November 18, 2019
This Wednesday, Dr. Stephen M. Hahn—the nominee to be the next U.S. Food & Drug Administration (FDA) Commissioner—will appear before the Senate Health, Education, Labor, and Pensions (HELP) Committee for a confirmation hearing on his nomination. The 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. Hahn will bring to this position.
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.
The FDA, often with congressional directives, has made important improvements in moving closer toward the goal of bringing the right treatment to the right patient, and at the right time. For example, treatments allowed under the federal Right to Try Act are the same treatments available to patients with the same disease who are lucky enough to qualify for a clinical trial. By removing the federal red tape, Right to Try is starting to make more treatments accessible for those patients whose lives hang in the balance.
In order to continue to build on this progress, Americans need to know about and understand if and how Dr. Hahn 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 answered this Wednesday:
Right to Try. The federal Right to Try law, signed by President Trump 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?
Off-label communications. Under current FDA policy, communicating about an approved treatment for a legal, off-label use can result in criminal prosecution and penalties even though using off-label treatments are an essential (and lawful) part of a physician’s toolbox. As a physician, you understand the treatment needs of patients but know 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 allow patients to access its Compassionate Use program. In fact, it seems to routinely deny FOIA requests related to the Compassionate Use program.
Not only are there years-long legal challenges involving these matters in federal court (including one notable one from the Goldwater Institute asking for the decision criteria used to grant an Ebola treatment to U.S. aid workers who received medical evacuation out of Liberia), but the FDA has staked out a position that is in stark opposition to the Freedom of Information Act (FOIA). In particular, the FDA has refused to answer As FDA Commissioner, what will you do to ensure that these life-or-death 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?
An FDA that embraces innovation is key to a healthcare future that works for patients, and we look forward to hearing more at this Wednesday’s hearing about we’re going to get to that future.