Right to die but no ‘right to try’
TERMINALLY ILL WANT ACCESS TO UNPROVEN THERAPIES
About a month ago, a group of people with terminal illnesses wrote to MPs, asking for legislation that would give the dying 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 people facing a foreseeable death have the legal “right to die” in Canada, they don’t have the “right to try” something that might prolong their lives, he says.
“We have the law that allows us to kill ourselves. We just don’t have the law to do something else.”
After meeting Perreault recently, Health Minister Jane Philpott said officials were studying the issue and would determine what actions were required.
The idea behind “rightto- try” seems a no- brainer. Surely only the hardest of hearts would deny a dying person one last shot at a longer or more comfortable life? More than 30 U.S. states have right- to- try laws. Now, the movement appears to be picking up steam in Canada.
Owen Thomas, 31, was one of the signatories of the letter. The Bridgewater, N. S., resident noticed stiffness in his legs in the summer of 2014 and was diagnosed with ALS in January.
The former avid drummer and weightlifter now uses a walker, needs help dressing and can’t cook for himself.
“Time is of the essence for terminally ill patients,” he said. “Right to try should be considered a human right.”
But opponents argue such laws are a mistake. Alison Bateman- House, a bioethicist at New York University, points out pharmaceutical companies are not obliged to make unapproved treatments available, even if right- to- try legislation is passed.
“You can’t make a private business provide a product in development, even in the case of a national emergency,” she said.
She also doesn’t believe there is a need to liberalize regimes. Pharmaceutical companies are often quite willing to make drugs in development available to patients on compassionate grounds, as long as the U. S. Food and Drug Administration or Health Canada requirements have been fulfilled, she says. Approval is usually given within days, even hours.
When a drug is at the end of 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,” Bateman-House said.
Companies may also make drugs available if they have proved to be successful against different conditions — for example, an antiviral that can treat several viruses. It gives the company anecdotal evidence the drug will work with 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?”
It ’s not unfathomable right-to-try will come to Canada, said 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 welcome. What he finds most concerning is that right- to- try campaigns are based on the belief regulators are 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 said.
“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 adds there is not any scientific evidence to show 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.”
Nonetheless, many desperate people will leave the country for clinics offering unproven therapies. Caulfield points to the now- discredited “liberation therapy” for multiple sclerosis. Driven by the power of testimonials, dozens of Canadians were treated abroad.
“Unfortunately, pressure from interest groups can trump science,” said Caulfield. “Patients aren’t the only ones who will lose out. There’s also harm to the legitimacy of the science.”
When he says publicly he is skeptical about treatments such as liberation therapy, he often gets jeered and shouted down, he added.
“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. Clinical trials are expensive and time- consuming, but they are necessary to maintain high standards for safety and efficacy.
One of the difficult things about right-to-try is that it is based on skepticism about government regulation and the sense the government is a barrier to people’s freedoms and rights.
“The underlying assumption is that ‘ There is a promising treatment but the government won’t let me have it,’ ” she said. “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?
That’s not always true. Some people have suffered devastating consequences as a result of experimental treatments. Last month, Juno Therapeutics Inc. of Seattle said two more patients had died after suffering brain swelling during a trial of an experimental genetically engineered leukemia drug. This extracted T- cells from a patient’s immune system and altered their DNA in the hopes these would better find and kill cancer cells.
So far, five people have died during trials of the drug, JCAR015. Early studies showed 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 early results because of a misunderstanding about what it means when a treatment is “showing promise,” said von Tigerstrom. About 90 per cent of treatments that show early promise fail to be approved for market.
But Thomas points out right- to- try applies to very few people, mostly those with ALS and some cancers. He adds it’s patronizing to suggest 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 right to try would be to encourage collaboration between pharmaceutical companies, researchers and specialist physicians. Thomas says a drug company could, with the support of a recommending physician, conduct an experimental trial without following a multi-phase, placebo-controlled regime.
He says U.S. states with right-to-try legislation haven’t gone far enough as most require Phase 1 approval. He wants the threshold to be lower in Canada.
‘ We believe that the fourphase 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 said.
“We think if there is less red tape, researchers would be more comfortable in investing in possible treatments.”
TIME IS OF THE ESSENCE FOR TERMINALLY ILL PATIENTS.