Congressional roundtable warns of AMR risks

A new week, and more reasons to support AMR R&D and the Pediatric Disease Priority Review Voucher (PPRV) program. (752 words, 3 minutes, 45 seconds)

Congressional roundtable warns of AMR risks, promotes legislative solution

Antimicrobial resistance (AMR) threatens our public health, economy, and national security, and legislation is required to address this threat, said members of a bipartisan congressional roundtable last week.

Why it matters: With drug-resistant “superbugs” contributing to nearly 5 million deaths annually, we need novel antimicrobials to fight infections. However, the market for these drugs is broken.

The challenge: “We have the best physicians, the best facilities, the best technology,” said Rep. Jodey Arrington (R-TX), Chair of the House Budget Committee, whose Health Care Task Force organized the roundtable, “but we just can't seem to put it all together.”

The public health threat: “Should safe and effective antibiotics disappear, innovative medical treatments will need to be reconsidered—and decades of medical technology advancements that improve health outcomes of patients will be undone,” warned Rep. Drew Ferguson (R-GA) in an Epoch Times editorial.

The economic threat: “The market doesn't work here,” said Rep. Scott Peters (D-CA), who co-sponsored the PASTEUR Act with Rep. Ferguson. “We are trying to figure out a way to make it make sense that people will create new antibiotics.”

The legislative solution:The PASTEUR Act would help provide financial stability to companies developing new antimicrobials and encourage and sustain more innovation in the AMR ecosystem. BIO supports the legislation.

Report demonstrates value of Pediatric Disease Priority Review Voucher (PPRV) program

The Pediatric Disease Priority Review Voucher (PPRV) program has a “tangible impact on the rare disease patient community” and needs timely reauthorization, according to a National Organization for Rare Disorders (NORD) report.

Why it matters: “As many as half of all people living with a rare disease are children, and the [PPRV] program offers a crucial incentive for companies to develop therapies for these particularly challenging to study patient populations,” says NORD’s report, released last week.

How it works: Companies gaining approval for drugs targeting rare pediatric diseases can be awarded a voucher granting expedited review of another drug in the future. Drug makers can also fund future research by selling the voucher to another company.

The key findings:

• “53 PRVs have been awarded across 39 rare pediatric diseases,” including typically fatal diseases.
• Before the PPRV program, “only three of these 39 rare pediatric diseases had any FDA-approved treatments.”
•  “23 PRVs have been redeemed for priority review of 21 different drugs for a variety of diseases, including six drugs to treat rare diseases.”
• The impact is increasing: “More than half of all [PPRV] designations, awards and redemptions occurred in the last four years.” 

BIO’s view: “The program demonstrates how smart government policy, combined with the scientific skill and dedication of America’s biotechnology industry, can lead to innovative new medicines,” says BIO President & CEO John F. Crowley. “As a father of two children with a rare disease, I am grateful that lawmakers are working together, on a bipartisan basis, to continue this vital program and hope it’s signed into law as soon as possible.”

What’s next: The 12-year-old program expires Sept. 30. Legislation to reauthorize it has been introduced in the House and Senate.

What Else to Know This Week

BIO member BeiGene opened its new flagship U.S. facility in Hopewell, NJ. BeiGene focuses on solid tumor and hematology drug development. The $800 million, 42-acre facility with 400,000 square feet of interior space will house integrated manufacturing and R&D facilities. The facility will add hundreds of jobs to the Trenton-Princeton region, which boasted 3,701 jobs in drugs and pharmaceuticals in 2021. Read more at Bio.News.

The recombinant vaccine for shingles significantly lowers the risk of dementia, a new study found. “Specifically, receiving the recombinant vaccine is associated with a 17% increase in diagnosis-free time, translating into 164 additional days lived without a diagnosis of dementia in those subsequently affected,” the study published in Nature found. The recombinant vaccine Shingrix, made by BIO member GSK, is currently the only approved vaccine available for shingles in the U.S.

Beltway Report: What's Ahead in Washington

President Biden’s Monday: Commemorating the 60th Anniversary of the Civil Rights Act with a visit to the LBJ Library in Austin, TX, before paying respects to Congresswoman Sheila Jackson Lee in Houston. He plans to unveil a proposal with term limits and other reforms for the Supreme Court, POLITICO reports.

Capitol Hill This Week: In the last week before recess, the Senate Commerce Science and Transportation Committee is scheduled to mark up dozens of bills Wednesday, including bills impacting innovation with artificial intelligence and one to establish the National Integrated Heat Health Information System within the National Oceanic and Atmospheric Administration. The Senate Appropriations Committee markup Thursday will include the Labor, Health and Human Services Appropriations Act.