Evidence map›Paper›PMID 41744015›Full record

ReviewIntractable & rare diseases research2026

Progress in the research and pharmacoeconomic evaluation of drugs and devices for rare diseases in China.

Shanlian Hu

Abstract readReview
In one paragraph

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.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

1 author.

Shanlian HuDepartment of Health Economics, School of Public Health, Fudan University, Shanghai, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The development, importation, and reimbursement of drugs and medical devices for rare diseases have become critical issues within China's healthcare system. Since 2018, China has issued two national Rare Disease Lists, covering 207 diseases. As of December 2025, 223 drugs for rare diseases have been marketed domestically, with 136 (61.0%) included in the national list for reimbursement by basic medical insurance scheme. Advances have also been made in diagnostic technologies and treatment equipment. This article also examines the issues with and factors influencing the pharmacoeconomic evaluation of rare disease therapies. Additionally, over 100 registered patient organizations contribute substantially to care, education, research, and advocacy. China has piloted multi-level healthcare security system, including national and local healthcare security systems. The introduction of a list of innovative drugs covered by commercial insurance in 2025 further supplements this system. These measures have collectively expanded reimbursement coverage. Despite progress in drug development, insurance coverage, and evaluation of drugs in terms of health economics, continued efforts are needed to enhance treatment accessibility and equity. Key measures include putting forward rare disease legislation, promoting research on health technology assessment, improving health utility measurement, encouraging domestic orphan drug development, and strengthening international collaboration. China's experience offers valuable insights for global rare disease prevention and treatment initiatives.

Indexed as

orphan drugspharmaceutical policypharmacoeconomic evaluationrare disease medical devicesrare diseases

Identifiers

PMID41744015
PMCPMC12932010

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.