iwewlkws When we hear about data integrity issues these days, the obvious perception is of a facility in India or another foreign country getting caught trying to manipulate results. However, for a change (pun intended) this week we are talking about Theranos, a diagnostic lab in California with branches in Arizona and New Jersey, with serious data … Read more
This week FDA’s Advisory Committee (Ad Com) voted to reject Sarepta Therapeutics’ new drug for Duchenne Muscular Dystrophy (DMD) despite emotional pleas from patient groups and physicians in support of the drug. DMD treatments are eligible for most of FDA’s expedited approval pathways such as orphan drug designation, fast track designation, priority review status, and breakthrough designation. Sarepta had … Read more
This week, the Seattle-based gene therapy company, BioViva, made a bold claim to have invented the world’s first “successful” gene therapy for aging. The evidence for efficacy and safety of its product was generated in a manner that raises serious concerns of ethics, good scientific practices, and regulatory oversight. What is clearly a publicity stunt may … Read more
Almost every week FDA make public announcements about dietary supplements with risky ingredients such as unapproved drugs, hidden drug ingredients, dangerous chemicals and other ingredients. FDA’s web-site lists more than 700 such dietary supplements that are sold in the US market. The list is published based on tests conducted at FDA’s labs. However, very few retailers and distributors of … Read more
Data integrity issues have plagued manufacturing sites in India for several years. Repeatedly GMP sites in India were found in violation of documentation practices, data manipulation, data omission, and other issues that raise major issues about reliability of data. Although sites in India are not alone in data integrity issues but arguably sites in India received … Read more
A recent report based on a survey of about 1200 physicians provides a critique on how little prescribers know about the process of drug approval by the FDA. According to the report about 90% of the physicians surveyed could not correctly answer all three questions asked about FDA approval process. The survey asked two sets of questions; … Read more
It is well known that the most challenging aspect of running a given clinical trial is finding patients to participate in it. The main reasons are negative public perception of clinical trial participation, poorly designed clinical protocols and lack of planning by the clinical trial managers. And regulators are trying to help change that. Last … Read more
Regenerative medicine was identified as one of the top priorities for the US government in a report from the Subcommittee for Advanced Manufacturing of the National Science and Technology Council that advises the US President. The report acknowledged that cell-based medicine holds great potential to address critical medical issues related to cells, tissues, and organs particularly in … Read more
Last month in a rare bipartisan move, a new bill was introduced in the Congress to provide conditional approval to regenerative medicine products intended primarily to affect how FDA regulates the Human Cell, Tissue or cellular or tissue-based Products (HCT/Ps). The proposed bill, called the “Reliable and Effective Growth for Regenerative Health Options that Improve Wellness”, or … Read more
Genetic tests indicating risk of diseases based on genetic markers have gained in popularity and consumer acceptance over the last decade although regulators have frequently questioned the accuracy and clinical benefit of such tests. Genetic tests on otherwise healthy individuals to look for genetic markers and predicting the likelihood of getting a given disease are … Read more
