need newer and better medicines,” said Crowley. “And that’s what we need to provide here, and we need to do that with a great urgency, to realize that time is precious.”
“It’s exciting to not only put patients at the center of everything we do, but to bring patients together as part of this great convention.”
As Crowley explained, BIO has a unique ability to convene, to be a thought leader, and to generate ideas, policies, partnerships, and solutions that could move medicine faster to people in need – watch:
This morning, alongside his family and fellow patient advocates, BIO President & CEO John F. Crowley led a morning conversation about the focus of biotech innovation: patients, families, caregivers, and people around the world. #BIO2025 pic.twitter.com/djSOpnJ1iC
— Biotechnology Innovation Organization (@IAmBiotech) June 18, 2025
How policy can support breakthroughs for rare disease
“Our goal is to spur investment in innovation and ensure that patients who can benefit from new medicines, new therapies, are ultimately going to have access to them,” said Sarah Alspach, Executive Vice President and Chief Communications Officer at BIO.
“One way we’re doing that is working on modernizing our regulatory processes and ensuring that our regulatory reviews match the speed of innovation,” she continued. “We’re also looking at…improvements and changes to the Inflation Reduction Act, which we know has had an impact on investment in research.”
For rare disease patients in particular, BIO is focusing on a number of key issues, such as the reauthorization of the Pediatric Priority Review Voucher and encouraging improvements and changes to the Inflation Reduction Act, including passage of the ORPHAN Cures Act.
‘The PRV means hope’
Several panels touched on the Priority Review Voucher program and why it was so impactful for the rare disease community.
“In one word, for the rare disease community, the PRV means hope,” said Karin Hoelzer, D.V.M., Ph.D., Senior Director, Patient Advocacy at BIO.
Only 5% of rare diseases have a treatment available today. And as Hoelzer explained, the parents of children with rare disease are dealt a one-two punch: the struggle of diagnosis, and – if they even get a diagnosis – the blow of likely hearing that there is no treatment.
“The voucher makes it economically feasible to develop these products,” she told Bio.News in an interview. Watch her explain more:
Director of the FDA’s Rare Disease Hub details mission
Amy Comstock Rick, J.D., Director of Strategic Coalitions at the Rare Disease Innovation Hub at the U.S. Food and Drug Administration (FDA), explained how companies can work with the Hub.
“The mission of the Hub is to advance therapies for rare diseases,” said Rick, “Particularly for the very small rare disease populations, as well as to enhance the coordination and consistency between the Biologic Center and the Drug Center at FDA. The Hub also has a mission of ensuring that there is appropriate, necessary, and helpful education available, not just to FDA and FDA reviewers, but really the entire rare disease community.”
As Rick notes, the Hub was created in response to the rare disease community’s need, as well as the FDA’s commitment to match the “lightning speed” that they are seeing when it comes to the development of treatments and drugs for rare disease patients.
“We are doing everything we can to make sure the FDA is embracing that and taking full advantage of it and working as one agency to do so,” said Rick.
The patients are why we do it
Storytelling has become a powerful tool in the biotech arsenal. Whether it is advocating on the Hill or inspiring researchers in the clinic, the patient story must always be at the center.
“When you look at the package insert for the medicines, you see an N, which represents a data point,” said Michele Oshman, BIO’s Chief Patient Advocate and Head, Patient Advocacy Center of Excellence. “But behind every data point there is a person, a family, and somebody for whom there’s a why.”
Sitting with a panel of patient advocates, researchers, and biotech entrepreneurs, it was clear: the why was the main driver of the work.
“Day One,” asserted Leslie J. Williams, MBA, Co-Founder, President, and CEO of HC Bioscience. “If you’re developing a drug, you’ve got a Day One. Know what? It’s not just the disease. You’re treating a person, a whole person, and there’s so much more to it, and the stakes become so personal.”
“The flood gates are opening for patients to be engaged at all levels of drug development,” said Rich Brennan, Vice President, Federal Affairs at The ALS Association.
