One year after Congress passed the 21st Century Cures Act with a bipartisan majority, the leaders of the National Institutes of Health (NIH) and the US Food and Drug Administration (FDA) testified to the Health Subcommittee of the House Energy and Commerce Committee yesterday about how they are implementing the law.
Many aspects of the legislation seemed to be getting off the ground, but questions emerged in Thursday’s session about funding for this ambitious project. For example, the Cures Act established an Oncology Center of Excellence at the FDA. This center’s work has already led to approvals of two gene therapy products targeting rare cancers, and the FDA is considering setting up similar centers for immunology and neuroscience, FDA Commissioner Scott Gottlieb, MD, told the committee. However, he added, Congress has not appropriated funds for the cancer center.
Similarly, the Cures Act requires that the Centers for Disease Control and Prevention (CDC) create a neurological surveillance system to track the prevalence and incidence of neurological conditions, including Parkinson’s disease. Rep. Gus Bilirakis (R-FL) asked NIH Director Francis Collins, MD, whether such a system could aid research on Parkinson’s. “If the data were available, we’d find it quite useful,” Dr Collins replied. But, because of funding issues, he said, the CDC hasn’t been able to conduct this surveillance.
Rep. Anna Eshoo (D-CA) noted that the Cures Act “authorized [funding] increases to FDA and NIH, but didn’t appropriate funding. Instead, it made it subject to the annual appropriations process. For all that the legislation calls for, how are your agencies doing with the appropriations process?”
Both Dr Collins and Dr Gottlieb replied that their agencies have been able to allocate the funds they have received to date. However, they didn’t get into specifics or say whether they were concerned about receiving adequate funds in the future.
Tax Bill Penalizes Grad Students
In Dr Collins’ prepared remarks, he made a big point about the need to strengthen the biomedical research workforce. With the 20% decline in NIH funding since 2003, he noted, the percentage of NIH awards that support early-career and mid-career investigators has declined. As a result of the Cures Act, he said, in June the NIH launched the Next Generation Researchers Initiative aimed at bolstering the workforce with a focus on early-career investigators.
However, Rep. Frank Pallone (D-NJ), the ranking member of the health subcommittee, pointed out that the House tax bill could make it difficult for some young people to become biomedical researchers because they would have to pay taxes on their research assistant stipends. He said that Rutgers University President Robert Barchi and other college leaders have said this tax change could make graduate biomedical education unaffordable “at a time when the US needs more students earning advanced degrees in the [science, technology, engineering, and mathematics] fields to remain competitive.”
Pallone then asked Dr Collins whether he agreed and whether he thought this might impede the discovery of new cures for diseases.
Dr Collins responded that graduate students are critical to the research effort. “We want to have the best and brightest who are interested in pursuing those careers to have the opportunity to do so. Anything that represents a major impediment in that regard is something we should take with great seriousness…. Given that science drives our economy, this is a very important area for continued investment. Anything that would diminish the interest of the next generation in joining that workforce is something we should be very cautious about.”
Drug Costs and NIH
Rep. Jan Schakowsky (D-IL) asked Dr Collins how useful new drug discoveries are if people can’t afford the medications because of high prices. The NIH director replied that his organization could help lower prices by making the drug discovery process as efficient and as accurate as possible, “so the failure rate for drug development is not so incredibly high. One reason why drug prices are so high is that the industry has to compensate for the failures, which are over 95%. If we had a success rate of 50% rather than 5%, the equation would look a lot different.”
Schakowsky than asked Dr Collins whether the NIH has exercised its statutory authority under its licenses of NIH intellectual property to drug companies to ensure that publicly funded drugs are “reasonably priced.”
Dr Collins said that the NIH looked at that issue in a couple of cases where it played a role in the medication’s early development. However, he said, the law seems to apply to circumstances where the drug is not available and then the NIH is “entitled to step in. This is different, where the drug is available but at high cost. Our legal experts don’t feel the law puts us in a position to step in.”
Continuous Manufacturing
Some subcommittee members were interested in the FDA’s progress in supporting development of “continuous manufacturing.” This is a next-generation approach to drug making that could help reduce drug shortages and recalls because of problems with product quality, according to Dr Gottlieb. It could also help attract some manufacturing to the United States from overseas locations, he said.
The FDA has given the University of Connecticut $1 million to study this process, he noted. Altogether, he said, Congress allocated $5 million for such grants.
The Cures Act also authorizes the FDA to evaluate the potential use of “real-world” evidence to support the approval of new indications of approved medical products or satisfy postapproval study requirements for marketed products. This evidence, Dr Gottlieb said in his prepared testimony, includes electronic health record data, claims and billing data, data from product and disease registries, and patient-generated data, including those from home and mobile devices.
Another topic that came up was the creation of rapid diagnostic tests that can speed up the detection and treatment of dangerous conditions. Dr Collins noted that the NIH is running a Cures Act-authorized competition to develop a test that, within 4 hours, can identify the organism responsible for a certain disease and determine whether it has multiple-drug resistance. “In terms of [urinary tract infections], pneumonia or sepsis, that would be a profound game changer,” he said.
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