The Harvard Business School’s Kraft Precision Medicine Accelerator is tackling a series of initiatives that will position patients to help drive improvements in both clinical care and clinical research.
The accelerator was founded in 2015 with a $20 million donation from the Robert and Myra Kraft Family Foundation to speed the development of precision medicine approaches to get the right cancer drugs to the right patients. The accelerator is co-chaired by HBS Senior Fellow Richard Hamermesh and Kathy Giusti, founder and board member of the Multiple Myeloma Research Foundation (MMRF) and the Multiple Myeloma Research Consortium.
In November, Giusti and Hamermesh published an update on the accelerator’s first project: to develop best practices for patients to obtain and share their own data. The aims of the project are to learn how direct-to-consumer marketing strategies can be used to guide patients throughout their cancer treatment, and enable access to data that scientists can use to discover and develop new therapies.
“We must better anticipate pivotal moments in patients’ journeys where they are likely to benefit from communications,” Giusti and Hamermesh wrote in their project update in cure, a magazine for cancer patients and providers. Providing value to patients in this way, they said, would encourage patients to share more data.
In addition, they wrote, patients need to be educated on the value of combining their data and personal health information with data from other patients, “particularly those who share similar characteristics, so that scientists begin to see patterns that inform treatment.”
Giusti told BioCentury most patients don’t recognize the value of this information and believe that doctors and researchers put the data to good use. In reality, most clinical and genomic data remains siloed within companies or fragmented across public databases.
The accelerator believes that to unlock the value of these data, patients need to know that the data exist, how to get them and when to share them.
The project team, led by former consumer products retail executive Lori Marcus, began by analyzing the patient journey from diagnosis to treatment in collaboration with MMRF, the Pancreatic Cancer Action Network (PanCAN), the Prostate Cancer Foundation, the Metastatic Breast Cancer Alliance and LUNGevity.
According to Giusti, the groups learned that the journeys of the patients they represent were similar enough that they could pool resources to conduct joint patient-awareness campaigns.
The five patient advocacy groups also met with executives at consumer retail companies to learn how they interact with consumers and to identify tools and methods that could be used to reach patients and keep them engaged.
Together, the groups are using this information to develop best practices that can be shared with other patient groups to improve patient engagement and data sharing. The results also will be shared with healthcare industry executives via case studies presented at HBS.
But first, Giusti and Hamermesh wrote in cures, “There must be a resource where patients can safely and easily share their data, be it via a standard customer relationship management (CRM) system or personal health information in an institutional review board (IRB)-approved patient registry.”
Giusti said the groups involved in the accelerator would share their experiences in how they built their CRMs and registries, which will then be integrated into the best practices.
A second project team is developing best practices for platform trials, including how to fund, design, launch and manage them.
Giusti said the accelerator believes platform trials can alleviate problems that keep patients from enrolling in clinical studies, including the sheer number of ongoing trials a patient must evaluate and choose among, and the chance of being randomized to a control arm.
Platform trials, which test multiple drugs in a single study using a master protocol, can be more efficient than running separate trials because they eliminate the need for separate protocol design, site enrollment, contracting and training. In addition, platform trials’ use of a single control arm dramatically reduces the number of patients needed and gives a greater proportion of patients a shot at a new therapy that could be better than SOC.
Despite the success of the I-SPY 2 platform study in breast cancer and the launch of additional platform studies in lung cancer and acute myelogenous leukemia (AML), it remains difficult to get these studies off the ground.
Reasons include difficulty securing funding, identifying and obtaining drug candidates and developing the master protocol.
“We all step into this stuff not knowing what we’re going to run into.”
For example, MMRF has spent more than a year setting up its Myeloma Developing Regimens Using Genomics (MyDRUG) platform trial. The Phase II study will match patients to an initial slate of nine treatment regimens based on their cancer’s genomic signature.
Giusti said MMRF spent most of its time identifying drugs to include in the study and then convincing the companies to participate. The organization also spent time working with FDA on the design of the master protocol.
MMRF plans to start MyDRUG in 2Q18 or 3Q18.
“We all step into this stuff not knowing what we’re going to run into,” Giusti said. “There is no easy way to sit down and share.”
The accelerator’s platform trial project team is therefore bringing together MyDRUG and two other platform trials -- PanCAN’s Precision Promise and GBM-Agile -- to determine what causes delays and how they can be avoided. The team is led by Dixie-Lee Esseltine, who was previously VP of oncology clinical research at Takeda Pharmaceutical Co. Ltd.
“As we try to get these studies off the ground, we are looking in real time at what are the delays, why are they continuing to happen, et cetera,” said Giusti.
Precision Promise will test an undisclosed number of drugs to treat pancreatic cancer. Patients will be assigned to treatment arms based on their tumor’s molecular profile, the tumor microenvironment and patient preference factors. The trial is expected to start enrolling patients in 1Q18.
GBM-Agile will test an undisclosed number of agents across biomarker-defined subgroups. The adaptive trial is designed to seamlessly move compounds that clear the Phase II efficacy threshold into Phase III within the same protocol. GBM-Agile is sponsored by the National Biomarker Development Alliance and four patient groups: Accelerate Brain Cancer Cure (ABC2), Cure Brain Cancer Foundation, the National Brain Tumor Society and the National Foundation for Cancer Research.
GBM-Agile is expected to start in early 2018.
The platform trials project team is also working with PIs who have successfully launched and conducted platform trials, including Laura Esserman, who is chief PI on I-SPY 2, director of the Carol Franc Buck Breast Care Center and co-lead of the breast oncology program at the UCSF Helen Diller Family Comprehensive Cancer Center.
The accelerator has already learned that the organization managing a platform trial must have a skilled CMO and clinical staff who can help design the study, select agents to test and address insurance and administration needs that arise.
It also has determined that how the trial is funded and whether it’s run with a partner or independently by a patient foundation could affect how quickly the trial is set up and the drug candidates the trial can use.
For instance, Giusti said, if a foundation isn’t trying to get partners to donate money at the same time it’s trying to secure drug candidates, it allows the organization to be more “thoughtful” about the study and “spread the net wide in terms of potential pharma companies and biotechs. The better we can operationalize and accrue, the more likely industry will give their highest priority drugs for testing.”
The group is also meeting with stakeholders from industry and FDA to get feedback on best practices for platform trials. Giusti said the accelerator will compile the best practices and share them via case studies at HBS and publications.
The accelerator also has two additional projects that are in the early stages focusing on data analytics and new finance models for precision medicine.
Accelerate Brain Cancer Cure, Washington, D.C.
Carol Franc Buck Breast Care Center, San Francisco, Calif.
Cure Brain Cancer Foundation, Surry Hills, Australia
Harvard Business School, Boston, Mass.
Kraft Precision Medicine Accelerator, Boston, Mass.
LUNGevity, Chicago, Ill.
Metastatic Breast Cancer Alliance, New York, N.Y.
Multiple Myeloma Research Foundation (MMRF), Norwalk, Conn.
National Biomarker Development Alliance, Scottsdale, Ariz.
National Brain Tumor Society, Newton, Mass.
National Foundation for Cancer Research, Bethesda, Md.
Pancreatic Cancer Action Network (PanCAN), Manhattan Beach, Calif.
Prostate Cancer Foundation, Santa Monica, Calif.
Robert and Myra Kraft Family Foundation, Foxborough, Mass.
Takeda Pharmaceutical Co. Ltd. (Tokyo:4502), Osaka, Japan
UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, Calif.
U.S. Food and Drug Administration (FDA), Silver Spring, Md.
Cukier-Meisner, E. “Yes we PanCAN.” BioCentury (2016)
Giusti, K., et al. “What patients with cancer can learn from savvy shoppers.” cure (2017)
McCallister, E. “I-SPY adapts.” BioCentury (2017)
McCallister, E. “A new beat for AML.” BioCentury (2016)
Unsigned commentary. “Back to School 2016: Haste not waste.” BioCentury (2016)