iwewlkws There are two ways patients can get access to experimental drugs outside of participating in a clinical trial: The Right-To-Try (RTT) access or the Expanded Access. FDA prefers patients to use the expanded access program over RTT since that way FDA can better monitor the non-clinical trial use of experimental treatments. FDA’s latest guidance on … Read more
A key application of digital medical tools is in diagnostic applications that rely on artificial intelligence (AI) algorithms. Experts opine that there are critical deficiencies in the methods used for training and validation of AI tools that might over-estimate the accuracy of these products, and hence make them less reliable. These deficiencies must be addressed … Read more
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
