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The Power of Inclusion: Navigating the FDA’s Diversity Action Plan

In December 2022, Congress passed the Food and Drug Omnibus Reform Act (FDORA), which requires, among other things, that drug and device sponsors submit diversity action plans for clinical trials that commence enrollment 180 days after publication of the final guidance. FDORA codifies a long-standing push by the US Food and Drug Administration (FDA) to…

Building a Better CAR: Emerging Technology and Safety Concerns Shake Up the Field

When chimeric antigen receptor (CAR)‑T cells first rolled off the assembly line 7 years ago, with the approval of Kymriah, they were the newest and most technically advanced immunotherapy against cancer, and they demonstrated the potential to elicit profound and durable responses in patients with relapsed/refractory hematologic malignancies. The patient’s own (autologous) T cells are genetically modified in…

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New Weapons Emerge in the Fight Against Respiratory Syncytial Virus (RSV)

Imagine your newborn baby has been hospitalized with a serious respiratory infection and is struggling to breathe. That’s a reality for thousands of US newborns and their parents every year during the respiratory syncytial virus (RSV) season. This common endemic virus doesn’t cause clinically significant disease in most children and adults, but it can cause…

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FDA Sets High Bar for Real-World Evidence in Rare Diseases

Real-world data (RWD) can be used to create historical control groups for clinical trials in rare diseases where a randomized controlled trial (RCT) is not feasible. But what happens when the US Food and Drug Administration (FDA) doesn’t accept it? Since passage of the 21st Century Cures Act in 2016, FDA has promoted the use…

COVID-19 Vaccines: When Will the Pandemic End?

As Pfizer, Moderna, and other pharma companies prepare to seek emergency authorization for their SARS-CoV-2 vaccines, the FDA has laid out a roadmap designed to ensure appropriate scientific rigor and help engender public trust. That plan was the subject of a special meeting of the Vaccines and Related Biological Products Advisory Committee (VRBPAC) on October…

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The Target Product Profile—Your Blueprint for Drug Development

When utilized to its full potential, the Target Product Profile (TPP) is a dynamic, living document that ensures all stakeholders—clinical, regulatory, quality and manufacturing, commercial, market access, and medical affairs—are working from the same blueprint. Unfortunately, the TPP often has a bad rap within industry because many people think it is too rigid for today’s…

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Mapping a Successful Path to Label Optimization

Bringing a drug to market is a long and expensive process. An analysis by the Tufts Center for the Study of Drug Development estimated the total cost of development from discovery to commercialization at $2.6 billion over the course of about 10 years (based primarily on big pharma companies). This represents more than a 10‑fold…

Making Sense of FDA’s Expedited Drug Approval Pathways and Designations – for the Non-Regulatory Professional

One of the fundamental responsibilities of the US Food and Drug Administration (FDA) is to approve effective medicines for people who need them, while upholding high standards for safety. That mission also demands that the FDA work efficiently and not delay approval of life-saving medical advances. Today, the FDA is reviewing applications for approval of…

Implications of ICMJE’s Data Sharing Proposal

On January 20, members of the International Committee of Medical Journal Editors (ICMJE) announced a proposal that would require the authors of clinical trial publications to share the deidentified individual patient data that support their published results within 6 months of publication. Announced in an editorial published simultaneously in multiple medical journals, this proposal is…

President Obama’s Cancer Moonshot 2020 Initiative: How Big Is the Challenge?

When President Obama’s White House Task Force meets for the first time this week, it faces a lofty charge: “Let’s make America the country that cures cancer once and for all.” His proposed Cancer Moonshot 2020 initiative will provide much needed funding for research, will encourage collaboration between industry and academia, and will undoubtedly lead…