The Prescription Drug User Fee Act (PDUFA), enacted in 1992, has transformed the U.S. drug approval process by providing the FDA with essential funding from biopharmaceutical companies. Critics often argue that this funding creates conflicts of interest, claiming it “corrupts” the FDA. One such narrative is presented in another book published this week, which misrepresents … Read more
Clinical trials with high-risk medical devices frequently involve historical controls and other criteria as concurrent control groups in such studies are not feasible or ethical. A survey of about 100 high-risk medical devices approved by the FDA over five years shows some interesting trends in using non-concurrent controls in medical device trials. Between 2019 and … Read more
Collecting data from both male and female volunteers is critical for appropriate evaluation of clinical trial outcomes. FDA’s latest guidance document outlines what the FDA expects when it comes to enrolling male and female participants, analyzing results by sex, and sharing this information in clinical study reports. Although this issue has been discussed extensively for … Read more
The FDA has announced the strengthening of a law signed in 2022 to reduce animal testing by creating a formal policy to require the use of AI models, lab-grown human tissues or organelles, and international safety data, if available, to replace traditional animal testing. The “shift” aims to “improve accuracy, reduce costs, and accelerate the … Read more
The laboratory testing industry is breathing a collective sigh of relief this week after a federal court struck down the Food and Drug Administration’s (FDA) final rule aimed at increasing regulatory oversight of laboratory-developed tests (LDTs). This ruling marks a significant victory for labs, who argued that the FDA overstepped its authority in attempting to … Read more
A lot has been written about the potential of Real World Data (RWD) and Real World Evidence (RWE) over the last decade. In the latest guidance from a regulatory agency, the European Medicines Agency (EMA) released a reflection paper on using RWD derived from non-interventional studies (NIS) to generate RWE for regulatory purposes. It emphasizes … Read more
Accurate data is crucial for the FDA to evaluate the safety of medical devices properly. If the data is flawed or falsified, it raises risks for patients, including serious health issues like toxic reactions or organ failure. This problem could ultimately compromise public health, as the FDA relies on trustworthy data to assess risks associated … Read more
In the field of medicine, the integrity of clinical data is crucial for ensuring that patients receive effective treatments. Randomized controlled clinical trials (RCTs) are considered the gold standard for evaluating the efficacy of new drugs because they offer the most reliable evidence by eliminating bias and allowing for direct comparisons between treatments. While the … Read more
For more than four decades, government grants have been a cornerstone of early-stage drug development, providing crucial non-dilutive funding that has fueled tens of thousands of programs. They’ve been vital in translating innovative scientific discoveries into life-saving therapies. However, the current political climate introduces an element of uncertainty regarding the future availability and consistency of … Read more
The FDA released a user guide, the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) based on case studies highlighting key concepts and regulatory considerations crucial for successful rare disease drug development programs. The case studies present successful approaches used by sponsors in designing and conducting their rare disease drug … Read more
