President Donald Trump’s choice to lead the Food and Drug Administration will inherit an agency undergoing staffing and leadership changes, as well as responsibility for regulatory decisions affecting people with cancer.
Trump has selected Dr. Heidi Overton, a White House policy adviser, to lead the FDA. Her appointment requires Senate confirmation. Overton is a physician and serves as deputy director of the White House Domestic Policy Council. She previously worked at the America First Policy Institute, a conservative think tank.
Overton received her medical degree from the University of New Mexico, trained in general surgery at Johns Hopkins and completed clinical investigation research training at the Johns Hopkins Bloomberg School of Public Health.
If confirmed, Overton would take charge of an FDA that has lost more than 3,000 employees over the past year, including division leaders. The departures, low morale and disputes over drug approvals have placed the agency under pressure to rebuild confidence among pharmaceutical companies and public health organizations.
For the cancer community, the leadership change matters because the FDA evaluates whether new cancer drugs and biologic treatments meet US requirements for safety and effectiveness. The agency’s Office of Oncologic Diseases oversees the development, approval and regulation of drugs and biologics used to treat cancer and hematologic malignancies.
The nomination therefore brings attention to a continuing issue in cancer regulation. How can the FDA provide timely access to promising treatments while requiring sufficient evidence of their safety and effectiveness?
The FDA commissioner’s role in cancer drug regulation
The FDA commissioner does not personally evaluate every cancer drug application. Those applications are reviewed by specialist offices within the agency, including the Office of Oncologic Diseases.
The Office of the Commissioner provides agency wide direction and management. It is responsible for ensuring that FDA programs operate within the agency’s regulatory framework and use available resources effectively.
The FDA’s Oncology Center of Excellence coordinates cancer related work across the agency. Its stated mission is to achieve patient centered regulatory decision making through innovation and collaboration.
In 2024, the Office of Oncologic Diseases approved 17 novel drugs for several cancer types. These included lung, breast, thyroid, gastrointestinal, gynecologic and genitourinary cancers, as well as leukemia, lymphoma and myelodysplastic syndromes. The FDA defines these novel drugs as new molecular entities or biologic products.
Oncology reviews can involve assessments of clinical trial design, tumor response, treatment safety and whether study results demonstrate clinical benefit for patients. The effect of the FDA’s recent staff losses on individual cancer drug reviews has not been established.
The debate over accelerated approval
The FDA’s accelerated approval pathway is among the regulatory programs that would fall under the agency’s next commissioner.
The program allows the FDA to approve drugs for serious conditions that address an unmet medical need based on a surrogate endpoint. A surrogate endpoint can be a laboratory measurement, medical image, physical sign or another measure expected to predict clinical benefit. It is not itself a direct measure of how a patient feels, functions or survives.
In oncology, accelerated approvals have frequently relied on overall response rate. This measures the proportion of patients whose tumors shrink or disappear after treatment. The FDA also considers the duration of those responses.
The majority of accelerated approvals issued since the pathway began in 1992 have involved oncology and hematology indications. The FDA says the pathway has made certain cancer therapies available a median of 3.1 years earlier than they would have been available through the traditional approval process.
Accelerated approval allows treatments to become available earlier, but uncertainty can remain about whether the initial findings will translate into clinical benefit. An effect on a surrogate endpoint does not by itself establish that patients will live longer, function better or experience an improved quality of life.
Sponsors of drugs granted accelerated approval must complete confirmatory studies to verify the anticipated clinical benefit. When a study confirms the benefit, the FDA generally ends the requirement. If a study fails to confirm sufficient benefit, the agency may withdraw the approval or change the drug’s approved indication.
Through Project Confirm, the FDA publishes information on oncology indications that have been converted to traditional approval, remain subject to ongoing requirements or have been withdrawn.
The pathway is intended to provide earlier access to treatments while requiring additional evidence after approval. The next commissioner would oversee how the FDA applies these requirements and responds when confirmatory studies do not verify the anticipated benefit.
Efforts to speed up oncology reviews
The FDA has developed programs intended to make oncology reviews more efficient while maintaining its requirements for safety and effectiveness.
The Real Time Oncology Review program allows FDA reviewers to begin examining selected trial results and datasets before a company submits its complete application. According to the FDA, the program is intended to improve review efficiency and quality, support earlier communication with applicants and make safe and effective treatments available to patients as early as possible.
The Oncology Center of Excellence launched Project Orbis in May 2019. The program provides a framework for the concurrent submission and review of selected oncology products by the FDA and participating international regulators.
Both programs are designed to make parts of the review process more efficient. They do not replace the independent evaluations conducted by participating regulators or the standards used to assess whether a treatment is safe and effective.
Leadership changes at the FDA
Overton would become the FDA’s permanent commissioner following the departure of Marty Makary, who resigned in May after 13 months in the position. Kyle Diamantas has served as acting commissioner since then. Makary’s departure followed disagreements with White House and health officials involving flavored vaping products, an abortion pill safety review and the handling of several drug and vaccine decisions.
The effect of these leadership and staffing changes on individual cancer drug reviews has not been established. If confirmed, Overton would oversee the FDA offices responsible for evaluating cancer treatments while the agency addresses its recent personnel changes.
Questions facing the next FDA commissioner
The announcement of Overton’s selection did not include a detailed oncology policy. It therefore remains unclear whether she would seek to change the FDA’s approach to cancer drug regulation.
Her confirmation process may provide more information about how she intends to manage the FDA’s scientific workforce and oversee programs designed to accelerate oncology reviews.
Another issue will be the FDA’s oversight of cancer drugs granted accelerated approval. These treatments require confirmatory studies to verify their anticipated clinical benefit. The agency may change or withdraw an approval when those studies fail to demonstrate sufficient benefit.
The Oncology Center of Excellence identifies patient centered regulatory decision making as part of its mission. The next commissioner would oversee the agency as it evaluates when cancer treatments have sufficient evidence for approval and how their benefits should be verified afterward.
For cancer patients and physicians, the main questions will be whether the FDA can maintain consistent scientific reviews, enforce its evidence requirements and continue operating its oncology programs during a period of staffing and leadership changes.
Written By Evelina Kachaturova