March 14, 2021 – Nearly a month after being sworn in for the second time as Food and Drug Administration (FDA) Commissioner, Robert Califf, M.D., has a lot on his plate, including the ongoing COVID-19 pandemic — and lessons learned about processes and the harms of misinformation – as well as a call for clinical trial reform and the need for an exhausted FDA to find a “new normal.”

As he approaches these issues, he is bound to use his experience with data to formulate the agency’s path forward, according to Nancy Bradish Myers, president, Catalyst Healthcare Consulting.

“When I think about what kind of commissioner Califf will be, I believe he will channel data,” Myers said at a recent Coalition for Healthcare Communication webinar, adding that one of her favorite Califf quotes is “In God we trust, all others must bring data.”

In Myers’ view, there are four key priorities for Califf to address in 2022:

• Managing the COVID-19 response and harnessing lessons learned to modernize the FDA;
• Rebuilding/maintaining the public trust in the FDA as the gold standard (which includes supporting regulatory decisions grounded in scientific evidence);
• Reforming clinical trials and research to include ensuring equitable access to clinical trials and the increased use of real-world evidence (RWE); and
• Supporting a fatigued agency by focusing on resources and staffing, helping the agency get back on track and handling the politics to allow FDA staff to do their jobs without distractions.

Califf also has publicly stated he will make tackling misinformation, the opioid crisis and tobacco issues top priorities as he retakes the helm at FDA.

COVID-19 lessons

The pandemic is not over yet, so the FDA is among the public health agencies that have to be prepared for either the next wave or the next non-flu pandemic, Myers explained. However, the agency also has begun to catalogue which improvements stemming from its experience with COVID-19 may remain. Califf will be responsible for determining which lessons should be implemented moving forward, and how broad those changes will be.

Speaking about priorities during his confirmation process, Califf committed to the tighter enforcement of requirements that sponsors conduct confirmatory clinical trials after receiving drug approvals through the accelerated pathway. Other candidates for continued use or evaluation include more frequent interaction between the FDA and sponsors to speed up drug development, the increased importance of post-market surveillance for approved products, and a tightening up of emergency use authorization.

Rebuilding trust in FDA

As FDA Commissioner, a significant part of Califf’s job is addressing how the public perceives the agency and the science behind its decisions, including the controversial accelerated approval of Aduhelm for Alzheimer’s disease (see related article, here), COVID testing confusion and vaccine approval speeds, Myers explained. There also is a push for greater transparency around reviews, as well as for the use of RWE and big data to more quickly inform outcomes.

Myers remarked that a reliance on data has been a hallmark of Califf’s professional life. “If you look back at his incredible career as a clinical trialist, as someone who is a cardiologist and who has been involved with groups like Verily, it’s all been about the data and high-quality evidence,” she told CHC webinar attendees.

Tackling clinical trial reform

Califf wants to see clinical trials become more efficient and coordinated, but it is unclear at this point how quickly that will happen and what shape reforms will take. According to Myers, COVID-19 “pressure tested” many concepts that Califf has championed during his career. These include:

• Leveraging RWE
• Permitting remote monitoring of patients
• Allowing decentralized clinical trials
• Valuing master protocols and adaptive trial design
• Centralizing national priorities and aligning clinical trial protocols

It is likely that Califf “will turn his technology experience into FDA priorities,” Myers predicted. “He has a new understanding of the data, its promise and current limitations,” she said, and “his experiences will underpin his problem solving.”

Managing a fatigued agency

Beyond policy and process, the people part of manning and managing an FDA that has been overextended, understaffed and working from home would be a heavy lift for any FDA Commissioner, but at least Califf is familiar with many of the staff members and knows the challenges they face, according to Myers. In the coming months, Califf will need to make management decisions about how remote work will continue and what tasks need to be handled face to face.

Califf also must devise a plan for attracting and retaining talent to keep the agency staffed and energized. Because of his past tenure at the agency and his experience in clinical trials, he does understand the current limitations of the FDA’s IT infrastructure, which is in the process of being modernized. “This is a critically important initiative that Califf is likely to address with hyper speed,” Myers said, because the agency is not technologically prepared to meet innovation in its current state.

It is worth noting that the Center for Drug Evaluation and Research’s Office of Prescription Drug Promotion recently established the Division of Promotion Policy, Research and Operation, which will provide additional support and increased focus on the regulatory counsel functions needed to develop sound and legally supportable policy documents – which is important in light of past challenges of FDA policies on First Amendment grounds, Myers observed during the webinar.

Communication is key

An important element that will help Califf with all of these priorities is communication – to staff, to Congress and to the public. “One of the differences you’ll see between 2016 Califf and 2022 Califf is the realization of the importance of communication,” Myers commented. Indeed, on the day he was sworn in as FDA Commissioner, Califf sent staff a memo addressing some of his goals and also shared those with the public through his new Twitter account: @DrCaliff_FDA

“In a world changing as fast as ours & facing as many disparate challenges as we do, FDA’s work is vital to protecting the public health,” he tweeted. “At the core of this work is this agency’s commitment to and reliance on the best available science & most rigorous data in reaching our conclusions.”