Lawmakers fight to cut red tape for terminally ill patients seeking experimental drugs

Should people fighting for their lives have to battle red tape?

That’s the question lawmakers are considering in four states where the “Right to Try” act is moving forward – proposed legislation aimed at giving terminally ill people access to experimental, but potentially life-saving, drugs before they have FDA approval.

Americans like Keith Knapp, of California, have fought hard for this kind of legislation.

Keith Knapp married his high school sweetheart Mikaela. He and Mikaela thought they had their whole lives ahead of them — until last year, when she was diagnosed with a terminal form of kidney cancer. This began a fight that the two of them never imagined, as they tried to gain access to a promising, but not-yet-FDA-approved, drug that was doing well in clinical trials. The couple learned that without being in one of these trials, current law would not allow them access to the experimental treatment.

From her hospital bed, Mikaela said recently: “People die from not being able to access these drugs all the time. I don’t want to be one of them.”

But sadly, she was. Despite her husband’s passionate efforts to lobby members of Congress, pharmaceutical companies and the FDA — and a huge media campaign — Mikaela lost her battle just two weeks ago.

“The amount of effort you have to put into doing this is just far too much at this time in our life when you really just want to slow things down and enjoy being together,” Keith said.

Currently, it takes the FDA about 10 years to complete a clinical trial on a new drug — and while many try, only 3 percent will gain access to a trial during that time. Meanwhile, 500,000 Americans died last year from cancer alone, with thousands more dying of other illnesses.

In states where the “Right to Try” act has been introduced, bill sponsors often have personal reasons for pushing the issue. In Missouri, state Rep. Jim Neely is trying to save his dying daughter. In Colorado , the law is sponsored by a clinical pharmacologist fighting for her dying brother. In Arizona, the driver of the bill is a man who lost his wife. A similar bill also has been introduced in Louisiana.

For many people who get a terminal diagnosis, they’re willing to try anything — but once a clinical trial is closed, patients cannot get access to the potentially life-saving medication until it is approved by the government.

A family in Vermont is facing a similar situation. Jennifer McNary’s two young sons Max and Austin have the same disease. Max got into a clinical trial for an experimental drug called eteplirsen and is doing better. Austin did not get into the trial, and is getting worse.

“If Austin is never given the chance to get on eteplirsen, we know with 100 percent certainty that he will die,” she said.

Austin wants access to the drug that he’s seen make a big difference for his little brother. “My brother Max can run and walk, I can only sit in my wheelchair and watch him. He’s been on eteplirsen for two years — it’s safe and effective and I want access,” he said.

Despite the heart-wrenching stories, many doctors warn against this — saying these drugs could actually lessen quality of life and heighten the risk of side effects.

“You don’t know that it’s better than nothing,” UCLA endocrinologist Dr. Stanley Korenman said. “You don’t know that this won’t reduce your life expectancy rather than increase your life expectancy because you don’t know what the side effects are.”

Other doctors agree, saying that many of these drugs turn out to be useless and give false hope. They also say it can lead to “snake-oil salesmen” taking advantage of desperate, dying people.

But the Goldwater Institute’s Darcy Olsen believes it’s time for a change. “Every day thousands of Americans are dying when there are potentially life-saving drugs that they could be taking if we simply got this regulatory process up to date and modernized,” Olsen said.

And Keith Knapp agrees.

“This is one area in which policy just does not match what the American people would want, and I would love to see that change so people don’t have to go through this in the future,” he said.