About a month ago, a group of people with terminal illnesses sent letters to every member of Parliament, asking for legislation that would give dying patients the right to access unapproved drugs and treatments.
“I’m going to die anyway,” said right-to-try campaign co-founder Jeff Perreault, who has ALS, also known as Lou Gehrig’s disease.
While competent patients facing a forseeable death have the legal “right to die” in Canada, they don’t have the “right to try” something that might prolong their lives, Perreault argued.
“We have the law that allows us to kill ourselves. We just don’t have to law to do something else.”
Federal Health Minister Jane Philpott met with Perreault about two weeks ago.
Jane Philpott, Minister of Health.
“I have asked my officials to continue this conversation so that his concerns can be addressed appropriately, and to determine what further actions are required,” Philpott said in a statement.
The idea behind “right-to-try” seems like a no-brainer. Surely only the hardest of hearts would deny a dying person one last shot at a longer or more comfortable life. In the United States, 32 states have already passed right-to-try legislation. Now, the movement appears to be picking up steam in Canada.
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Owen Thomas, 31, was one of the signatories of the letter. The man from Bridgewater, N.S., noticed stiffness in his legs in the summer 2014. He was diagnosed with ALS last January and retired from his law practice this September.
Once an avid drummer and weightlifter, Thomas can now do neither. He uses a walker, needs help dressing and can’t cook for himself.
“Time is of the essence for terminally ill patients,” he says. “Right to try should be considered a human right.”
Owen Thomas is a Nova Scotia lawyer who was diagnosed with ALS in January 2016.
But opponents argue it would be a mistake to legislate right-to-try in Canada.
“Right-to-try has been very emotional. Why not give these people a chance? Because what happens to these people doesn’t affect just these people, but thousands of other faceless downstream patients,” says Alison Bateman-House, a bioethicst at New York University who has been keeping an eye on the movement in the U.S.
“It’s very scary to see that there is a movement to roll back protections that are there for a reason.”
First, pharmaceutical companies have no obligation to provide access to unapproved treatments, even if right-to-try legislation is passed, Bateman-House says. “You can’t make a private business provide a product in development, even in the case of a national emergency.”
She says she doesn’t believe there is a need to liberalize existing regimes to offer access.
Pharmaceutical companies are already often quite willing to make drugs in development available to patients on a compassionate basis, as long as the U.S. Food and Drug Administration or Health Canada requirements have been fulfilled. It is usually a matter of days or hours for a patient to learn whether they will have access to an unapproved drug, she says.
When a drug is at the end of its clinical testing — and there is a good supply of the drug, which is not always the case — companies are often quite willing to allow access.
“It’s good word-of-mouth, and a boost for marketing.”
As a bioethicist, Alison Bateman-House says she sees the problems and the slippery slope posed by right-to-try.
Companies may also be willing to make drugs available if they have proved to be successful against a variety of conditions — for example, an anti-viral that has been proven for a number of viruses, and the manufacturer believes it will also be successful in other cases. It gives the company anecdotal evidence that the drug will work in another class of viruses.
But the opposite can also happen and it makes sense that drug companies want to be cautious.
“If they give out a drug and it doesn’t work, then that word gets out,” said Bateman-House. “It also has to be reported to regulators. Drug companies spend millions of dollars and 10-plus- years developing a drug. Why risk having a black mark against the drug?”
She said she can see why patients get frustrated. Drug companies have no uniform rules when it comes to providing drugs on a compassionate basis. Some allow access under transparent policies, some deny access. Some change their practices under public pressure. It can vary depending on the drug.
It’s not unfathomable that right-to-try will come to Canada, says Timothy Caulfield, the Canada Research Chair in Health Law and Policy and a professor in law and public health at the University of Alberta. That said, it’s not a step he would like to see.
There’s no evidence that efficacious treatment is being withheld from patients, says Timothy Caulfield, a professor of law and public health.
What Caulfield finds most concerning is that right-to-try campaigns work on the underlying idea that the regulatory framework is withholding useful therapies.
“The steps needed to get something to clinical trials are treated as bureaucratic hurdles, not steps in a scientific process. Once something is on the market, it’s hard to get it off the market,” he says. “It’s one thing to access a treatment with no scientific basis. It’s another thing to create a regulatory framework that allows it.”
Caulfield argues there isn’t any scientific evidence to back up the idea that efficacious treatments are being withheld from people who would benefit from them.
“Exciting stuff is happening in stem-cell research. But there are few treatments that are ready for clinical applications. They’re just not ready yet.”
The list of stem cells therapies that are available now is very short: bone marrow treatments for some types of cancer; skin grafting and some eye treatments, he says. “That’s about it. There’s exciting stuff going on in multiple sclerosis, but it’s not ready yet.”
Many desperate patients will nonetheless leave the country to access clinics that offer unproven treatments. He points to the example of the now-discredited “liberation therapy,” a treatment for multiple sclerosis patients. Driven by the power of testimonials, dozens of Canadians left the country to be treated.
“Unfortunately, pressure from interest groups can trump science,” says Caulfield, who has studied the effect celebrity health trends have had on ordinary health consumers.
“A lot of these clinics offer false hope. Therapies are expensive, and there’s no evidence to suggest they’re effective,” he says. “Patients aren’t the only ones who will lose out. There’s also harm to the legitimacy of the science.”
Caulfield says when he is skeptical about treatments such as liberation therapy in public appearances, he often gets jeered and shouted down.
“There’s this idea that I’m taking away hope. But I don’t have a horse in this race. I don’t think people should be deceived.”
Barbara von Tigerstrom, a law professor at the University of Saskatchewan who specializes in health law, doesn’t see Health Canada’s regulatory requirements creating a barrier for people. Clinical trials might be expensive and time-consuming, but they are necessary to maintain high standards for safety and efficacy, she said.
Law professor Barbara von Tigerstrom specializes in health law.
One of the difficult things about right-to-try is that it rests on skepticism of government regulation and the sense that the government is a barrier to people’s freedoms and rights, she says.
“The underlying assumption is that ‘There is a promising treatment but the government won’t let me have it,'” she says. “I can understand. People are frustrated by the lack of options for treatments. People seem to be under the misconception that there are effective treatments out there that don’t really exist.”
But there’s no harm in trying, right?
Actually, that’s not always true, she says. Some patients have suffered devastating consequences as a result of trying experimental treatments. Last month, for example, Juno Therapeutics Inc. of Seattle reported that two more patients had died after suffering brain swelling during a trial of an experimental genetically engineered leukemia drug that extracted T-cells from a patient’s immune system and altered their DNA to allow them to better find and kill cancer cells.
The two deaths brought the total number of patients who have died to five. In July, three other patients had died from brain swelling in the same trial testing the drug, called JCAR015. Early studies had shown that CAR-T therapies can eliminate blood cancers, such as leukemia and lymphoma in 40 to 90 per cent of patients.
Patients and physicians may give too much weight to the results of very early studies because of a misunderstanding about what it means when a treatment is “showing promise,” says von Tigerstrom. About 90 per cent of treatments that show early promise fail to be approved for market.
Thomas, however, argues that right-to-try applies to only a small fraction of the population, mostly people with ALS and certain types of cancer. And he says it’s patronizing to suggest that patients can’t decide for themselves.
“You’re terminally ill, but you’re still a rational being. I think the vulnerability argument has been blown out of proportion.”
One of the goals of the legislation would be to encourage collaboration between pharmaceutical companies, researchers and specialist physicians. As Thomas sees it, a drug company could, with the support of a recommending physician, conduct an experimental trial without following a multi-phase, placebo-controlled regime.
Thomas believes the U.S. states that have right-to-try legislation haven’t gone far enough. Most U.S. states require what’s called Phase 1 approval. He wants the threshold to be lower in Canada.
“We believe that the current four-phase trial currently being used for most drugs isn’t suited to ALS patients. It sometimes takes four to five years. The average lifespan for an ALS patient is two to five years,” he says. “We think if there is less red tape, researchers would be more comfortable in investing in possible treatments. We think we could be a global leader.”
Thomas has never sought to access any drugs or treatments through Health Canada’s Special Access Programme. He’s interested in an experimental stem cell research being done in Canada right now, although he has been advised the treatment can’t be accessed through SAP.
If right-to-try existed in Canada, Thomas says, he would want to try stem cell treatment and other options, including a drug called NP001, which has shown some benefit for a subset of ALS patients. He has already applied for a stem cell treatment in Israel, but he doubts he will be accepted as there are only limited numbers of spots.
Dying patients have argued that they are dying anyway. But Bateman-House sees this as a slippery slope. “What’s next? People who have two years to live? Five years?” And what about the argument that the only life they are risking is their own?
“I wouldn’t disagree with that,” says Bateman-House. “However, they have to understand that what they do with their lives has implications for other people.”
In the U.S., Bateman-House sees right-to-try as “a solution in search of a problem.”
She has scoured media accounts and used her Twitter account in an attempt to solicit case stories from people who have been helped by right-to-try legislation. So far, she has found no verifiable cases.
“This law has helped no one,” she says. “The law promised wonderful-sounding things to people who are desperate, but it won’t actually give them anything.”
jlaucius@postmedia.com