Exceptional medications don’t emerge from thin air

The globe’s most notable medical treatments frequently arise from unexpected origins.

It would have been nearly impossible to foresee that examining the distinctive pancreas of an anglerfish might lead to groundbreaking drugs for diabetes or that the identification of microRNA in a minute worm could inspire an entirely new category of therapies.

Yet, while the narrative of any specific advancement might appear improbable, the trend persists. Fundamental research — frequently financed by the government and carried out by university faculty and students — results in transformative drugs and technologies.

Given the recent withdrawal of research grants, specialists in medical science express concern that the U.S. could forfeit its longstanding advantages in research and development for many years to come.

“If these grants are not reinstated, it wouldn’t surprise me if a decade from now, we witness a significantly lower number of therapies reaching the market,” stated Robert Huckman, the Albert J. Weatherhead III Professor of Business Administration at Harvard Business School and the Howard Cox Faculty Chair of the HBS Healthcare Initiative. “That represents a genuine risk.”

Isaac Kohlberg, senior associate provost and the University’s chief technology development officer, concurred.

“If you undermine the base of the structure, the structure will collapse,” he remarked. “Academic innovations form the underpinning of countless products and services that consumers obtain. If we neglect investment in essential resources to foster fundamental discoveries, university laboratories may diminish, and the U.S. innovation landscape will falter.”

These challenges initiate from the beginning of the innovation funnel.

“To address disease, we must comprehend how it functions,” Huckman asserted. “And grasping the mechanisms of disease is a complex issue.”

By the time a company opts to invest in a clinical trial for a novel treatment or intervention, years — or even decades — of government-funded research typically precede it.

“You need to consider it as a foundational piece,” emphasized Kohlberg. “Without fundamental research, there is no translational research.”

Currently, few companies are structured to finance this research. Although a report from the nonprofit United for Medical Research indicates that each dollar of research backed by the National Institutes of Health generates $2.56 in economic activity, firms generally lack the initial capital — and long-term economic perspectives — to support expansive early-stage research.

“It isn’t the responsibility of companies to fund basic research,” Kohlberg pointed out. “Businesses are designed to generate profit, to produce products. Their focus is on immediate outcomes.”

Nonetheless, they do attentively track government-financed research, the results of which must be accessible to the public.

A variety of firms, for instance, capitalize on the groundbreaking gene-editing technology CRISPR or develop GLP-1 medications — even though parts of the foundational research behind these technologies were conducted at Harvard.

“You need to have a model in place to tackle significant issues,” Huckman noted. “And I believe that the partnership between government and academic institutions has historically facilitated these significant moves.”

When funding for early research diminishes, so too does the chance to commercialize promising advancements.

“We could begin to observe a constriction of research portfolios that might affect both the research facilities and those entities aiming to commercialize the discoveries those labs could have uncovered,” Huckman elaborated.

Biotech companies aspiring to explore technologies that might assist in treating various diseases may witness cuts to basic research and opt to chase less ambitious objectives.

“There’s a sequence of activities necessary to transition from understanding basic science to an approved treatment,” elucidated Huckman. “If we disrupt or weaken any link in that chain, we jeopardize the entire process.”

Kohlberg envisions a similar downturn: With decreased research funding, fewer concepts will emerge from universities; startups will struggle to develop those concepts; and larger firms traditionally involved in identifying, acquiring, and funding promising startups will have reduced opportunities to do so.

“It’s akin to a funnel,” he remarked. “Ultimately, patients will have fewer choices.”

And those choices will be pricier.

Huckman emphasized that if pharmaceutical companies must engage in riskier, early-stage research, they will need to recover those costs when pricing the medications they can market.

“If one believes pharmaceutical prices are elevated currently, it is reasonable to anticipate they will climb even more due to these funding reductions,” he stated.

For both Kohlberg and Huckman, severing the university-government collaborations could disrupt the entire framework of American innovation.

“These cuts signify a lack of support for the research sector,” Kohlberg warned. “This will thoroughly undermine the future of innovation.”