Janet Woodcock

Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial

Marshall L. Summar, Janet Woodcock

Clinical trials for rare diseases face a fundamental mathematical challenge that conventional randomized controlled trial (RCT) designs cannot overcome. With approximately 95% of the estimated 10,000-16,000 rare diseases lacking approved therapies, and drug development programs failing at rates exceeding 75% in non-oncology …

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The Placebo Effect in Rare Disease Clinical Trials: Measurement, Impact, and Statistical Approaches for Patient‐as‐Own‐Control Designs

Marshall Summar, Janet Woodcock

A frequently cited concern regarding patient-as-own-control trial designs in rare disease is the potential for placebo and related effects to inflate apparent treatment efficacy. Whether this concern is disqualifying or manageable has not been systematically evaluated. We reviewed meta-analyses quantifying placebo effect …

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A Threat to Evidence-Based Vaccine Policy and Public Health Security at the FDA

Robert M. Califf, Scott Gottlieb, Mark McClellan, Brett P. Giroir et autres

Twelve former commissioners of the FDA express concern that the agency’s recent moves will undermine a regulatory model designed to ensure vaccine safety, effectiveness, and availability.

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A Threat to Evidence-Based Vaccine Policy and Public Health Security at the FDA

Robert M. Califf, Andrew C. von Eschenbach, Michael Friedman, Brett P. Giroir et autres

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The US regulatory and pharmacopeia response to the global heparin contamination crisis

David A. Keire, Robert J. Linhardt, Roger Williams, Christian Viskov et autres

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The NIH-led research response to COVID-19

Francis S. Collins, Stacey J. Adam, Christine M. Colvis, Elizabeth Desrosiers et autres

Investment, collaboration, and coordination have been key.

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Benefits of Streamlined Point-of-Care Trial Designs

Robert M. Califf, Patrizia Cavazzoni, Janet Woodcock

This Viewpoint discusses the benefits of streamlined point-of-care trial designs in clinical research, using lessons learned from the UK RECOVERY study of dexamethasone dosing for patients with COVID-19 and hypoxia.

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Correction: Applicability of in vivo staging of regional amyloid burden in a cognitively normal cohort with subjective memory complaints: the INSIGHT-preAD study

Fatemah A. Sakr, Michel J. Grothe, Enrica Cavedo, Irina Jelistratova et autres

Background Current methods of amyloid PET interpretation based on the binary classification of global amyloid signal fail to identify early phases of amyloid deposition. A recent analysis of 18F-florbetapir PET data from the Alzheimer’s disease Neuroimaging Initiative cohort suggested a hierarchical four-stage …

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COVID-19 Vaccination—Becoming Part of the New Normal

Peter Marks, Janet Woodcock, Robert M. Califf

In this Viewpoint, authors from the FDA discuss how COVID-19 vaccination is an integral part of establishing a new normal, now that SARS-CoV-2, the virus that causes COVID-19, will likely circulate worldwide for the foreseeable future.

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Racial and Ethnic Representation of Participants in US Clinical Trials of New Drugs and Biologics—Reply

Milena Lolic, Richardae Araojo, Janet Woodcock

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Racial and Ethnic Representation in US Clinical Trials of New Drugs and Biologics, 2015-2019

Milena Lolic, Richardae Araojo, Melvyn Okeke, Janet Woodcock

Racial and Ethnic Representation in US Clinical Trials of New Drugs and Biologics, 2015-2019Other Pacific Islander, unknown/unreported/missing, other race, and multiracial.c The percentages of the categories Native Hawaiian or Other Pacific Islander and 2 or more races are not presented in the …

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Reducing Sodium Intake in the US

Susan T. Mayne, Robin A. McKinnon, Janet Woodcock

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