With billions of dollars spent on the development of precision medicine and related cancer research over the last decade, a recent partnership seeks a new way to bring these treatments to patients more quickly: through a better business model.
Founded in 2016 with a $20 million gift from the Robert and Myra Kraft Family Foundation, the Kraft Precision Medicine Accelerator is a partnership between the foundation, Harvard Business School, and the Broad Institute of MIT and Harvard. The alliance is working to eliminate inefficiencies and bottlenecks in the precision medicine development system to speed delivery of targeted therapies to the marketplace.
“Early on, people would ask us why the (Harvard) business school, and not the medical school, was the recipient of this gift,” says accelerator co-chair and HBS senior fellow Richard Hamermesh. “Nowadays, I don’t get that question—it’s clear.” Simply put, inefficiencies in the development of precision medicine can best be addressed by a business-analysis approach.
With the mapping of the human genome completed 15 years ago, the sci-fi concept of using a cancer patient’s genetic information to create personalized, FDA-approved therapies has become a reality, as have immunotherapies that induce or suppress a body’s immune response to treat disease. Yet, this relatively swift progress has not been fast enough for millions of other patients who still lack effective treatments that take into account their genetic or immune makeup.
While oncology currently leads the way in precision medicine, therapies are also available to treat cystic fibrosis, for example; and the field has the potential to impact treatment of all diseases, including crippling neurological conditions such as Parkinson’s and Alzheimer's.
“The Kraft initiative was founded with the goal of optimizing the business plan in precision medicine to accelerate new therapies to oncology patients,” says co-chair Kathy Giusti, adding that much of that work has entailed identifying high-performing organizations in the precision medicine ecosystem that are willing to collaborate and drive progress. Giusti is a senior fellow at Harvrd Business School.
“We bring together the most disruptive and thoughtful leaders in this area and engage them to identify the greatest challenges and solve for them,” she says. “As we do that, we write the business plan to help accelerate treatments.”
(An early mover in the field of precision medicine, Giusti founded the Multiple Myeloma Research Foundation in 1998, where she now serves on the board of directors.)
After joining the initiative in February 2016, Hamermesh and Giusti identified four work streams to address blockages that currently exist in the system:
Direct-to-patient
The Kraft team realized an acute need to reach more patients and encourage them to share their data. “Without access to their tissue and data, you will never understand what the new targets might be or how to use currently available drugs,” says Giusti.
Working with leaders at direct-to-consumer enterprises and five leading cancer organizations, they drew on the experience of businesses including Keurig, Peloton, and Rent the Runway to develop more effective strategies to engage and educate patients about precision medicine and the need for genetic testing.
Business techniques employed include developing an emotive brand to draw people in; using more social media; employing jargon-free language; and “making everything simple and straightforward, from our registration process to the questions we ask,” Giusti says. In other words, create a better end-to-end consumer experience, with the result being a larger, deeper pool of data to draw on and drive progress in new therapies.
Data and analytics
“It takes an abundance of data to answer the questions that every patient has about their specific, precise journey,” says Giusti. “Cancer is so heterogeneous that we have to build robust, standardized datasets that can be thoughtfully analyzed to answer those questions.”
Once they have it, however, most research organizations protect their data, as it represents a substantial investment of time and money. How to circumvent this stickiest of bottlenecks? Create business partnerships by framing the right questions, says Giusti, and then identifying and combining existing datasets for analysis to answer those questions. The organization also rallies individual groups around the importance of a particular question, providing incentives and protecting proprietary information as needed—then leveraging the latest in artificial intelligence and machine learning through entities such as GNS Healthcare and IBM’s Watson to find an answer.
Clinical trials
In the age of precision medicine, it’s even harder to find people to fully enroll in clinical trials, Hamermesh says. “We’re not just looking for anyone with pancreatic cancer—it’s patients with these three particular mutations.”
That underlines the importance of making patients aware of the benefits of knowing their cancer subtype, he notes, in addition to the benefits of sharing data, which can then be used to more quickly identify the most pressing targets and design trials to address them.
Innovations to the typical clinical trial model are also being used. In an adaptive platform trial, for example, different drugs are simultaneously tested in multiple arms of the same trial. Drugs that aren’t working may be more quickly eliminated; patients can be moved over to the arms of the trial that are seeing a response. Challenges are also addressed more immediately and published in real time in order to optimize the dissemination of best practices for trials in startup mode.
Currently the Kraft team is tracking three ongoing trials for pancreatic cancer, glioblastoma, and multiple myeloma, and has met with the US Food and Drug Administration to discuss standardizing platform methodologies.
Investment
Innovative trial designs require significant funding. The Kraft team is conducting interviews to evaluate models at the Cystic Fibrosis Foundation (philanthropy); at Stanford’s StartX and MIT’s Sandbox (incubator); and at Breakout Labs and Root Capital (venture philanthropy).
“We’re exploring new, venture-based models for creating and delivering personalized medicine, among other approaches,” Giusti says. “For example, how would we design a model to allow investors the return they’re seeking?”
Hamermesh also cites the possibility of funding open source challenges as a less expensive, faster way to develop initial answers to tough questions.
“Maybe you don’t get the perfect answer,” he says, “but for $50,000 you get three-quarters of the way to an answer that otherwise would have cost $500,000.” Or perhaps philanthropists who underwrite a drug’s development could receive royalties from it.
A more powerful investment
All of these approaches require a shift in the traditional philanthropy mindset, Hamermesh says, from funding the construction of a new cancer pavilion to financing the development of specific therapies for cancer subtypes—a less visible, but potentially more powerful, investment.
These four work streams—with the necessary added ingredient of time—will make precision medicine a reality for millions of more patients, the Kraft team believes.
“The accelerator acts as a catalyst,” Hamermesh says. “It’s causing a reaction to happen faster, and with less friction, so we achieve our end goal of making more targeted and immune therapies available to patients as quickly as possible.”
The next step for the KPMA is to launch an HBS Executive Education program, Accelerating Innovation in Precision Medicine, in September with the goal of helping organizations build business plans in precision medicine.