Washington Watch
Congress managed to pass well-intentioned legislation recently allowing people with life-threatening illnesses to bypass the Food and Drug Administration (FDA) to obtain experimental medications. Unfortunately, it won’t do much to help the people who need the unapproved therapies the most.
The “right to try” legislation, pushed by Sen. Ron Johnson (R-Wis.) and Reps. Andy Biggs (R-Ariz.) and Brian Fitzpatrick, R-Pa., which Congress passed and President Donald J. Trump signed into law in May, gives terminally ill patients the right to seek drug treatments that remain in clinical trials and have passed phase one of the FDA’s approval process, but have not been fully approved. The new law allows patients and doctors to ask drug companies directly for access to the experimental drugs, rather than wait for approval by the agency.
But it does not require the drug companies to provide these medicines, leaving it more accurately like a right to ask drug companies for help instead of a right to try. The measure is really nothing more than a symbolic gesture, allowing politicians in an election year to say they’ve done something to help people, without doing much to actually help those people. Critics warn it could be dangerous and counterproductive.
Nearly everyone can empathize with these mostly-elderly patients’ desperate hopes and desires to try new therapeutic approaches to extend their lives. When you have nothing left to lose, any step helps. Facing terminal illness, most people would do everything possible to get access to any experimental therapies that might extend our lives or those of a loved one.
In recent years, the FDA has streamlined its complex procedures to make it much easier for terminally ill people to get access to experimental treatments. So what was a barrier when advocates started pushing right to try laws five years ago is no longer really a significant impediment to access.
But medical experts suggest that the effect of a new federal right to try law gives patients false hope and actually could put them at increased risk, undermining the ability to determine how well these treatments work.
Currently, 40 of the 50 states already have their own state right-to-try laws on the books (Alaska does not) and efforts are underway in the remaining 10 states. What the federal law will do is assist people in the states where no state law exists by instituting a federal law.
There is consensus that the bill will increase awareness about other options for the terminally ill and that could result in more data about experimental drugs being eventually made available. But that’s where agreement ends.
Let’s be clear -- people with terminal illnesses who want to live, should be able to get access to medicine that might be able to help them. No one wants to undermine the determination of people to live and if this gives them hope, that’s wonderful. But other laws already give them access, and the drug companies are not required to give them these untested drugs. The huge costs of these drugs and the need for the drug companies to provide them remain the biggest barriers.
Right now, drugs in the U.S. are regulated by the Food and Drug Administration. When a drug company develops a new drug intended for patient use, the medication goes through three phases of clinical trials that often take years to complete.
The first phase requires the company to prove the drug is relatively safe for humans, making sure that the drug itself will not poison the patient. These phase 1 trials are often conducted on as few as 30 patients.
According to the FDA, about 70 percent of these drugs move onto the next phases. Phase 2 and phase 3 clinical trials test whether the drug is effective at treating the condition which it is intended without problematic side effects. The later phases involve lots more people, and are where the vast majority fail because many drugs turn out to be ineffective or to cause severe side effects.
Expanded access
“We will be saving – I don’t even want to say thousands, because I think it’s going to be much more, thousand and thousands, hundreds of thousands,” President Trump said when he signed the measure into law. “We’re going to be saving tremendous numbers of lives.”
The right-to-try advocates say over a million Americans die annually from a terminal illness. Actually, there doesn’t appear to be anyone with a credible estimate of how many people might actually be able to use the new law. One of the reasons no numbers are available is because the vast majority of states do not have any central reporting requirements on when or how people apply to use the law.
The FDA, which regulates access to the approval process for these drugs, believes its current regulations adequately cover everyone who might need this. In the 1970s, the FDA set up its compassionate use or expanded access program. That allows patients with a serious disease or condition to obtain experimental medicines.
But for decades, people and doctors complained the approval process was burdensome and took about 100 hours to complete a single application. Those application forms called for 26 types of information and required seven attachments.
In addition to the excessive paperwork, the FDA required a 30-day delay, which dissuaded patients and doctors from petitioning. One reason for the long delays was that the FDA was doing its job instead of simply rubber stamping the applications. The FDA usually provides important feedback on safety monitoring, dosages and informed consent documentation.
These long holdups prompted states so start seeking right to try laws. Colorado was the first state to approve its, in 2014.
To address the problem, the Obama administration’s FDA streamlined the application process in 2015, and a 2016 report from the New York University School of Medicine found that the new application takes doctors approximately 45 minutes to complete.
Who wins, who waits, who loses?
Supporters of the right to try law argue that fewer than three percent of terminally ill patients gain access to these drugs through clinical trials. Right to try was designed to help the other 97 percent, according to Righttotry.org, the advocacy group’s website. They point to the comparatively low number of expanded access applications as an indication that the FDA process remains overly cumbersome and time consuming.
But the numbers don’t support that position. Last year, 99 percent of the almost 1,800 requests were approved, many in just hours or days.
An earlier version of the bill passed in the House in March. That included more safeguards, such as greater FDA oversight, a narrower definition of who is eligible to receive these drugs and the Expanded Access program’s patient-safety measures, and things like mandatory reporting requirements to follow up what happened to people who took these drugs. But in order to get legislation through quickly and signed into law, lawmakers took up the Senate version, and dropped the stronger requirements.
Opponents of the bill, including a group of more than 100 patient and provider advocacy groups like the American Cancer Society Cancer Action Network, Friends of Cancer Research and the National Organization for Rare Disorders, say the bill intentionally undercuts the FDA’s power.
Under the new law, the FDA cannot compel a pharmaceutical company to supply an experimental therapy to a patient. Drug companies can decline to because of concerns over liability, additional costs or limited supply. Companies warn that providing drugs via this experimental procedure could undermine their ongoing drug development process and could jeopardize formal approval down the road.
Opponents fear the bill would have a detrimental effect on how the FDA safeguards people’s health. “By removing the FDA oversight, you’re counting on physicians and manufacturers to serve as the gatekeeper and protector of our patients. I simply don’t buy that that’s going to work, argued Rep. Frank Pallone Jr., D-N.J., the ranking Democrat on the House Energy and Commerce Committee, on the floor shortly before the vote.
Michael Becker, a former biotech executive who suffers from terminal neck cancer, said that “the gullible will applaud” the passage of the measure but that he is worried about those who may be “targeted by unscrupulous snake oil salespeople seeking profits.”
Some doctors worried that removing the FDA from the equation leaves too much uncertainty surrounding drugs that haven’t been tested, especially when it comes to medically fragile patients taking drugs with unknown risks and benefits.
Doctors are flying blind on things like how much of the drugs to give, how to give it and what kind of side effects to look for, Dr. Steven Joffe, a medical ethics professor at the University of Pennsylvania told CNN. “So by cutting the FDA out of the loop, you are failing to take advantage of the knowledge that it has about how to use the drug.”
Unfortunately, the Right to Try Act won’t remove the primary roadblock facing terminally ill patients who want to test unapproved, investigational therapies: access to the drug by its developer. Art Caplan, director of the Division of Medical Ethics at the New York University School of Medicine, argues the law “is targeting the wrong obstacle. It’s basically saying the FDA is in the way, and that’s why people can’t try unapproved medicines,” he told CNN recently.
“It’s companies that control access to their drugs,” he added. Caplan argues that the FDA doesn’t act “until a doctor goes to a big pharmaceutical company and that company says they will provide the drug. If they don’t say yes, there is no FDA. So let’s be clear where the obstacles are.”
Leaving it up to the drug companies is one of the bill’s weaknesses and one of the reasons it’s more of a political accomplishment than something that will help lots of people. Drug companies have no obligation to honor right to try requests and major drug companies like Merck & Company, and Johnson & Johnson’s Janssen subsidiary have taken a negative view of the law.
The new law also exempts physicians and drug companies from any liability related to the prescription or dispensing of these unapproved therapies and does not require insurance companies to cover the costs of these drugs. So actually getting the medicines from manufacturers could be more cumbersome than going through the FDA’s program.
Who’s behind all this?
The hidden political motive behind the legislation appears to be to strip power away from the FDA, something that falls in line with the Trump administration’s deregulation push.
“A lot of this is smoke and mirrors for some other agenda,” said Andrew McFadyen, executive director of the Toronto-based Isaac Foundation, which assists U.S. and Canadian patients seeking access to medications. “A weaker FDA is what they are after,” he told the Washington Post.
Even the bill’s chief Senate author indirectly acknowledges that motivation. In a letter to FDA Commissioner Gottlieb, Wisconsin Republican Sen. Ron Johnson wrote “[This] legislation is fundamentally about empowering patients to make decisions in cooperation with their doctors and the developers of potentially life-saving therapies. This law intends to diminish the FDA’s power over people’s lives, not increase it.”
Also contributing to this report were: Washington Post, Stat, CNN, NPR, Vox and the New York Times.
