The LEAPS Project in the MIT Center for Biomedical Innovation’s NEWDIGS Initiative held its third Design Lab June 18–19 at MIT. It was well attended by leaders and innovators across the spectrum of healthcare system stakeholders involved in rheumatoid arthritis (RA) research and patient care.
LEAPS is the newest project of the NEWDIGS Initiative, building on its 10-year record of action-oriented, inclusive collaboration on system-wide innovation. The fundamental question driving the project is, what will be the fate of biomedical innovation in a world of value-based healthcare? Like other NEWDIGS projects, LEAPS is aimed at driving more value from biomedical innovation to patients faster, in ways that work for all system stakeholders.
The project’s first goal is to design a Massachusetts-wide pilot project for a rheumatoid arthritis “learning engine” for better evidence-driven outcomes for patients in ways that are more efficient for the system. The project team anticipates launching pilot modules in 2020, accompanied by a “playbook” of generalized principles that guide the project and which could be applied to other disease areas and in other geographies.
“The stakes of getting treatments wrong are high, as patients suffer irreversible progression of the disease,” said Gigi Hirsch, MD, Executive Director, MIT Center for Biomedical Innovation and Director of the NEWDIGS Initiative. “The market for innovators is crowded and undifferentiated, which makes commercialization risky…and a lot of what's being used and paid for isn't working well—that's a lot of waste and inefficiency.”
“The costs of getting it wrong fall disproportionately on patients and their families,” she continued. “They feel most strongly the cost offsets of being on the wrong treatment. That matters because the incentives aren't necessarily aligned for a major disruptive change, which means LEAPS can catalyze something that might not otherwise happen.”
The challenge and opportunity of downstream innovation
Driving change will mean redirecting actions of many parties—patients, payers, providers, developers, and many others, without any coordinating authority. “In LEAPS we're dealing with a complex adaptive system—many players, no single boss. How do we grapple with that? We need to determine how to align incentives, and how to make it easier for everyone to do the right things for patients.”
The premarket—upstream—innovation path is guided clearly by a single authority, the regulator. Fundamentally, the regulator’s responsibility is to determine whether a new medicine, considered alone, offers more benefits than risks to the “average” patient in a clinical trial. LEAPS focuses on generating evidence that is missing when the product enters the market, and is critical for choosing which medicines—in what combination or sequence—are likely to be best for a given patient, in the context of their particular disease experience over time. This evidence requires different types of data, collected, linked, and analyzed in variety of ways, than the evidence required for regulatory decision making.
The Design Lab series convenes leaders across the RA innovation ecosystem (upstream and downstream) toaddress these critical knowledge gaps, by designing new scalable evidence generation platforms, aligning incentives, and leveraging big, real world data, emerging technologies, and advanced analytics, in new ways. The diversity of expertise and perspectives, harnessed through proven Design Lab methods, will guide LEAPS toward solutions fit for the complexity of the challenge—a winning combination that has already driven change in past NEWDIGS programs focused on Adaptive Licensing and financing strategies for curative therapies.
Perspectives of the healthcare innovation leaders involved in the project
"LEAPS is addressing knowledge gaps to improve care delivery from the perspectives of multiple stakeholders. Today there's no way to determine which medicine is appropriate for a given patient at a given time, so we risk wasting patients' time and they live with symptoms longer than they need to. We need better information to guide decision. LEAPS incorporates important stakeholder viewpoints and perspectives…all the people affected by decision-making…I haven't seen a group assembled like that."
Pharmaceutical clinical researcher and rheumatologist
"I'm intrigued by the potential of the process to lead to better outcomes, from both patients' and payers' perspectives. We are keenly aware of the personal response to medicines, and we can see the variation of those responses across the [rheumatoid arthritis] patient population, but we also must manage efficiency and fiduciary responsibility. LEAPS could give us the opportunity to determine the best first treatment, which will benefit patients and payers alike."
Public payer organization executive
"I find LEAPS exciting because I often see attempts at getting to precision medicine in RA as having very limited scope such as focused on one clinical trial or one set of potential biomarkers. This approach is much more global in scope and the breadth and depth of expertise —including third party payers, pharmaceutical companies, patient advocates, clinical and outcomes researchers—is exactly what we need to start this process of determining the right patient, right drug, and right time. We can start with larger questions using resources, methods, and input from all parties, rather than starting small and trying to generalize."
"What's so compelling is the motive to find the right medicine for the right patient at the right time. This will add incredible value to what I offer patients, because what we do now is start with the gold standard, which works for 25–30% of patients, but some people need more…we have lots to offer, but it's trial and error. It's a privilege to be part of this project that will help all rheumatologists take better care of their patients. It appeals to my curiosity about medical research—to learn how how to take better care of patients, and how to be the best rheumatologist possible."
Rheumatologist and program director for rheumatology fellows
"At FDA, we are aware of many pilot projects that aim to leverage RWE for particular diseases or therapeutic areas. Some of these pilot projects may come to fruition and sponsors might want to use a project’s generated RWE to ask the FDA for changes to their product’s label, such as adding an indication, adding a new patient population, or possibly to demonstrate comparative safety and efficacy as is allowed by the 21st Century Cures Act. So FDA wants to be in that conversation early, instead of saying 'you should have done this or that' at the end when we receive the RWE to make a regulatory decision."
FDA medical officer
"The LEAPS RA pilot is a great way to highlight Massachusetts' ability to collaborate on innovations that deliver value to the whole healthcare system. It builds on our past achievements—such as our value-based care initiatives and healthcare coverage requirements—and will show how we can generate and use valuable evidence to inform the development of new therapies and improve the clinical care our providers deliver."
Massachusetts state official
"LEAPS is important because it's targeting the same issues that we're working on every day. It will help get the right medications to patients, reduce barriers to access, and improve their outcomes and quality of life. And, as an arthritis patient, I believe patient engagement is especially important in designing a system around us…we need to be in the room to co-create it. We have important evidence to contribute, and we are a constant factor across all clinical encounters and everything around them."
Patient advocacy foundation leader
"LEAPS is a concept to change how we work in the industry, a guiding light building a shared understanding…NEWDIGS attracts the most diverse group of stakeholders and keeps them together and collaborating for a long time, which the deliverables require. From a physician's point of view, this work is extremely important and innovative, with tremendous impact on patients."
Physician and pharmaceutical pharmacovigilance executive
"We're building real-world models for doing things better; that's my fundamental objective as a regulator. NEWDIGS is an intellectual oasis where we can really get down into details, a unique combination of people and process, and a meta-model of what we're trying to build. The long-term commitment to the long-term view is rare. It’s the approach we need in this transitional era."
National regulatory official