iwewlkws A “strange” news release by Zogenix announced that it received a Refuse-to-File (RTF) for its NDA for Fintepla, a breakthrough therapy designated (BTD) drug for epilepsy. There are many clues in this announcement that could be lessons for others. The strangeness of the news is in the details of the RTF issues: missing chronic non-clinical … Read more
This week FDA reiterated its goal to enforce strict regulation of all stem cells treatment. In the strongly worded blog, FDA emphasized that autologous stem cell treatments are also regulated under the regulations for INDs and BLA and that it is false to claim that FDA jurisdiction over such treatments is not applicable. FDA also … Read more
A joint blog by the FDA Commissioner and the Center Directors for Drug and Biologics this week highlighted the important role expanded access (also known as Compassionate INDs) plays in the treatment of patients with no available options, and the benefits it may provide the manufacturers. Besides being potentially helpful to needy patients, FDA noted … Read more
Minorities represent a small fraction of subjects participating in clinical trials. A good way to improve minority participation in clinical trials could be through investigators from these communities. In an essay published this week, Dr Kyle Mack, a Chicago doctor, who is African-American describes how being from the community opens doors for him to get … Read more
It is hard to conduct placebo-controlled clinical trials, particularly in rare diseases. FDA released a new guidance document last week that provides ways to use historical controls to reduce and possibly eliminate placebo controls in all clinical trials. All diseases have a natural course under available standard of care which can be charted and used … Read more
FDA prefers that legacy clinical trial programs where a given product is tested through long clinical trials with generalized designs be replaced with modernized clinical development programs containing enrichment strategies, adaptive designs, master protocols, and operations supported by mobile technologies. Last week, FDA finalized two older guidance documents, one on enrichments strategies describing ways to … Read more
A recent study by researchers from MIT and Harvard University raise concerns about the risks of adverse reactions due to excipients in drugs, reporting that more than 93% of drugs containing inactive ingredients that could cause allergic reactions. The study, though well-intentioned, could create unreasonable anxiety and fear in patients taking the same drugs. All … Read more
Last week FDA launched a new online portal for patients, physicians, patient advocates and support groups can formally and directly request meetings with the FDA to discuss their disease and ask about specific FDA regulated products. Over the last few years, patients have been able to interact with FDA directly and that has led to … Read more
The clinical trial recruitment criteria must be modernized to accurately reflect measures needed for the safety of the participants, and to diligently evaluate the investigational product. This week FDA published five new Guidance Documents to rebut common exclusion criteria used for all clinical trials, specifically trials in cancer patients. The goal is to eliminate common … Read more
Medical device manufacturers will be able to send their proposed actions to address audit findings to FDA before formally implementing them, thereby avoiding unnecessary back and forth with the FDA. An FDA 483 lists all the findings of non-compliance at the audited site with the requirement that the manufacturer address all findings adequately within a … Read more
