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Is Right to Try Saving a ‘Lot of People’?


President Donald Trump last year signed a bill — the Right to Try Act — that provided another avenue for terminally ill patients to gain access to unapproved medications still in development. But there’s no evidence that the new law is saving “a lot of people,” as the president claims.

The U.S. Food and Drug Administration, which is responsible for collecting data on patients treated with so-called investigational medicines under the Right to Try Act, told us that it has no information on the number of people who have used the law to gain access to the medications. A group that advocated for the new law has publicly identified two people who have used the law.

The president made his remarks at a White House meeting on another health issue, e-cigarettes.

Trump, Sept. 11: [W]e’ve done more than any administration probably in the history of the country. You just look at one point after another point, whether it’s regulation cuts, whether it’s tax cuts. You look at Right to Try … where people are able to use some of the incredible innovations that we’ve developed with the greatest labs and the greatest doctors in the world. And they can use them, instead of being forced to move to — and leave — to other countries that don’t have a clue, compared to us. And now they have Right to Try.

And, by the way, a lot of people are being saved. A lot of great things are happening with Right to Try.

He brought it up again two days later in a speech to House Republicans at their annual conference retreat, which was held this year in Baltimore. It was the third time this month that the president touted the success of the program. In Baltimore, the president congratulated the Republicans for passing the bill, which he signed more than a year ago on May 30, 2018.

Trump, Sept. 13: And you have to see the success. People used to go to Asia. They’d go to Europe. They’d go all over the world looking, if they had money. Most people didn’t have the money, so they’d go home and they’d die. Now they have the Right to Try.

First, to be clear, the FDA for years has approved applications from patients seeking access to investigational drugs through the agency’s “expanded access” program, which oversees the use of such medicines for patients who are not able to participate in clinical trials.

The FDA’s “history of facilitating access to investigational therapies reaches back to the 1970s; however, regulations did not specifically describe a pathway for access to unapproved drugs until 1987,” FDA officials write in a May 2017 paper on the history of the program. “FDA later published revised regulations in 2009, further clarifying the expanded access process.”

As we have written before, then-FDA Commissioner Dr. Scott Gottlieb told Congress in October 2017 that the FDA had approved 99 percent of the more than 1,000 annual applications it has received in recent years for “expanded access to treat patients with investigational drugs and biologics.”

The new law provides a new path to investigational medicines. It circumvents the FDA and gives terminally ill patients access to unapproved drugs more quickly than through the FDA’s expanded access program. But we can’t find any evidence that “a lot of people are being saved” as a result of it.

The Right to Try Act requires drug manufacturers to submit an annual summary report of any use of investigational medicines. The summary must include information on “the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events,” according to the law.

The law also requires the FDA to post that information on its website. But no information is available.

So far, the FDA has only published a public notice saying that the agency intends to propose a deadline for drug manufacturers to submit annual reports. But it hasn’t proposed a deadline for those reports yet.

“The FDA does not currently have information on the number of patients who have accessed drugs, or the drugs that have been sought for use, under Right to Try,” FDA spokeswoman Sandy Walsh told us in an email.

The Goldwater Institute – a proponent of the law – has disclosed examples of two people being helped by the new law in the first year. The institute told us that it expects later this month to announce more patients who were helped.

One of the two people helped by the law so far is Matt Bellina, an ALS patient who advocated for the law and attended the bill-signing ceremony. Bellina, whose name appears in the bill’s title, used the new law to gain access to NurOwn — an experimental drug that is being developed by an Israeli-based company called BrainStorm Cell Therapeutics.

But that company announced a year ago that it will not provide access to any other patients, citing costs. The new law doesn’t require insurance companies to pay for such treatments.

“As we were unable to identify a practical funding solution, we unfortunately are not in a position to initiate access to NurOwn under RTT at this time,” Chaim Lebovits, president and CEO of BrainStorm, said in a June 26, 2018, statement.

Lebovits told Reuters at that time that BrainStorm would provide NurOwn under the new law only to Bellina, and that Lebovitz would personally pay for it.

Other companies have decided not to make their investigational medicines available under the new law, either, because it does not require FDA oversight.

At least two major pharmaceutical companies – Pfizer and Janssen, a Johnson & Johnson company – have said that they will make their investigational drugs available only through a process that involves FDA oversight, referring to the FDA’s existing expanded access program, according to Alison Bateman-House, co-chair of the NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access. Bateman-House has advised both Pfizer and Johnson & Johnson on investigational medicines.

When the Right to Try Act was being considered by Congress, Pfizer issued a position statement that said, “Pfizer supports policies that protect the integrity of clinical trials and FDA oversight to maintain the best interests of patients, with an emphasis on safety. … Pfizer’s overarching policy principle with respect to Federal Right to Try Legislation is to protect the FDA oversight of expanded access, safety, and the integrity of clinical trials.”

Pfizer’s statement noted that in 2017, before Right to Try was enacted, the company “received 4,818 requests for preapproval access to 24 investigative medicines from 59 countries; approximately 98 percent of requests were granted.”

Janssen issued a similar policy statement on investigational medicines prior to the adoption of the new federal law that stressed its support for FDA oversight.

Janssen’s policy, in the form of a question and answer, including this: “Does Janssen accept Right to Try requests? We are committed to helping patients with serious illnesses and their families request access to our investigational medicines. We support these requests through our established review and evaluation processes, which includes independent review by the FDA to assure full consideration of available safety data of which the FDA may be uniquely aware.”

In an email, Bateman-House told us: “My educated guess is that the number of patients utilizing Right to Try is a mere trickle, if that.”

We don’t know that that is the case, either. All we can say for sure is that two people so far have accessed investigational drugs using the new system, according to a group that supports the new law.