Patient advocates urge PPRV renewal

Patient advocates explain why the Pediatric Priority Review Voucher (PPRV) Program must be reauthorized, and National Ag Day highlights the links between biotech and agriculture. (982 words, 4 minutes, 52 seconds)

P.S. If you’re in D.C., BIO is sponsoring today’s AgriPulse 2025 Ag & Food Policy Summit, "Navigating Taxes, Trade and Technology: What's Ahead for Food & Agriculture." BIO members BASF and Pivot Bio will also speak.

Patient advocates urge PPRV renewal

The Pediatric Priority Review Voucher (PPRV) Program to encourage treatments for rare diseases must be reauthorized, said panelists during BIO’s March Patient Advocacy Coffee Chat.

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 to fund research. Renewed with bipartisan backing since its initiation in 2012, the program sunset at the end of last year.

Why it matters: As many as 60 drugs have earned a voucher to date—the vast majority for rare pediatric diseases without treatment options before the program’s inception, Bio.News reports. And the program costs taxpayers nothing.

Why delayed reauthorization is bad: “It’s creating uncertainty. And this means that people aren’t getting resources, and then it means that children are going to have to wait longer to get important medicines,” said Duke University's Dr. David Ridley, who was instrumental in developing the program.

“Last year, the House passed unanimously a bill called the Give Kids a Chance Act,” which included PPRV reauthorization and other beneficial legislation, but it was dropped at the last minute from December’s spending package, said Jamie Sullivan of EveryLife Foundation for Rare Diseases.

“One thing that we can all agree on is the need to give our children a fighting chance,” BIO President & CEO John F. Crowley wrote to Congress. “This program will provide greater certainty to small biotech companies, but more importantly, hope to children and families living with rare diseases.”

Read more on Bio.News.

National Ag Day: Biotech and agriculture grow together

As we celebrate National Ag Day on March 18, we recognize how biotechnology and agriculture have grown together.

Since farming began, we’ve worked to improve plants, animals, and soil, and impact microbes.

Biotech enables rapid improvements that would otherwise require generations of crossbreeding:

• Plants: Biotech is improving productivity per acre for crops like corn, rice, and potatoes; the nutritional value of tomatoes, sweet potatoes, and soy; and increasing the shelf life of strawberries, potatoes, and apples.
• Animals: Biotech fights disease among chickens and pigs, including the development of avian flu vaccines for birds and cattle. BIO member Acceligen enables mixing productive Holstein dairy cattle with heat-tolerant breeds in tropical zones.
• Microbes and soil: Biostimulants, made from altered microorganisms or other substances, are placed in the soil or on plants to help them grow.

Biotech shares growers’ policy concerns, including the need to pass the Farm Bill and encourage agricultural research.

BIO’s view: “We are now at the lowest spending level since 1970 in terms of agricultural research. China outspends the U.S. two times in this area,” according to Sylvia Wulf, BIO’s Interim EVP & Head of Agriculture and Environment. “What we want to do is make sure that this administration and Congress understand what they need to do, which is funding agricultural research so that we can continue to lead the world.”

Read more on Bio.News.

BIO is proud to partner with Corporate Traveler as its preferred travel management provider, offering members exclusive perks to simplify business travel and drive cost savings.

Through BIO Business Solutions, Corporate Traveler provides a high-touch service model backed by intuitive technology, ensuring biotech companies can optimize their travel programs while staying focused on groundbreaking research.

As a member company, your organization gains access to a customized travel management program with no startup fees—designed to help you travel smarter, reduce costs, and maximize efficiency.

Optimize your travel program today. 

What Else to Read This Week

BIO President & CEO John F. Crowley discusses working with the Trump administration in a BioCentury interview: 

• On tariffs: “‘Tariffs could have significant consequences in the near term—on drug availability, pricing, and investment in the biopharma ecosystem,’ Crowley told BioCentury. A key point he’s making to policymakers is that securing the U.S.’s pharmaceutical supply chain requires a global perspective. ‘The United States can once again become the epicenter of biopharma manufacturing, but we can’t achieve this renaissance solely within our borders,’ Crowley said.” 
• On FDA: “We need a strong, modernized, and reformed FDA, but don’t break the FDA. Don’t lose key people. Don’t destroy the culture.” 
• On working with the administration: “Crowley told BioCentury his approach to Trump 2.0 is based on ‘constructive engagement, building relationships, and finding common ground and areas of disagreement.’ BIO, he said, needs to have a seat at the table where decisions about the industry are being made, and to have credibility in the discussion it must ‘come armed with data, case studies and anecdotes that humanize’ its arguments.”

Scientists report correcting a gene mutation causing a rare disease with a new technique. Instead of adding or removing a gene as some current therapies do, this approach “involved fixing a spelling error involving the four base sequences — G, A, C and T — in DNA,” The New York Times reports. Dr. Kiran Musunuru, a co-founder of BIO member Verve Therapeutics, led the research.

Patients face ‘wide range of challenges’ in navigating access to care, says the PAN Foundation’s Center for Patient Research 2025 State of Patient Access Report. “For the second year in a row, Access to Treatment through Healthcare Plans was the lowest rated measure, scoring just a ‘D-.’”

The EU took a step toward legislation loosening restrictions on gene editing in food and feed. The Council of the EU announced Friday it received a negotiating mandate to push for legislation stating “new genomic techniques” that “could occur naturally or through conventional breeding methods” would be exempt from strict restrictions the EU places on genetically modified organisms. CropLife Europe praised the decision. Final approval could still take some time, though more is needed to allow Europe to benefit fully from agricultural innovation.

Sylvia Wulf, BIO’s Interim EVP & Head of Agriculture and Environment (pictured above left), moderated a discussion at the World Agri-Tech Innovation Summit. According to Pairwise—whose CEO Tom Adams spoke on the March 11 panel—it was “a dynamic panel that touched upon the importance of consumer transparency around gene-edited crops and how CRISPR unlocks year-over-year yield improvements and disease resistance.”

BIO member Pivot Bio’s CEO Chris Abbott joined AgriPulse’s Open Mic Podcast. Abbot explains how his company uses soil microbes to increase nitrogen uptake and improve plant nourishment without using chemicals.

Are you drinking green beer today? Thank biotech! To celebrate St. Patrick’s Day, check out our Bio.News archive piece about beer making as a basic biotech art and the origin of the first green beer!

Beltway Report

The House and Senate are off this week after approving a bill on federal funding.