June 14, 2012 (Washington, DC) — One of the challenges in the development of therapeutics today is the inability to move drugs in a reasonable manner to the patients who need them. So said Siddhartha Mukherjee, MD, author of The Emperor of All Maladies: A Biography of Cancer, assistant professor of medicine at Columbia University, and staff physician at Columbia University Medical Center in New York City.
He was speaking here at Turning the Tide Against Cancer Through Sustained Medical Innovation, a conference held June 12 to examine the changes needed to promote progress in personalized cancer medicine. Wide-ranging discussions at the meeting touched on the challenges and innovations involved in moving from cancer science to better care. The conference was cohosted by the Personalized Medicine Coalition, the American Association for Cancer Research, and Feinstein Kean Healthcare.
It is an embarrassment.
To illustrate his point, Dr. Mukherjee described how he and a colleague published a paper suggesting that a specific medicine could be effective against ovarian or breast cancer. He then spent 2 years trying to get institutional approval for a clinical trial, but had no luck. Six months ago, he called up a colleague in India and sent over all the human subject data and preclinical data he had. Within 3 months, the colleague in India enrolled the first patient in a trial.
"It is an embarrassment that in the United States we cannot put patients on trial over the course of 3 years, whereas in India, you can put patients on trial in 3 months," said Dr. Mukherjee. He noted that he wasn't talking about putting holes in the safety apparatus.
In another talk, John Mendelsohn, MD, past president of the University of Texas M.D. Anderson Cancer Center in Houston, asserted that "data balkanization" is blocking personalized drug development. He is also director of the Khalifa Institute for Personalized Cancer Therapy at M.D. Anderson.
Companies, academia, and even departments in a cancer center are the same. "We all hold onto our data; we don't like to share," he said. "It's time for us to say: 'Okay, time out. It's the patient that counts." He called on researchers to share data and share the methodology used to get the data. For mutations that are present in 2% or 3% of cancer patients, each institution is going to be treating just a handful of such patients, he explained. To get meaningful answers about whether a treatment works, he said, "we're going to have to pool data."
Some innovators have managed to push beyond data-balkanizing forces. In a session on new models in cancer research and care, Kathy Giusti, MBA, founder and CEO of the Multiple Myeloma Research Foundation and Multiple Myeloma Research Consortium (MMRC), described how she realized early on that the system was not enabling the collaborative efforts needed to study an uncommon cancer. Each year, about 20,000 people are diagnosed with multiple myeloma in the United States. When a cancer is that uncommon, she said, you need a critical mass of centers working together.
With the help of experts, she decided to build a clinical network. The MMRC designed the model everybody would use, the membership criteria, the standard operating procedures for tissue banking, the way trials would be selected, and the way complications would be handled. MMRC decides who gets to join and everybody is held accountable for the metrics, she explained.
"If you do a great job, you get support at your center," said Giusti. "If you don't do a great job, we have to ask you to leave."
Besides building its own tissue bank, the clinical network followed a collaborative model on genomics. Data from its genomic sequencing initiative were placed in a public portal that more than 1000 scientists are using to study myeloma genomics. Giusti noted that "everybody did really want to hold on to their data. It was hard to put them out there in the public domain."
Collect Data as You Go
To run better clinical trials, research and clinical care should be one integrated system, said Laura Esserman, MD, MBA, professor of surgery and radiology at the University of California, San Francisco (UCSF), director of the UCSF Carol Franc Buck Breast Care Center, and coleader of the breast oncology program at the UCSF Helen Diller Family Comprehensive Cancer Center. "Research can't be an afterthought," she explained, adding that it's better to collect data as you go.
"Each time someone comes in, I should be informed by everything I've done to date," she said. "But that's not how our systems work."
All the information technology systems are optimized, integrated, and developed specifically for institutions, not networks. "It's not patient-centric," she said, which "does not maximize learning."
Dr. Esserman said her fantasy is a world in which new registries would contain mission-critical data needed to care for patients. The data would be collected once and become part of the registry. You could verify it electronically, and it could drop into your clinical trials. Once the drug is on the market, the registry could help you check its toxicity or long-term efficacy.
Imagine you had accelerated approval, she said. Now you need to conduct research on 1000 or 2000 patients. "Anyone who wants access to that drug better figure out how to collect toxicity and outcomes data," she said. "All of the sudden, you've got the driver to make people develop the systems we really need to transform our cottage industry of medicine into something that is a healthcare learning system. I think that is what we need."
Medscape Medical News © 2012 WebMD, LLC
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Cite this: Calls for Collaboration to 'Turn the Tide on Cancer' - Medscape - Jun 14, 2012.