Skip to content

Has the National Institutes of Health Lost Its Way?

Written by Dr. Katherine Roe
March 2023

The National Institutes of Health’s (NIH) mission is “to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability.”

Sounds good.

Unfortunately, NIH seems to be so focused on the first part of its mission that it has lost sight of the second.

NIH spends billions of taxpayer dollars each year seeking fundamental knowledge about nonhuman living systems. And we can certainly credit it with our greater knowledge about the atypical physiology and behavior of laboratory animals living in stressful, unnatural, and impoverished laboratory environments than we had several decades ago.

But what about the second part of its mission? The part about enhancing health, lengthening life, and reducing illness and disability? Where are the new preventions, treatments, and cures for mental health conditions, cancer, Alzheimer’s, sepsis, heart disease, and strokes?

They’re lost in translation.

A full 95% of new drugs deemed safe and effective in animals turn out not to be safe or effective in humans. Animal tests fail to detect the potential side effects of drugs in humans 81% of the time, and 90% of basic research, which NIH deems “fundamental,” has failed to lead to any human therapies within 20 years. Data from up to 89% of experiments can’t even be reproduced in different laboratories, resulting in approximately $28 billion a year wasted on preclinical research that may be inaccurate or misleading. Treatments for strokes and sepsis tested in animals have had a devastating 100% failure rate in humans. Alzheimer’s disease treatments developed in animals fail 99.6% of the time in humans. Oncology drugs tested in animals have a success rate of only 3.4%. Decades of harmful and expensive experiments that involve infecting monkeys and other animals with HIV and similar viruses have yet to produce an effective vaccine for humans.

The abysmal failure rates of animal-based fundamental research to translate into health benefits for humans should set off alarm bells for patients and taxpayers alike. Taxpayers have paid heavily to fund NIH—shouldn’t it return that favor by at least trying to produce meaningful returns on this multibillion-dollar investment?

Despite pleas from patients, animal experts, and forward-thinking scientists, NIH’s research priorities haven’t changed. It seems that somewhere along the way, the agency forgot, or just gave up on, its mission to “enhance health, lengthen life, and reduce illness and disability” in humans.

As Lewis-Fernández et al. wrote, “Publicly funded government agencies are custodians of research for the public good. A diversified research portfolio, balanced between longer- and shorter-term payouts.” A 2021 report by E. Fuller Torrey et al. in Psychiatric Services revealed that 90% of the National Institute of Mental Health’s resources were being spent on basic research and that funding for treatment trials for schizophrenia, bipolar disorder, and major depressive disorder had been reduced by 90% between 2003 and 2019.

The good news is that the newly minted Advanced Research Projects Agency for Health (ARPA-H) program is a beacon of hope for millions of patients who have been waiting too long for meaningful treatments and cures. Patients with rare diseases or diseases that disproportionately affect Black people, indigenous people, other people of color, or women hope the agency will finally make funding research to alleviate their ailments a priority. It should be a breath of fresh air, encouraging innovative, transferable, and usable scientific discoveries—which would be laudable.

What no one seems to be talking about, however, is an obvious but inconvenient truth: We need ARPA-H because NIH, the world’s largest funder of biomedical research, is failing its mandate. Maybe we should just rename it the “National Institutes of Basic Research,” since health is no longer its priority. Maybe it never was.