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Seeking the Right to Try

November 18, 2014

Originally in US News

Christine McSherry’s son Jett is a typical college freshman trying to push for as much independence as he can. But Duchenne muscular dystrophy, a disease that tears away muscles, has taken his ability to walk. He relies daily on aides to help him with homework, eating and carrying classroom materials.

“He feels he has regressed to being a young child,” McSherry says. “It’s a horrible paradigm to be in.”

Jett is planning on majoring in history and is enjoying the social aspects of his college outside of Boston. Soon, he’ll begin a clinical trial that he’s been waiting two years to participate in, and his family hopes the experimental drug – eteplirsen, made by Sarepta Therapeutics – will stabilize his condition and give him a longer life.

There is no cure for Jett’s condition, which is fatal, but parents like McSherry believe there is hope if they can bypass government bureaucracy and gain access to medications still being tested by scientists. “If there was a drug to treat my son’s disease, then he would at least have the right to try it without long and expensive waits for it,” she says. 

Some states are making this process easier, passing laws that allow terminally ill patients access to drugs that have not been approved by the government. In Arizona, such a provision quietly won approval during the midterm elections.

Patients and their advocates are delighted they could have access to potentially lifesaving treatments, while some say the law is more symbolic than effective.This type of measure – passed also in Colorado, Louisiana, Michigan and Missouri – is known as a “right to try” statute. It allows dying patients to request access to treatment that has not been approved by the Food and Drug Administration, the federal agency that reviews testing by drug companies to decide whether medications are safe and effective. The laws allow doctors, hospitals and manufacturers to bypass the FDA and protect them from prosecution, even if they request medication from a drugmaker in another state.

Protecting someone from prosecution is only half the battle, critics say: Right-to-try laws undermine the FDA approval process, and patients could be putting themselves in harm’s way. The intention of drug review procedures is to make sure a drug can be distributed to a wider population, by assessing in part whether the benefits outweigh any known risks, according to the FDA.

Drugs and medical devices undergo three phases of clinical trials before they go to the FDA for review and, if approved, hit the market. The process can take more than a decade and costs pharmaceutical companies $1.2 billion per drug.

“The [right-to-try] laws are a simplistic way of going after much more complicated issues,” says R. Alta Charo, a professor of law and bioethics at the University of Wisconsin-Madison.

The laws allow patients to request drugs that have passed the first clinical trial stage – the one during which scientists determine whether a drug hurts people, not whether it can cure them. Just because a drug passes the first clinical trial does not mean it eventually will gain FDA approval.

Doctors covered by the laws can bypass the FDA and go directly to drugmakers to discuss what risks exist. They then decide with the patient whether they will request access. 

That lack of bureaucracy is needed when it’s literally a matter of life and death, advocates say. “When a patient is going to face certain death unless an action is taken, then all those actions are important in saving a person’s life,” says Victor Riches, vice president of external affairs for the right-leaning Goldwater Institute, which is leading the effort to pass right-to-try laws.

Other than clinical trials, which are difficult to qualify for, the FDA has provisions for patients who want to access experimental drugs under its compassionate use process. But advocates of right-to-try laws say the process is lengthy and time-consuming: The FDA quickly approves nearly every request it receives, but getting to that point can take doctors more than 100 hours of paperwork and the process can take months to navigate. Patients must demonstrate that they are terminally ill and have tried available alternatives.

Last year, the FDA gave nearly 1,000 people compassionate use approval, but that still doesn’t obligate drug companies to provide medications early.

“FDA’s oversight of [compassionate use] provides important protections for individual patients, while also ensuring the collection of the data needed to support FDA approval of safe and effective therapies,” a spokeswoman for the agency tells U.S. News in an email. The FDA has not taken a position on right-to-try bills.

For their part, industry members appear hesitant to look outside of federal requirements. “Legislation at the state level, however well-intentioned, is unlikely to add any meaningful new approaches that can optimize the federal expanded access process overseen by FDA,” says Sascha Haverfield, vice president for scientific and regulatory affairs at Pharmaceutical Research and Manufacturers of America, the industry’s trade group. “We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the FDA and clinical trial process, which is not in the best interest of patients and public health.”

“They may not want to be inundated by requests,” Charo says. Clinical trials are controlled environments with a homogeneous population, she explains, and people who are terminally ill have complicated medical conditions – which is why they often are not accepted to participate in such trials. “The chance of having an adverse event is pretty high,” she says. “When the FDA gets that information, they have to take it into account.”The laws do not force drugmakers to give treatment to patients, and many companies are reluctant to distribute their products if they have not gone through the approval process – either because they worry about the cost or liability, or because they have a limited supply. Any adverse reactions to the drugs used outside of clinical trials also would need to be reported to the FDA.

For Jett and his family, this became a stark reality: He nearly was booted out of the clinical trial because his condition has worsened during recent months. “If it had been another month or two, there is a good possibility that he may not have made it,” McSherry says. She says she wishes he had access to the drug when they first requested it nearly two years ago, but doesn’t worry about the outcome of the trial because they will at least have tried. She has seen other children become stronger with the experimental drug, and hopes it will do the same for Jett, buying him more time as drugs continue to hit the market.

The Goldwater Institute is working with advocates like Frank Burroughs, who’s supported right-to-try measures since 2001, when his daughter Abigail died from cancer at the age of 21. Shortly after her death, Burroughs founded the Abigail Alliance, a Virginia-based group dedicated to helping patients with life-threatening illnesses have better access to experimental drugs.

Abigail’s doctors had reviewed clinical trials and requested Erbitux, a drug that was still being tested. Her family pushed pharmaceutical companies and Congress to try to get access to the drug, and their mission gained media publicity. But the FDA did not approve the drug for Abigail’s type of cancer until more than four years after she died.

“Terminal patients shouldn’t have to ask the government for permission to try to save their own lives,” Darcy Olsen, president of the Goldwater Institute, said in a statement.

And for patients who are terminally ill, time – obviously – is running out. “It’s hard to argue that a terminally ill person is taking huge risks,” says Arthur Caplan, director of the Division of Medical Ethics in NYU Langone Medical Center’s Department of Population Health. “The drugs could make you die faster or make you miserable, but you’re already headed toward a bad outcome.”

Burroughs has similar feelings. “Patients going in understand there is a possibility of unforeseen side effects,” he says, adding that he does not think most medications doctors will request will be fatal because they will have carefully examined risks with patients and their families before deciding to participate. 

Dr. Ezekiel Emanuel, chair of the Department of Medical Ethics and Health Policy at the University of Pennsylvania, says he doesn’t think states should be passing right-to-try laws, though he is sympathetic to the motivations behind them.

“You’d like to turn over every rock to make sure [someone] gets a chance to live,” he says, “but from the standpoint of actually curing people it is not helpful. The most helpful thing we can do is to get the data … You don’t learn anything useful from the one-off cases.”

Emanuel says the meticulous clinical trial process is necessary to collect that data over time and determine whether drugs are working. “We’re asking the FDA to do two jobs at once,” he says. “We want them to protect us from drugs that might cause harm … but on the other hand, we want them to do it fast.”

But Burroughs called current requirements for FDA approval archaic, saying the process should be faster when someone’s life is at stake. FDA laws originated during the 1960s, when Americans became outraged over a sedative prescribed to pregnant women. The drug was intended to help alleviate morning sickness, but later was found to cause birth defects.

In 2005, a bill was introduced in Congress to increase terminally ill patients’ access to promising treatments undergoing clinical trials, but the measure never got a hearing.

The Goldwater Institute agrees that the process is dated. Next year, it will focus its efforts on passing right-to-try laws in Florida and Texas. Eventually, it hopes to extend the right to every state, Riches says, adding that the issue has consistently received bipartisan support. 

But questions remain about how much help such laws can be to patients. “They promise more than they can deliver,” Caplan says. “These laws are easy to vote for but accomplish almost nothing. They’re feel-good laws.” He explains that even if someone were to get access to a drug, he or she likely would need to travel to get it, and would need to be able to afford it. The decision of what to charge for the drug before it has been FDA-approved would be up to the company manufacturing it, and it is not clear whether it would be covered by health insurance.
Emanuel also worries that the laws could create a greater gap between the rich and poor, saying that those who would be able to obtain medications are likely to be well-connected and well-resourced.

“They are widening disparities rather than helping people get access to medication that works,” he says. He cites experimental treatments used on Americans to fight Ebola, an illness that has been highly publicized and placed U.S. health agencies under tremendous political pressure. “Everyone has a right to live,” he says. “I don’t like it for that reason. It’s implicitly discriminatory.” 

Riches thinks companies might provide treatment without cost, and says the option of traveling within the U.S. is still preferable to traveling outside the country for treatment. He admits he is not yet aware of patients who have successfully used an experimental drug through right-to-try laws in states where they have been passed, though he says they are still new and many patients are not yet aware of the option.

Patient barriers drove the organization to push for the laws in the first place, he points out, and patients have expressed interest in using the new provisions.

“People in this situation investigate every possible way to save their lives and the lives of their loved ones,” he says



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