Social pharmaceutical innovations (SPINs) in an era of orphanisation

We first met with the social pharmaceutical innovation (SPIN) team at the École Nationale Supérieure des Mines (Paris School of Mines - PSL University) in May 2022, and have been working to establish collaboration with them ever since. Our project examines what the process of orphanisation means for pharmaceutical access and pricing; how markets, industry parties, rare disease patients, and many others are adapting. In compliment, the SPIN project takes an approach equally steeped within social science to examine medical product innovation cycles and potential to change them via various SPINs i.e., by including a broader range of stakeholders and inclusion of societal value within pharmaceutical development and marketing. Overlap between our two bodies of work can be seen in the mutual generation of a global pharmaceutical studies and society network (GPSS), which will continue far beyond the funding lives of either project. With that in mind, we attended the SPIN final conference ( 09-Mar-2023 and 10-Mar-2023). There, we were brought together a variety of like-minded clinicians, health economists, medical regulators, and sociologists for three days in Utrecht's aptly named Social Impact Factory. The conference followed a call for collaboration in the SPIN team's latest position paper on Orphanet and revolved largely around an extended discussion of themes within the SPIN project findings. In responding to the call, attendees had journeyed from Brazil, Canada, and almost all corners of Europe to attend, lending the event a real sense of purpose and momentum. Whilst it the event marked the end of the SPIN project's Trans-Atlantic Platform (T-AP) funding, it felt more a tentative step along a much longer path, and highlighted both an appetite and need to fully understand the range of changes currently taking place within the pharmaceutical industry, such as the increasing cost of many treatments (especially for rare disease) and how innovation could and ought to be adjusted to foster more SPINs. Working to do so, we later saw a few attendees at both the (Re)Act Congress in Berlin a week later, and the World EPA Congress a week after that.   

Throughout the event we saw: clinicians and pharmacists question why hospitals were not developing and/or formulating more treatments in-house, or making more use of drug repurposing (a process of finding alternative uses for approved and/or investigational medicines). Also on what sort of regulatory mechanisms and support they would need to do so; others called for international collaboration, both to abate the high prices generated separately within regional and national markets, and as a form of Increased knowledge exchange; elsewhere, discussion over the use of clinician time spent on gathering real-world data (RWD) led to debate over who should hold responsibility to gather, process, and own it. Here, RWD was marked as a potential tool for ensuring the patient voice could be included within the development and pricing of medical treatments in a more meaningful ways; a further debate on how best to approach value saw value-based pricing become a key point of discussion, with both positive and negative attributes put forward. Around the topic, qualitative life-adjusted years (QUALY) and performance-based models were compared with more utility focussed models, with a loose consensus formed that fairer pricing is needed, and that international collaboration could be a way forward. Overall, the conference ended with an understanding that for SPINs to become embedded, there need to be far more inclusion of patient groups/organisations, clinicians, and a wider body of industry partners alongside academics. For our project, the main take-away point was that in an era of orphanisation, where the prices of rare disease treatments are rapidly increasing, collaboration amongst various stakeholders Is vital. This would mostly likely take place across countries and could open new approaches to valuation, with the possibility of various SPINs bringing greater focus on social value. To do so, however, will require a great deal of work from all stakeholders involved.