RESUMO
Public-Private Partnerships (PPPs) have been crucial in medicine research and development (R&D) for decades. Initially, PPPs involved private and academic innovators working in bilateral collaborations to advance pharmaceutical innovation. Later, a precompetitive open innovation environment was created, where multiple public and private innovators collaborated on mutual interests. The entry of regulators and patient interest organizations into PPPs has triggered a third shift from an innovator result-driven to a multi-actor impact-oriented partnership model. Using the second Innovative Medicines Initiative program (IMI2) as an example, this chapter focuses on the increasing roles of patient interest organizations in PPPs in roughly the last decade.Most IMI2 partnerships focused on raising awareness and sharing information tailored to patient needs (listener role) and inviting patients to share their experiences and needs (co-thinker role). Many partnerships also integrated the patient perspective by implementing patient advisory bodies (advisor role) or including patients as equal partners in steering the project (partner role). Notably, partnerships like EUPATI and PARADIGM showed that patient interest organizations can lead initiatives, especially those aiming at advancing patient engagement across the medicine R&D lifecycle (decision-maker role). While the overall impact of patient involvement in the IMI2 program is still being assessed, it has exposed many innovators and regulators to the patient perspective and created a community of patient experts with access to tools and guidelines for meaningful involvement.The PPP model continues to evolve, shifting from a treatment-only to a comprehensive diagnosis, treatment, and monitoring approach by incorporating digital and medical technology actors. This development, alongside continued patient and public integration could revolutionize the R&D and accessibility of new treatments and diagnostics.
RESUMO
"Non-biological complex drugs" (NBCDs), such as liposomal formulations, iron-carbohydrate complexes and glatiramoids, gained increased interest from a regulatory perspective in recent years. Similar to biologics, the quality of NBCD products is highly dependent on a robust and well-controlled manufacturing process. This provides challenges for generic drug developers to replicate NBCD products once market exclusivity of the originator product is expired. However, unlike biologics for which a consistent regulatory framework was established with the biosimilars pathway, NBCDs are not recognised as a distinct category of medicines and hence no formal regulatory pathway for their approval is defined. Currently, a "case-by-case" approach is applied for regulating NBCD follow-on products in the EU. Furthermore, NBCDs can follow a non-centralised authorisation procedure, leaving regulatory approvals to national competent authorities. This can lead to heterogeneity in the regulatory approach and outcomes when assessing NBCD follow-on products throughout the EU, which for some product classes has already resulted in some safety and efficacy implications. Here, we explore the regulatory landscape of NBCDs and their follow on products. This study shows that almost all of the 85 NBCD follow-on products available in the EU in 2018 have been approved via various non-centralised procedures. Although most NBCD follow-on products followed an Article 10(1) procedure, we clearly see a recent increase of the use of the hybrid pathway via Article 10(3). This study shows the heterogeneity in the regulatory approach taken for many NBCD follow on products. To what extent this may have consequences for their safety and efficacy evaluations is unknown and needs to be further investigated. The present study should stimulate the rethinking to design prudent regulatory pathways for NBCD follow-on products.