When Lorraine Heidke-McCartin ran out of options to treat her aggressive strain of breast cancer — which was diagnosed in 2006 — she did what many women in her position would do. She and her husband started looking for alternative ways to save her life.
And like other people facing the ticking clock of a terminal illness, Heidke-McCartin turned to experimental treatments.
They included a drug, called T-DM1, which was still undergoing clinical trials and had not yet been approved by the Food and Drug Administration (FDA).
That meant there were only two routes open for her to get this potentially life-saving drug. The first was to enroll in one of the ongoing clinical trials. But these only have room for a fraction of the number of people who stand to benefit from the drug once it’s shown to be safe and effective.
The other option would be to request the drug through an expanded access program, which allows “compassionate use” of experimental drugs by terminally ill patients. At the time, the drug’s maker, the Genentech division of Roche Holding AG, had this kind of program for T-DM1.
But before Heidke-McCartin was able to request the drug, the company shut down the expanded access program in Boston, citing delays in the approval process by the FDA.
Among people with a terminal illness and few options for treatment, Heidke-McCartin’s story is not uncommon. It’s also one of several highlighted on the website of the Arizona-based Goldwater Institute, a conservative and libertarian think tank.
The organization is encouraging states to pass “Right to Try” laws that allow patients, like Heidke-McCartin, who are ineligible or unable to participate in clinical trials to gain access to experimental drugs. To do so, patients must have a doctor’s recommendation and have first exhausted all other treatment options.
“Our goal is really to help increase access to potentially life-saving drugs,” said Kurt Altman, the director of national affairs and special counsel for the Goldwater Institute.
Altman and a colleague drafted the legislation that a growing number of states have used as a model for their own initiatives. According to the Regulatory Affairs Professionals Society, 19 states so far have passed Right to Try laws, with Arizona approving it by a ballot measure last November. In addition, legislation is waiting for the governor’s signature in three states.
Unknown Side Effects Could Worsen Quality of Life
On the surface, few people would disagree with a law designed to speed up getting drugs to terminally ill people who have nowhere else to turn. But once you wade into the details, not everyone in the medical community agrees that Right to Try legislation will help all patients.
One of the main concerns is that these laws target drugs that are still in clinical trials and have not yet been approved by the FDA. That means their safety and effectiveness are not completely known.
“There’s an idea at that point that they are not going to kill somebody. That they’re relatively safe,” said Craig Klugman, Ph.D., a bioethicist and medical anthropologist at DePaul University. “But there may be a lot of side effects and we’ll have no idea what they are.”
The earliest type of clinical trials — known as phase I — are designed to test safety, identify major side effects, and determine the best dose of a drug to use. But these studies involve only a small number of people.
Once a drug makes it through that stage, larger trials are needed to really test how well a drug works. These later phases also may catch side effects that don’t show up in smaller trials.
Although terminally ill patients may have nothing left to lose, unknown side effects could worsen their quality of life toward the end.
“Whatever time that people have left could be made much worse,” said Klugman. “It could increase pain, it could increase suffering, and make whatever time they do have left be a rather miserable experience — on a pipe dream.”
Critics Worried About Drug Free-For-All
In theory, Right to Try would allow patients to request an experimental drug at any stage in its development process. But some promoters of earlier access to drugs say that the focus should be on pushing promising drugs along faster — those that have been shown in clinical trials to work well with minimal safety concerns.
“The Abigail Alliance has never endorsed a free-for-all,” said Frank Burroughs, founder of the Virginia-based Abigail Alliance, a nonprofit organization that pushes for earlier access to new drugs under development.
His daughter, Abigail, died from head and neck cancer at age 21 in 2001. Before she died, her doctors told her family about an experimental drug that targeted the same type of cancer that she had. The drug was performing well in clinical trials at the time, but those studies were for colon cancer. So she was not able to participate.
Her family pushed the pharmaceutical companies and Congress for access to the drug. They also mounted an extensive media campaign. In spite of the publicity, the FDA did not approve the drug until four years after she died.
Burroughs is not concerned about Right to Try leading to a free-for-all because he has found that many terminally ill patients do their homework on what might work for their disease.
“We hear over and over from the patients that call us, that send us emails — and sometimes it’s groups of patients — that they’re looking at a drug that has shown promise in early clinical trials, not just any drugs,” said Burroughs.
FDA’s Compassionate Use Program Too Slow
Since states started introducing Right to Try legislature — many of them only in the current session — the movement has gained a lot of traction.
“Hopefully by the end of summer we’re beyond half the states having passed laws like this,” said Altman.
The FDA has had rules in place since 1987 that have allowed patients to access experimental drugs under certain circumstances.
However, critics of the FDA’s expanded access program say the process requires too much of the one thing that terminally ill patients don’t have enough of — time.
There is some sign of progress in this area. The FDA announced earlier this year that its expanded access form will only take 45 minutes for a doctor to fill out. But this change has yet to be implemented.
“Even though the FDA has proposed a rule change, to this day it hasn’t taken effect,” said Altman. “It still takes just the physician over 100 hours to complete the required paperwork to apply through the [FDA’s] compassionate use program.”
And this doesn’t even take into account the time required for the drug company to respond or for the FDA to review the request, which can add up to months. Right to Try advocates hope to speed up the entire process of getting experimental drugs to patients.
“We set out at the state level to attempt to cut that two to four months down to two to four weeks, and eventually — hopefully — to two to four days,” said Altman.
Faster Drug Approval May Help More Patients
For some advocates of Right to Try, the legislation takes the FDA out of the process and leaves decisions about medical care where it belongs — between a patient and doctor. But others are concerned that doing this will only compromise safety.
“The problem here is that [Right to Try] basically undermines the system we have that works,” said Klugman. “We have this FDA review system that reviews drugs to make sure they’re safe and make sure they’re effective. It prevents people from being harmed by drugs for the most part.”
Right to Try, though, is not the only avenue for getting treatments to terminally ill patients. Although the Abigail Alliance supports Right to Try legislation at the state level, it focuses more of its energy on encouraging the FDA and Congress to speed up the approval process of promising drugs.
“When you have promising drugs that are showing efficacy and safety in early clinical trials, we need those drugs approved earlier,” said Burroughs. “That’s the way to help the vast majority of people who’ve run out of FDA options and can’t get into clinical trials.”
Speeding up the overall approval process may also help terminally ill patients in other ways.
Right to Try laws allow patients to request an experimental drug from a pharmaceutical company. But compared to a full-scale rollout of an FDA-approved drug, only limited batches of experimental drugs are made for clinical trials. This makes them expensive.
These drugs are “not covered by insurance because experimental treatments, in general, are not covered by insurance,” said Klugman. “It’s not covered by your federal programs. It’s not covered by your state programs. So you have to pay for this out of pocket.”
There is also the problem of limited availability. Just because a patient asks for a drug through Right to Try, it doesn’t mean that a company has enough available to give the drug to people outside of its clinical trials.
This means that drug companies need to decide which patients receive experimental drugs through compassionate use programs. Which can leave them open to pressure or attacks from high-profile social media campaigns by patients and their families.
In response to the challenges of running compassionate use programs, Johnson & Johnson announced recently that it would create an independent advisory board. This group — composed of physicians, bioethicists, and patient advocates — will review requests to the company’s pharmaceutical division for access to experimental drugs.
Right to Try Signals Public Push for Change
It’s too soon to know how the Right to Try movement will play out, but Burroughs says one thing is certain:
“The Right to Try laws keep sending a powerful signal to the FDA and to Congress, saying the public wants this change,” said Burroughs. “We want these drugs approved sooner.”
Faster approval might have helped Burroughs’ daughter. And it might’ve also sped up Heidke-McCartin’s recovery.
After the drug company closed the expanded access program in Boston, Heidke-McCartin was able to get T-DM1 at another site in Virginia. This required taking more than 16 trips back and forth for treatment before the FDA finally allowed her to take the drug in Boston.
In a YouTube video about her experience, Heidke-McCartin sums up the feelings of many patients with terminal illnesses, their families, and those advocating for faster approval of promising drugs.
“If there are drugs that are available, and they’ve passed the safety standards,” she said, “there doesn’t seem to be a reason why you should hold them back from somebody.”