Medicines Adaptive Pathways to Patients (MAPPs): A Story of International Collaboration Leading to Implementation

Abstract

After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Although it is a significant achievement to see the first asset being placed into human trials under an adaptive pathway, there is much to be learned regarding the multinational and multi-stakeholder effort that has driven the growing acceptance of MAPPs as a methodology and concept, as well as the need for continued and increasing international collaboration to foster the wider adoption of MAPPs. Changes in available science and technology, as well as a number of challenges in the current system, outlined in this paper, are transforming approaches to medicines development and approval. It is these challenges that have led directly to the groundbreaking MAPPs collaboration between the Massachusetts Institute of Technology Center for Biomedical Innovation's New Drug Development Paradigms Initiative, the EMA, patient, payer and health technology assessment groups, the European Federation of Pharmaceutical Industries and Associations, and the Innovative Medicines Initiative-a European public-private partnership. This article examines the development of MAPPs, from inception of the concept, to the establishment of this trans-Atlantic initiative, and examines challenges for the future.

Current challenges for assessing the long-term clinical benefit of cancer immunotherapy: a multi-stakeholder perspective

“…Separate trends in adaptive licensing at a regulatory level, and conditional reimbursement pathways, reflect this. 84 85 RWE in assessing long-term survival benefit of IOs is not yet commonly used in HTA 86 87 but has two potential applications: first, to provide data for modeling the survival benefit that can help generalize clinical trial data to real-world clinical practice 88 89 ; and second, to validate externally both the clinical trial data, particularly from later stages of follow-up, and any predictive modeling of long-term survival benefit. 90 91 …”

Improving Interactions Between Health Technology Assessment Bodies and Regulatory Agencies: A Systematic Review and Cross-Sectional Survey on Processes, Progress, Outcomes, and Challenges

“…Moreover, the success of APs requires a more "system-wide" approach including, for example, the willingness of patients to participate in clinical research to evaluate benefit/risk and determine if new medicines were effective and how different stakeholder perspectives are reflected in the decision-making process (90). As highlighted by Schulthess et al (93), the bigger challenge facing the adoption of APs borders on how to incorporate different decision-making processes into AP methodologies "to ensure the appropriate balance is struck between earlier access to new medicines, a given regulator's willingness to facilitate that to occur, a healthcare provider's willingness to accept more focused data before prescribing a new medicine, as well as the provider's correlating willingness to restrict their off-label prescribing practices and to participate in real-world clinical research to progressively reduce uncertainties, a given payer's willingness to purchase such medicines, and having strong multifaceted postauthorization systems in place to facilitate all of this in as safe and dependable a manner as possible. "…”

Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?

“…This is at the heart of “Why” stakeholders wish to engage in MAPPs. For an in‐depth description of this conundrum and of the principles and goals underpinning the MAPPs approach, please refer to previous work …”