Developing treatments for ultra-rare diseases has always been one of the toughest challenges in drug development. With small patient populations and limited trial options, many promising therapies stall before reaching patients. The FDA’s new Rare Disease Evidence Principles (RDEP) aim to change that by offering a clearer, faster, and more flexible path to approval. This … Read more
When patients report side effects to the FDA, their experiences become part of a powerful drug safety system. Until recently, those reports took months to appear in the public record, leaving a long gap between patient experience and public awareness. Now, with daily publication of adverse event data through the FDA’s FAERS database, the agency … Read more
Natural products have been trusted for centuries to ease pain, boost immunity, and restore balance to the body. Yet, modern medicine often highlights rare adverse reports while overlooking the vast history of safe, beneficial use. It’s time to reframe the conversation and recognize that “natural” doesn’t always mean unsafe. A commentary published in MedScape raises … Read more
Medical technology is advancing faster than ever, but many lifesaving devices often face years of regulatory hurdles before reaching patients. The FDA’s Breakthrough Devices Program is designed to change that, offering innovators a faster pathway to market while maintaining rigorous safety standards. With over 1,100 designations and 160 authorizations to date, this program is transforming … Read more
In cancer research, nothing is more important than proving whether a treatment helps patients live longer. That’s why “overall survival” (OS) is considered the gold standard in oncology trials—it’s clear, objective, and clinically meaningful. The FDA’s new draft guidance sheds light on how sponsors should design, analyze, and report OS data to ensure that cancer … Read more
Twenty years ago, U.S. politics—not science—slammed the brakes on embryonic stem cell research, delaying lifesaving breakthroughs for a generation. Now, the same playbook is being dusted off—this time targeting mRNA vaccine technology, the very innovation that helped save hundreds of millions of lives during COVID-19. If history is any guide, this short-sighted decision will cost … Read more
Imagine walking out of your doctor’s office with a grocery list and a cooking plan instead of another prescription. At UT Southwestern Medical Center, that’s exactly what’s happening. Through a pioneering culinary medicine program, doctors and dietitians are teaching patients how to cook their way to better health—and the results are turning heads in the … Read more
Investing in quality management isn’t just about regulatory compliance; it’s a strategic move that boosts efficiency, profitability, and public health outcomes. An FDA white paper presents a systematic analysis of how mature quality systems can provide both economic and public health benefits to the industry. There are no surprises in the paper; it provides the … Read more
Observational studies play an important role in medicine by providing insights not available from controlled clinical trials, but many suffer from poor transparency and inconsistent methods. Their potential is often compromised by bias, data dredging, and unverifiable conclusions. Could registering the observational studies address the concerns with their reliability and unlock their full potential to … Read more
Artificial intelligence is increasingly powering your doctor’s decisions. But as AI advice becomes a staple in modern healthcare, a surprising trend is emerging: patients aren’t quite ready to trust it. Artificial intelligence (AI) is making its mark on healthcare, offering unprecedented support in diagnostics, treatment planning, and even behind-the-scenes administrative work. But new research reveals … Read more
