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…
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…
CRISPR is a powerful gene-editing tool that enables targeted therapeutic gene editing, with clinical applications for improving treatment of inherited and/or rare genetic diseases, viral infections, and arresting the progression of cancer. CRISPR gene-editing technology has the potential to revolutionize the treatment of rare genetic disorders, with the first product poised for FDA approval in…
The field of neurology is experiencing a significant upswing in innovative therapeutic development, propelled by advances in genetics, neuroimaging techniques, and biomarker research. However, neurological diseases are inherently difficult to treat, and there remains an urgent need to rapidly translate these advances into more effective treatments. It is timely then, that several recent drug approvals…
Artificial Intelligence (AI) is rapidly transforming how we work, and the pharmaceutical industry is no exception. In particular, the emergence of Generative Pre-trained Transformer (GPT) models has the potential to revolutionize several aspects of the biotech business, including drug discovery and clinical trials. According to a recent survey, 39% of healthcare professionals see AI (including…
Traditional clinical trials are typically conducted at central locations or sites that patients must travel to in order to be evaluated and treated by trial investigators. These are referred to as centralized clinical trials. In contrast, a decentralized clinical trial (DCT) has no set location for patients to report to. Rather, clinical trial activities are…
On September 30, 2022, President Biden signed into law the reauthorization of the Prescription Drug User Fee Act (PDUFA VII), which will be in place for the next 5 years. Despite extensive bipartisan efforts to include reforms of the accelerated approval (AA) pathway as so-called “policy riders” in the bill, ultimately a “practically clean” version…
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…
The United States is facing an avalanche of Alzheimer’s disease (AD). An estimated 12.7 million Americans over the age of 65 are projected to suffer from AD dementia by 2050,1 and yet, despite more than 30 years of intensive research, we have yet to develop a drug that provides a clinically meaningful slowing in cognitive…
Study protocols are required for every clinical trial. Approximately 20,000 are submitted and posted to www.clinicaltrials.gov every year1—each one different. The format and core content can vary from sponsor to sponsor, costing the US Food and Drug Administration (FDA) time and resources to interpret, review, and ultimately, approve each uniquely complex protocol. This process, as…
