PPRV ‘means hope’ – plus, the 'biological century' is ahead

BIO 2025: The PPRV ‘means hope’

The Pediatric Priority Review Voucher (PPRV) is vital to biotechs developing rare disease treatments and patients needing those treatments— and must be reauthorized, said panelists at BIO 2025.

What it is: Companies gaining approval for rare pediatric disease drugs can receive a voucher granting expedited review of another drug in the future; the voucher can also be sold to another company. The funds can be reinvested to fund other critical research. Renewed with bipartisan backing since its initiation in 2012, the program sunset late last year.

Why it matters to patients: “In one word, for the rare disease community, the PPRV means hope,” said Karin Hoelzer, D.V.M., Ph.D., Senior Director, Patient Advocacy at BIO. The voucher has enabled the commercialization of dozens of treatments for previously untreatable diseases.

Why it matters to small biotechs: The funds provide an important additional revenue stream. As the panelists explained, Travere Therapeutics, for example, sold a voucher to Sanofi in 2015, providing funding to start developing a rare kidney disease treatment that is now available for patients.

Why it matters to ALL biotechs: At no cost to taxpayers, a voucher expediting a drug’s approval means earlier revenues, which can make investors decide to greenlight a new drug, said Barry Datlof of the U.S. Army Medical Research and Material Command.

“Tsunami” of support: “One of the really amazing things about the rare disease community is that we have a tsunami of advocates,” Hoelzer said. “We’ve seen so many families raising their voice to protect the PPRV program.”

Watch Karin Hoelzer explain:

What BIO is doing: The PPRV is a critical issue that BIO's advocacy efforts remain focused on, said BIO's Chief Communications Officer, Sarah Alspach, in an interview at BIO 2025. The PPRV "provides an important incentive to invest in rare disease research, knowing the many thousands of patients who are still waiting for their treatment or their cure." Watch:

BIO 2025: The world can’t wait – it needs to collaborate

Biotech is a global business, and we’re better for it, said experts at the closing mainstage panel.

Why it matters: 45% of the participants at BIO 2025 were from outside the U.S., said moderator Joseph Damond of Crowell Global Advisors. Six panelists, including biotechs, investors, and an EU official, explained how global business enables new treatments.

Case in point: Venture capitalist Bibhash Mukhopadhyay said a global community of doctors, biotechnologists, hospitals, and patient organizations enabled a CRISPR-based breakthrough that saved a baby and may revolutionize rare disease treatment. “Our industry needs that collaboration,” he said. “No one individual, one vertical has that.”

COVID highlighted challenges for our global supply chain. While we can’t build new infrastructure in every country, we need to ensure each region has a resilient supply chain, said Steffen Thirstrup, MD, CMO of the European Medicines Agency.

Tariffs interfere with global trade, “restrict progress,” and do not improve access to treatments, said Generate:Biomedicines CEO Michael Nally. Improving access requires changing America’s pricing system, where “the manufacturer basically is only getting about half the value while middlemen receive the other half.” (Read more about BIO's take on tariffs.)

“This will be the biological century,” Nally predicted. Panelists agreed that global collaboration and AI mean remarkable breakthroughs will come at a faster pace—and the world can’t wait.

What Else to Read

FDA approves Gilead Science’s HIV prevention drug. The BIO member’s twice-a-year injection “was found to dramatically reduce the risk of infection and provide near-total protection against HIV,” reports CNN. “Gilead scientists have made it their life’s work to end HIV and now, with the FDA approval of Yeztugo and in collaboration with our many partners, we can help to make that goal a reality,” said Gilead CEO Daniel O’Day.

FDA’s lead CGT reviewer Nicole Verdun exits the agency. She led the office that reviews cell and gene therapies at the Food and Drug Administration (FDA), as STAT News notes. Biotech companies and advocates praised her for accelerating the development of rare disease therapies.

HHS Secretary Robert F. Kennedy Jr.: ‘Make American Biotech Accelerate’. In a post on X, Secretary Kennedy said, “The mission to Make America Healthy Again (MAHA) includes MABA — Make American Biotech Accelerate. President Trump showed in his first term what happens when you unlock American science — breakthroughs happen fast. Now, we’re going to do it again. We know the power of U.S. biotech.” Read the whole post.

Did you miss BIO 2025? We’re continuing to publish recaps, interviews, and thought leadership on Bio.News all summer long!

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