Right-to-try laws challenge FDA's bottleneck on a promising treatment.

‘I met my wife in sixth grade,” says Keith Knapp, age 24. “She transferred to this small little school I was going to, and I liked her right away. I stopped playing basketball at recess and instead started swinging with her.” Life moved fast for Keith and his beloved Mikaela ever since — the two started dating in 2005, at age 15. In 2011, they got married in Hawaii. This year, he’s fighting to save Mikaela’s life.


Last year, Mikaela noticed a painful lump on her ribs. At first, her doctor diagnosed it as a fluid buildup and sent her home with painkillers and steroids, which seemed to help. But by fall, the bump was back and bigger. A CT scan revealed numerous tumors, which were at first diagnosed as lymphoma.

But after a pathology test, “the oncologist came in looking very grim and told us it was kidney cancer,” Keith says, recalling how he immediately began researching the illness on his computer. “It went to way over 90 percent chance of not living over a year. And over the course of the next couple days, we found out it had spread to her bones, her brain, and to her lungs. . . . It was a nasty type. It wasn’t even just generic kidney cancer. It was a rare subtype. There have only been a couple hundred cases in the medical literature. So every day, we’d get woken up at like six in the morning [at the hospital], and they’d just be dumping new bad news on us.”

In the six months since 25-year-old Mikaela’s diagnosis, she and her doctors have exhausted all treatment options — save one. An experimental cancer drug called anti-PD-L1 immunotherapy is currently in clinical trials and has shown great promise in treating cancer, even in advanced stages like Mikaela’s.


There is no guarantee that the experimental drug would save Mikaela’s life, but “just that little chance of a miracle happening — it’s so much more to latch on to than what we’ve had up to this point,” Keith says. “So if there’s some little, tiny chance that it may cure her cancer — an infinitesimally small number that’s larger than zero — that’s still greater than zero.”


The problem is, red tape has prevented Mikaela from even trying this treatment.

Clinical trials accept only “typical” patients — Mikaela’s rare form of kidney cancer has ruled her out, but for others in her situation, complications as common as diabetes or high blood pressure could also be reason for ineligibility.

And the Food and Drug Administration’s approval process for “compassionate use” of an experimental drug outside of clinical trials is extremely arduous.


First, a pharmaceutical company has to be willing to provide the experimental drug — a high-risk proposition, given that an atypical result in an atypical patient can prompt the FDA to delay approval or require significant and expensive additional testing. It can take more than a decade and $1 billion for pharmaceutical companies to bring a new drug to the market, so few are willing to risk further delays.

And approval from a pharmaceutical company is just the first step. Next, patients and doctors must fill out an exhaustive paperwork disclosure to submit to the FDA, which is estimated to take at least 100 hours to complete.


The FDA then begins a review, which is supposed to take no longer than a month — but if the agency’s reviewers have any additional questions or need more information, that 30-day clock is reset.

Finally, the hospital’s institutional review board has to give approval — and if a patient is being treated at a small hospital that lacks such an administrative panel, the case has to be reviewed by a bigger facility, often one unfamiliar with the patient.


But there is evidence that immunotherapy treatment is effective. A Roche study of 85 locally advanced or metastatic lung-cancer patients saw very high disease-control rates.

In Mikaela’s case, the drug companies have not been willing to take the FDA gamble to supply the treatment. So Keith has launched a massive campaign, including a Change.org petition that has garnered more than 362,000 signatures, to change their minds.

“It’s not something I want to do,” Keith says. “I don’t know how much more time Mikaela has, and it kind of sickens me that I have to leave her side to push the story on this. But she’s assured me that she wants me to keep fighting until the very end. I’m not getting to bed until like 3 to 5 a.m. nowadays. You add all this responsibility to taking care of her — this period in my life, I feel like I have the least free time to lead a campaign like this, but it’s the time I most need to. It’s the paradoxical thing.”

Ideally, Congress would pass reforms that allow terminally ill patients easier access to experimental treatments. Though bills that would streamline the process have been introduced every year since 2008, none have even received a committee hearing.

But several state legislatures have recently proposed “Right to Try” legislation, which would simplify the process for compassionate use, helping patients like Mikaela.

In the past week, the state houses in Louisiana and Colorado both unanimously passed Right to Try bills. In Missouri, the bill has won unanimous support in both the House and a Senate subcommittee. Another bill won approval in the Arizona house recently, and the state senate is expected to vote on the legislation in the next few weeks.

“You shouldn’t be putting a bureaucrat in the middle of medical decisions, especially when you’re talking about terminal patients,” says Christiana Corieri, a health-care policy analyst for the Goldwater Institute, which helped draft the legislation. “There’s no right that could be more fundamental than the right to try and save your own life.”

— Jillian Kay Melchior writes for National Review as a Thomas L. Rhodes Fellow for the Franklin Center. She is also a Senior Fellow at the Independent Women’s Forum.