ReviewTherapeutic innovation & regulatory science2025
Insights into Support Systems for Orphan Drug Development: A Comparative Study.
Review in Therapeutic innovation & regulatory science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
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Authors and funding
9 authors.
Funding
Abstract
Due to great unmet medical needs, orphan drug development is a common issue of high priority for policymakers, industry leaders, researchers and patients worldwide. The establishment of a harmonized support system is the way forward to address the dilemma facing rare diseases (RDs). This study pioneers the proposal of a framework and dimensions of support systems affecting orphan drug development, covering legal (political and legal basis), technical (regulatory guidance and acceleration), motivational (economic incentives and innovation returns) and logistical (fundamental infrastructure) factors. This study, based on the framework, shows that the conditions for orphan drug development above are basically developed in the US, the EU and China. Additionally, China lags behind in terms of economic incentives and fundamental infrastructure. Continuous improvements in pricing and reimbursement, as well as the acceleration of real-world data (RWD) database and biobank repository integration, are expected in China. Based on these findings from the three study regions, action plans with three strategies (national plans and strategies, a patient-centered health system, global governance and collaborations) and eleven actions are suggested for strengthening synergies between initiatives and stakeholders to satisfy the medical needs of RD patients and families. This study can provide a reference not only for orphan drug development in the three study regions but also for all other countries worldwide, especially for those with a late start in addressing RDs.
Indexed as
Identifiers
40658166What 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.