In Their Own Words

These Patients Have Nothing Else. Should That Be Enough for FDA Drug Approval?

Despite Streamlined Processes, FDA’s Role in Rare-Disease Drug Development Draws Continued Scrutiny

October 21, 2025

By:

Holly Fernandez Lynch, JD, MBE
and Remsha Ramachandran, MD, MPP, MHS

Print:

Four pills sitting on top of a magnifying glass with a clock in the background.

This op-ed was published in the Los Angeles Times on October 19, 2025.

If you’re faced with a serious disease, you better hope it’s not a rare one.

After an often tortuous path to diagnosis, people with rare diseases are likely to find that good treatment options don’t exist and none is on the horizon. Many of these conditions are poorly understood, and conducting studies in tiny patient populations can be practically impossible. Most drugs won’t pan out, and those that do will have little demand and financial payoff, no matter how beneficial they are. Drug companies usually direct their attention elsewhere.

Recognizing these challenges, policymakers have worked since the 1980s to encourage rare-disease drug development. They’ve earmarked federal research funding, established dedicated programs at the Food and Drug Administration, extended protection against competition to help secure profits, and awarded lucrative incentives for rare pediatric disease drugs. Still, 95% of rare diseases, which affect an estimated 30 million Americans, lack any approved therapy.

Some blame the FDA. They say the agency is too rigid, imposing impossible requirements and demanding unreasonable proof of effectiveness and safety for rare disease drugs. Some suggest patients would be better off if the FDA just got out of the way — not only for rare disease treatments, but also more broadly.

The current administration now seems determined to do just that, at least for products that fit the “Make America Healthy Again” agenda, like stem cells and psychedelics (never mind recent intrusions on COVID vaccines and abortion drugs).

Large swaths of FDA experts have been DOGE’d or otherwise forced out of the agency. Commissioner Martin Makary has proposed approving drugs based just on their scientific plausibility, while the agency’s chief medical and scientific officer pledged to “take action at the first sign of promise for rare diseases” — potentially making treatments available far sooner, even though many drugs that look promising at the start turn out not to work.

Just last month, the FDA announced approval of a drug the commissioner claimed would help “hundreds of thousands of kids” with autism, not based on a clinical trial but on published case reports of 40 patients with a potentially related condition — alarmingly and unprecedentedly accepting anecdotes as evidence of efficacy.

The call to disarm the FDA is coming from inside the house.

Read the full op-ed here.