November 12, 2021
By Naomi Lopez

Breakthrough technologies. Personalized medicine. Modern communications tools. The ingredients are all there to build a more flexible healthcare system that’s truly centered around patients’ needs. But how will the next U.S. Food and Drug Administration (FDA) Commissioner help bring that system to life?

President Biden has nominated former FDA Commissioner Robert Califf to be the next leader of the agency. While there is no question that Dr. Califf is a capable candidate, having previously led the agency, daily decisions of the FDA Commissioner have the potential to shape American healthcare for decades to come, and so it is essential that Americans hear and understand the philosophy Dr. Califf will bring to this position especially in these difficult times.

While the FDA has been frequently criticized for its slowness and risk aversion, it has made important improvements in moving closer toward the goal of delivering better, more timely care to patients. But to continue building on this progress, Americans need to understand whether Dr. Califf will move closer to the goal of ensuring that Washington doesn’t stand in the way of patients accessing the care that they and their physicians believe will best meet their medical needs and personal preferences. Here are some questions we’d love to see the nominee address:

Right to Try. The federal Right to Try law, signed into federal law in May 2018, is saving lives today. What will you do to protect and expand the ability of patients to access promising investigational treatments as they work their way through the years-long FDA approval process? And do you fully support the medical autonomy of patients, under their physicians’ care, to seek investigational treatments?

Off-label communications. Off-label prescriptions are extremely common in the United States, accounting for about one in every five prescriptions. But under current FDA policy, communicating about an approved treatment for a legal, off-label use can result in criminal prosecution and penalties. As a physician, you understand the treatment needs of patients, but you’re well aware that not all physicians have access to full information about treatment options, especially when addressing with rare disease or aggressive forms of cancer, due to these FDA-imposed gag rules. What will you do to ensure that physicians and insurers have the most accurate, truthful information about how off-label treatments are being used to treat patients?

Reciprocity. Some of the world’s leading medical treatments are increasingly found in other countries. Do you believe that there should be a pathway for harnessing the important scientific work that has already been undertaken elsewhere on behalf of patients? And, if so, what approaches might you take to achieve patients in the U.S. have the broadest access to the world’s most innovative treatments?

Transparency. The FDA has a long and well-documented history of obscuring the decision-making process it uses to make life-or-death decisions that would allow patients to access its Compassionate Use program. In fact, it seems to routinely delay and deny FOIA requests related to the Compassionate Use program. As FDA Commissioner, what will you do to ensure that these crucial decisions are not government secrets?

Medical autonomy. Decisions about one’s medical treatments are among the most personal. When a patient’s life hangs in the balance, who should hold the ultimate authority over the best path forward: the patient under their doctor’s care or the federal government?

We are in the midst of a rapid period of medical innovation that holds the promise of delivering personalized treatments and curing diseases that, just a few short years ago, were death sentences. And there is a growing realization that today’s FDA, which was established in its current form in the 1960s, is not keeping pace with the innovations now taking place.

Perhaps now more than ever, an FDA that embraces innovation is key to a healthcare future that works for patients, and we look forward to hearing more soon about how we’re going to get to that future.

You can read the Goldwater Institute’s set of landmark FDA reform proposals, Putting Patients First: Unleashing Innovation in American Healthcare, here.

Naomi Lopez is the Director of Healthcare Policy at the Goldwater Institute.

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