Abstract
Advanced Therapy Medicinal Products (ATMPs) comprising cell, gene, and tissue-engineered therapies have demonstrated enormous therapeutic benefits. However, their development is complex to be managed efficiently within currently existing regulatory frameworks. Legislation and regulation requirements for ATMPs must strike a balance between the patient safety while promoting innovations to optimize exploitation of these novel therapeutics. This paradox highlights the importance of on-going dynamic dialogue between all stakeholders and regulatory science to facilitate the development of pragmatic ATMP regulatory guidelines.
Highlights
Navigating an Investigational Medicinal Product (IMP) through the regulatory maze to the clinic is time-consuming and expensive, with many stakeholders involved
The regulatory agencies, national competent authority (NCA) and the developers face a quandary in how to achieve balance between flexibility while aiming to provide clarity. We suggest this impasse could be circumvented by intensive interactive discussions throughout e.g., the early development process involving experts during the clinical trial application (CTA) review process
Heterogeneous Advanced Therapy Medicinal Products (ATMPs) continue to evolve at a rapid pace, providing options for unmet clinical needs
Summary
Navigating an Investigational Medicinal Product (IMP) through the regulatory maze to the clinic is time-consuming and expensive, with many stakeholders involved. In consultation with the stakeholders, EMA refined their strategy and included a number of recommendations in the “Regulatory Science to 2025 strategy” [37] This comprises (a) establishing a multi-stakeholder forum to foster innovation in clinical trials; (b) re-enforcing relevance of patients for evidence generation; (c) promoting the use of real-world data and big data in decision-making processes; (d) providing a feasible legislative framework as well; (e) contributing to a better HTAs’ preparedness and decision-making at national levels to foster innovative medicines development while enhancing translational dialogue with payers to enhance accessibility. The advantages of translational hubs is exemplified by the RESTORE and ReSHAPE consortia [45, 46], which led to the development and translation of ground-breaking cellular therapies, including T cells, to modulate the immune systems in living donor transplant recipients enabling reduction of dependency upon toxic immunosuppressive drugs [47, 48] This required development of new GMP compliant procedures through frequent discussions in how current regulatory guidelines should be applied and adapted where necessary. The most important criteria for selecting a clinical trial site and country are the expertise of the health care professionals, quality of review and the expertise of regulatory authorities [50]
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