ReviewPharmacoEconomics2025
HTA Evidence in Rare Diseases: Just Rare or Also Special?
Review in PharmacoEconomics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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
8 citing papers in PubMed.
- Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases.Applied health economics and health policy · 2026Review
- Comparing National Health Technology Assessment approaches and the new European Joint Clinical Assessment framework: The case of onco-hematological medicines.European journal of clinical pharmacology · 2026Article
- Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies.Journal of market access & health policy · 2026Article
- Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations.Journal of market access & health policy · 2026Article
- The Importance of Structured Expert Elicitation to Inform Outcomes Following Treatment Discontinuation: Evidence Assessment Group Perspective.PharmacoEconomics · 2026Article
- Health technology assessment and medical insurance access for rare disease drugs in China: A policy review with quantitative insights from publicly available data.Intractable & rare diseases research · 2026Review
- Five-year Incidence Trends and the Status of Major Rare Diseases in 2023 in the 「Annual Report on the Rare Disease Patients in Korea」.Public health weekly report · 2026Article
- The PBC Ireland patient registry: study protocol for a national platform on primary biliary cholangitis.Therapeutic advances in rare diseaseArticle
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.
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.