Advanced therapy medicinal products (ATMP) offer hope for health benefits in all situations where traditional methods of therapy fail or cannot be used for various reasons. The main purpose of this article is to analyze the concept of innovation as applied to the biotechnologies employed in ATMP. In the analysis of the concept, five main contexts of meaning that contribute to its understanding will be distinguished: a change in the way of thinking about the available (...) spectrum of medical procedures, the short time of recognition of technologies in the experimental and theoretical models, lack of clinical data in advance of used technologies, increase of technologies’ complexity and diversity of substrates used in them. The conducted analysis indicates the need to adopt an attitude of far-reaching caution in the evaluation of the safety of ATMP therapies in which innovative biotechnologies are used. (shrink)

The following text is a voice in the discussion around normative problems of innovative therapies. It particularly refers to the meaning of the concept of innovation, also discussed in this issue in the article by Tomasz Rzepiński "Advanced therapy medicinal products (ATMP) as products of innovative biotechnologies.".

The need to better balance the promotion of scientific and technological innovation with risk management for consumer protection has inspired several recent reforms attempting to make regulations more flexible and adaptive. The pharmaceutical sector has a long, established regulatory tradition, as well as a long history of controversies around how to balance incentives for needed therapeutic innovations and protecting patient safety. The emergence of disruptive biotechnologies has provided the occasion for regulatory innovation in this sector. This article investigates the regulation (...) of advanced biotherapeutics in the European Union and shows that it presents several defining features of an adaptive regulation regime, notably institutionalized processes of planned adaptation that allow regulators to gather, generate, and mobilize new scientific and risk evidence about innovative products. However, our in-depth case analysis highlights that more attention needs to be paid to the consequences of the introduction of adaptive regulations, especially for critical stakeholders involved in this new regulatory ecosystem, the capacity and resource requirements placed on them to adapt, and the new tradeoffs they face. In addition, our analysis highlights a deficit in how we currently evaluate the performance and public value proposition of adaptive regulations vis-à-vis their stated goals and objectives. (shrink)