By Michelle McMurry-Heath
Scientists had an astoundingly productive 2021. The Food and Drug Administration fully approved more than 50 new drugs — one of the best years on record — and granted emergency use authorization to numerous other therapies, including antiviral pills that reduce the risk of death from COVID-19 by up to 89%.
Those COVID therapeutics and vaccines generated the biggest headlines. But other breakthroughs were just as scientifically impressive.
Consider the new once-a-month injectable treatment for HIV-positive adults. It will make it easier for folks to adhere to their medications, stay healthy and avoid infecting others.
Then there is a first-of-its-kind treatment that protects bone marrow from damage caused by chemotherapy. The drug could eliminate one of the most harmful side effects of chemotherapy, ensuring patients can complete their treatment regimens.
We also had the first new treatment for Alzheimer’s since 2003. Millions of patients, and their families, are hoping advances in new treatments will help them beat back this fatal disease.
Researchers made huge strides in the fight against rare diseases, too. Early last year, federal regulators gave the green light to the first drug to treat a specific genetic mutation present in 8% of patients with Duchenne muscular dystrophy, a fatal disease that causes patients’ muscles to gradually waste away.
This year could prove even more transformational than 2021.
Researchers are on the verge of a breakthrough in treating amyotrophic lateral sclerosis (ALS), often known as Lou Gehrig’s disease. Late last year, the FDA granted priority review to the medicine, and a decision could be made by this summer.
And there’s a real possibility that we will finally achieve a vaccine for HIV. Scientists announced in December that they had developed a vaccine for a virus similar to HIV that proved safe and effective in animals. Preliminary results showed that the jab reduced risk of infection by 79%.
With scientists on the verge of so many breakthroughs, it’d be enormously counterproductive for policymakers to press forward with well-intentioned but poorly conceived legislation and executive actions that would cause private-sector investment in research endeavors to dry up.
That would be cataclysmic for patients, since private capital drives nearly all drug development. According to a recent study, “23,230 NIH Grants in the year 2000 were linked by NIH-supported patents to 41 investigational drugs, only 18 of which gained FDA approval by 2020.” Of those 18 medicines approved in 2020 that benefited from federal grants, $44.2 billion of the funding needed to bring them to market came from the private sector, and just $670 million came from the NIH.
In other words, private companies funded 98.5% of the cost of developing those drugs.
Lifesaving medical breakthroughs don’t happen by accident. They’re a direct consequence of good public policy. Skeptics need only look at Europe — which used to develop over half the world’s new medicines as recently as the 1970s, but now invents barely a third, while America creates nearly two-thirds of new drugs — for proof of the damage wrought by poorly thought-out policies.
America’s scientists are poised to make 2022 another banner year — as long as policymakers don’t throw a wrench in their work.
Dr. Michelle McMurry-Heath is a physician-scientist and president and CEO of the Biotechnology Innovation Organization.
This piece was originally published in Modern Healthcare (ModernHealthcare.com).
