“What is the biggest barrier to progress in personalized medicine?” asked moderator Anna Barker, PhD, leading a panel discussion at a recent meeting convened by the Washington-based advocacy group, the Personalized Medicine Coalition, with the American Association for Cancer Research and Feinstein Kean Healthcare.
Five well-known experts responded without hesitation:
“A greater understanding of the disease,” said Richard Pazdur, MD, from the FDA. “This will lead to more effective drugs.”
“On my side, it’s payment issues,” said Aetna’s Ira Klein, MD.
“There is a fundamental mismatch with that incremental nature [of progress] and the time frame and focus of the public policy progress,” added John J. Castellani, President and CEO of the Pharmaceutical Research and Manufacturers of America.
“There’s no business model for developing the diagnostic tests that guide the use of targeted drugs,” said David Parkinson, MD, a Venture Partner at New Enterprise Associates. “Who’s going to pay for the development of companion diagnostic tests?”
“Complexity,” responded academic researcher, Kenneth C. Anderson, MD, from Dana-Farber Cancer Institute. “We need to identify not just targets but accessory pathways.”
Dr. Barker herself, who leads two initiatives to develop innovative research models at Arizona State University, noted that “increased costs are a major barrier to achieving personalized cancer medicine.” Indeed, she continued, “rising costs are a problem on many fronts—but for the introduction of innovative molecularly based interventions for cancer, they could be a catastrophic barrier.”
Long List of Issues
These and other viewpoints emerged repeatedly in talks and panel discussions throughout the symposium, Turning the Tide Against Cancer Through Sustained Medical Innovation, held June 12 in Washington, DC.
The meeting’s purpose was to focus attention on regulatory, reimbursement, and education issues. But panels and speakers often took a broader look at the challenges confronting developers of targeted cancer drugs. The morning keynote speaker, John Mendelsohn, MD, Director of the Khalifa Institute for Personalized Cancer Therapy at The University of Texas MD Anderson Cancer Center, ran down a long list of issues, from the heterogeneity of cancer cells and genes to clinical trial design to ethical concerns.
“The biggest bottleneck,” he said, “is information management.” Massive amounts of data need to be analyzed and stored in personalized medicine research, and that is made more difficult, by “data balkanization”—the tendency toward territoriality and competition.
Data-sharing Models
How to counter that tendency and encourage data sharing became the theme of the panel assigned to discuss innovative models in cancer research and care, moderated by Ramsey Baghdadi, Editor of The RPM Report. Panelist Laura Esserman, MD, noted that data sharing is vital to the success of the I-SPY 2 breast cancer trial, which is designed to speed drug development by testing multiple phase II drugs. The trial incorporates precompetitive collaboration for biomarker identification and makes study data publicly available.
“It’s an illusion that having data kept to yourself is an advantage, because now time to market will become much more important (now that there is a neoadjuvant path for accelerated approval),” said Dr. Esserman, who is Director of the Breast Care Center at the University of California, San Francisco, and principal investigator on I-SPY2.
But data sharing can be a hard sell, said panel member Kathy Giusti, MBA, Founder and CEO of the Multiple Myeloma Research Foundation. Her organization has launched a landmark study to collect data on the molecular and genetic changes underpinning the evolution of myeloma by closely following newly diagnosed patients for at least 5 years.
All inventions from the study are dedicated to the public domain. At first, academic centers were reluctant to relinquish rights to intellectual property, Ms. Giusti said, so her advocacy group turned to the community cancer centers, such as those run by US Oncology, as the first sites to participate in the study. Now academic centers and industry are also participating in a precompetitive consortium where all data generated from the study will be placed onto a free public portal prior to publication.
J. Leonard Lichtenfeld, MD, MACP, Deputy Chief Medical Officer of the American Cancer Society, also highlighted the importance of community cancer centers and stressed the need for access to the best medicine for all patients, including the disadvantaged. We have to make data “accessible and actionable” at the clinical level, he said.
Determining Value
The other panel of the day considered how to demonstrate and measure the value of targeted therapies. Moderator Kathleen Foley, PhD, of Thomson Reuters Healthcare, began by asking panelists how they would define value.
Gwen Darien of the Pathways Project and William S. Dalton, PhD, MD, Director of the H. Lee Moffitt Cancer Center and Research Institute, suggested that discussions of value should be framed around individual need. “You can’t individualize coverage as a payer,” pointed out Lee Newcomer, MD, Senior Vice President at UnitedHealthcare. “We need to prospectively define the standards of evidence for coverage to provide fairness to all our members.”
The need for both evidence-based medicine and personalized care—a system that combines evidence and expectation, as Dr. Dalton put it—continued to occupy this panel. The move to care based on individual circumstances and preferences will influence how we look at data, comparative effectiveness, and clinical trial design noted Al B. Benson III, MD, FACP, of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. He advocated “constant discussion” on how to get good evidence and on what is truly appropriate care.
The day ended with a wrap-up by Duke University’s Amy Abernethy, MD, of recurrent themes, such as the need for collaboration and sharing among companies and agencies; for a public dialogue on costs and reimbursement; and for more research on the basic science of targets and pathways. And, she noted, there was the overriding theme that emerged from the discussion on determining the value of targeted drugs: We need data. ■
Disclosure: Dr. Abernethy has received research funding from the the National Institutes of Health, Agency for Healthcare Research and Quality, Robert Wood Johnson Foundation, Pfizer, Helsinn, Amgen, Kanglaite, Alexion, Biovex, DARA, and MiCo, has served as a consultant for Novartis and Pfizer, and is on the corporate board of directors for Advoset and Orange Leaf Associates, LLC. Drs. Barker, Pazdur, Anderson, Mendelsohn, Esserman, Lichtenfeld, Newcomer, and Benson, and Ms. Giusti reported no potential conflicts of interest.
