Getting New Drugs to Terminally Ill - More to Story than Bashing FDA

Laura McLinn and her son Jordan during a “Right To Try” rally at the Capitol in D.C. on June 16. Jordan has Duchenne muscular dystrophy. The rally advocated easier access to experimental drugs that have not been fully approved by the Food and Drug Administration. (Family photo)

Laura McLinn and her son Jordan during a “Right To Try” rally at the Capitol in D.C. on June 16. Jordan has Duchenne muscular dystrophy. The rally advocated easier access to experimental drugs that have not been fully approved by the Food and Drug Administration. (Family photo)

By Joe Davidson

Their stories were heartbreaking and inspiring — but ultimately fatal.

The terminally ill rallied on the West Lawn of the Capitol on Thursday afternoon, along with their families, supporters and members of Congress, demanding the “Right to Try” drugs that have not yet been approved by the Food and Drug Administration (FDA).

They pushed legislation in the House and Senate that says “the Federal Government shall not take any action to prohibit or restrict” the use of “an experimental drug, biological product, or device” that has been authorized by state law and has completed the first phase of a multi-level approval process. Thirty states have approved right to try legislation.

On a gut level, that makes such good sense. Why not allow the terminally ill the ability to do what they can to extend their lives? How could anyone refuse Jordan McLinn of Indianapolis, an impossibly cute 7-year-old, in an honorary firefighter’s hat? “I love my mommy,” he said just before his mother, Laura, told the crowd there is an unapproved drug for his condition, “but Jordan can’t access it.” His radiant personality masks the Duchenne muscular dystrophy that will cripple then take his life.

“Today, our civil rights are being denied,” said Frank Mongiello of Yardley, Pa., his voice determined, yet distorted by ALS, amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease. There is no cure. “We’re being denied a fighting chance because of all the bureaucracy.”

“I will fight for my right to save my life …” he added to cheers, his spirit strong.  “I don’t need the FDA to protect me from death.”

His story is compelling, but it is not the whole story.

The problem is not the FDA, according to Arthur Caplan, director of medical ethics at New York University School of Medicine.

Speakers at the rally, organized by the Have a Heart Foundation and cosponsored by theGoldwater Institute, repeatedly blamed the FDA. “But nothing could be further from the truth,” Caplan said in an interview. “In fact, it is companies that control access. They have the drugs, or the devices or whatever. … It’s up to a company to decide if they are going to give anything away. Unless Congress is ready to start making companies give away their products, which I think is somewhere between unlikely and never going to happen, talking about the FDA doesn’t do anything to get people access.”

Caplan doesn’t favor the legislation because “it is simply feel-good legislation that will disappoint Americans who think they’re getting a new right when they are not. That’s not something I’d want to do to the terminally ill.”

Then there’s the question of who would pay for the drugs? Ultimately it will be between the patient, the doctor and the drug manufacturer to come to an agreement, according to the office of Sen. Ron Johnson (R-Wis.), sponsor of the Senate bill.

Earlier this month, FDA Commissioner Robert Califf said the agency streamlined expanded access for “compassionate use” of so-called investigational drugs. “One way we hope to make things simpler for physicians is to reduce the amount of time they spend filling out a request for access to an investigational drug,” he said.

“It should take about 45 minutes for a physician to complete the new form.”

Permission to use investigational drugs was granted in more than 99 percent of expanded access requests received during fiscal years 2010-2015, according to FDA. “The agency often allows these submissions to proceed quickly,” said spokeswoman Sandy Walsh, “often in a matter of days and in the case of emergencies, typically provides authorization over the phone in a matter of hours.”

Johnson has a starkly different, even sinister view of the agency.

He spoke warmly about giving people with terminal illnesses “the opportunity to decide for themselves, to avail themselves of experimental treatments or experimental drugs.”

Then he launched a cold attack on the Affordable Care Act, a.k.a. Obamacare, as “the single greatest assault on our freedoms in my lifetime.” He said the FDA “has grown out of control. … It is reducing our freedoms.” Suddenly, his passionate remarks on behalf of the ill had mutated into a political hit. The FDA, Johnson said, is a once “well-intentioned agency that has now turned and is no longer looking out for the best interests of America and its people.”

None of the other speakers, not even Sen. Ted Cruz (R-Tex.), chose to go there during an event that otherwise was more compassionate than partisan. Remarks like Johnson’s might be one reason his bill has no Democratic sponsors.