Increasing costs of drugs for rare diseases have raised concerns about health systems’ sustainability and equitable access to these therapies. Newer and highly effective orphan drugs, such as Chimeric Antigen Receptor T-Cell (CAR-T) therapies, highlight the need for alternative innovation models that can offer greater affordability and accessibility. This case study examines Hospital Clínic Barcelona’s (HCB) alternative innovation model to develop ARI-0001 (varnimcabtagene autoleucel), a novel CAR-T therapy for certain forms of leukemia, at a price two-thirds lower than comparable therapies from the pharmaceutical industry. We conducted background research, and performed twenty-one semi-structured interviews with HCB staff, representatives of regional Health Departments, the Spanish Ministry of Health and regulatory agency, academics, civil society and patient groups. We used a framework drawing on concepts from complex adaptive systems, incorporating the resources used (i.e., funding, knowledge, and manufacturing capacity), the practices implemented (i.e., knowledge management, access practices and transparency), and the rules and norms that shaped HCB’s interactions with other actors in the system. We then identified the concept of institutional logics as well-suited to explain how HCB’s model overcame various barriers. HCB’s model builds from three competing institutional logics – healthcare, academic and industrial – while also addressing each logic’s weaknesses. The healthcare logic shaped HCB’s affordable pricing of ARI-0001, but also posed organizational challenges. The academic logic drove HCB’s willingness to share knowledge and technology with peers, but posed barriers to fund late-stage clinical trials and regulatory processes. Lastly, HCB adopted an industrial logic by identifying in-house regulatory expertise and developing partnerships for production. The hospital exemption clause, a European regulatory pathway that allows hospitals to develop and produce treatments under certain conditions, allowed HCB to span the boundaries of the three competing logics. Our findings underscore the potential of academic hospitals to develop more affordable advanced therapies, the importance of conducive regulatory frameworks, and the challenges that academic hospitals face to expand this model. Through increased regulatory and financial support to academic hospitals, strengthened coordination, and public funding with requirements for access, this model could achieve innovation with affordability in a cutting-edge technological area.
Ruiz et al. (Fri,) studied this question.