New Drugs: Getting Them to Cancer Patients Faster
David Lassiter knows he is running out of options. The new drug Avastin is keeping him alive, staving off his colorectal cancer that recurred three years ago. But already the drug is showing signs of diminishing success. When it stops working, there are no drugs on the market to keep his cancer at bay.
"I'm not convinced the National Cancer Institute and the Food and Drug Administration are effective at getting new drugs to patients quickly," said Lassiter, a patient at the Duke Comprehensive Cancer Center. "I know there is a need to balance the risks versus the benefits, but we patients are less concerned about the potential side effects than the fact that the drugs may save our lives, which is the ultimate goal."
Currently, it takes 12 to 15 years and an estimated $1.5 billion from the time a drug is discovered until a safe and effective product reaches the market, said H. Kim Lyerly, M.D., director of the Duke Comprehensive Cancer Center. The drug development process hasn't kept pace with the rapid scientific advances in cancer biology and genomics, but the problem isn't solely a government regulatory issue, he said.
"We need to devise tangible solutions to accelerate drug development so that cancer patients have access to safe and effective drugs more quickly," Lyerly said. "The responsibility doesn't rest with a single player - academia, industry and government are collectively responsible."
Lyerly has teamed with government, industry and cancer groups to create an open forum for scientists and regulators to discuss the testing and validation of new drugs, free of the regulatory constraints that necessarily occur during official drug approval process. In 2004, he organized what is now an annual workshop with the FDA, the National Cancer Institute, the American Association for Cancer Research and the American Society of Clinical Oncology to train early-career scientists how to effectively develop and validate new anticancer drugs.
Contrary to assertions that the FDA closely guards its drug approval guidelines, Lyerly said the agency has embraced the opportunity to educate investigators on how to best approach drug development and approval. The problem has been largely one of communication, not privacy, he said. Scientists and companies design clinical trials and test new drugs without prior input from or collaboration with the FDA. When it comes time to seek regulatory approval, scientists may lack critical data that the FDA needs, sending the process back to the testing phases.
"Some of the very best scientists don't necessarily have the tools or the training to develop their drugs and move them through the approval pipeline," Lyerly said. "With this workshop, we're removing the barriers to constructive dialogue by linking academic and industry scientists together with FDA officials in a creative way to talk about new strategies for accelerating drug discovery."
In the training workshop, "Accelerating Anticancer Agent Development and Validation," seasoned cancer researchers from industry, academia and government - along with senior representatives from consumer groups - will share their knowledge of drug development with early-career cancer scientists from the United States and abroad. Workshop participants will bring their data about a promising anticancer agent or new therapeutic strategy. Senior scientists will mentor participants as they design strategic plans for fully developing and validating their anticancer agents.
Senior FDA officials will be among the more than 60 mentors - all volunteering their time - who will help train participants at this year's workshop, to be held May 17-19 in North Bethesda, MD.
"In this era of targeted drug development, it is imperative that there be an open dialogue among representatives of industry, academia, and the FDA regarding ways to optimize the development of these novel therapeutics with the ultimate goal of enhancing treatment options for cancer patients," said Neil Spector, M.D., director of Exploratory Medical Sciences-Oncology at GlaxoSmithKline. "This workshop provides an opportunity for researchers to obtain guidance in a much less formal setting, where the discussion can be more open and free of the constraints that might otherwise stymie the process if delayed until a later stage."
The workshop's primary goal is to encourage what is known as translational research -- transforming scientific discoveries made in the laboratory into viable treatments for patients, said Richard Pazdur, M.D., director of the FDA's Office of Oncology Drug Products.
"The workshop allowed me to converse with regulatory experts and better understand the requirements and expectations of the FDA and other agencies," said Antonio Wolff, M.D., associate professor of oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. Wolff attended the conference in 2004.
"In addition, the opportunity to present my proposal to a large group provided me with instant peer review and valuable critique of my plans and ideas," Wolff said. "In my specific case, it helped me and my colleagues fine-tune a large project prior to its submission to the National Cancer Institute."
Duke oncologist Kimberly Blackwell, M.D., who participated in previous workshops, said this specific type of training in drug development is essentially unavailable in cancer research.
"Bringing together these groups of people for collaboration makes this workshop an incredibly productive environment for cancer researchers," said Blackwell, assistant professor of medicine-oncology at the Duke Comprehensive Cancer Center. "The tools and insight I've gained have helped me to navigate the confusing pathway of drug development, and this has proved invaluable as I work to develop new therapeutics for my patients."