Key Takeaways
- FDA's expanded access program lets seriously ill patients, small groups, or larger populations obtain an investigational drug outside a clinical trial when certain conditions are met.
- Doctors may generally prescribe an approved drug for an unapproved use as part of the practice of medicine, though they can face malpractice liability.
- Whether FDA can penalize drug companies for truthful, non-misleading off-label promotion remains contested, and the Supreme Court has not resolved it.
When public figures tout an untested drug as a possible cure, what does the law actually say about getting access to it?
In this TalksOnLaw Brief, Glenn Cohen, Deputy Dean and the James A. Attwood and Leslie Williams Professor of Law at Harvard Law School and faculty director of its Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics, explains the rules governing unapproved drugs and unapproved uses of approved drugs. He begins with a caution about the risks of taking drugs that have not been cleared for a particular use, recounting the tragedy of a man who died after consuming a form of a drug meant for fish in a koi pond.
FDA Approval and Expanded Access
Professor Cohen explains that the Food and Drug Administration has primary authority to approve drugs in the United States. It reviews three phases of clinical trials designed to show that a drug is safe and effective for a particular indication with an acceptable side effect profile. The process is long and expensive, and many drugs never make it through, but it is time-tested.
FDA recognizes that some patients, such as the terminally ill, cannot wait. Through its expanded access program, often called compassionate use, it can permit use of an investigational drug for an individual, a small group, or even widespread use. He also describes Right to Try laws, adopted in many states and then by the federal government, which offer an even more capacious route and shift much of the decision-making and regulatory burden away from FDA.
Off-Label Use
Drugs already approved for one use can be prescribed for another. FDA treats that as the practice of medicine, which it does not regulate, so a physician who prescribes a drug approved for one condition to treat a different one does not violate the Food, Drug, and Cosmetic Act, though tort liability remains possible.
Off-Label Promotion
Promotion by drug companies is trickier. FDA has long claimed authority over the promotion of off-label uses, but it has lost a number of cases, beginning in the lower federal courts, on First Amendment grounds, and the government has not pushed the issue to the Supreme Court. He flags it as a question a future Court might take up.
What to Know Now
The early COVID-19 drug debates moved quickly. FDA issued an emergency use authorization for hydroxychloroquine and chloroquine in March 2020 and revoked it that June, concluding the drugs were unlikely to be effective, while remdesivir became the first FDA-approved COVID-19 treatment in October 2020. Emergency use authorization, a pathway separate from expanded access, became the main legal route for pandemic products. The expanded access regulations and the federal Right to Try Act of 2018 remain in place. On off-label speech, United States v. Caronia (2d Cir. 2012) and Amarin Pharma v. FDA (S.D.N.Y. 2015) are still the leading decisions protecting truthful, non-misleading promotion, and the Supreme Court has not addressed the question. A related dispute involved ivermectin: in Apter v. HHS (2023), the Fifth Circuit let physicians pursue claims that FDA's public messaging against using the drug for COVID-19 exceeded its authority, and the case later settled with FDA removing those statements.
