DOI: 10.15131/shef.data.29503808
title: Orphan Drugs - Dataset 3: US interviews with rare disease patients and patient organisation leaders on access and coverage for treatment

description: These 21 interview transcripts have been deposited on an open access (CC-BY-NC 4.0) basis as part of larger project titled "Exploring the emerging sociotechnical imaginaries of US patients’ access to orphan drugs". It gained ethical approval from the University of Sheffield on 23-November-2023 under reference 057267. In total, 26 semi-structured qualitative interviews were conducted for this project, all as a core activity of workstream 2 of the larger Wellcome Discovery award funded (219875/Z/19/Z) ‘Orphan drugs: high prices, access to medicines and the transformation of biopharmaceutical innovation’ project held by Professor Paul Martin. Five participants chose not to allow their datasets to be made open access.<br><br>Overall, the 26 semi-structured interviews were all conducted online, with one questionnaire using an adapted version of the interview topic guide. All were conducted between July 2024 and March 2025. Participants included rare disease patients, parents of patients, patient organisation leadership (many crossing categories), and one genetic counsellor.<br><br>Despite my best attempts at purposeful sampling, there is a gendered (as self-identified) bias in the sample, with only five males to 21 female participants. The latter reflects a known gendered division of labour in research participation within the rare disease community (Le Hénaff and Héas, 2023). Participants were located across all US regions, including both urban and rural areas, as well as both land-locked and coastal states. Participants have each had the opportunity to review and approve their own transcripts and early findings. Many challenged and extended my thoughts, leading to an iterative revision of findings, and a finished understanding that represents consensus across accounts.<br><br>My three-stage applied thematic analysis (Guest et al, 2014) of verbatim interview transcripts was partly informed by a four-year ethnography of pharmaceutical and ethnographic conferences undertaken as part of the Wellcome project's workstream 2 activity, alongside a meta-narrative literature review of orphan drug pricing debates (Hanchard, 2025a), and a large-scale digital methods analysis of Facebook posts on the mobilisation of lay therapeutic expertise in discussion of rare disease determinants (Hanchard, 2025b). Past experience of similar work undertaken in the UK for a project on rare disease patients’ perceptions of access to treatment helped shape my initial thoughts on policy barriers too.On positionality, I entered the interviews with the intention of asking participants to connect their access/coverage for treatments with national economic and health policy. I planned to do so by a drawing on Jasanoff's (2015) notion of sociotechnical imaginaries. As the analysis continued, my coding shifted and the data lead me towards Bourdieu's three forms of capital instead, and by extension to cultural health capital alongside sociologies of lay expertise.As a background context, the interviews took place before, during, and after the US election where the Biden administration conceded to Trump's second term as US president. They came in the wake of the Inflation Reduction Act (2022) and prior to JD Vance's large-scale cuts to US medical regulatory agencies.The dataset contains 21 verbatim transcripts in .docx format, a list of qualitative codes generated in .docx (accessible in various qualitative data analysis software (such as QSR NVivo), and Participant face-sheet setting out detail on the transcripts. The latter includes dates on time and date of interview, rare disease severity and grouping, and the participation's organisation type, geographical remit, and their role within it.<b>References</b>Guest, G., Macqueen, K. M. and Namey, E. E. (2014) ‘Planning and Preparing the Analysis In: Applied Thematic Analysis Learning Objectives’, in Applied Thematic Analysis. SAGE Publications, Inc., pp. 21–48. doi: 10.4135/9781483384436.Hanchard, M. (2025a) Debates over orphan drug pricing: a meta-narrative literature review. <i>Orphanet: Journal of rare disease</i>, 20(107). TBC. doi: https://doi.org/10.1186/s13023-025-03634-2 7Hanchard, M. (In preparation/2025b). The framing of amyotrophic lateral sclerosis (ALS) treatments on Facebook. Social Science & Medicine, TBC(TBC). TBC. doi: TBC.Jasanoff, S. (2015) ‘Future imperfect: Science Technology, and the Imaginations of modernity’, in Jasanoff, S. and Kim, S.-H. (eds) Dreamscapes of modernity: Sociotechnical imaginaries and the fabrication of power. London: University of Chicago Press.Le Hénaff, Y. and Héas, S. (2023). ‘Gender and care in the context of rare diseases: exploring nuances of emotional support and the division of household labor’. Health Sociology Review, 32 (3). pp. 357-371. doi: 10.1080/14461242.2023.2199729