1:45 PM (PT)
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A longstanding priority for both Congress and FDA, there has been a significant evolution in recent years in the ways in which patients’ experiences and preferences have been incorporated into the dru ...Breakout Session
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Orphan and Rare Disease
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In recent years, the US public health apparatus has weathered mounting fiscal stress, shifting political priorities, and deep cuts at the federal level. Proposed reductions to the Centers for Disease ...Breakout Session
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Infectious Disease and Vaccines
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Despite growing calls for pediatric inclusion, many clinical trials, especially in rare diseases, continue to exclude children or delay their enrollment until late stages. Yet, in many rare diseases, ...Breakout Session
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Science and Regulatory Innovation
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3:00 PM (PT)
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HBO Max’s “The Last of Us" captivated audiences with its gripping portrayal of a post-apocalyptic world ravaged by a fungal infection. While fictional, the threat posed by fungal pathogens is real. Fu ...Breakout Session
- Infectious Disease and Vaccines
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The biotech and pharmaceutical sectors are undergoing rapid transformation, driven by advances in digital health, artificial intelligence, personalized treatments, and cell and gene therapies. These b ...Breakout Session
- Science and Regulatory Innovation
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4:15 PM (PT)
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As the biopharmaceutical industry strives to address areas of high unmet medical need, there is increasing reliance on novel endpoints to accelerate and support drug development. In rare diseases, tra ...Breakout Session
- Science and Regulatory Innovation
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More than 10 million Americans live with osteoporosis. For decades, proving whether a new osteoporosis drug worked meant running massive clinical trials that followed thousands of people for 10–12 yea ...Breakout Session
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Translational Research
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Influenza has long served as the backbone for the United States’ annual respiratory disease response and pandemic preparedness efforts. Unfortunately, the flu continues to claim hundreds of thousands ...Breakout Session
- Infectious Disease and Vaccines
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Over the past year, the Federal government has prioritized action and changes to federal vaccine policy. These recent shifts—including changes to vaccine recommendations, decision-making processes, , ...Breakout Session
- Infectious Disease and Vaccines
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11:00 AM (PT)
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Europe is actively working to improve the clinical research environment, with particular emphasis on supporting biotechnology innovation and adapting to evolving scientific and societal needs. As new ...Breakout Session
- Science and Regulatory Innovation
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Messenger RNA (mRNA) medicines are increasingly recognized for their transformative power to improve patient health, as well as a strategic asset with implications for national and global security. Th ...Breakout Session
- Infectious Disease and Vaccines
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1:45 PM (PT)
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Recent changes in FDA’s leadership and approach have caused some concern among industry leaders and patients about the stability, predictability, and reliability of our regulatory framework. While our ...Breakout Session
- Science and Regulatory Innovation
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Clinical trials in small populations, particularly those with progressive or degenerative rare diseases, face scientific and ethical hurdles that challenge traditional trial frameworks. High phenotypi ...Breakout Session
- Orphan and Rare Disease
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3:00 PM (PT)
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This session explores how patient preference studies (PPS) are transforming regulatory strategies in drug development, aligning with the upcoming ICH E22 guideline - the first international guideline ...Breakout Session
- Science and Regulatory Innovation
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Respiratory syncytial virus (RSV) is a leading cause of severe respiratory illness globally and a major driver of hospitalization in infants under one year. The introduction of RSV immunization for in ...Breakout Session
- Infectious Disease and Vaccines
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4:15 PM (PT)
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HHS secretary mandates NIH and FDA federal agencies to implement immediately the FDA Modernization Act which aims to establish a new roadmap to reduce animal testing in preclinical safety studies. Suc ...Breakout Session
- Translational Research
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Congress created the Rare Pediatric Disease Priority Review Voucher (PRV) program to incentivize the development of drugs for serious or life-threatening rare diseases in children. Vouchers are earned ...Breakout Session
- Orphan and Rare Disease
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The landmark ORPHAN Cures Act has catalyzed investment in rare disease research since its passage last year, bringing hope to millions of patients and their families. Early data demonstrates renewed p ...Breakout Session
- Orphan and Rare Disease
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Super SessionJoin us for a moderated discussion with the acting directors of the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) of the U.S. Food and Drug ...Super Session
- Science and Regulatory Innovation
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11:00 AM (PT)
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New Approach Methodologies (NAMs) are playing an increasingly central role in biomedical and life-science research, while also breaking scientific and regulatory boundaries by moving beyond early adop ...Breakout Session
- Translational Research
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The role of vaccines in preventing and eradicating numerous infectious diseases is unparalleled; however, the past year has presented some new challenges for the industry, calling into question the sc ...Breakout Session
- Infectious Disease and Vaccines
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Super SessionThis super session will explore the latest developments in UK policy and advances in the UK's regulatory approach to biotechnology and biopharmaceutical innovation. It will feature a moderated discuss ...Super Session
- Science and Regulatory Innovation
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1:45 PM (PT)
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The April 2025 Food and Drug Administration Roadmap to Reducing Animal Testing in Preclinical Safety Studies is a signature effort to shift the prevailing drug development paradigm. Aiming to reduce c ...Breakout Session
- Science and Regulatory Innovation
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4:15 PM (PT)
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Enacted in 1992, the Prescription Drug User Fee Act (PDUFA) establishes how regulated industry fees support the human drug review program at the Food and Drug Administration (FDA) and associated Agenc ...Breakout Session
- Science and Regulatory Innovation
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9:00 AM (PT)
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This session brings together leaders from early-to-late-stage biotech’s and the investment community to explore practical frameworks for transforming early-stage discoveries into development-ready the ...Breakout Session
- Translational Research
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Even though orphan drugs currently account for almost 50% of all new medicine approved in USA and EU, many conditions still lack therapeutic options, and it has been estimated that 90-95% of the more ...Breakout Session
- Orphan and Rare Disease
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10:15 AM (PT)
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The path from academic discovery to patient impact has never been more complex. Federal funding shifts, market turbulence, and evolving expectations for commercialization are challenging traditional m ...Breakout Session
- Translational Research
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Sponsors routinely collect patient experience data (PED) to better understand disease areas and ensure development programs meet patient needs. To advance patient-focused drug development effectively, ...Breakout Session
- Science and Regulatory Innovation
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