Compassion for desperately ill patients who need immediate access to experimental medicines
To make experimental medicines available to patients who have exhausted all options for treatment and clinical trials, the Food and Drug Administration (FDA) offers an expanded access (compassionate use) program. However, the process for approval can be cumbersome and costly. As a result, patients often face yet another delay at the worst possible time. Clinical Research Pathways seeks to give these patients another chance by:
- Helping them learn about the approval process to obtain experimental medicines.
- Making it easier for them to gain access to experimental medicines by streamlining IRB procedures for review of expanded access requests.
We work closely with government officials, pharmaceutical and biotechnology companies, and others involved in drug development to make information about experimental drugs more available to physicians and patients. We also partner with pharmaceutical companies and government agencies to streamline the approval process.
One patient’s story: How we helped speed approval for experimental treatment
It took more than two years for Tyson Fowler to receive an accurate diagnosis of the cause of his motor neuron symptoms. By then, chronic Lyme disease had taken its toll. The once healthy, athletic 30-something was confined to a wheelchair, unable to move on his own. His condition was worsening rapidly, and the prognosis was grim.
At the recommendation of one of the nation’s top Lyme-treating physicians, in 2013 Tyson and his wife, Ann, began making periodic trips to India so he could receive experimental stem cell treatments. For the first time since he was diagnosed with Lyme disease, Tyson and Ann saw progress—and cause for hope.
Clinical Research Pathways literally opened the door for us. I don’t know where we’d be today without them.
Then came a major setback. In February 2015, Tyson suffered cardiac arrest. He came home from the hospital on a ventilator, in need of constant care and too weak to continue to travel to India to continue the experimental therapy. The only option was to request permission—under the FDA’s expanded access program—to import the biological materials and be treated in the U.S.
“I spent the better part of two years navigating the complete unknown and asking so many people for help,” Ann says, only to face one roadblock—and rejection—after another. This summer, in what she terms a “Hail Mary Google search,” Ann typed in the words “independent IRBs” and “compassionate use,” and the WCGF website popped up.
Within 10 days of WCGF’s involvement, an IRB had reviewed and approved the Fowlers’ request. The first shipment has arrived in the U.S., and Tyson has resumed treatment. Already, he’s showing signs of improvement.
“Before WCGF, we were at a standstill and, even, moving backward,” Ann says. “It was such an awful, defeated feeling. Now, we’re hopeful—and I know to point people to WCGF so no one has to go through what we did.
“WCGF literally opened the door for us,” she adds. “I don’t know where we’d be today without them.”
The Fowlers’ experience illustrates both the importance of the FDA’s expanded access program and the value of our expertise to those who need help navigating the expanded access process. Note: In 2017, when we worked with the Fowlers, Clinical Research Pathways was known as WCG Foundation (WCGF).
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For more information on expanded access:
For treating physicians:
- Your role in the expanded access process.