- Associated Press - Wednesday, March 4, 2015

INDIANAPOLIS (AP) - A 5-year-old honorary firefighter who is battling a terminal form of muscular dystrophy pleaded with Indiana legislators Wednesday to expand patient access to experimental drugs, including one his family hopes could give him decades longer to live.

In December, Jordan McLinn landed his dream job when the Indianapolis Fire Department made him its youngest-ever recruit. Sporting his black-and-yellow firefighter costume, he addressed members of the Senate health committee and had to lift himself up off his seat to reach the microphone.

“Please say yes,” Jordan said of the bill, which would give him and other terminally ill patients the chance to try medicines that have cleared basic safety tests, so long as the patient’s doctor and the drug manufacturer approve.

The “right to try” legislation passed through the state House last month. Indiana is one of nearly two dozen states that are considering the legislation. Michigan, Colorado, Missouri and Louisiana have already passed similar measures, and Arizona put a proposal in place through a voter referendum.

Jordan is battling a terminal form of muscular dystrophy called Duchenne, and he’s not expected to live past the age of 20. A clinical trial drug has the potential to give him another 50 years, but it hasn’t received approval from the Federal Drug Administration and is therefore off limits.

“We have to make his dreams come true at a really young age,” said Jordan’s mother, Laura McLinn, because by the time he’s old enough to really become a fire man “he might be living in heaven.”

Under current law, patients have some options to obtain investigational drugs. They can enroll in a clinical trial or ask their doctors to file an application with the FDA for a single investigational or compassionate use.

Kurt Altman, the national policy adviser and general counsel for the Goldwater Institute that supports the measure, said only about 3 percent of those who are eligible for a clinical trial are accepted, and the compassionate use application process is lengthy and has no guarantee of success.

Patients also can wait for drugs to enter the market after they receive federal approval, but the entire process includes three phases and can take about 10 to 15 years to complete, Atman said.

“That’s time terminally-ill patients diagnosed late in their diseases just don’t have,” he said. “They don’t want to ask permission anymore to try and save their lives and the current system that’s what they have to do.”

If passed, patients could get access to drugs that have passed at least Phase-1 of the FDA’s approval process and are still in the experimental stage.

No one spoke in opposition at the committee hearing Wednesday, but lawmakers expressed some concerns about how the state would effectively track and monitor the drug usage. The bill would also need to ensure that all medical professionals who treat these patients would be immune from liability.

Committee Chairwoman Patricia Miller, R-Indianapolis, said she would not hold a vote on the measure until next week to allow changes to be made.

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