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Although the three-day event focussed on developing new and novel approaches to expanding market access from an industry perspective, the offer of free attendance/registration for academics was greatly appreciated. It felt as though a corner had been turned, where social scientists were to be openly engaged with a broad array of concerns - no longer seen as critical opposition, but as balanced contributors both to industry practices and public knowledge. Walking around the luxurious RAI convention centre, we saw various stands in the lobby, each vying for attendees’ business, each looking to sell their latest software, offer consultancy services, or provide intermediary brokerage - often for real-world data. As might be expected from the , this lent a strong commercial air to the event. However, beneath the surface lay some deeply substantive debates, and care for patients; it was not all about profit margins and sales. The congress plenary session, for instance, opened at 8.55am sharp on 21-Mar-2023 with Ana Plata (AstraZeneca’s Global Head of Pricing Implementation) setting equity as a key term for the coming days. Following on, Christoph Glaestzer (Chief Global Value and Access Officer for Janssen Pharmaceutical’s Johnson & Johnson) posited that “market access is not a function, it is a strategy”, seemingly holding patients as end-points in the process. He added that “intent and action divide at the point of decision-making” before moving on to say that the patient voice is only being heard in some facets; little or no patent input goes into decisions about delisting, changes in coverage, on clinical guidelines, or on prescription decisions. What Glaestzer added to the event then, was a steer towards greater inclusion of the patient voice across all facets of healthcare. The final opening plenary speaker, Alberto Briones (Senior Consultant for event sponsor Lifescience Dynamics), introduced the notion of framing archetypes of market dynamics, and warned of potential for aggressive discontent over pricing when poorly managed. For the EU market, Briones noted how archetyping (something akin to Goffman’s media frame analysis) could help build better strategies for market access. These archetypes clustered around high to low competition and pricing pressures on one axis and national decision-making approaches on the other. The theme he suggested for the congress to take forward was clearly about understanding the nuance of different healthcare markets and adapting accordingly. With so much covered in the opening plenary, by 9.40am the event was set up early to expand on equity, patient voice, and adaptation to nuanced markets with an overall focus on market access.

The event was then split into various tracks, with our team staying primarily within the ‘Rare Disease’ one. In venturing out to the ‘Big Data and Digital Health’ track for half a day, we found that the industry itself seems ill-prepared for many changes taking place. In one talk, for instance, Fausto Artico (GSK’s Director of Innovation and Data Science) a double-doctorate in Information Engineering and Computer Science no less, remarked that: “pharma is a very conservative environment…” and that with digital technologies it would do well to “...try new things and de-risk”. In a statement aimed at potential startups, Artico went on to note that proposals from small agile teams were often more successful when their product met a very niche market and had built a relevant community. His main takeaway; that you can always be more niche - a prescient reminder that we are now entering an era of personalised medicine. However, Artico soon added that the model for reimbursement over new digital medical technologies was unclear at times. Echoing this sentiment, Zsombor Zrubka (Associate Professor in Health Economics at Obuda University) noted that on RWE there is still “global work needed to standardise” both in terms of processes and data standards. As an example, Zrubka noted that there is ‘no standard sample of rare disease paediatric diabetes [patients]; how can we train AI on the original sample if it is not representative?”, opening key questions about how different sets of actors might collaborate to do so. Seen through a sociological lens, unpacking the sets of alliances (each laden with power relations) come to mind as a key topic here, to better understand data power in the development of medicine. As a separate point, Zrubka went on to suggest that “expensive products could be expanded to [greater] use through gamification of habit-based costs”, again reinformeding the view that healthcare is undergoing a move towards personalisation. His viewpoint suggested that healthcare appears to be moving towards an ego-centric ontology, where individuated patients and healthcare systems are held to interact in duality without consideration of either a societal midlayer, or of any dialectic between agency and structure. In the same track, a more material limitation was highlighted when Jesper Bjerggren (Teva Pharmaceuticals’ European Market Access Lead for Digital Health & Respiratory) remarked that it was still unclear, in regulatory and terms and within many businesses what to do when “a new iOS version or a new feature [of a product] is released.  Is a new compliance [assessment] needed? I don’t know!” And so, while the track highlighted a clear need for regulatory guidance over the development and updating of digital technologies, it also pointed at the increasing personalisation of medicine as a key driver for change, and with it and the need to engage with patients across the entire drug development and distribution cycle.

Our main interest, however, was in the ‘Rare Disease’ track, where discussions about digital technologies and/or big data rarely featured. Instead, we saw a plethora of talks that each spoke the opening plenary themes in different ways. Early on in the track, a health economics discussion saw incremental cost-effectiveness ratios (ICERs) lamented by Philippe Ghyssels (Vice president of Corporate and Global Public Affairs at Ipsen), both for their culmination in health rationing (and thus inequity of access) and - in a French context - for having been set in 2004 without accounting for inflation. Others sought business buy-in, for example Lavni Varyani (a founding partner of Pharma Business Partners) beckoned pharmaceutical companies to invest in Dubai as part of the Saudi Vision 2030; there, an increased focus has been placed on genomics and for ATMPs in particular, marking a burgeoning turn towards genomics and personalisation of medicine across the Middle East. Other talks revolved around patient advocacy, urging clinicians to work with patients, and not ‘on them’ as abjectifed, objectied, or instrumental bodies…”; and instead to do as Josie Godfrey (Co-Founder and CEO of Realise Advocacy) says, and “meet patients where they are” -  statements prime for any epistemologist or ethicist to dig in to. Elsewhere, Godfrey went on to argue that “natural history would not look the same without patient input”, bring the fore the value of including the patient voice throughout all stages of the treatments development and delivery process. Elsewhere, patient advocates offered less systematic talks, and instead focussed on the particular, and its relation to access barriers. As one example, Juliette Vila Sinclair Spence (Founder & Chairman [chairperson] at Acanthamoeba Keratitis Eye Foundation) drew on her life-altering personal experience of developing acanthamoeba keratitis (AK) from a parasitic infection caused by swimming pool water being caught behind her contact lens. As a young disease with no licensed treatment, Spence discussed the lengthy journey of gaining diagnosis and then of gaining access to treatment. In a neat conference curatorial manoeuvre, her talk was quickly followed by Luca Tofani (SIFI spa’s Director of Pricing and Market Access) who presented a new treatment with an 86.7% cure rate for AK at phase III clinical trials. Rather than going straight from evocative testimony into a sales pitch, Tofani offered a discussion about some of the practical issues around early access programmes for ultra-rare acute diseases when they need urgent treatment. Here, he added that while there are generics, they can take between 14 and 51 days to access. For AK patients, that can mean months without being able to move, speak, see, and constant pain. Meanwhile, physical logistics could allow medicines to be accessed within two days. In referring to SIFI spa’s own treatment for AK, polihexanide (PHMB), Tofani noted that because the medicine was produced in Italy, import regulations mean that it has to be taken outside the county and then bought back from an external country for Italian patients to access it. As such, his talk echoed many others, from both the ‘Big Data and Digital Health’ and ‘Rare Disease’ tracks, in pressing for regulatory reform - either to speed up the approval process, to put mechanisms in place for urgent care needs, and for greater clarity over the compliance requirements for digital medical products.      

Overall, the congress marked a sharp change in tone from the previous year. The new building and revised set of amenities certainly provided a more corporate feel, but more than that, it seemed that industry had moved away from an overarching push to appease investors by shoring up their books. The previous post-pandemic push for sustainability and stability in the healthcare market had given way to one on examining how change can take place, what forms of innovation are needed, and the barriers faced both in increasing equity (health justice) and finding meaningful ways to include patients. Beneath it all lay an unspoken assumption that we are moving towards personalisation. What we took away from the event as a social science project was a firm commitment to: understanding how these actors are reformulating their relations with one another, and on mapping those relations as they shift; as well as understanding how pharmaceutical prices (orphan drugs in particular) and approaches to valuation are changing; and how patients narratives feed into these sets of changes in differing ways. As an ongoing engagement, Matthew Hanchard has now been invited to speak at EPA Congress 2024 in Amsterdam, where he will present some of the drug orphanisation project work on the three narratives of orphan drug pricing and a call for regulatory reform.