Unbiased Analysis of Today's Healthcare Issues
FDA wants drugs to be more targeted to factors that impact patient’s lives in ways that they care about. To achieve this goal, last month FDA released a third guidance document on patient focused drug development generally applicable to a variety of clinical outcome assessments (COAs), including patient-reported outcome (PRO), observer-reported outcome (ObsRO), clinician-reported outcome…
When new drugs are approved, FDA limits their on-label use to the treatment of specific diseases. A key question is whether the definition of a disease should be narrower or broader. Make the disease definition too broad, then there may not be strong evidence from the clinical trial to support its use among these types…
What disease components are most burdensome to patients? How do researchers find this information out? To answer this question, the Food and Drug Administration (FDA) recently put out its second guidance document on Patient-Focused Drug Development: Methods to Identify What Is Important to Patients. A few qualitative approaches they recommend to collect this information include:…
Today, the Office of Generic Drugs (OGD) at the Food and Drug Administration (FDA) released its 2021 Annual Report. As noted in the report, 90% of all drugs dispensed in the US are generic. In 2021, there were 776 approved generic drugs through the Abbreviated New Drug Applications (ANDA) process. Approvals by month are below.…
Recently, the Food and Drug Administration (FDA) approved Prograf (tacrolimus) in combination with other immunosuppressant drugs for the new indication of preventing organ rejection for patients who have received a lung transplant. While the FDA approving a new indication isn’t typically news, this is a noteworthy event because (i) it marks the first approval of…
FDA draft guidance published this month says you should. In most cases, adjusting for covariates is not necessary. Randomization generally insurers that covariates are balanced across clinical trial arms. Randomization, however, may not always result in perfectly balanced trial arms. In these cases, the FDA notes that covariate adjustment is perfectly acceptable. There are some…
Diversity in clinical trials Recently, the FDA’s Center for Drug Evaluation and Research’s (CDER’s) released its 2020 Drug Trials Snapshots Summary Report. Part of the report examines the demographics of participants in clinical trials for the 53 drugs that FDA approved in 2020 either as new molecular entities (NMEs) under new drug applications (NDAs) or…
This is the question that a recent paper by Chorniy et al. (2020) attempt to answer. This issue is clearly very relevant as the UK recently has approved the Pfizer/BioNTech vaccine for COVID-19 before the US. Unlike most studies that attempt to examine the relationship between FDA review time and R&D investment dollars, the authors…
FDA faces an near existential crisis: digital. New digital technologies have the capability to improve the way care is delivered to patients. The question is, how do we make sure it is safe? The FDA is certainly good at making sure drugs and devices are safe; in the case of digital however, it is less…
The priority drug review process was enacted in 2012 in order to stimulate drug development for rare pediatric diseases. Drugs granted priority review had the typical ten-month FDA review timeline shortened to 6 months. For a drug to be eligible for this program, it must not only be for a rare disease but it must…