Abstract
Advanced therapy medicinal products (ATMPs) are a group of innovative and complex biological products for human use that comprises somatic cell therapy medicinal products, tissue engineered products, gene therapy medicinal products, and the so-called combined ATMPs that consist of one of the previous three categories combined with one or more medical devices. During the last few years, the development of ATMPs for the treatment of eye diseases has become a fast-growing field as it offers the potential to find novel therapeutic approaches for treating pathologies that today have no cure or are just subjected to symptomatic treatments. Therefore, it is important for all professionals working in this field to be familiar with the regulatory principles associated with these types of innovative products. In this review, we outline the legal framework that regulates the development of ATMPs in the European Union and other international jurisdictions, and the criteria that each type of ATMP must meet to be classified as such. To illustrate each legal definition, ATMPs that have already completed the research and development stages and that are currently used for the treatment of eye diseases are presented as examples.
Highlights
Advanced therapy medicinal products (ATMPs) are a large and diverse group of therapeutic agents for human use
Because of the complexity and novelty of these innovative products, the regulatory procedures have the potential to be excessively rigid and complex, creating new challenges to both developers and regulators. These challenges can be especially significant for small ATMP developers that often have limited budgets and regulatory expertise and for whom a deep understanding and compliance with ATMP regulations can be difficult
Developers normally feel overwhelmed with the regulatory requirements, because they increase both the financial and administrative burden and hamper the market access of ATMP products
Summary
Advanced therapy medicinal products (ATMPs) are a large and diverse group of therapeutic agents for human use. As with other existing and often less-complex medicinal products, ATMPs must meet the same high standards for scientific, methodological, and regulatory requirements: (1) the safety and efficacy must be demonstrated through both preclinical studies and human clinical trials. The anatomical structure is compartmentalized, limiting the distribution of medicinal product to non-target tissues It has good accessibility for applying treatments and examining outcomes. During the last few years, the development of ATMPs for the treatment of eye diseases has become a fast-growing field It is important for all professionals working in this field to be familiarized with the regulatory principles associated with these types of innovative products. We identify and describe the ATMPs for the eye that have completed the research and development stages and are currently being used for the treatment of ocular diseases
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