Will we maintain the momentum on new therapy breakthroughs?


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—and could be just as transformative for patients.

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 Alzheimer’s treatments will help them beat back this fatal disease—although they were recently dealt a blow when the Centers for Medicare and Medicaid Services proposed making the class of drugs that target amyloid off-limits for all but a tiny number of Medicare patients enrolled in CMS-approved clinical trials, which could take years to set up.

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. The vast majority of patients are boys and young men who typically don’t survive past their late 20s.

This year could prove even more transformational than 2021.

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Scientists and doctors just transplanted a genetically modified pig heart into a human patient—a technological breakthrough that, if it proves widely replicable, could effectively eliminate wait lists for organ transplants and save hundreds of thousands of lives.

Researchers are also 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%. This experimental HIV vaccine uses an mRNA platform, the same technology that underpins the two most popular COVID-19 vaccines.

These and other success stories are possible only because of a regulatory and policy environment that supports investment in new treatments. 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—from weakening intellectual property protections to prohibiting companies from earning a return—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 (National Institutes of Health) 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.



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