The Right to TryJuly 2016
By Astrid Derfler Kessler
How the Federal Government Prevents Americans from Getting the Lifesaving Treatments They Need
An Interview with Darcy Olsen
In 2015, Darcy Olsen, the president and CEO of the Goldwater Institute, a national policy and litigation organization that has changed more than 200 laws nationwide, released a book titled The Right to Try: How the Federal Government Prevents Americans from Getting the Lifesaving Treatments They Need. Throughout the book, Olsen states that the FDA’s approval process for new lifesaving medications is needlessly costing tens of thousands of American lives each year. She reveals disturbing statistics that underscore the depth of this worsening problem.
“The FDA,” says Olsen, “takes as long as 15 years to bring a new medicine to market and Americans are waiting 60% longer for the FDA to approve lifesaving devices than they did just seven years ago. Instead of speeding medical innovation, the FDA is slowing it down—demanding more data, more tests, and more procedures on more subjects before it will approve a drug.”
The book begins with the story of Ted Harada, a father of three who was diagnosed with amyotrophic lateral sclerosis (ALS) at age 38. Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is 100% fatal, with death occurring within two to five years.
But Harada was fortunate. In 2011, he underwent experimental stem cell surgery at Emory University in Atlanta, Georgia, that reversed his symptoms completely. A second surgery was done in 2012—and two months later, Harada had recovered enough to participate in an ALS fundraising walk.
Then the FDA intervened and decided Harada could not have any further surgeries. He is also unable to undergo surgery outside of the study because the treatment has not yet been approved by the FDA. This lifesaving surgery is also unavailable to the nearly 24,000 people who have died since the clinical trials began. Every 90 minutes, someone with ALS dies.
In an interview with Darcy Olsen, Harada posed the question: “If I have twice shown the surgery is safe and effective, why should I have to ask the FDA for permission to do it? If I have a doctor and a drug company willing to provide this to me, and I obviously have informed consent, why should I ever have to be in a position to go hat in hand to the FDA asking for their permission for something that has been shown to work for me twice? Why should my life rest in their hands?”
LE: You became involved in advocating for Right to Try after the then-CEO of Cancer Treatment Center of America, Steve Bonner, came to the Goldwater Institute for help in getting access to innovative treatments that could save lives.
DO: As I listened to Bonner and his colleagues, I immediately thought of my uncle Kenny. He died when I was about four from Hodgkin’s lymphoma—a form of cancer for which there are now multiple treatments with high cure rates. He was my father’s only brother, and I remember my dad saying that Uncle Kenny died just a few months before a new treatment was approved. At that moment it hit me: If Kenny had been allowed to try that treatment earlier, my father might still have his brother and my cousins might still have their father. The idea that the federal government was standing in the way of people fighting for their lives was infuriating. It’s one thing for people to die because science has not come up with a treatment for their illness yet. It’s quite another for someone to die when a promising treatment exists, but a patient can’t get access to it because of government obstacles. If Washington would not fix the problem, then it was our job to step in.
LE: You compare your plan of action, taking the fight to the state level as opposed to the federal level, with medical marijuana laws and the right of assisted suicide. Can you explain your thought process?
DO: I am a firm believer in federalism, the constitutional authority and responsibility of states to check and balance Washington’s overreach. If states have the authority to give their citizens access to marijuana and drugs to end their lives, certainly they have the authority to allow patients access to investigational medicines to save their lives. If you have the Right to Die, you have the Right to Try. And you don’t have to wait for Washington to secure it.
LE: What was involved in the initial draft law?
DO: It would protect a person’s fundamental right to try and save his own life by expanding access to promising new drugs that await the FDA if:
- The patient has a terminal diagnosis and has exhausted all treatment options
- The patient’s doctor has advised the use of an investigational medication
- The medication has successfully completed basic safety testing and is part of the FDA’s ongoing approval process
- The patient has provided “informed consent” acknowledging the potential risk of the drug and
- The company developing the medication is willing to make it available to the patient.
LE: The first Right to Try law passed in May 2014. In just a little over a year from that date, Right to Try has passed in 24 states and counting. When it comes to legislation, the speed with which it was voted into law is astounding.
DO: In the state legislatures, there have collectively been 3,045 votes in favor of Right to Try and just 26 against. To put that in perspective, if those were the votes in an election, Right to Try would have won by a margin of 99% to 1%. The only place in the world you’d find results more lopsided than that is North Korea!
LE: The FDA argues that they have what is called Expanded Access or a compassionate use program that allows terminal patients access to clinical trials. In fact, Richard Klein, the director of the FDA’s patient liaison program said, “The agency has a pathway. It seems to work quite well and I’m not sure what the state Right to Try bills really add to that.”
DO: Except they don’t work. Just ask the hundreds of thousands of Americans stymied each year in their efforts to get the drugs they need to save their lives. The FDA has buried its head in the sand. Its expanded access system is badly broken. Not only has the FDA created obstacles for patients and doctors, it created a system that discourages drug companies from offering innovative treatments on a compassionate basis.
LE: How many patients per year, on average, are accepted under the Expanded Access program?
DO: [There are] an average of 1,200 requests per year. The FDA says it has high approval rates [when it comes to compassionate use]. But what is striking is the infinitesimally small number of requests. About 1,615 people die of cancer every single day. Yet the FDA receives only 1,200 requests for compassionate use of investigational drugs in an entire year! According to research, 40% of all cancer patients attempt to join a clinical trial. But only 3% of adult cancer patients actually succeed in getting into a trial. And yet the FDA says it’s a system that “works quite well!”
Compassionate use is so rare that a leading cancer specialist at one of the nation’s leading cancer research hospitals has never seen it happen in the last 10 years.
LE: A complaint regarding the FDA’s compassionate use program is that the application is difficult to understand and extremely difficult to fill out. In February 2015, the FDA pledged to “simplify and accelerate” the application process.
DO: The agency promised a new application process that would take less than an hour to complete. Sadly, the agency hasn’t fulfilled that promise.
LE: One family’s experience sums up so poignantly why Right to Try is necessary to overcome government roadblocks and save lives. Let’s discuss the McNary family—Jenn and her two sons Austin and Max and the tragedy that surrounded them.
DO: Imagine the joy of watching your dying son experience a miraculous recovery thanks to an experimental medicine. Then imagine the horror of watching your other son slowly die from the exact same disease because the federal government prevented him from receiving the same lifesaving treatment as his brother. That’s the nightmare my friend Jenn has lived for the past three years.
One day Jenn began to notice that Austin was not keeping up with his friends [or] meeting developmental marks. The doctor told her that Austin had Duchenne muscular dystrophy, a disorder that leads to muscle degeneration and eventually death. When she discovered the disease was fatal, she had her 3-month-old son Max tested—and to her horror, found out he also had Duchenne.
LE: Austin didn’t qualify to join any of the clinical trials testing an experimental drug called eteplirsen, which is designed to partially repair one of the common genetic mutations that causes Duchenne. Until the Food and Drug Administration approves it, no one can receive eteplirsen outside of an approved clinical trial. What happened with both boys, who were in wheelchairs with limited mobility?
DO: Max was enrolled in a double-blind, placebo-controlled trial. About 16 weeks after his infusions began, Jenn started noticing changes in Max. He asked to play outside [and] started regaining his fine motor skills. After a year, he abandoned his wheelchair and hasn’t used it since. He was getting better.
LE: And Austin?
DO: Jenn’s joy was bittersweet. At the time she was watching one beloved son recover, she was watching her other beloved son continue to deteriorate. “Austin is very angry right now,” [Jenn said to me]. “He doesn’t understand why the grown-ups in his world can’t figure this out and make things happen faster for him. It is one thing if your child is dying from an untreatable disease, but to have your child dying from a treatable disease is devastating. I never thought it would be the government I’d be working against.”
LE: Three years after Sarepta [the pharmaceutical company that developed eteplirsen] first demonstrated that it’s safe and helping Duchenne kids, the FDA still hadn’t invited Sarepta to apply for approval of the drug. How many kids were hurt by these delays?
DO: There are about 15,000 to 20,000 boys in the US with Duchenne, and eteplirsen can help about 13% of them. That is about 1,950 to 2,600 boys who desperately need this drug. But Sarepta estimates that its broader portfolio of compounds based on same technology as eteplirsen can help about 80% of these kids. That’s 12,000 to 16,000 kids in the United States alone—and tens of thousands more across the globe. Jenn says there [were] only 12 children in the world receiving the medicine. The rest are wasting away and dying.
LE: After 39 months of fighting, Jenn finally succeeded in getting Austin into a clinical trial. Of course, the drug cannot reverse the debilitating declines Austin suffered while fighting for access. But how is he doing now?
DO: [According to Jenn], it’s not going to make him walk again, but it could stabilize his progression so he doesn’t get worse. For 170 weeks, Austin watched his brother get better while he got worse. He lost the ability to transfer himself from chair to bed, from chair to toilet, to wash his hair, dress himself, cut his food… The most frustrating part of the FDA is that they already have the tools they need to approve a drug like this. We weren’t asking them to go against the rules, against the law, or make an exception.
LE: What is happening in the approval process now?
DO: In April 2015, the CEO of Sarepta resigned—a casualty of the company’s struggles with the FDA. A few weeks later, the FDA invited Sarepta to submit a new drug application. If all goes well, and the drug is approved, it will be because an innovative CEO and a group of moms joined forces to fight for faster approval.
LE: Why do you think the Right to Try has taken the country by storm?
DO: It’s simple. Almost everywhere it has passed, courageous Americans fighting terminal illnesses have stepped forward to fight for it. These patients and their families have testified before state legislatures, lobbied their elected leaders, and shared their stories with the media—asking their fellow citizens to stand with them in the fight to save their lives. Their stories captured the imaginations of legislators of both parties and inspired millions to rally around the terminally ill. These Americans moved hearts—and votes—in support of the principal that every American has a fundamental right to try to save his or her own life.
LE: Thank you for sharing your experience and the work you’re doing.
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Darcy Olsen is the president of the Goldwater Institute, a national research and legal organization that has changed over 200 laws throughout the US. An authority on education reform, economic policy, and government reform, Olsen is a regular guest on national media programs. Her opinions have been widely published in outlets such as the Wall Street Journal, USA Today, and National Review. Olsen has received numerous awards and recognitions including a 2014 Bradley Prize, which recognizes individuals of extraordinary talent and dedication who seek to preserve and defend the institutions of free government and private enterprise, and the national Roe Award for achievement in public policy. To contact the Goldwater Institute, please visit www.goldwaterinstitute.org or call 1-602-462-5000.
To order a copy of The Right to Try, call 1-800-544-4440.