ArticleExploratory research in clinical and social pharmacy2023
A qualitative study on the views of experts on the social impact of the high-priced orphan drug nusinersen.
Article in Exploratory research in clinical and social pharmacy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
3 citing papers in PubMed, 1 synthesis or guideline pooled it, 4 citations in OpenAlex.
- Bioethics at the intersection of politics, society, and healthcare: the significance of media debate analyses.BMC medical ethics · 2025Pooled it
- Implicit ethical choices in ultra-rare therapy development: researchers' perspectives on patient selection and communication, therapy value, and accessibility.BMC medical ethics · 2026Article
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Escalating medical costs due to the increasing occurrence of high-priced orphan drugs is a topic discussed in the media and specialist literature. However, there is no study investigating the social impact of such drugs through the views of experts. Objectives: The aim was to demonstrate the social impact of the orphan drug nusinersen based on the views of experts within the community. Methods: The study was conducted using two methods for data collection: a media analysis and qualitative semi-structured interviews. In the media analysis, expert comments on nusinersen were extracted from the Finnish media. Interviews were conducted with experts from the fields of pharmacy, medicine, politics/academia, law/economics, hospital management and patient organisations from different parts of Finland, who encountered nusinersen in their profession. Participants were recruited through purposive and snowball sampling. Interviews were audio-recorded, transcribed verbatim, and the overall data were analysed thematically. Results: Twenty-nine media references were collected, and 16 interviews conducted. Three main themes were identified: ethical aspects, financial aspects, and call for new strategies. Expert views were divided between the ethical and financial aspects of nusinersen. These existed alongside each other, showing that different attitudes and values compete with each other, and may be classified in different ways depending on the situation. However, the discussion quickly evolved into a call for new strategies in order to find solutions to issues concerning orphan drugs and the social impact created as a result. Conclusions: This study reveals the social impact of nusinersen thus far within the community and it appears somewhat different when seen from the perspectives of patients and decision-makers. Even though impact has been created, such as the establishment of a disease-specific patient organisation, other issues still require further research. Among these are the potential establishment of international collaboration forums for price negotiations with pharmaceutical companies.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.