World Orphan Drug Congress USA - Boston 2024

Having attended several Terrapin events over the last three years as part of our ongoing ethnography of pharmaceutical conferences - speaking at the World Orphan Drug Congress (WODC) in Boston, USA held a slightly different feel. In Europe, we have covered how the World Evidence and Pricing (EPA) Congress in 2022 and 2023 in previous blog posts - both of which held a strong focus on market access, bringing together industry, patient organisations (POs), and regulators. At the WODC, the focus seemed to flow back to market access, but gravitated too towards finding solutions to core issues like the diagnostic odyssey, the pricing wrangle over orphan drugs, regulatory hurdles left to overcome. Discussion felt polemic. The shift in tone followed into the stalls too, where stands like PCMtrails selling at home testing kits for ‘decentralised clinical trials’, jostled with various real-world data and consultancy services of all kinds, through to pre-constituted survey panels, and several PO’s. Yet curiously, unlike the World EPA Congress, there were no biotech and pharma companies there (big, SME, or startup) to set up shop in the lobby. This made it it a little unclear on who exactly the Clinical Research Organisations’ (CROs’) target clients might be! Surely, CROs cannot sell simply to other CROs alone? The conference talks deviated slightly from the World EPA Congress too in their scope of discussion. Rather than a track on ‘Rare Disease’ it held one on ‘Rare Oncology’. Meanwhile, ‘Market Access’ became ‘Global Market Access’, and ‘Big Data and Digital Health’ became ‘AI and Digital Health’ - subtle but notable differences in framing.  The WODC also held a much larger poster session with space for a patient art exhibition, giving attendees time to engage with cutting-edge research, becoming far more of a site for recruitment and headhunting of future staff. 

Beyond surface appearances, the content of discussion at the event also differed from the World EPA Congress. Over the past three years, the World EPA Congress has moved from a post-pandemic concern for maintaining stability and sustainability in the market through increasing access onto a recent focus on addressing policy wrangles around how to ensure patients gain equitable access to orphan drugs (despite their high price). The WODC somehow felt more solution-focussed, with health economists and activists thrashing out new and novel potential ideas; albeit interspersed with very clear sales pitches for products and services. Within this, some sage perspectives came forward. Championing industry, within a panel worryingly titled ‘Monetising Hope’, Patroski Lawson (CEO of KPM Group DC) noted that “...about 75% of innovation comes from small companies and SMEs…”, likening them to successful family-run restaurants that fail when trying to scale-up, Patrowski added that “....we still need big pharma - they talent scout and offer buy-outs, but how can we get everybody to win?” By contrast, a counterpart panel respondent, Kevin Bagley (former Medicaid Director for the State of Nebraska, now at Strategic Consulting) raised the question that “Medicaid covers around 50% of all [US] births, so who will get less money…[if we increase cover for orphan drugs]?”, referring to an ongoing debate over resource allocation linked to the rule of rescue. Later, Bagley added that “availability is not the same as access - we need all sides to bridge the gap” beckoning collaboration and alliances to improve processes. In short, resolving access issues requires new models and levels of support. On innovation, Bagley went further, boldly stating that “Innovation is at risk! We have gone down a pathway where SMEs push innovation, but then often go out of business. Congress has prioritised innovation but failed to follow through”. Here, a careful juxtaposition between urging more state intervention and stepping back to allow free-market freedoms seemed to be at stake - but with little resolution on its conflicts; if ever a space for social science were to be opened!  

This sentiment followed in part beyond industry too, and into an ‘Advocacy support’ seminar, with patient organisations questioning “whether we are competitors or collaborators…with undue pressure [from] everybody going for the same pot [of money]” (Frank Rivera, President of Stronger Than Sarcoidosis). The latter saw Josie Godfrey (Co-founder and CEO of Realise Advocacy) - a long-standing mainstay speaker at both World EPA and WODC - and key figure for developing rare disease patient organisations, advised that “...having worked with two patient organisations where people [leaders] would not sit in the same room, it is often better to start small, and work slowly“ when building collaborations. Thus, a considered and slow approach to community-building is key. 

Meanwhile, a few speakers looked to pragmatic concerns the industry faces, in the ’Pricing and Reimbursement’ panel for example, Marshall Summar (CEO of Uncommon Cures) noted for instance that the US has only around 120 people able to run and/or conduct rare genetic clinical trials, leaving a large skill gap that brings delay to innovation - there are simply too few physicians to meet industry need. Yet few proposed solutions to many of the issues that industry faces. Elsewhere, as keynote Mark Trusheim (Strategic Director of NEWDIGS, at the Center for Biomedical System Design, Tufts Medical Center) proposed that there should be a “risk-based system where insurers’ licenses could be removed at state level if they fail to provide coverage that has been paid for” - albeit a position at odds with upcoming changes that allow Medicaid coverage for treatments out of state.    

In our own contributions, our team presented research on the value of social media data for diagnosis (Matthew Hanchard) in the ‘Diagnosis’ panel and a comparison of EU and US orphan drug submissions versus approvals and authorisations (Jin Ding). As a result, we remained largely tied to our respective panels as attendees. But within each panel, and others that we delved into, Jin and I felt that the event and much of its discussion was framed by rapidly shifting policy amidst the run-up to the next US Presidential election, but still firmly steeped in a normative understanding of policy. As such, our presentations - coming from a social science origin - required that we initially justify social science and the value of theory before making our case. As academics, this led us to address a different set of ‘So What..?’ questions; less on what can this tell us, or why is this significant, and more instead on how does this improve either profit margin or equitable access to healthcare. It will certainly be interesting to trace these debates as they evolve through further conferences in the run-up to the US presidential election, and in the same year as the UK general election and many others across the EU.