Question 410 of 433
How do I read a “breakthrough” headline?
The short answer
Find out what stage the research is in. Most breakthroughs you read about are years from your pharmacy, and most never arrive.
Laboratory or animal study: an idea. Translation to patient benefit is uncertain and often unsuccessful.
Phase 1: a few dozen people, testing safety.
Phase 2: a few hundred, looking for signs that it works.
Phase 3: larger confirmatory studies, sometimes with hundreds rather than thousands, using clinical or accepted surrogate endpoints and an appropriate comparator. The endpoint still matters.
FDA approved: it works well enough and safely enough for the stated use.
In the guidelines: doctors broadly agree it should be used.
Many candidates that enter human testing never reach approval; estimates vary by disease, period, and definition. So when I describe something below as Phase 1, read it as “interesting,” not as “soon.”
“This book is for education. It is not medical advice, and reading it does not make you my patient or create a doctor-patient relationship.” From the book’s medical disclaimer. Full disclaimer
RESEARCH FRAMEWORK — Trial phases do not guarantee fixed sample sizes, endpoints, or eventual approval.Evidence grade (draft mapping)JClinical judgment / author synthesisPart 28, Q410 · Part sources
From Longevity Unmasked by Afshine Ash Emrani, MD, FACC. Medical disclaimer. This book is for education. It is not medical advice, and reading it does not make you my patient or create a doctor-patient relationship. Nothing here is meant to diagnose, treat, cure, or prevent any disease. Medicine is personal: a test, drug, supplement, or exercise plan that helps one person can harm another. Do not start, stop, or change any medication or treatment because of something you read in these pages. Talk with a licensed clinician who knows your history first. If you think you are having a medical emergency, call 911. The author disclaims responsibility for any loss or harm arising from use of the information in this book.