SynthesisFrontiers in medicine2025
Societal participation in the development of orphan drugs: a systematic review.
Synthesis in Frontiers in medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
1 citing paper in PubMed.
- Orphan medical devices: addressing the regulatory and access gaps in the EU and US.Frontiers in public health · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The development of orphan drugs (ODs) remains constrained by structural barriers, including limited scientific knowledge, high clinical uncertainty, and fragile commercial incentives. In parallel, an alternative ecosystem has emerged in which non-commercial societal actors (patients, advocacy groups, and philanthropic entities) contribute to the design, evaluation, and dissemination of therapies for rare diseases. This systematic review provides the first structured synthesis of empirical evidence on societal participation in OD development, with a specific focus on neuromuscular and neurodegenerative disorders. Twenty-one peer-reviewed studies were included, spanning a range of designs from case reports to stakeholder surveys. A functional typology of societal roles was developed, identifying four primary modalities
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What OpenQuestion holds
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 OpenQuestion graph.