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Regulatory blind spots in cellular therapy translation experience from Australia

      Regulators are in a conundrum. While reluctant to approve novel cellular therapies due to the lack of substantive clinical evidence, regulators are nonetheless motivated to expedite approvals because the negative effects of delaying a potential cure is perceived to be more harmful than the adverse effects of the product itself. In the face of mounting pressures for curative treatments for terminal and debilitating diseases, clinical trials for cellular therapies proceed on humans even though the mechanisms behind those effects are not fully understood (i.e. therapeutic effects are based on preclinical animal models). At the time of writing, FDA has only approved cord blood derived haematopoietic stem cell therapies for a narrow spectrum of applications (e.g. some blood cancers such as leukaemia). Regulators have in general subjected emerging cell therapies to the same clinical governance framework as that for small molecule drugs and biologics. While this is applicable to most cell therapies, we have concerns that high-risk autologous applications are falling outside existing regulation, e.g. being labelled a ‘non-therapeutic good’ and thus not subjected to regulation. If the future for cellular therapies (allogeneic/autologous) is to provide for efficacious solutions that engenders public support, not having sufficient regulation to capture autologous applications could curtail the development of the industry on a number of fronts (e.g. inability regulate practice, false advertising and deception, limited scientific progress, limited collection & analysis of patient outcomes, patient protection, health system reimbursement). By reviewing regulatory profiles of a range of industries and adopting a case study approach, our investigation argues to the benefits for a distinctive regulatory framework for autologous therapies. We argue for a regulatory stance that would satisfy the efficacy, safety and governance mission of regulators, while providing for rapid translation of science to patient treatment.
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