Experimental treatments at the end of life

WASHINGTON—At a rally on Capitol Hill on Thursday, advocates revived the push for federal right-to-try legislation allowing terminally ill patients to try experimental treatment methods after exhausting other options.

“This isn’t a Republican or Democrat issue, it is a human rights issue,” said Sen. Ron Johnson, R-Wis., the sponsor of the Senate’s right-to-try bill. “Across the nation, we are seeing a groundswell of support for the idea that patients deserve the right to try.”

Thirty states have already passed right-to-try legislation. Johnson’s bill, which mirrors House legislation from Rep. Matt Salmon, R-Ariz., seeks to ensure the Food and Drug Administration cannot interfere with those state laws. Right-to-try bills afford patients with incurable prognoses the option to try investigational treatments that have passed phase one of the FDA’s three-phase approval process. But progress on federal legislation has been slow, with some fearing it offers false hope and could lead to a lack of oversight and abuse.

The Goldwater Institute, a libertarian think tank based in Arizona, has promulgated right-to-try legislation for years, claiming the FDA hinders possible treatment for patients who are down to their last resort.

On its promotional website, Goldwater tells the story of Jordan McLinn, a 7-year-old from Indiana with dreams of becoming a firefighter. Jordan has Duchenne muscular dystrophy and a life expectancy of fewer than 20 years. The disease has a 100-percent fatality rate, and there are few treatment options to slow its progressive destruction of his body.

His mother, Laura, spoke at the rally Thursday, saying she doesn’t know if experimental treatments will cure her son, but if right-to-try legislation helps just one person it will all be worth it.

The FDA offers clinical trials to terminally ill patients for drugs still awaiting full approval, but according to Goldwater only 3 percent of the 1 million terminally ill patients who die each year get to take part in clinical trials.

Some medical researchers fear right-to-try laws could open the door to a host of new ethical questions and future problems.

“Most right-to-try laws do not set qualifications for either the healthcare provider making the attestation of terminal illness or the physician recommending experimental treatments,” opponents wrote in an op-ed in the Annals of Internal Medicine last fall. “As such, patients are not assured of an optimal (or adequate) assessment of their condition, prognosis, or chance of benefit from an experimental treatment. Worse, the door is left open for the unscrupulous or inept to prey on desperately ill patients and their families.”

Because the drugs are not yet fully approved, patients would be responsible for the full costs of investigational treatments under right-to-try legislation. That requirement could potentially drain the bank accounts of the terminally ill.

“I personally am in favor of offering people hope, however, small it may be,” said Catherine Glenn Foster, associate scholar at the Charlotte Lozier Institute and executive director for the Euthanasia Prevention Coalition USA. “When it comes to right to try, it works toward a culture of life, a culture of medical progress, a culture of cure, and promoting scientific inquiry.”

Along with the McLinns, several patients with Lou Gehrig’s disease joined the rally yesterday. The illness, also called amyotrophic lateral sclerosis (ALS), is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. It has no cure and only some treatments to slow its progression and manage discomfort.

Johnson, who introduced his right-to-try legislation last month, said there is bipartisan support to make it federal law. But so far, his legislation has made few waves in the Senate, garnering only Republican co-sponsors.

One of Johnson’s supporters, Sen. Ted Cruz, R-Texas, also spoke at the rally. The former presidential candidate called the FDA a large bureaucracy that needs to remove its barriers so more people can have access to lifesaving drugs.