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We’re back on a Tuesday, with news about the Rare Pediatric Disease Priority Review Voucher program and the BIOSECURE Act—plus, the season opener of the I am BIO Podcast. (725 words, 3 minutes, 37 seconds) |
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CSBA urges passage of PPRV program for rare diseases in children |
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The Council of State Bioscience Associations (CSBA) sent a letter to congressional leaders yesterday urging the reauthorization of the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program before it expires at the end of September.
Why it matters: “Rare diseases affect a significant number of individuals, with nearly half of those affected being children. While treatments exist, options remain limited due to high cost of development and utilization by a smaller section of the patient population,” said CSBA, a coalition of independent state and territory-based non-profit trade associations affiliated with BIO.
How it works: “A drug developer who receives approval for a drug or biological product for a rare pediatric disease may qualify, if it meets certain criteria, for a voucher that can be redeemed to receive priority review for a different product.”
The benefits: Faster market access from priority review benefits drug makers. Vouchers can also be sold to another drug maker, and the funds can support further development.
How it has helped: “Since the start of the RPD PRV program, 53 vouchers for 39 rare pediatric diseases have been awarded”—36 of which had no previous treatment. Over 200,000 patients have benefitted, according to the letter.
The program has broad support: “This program has been reauthorized with bipartisan support twice since its inception, both in 2016 and 2020,” said the letter.
The next steps: “We urge your timely support in reauthorizing the bipartisan, bicameral Creating Hope Reauthorization Act before it expires on September 30, 2024. A lapse in this program will negatively impact some of the most vulnerable patients suffering from diseases for which there are no cures or treatments,” said the letter.
More reading: Joe and Courtney Dion, the parents of two children with limb-girdle muscular dystrophy 2C, explain in STAT News why the program needs to be reauthorized. |
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I am BIO Podcast: A doctor fights to save his own life |
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AI can match existing medicines with known diseases to discover new treatments, explores today’s season opener of the I am BIO Podcast.
It began with one doctor: After discovering he had Idiopathic Multicentric Castleman disease in med school—a disease that brought him to the brink of death five times—Dr. David Fajgenbaum researched his own body. “I got a signal that a particular communication line in the immune system called mTOR was turned into overdrive,” he says.
It got bigger: Dr. Fajgenbaum has been able to treat himself with an existing drug that addresses mTOR for another condition. He was inspired to start Every Cure, a non-profit that is finding new uses for existing drugs.
Then it got faster, using AI: Applying five different algorithms to match “all 3,000 drugs against all 20,000 diseases, meaning that within one day we can calculate 60 million scores,” says Dr. Fajgenbaum. “If we did that manually the way that we do things in my lab, it would take thousands of years.”
Get the episode at www.bio.org/podcast or your favorite podcast app. |
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“We’re all here today because we know we’re racing against time,” said Emily Wheeler, BIO’s Vice President of Infectious Disease Policy, in her keynote at last week’s World AMR Congress. “Antimicrobial resistance isn’t some far-off problem—it’s here, it’s now, and it’s serious.” Wheeler discussed the importance of passing the PASTEUR Act: “The market for new antimicrobials is fundamentally broken,” she said. “What if we paid for antibiotics based on their value to public health instead of how much we use them? The PASTEUR Act, a bipartisan bill before Congress, offers such a model.” Read more at Bio.News.
BIO joins letter urging congressional leaders to pass the Farm Bill. BIO has been a strong advocate of the Farm Bill, which would support biotech advances in agriculture, promoting food security, sustainable agriculture, and cleaner energy. The letter is signed by 304 national, state, and regional agriculture groups. Read the letter here. |
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BIO's Emily Wheeler speaking at the 2024 World AMR Congress in September 2024 |
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Beltway Report: What's Ahead in Washington
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Yesterday, the House passed the BIOSECURE Act, which requires federal contracts with a “biotechnology company of concern” to end by 2032. When the bill was introduced in the House on May 9, BIO noted that the legislation “highlights a key vulnerability in our global supply chain and importantly provides a reasonable timeframe for companies to decouple their reliance on China-based biomanufacturing.” The Senate must pass the bill before it can become law.
Today, Vice President Kamala Harris will debate former President Donald Trump in Philadelphia, at 9pm ET. |
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