Prahsant Kumar A new drug can affect multiple aspects of the disease it is intended to treat, and hence clinical trials are designed to evaluate multiple efficacy endpoints compared to the controls. However, evaluating multiple endpoints in a clinical trial should involve appropriate statistical adjustments to account for the existence of multiple comparisons, called the multiplicity effect. … Read more
Today FDA announced a new program for advancing Real World Evidence (RWE) aimed to develop new approaches for using RWE for labeling changes, new indications for previously approved drugs, or to support post-market commitments. The program will select a few sponsors who want to use RWE for the above purposes and use the experience and … Read more
A diabetes drug with a side-effect of weight loss has become popular for its off-label use as a weight-loss drug presenting a classic test case where medical experience could have potential applications for RWE-driven new indications. The drugs of the class GLP-1 RA (Glucagon-Like Peptide 1 Receptor Agonist) are approved for the management of Type … Read more
For the last few years, rules governing clinical trials funded by government grants are being harmonized with the rules for privately funded trials. Most of the government funded trials have traditionally required additional burdensome processes that limited their patient access without additional subject protection or other benefits. The rules changes were designed to address those … Read more
Reviewers of papers submitted to journals can be biased towards authors with established credentials; for example, an article with a Nobel Laurette as an author is about 6 times more likely to get published without any significant comments compared to one by novice authors. This can be addressed by anonymizing the authors of the publication. … Read more
For developers of medical devices using artificial intelligence (AI) and/or machine learning (ML) tools, precedent information is critical. Being a new field, there are few public resources to plan and strategize development steps for FDA approval. FDA’s list of AI/ML-enabled medical devices is hence a priceless gift to the developers. FDA has been maintaining this … Read more
This week the US Congress unanimously cleared the Bill S.5002 which requires FDA to allow alternatives to animal testing for drug and biological applications. While there is a strong bi-partisan political support in the US for this bill, in practicality it may have minimal impact on the animal tests required by FDA. Animal testing is … Read more
Including children in clinical trials poses unique challenges and FDA’s latest guidance document on the topic acknowledges and highlights all the reasons why so few trials are done in pediatric subjects. The guidance confirms the conventional wisdom that trials in children must be adequately justified, properly designed to minimize risk to children, and avoided if … Read more
This week, FDA formally announced that it has stopped accepting Emergency Use Authorizations (EUAs) for most Covid diagnostic tests. While this should not come as a surprise to most, this announcement should settle this issue for anyone still hoping to submit an EUA for such tests. EUAs are an important tool for rapidly releasing unapproved … Read more
This week, President Biden’s statement that the “pandemic is over” created a media storm. Public health experts were split on if the statement was premature or if it is time to move on from the classical definition of the pandemic. From a regulatory point of view, the statement from the President could indicate a significant … Read more
