BIO Supports and Seeks Refinements to FDA’s Plausible Mechanism Framework
The FDA has issued draft guidance introducing a Plausible Mechanism Framework to evaluate safety and efficacy of individualized, disease‑targeted therapies when traditional trials are infeasible. The Biotechnology Innovation Organization (BIO) issued a supportive comment letter, praising the agency’s direction while urging practical refinements. BIO’s recommendations call for a broader, modality‑agnostic scope, clearer definitions, flexible CMC expectations, and fit‑for‑purpose post‑approval safety plans. The trade group also seeks operational guidance on how the framework aligns with existing expedited programs.
Parkinson’s Disease in Women: Research Gaps, Treatment Challenges, and New Hope Through GEM-PD
In Women’s Health Month, Bio.News highlighted the systemic gaps affecting women with Parkinson’s disease, from delayed diagnoses to under‑representation in clinical trials. Ragasudha Botta, senior scientific director at C‑Path’s Critical Path for Parkinson’s (CPP), explained how biological sex differences influence...
Patient Advocacy Drives Innovation: James Roe’s Asthma Story Comes to BIO 2026
James Roe, an INDY NXT driver who lives with type 2 asthma, will speak on the BIO Storytelling Stage at the 2026 BIO International Convention in San Diego on June 24. His appearance follows a partnership that places his Topcon‑liveried car, bearing...
State of Play: BIO Coffee Chat Covers How State Policies Impact Access
At BIO’s April Coffee Chat, senior advocates highlighted how state legislation is increasingly shaping patient access to biotech therapies. They cited the ALS Association’s success in securing a CMS directive that bars Medicare Advantage plans from labeling an FDA‑approved accelerated‑approval...
What Is PDUFA—And Why Does It Matter for Biotech Innovators, FDA & Patients?
Congress will reauthorize the Prescription Drug User Fee Act (PDUFA) next year, renewing the FDA’s authority to collect fees from biotech firms for drug reviews. The user‑fee model supplies a stable budget that underpins faster, more predictable approval timelines—about ten...
BIO on the American Road Tours Gene Therapy Hub in Ohio
BIO President John F. Crowley toured Ohio on April 28, spotlighting the state’s emerging gene‑therapy hub. Researchers at Nationwide Children’s Hospital have delivered two of the FDA’s first eight approved gene therapies for Duchenne muscular dystrophy and spinal muscular atrophy type 1....
BIO Panel at HERS Explores Women’s Health Biotech Ecosystem
The Biotechnology Innovation Organization (BIO) convened a panel at the inaugural Health Executive and Research Summit in San Diego to spotlight the women’s health biotech ecosystem. Speakers noted that only six percent of private healthcare investment goes to women’s health,...
BIO Is Expanding Its Work to Defend IP
The Biotechnology Innovation Organization (BIO) is scaling its intellectual‑property advocacy by creating an IP Task Force and a new Economic Growth, Innovation, and Intellectual Property Committee to steer strategy. BIO is actively lobbying on Capitol Hill, filing amicus briefs, and...
Q&A with National Health Council: Driving Patient-Centred Policy
The National Health Council (NHC), a century‑old coalition of health stakeholders, is championing patient‑centered policy as the U.S. grapples with new payment models and drug‑price reforms. Its advocacy secured patient input in the Inflation Reduction Act’s Initial Price Applicability Year...
BIO Launches ‘Fight of Our Lives’: The Real Stories, Power, and Promise of American Biotech at a Defining Moment
The Biotechnology Innovation Organization (BIO) has launched the "Fight of Our Lives" campaign, using real patient narratives to underscore the impact of American biotech. The initiative features three inaugural stories—a rare‑genetic condition treated with targeted gene therapy, the first CAR‑T...
BIO Coffee Chat Explores Venture Philanthropy
The Biotechnology Innovation Organization (BIO) hosted a Patient Advocacy Coffee Chat highlighting the growing influence of venture philanthropy in biotech. Patient groups are now acting as investors, using capital and disease expertise to de‑risk early‑stage programs and guide trial design....
BIO Coffee Chat: Price Controls Like MFN Harm Access, Increase Costs
BIO’s March Coffee Chat highlighted how recent U.S. drug‑price policies, including the Inflation Reduction Act’s out‑of‑pocket cap and proposed Most Favored Nation (MFN) pricing models, are unintentionally raising patient costs and tightening access. Evidence shows beneficiaries often pay more than...
BIO’s Comments for USTR Report Highlight Global Threats to Intellectual Property
The Biotechnology Innovation Organization (BIO) submitted comments to the USTR’s 2026 Special 301 Report urging stronger global enforcement of intellectual‑property (IP) rights for biotech. BIO argues that weak IP protections in markets such as Colombia, Russia and Brazil create barriers...
Biotech Innovation Makes Inroads Against Bleeding Disorders
Biotech breakthroughs have transformed bleeding disorders from fatal diagnoses into manageable chronic conditions, with extended‑half‑life clotting factors, subcutaneous non‑factor drugs, and emerging gene therapies extending dosing intervals to weeks or months. The National Bleeding Disorders Foundation’s Pathway to Cures fund...
BIO CEO Hails Congressional Report on Threat of China Dominating Biotech
At a March 25 reception marking the first anniversary of the National Security Commission on Emerging Biotechnology report, BIO President John F. Crowley warned that China’s rapid biotech expansion threatens U.S. health security. The commission’s findings cite a 400‑fold rise...