The Right to Try Act May Expand Use of Experimental Treatments

Barriers are coming down and expanding access to experimental treatments for patients with terminal illnesses is on the horizon. Last week, the Senate approved funding for the US Food and Drug Administration (FDA) and the Right to Try Act of 2017. This bill requires the federal government to allow unrestricted manufacturing, distribution, prescribing, and dispensing of experimental drugs, biologic products, and medical devices that are intended to treat a patient who has been diagnosed with a terminal illness. It also calls for allowing unrestricted possession and use of such treatments by patients if they are certified by a physician as having exhausted all other treatment options.

The bill also states that a manufacturer, distributor, prescriber, dispenser, possessor, or user of such a treatment has no liability regarding the treatment. However, the treatment must have completed a phase I clinical trial, among other things. “The pace of the scientific advancements is progressing at a very rapid rate and now there are new drugs coming out that are fundamentally different and so there needs to be a speeding up of the process,” said Robert Hayashi, MD, director of the division of pediatric hematology/oncology at St. Louis Children’s Hospital and of the department of pediatrics at Washington University in St. Louis.

He noted that computer and genomic advancements have changed the paradigm for treating cancer and there is an urgent need to improve access to newly identified therapies. Scott Borinstein, MD, PhD, associate professor of pediatrics and the director of the adolescent and young adult oncology program at Vanderbilt University in Nashville, said that the system is in need of improvement. “We are in a great time in medical history in which we have all the great drugs coming out and we are still figuring out how to use them,” he said in an interview with OncoTherapy Network.

Borinstein said that when it comes to the right to try issue, it needs to be examined very carefully, and the potential for causing more harm to someone at the end of life needs to be thoroughly investigated. “We need to make sure we do things in a safe manner,” he said.

The Senate also approved the RACE for Children Act as part of the FDA Reauthorization Act, which will allow children with cancer to have access to many more novel drugs and will help open clinical trials. It will also help pediatric oncologists to gain new information about which cancer drugs might help their patients.

Hayashi noted that major changes are warranted; while genomic advances are creating new treatments for cancer, one of the difficulties is getting these potential therapies to children. Because childhood cancers are so rare, pharmaceutical companies have very little financial incentive.

“Children are different. There are medications that are handled differently when you are a child than when you are an adult. There is a little reluctance to embark on activities where a severe reaction could occur in a child. That could bring a lot of unfavorable publicity,” Hayashi told OncoTherapy Network.

Currently, there are hundreds of drugs in the pipeline for adult cancers. However, that is not the case for childhood cancers. “We hope we will see many more clinical trials for children,” said Nancy Goodman, executive director of the advocacy group Kids v. Cancer, who worked on the RACE bill. “Pediatric oncologists are often left guessing on the dosing and scheduling and they have no idea about the toxicities.”

RACE passed on the House floor on July 12 and the Senate floor on August 3. The bill just needs the president’s signature. “The pediatric oncologists know that access to novel therapies is one of their greatest challenges,” said Goodman. “This will now be several steps forward in addressing this issue.”