In a wide-ranging hearing on Thursday, the US Senate Committee on Health, Education, Labor and Pensions questioned Francis Collins, MD, director of the National Institutes of Health (NIH), and Scott Gottlieb, MD, commissioner of the US Food and Drug Administration (FDA), on the progress they have made in implementing the 21st Century Cures Act in the year since the legislation was passed.
While the tone of the hearing was mostly upbeat and congratulatory, some Democratic senators raised the same questions about funding for the Cures Act implementation that arose in the recent House subcommittee hearing on the same subject.
Dr Collins and Dr Gottlieb said that the law has helped their agencies more quickly push forward medical research and approve new therapies, respectively. This acceleration is especially important, they said, at a time when researchers are making rapid advances in such fields as gene therapy, regenerative medicine, and precision medicine.
For example, the Cures Act has given the FDA the ability to expedite reviews of certain regenerative medicine products — a new program on which the FDA has just provided draft guidance. These treatments include gene therapies that can be used to treat illnesses such as cancer and hemophilia.
“Over the next several years, we’ll see this [gene therapy] approach become a mainstay of treating, and probably curing, a lot of our most devastating and intractable illnesses,” Dr Gottlieb said. “At FDA, we’re focused right now on establishing the right policy framework to capitalize on this scientific opening.”
The Cures Act also provided the NIH with “other transactions authority” (OTA) to help it move flexibly and quickly in areas of high need. “It means we can bring partners together quickly without waiting a whole year that it often takes to issue grants,” Dr Collins said. This approach could be especially helpful in addressing the opioid crisis, he added.
Committee chair Lamar Alexander (R-TN) asked Dr Collins to explain what he meant. The NIH chief said that a critical part of the solution to the opioid crisis is to develop nonopioid pain medicines for the 22 million people who suffer from chronic pain. Working hand in hand with biomedical companies and academic medical centers, he said, the NIH could use the OTA to dramatically accelerate the development of these new therapies.
Dr Gottlieb said the FDA is trying to speed up the drug approval process with new scientific tools to determine the safety and effectiveness of candidate drugs. He cited a new “tissue on a chip” initiative designed for toxicology.
Insurance coverage is vital to the success of a drug after it has been approved, and Dr Gottlieb noted that the Centers for Medicare and Medicaid Services (CMS) has started cooperating with the FDA in this regard. For example, the FDA recently approved a diagnostic test to detect genetic mutations that can cause cancer, he said. At the same time, CMS approved Medicare coverage of this “breakthrough” test. This will not be the last time that approval and coverage go hand in hand, he predicted. “What CMS said in their policy is that [test] panels that come through FDA for voluntary approval now will automatically receive a national coverage determination.”
Other FDA Initiatives
The FDA is also pushing forward on other Cures-related fronts, Dr Gottlieb said. He cited the agency’s just-released guidance on the development of clinical decision support tools, the guidance it released in August on improvements in medical device reprocessing, and its continued monitoring of mobile health apps.
Regarding wearables and other kinds of mHealth devices and apps, Dr Gottlieb said, “We continue to receive adverse event reports, even for products we might exclude from regulation under the parameters in the Cures statute. If we did receive an adverse event report that led to a recall of an excluded product, that would be a reason for us to bring that product back under regulation through the provisions in Cures…. That tells us it should not have been excluded in the first place. So we continue to monitor products, even if they’re not within our scope of regulation.”
Puerto Rico Disaster
Sen. Robert Casey (D-PA) noted that there have been shortages of intravenous (IV) solutions in his state because some of these products are manufactured only in Puerto Rico, which is still recovering from Hurricane Maria. He asked Dr Gottlieb what is being done about the problem and whether “outsourced” compounders could help solve it.
Dr Gottlieb replied that the FDA is concerned about the situation and that it has “taken steps” with manufacturers and the Puerto Rican authorities to alleviate the shortage. However, he said, it will not improve significantly until early next year. As for the compounders, he said they can make the IV fluids but lack the proper plastic bags, which are also made in Puerto Rico.
Lack of Funding
Sen. Chris Murphy (D-CT) brought up the same Cures Act funding questions that emerged at the House hearing. He asked Dr Gottlieb about the Oncology Center of Excellence, a new FDA unit designed to pull together the clinical data and knowledge the agency has acquired in reviewing cancer drugs. Dr Gottlieb said that the center could do more if it were fully funded, and Murphy said the committee should look into that.
Murphy also questioned Dr Collins about the importance of tracking the incidence and prevalence of neurological conditions, as required by the Cures Act. Dr Collins again said that it was important for the Centers for Disease Control and Prevention (CDC) to conduct the mandated study, but that the CDC lacked funding for it.
Sen. Elizabeth Warren (D-MA), while applauding the intent of the Cures Act, said it fell short on funding because it didn’t include new money to implement its provisions. Instead, she said, it relied on future Congresses to fund the initiatives.
“So far, Congress has been increasing NIH funding,” she said. “But I don’t think it’s time for us to pat ourselves on the back for where we are in funding the NIH.”
Responding to a question from Warren, Dr Collins said that NIH funds only 19% of research grant applications. That isn’t because all the rest are a waste of money, he said. In 2000, the NIH was funding 30% to 35% of grant applications. NIH studies have shown that that there is no difference in the productivity of grants in the 15th percentile and the 30th percentile in order of priority, he said. So it would be better for science if the NIH could get enough money to fund at least 25% of grant applications.
Warren said that is one reason why she and the committee’s other Democrats are introducing the National Biomedical Research Act, which would provide $50 billion in new funding for the NIH and the FDA.
In response to Warren’s comments on the resources available for the Cures Act, Alexander noted that Congress has increased NIH’s funding by $2 billion a year for the last 2 years, and is on track to do the same in 2018. The Cures Act, he said, also provided $4.8 billion in additional funding for precision medicine, regenerative medicine, the BRAIN initiative, and the Cancer Moonshot.
“The chances of that happening again are close to zero,” he said. “The real money that goes to NIH and FDA comes from the [Senate] appropriations committee and they’ve been doing a tremendous job for the last few years.”
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