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Regulatory News | 18 January 2022 | By Michael Mezher
In correspondence with two Republican senators before his nomination was advanced in the Senate last week, FDA Commissioner-nominee Robert Califf committed to promoting the use of real-world evidence (RWE) and cited the need for better postmarket evidence generation.
The senators, Roger Marshall (R-KS) and Mike Braun (R-IN), were two of the eight lawmakers on the Senate Health, Education, Labor, and Pensions (HELP) committee who voted against Califf’s nomination. Facing some Democratic opposition, Califf will need a handful of votes from Republican senators if he is to be confirmed to the post. (RELATED: Senate committee advances Califf’s nomination as FDA commissioner, Regulatory Focus 13 January 2022)
The two senators questioned Califf on a range of topics, including RWE, FDA’s accelerated pathways, digital health and software-based medical devices and the opioid epidemic. Focus obtained a copy of the questions, and Califf's responses, from the Senators' offices.
“During my last tenure as Commissioner, I helped lead the agency forward toward more widely using real-world evidence in its regulatory decision-making processes. I can assure you that, if confirmed, I will continue this trend, including evaluating all evidence, including RWE, for products that have received EUAs as they work toward full clearance or approval,” Califf told Marshall.
Califf said that part of his agenda will be to develop additional systems and policies for RWE and real-world data (RWD) and that FDA should “continue considering RWD/RWE that is fit-for-purpose.” The former commissioner pointed to existing FDA initiatives and recent draft guidance on the use of RWE in drug development and cited the need for improved post-market evidence generation.
Califf also committed to working with FDA and the global community to “optimize the quality and reliability” of digital health records in response to a question from Marshall on using RWE from other countries to make faster decisions about products during the pandemic, without compromising FDA’s standards. But he cautioned that “building a system to curate and use the significant amount of health data stored throughout the country is essential to making decisions quickly. However, this data will not and cannot be used without careful planning for study design followed by high quality analyses being conducted.”
In response to Braun, Califf dodged questions on electronic prescribing information and whether he would “reign in CDRH” in its negotiations with the medical device industry on the next iteration of the Medical Device User Fee Amendments (MDUFA V) program. Recent reports indicate that FDA missed its deadline to transmit the MDUFA V package to Congress, in part due to a disagreement with industry about how much the program will cost.
Califf told Braun he is not privy to the MDUFA negotiations but committed to working with the HELP committee on reauthorization “to assure the agency is resourced.” Califf also said he would “take a closer look” at FDA’s biocompatibility testing requirements.
Braun also asked Califf how he would integrate programs such as the agency’s pilot on model-informed drug development into the agency’s routine approach to drug development.
“If confirmed, I am committed to ensuring that product development and review is completed in the most efficient way possible to ensure the Agency gets to the critical questions of safety and efficacy of products. This includes integration of methods from pilot programs, like the one you mentioned, if they prove to improve clinical trial efficiency, enhance the probability of regulatory success, and optimize other aspects of drug development,” Califf responded.
Califf also championed digital health technologies and said he is “highly supportive of the efforts of FDA’s Digital Health Center of Excellence, and see[s] it as an important way to empower stakeholders to advance health care by fostering responsible and high-quality digital health innovation, including for software-based medical devices.”
On opioids, Califf committed to “reviewing all aspects of FDA’s role in combating the addiction and overdose crisis” during his first year back at FDA, acknowledging that not enough has been done to stem the epidemic.
Califf also told Braun he would work with the HELP committee to hone and advance the VALID Act, which was reintroduced in the House and Senate last year and would overhaul the regulation of in vitro diagnostics (IVDs) and laboratory developed tests (LDTs). A discussion draft of the bill was first released in 2018 and follows years of stalled efforts to reform FDA’s oversight of LDTs in the wake of increasingly complex tests. “I strongly support the need to create a modern framework for oversight of all in vitro diagnostic tests,” Califf said, adding that he would work with the committee to “ensure any legislation strikes the balance needed to promote innovation and development of diagnostic tests, including for unmet needs, while adequately protecting patients and the public health.” (RELATED: FDA leadership calls for LDT reforms, Regulatory Focus 6 December 2018)
Additionally, the former commissioner signaled his support for the use of change control plans for software-based medical devices to reduce the regulatory burden on developers by allowing them to lay out planned changes to a product in a premarket submission and execute those changes without requiring a new submission to FDA.
In his questions, Braun noted his concern that FDA inconsistently applies its programs and flexibilities intended to facilitate the development of treatments for certain rare diseases. Califf responded by saying that advancements in biomedical research and recent innovations in clinical trial design, along with FDA’s accelerated pathways are key to rare disease drug development. Califf added that the “post-market phase deserves particular attention to ensure that we understand the long-term effects of interventions,” and that digital health technologies and electronic health records can “enable a much more thorough evidence base for effective treatment.”
Responses to Braun, Marshall