Writing about new drugs sounds easy. The Food and Drug Administration (FDA) has approved more than 700 drugs in just the past two decades. But how to describe this phenomenal accomplishment in just a few words? Describing a new epilepsy drug, for example, can consume thousands of words.
So let's make the task selective. Let's not talk about vaccines (whatever happened to smallpox?) or fancy injectable drugs like pembrolizumab (it's really good for cancer, but it's hard to pronounce!). Let's just talk about plain old pills — pills we just take by mouth and swallow.
Most of those 700 approvals are pills. Aspirin is a pill — and one aspirin tablet contains about 1,670,000,000,000,000,000,000 molecules. The challenge to the pharmaceutical world is to design new molecules that make a different pill that will, in some way, alleviate human disease. In the past twenty years we learned how to do this the way an engineer designs a key, with a specific lock already in mind.
Let’s focus on just two new pills that are absolute game-changers — one you will know, and one that most people have never heard of.
The first is Wegovy and Ozempic. You have heard these names. These are GLP-1 drugs, and the newest versions are pills. They work by mimicking a hormone your intestine releases after eating — telling your brain that you are full. These pills can decrease body weight by fifteen to twenty percent, numbers previously seen only with surgery. About 40% of American adults are obese, and almost 75% are overweight — driving heart disease, diabetes, joint failure and more, at a cost to the US of more than $100 billion annually. These pills improve diabetes, reduce heart attacks, and even improve sleep apnea. They are already revolutionizing health care — and we are just getting started.
The second pill is Harvoni — and this is the one most people have never heard of. Harvoni is a specific antiviral pill that targets Hepatitis C, a virus that silently attacks the liver for decades, causing cirrhosis, liver failure, and death. Before 2014, an estimated 3.5 million Americans were living with the infection — most without knowing it — and nearly 20,000 were dying from it every year. It was the leading cause of infectious disease death in the United States. Then came Harvoni, a once-daily pill that shuts down the Hepatitis C virus's inner machinery with extraordinary precision. Cure rates above 95% with eight to twelve weeks of treatment. Since its arrival, Hepatitis C death rates have fallen by nearly 40% — and are still dropping. A disease that was killing 20,000 Americans a year is headed toward elimination — with a pill.
We have only discussed two pills out of more than 700 approved by the FDA in the past twenty years. The pipeline ahead — targeting Alzheimer's, cancer, rare genetic diseases and more — will surely make even these two remarkable pills look like just the beginning.
Dr. Roger J. Porter, MD, is a neurologist who spent 20 years at the National Institutes of Health and 10 years as a vice president at Wyeth, then a multibillion dollar pharmaceutical company. He has written more than 200 scientific articles and 13 books, mostly on advances in epilepsy diagnosis and therapy. He also created and managed a White House Committee on brain research. He and his wife Candace live in Fayston.
