Califf Hopeful on Alzheimer’s, Obesity Drug Data
FDA commissioner Robert Califf says he’s cautiously optimistic that the U.S. could be on the verge of seeing major impacts from new drugs for treating Alzheimer’s and obesity. Presumably addressing Eli Lilly’s recent and promising news on its Alzheimer’s drug donanemab (see story) and its diabetes drug Mounjaro (tirzepatide) that is being studied as an obesity treatment (see story), Califf said the agency is awaiting final submissions, but if the submitted data matches up with the press releases, it is possible these drugs could be "major game changers in our ongoing battle with chronic diseases that are causing the vast majority of death and disability in our country.” Califf made these remarks at the Food and Law Institute’s annual meeting 5/17.
Hinting at previous reimbursement restrictions attached to any Alzheimer’s drug approved under the accelerated approval pathway (see earlier story), Califf said that advancements in Alzheimer’s and obesity “will raise major questions about the interface between approval by FDA for an intended use and broader use for tens of millions of Americans. This is good problem to have, and we’re having productive discussions with our CMS colleagues, for example, but we all need to be working on these issues.”
While the U.S continues to be the number one innovator in medical products around the world, Califf said FDA needs to “strengthen our structures, methods, data collection and science to allow us to be even more efficient, flexible and effective in the exercise of our regulatory responsibilities” to help industry retain this leadership. One example Califf offered is the evolving role of advisory committees as an important tool for obtaining independent advice regarding scientific, technical or policy questions. “As a former long term advisory committee member,” he told FDLI, “I believe strongly in the value of interchange among our experts at the FDA and the experts who work outside the agency. For certain complex issues, seeking the advice of an advisory committee not only makes our decisions better, but also adds an element of transparent societal discussion that’s important. But I also believe that our advisory committee system can be improved to enable our experts to get the best advice possible. Stay tuned for developments in this area.”
In closing, Califf addressed yet again what he sees as one of the agency’s biggest challenges — misinformation/disinformation. “One of the most basic responsibilities of the FDA is to disseminate facts about science and medicine to the public in order to help Americans make informed choices about their health,” he said. “But by undermining confidence in science, purveyors of misinformation make this much more difficult by weakening faith in governmental and other institutions, including the FDA, and in turn endangering the American public health.”
FDA has relaunched an updated “FDA Rumor Control” Web page, which “provides a place for people to find the facts on hot topics and learn more about how to spot and address misinformation,” Califf said. Additionally, he asked attendees to play a role in addressing misinformation. “Indeed, our fundamental authority to regulate the safety and efficacy of products has been challenged,” he said without mentioning any specific case. What I want to ask you to do as lawyers intimately familiar with and occasionally advocating for the crucial role the FDA plays is to stand strong for the work and principles of FDA and to not let them be distilled or destroyed through misinformation, disinformation, or partisan legal challenges.
“Many of you have worked at the FDA often in key positions,” Califf continued. “Some of you have litigated on behalf of the agency… I’m not asking you not to be advocates for your clients. My hope, however, is that in your current work, you go beyond simply abiding by the ethical rules governing lawyers who leave federal government practice for the private sector and seek to strengthen the environment in which we all operate. Each of you have the ability to help shape the debate and discussion to promote communication and collaboration and to work productively to reinforce the importance of our institutions. No matter what side of the legal proceeding you are on in taking this honorable path, you can help make sure that facts win out over misinformation and disinformation, that the principles we stand for are not eroded and that our ability to protect patients and consumers is reaffirmed and preserved.”