Sharing Strategies to Navigate the Drug Development Pathway
The era of molecular medicine means different things to different people. To cancer patients, it holds the promise of tailored treatments to match their particular pathobiology. To drug companies big and small, it means that thousands upon thousands of potential "new agent recipes" have been published in the human genome, exponentially increasing the number of drug targets. To Dr. H. "Kim" Lyerly, it is a call to arms: "This new era of drug development requires us to reinvent the way we perceive, pursue, and communicate about these opportunities."
Dr. Lyerly is the director of the Duke Comprehensive Cancer Center (see Conversation with Dr. Lyerly). Three years ago he organized a training workshop around the premise that young investigators - having already embarked on the discovery-development-delivery pathway with a promising cancer drug or technology - often need the kind of strategic advice that only seasoned veterans are likely to possess and that is routinely unavailable in the American system of cancer research.
This spring nearly 50 early-career cancer scientists from the United States, France, Italy, Belgium, South Korea, and England came to the third annual "Accelerating Anticancer Agent Development and Validation" workshop in Bethesda, Md. These researchers arrived at the workshop with a wide variety of projects at various stages of development.
What they had in common, however, was quality: Their projects were competitively selected for merit and promise. "Yet some of the very best scientists don't necessarily have the tools needed to develop their drugs and move them through the approval pipeline," explained Dr. Lyerly.
What the participants needed most was the kind of focused mentoring that the workshop's senior faculty - more than 50 leaders in clinical and translational research from academia, industry, NCI, and the Food and Drug Administration (FDA) - had volunteered to provide.
Dr. Jerry Collins, associate director for Developmental Therapeutics in NCI's Division of Cancer Treatment and Diagnosis (DCTD), spoke in one of the opening sessions on the preclinical aspects of drug development. "The workshop delivers exactly what it promises: a concentrated experience in which learners can pick the brains of senior drug developers on how to navigate from an 'idea-and-a-molecule' to a drug and therapy for patients," said Dr. Collins. And the atmosphere was surprisingly collegial, he found. "Even the most experienced teachers can learn from the fresh approaches brought forward by talented students."
More than 9 hours of small-group sessions were spaced throughout the 3 days so that each of the participants had a chance to conceptualize a strategic plan for developing and validating the molecule they were working with as a biomarker, therapeutic agent, or cancer prevention agent.
In each of the breakout groups, four to six researchers presented their projects to an expert panel on drug development, consisting of an FDA representative; a biostatistician; senior scientists from NCI and FDA; and at least one academic, clinical, or industrial investigator; members of the advocacy community; and others experienced at matching projects and funding sources.
The researchers found themselves in a privileged position, but also occasionally in the hot seat. Some were cross-examined about possible scientific flaws in their model or misconceptions they might have about moving a product through the development pipeline. Most were asked to demonstrate their agent's potential impact and what its value might be to different constituencies.
Not every drug will target tens of thousands of patients, nor offer the kind of benefit that warrants millions of dollars in drug company investment. But other avenues of development exist: NCI has programs, initiatives, and projects to foster the development of some of these agents and strategies. Many of the participants learned of alternative opportunities for support and investment.
"The course provides a comprehensive view of the drug development process," said faculty member Dr. Richard Pazdur, director of FDA's Office of Oncology Drug Products. "Few individual investigators possess this viewpoint, and the workshop illustrates the interplay of the many stakeholders throughout the drug development process." And of course the FDA oversees more than just oncology drugs, added Dr. George S. Johnson, of DCTD's Developmental Therapeutics Program, who called the workshop "a model for translational research fully applicable to other diseases."
Information about the workshop is available at http://www.acceleratingworkshop.org. Major sponsors of the event included the American Association for Cancer Research, the American Society of Clinical Oncology, FDA, NCI, and the Duke Comprehensive Cancer Center.
By Addison Greenwood