Promoting Equitable and Affordable Patient Access to Safe and Effective Innovations in Donation and Transplantation of Substances of Human Origin and Derived Therapies.

Abstract

Innovation is a hallmark of organ, tissue, and cell transplantation. The development of new treatments derived from these substances of human origin (SoHO) has rapidly evolved in recent years. Despite the great benefits that these innovative therapies could bring to patients, significant difficulties have arisen in making them equitably and widely accessible. Herein, we identify and address 4 challenges to promote innovation in this field in a collaborative, sustainable, and transparent manner and propose some concrete solutions applicable to SoHO-derived treatments, ranging from cell therapies to solid organ transplantation. Regulators, health policymakers, and government officials are recommended to incorporate specific elements into the regulatory frameworks of their respective jurisdictions, although regulatory convergence and equivalent quality and safety standards applicable to SoHO at a global level would be needed. An innovation-driven regulatory environment, respectful with the human origin and in accordance with the altruistic donation of SoHO, should be encouraged to improve the safety, effectiveness, accessibility, and affordability of SoHO and to promote collaboration between countries and between public and private sectors. This overview is the outcome of a working group focused on "Innovation in the donation and clinical application of SoHO" as part of the international Summit "Towards Global Convergence in Transplantation: Sufficiency, Transparency and Oversight" convened by the Organización Nacional de Trasplantes under the Spanish Presidency of the Council of the European Union in November 2023 and cosponsored by the Council of Europe, the World Health Organization, the Transplantation Society, and the European Society for Organ Transplantation.