Breath from Salt by Bijal P. Trivedi & Bijal P. Trivedi

Author:Bijal P. Trivedi & Bijal P. Trivedi [Trivedi, Bijal P.]
Language: eng
Format: azw3
ISBN: 9781950665501
Publisher: BenBella Books, Inc.
Published: 2020-09-07T16:00:00+00:00

CHAPTER 33

A Network for Developing Therapeutic Drugs

1997 –1998

If you build it, they will come.

—Field of Dreams

With the successful launch of two drugs, Cystic Fibrosis Foundation president Bob Beall now had a new agenda and strategy: he would work directly with biotech and pharmaceutical companies to design medicines. Anticipating that this new venture would eventually yield the foundation a drug to test, he wanted to address a looming problem that haunted all clinical trials: recruiting patients.1 Pharmaceutical companies frequently struggled to fill their clinical trials, even for common diseases, and the problem was much worse for rare diseases like CF. Beall needed a system to rapidly identify the right patients for a trial and funnel them to a nearby center where they could participate. And he knew Bonnie Ramsey was the mastermind for the job.

Beall and Ramsey had been friends and colleagues for some time when, in 1997, Beall came to Ramsey with a proposal. He wanted her to lead a new project for the foundation, one she had been pushing him to launch for a decade: a permanent infrastructure for future CF drug trials, through which physicians could recruit patients quickly and perform those trials more efficiently.

In the past, each time Ramsey completed a clinical trial, the entire network of nurses and scientists and physicians disbanded. When another began, they had to reassemble the team, hire and retrain nurses and trial coordinators, and establish new drug testing protocols. Wouldn’t it be more efficient, Ramsey had asked Beall after the Pulmozyme trial, if the foundation had a permanent network of sites dedicated to clinical trials? One staffed with nurses and lab technicians familiar with all the nuances of cystic fibrosis, experienced and comfortable with the complicated breathing measurements and chloride tests—professionals with a deep knowledge and appreciation of the disease and its quirks?

The model Ramsey envisioned for this network was the NIH-funded Children’s Oncology Group,2 which began in 1955 as a cooperative of cancer clinical-trial groups working to find treatments and cures for infants, kids, teens, and young adults. The COG’s strategy had three prongs: clinical trials, long-term follow-up with patients, and basic research into the biology of cancer. Over the next fifty years, the network of medical centers participating in COG studies and trials rose to 230 in six countries and involved more than 5,000 pediatric cancer specialists. Within the same period, the survival rate for patients diagnosed with childhood cancer skyrocketed from just 10 percent—a death sentence—to 80 percent, transforming the field.3 The key to this turnaround was the multidisciplinary patchwork of physicians, nurses, biologists, pharmacologists, psychologists, and other specialists who treated patients and studied them through each new trial and beyond. Ramsey wanted to do the same for cystic fibrosis.

Before Pulmozyme and TOBI, cystic fibrosis treatments had a long history of being tested in small, weak clinical trials at individual clinics.4 In about 80 percent of these studies, it was hard to tell whether the drug or therapy was successful, because the studies either recruited too few patients or were poorly designed.

Download

Copyright Disclaimer:
This site does not store any files on its server. We only index and link to content provided by other sites. Please contact the content providers to delete copyright contents if any and email us, we'll remove relevant links or contents immediately.