Bob Bardone and his wife, Peggy, dreamed of retiring under the warm skies of Florida and taking long walks on the beach. Then last summer, a month before they were set to move, Bob Bardone, 67, found out he has amyotrophic lateral sclerosis, or Lou Gehrig’s disease. He was given two to three years to live.
The couple went ahead with their long-held plans, but Peggy Bardone struggled to care for her husband. They decided to move back to St. Louis in March to be closer to family.
“My dad’s body is slowly giving out on him, and we have to watch,” said one of the couple’s four children, Cadie Connors, 44, of Keller, Texas. “We can’t even give him any medicine until the end of his life, which does very little to help. It’s absolutely brutal.”
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The move to St. Louis happened to bring them hope. They learned Missouri had become the third state to enact “right to try" legislation, which went into effect in August and allows terminally ill patients to try promising drugs yet to be approved by the FDA.
The family had been closely following news about a drug, GM604. Its maker reported that it significantly slowed the progression of ALS in 12 patients over 12 weeks with no dangerous side effects, though some scientists question the findings. The drugmaker, Genervon Biopharmaceuticals, is seeking fast-track approval from the FDA to sell the drug before spending years studying it in larger numbers.
“My dad had hope for the first time since diagnosed with ALS when he found out Missouri was a right-to-try state,” Connors said. “He thought that because he was moving back to Missouri, that he could try this drug.”
Bardone’s neurologist tried to get more information about the drug, and the family made emotional requests to the company. Genervon, however, refused to provide it.
After months of trying, the family is disappointed and frustrated, and Bob Bardone’s health is worsening.
“Can you imagine the hope we felt?” Connors said. “This entire process puts the most emotional time in our lives on even a more emotional roller coaster.”
FALSE HOPE
Right-to-try laws are sweeping the country. Seventeen states have enacted legislation, and more than 20 others are considering similar bills. But the efforts don’t appear to be getting drugs into the hands of patients.
The laws mostly follow a model designed by the Goldwater Institute, a free-market advocacy group that argues right-to-try laws improve terminal patients’ access to drugs by “dramatically reducing paperwork, wait times and bureaucracy.”
The legislation allows access to drugs that have passed the first phase of testing in humans, looking for dangers in a small number of people. To qualify, patients must be terminal, have exhausted other available treatments, get a doctor’s approval and sign a consent form acknowledging risks.
But right-to-try laws may be meaningless because companies can refuse to provide their experimental drugs. They may only create a false sense of hope among desperate families.
“It gives people a false impression that somehow because it’s a state law, it mandates this, but really it just gets everyone up in arms,” said Dr. John DiPersio, the deputy director of the Barnes-Jewish Hospital Siteman Cancer Center. “All it’s doing is causing commotion and confusion.”
Connors said her family doesn’t understand why a law would say a patient has a right to try a drug but then a manufacturer can turn around and refuse to give it to an eligible patient.
“It’s very hard knowing that there is a drug out there that can save my dad’s life. The law says he can have it … But a drug company says he can’t?” Connor said. “Who’s the law here? The drug company or the state of Missouri?”
The bill’s sponsor, Rep. Jim Neely, a Republican legislator and physician from Cameron in northwestern Missouri, said at the time he wanted to give patients as many options as possible.
A press release from the Goldwater Institute revealed that Neely’s daughter Kristina Brogan was pregnant when she was diagnosed with colon cancer in spring 2013, which made her ineligible to enroll in any studies. After safely delivering the baby early, liver failure from her rapidly progressing disease left her with fewer options. The mother of five died last month at the age of 41. Neely did not respond to a request for comment.
“I think that right-to-try laws are the wrong solution to a terrible problem,” said Nancy Goodman, founder of Kids v Cancer, a nonprofit group that promotes pediatric cancer research. “The problem is that most of the time, when patients can’t get access to drugs they so desperately need, it’s because the company decides not to share its drugs with them. It’s not because the FDA refuses to approve it.”
Genervon said in an email that the company had been overwhelmed by requests from ALS patients for its drug. It chose to focus its efforts on getting fast-track approval, which “would give immediate access to all ALS patients, require doctors to prescribe and monitor patients’ progress, allow us to continue gathering data ... and the cost would be supported by health insurance.”
DiPersio said companies have valid reasons for not making drugs available. Some are small ventures with no resources to dedicate to fulfilling individual requests. Supplies may be limited with no system to decide who gets the drug and who doesn’t. Others don’t want to reveal their drug is not yet safe and scare off investors. Some have spent millions over two decades to develop a drug, which they can’t risk providing outside of a controlled study. One bad outcome could cause a delay in what could be a good treatment for many.
The Goldwater Institute argues the FDA should assure companies that outcomes of patients who receive drugs for compassionate use will not negatively affect a drug’s approval.
But DiPersio points out, “It’s very expensive to have a compassionate-use program and to give out drugs willy-nilly. ... You have to track the data. It’s risky and complicated and expensive, and a lot of companies don’t have the stomach for it.”
FDA NOT THE PROBLEM
Critics of right to try also argue the FDA already has a process for approving experimental drugs for individual compassionate use or “expanded access.” Doctors work with willing drugmakers to submit expanded access applications to the federal agency, and 99 percent are approved. Last fiscal year, the FDA approved nearly 1,900 applications, some involving groups of patients.
Most applications are handled within four days. The application is time-consuming. However, in February, the FDA drafted a simplified application that when finalized, physicians will be able to complete in 45 minutes.
The FDA has not taken on a position on right-to-try laws, but an email from press officer Sandy Walsh says the agency is not a barrier.
“In recent years, the agency has been urging drug companies to consider Expanded Access programs, but we recognize that companies might have their own reasons to turn down requests for their investigational drugs,” Walsh wrote. “It is ultimately up to the companies to decide whether to make investigational drugs available for Expanded Access.”
Area doctors say companies with promising drugs are usually willing to provide them when appropriate.
“There may be an occasional instance when companies are not as flexible as they could be, but that is not the rule,” said DiPersio, who submits compassionate use requests almost weekly. “Most of the time, these guys, if they can, will do everything they can to help.”
Dr. Rob Hanson, an oncologist at Mercy Children’s Hospital St. Louis, said in reality, miracle treatments are rare.
“The thought that there is a drug out there that a child could be getting but regulations are standing in the way — you have to be realistic on how likely that is,” Hanson said. “If it really is a promising drug, we will know very fast, but unfortunately the vast majority don’t fall into that category.”
Doctors say the state laws have sparked important debate about how to get drugs to the public more quickly, but they argue that legislative efforts should focus on increasing funding for research and helping patients pay for drugs that are available.
Goodman takes the debate a bit further. Last year, she helped lead a social media campaign that pressured a company to make a drug tested in adults available for a 7-year-old suffering from a deadly infection after a bone marrow transplant. The boy survived because of the drug. She does not think companies should be deciding who qualifies to participate in a study or who can get a drug before it’s approved.
“My question is, ‘Do we as society want to give companies the opportunity to withhold drugs from people who are dying?’” she said. “Maybe that’s a decision we should consider.”
In the meantime, families with little time left must wade through the confusion of expanded access, compassionate use, accelerated approval, clinical trials and right to try.
“It’s important for people to know what they may be facing and to not have false hope,” Connors said. “I wouldn’t want anyone to go through what we have gone through.”
