CAMBRIDGE, MASSACHUSETTS—The MIT NEW Drug Development ParadIGmS (NEWDIGS) initiative, an international biomedical innovation “think and do tank” involving a range of stakeholders in the healthcare system, hosted a collaborative Design Lab to explore the challenges and opportunities presented by Efficacy to Effectiveness (E2E) strategies for the development and delivery of new medicines. 42 people, representing pharmaceutical companies, patient advocacy groups, regulators, payers, healthcare providers, and policymakers, participated.
The Design Lab, held July 19 and 20 at the Massachusetts Institute of Technology, was part of an ongoing NEWDIGS project called WISDOM. The project is focused on the planning and generating of evidence, to improve decision-making for all stakeholders across the lifecycle of innovative therapies.
The goals of the Design Lab were to understand design and implementation considerations of E2E strategies; to assess opportunities for E2E approaches in the context of case studies; and to review the evolving NEWDIGS Collaborative Design Toolkit.
“To date, all E2E work has been at the single product level,” said Dr Kay Larholt, NEWDIGS Director of Integrated Knowledge Solutions and one of the Design Lab organizers. “In the Design Lab we used concrete examples of new therapies in development to think about generalizable lessons.” The broad vision is to extend knowledge generation and analysis beyond the product level to at least disease-level for better end-to-end decisions. “A major problem we have is that there are so many one-off systems and data infrastructures…we need to move beyond asking what the data [in a given system] can tell us, start asking what we want to know, and look for the information we need,” she continued.
“We had great conversations about how decisions are made—and how those practices won’t suffice going forward,” said Dr Gigi Hirsch, Executive Director of CBI and Director of the NEWDIGS initiative. “We’re entering an exciting new era of collaboration, so we’re learning how to use data better, to make decisions informed by richer evidence, and to manage uncertainty throughout the innovation process.”
In order to encourage openness and collaboration in a safe haven environment, the workshop was held under strict confidentiality terms and Chatham House Rule (which assures participants of confidentiality to encourage openness and information sharing). Participants used an asset under development by a biopharmaceutical company as a case study, enabling discussion of E2E at both product and system scales. “The [tangible] case made all the difference for understanding the platform problem,” said one participant who works at a federal research organization. “It allowed for people [with different perspectives] to chime in at different points. …Especially with rare diseases, we need to come together early on to establish relationships and identify how to work together before Phase 1 trials to identify measures and desired outcomes.”
Another participant, from a national medicines agency in the European Union, agreed that the openness of the workshop was critical. “You could open up all the problems, share your perspective, and view them from others’ perspectives. It is a very creative, open-minded approach. And because I’m representing only my opinion, not my country’s official position, we could talk about the elephants in the room…we could share wisdom from past experiences.”
Participants were also able to examine and address assumptions around E2E. “The workshop made us aware of the E2E problems,” continued the European participant. “Things people might not recognize without definitions. E’s can overlap, be indistinct, or have gaps. By putting the problems into words …we can establish a framework for discussion. We can understand instead of assume.”
The workshop also included a public “Commons” sessions to look at tools and systems that cross sectors and stakeholders. One session, a panel called “The Role of Existing Evidence Generating Infrastructures in Evaluating Effectiveness,” included representatives from Harvard Pilgrim Healthcare Institute and the FDA. They discussed the capabilities—and limitations—of systems like Sentinel (The FDA’s system for safety monitoring) and PCORnet (a network of networks designed to improve healthcare decision-making by patients, clinicians, and other stakeholders) to support prospective research and development.
In the weeks ahead, working groups will produce detailed Design Lab summaries on the case as well as on high-level lessons that can be generalized. They will continue to refine the planning and evaluation toolkits, with the first goal to retroactively apply them to real-world cases from the pharmaceutical literature to explore how greater coordination in evidence planning between stakeholders (if it had been possible) might have delivered improved outcomes for patients.
The Design Lab was the latest in a series of workshops for NEWDIGS programs collectively aimed at advancing Adaptive Biomedical Innovation practices. “Biomedical innovation occurs in a complex adaptive system,” said Dr Hirsch. “Decisions made by one stakeholder can affect all other stakeholders, sometimes in ways that are negative and unpredictable. Adaptive biomedical innovation practices like those we are developing in NEWDIGS will help players in the system understand the consequences of their decisions, and make decisions based on more complete and relevant information to benefit not only their own organizations but also other stakeholders…after all, we’re each trying to ultimately achieve patient impact.”
“There is tremendous value [in this process] beyond what people realize,” said a veteran industry executive and longtime NEWDIGS community member. “This is far from an ‘academic’ initiative. Adaptive biomedical innovation practices can really change the way we develop and use biopharmaceutical and medical device innovations in medical care. The Adaptive Pathways approach in the EU is an example of what this community and process can produce.”
E2E strategies will be instrumental to successful platform clinical trials, which in turn are key to adaptive biomedical innovation. “Our current mechanisms for decision-making can’t keep up with the emerging science,” Dr Hirsch concluded. “This Design Lab has shown the biomedical innovation community’s readiness for new practices and technologies, which we will discuss in our Next Wave Forum this December.” The gathering of about 200 senior executives and thought leaders, planned for December 12–13 at MIT, will build on work across the NEWDIGS projects, and focus on advancing platform trials into end to end rapid learning systems. More on the Next Wave Forum can be found at nextwave.mit.edu.
About MIT NEWDIGS
MIT NEW Drug Development ParadIGmS (NEWDIGS) is an international “think and do tank” dedicated to delivering more value faster to patients, in ways that work for all stakeholders. NEWDIGS designs, evaluates, initiates, and connects advancements that are too complex and cross-cutting to be addressed by a single organization or market sector. Its members include global leaders in research, development, insurance, regulation, and patient advocacy. For more information, visit http://newdigs.mit.edu.