NEW YORK – The Verifying Accurate Leading-edge IVCT Development (VALID) Act passed out of the Senate Health, Education, Labor, and Pensions (HELP) committee on Tuesday as part of the larger Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act.
Support for VALID, which will now move to the full Senate for consideration as part of FDASLA, could be weaker than anticipated, however, as one of its main proponents, Sen. Richard Burr, R-N.C., voted against FDASLA in committee and raised questions about whether it was an appropriate time to give the US Food and Drug Administration new authority given its recent struggles — its failure to ensure the safety of the domestic baby formula supply, in particular.
During his remarks at the opening of the Tuesday HELP committee session, Burr said that while FDASLA gives "the FDA more resources and more authority… it has become clear that the agency may not deserve our trust."
He called out the VALID Act specifically, acknowledging that he had originally pushed to include the bill in the larger FDASLA package, but that he now worries "that we are rewarding bad behavior" at FDA.
The VALID Act aims to resolve decades-long questions around the FDA's authority to regulate laboratory-developed tests by placing them within the agency's purview. The legislation creates a risk-based framework for in vitro clinical test (IVCT) regulation, with high-risk tests, such as novel assays, required to go through premarket review, while lower-risk tests could go to market after passing through technological certification. The law would grandfather in LDTs currently in clinical use.
Burr is co-sponsor of the bill and has been one of its key advocates in the Senate.
"Is this the time to expand FDA authority in any space, including diagnostic tests?" he asked. "When FDA fails to do their fundamental job on infant formula, can they be trusted with new responsibilities?"
Later in Tuesday's session, as the committee considered an amendment by Sen. Tommy Tuberville, R-Ala., that would exempt LDTs developed at academic medical centers from FDA regulation under VALID, Burr defended the bill and opposed Tuberville's amendment (which was ultimately tabled by Senate HELP chair Patty Murray, D-Wash., meaning it was not included in the bill).
Notably, however, Burr did not frame his support for VALID as an argument that FDA should necessarily have authority over all diagnostic testing but only that all diagnostic testing should be under some sort of unified regulatory regime.
"If you've got two different entities creating the same thing, one can't be regulated by one [body] and one regulated by another," he said. "It's all got to be under the same umbrella. Maybe instead of VALID, the private sector labs should be approved by CLIA. I don't know."
It's unclear whether Burr's comments and vote against FDASLA were simply meant as a warning shot at FDA or if they suggested he may vote against the bill or seek to strip VALID from the package when it comes up in the full Senate.
"We still have a long way to go on this bill before it becomes law," Burr said. "And I will be watching the actions of FDA all the way to the end to see if they rise to the challenge and decide whether they deserve an ounce of new authority."
The bill passed out of the HELP committee with 13 senators voting for it and nine voting against. While FDASLA, which reauthorizes FDA to charge the user fees that provide a substantial portion of its funding, is widely viewed as must-pass legislation, various components of the package, like VALID, that are not central to the user fee reauthorization, could be taken out or amended.
VALID also recently suffered a setback in the House of Representatives, where it was not included in the Food and Drug Amendments of 2022 bill (which reauthorizes the FDA user fee program) that passed earlier this month 392-28.
Rep. Larry Bucshon, R-Ind., one of the sponsors of VALID in the House, said that he had tried to get the bill included in the larger Food and Drug Amendments of 2022 package but that legislators could not agree on whether to include various provision, including VALID.
"And so I think the decision was made on the House side at least to live to fight another day and get a relatively clean [user fee bill] across the finish line and then to work with the Senate," Bucshon said in an interview.
He said that despite the decision not to include VALID in the House bill, he expects the House would vote to include it in the final bill provided it is included in the Senate bill.
An industry observer concurred with Bucshon's assessment that the House would include VALID in the final version of the user fee bill if it was attached to the Senate bill.
This observer suggested, though, that Burr's opposition could complicate the passage of VALID in the Senate, noting that members of the same party often take their lead from a bill's co-sponsor, especially when that individual is also the ranking member on the committee with jurisdiction over the legislation.
As for the bill itself, both proponents and opponents have taken issue with various aspects of the Senate version of VALID. Among the contentious portions are exemptions the bill provides for tests for diagnosing rare diseases or conditions and for custom or low-volume tests.
For the rare disease exemption, the bill sets a threshold of "use for a diagnostic purpose for a disease or condition [that affects] not more than 10,000 (or such other higher number determined by the Secretary) individuals in the United States per year)," provided the test is not meant for screening or for diagnosing a contagious disease with a potential public health impact.
For the custom or low-volume exemption the bill sets a threshold of "a test system performed for not more than 5 patients per year (or such other higher number determined by the Secretary)," and "for which no other in vitro clinical test is commercially available in the United States."
Liz Richardson, director of the health care products team at The Pew Charitable Trusts, which has advocated strongly for VALID, said that while the organization sees the custom test exemption as giving labs and providers needed flexibility, it is concerned that the rare disease exemption could prove a sizable loophole.
"That one raises a few more eyebrows for us," she said. "If you set it at 10,000 people or fewer are subject to this diagnosis per year, that is still a lot of people potentially getting a result from the test."
She said Pew considered it important that the bill retain the language clarifying that the exemption did not apply to screening or infectious disease testing, "because otherwise you could have a ton of tests getting out to market through that program."
Meanwhile, the American Association for Clinical Chemistry, one of the more vocal opponents of VALID, is concerned that the 10,000 threshold is too low, with Stephen Master, AACC president, and chief of the division of laboratory medicine at Children's Hospital of Philadelphia, saying in an email that the organization "would strongly prefer that a higher threshold be set in the bill, because this could have a profound impact on the availability of tests for a number of rare diseases."
Bucshon said that he shared Pew's concerns, noting that, personally, he didn't think "there should be very many exemptions at all," but added that such compromises are a part of legislating.
There are also questions around the law's "technology certification" pathway through which labs can launch low-risk tests without going through premarket review. Pew's Richardson specifically highlighted its language describing the breadth of a technology certification order, which says that such orders "shall be no broader than a single technology type; or a fixed combination of technologies where multiple in vitro clinical tests utilizing the technology do not significantly differ in control mechanisms, energy sources, or operating principles and for which development, analytical and clinical validation, of the in vitro clinical tests would be addressed through similar procedures."
Richardson said Pew's concern is that, as written, the law could allow a developer to bring "hundreds" of tests to market on a given technology without premarket review once it has received a technology certificate order.
AACC's Master, on the other hand, said the organization would "strongly object" to a narrower reading of technology type. He added that "the fact that there are even two sides on this issue proves that these things are going to have to be clarified in the legislation" and "reinforces the fact that, as it stands, this legislation is faulty and should not be shoe-horned into a must-pass vehicle like the FDASLA Act without going through appropriate debate and review."
In a May letter to Murray and Burr, the American Clinical Laboratory Association – which has indicated that, despite its reservations, it considers the VALID act the likeliest route forward regarding LDT regulation – also advocated for a broader scope for technology certifications. It also asked that tests put on the market via technology certification be "deemed approved" as opposed to "exempt from premarket review" to ensure that they "receive equal treatment under other provisions of law, such as related to reimbursement."
The question of funding FDA's new responsibilities under VALID is also an issue, with organizations including AACC and the National Independent Laboratory Association raising concerns that user fees required to pay for agency review of tests will prove onerous for academic and small independent labs.
Master predicted a scenario where labs without the financial resources to pay user fees will stop offering LDTs, leading to less user fee revenue and, ultimately, "a vicious cycle where you have a new, costly regulatory structure with no users to pay for it."
Pew's Richardson, on the other hand, expressed concern about how FDA would fund sufficient post-market review of the new categories of tests it would take on under VALID, noting that this is not paid for by user fees.
Bucshon dismissed concerns about the burden of user fees on labs but said the question about funding for post-market review is "a legitimate concern" and one that Congress would need to address.
"We have seen with the baby formula stuff and the difficulty in staffing that the FDA has that they do need more financial resources," he said. "That doesn't have to completely be within the user fee agreement. As far as appropriations, I think we will make sure the FDA has the ability to do what they need to do."