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Families lobby lawmakers for 'right to try' investigational drugs, devices

A bill that would allow terminally ill patients who have exhausted traditional treatments to try drugs and devices that have not yet received full FDA approval is before the Utah Senate after a Senate committee gave it unanimous approval.
A bill that would allow terminally ill patients who have exhausted traditional treatments to try drugs and devices that have not yet received full FDA approval is before the Utah Senate after a Senate committee gave it unanimous approval.
Alexander Raths, iStock

SALT LAKE CITY — Monday was one of those nights when nothing soothed the unrelenting pain Talan Summers was experiencing.

"My wife was up all night massaging his arms and his legs for the plain fact we don't have options, either," said Stan Summers, whose 24-year-old son has a rare autoimmune disease.

Stan Summers, a Box Elder County commissioner, addressed a legislative committee Tuesday, urging support of the latest version of HB94, which would create a mechanism for patients with terminal illnesses who have exhausted traditional treatments to try investigational drugs and devices that do not have full federal regulatory approval.

The Senate Health and Human Services Committee voted unanimously to send the bill to the Senate for its consideration. The bill, sponsored by Rep. Gage Froerer, R-Huntsville, passed the House on a 72-1 vote.

Not only might the legislation help his son, who has immunoglobulin G4-related sclerosing disease, but Stan Summers told committee members it would have also helped a family friend who had to travel to Mexico for colorectal cancer treatments not approved in the United States.

"This was seven years ago. She goes to Mexico once a year now to make sure everything is OK," he said.

Stan Summer thanked Froerer for his work on legislation he believes will help people who have run out of options.

"Heaven forbid you ever get to the point that you are, where we are, that you're looking for the next 'thing,' the next 'breakthrough,' the next drug that can help you have quality of life, where you can take your wife out on a date or your son can run across the yard or go to school," he said.

But others, including Matt Fairbanks, who said he started his law enforcement career in Dallas and has also worked in Utah, questioned whether the bill would open the door to marijuana use.

"The Food and Drug Administration has not approved any product containing or derived from botanical marijuana for any indication," its website states.

Froerer said Congress would have to act to permit the FDA to conduct clinical trials with marijuana plants or products derived from the plants.

"There are those who think you're trying to sneak marijuana in under a different path," said Sen. Allen Christensen, R-North Ogden.

"That is not the case," Froerer responded.

Cristina Might, whose 7-year-old son, Bertrand, has the first documented case of N-glycanase deficiency, a genetic metabolic disorder that causes developmental delays, abnormal tear production and liver disease, also testified on behalf of the bill.

Might said many of the FDA's regulations were enacted before the development and use of technology that makes it possible to diagnose and develop drugs to treat certain diseases.

Bertrand Might and 24 other children who have been diagnosed with the same genetic disorder would likely benefit from a drug that is now in Stage 3 FDA approval.

"There is miracle drug we know will work for our kids," the boy's mother said. "Two years ago, if he'd had access to this drug, he might be developing normally. He might not be having seizures. We wouldn't have to be worrying about him passing away at night."

Cristina Might said she hopes the FDA will change some of its rules in the near future to give patients greater access to treatments that would help them.

"Until then, patients and their families should have the right to try these potentially lifesaving drugs," she said.

Froerer said the bill contains a number of protections. Drugs must have passed Phase 1 FDA approval. Patients or their family members must provide informed consent to their treating physician and pharmaceutical companies acknowledging potential risks of drugs. The manufacturer must consent to dispense the drug.

The bill also provides some protections to insurance companies.

"It gives hope to the hopeless," said Jonathan Johnson, Ovestock.com chairman and chairman of the political action committee Promote Liberty.

Sen. Brian Shiozawa, R-Cottonwood Heights, who is a physician, applauded Froerer and Johnson who earlier this month launched the Right to Try Foundation, which would provide financial assistance to terminally ill patients who want to try experimental drugs but cannot afford them.

"This a great thing you're doing for patients. It gives them hope," Shiozawa said.

Email: marjorie@deseretnews.com