'Right to Try' Bill in Senate for Terminally Ill Patients

Roxanne Nelson, BSN, RN

May 16, 2016

A new bill introduced in the Senate would give terminally ill patients in the United States greater access to experimental drugs.

The Trickett Wendler Right To Try Act of 2016, introduced by Sen Ron Johnson (R-Wisconsin), gives dying patients the right to investigational agents outside the confines of a clinical trial, and circumvents the need for approval from the US Food and Drug Administration (FDA).

It joins a companion bill that was introduced last year in the House. Both bills would prevent the FDA from taking action to prevent patient access. The bill was named after Trickett Wendler, a young Wisconsin woman who died last year from amyotrophic lateral sclerosis (ALS).

"Many of us have felt that sense of desperation — of urgency — when we learn that we or someone we love is fighting for their life," Sen Johnson said in a statement. "In 2014, I met with a brave Wisconsin woman, Trickett Wendler, who was fighting ALS. Trickett passed away last year, but her spirit and her fight are among the reasons I am passionate about this issue — because I know that today, and every day, millions of Americans are fighting similar life-and-death battles to save themselves and their loved ones."

Sen Johnson noted that in this "era of unprecedented medical innovation, we have to do more to ensure that patients facing terminal illnesses have access to potentially life-saving treatments."

Many patients are frustrated by the lengthy drug-approval process, especially those who are facing lethal illnesses. "These patients don't have the time to wait — often years and years — for the FDA to grant final approval. For Trickett, and for countless others facing terminal illnesses, now is the time to give more patients a chance to save their lives — to give them hope," he explained.

A growing number of states have already begun to implement their own right-to-try laws. Currently, 28 states have passed such laws, and legislation is pending in about 15 others.

For example, the House in Rhode Island unanimously approved right-to-try legislation. It now needs approval from the state Senate, and then can be signed into law by the governor.

But investigational drugs are not completely off limits to patients outside clinical trials. The FDA does allow patients who are not eligible for or who are unable to participate in a clinical trial to obtain a drug though expanded-access or compassionate-use programs.

Last year, after pressure to expedite compassionate-use approvals, the FDA streamlined the process by simplifying the application form.

Offers Hope But Doesn't Deliver

However, laws that seek to bypass the FDA are fraught with problems and do nothing to guarantee access to a drug, according to Art Caplan, PhD, Drs William F. and Virginia Connolly Mitty Professor and head of the division of medical ethics at the NYU Langone Medical Center in New York City.

First, it is highly unlikely that Sen Johnson's bill has any hope of passing in the near future. "This is an election year, and it's a very contentious election. The odds of this passing during an election year are zero," Dr Caplan told Medscape Medical News.

Second, state laws have been completely ineffective and have not allowed a single person access to an experimental drug. "These laws are targeting the FDA, and trying to go around the FDA, but the FDA doesn't control access to experimental drugs," he pointed out. "The manufacturer controls access, and the FDA only gets involved once the company grants access for compassionate use."

In other words, state laws and the proposed federal laws do not create any obligation or duty on the part of drug companies to offer experimental drugs.

Many smaller companies do not have the resources to run compassionate-use programs, and even with larger manufacturers, the drug might be in limited supply and needed for clinical trials, he explained. "There is nowhere in these laws that says the company must supply the drug if a patient asks for it."

Another issue is cost. "None of the state laws provide any assistance for paying for the drug," Dr Caplan said. "Patients may have to travel to get the drug, or it may have to be shipped, and there may be other expenses. The legislation looks good, but no one is willing to pay for it."

 
These laws offer hope but they don't really deliver.
 

In addition, dying patients who start using an experimental drug might no longer be eligible for hospice. "Once in hospice, you have to give up medical treatment," he said. "So if a dying patient starts using an experimental drug, it may mean leaving hospice. That is something that also needs to be addressed."

Overall, the federal law is not any better than the current state laws. "If the proposed bill were to pass, it would invoke a Constitutional crisis, as the FDA is mandated to regulate drugs and devices," he said. "It could take years going through the courts, unless Congress decided to completely revamp the FDA."

"These laws offer hope but they don't really deliver," Dr Caplan explained.

Better Framework Needed

Another expert agrees.

"These right-to-try laws are being enacted with the best of intentions but will likely do more harm than good," said Cary Gross, MD, professor of medicine and codirector of the Robert Wood Johnson Foundation Clinical Scholars Program at Yale University in New Haven, Connecticut.

"We all want to ensure that our loved ones have every possible chance to receive treatments that are potentially helpful when facing a deadly illness," he told Medscape Medical News. "But without a robust system of research, we will not be able to determine which drugs actually work and which ones don't."

He pointed out that if there are no safeguards in place to prevent exorbitant pricing, even when there is no evidence that a particular drug is helpful, patients and their families can be facing financial ruin.

Dr Gross acknowledged that there are many frustrations with the FDA drug-review process, but even so, "the solution is not to completely circumvent efforts to conduct research and critically review the safety and benefits of new drugs."

"And that is where we are headed with a right-to-try framework that is narrowly focused and short-sighted," he noted.

A better option would be if patients were allowed to access experimental treatments in the context of a framework that allows for the ethical review of a particular drug/indication that does not undermine efforts to enroll people into clinical research studies and ensures that outcomes of the experimental drugs can be used to inform subsequent studies, Dr Gross added.

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