Science is advancing in exciting ways, bringing long awaited cures increasingly within reach. In many ways, though, the science is advancing faster than the “system” – the policies, processes, and structures that shape the path for new medicines to patients. This evolutionary lag creates challenges for all stakeholders in the biomedical innovation ecosystem – and most importantly, it can delay or prevent access for patients to new, needed treatments.
The traditional innovation system needs to be modernized, but closing the evolutionary gap between the pace of scientific advancements and the system that shepherds innovation through to impactful medical treatments is a massive undertaking in terms of scope, scale, and complexity. The system is evolving, but in fragmented ways across traditional stakeholder, expert, and geographic siloes. “Adaptive Biomedical Innovation” (ABI) – a term coined by NEWDIGS – offers a shared vision and general principles to foster greater efficiency, coordination, and effectiveness as this evolution continues to unfold.
ABI focuses on aligning stakeholders in ways that drive sustainable patient-centered innovation, applying the principles of continuous learning and improvement across the product life span in ways that: (1) optimize benefit/minimize harm for patients, (2) progressively enhance knowledge and reduce uncertainties about the treatment, and (3) incorporate acceptable tradeoff decisions for all stakeholders.
NEWDIGS’ first project (2010-2013) in ABI focused on “Adaptive Licensing”. Findings have been widely disseminated through publications and conferences and have helped to inform the European Medicines Agency (EMA) ‘Medicines Adaptive Pathways to Patients’ (MAPPs) pilot rogram which was initiated in March 2014. MAPPs is a version of ABI that has been tailored for Europe, but many of its underlying principles have universal relevance. NEWDIGS remains actively involved supporting related activities in the EU while also enabling coordinated advancements and shared learning in ABI across regions globally.
Adaptive Biomedical Innovation (ABI) requires that there be a more seamless knowledge continuum spanning the pre/post market phases of product innovation.
Evidence generated from data sources beyond traditional Randomized Clinical Trials (RCT) is becoming increasingly important in biomedical innovation in the era of precision medicine and value-based reimbursement models. While there is much excitement about the increasing availability of “big data,” our ability to deliver meaningful patient value from it so far remains elusive. Shifting our focus from inputs to outcomes requires that we better understand the requirements for actionable evidence as defined by key decision-makers (regulators, payers, providers, and patients). In other words, the new evidence must be ‘fit for purpose’ as determined by stakeholders upon whose actions innovation success will depend.
The NEWDIGS WISDOM Project seeks to describe and demonstrate how new kinds of evidence integrated with that from traditional RCTs could impact (1) specific decisions regarding biomedical innovation marketing authorization, access and use, and (2) outcome metrics that are meaningful to each stakeholder.
Detailed information can be found in the WISDOM Project Prospectus.
Financing and reimbursement of Cures in the US
Medical research is making significant advances toward treatments that cure major diseases, but these treatments will often entail very high up-front costs. The FoCUS Project targets the need for new innovative financing and reimbursement models for curative medicines in the US that ensure (1) patient access for needed treatments; (2) affordability for public and private payers; and (3) the sustainability of innovation by manufacturers.
FoCUS activities center around the design, rapid cycle prototyping, and piloting of financing and reimbursement models for curative therapies in the US, with targeted pilot launches occurring throughout the life of the project. All financing and reimbursement designs will be derived from work done by disease specific multi-stakeholder Target Area Groups (TAGs) in coordination with NEWDIGS team members, MIT Faculty and others. Designs will be rigorously evaluated through modeling and simulation exercises, evaluated through product specific case studies in NEWDIGS Design Labs and vetted. Learnings will be disseminated broadly to inform the stakeholder community, pilot implementations and scale up. Initial priorities for action are curative gene therapies and durable oncology treatments.
Detailed information can be found in the FoCUS Project Prospectus.
NEWDIGS success in advancing its AL/MAPPs initiative was built around a methodology of interactive multi-stakeholder, case-based simulation exercises.
These exercises, conducted in a safe haven environment and based on proprietary data from real world assets, provided specific insights into the implications of AL/MAPPs for all stakeholders and facilitated stakeholder decision making. The JANUS project is developing a next generation set of linked simulation tools and processes to provide increased scalability, rigor and quantification in evaluating case-based implications of AL/MAPPs and other accelerated development and access pathways based on each stakeholder’s perspective.
Over the past decade there has been a proliferation of multi-stakeholder consortia and collaborations aimed at addressing a diverse set of major global challenges in biomedical innovation.
Although there have been some descriptive studies of these collaborations, to date there has not been an organized effort to characterize them and, translate experience into continuous improvement. The NEWDIGS Science of Collaboration Program focuses on establishing a conceptual framework, tools, and knowledge to enhance our capacity to reliably and sustainably deliver value in distributed innovation environments.