Adaptation through Collaboration: Developing Novel Platforms to Advance the Delivery of Advanced Therapies to Patients

Abstract

For the nascent field of advanced therapies, collaboration will be a game-changer, turning scientific progress that was once unimaginable into transformative medical practice. Despite promise for lifelong management and even cure of disease, skepticism remains about the feasibility of their delivery to patients, fueling investment risks. With the potential for long-term effectiveness in need of frequent reassessment, current approaches to predict real-life drug performance bear little relevance, necessitating novel and iterative schemes to monitoring the benefit–risk profiles throughout the life span of advanced therapies. This work explains that reinventing an adoption route for Advanced Therapy Medicinal Products is as much about the scientific and clinical components, as it is about the organizational structures, requiring an unprecedented level of interactions between stakeholders not traditionally connected; from developers and regulators, to payers, patients, and funders. By reflecting on the successes and lessons learned from the growing space of global precompetitive consortia and public–private partnerships, as well as a number of emerging accelerated development pathways, this work aims to inform the foundations for a future roadmap that can smooth the path to approval, reimbursement, and access, while delivering value to all stakeholders. Echoing the growing demands to bring these transformative products to patients, it provides critical insights to enhance our capacity in three fundamental domains: deploying the operational flexibilities offered by the growing space of collaborations, utilizing emerging flexible and accelerated pathways to tackle challenges in quantifying long-term effectiveness, and building the necessary digital and clinical infrastructure for knowledge development.

Principal Criteria for Evaluating the Quality, Safety and Efficacy of hMSC-Based Products in Clinical Practice: Current Approaches and Challenges

“…The in vitro expansion process involves substantial manipulation of hMSCs, which entails an important modification of the biological characteristics, physiological functions and structural properties of cells [28]. This stage is considered critical, since any minimum alteration could affect the intrinsic characteristics of cells [29]. Therefore, it will be crucial to control the entire process.…”

Addressing Pressing Needs in the Development of Advanced Therapies

“…However, very little has thus far been done to reliably demonstrate the bioequivalence of these bioreactor products with the manual procedure. Recent changes in the manufacturing guidelines have assisted in this critical gap in ATMP production but manufacturers have still failed to sufficiently address the critical requirements to meet increasing quality and safety concerns (Hourd et al, 2014 ; Papadaki, 2017 ).…”

“…Sometimes, as exemplified by ambiguity in the difference between senescence and precancerous stage, basic science also needs to be further advanced. Such gaps in understanding makes the development time uncertain and the process risky (Papadaki, 2017 ; Phacilitate, 2017 ).…”

“…In Japan, the restructuring of the Regulatory Agencies was coincidental with strong government support for the application to medicine of iPS and the creation of a network of universities dedicated to translational medicine. However, both areas as well as Australia and Canada, have a similar regulatory framework in place for Advanced Therapies to that of Europe, with no substantial differences (Ecorys Nederland and University Utrecht, 2016 ; Papadaki, 2017 ).…”

“…As with manufacturing, an ATMP adoption pathway strategy in establishing viable funding and stakeholder engagement routes to address the issue of reimbursement is required. Innovative partnerships with commensurate access to resources are required to formulate strategies to ease this roadblock to the clinical utilization of these therapies (Advanced Therapies Manufacturing Taskforce, 2016 ; Papadaki, 2017 ; Phacilitate, 2017 ).…”