Short abstract:

Using a qualitative event history analysis, we study how we can understand the transformative nature of initiatives in the gene and cell therapy field which challenge traditional regulatory frameworks, access conditions and business models, as well as associated value orientations of actors.

Long abstract:

Technological advancements in understanding and modifying the human genome have enabled the emergence of the gene and cell therapies (GCTs) field. GCTs hold the potential to treat rare genetic diseases, cancers and infections, many of which were previously hardly treatable or untreatable. Fairly early on in the emergence of the field, gene therapies started to be regulated as pharmaceutical products. Consequently, gene therapies have been developed within the traditional linear regulatory and access frameworks based on traditional business models in the sector. However, these traditional models are failing to deliver the quantity of therapies needed for rare genetic diseases, as well as access to the ones that are developed. Initiatives have emerged in the GCT field which aim to challenge traditional regulatory frameworks, access conditions and business models. Examples include patent opposition filed by NGOs against the patent of gene therapy Kymriah or academic hospitals using the hospital exemption in direct competition with a licensed product. These initiatives often involve public actors (academia, hospitals, civil society) and reflect public values (not-for-profit, transparency, equity). They create frictions with dominant regulatory and access frameworks that reflect the values of the traditional pharmaceutical model (for-profit, IP protection, economies of scale). In this paper we study how we can understand the transformative nature of these initiatives and associated value orientations of actors. We identify initiatives based on a qualitative event history analysis of the GCT field and specifically examine how these initiatives can be transformative.

co-author: Lourens Bloem (Utrecht Institute for Pharmaceutical Sciences)