Biomedical research institutions and companies in the Triangle could get a boost of extra funding if a bill passed this month by the U.S. House of Representatives is approved by the Senate.
In a rare display of bipartisan harmony, the House voted 344-77 in favor of the 21st Century Cures Act, which would increase federal money for medical research and would speed up the delivery of new treatments to the public.
The bill would provide the National Institutes of Health with more than $1.5 billion extra each year for the next five years.
“This is a huge positive shot in the arm, so to speak, for the biomedical research community,” said Steven Patierno, deputy director of the Duke Cancer Institute in Durham.
If the NIH budget increases, the money flowing into the biomedical research community in North Carolina would likely increase proportionally, said Norman Sharpless, director of the UNC Lineberger Comprehensive Cancer Center in Chapel Hill.
“North Carolina gets about a billion dollars from the NIH, and almost all that comes to (the Research Triangle),” Sharpless said. While most of that money goes to institutions, some is made available to private startup companies, he said.
The budget for the NIH, a federal agency that funds medical research in the United States and internationally, has been relatively stagnant since 2003. Accounting for inflation, the NIH says its purchasing power has decreased by 22 percent.
Scientists compete for NIH grants to fund their research. Before 2003, the NIH funded about 30 percent of grant applications; last year, that number was just 18 percent. David Pugach, a government liaison with the American Cancer Society, says the decrease reflects cuts to the agency’s budget.
That makes competition for these grants steep, said Patierno, with “chilling” consequences.
“We’ve lost probably two generations of cancer researchers who have left a career in science because of concerns that there was very little future other than an intensively competitive one,” he said. “And we’ve had a major round of attrition of successful scientists who have found themselves unable to compete for this increasingly rare resource.”
The bill would create an “Innovation Fund” to provide extra money to the NIH outside of what Congress budgets for the agency. The Obama administration requested $31.3 billion for NIH in 2016, $1 billion more than the agency’s budget in 2015.
This is “the strongest recognition and demonstration of support that we’ve seen in a long time for the importance of increasing medical research funding,” said Pugach. And, he added, it’s coming from Republicans and Democrats alike.
Critics of bill
But the 21st Century Cures Act has not been immune to criticism.
Dave Brat, a Republican from Virginia, proposed an amendment to change the bill’s funding from mandatory to discretionary, meaning congressional committees would have had to approve the proposed budget every year.
That change would have effectively killed the bill, said Pugach.
Among those who applaud the extra funding, some object to the bill’s proposed changes to the way medical innovations are regulated.
The Food and Drug Administration typically approves a drug based on the results of large, randomized clinical trials. But the bill would speed up the process of getting a drug to market by allowing the FDA to consider information gathered from less rigorous sources, such as observational studies.
That loosening of regulations worries Aaron Kesselheim, a health policy researcher at Harvard.
“There’s this misperception out there that there’s some major massive government bureaucracy that is keeping cures away from people,” Kesselheim said. In fact, he said, the FDA approves drugs very quickly “when it gets evidence that the drug does work.”
Kesselheim is also concerned about several other proposed changes. The bill would encourage the FDA to use short-term indicators of health, such as tumor progression or bacterial counts, to assess a drug’s efficacy, as opposed to longer-term assessments of patients’ health; it would allow new, untested antibiotics to be prescribed to ailing patients; and it would permit medical device makers to bypass FDA approval for changes made to their devices if approved by an outside consultant.
“If you lower the FDA’s standards for antibiotics or medical devices, as this bill does, you get a risk of getting a lot of products that don’t work” and waste money, Kesselheim said.
Cutting red tape
Patierno, the deputy director of the Duke Cancer Institute, believes that these changes will cut unnecessary red tape.
“I think there are enormous and highly iterative and redundant protections in place that protect our patients extensively,” he said. “The amount of effort – time, money, and otherwise – that institutions have to put into regulatory compliance is staggering.”
The requirements should be flexible depending on the drug being funded, Sharpless said. “Often patients with cancer can’t wait five years for the approval process,” he said.
Another proposal in the bill would encourage research institutions to share patient information and work together on clinical trials.
Paula Brown Stafford of Durham-based Quintiles, a company that coordinates clinical trials, testified before Congress last year about the need for “modernizing” those trials. Earlier this month, Quintiles released a statement largely praising the bill passed by the House, saying that it “removes barriers that led to redundant efforts or restricted data flow.”
Encouraging institutions to share information and work together would accelerate the development of “precision medicine,” Patierno said.
Some new drugs target specific genetic changes that may not be shared by all patients diagnosed with a particular disease, “and that means it becomes much more difficult for any single institution to find enough patients with that specific mutation” to be enrolled in clinical trials, Patierno said.
Doctors hope that precision medicine would mean “prescribing the right medicine at the right dose to the right patient at the right time,” he said.