Paper short abstract:

We present a longitudinal case-study of a European policy instrument that facilitates early patient access to pharmaceuticals that address unmet medical needs. We focus on the boundary work of companies and regulators to delineate levels of evidence for market access and define unmet medical need.

Paper long abstract:

We conducted an in-depth longitudinal case-study of a European policy instrument to facilitate early patient access to pharmaceuticals that address unmet medical needs. Following institutional theory, we argue that rules to evaluate techno-scientific products always contain ambiguities. Actors will interpret, contest and act upon these ambiguities in different ways depending on their strategic interests. In line with an interest-based approach (Abraham and Davis 2013), we expect that regulators aim to protect public health and will therefore consistently reduce ambiguities in that direction, while companies have a commercial interest and thus exploit ambiguities to obtain early market access for their products.

We examine this process using a mixed qualitative-quantitative approach based on interviews, archival documents and quantitative indicators of marketing authorisation applications by companies. The various methods allow us to study how companies and regulators delineate 'sufficient levels of evidence' to gain market access and how they define 'unmet medical need'.

Our results indicate that the boundary work by regulators and companies has a regulative component (legal definitions of evaluation criteria), normative component (ordering of evaluation criteria) and cognitive component (construction of evaluation routines). The work done by actors has led to two divergent logics by which pharmaceuticals reach the market early. For some pharmaceuticals there is consensus about unmet medical need after which evidentiary standards become the subject of negotiation between regulators and companies. For others, companies present pharmaceuticals with lower levels of evidence to regulators after which unmet medical need is to be actively constructed to legitimize market access.