President Donald Trump’s brief foray into medical issues this year sounded almost reasonable. People do want lower drug prices, as he promised. And who could argue when he said he would help people with incurable diseases?
But there’s a problem with the channel he endorsed — the so-called right-to-try law that passed the U.S. Senate last summer. Experts who have been pushing to improve terminally ill patients’ access to experimental drugs say this law isn’t based on any realistic assessment of the problem. The law aims to limit the powers of the Food and Drug Administration, but FDA is doing much more good than harm in advancing medical science and promoting the interests of patients.
For decades, patients have been getting access to experimental treatments through what’s called a compassionate use or expanded access policy. A patient’s doctor can request such drugs from the manufacturers, and then get final approval from FDA. About 11,000 such requests have been granted in the last decade, according to the Washington Post.
A right-to-try law would change the current situation, mainly by taking FDA out of the equation. A libertarian nonprofit called the Goldwater Institute drafted model legislation for 38 state right-to-try laws as well as a federal bill.
Trump’s plug for the law implied a scandal that doesn’t exist: “People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home,” he said during his State of the Union address. But the FDA is not withholding a cache of miracle cures from the American public. Most experimental drugs for terminal diseases, both here and abroad, offer at best a small chance of a little more time. And patients are already able to get them.
One critic of the bill, New York University bioethics professor Alison Bateman-House, has been working on a panel devoted to helping terminally ill patients get access to experimental drugs. She’s therefore on board with the ostensible goal of right-to-try. But in her research, she’s found that the barrier — when there is one — isn’t FDA red tape but refusal on the part of drug companies or doctors. Under the current system, she said, the FDA approves 99 percent of requests within one day for emergencies and four days for non-emergencies.
And the FDA sometimes offers guidance to patients. It may suggest another more promising possibility or a different dosing rate, said Christopher Robertson, a law professor specializing in medical ethics and drug policy at the University of Arizona. “They are privy to all sorts of information that your average local doctor might not know about,” he said.
The Senate bill would also limit the ability of patients getting experimental drugs to sue drug companies or doctors if things go wrong — but the experts worry that there are better ways to encourage greater access. Bateman-House said many doctors simply don’t know how to get their patients such drugs. And doctors may not have the data to know which ones would be safest and most likely to help. The other barrier is with drug companies, who would retain the power to refuse patient requests under the Senate bill. Patients would not, as the bill’s name implies, really gain a right to try anything.
There’s an assumption behind the right-to-try law that patients have nothing to lose. But even patients diagnosed with terminal conditions may have months or even years to live using currently approved treatments.
While there’s inefficiency in every bureaucracy, FDA is in the business of enforcing the use of well-established scientific methods to determine whether drugs are reasonably safe and have any probability of doing anyone any good. I talked about this with Stanford researcher Matthew Porteus, who is waiting for FDA to give him the green light to start testing the novel technology known as Crispr on patients with sickle cell disease. He said that while Chinese researchers have already started human trials for various Crispr-based therapies, he thinks the more stringent U.S. rules are better not only for protecting patients, but for advancing science by ensuring data collection is done openly and coherently.
And as University of Arizona’s Robertson pointed out, most drugs aren’t cures. Many approved cancer drugs show minuscule effects in slowing the growth of tumors or improving blood-test results. Drugs don’t even have to demonstrate that they extend patients’ lives to be approved.
A better way to help terminal patients would be to expand clinical trials, Robertson suggested. That way more people could get experimental drugs and their data would be tallied to help further medical understanding. Some patients can’t get enrolled in a trial because they live in rural areas, or they have secondary conditions — diabetes in addition to cancer, say.
There are reasons for excluding people from trials: It’s easier for researchers to get a clear signal when there’s only one condition, for example. But people with secondary conditions could be included in separate arms of a clinical trial, in a way that their data would still be of use.
In his speech, Trump praised the FDA for approving a record number of drugs in the last year. But it makes no sense to measure the FDA’s performance this way. If they stopped doing their jobs altogether, they could get an even higher number.
One of the FDA’s historic achievements was holding up approval of thalidomide, asking for more safety evidence while use in other countries led to a rash of birth defects. The strongest recent criticisms of the FDA have centered not on holding back cures but on being too lax in approvals of opioids. In the same speech, Trump said he was committed to the opioid crisis and would fight for those who had been hurt by drugs — without seeming to recognize the contradiction.
Faye Flam is a Bloomberg View columnist.