ReviewIntractable & rare diseases research2026
Health technology assessment and medical insurance access for rare disease drugs in China: A policy review with quantitative insights from publicly available data.
Review in Intractable & rare diseases research, 2026. 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
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
To ascertain the status and propose optimization strategies for rare disease drugs (RDDs) value assessment in the scenario of the National Reimbursement Drug List (NRDL) dynamic adjustment in China, we conducted a narrative policy review that synthesized published literature and policy documents, supplemented by a secondary descriptive statistical analysis of publicly available 2022-2024 year NRDL negotiation data to contextualize recent reimbursement practices for rare disease drugs in China. This study found that value assessment of RDDs largely aligned with the traditional framework, encompassing five key dimensions: safety, efficacy, economic evaluation, innovation, and equity. Considering disease severity and the competitive landscape, innovative RDDs tend to receive higher clinical value ratings, higher willingness-to-pay thresholds, and broader policy support across the healthcare system. Between 2022 and 2024, a total of 60 RDDs applied for NRDL inclusion, with 43% successfully reimbursed. Most applicants were either original research drugs already approved overseas or modified new drugs launched domestically and abroad. Notably, 42% of the drugs had achieved global first launches before 2015, thereby accumulating extensive clinical evidence, and 58% submitted randomized controlled trial (RCT) data. The proportion of drugs supported by RCT evidence in the reimbursed group was significantly higher than the figure in the non-reimbursed group, whereas the proportion of drugs with pediatric indications were relatively lower in the reimbursed group. No significant differences were observed in other value assessment dimensions between successful and unsuccessful applicants. It is recommended that China develop detailed health technology assessment (HTA) guidelines and real-world evidence (RWE) guidance tailored for RDDs, facilitating the generation of high-quality evidence and decreasing decision-making risks associated with the value assessment of innovative RDDs.
Indexed as
Identifiers
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.