ReviewRMD open2018
Rare diseases under different levels of economic analysis: current activities, challenges and perspectives.
Review in RMD open, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 3 of them syntheses that pooled 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.
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
28 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Institutional Priority-Setting for Novel Drugs and Therapeutics: A Qualitative Systematic Review.International journal of health policy and management · 2024Pooled it
- Spectrum of Genetic Diseases in Tunisia: Current Situation and Main Milestones Achieved.Genes · 2021Pooled it
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- SMART Planning of Early-Stage Health Economic Decision Models for Mechanism-Based Precision Treatments in Rare Cancers: An Application and Tool Update.PharmacoEconomics · 2026Review
- European Reference Networks - a flagship activity of the EU in the field of rare and complex diseases: from 2017 to 2025.Orphanet journal of rare diseases · 2026Review
- The organizational dimension in rare and complex diseases care management: an application of RarERN PathBMC health services research · 2025Article
- Challenges in evaluation and reimbursement of drugs for rare diseases in Saudi Arabia: a Delphi expert consensus.Journal of pharmaceutical policy and practice · 2025Article
- The impact of rare diseases on the quality of life in paediatric patients: current status.Frontiers in public health · 2025Review
- Development of an multicriteria decision analysis framework for rare disease reimbursement prioritization in Malaysia.International journal of technology assessment in health care · 2024Article
- Priority-setting for hospital funding of high-cost innovative drugs and therapeutics: A qualitative institutional case study.PloS one · 2024Article
- Estimated costs for Duchenne muscular dystrophy care in Brazil.Orphanet journal of rare diseases · 2023Article
- Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges.International journal of environmental research and public health · 2023Review
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Cost-effectiveness of miglustat versus symptomatic therapy of Niemann-Pick disease type C.International journal of clinical pharmacy · 2022Article
- Recommendations from the IRDiRC Working Group on methodologies to assess the impact of diagnoses and therapies on rare disease patients.Orphanet journal of rare diseases · 2022Article
- The Value of Genomic Testing: A Contingent Valuation Across Six Child- and Adult-Onset Genetic Conditions.PharmacoEconomics · 2022Article
- State of the art and future directions in assessing the quality of life in rare and complex connective tissue and musculoskeletal diseases.Frontiers in medicine · 2022Review
- The Financial Burden of Surgery for Congenital Malformations-The Austrian Perspective.International journal of environmental research and public health · 2021Article
- Being a caregiver of a Behçet's syndrome patient: challenges and perspectives during a complex journey.Orphanet journal of rare diseases · 2021Article
- Left to themselves: Time to target chronic pain in childhood rare diseases.Neuroscience and biobehavioral reviews · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Rare diseases imply clinical and economic burden as well as a significant challenge for health systems. One relevant objective of the activities planned within the European Reference Network on Rare and Complex Connective Tissue and Musculoskeletal Diseases (ERN ReCONNET) is to address the economic dimensions of rare diseases to identify, develop and suggest strategies to improve research and patients' access to orphan drugs (ODs) and highly specialised health technologies. This paper presents a preliminary review of the existing policies on rare diseases in the countries of the Network members. It also introduces and discusses the theme of
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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.