Califf: New systems needed for bringing rare disease treatments, at-home devices to market

WASHINGTON – Robert Califf, commissioner of the US Food and Drug Administration (FDA), said treatments for rare diseases and devices used to monitor people at home require new systems and regulatory pathways because the current ones are not suited for the job. He also said he’s optimistic the agency will be able to implement its proposed Human Foods Program and Office of Regulatory Affairs (ORA) reorganization as planned and that he wishes Congress had passed diagnostics reform legislation.

On 14 February, Califf spoke at a luncheon at the National Press Club, where he talked about how medical misinformation is having a detrimental effect on Americans’ health and took questions from journalists about his priorities for FDA. He said journalists have a responsibility to combat medical misinformation, but also noted that the medical products industry should do its part too. (RELATED: Califf says major effort underway to reform advisory committee meetings, Regulatory Focus 1 February 2024)

"I think the whole business community needs to pay more attention to this because it affects their employees and the families of the people who work for them,” said Califf. “I do think coalitions from the private sector need to gather up reliable information and make sure the people that work for them, and their communities are getting good information… We’re all accountable."

When asked about the Operation Warp Speed for Rare Diseases pilot that was launched by the Center for Biologics Evaluation and Research (CBER) last year, he said that researchers have shown promising new treatments for rare diseases, but now regulators have to come up with ways to evaluate such treatments in very small populations. (RELATED: CBER chief says pilot program will justify increasing reviewer headcount, Regulatory Focus 4 December 2023)

“If you had told me 10 years ago that we could identify the exact gene causing a disease in a child and go in and sniff out that gene and put in a new one, I just would have said that's science fiction,” said Califf.

The commissioner noted that there are several cases where that is exactly what’s happened, based on promising findings in journal studies. However, he added that regulators must develop a new regulatory system that can handle situations where the sample size of patients is very small because the traditional regulatory pathways are not suited for such situations.

“There’s already progress that’s been made here, but we’ve got to invent that system,” said Califf. He added that researchers also need to develop networks to identify patients with rare diseases and enroll them in clinical trials, while noting that there isn’t an economic model to encourage investment in rare disease research.

“The good news is in our user fee agreements, we got money to hire a lot of people [to address such issues], and we’re doing that,” said Califf. “But stay tuned, we’re working on [creating new] systems.”

Califf talked about the need to improve the quality of life for people regardless of age as the life expectancy in the US is decreasing. He pointed to places such as Singapore and Scandinavia that are taking proactive steps to ensure the elderly are taken care of and the importance of technology in ensuring people’s health is monitored at home.

During his five-year stint at Alphabet between his commissionerships at FDA, Califf said he saw proposals for new devices that could help people improve their quality of life but that were not useful unless they were integrated with other devices in the home. However, he noted there currently isn’t a defined regulatory pathway for such devices.

“If you think about device development, like sensors, if you do it one at a time, you'll never put a whole suite together into something that fits together in the [home] environment,” said Califf. “[The Center for Devices and Radiological Health (CDRH) is] working on a strategy on how to create regulatory pathways to help make that happen."

During his talk, Califf didn’t give an update on when he thinks a recently proposed rule to regulate laboratory-developed tests (LDT) would be implemented but noted that he’s had family members who have recently been diagnosed with cancers and it disturbs him that the test results could vary based on who is administering them. He again noted that he would have preferred it if Congress had passed the Verifying Accurate, Leading-edge IVCT Development (VALID) Act, but the rule will have to suffice. (RELATED: Experts expect opponents of LDT rule to ramp up Congressional lobbying, Regulatory Focus 14 December 2023)

“I've been very involved in the rulemaking and I'm completely behind it,” said Califf. “I wish Congress had done its job, that would be the best because we would do better with (an in-vitro diagnostics) pathway that had the things in it.”

“Rulemaking is limited in what we can do, but it's a good start, and I think it's a very important start,” he added.

Califf also said he couldn’t provide an update on when he thinks FDA’s plan to reorganize its Office of Regulatory Affairs (ORA) will happen but noted that the process is very complicated and involves sign-off from the executive branch and input from Congress. He noted, however, that it has been well-vetted by the thousands of agency employees and other stakeholders who are affected by it. (RELATED: FDA leaders detail reorg plans, say 1,500 ORA staff will be reassigned, Regulatory Focus 19 January 2024)

"Since it's under consideration now, I have to stay quiet, but I'm very optimistic because I think it's a great plan,” said Califf. “We have to get through all the hurdles before we can finalize the reorganization, but we've got thousands of people interested in working on it."

During a recent interview with Focus, former FDA Principal Deputy Commissioner Janet Woodcock said FDA needs Congressional authority to force manufacturers to initiate a recall. (RELATED: Interview: Janet Woodcock’s lasting influence on FDA, Regulatory Focus 29 January 2024)

After the luncheon, Califf told Focus he agreed with that view, and it’s an issue the agency is actively working on in their discussions with Congress. As an example, he pointed to the delay in getting potentially contaminated eyedrops made by Kilitch Healthcare India Limited off the market that unnecessarily put Americans at risk for months.

"Pretty much across the board, when something goes wrong with a product, I think it's important that FDA has the ability to act quickly before too many people are injured by that product,” said Califf. “Historically, there's been resistance from the industry that has kept this from happening. It's just frustrating when we're limited in what we can do.”

Califf: New systems needed for bringing rare disease treatments, at-home devices to market

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