NIH targeting rare diseases, director tells U.S. House funding panel
Republished from Kentucky Lantern
WASHINGTON — The director of the National Institutes of Health testified before Congress on Tuesday the agency is looking to rebuild trust following the COVID-19 pandemic by solving some of the biggest health challenges facing the country.
Monica M. Bertagnolli told the House panel in charge of the agency’s funding that one of those focus areas is rare diseases, since private, for-profit companies often won’t take on the financial risk of developing gene therapies.
“What if you’re a parent of a child who has this rare disease?” Bertagnolli said. “We know if we work hard, our technology will be able to cure that child.”
Bertagnolli said during the two-hour hearing that NIH plans to soon begin “delivering more out of our pipeline for gene therapies for rare disease” and is collaborating with the U.S. Food and Drug Administration to ensure a smooth process.
“We will manufacture and test them within the clinical center at NIH. And then when they are ready for marketing and production — at that point after everything is completely de-risked — we will hand them over to the for-profit sector with the absolute requirements that the people who need them are able to access them,” Bertagnolli said.
The NIH, she told lawmakers, is responsible for the health of all Americans and will partner with the private sector on projects, but “can’t abandon these patients and these families.”
Bertagnolli told the House Labor-HHS-Education Appropriations Subcommittee that working to solve health challenges is one of several ways the NIH is working to rebuild trust with Americans following the global pandemic.
“We do not get people’s trust by putting out public service announcements. We do not gain people’s trust by saying ‘We’re smart, we know what to do, we’re the scientists,’” Bertagnolli testified. “We get people’s trust by solving the problems they need to have solved.”
Science, not politics
Bertagnolli also sought to reinforce basic scientific understanding about public health during the hearing, pressing back against political notions of researching infectious diseases.
“First and foremost, NIH concentrates on science, not on politics,” Bertagnolli said. “We actually have an integrity mandate against political interference in our work. That is the law for us and we abide by that completely.”
Bertagnolli noted that if NIH ceased research into diseases, death rates would rise.
“We are facing some serious threats. I mean, look in the news today, H5N1 is scary for us and we are all over risk assessment, mitigation, working with our other federal agencies to make sure that we can protect and mitigate anything that happens with that virus doing something evil,” Bertagnolli said. “We cannot afford to let down on that, people will die.”
H5N1, also known as highly pathogenic avian influenza, has caused significant disruptions to U.S. poultry farms for years, but showed up in dairy herds this March, raising alarm bells for public health experts.
Fifty-three people have been diagnosed with the virus so far, though the risk to the general public is low.
NIH duties
The NIH is made up of 27 different centers and institutes, including the National Cancer Institute, National Institute on Aging, National Institute of Neurological Disorders and Stroke, and the NIH Clinical Center that’s also referred to as America’s research hospital.
Congress approved $48 billion in discretionary funding for NIH in March as part of a full-year spending package.
The NIH writes on its budget website that 84% of its funding goes to “extramural research, largely through almost 50,000 competitive grants to more than 300,000 researchers at more than 2,500 universities, medical schools, and other research institutions in every state.”
“In addition, approximately 11% of the NIH’s budget supports projects conducted by nearly 6,000 scientists in its own laboratories, most of which are on the NIH campus in Bethesda, Maryland,” it states. “The remaining 6% covers research support, administrative, and facility construction, maintenance, or operational costs.”
Bipartisan support for funding
The NIH has long had broad bipartisan support in Congress, especially from members of the Appropriations committees.
That continued Tuesday with the majority of Democratic and Republican lawmakers on the panel asking genuine questions about NIH research, funding and future plans.
Alabama Republican Rep. Robert Aderholt, chairman of the subcommittee, said there was “no doubt every life is touched by the discoveries made by the investments at NIH.”
Aderholt encouraged Bertagnolli to make it a top priority to rebuild “confidence in the NIH as a leader in unbiased, nonpartisan, objective, basic science.”
A Pew Research Center survey published earlier this month showed 76% of Americans held a fair amount or a great deal of “confidence in scientists to act in the public’s best interests.” That represents a slight rebound for public faith in scientists after a steady decline since the start of the pandemic, according to Pew surveys.
Eighty-nine percent of those surveyed said research scientists were intelligent, while 65% said they were focused on solving real problems.
Connecticut Democratic Rep. Rosa DeLauro, ranking member on the panel, applauded Bertagnolli for “ensuring NIH’s life-saving research reaches more places and more patients, especially in rural communities.”
“And she understands that health policy must be first and foremost based on science and data,” DeLauro said.
NIH funding, she said, was going toward better understanding numerous diseases facing Americans, including ALS, Alzheimer’s, cancer, diabetes and mental illness.
The agency is also dedicating research dollars to address several health challenges facing women, including maternal mortality, as well as other areas that historically have been ignored or underfunded.
“NIH supported research has us on the cusp of curing endometriosis and they are ramping up investments for menopause, which will ultimately impact half of our country’s population,” DeLauro said. “But there is still a long way to go.”
Last updated 4:54 p.m., Nov. 19, 2024
Kentucky Lantern is part of States Newsroom, a network of news bureaus supported by grants and a coalition of donors as a 501c(3) public charity. Kentucky Lantern maintains editorial independence. Contact Editor Jamie Lucke for questions: info@kentuckylantern.com. Follow Kentucky Lantern on Facebook and Twitter. Kentucky Lantern stories may be republished online or in print under Creative Commons license CC BY-NC-ND 4.0.
Donate to Kentucky Lantern here.
