The Hunts were preparing end-of-life care for their middle son, Zander, when his doctor at Cook Children’s offered a last resort: Treat the 9-year-old with a cancer drug approved only for adults.
The option, called compassionate use, becomes available when standard treatments like chemotherapy, radiation and surgery fail. It requires a slew of permissions; the family, hospital, drug company and U.S. Food and Drug Administration must provide consent. For children like Zander, whose life hangs in the balance, the approval process must be quick and efficient.
Working around the clock, sometimes nights and weekends, to make that happen is a three-woman cadre called the research pharmacy team.
“To have one tiny possibility, one more choice that could possibly help him — I think ‘gratitude’ is the word that comes to mind,” Emily Hunt, Zander’s mom, said. “I’m so grateful that they’re willing to do what they do.”
‘Who knew you could call a drug company and ask for free drug?’
In the research pharmacy on the fifth floor of Cook Children’s outpatient center, saving lives starts and ends with paperwork.
The process begins when a physician asks the team to seek compassionate use approval for a specific drug. For cancer patients like Zander, Dr. Micha Koentz, a pharmacist who leads the team, takes the helm; Dr. Jamie Haswell, the team’s second pharmacist, oversees the application for everyone else.
Koentz turns, first, to Google to find out which company makes the drug and which contact person to “badger.” She seeks out cell phone numbers; she can and will call a drug representative during dinner. Then, like an attorney, she prepares her case.
“I have to say, ‘We have this mutation, this trial won’t work, the patient can’t swallow, we need the liquid form,’” she said. “We try to have all of our ducks in a row and really tug at the heartstrings when we plead our case, because we’ve really only got one shot.”
She has one, maybe two minutes to sway the company to provide a drug — for free — for a purpose it wasn’t intended. The company’s central incentive is, she thinks, altruism. However, the data gleaned from a single patient like Zander, whose treatment serves as a case report, can spur the company’s own investment in new pathways for its drugs.
She waits. This is the hardest step, she said. If the company decides to approve her request, they provide a letter of authorization.
How can a patient advocate for compassionate use?
Discuss the option with your physician, Dr. Kelly Vallance recommends. Ask if any targeted therapies exist that might fit your case.
Koentz passes the baton to Danelle Morgan, the research pharmacy team’s technician. Morgan describes herself as “regulatory-minded.” After years of submissions, she’s on a first-name basis with department contacts at the FDA. She bundles the paperwork — the company’s letter of authorization, a consent form for the family, a protocol for treatment — and passes it along.
For cancer patients, the FDA’s turnaround is usually 24 hours. For non-cancer patients, it can take a month. Still, both Koentz and Morgan estimate that the FDA approves 99% of these applications. “You think of the big, bad FDA, like they’re going to be a big hurdle, but they’re not,” Koentz said. “They really do want us to try.”
With FDA approval in hand, Morgan submits a separate application to the hospital’s Institutional Review Board, an ethics committee tasked with monitoring medical research that involves people. For emergency cases like Zander’s, the process is expedited: Only the chairperson of the committee, Dr. William Stigall, needs to review and approve the request.
If each domino falls as it should, Koentz returns to the drug company with the approvals her team has gathered. The company dispenses the drug and the patient begins treatment. For cancer patients, the entire process typically takes less than two weeks.
“This is (a drug) that they truly are getting to add on quality time to their life,” Koentz said. “It’s just a really cool avenue of drug access I didn’t know existed whenever I was in pharmacy school. Who knew that you could call a drug company and ask for free drug?”
‘Any kind of hope, any solution’
Before Zander turned 10, he’d undergone an aggressive regimen of chemotherapy, radiation and surgery for the kidney cancer diagnosis he’d received as a 7-year-old. By the middle of the first year of the pandemic, his prognosis was stark.
After a patient exhausts the “standard of care,” or treatment avenues most likely to be effective, their options veer into the investigational. Some patients enroll in clinical trials. Zander wasn’t eligible, so in September 2020, his oncologist at Cook Children’s offered the family a rare glimmer of hope: compassionate use.
What’s the difference between a clinical trial and compassionate use?
A clinical trial is a research study where patients receive a treatment to determine if it’s helpful, harmful or no different from available alternatives. The main purpose of a clinical trial is to systematically collect data.
Compassionate use access is an opportunity for a patient with a life-threatening illness to receive a treatment that hasn’t been approved for that patient’s specific needs after standard options have failed. The main purpose of compassionate use is to treat the patient — and collecting data is a bonus.
A member of the Children’s Oncology Group kidney committee, Dr. Kelly Vallance, had heard from colleagues about an adult drug with low side effects that might work for Zander. “I never promise cure,” she said. “You can’t say that this is going to help, but maybe it will.”
By then, Zander’s parents had reached a “point of complete desperation,” Hunt said. “We’re just looking for any kind of hope, any solution.” She and her husband, Aaron, spoke to Zander about compassionate use; as a family, they agreed to move forward with the treatment.
Once the required groups approved compassionate use for Zander, the research pharmacy team became a liaison between his family and the drug company. They monitor his side effects and dosing and submit regular reports about both.
For example, his original dose, a pill, was too strong. Koentz and her colleagues used graduated cylinders (“like scientists,” she said) to dissolve the pill, diluting it with water and berry citrus syrup for flavor. These days, Zander sips the medicine from a syringe.
Some people misconstrue compassionate use treatment, Morgan said. They think someone who receives it is merely a “research rat.” But that’s not true, she and Vallance said. The first priority in trials like Zander’s is protecting the patient. When the drugs produce unwanted side effects, for example, the treatment stops.
But when a treatment works well, the benefits reverberate. The data the pharmacy research team collects helps not only the drug companies but the hospital. Vallance recently submitted Zander’s case report for publication in a peer-reviewed medical journal.
“It’s the best possible mesh of pharmaceutical research and patient need,” Hunt said.
A world people don’t know exists
Zander is 11 now. Medically, he’s cancer-free. After nearly two years of treatment with the investigational drug, he’s transitioned to “normal, fifth grade boy,” Hunt said. For him, that looks like school and soccer and the fourth Harry Potter book and playing outside until nightfall.
“It’s very typical,” she said. “I didn’t know how grateful I would be for ‘typical.’”
Zander’s binder on the drug-laden shelf of the research pharmacy is slim and robin’s egg blue. The color indicates compassionate use; Cook Children’s has eight of these cases right now, Morgan said. That number has increased over the past few years. Vallance attributes the expansion to the efforts of Koentz and her colleagues.
The vast majority of other binders are white — for clinical trials. The research pharmacy team oversees medicines for both types of investigational drugs.
Their other duties are manifold: They maintain a library of binders for studies already completed, monitor temperature in the pharmacy to protect the medicine, safeguard a locked cupboard of narcotics even physicians can’t access, counsel patients about drugs and parley with FDA auditors who come to assess their data collection.
For some clinical trials, the team is responsible for “blinding” the medicine — delivering a drug to some patients and a placebo to others without anyone knowing which is which. Sometimes that requires providing saline on a Saturday, Morgan said. Weekend work isn’t uncommon; once, they were called into the pharmacy to mix medicine for a malaria patient in the middle of a Sunday night, she said.
Haswell’s role mimics Koentz’s, but for non-cancer patients. The drugs she oversees are meant for improving a patient’s quality of life.
She originally pursued pharmacy for children because children in research tend to be overlooked. Cancer, for example, is far more common in adults, so drug companies can make more money from pursuing adult treatments. Furthermore, it’s difficult to persuade families to enroll their children into clinical trials. Put simply, her team works in a “world that a lot of people don’t really know exists.”
Behind the scenes, in a pharmacy painted Easter purple and brimming with binders and drugs, she’s grateful for the work they do.
“It’s not always a happy ending,” she said, “but a lot of times, it is.”
Alexis Allison is the health reporter at the Fort Worth Report. Her position is supported by a grant from Texas Health Resources. Contact her by email or via Twitter. At the Fort Worth Report, news decisions are made independently of our board members and financial supporters. Read more about our editorial independence policy here.