NEWDIGS has launched several initiatives with the ultimate goal of more reliably and sustainably delivering new, better, affordable therapeutics to the right patients faster. These initiatives are based on the proven NEWDIGS approach, which convenes all key stakeholders in a ‘safe haven’ environment to explore global capacity building through systems innovation.
Adaptive Licensing (AL)/Medicines Alternative Pathway to Patients (MAPPs)
NEWDIGS’ initial program centered on catalyzing the evolution of global pharmaceutical regulation and access, and specifically adaptive licensing (AL) also known as Medicines Alternative Pathway to Patients (MAPPs). AL offers an approach to refining the management of tradeoffs between access and evidence development. The AL model envisions a prospectively designed process based on early registration for a restricted patient population followed by expanded market authorization informed by iterative cycles of evidence gathering. It builds on a more explicit acknowledgement of the need for the progressive reduction of uncertainty around safety, efficacy and effectiveness over the lifecycle of a product. This paradigm is based on a coordinated, yet flexible, multi-stakeholder (e.g. manufacturer, regulator, reimbursement authority, patient and clinician) process that can be deployed using existing regulatory and reimbursement frameworks. This approach is designed to maximize the public health benefits of new drugs while balancing the need for information on benefits and risks.
NEWDIGS investigated the feasibility of AL/MAPPs over the past four years through the precompetitive collaborative analysis of a series of case studies involving proprietary products currently under development across the global industry. Findings have been widely disseminated through publications and conferences and have informed a European Medicines Agency (EMA) AL pilot program which was initiated in March 2014.
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 program is developing a next generation set of linked simulation tools and processes to provide increased scalability, rigor and quantification in evaluating the implications of AL/MAPPs based on each stakeholder’s perspective.
A key requirement for the successful implementation of AL/MAPPs and other early access paradigms is the ability to reliably collect and analyze data throughout an agent’s lifecycle to reduce uncertainty in a timely manner. While much data is currently available, it may not adequately meet stakeholder requirements for decision making. The NEWDIGS Data Program proposes to identify, characterize and address issues regarding healthcare data and systems that are necessary for the success of an adaptive approach to the lifespan management of drugs. This includes the exploration of processes, technologies and policies needed to optimize quality, interoperability and data sharing across boundaries. In addition, the program will specifically explore the issues underlying international data cooperation in the context of AL/MAPPs, as well as rare disorders and precision medicine.
The Science of Collaboration in Biomedical/Healthcare Innovation
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.