ReviewOrphanet journal of rare diseases2018
Compounded medication for patients with rare diseases.
Review in Orphanet journal of rare diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed, 69 citations in OpenAlex.
- Extemporaneous Formulations for Pediatric Patients: Global Necessities, Challenges and Opportunities.Pharmaceutics · 2026Review
- From precision interventions to precision health.Nature communications · 2025Review
- Safety of Compounded Medications.Cureus · 2025Article
- Article
- Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.Journal of inherited metabolic disease · 2025Article
- Three-Dimensional Printing of PVA Capsular Devices for Applications in Compounding Pharmacy: Effect of Design Parameters on Pharmaceutical Performance.Pharmaceutics · 2024Article
- Global health for rare diseases through primary care.The Lancet. Global health · 2024Article
- Evaluation of the type and frequency of extemporaneous formulations dispensed in hospital and community pharmacies.Exploratory research in clinical and social pharmacy · 2023Article
- Paediatric capsule compounding in hospital practices: by weight or by volume?European journal of hospital pharmacy : science and practice · 2023Article
- Tuberculosis medicines for children in Europe: an unmet medical need.ERJ open research · 2023Article
- Social pharmaceutical innovation and alternative forms of research, development and deployment for drugs for rare diseases.Orphanet journal of rare diseases · 2022Article
- The Role of Pharmaceutical Compounding in Promoting Medication Adherence.Pharmaceuticals (Basel, Switzerland) · 2022Review
- Systems Challenges in Accessing Medicines among Children under Thailand's Universal Health Coverage: A Qualitative Study of a Provincial Public Hospital Network.Children (Basel, Switzerland) · 2022Article
- Medicine manipulation: An alternative to mitigate therapeutic gaps in the Brazilian Unified Health System?PloS one · 2022Article
- Treatable inherited metabolic disorders causing intellectual disability: 2021 review and digital app.Orphanet journal of rare diseases · 2021Review
- Vitamin D intoxication in patients with cystic fibrosis: report of a single-center cohort.Scientific reports · 2021Article
- Assessing a Mass-Based Method for the Preparation of Low-Dosed Paediatric Capsules with Baclofen and Spironolactone.Pharmacy (Basel, Switzerland) · 2021Article
- Pharmacy Compounded Medicines for Patients With Rare Diseases: Lessons Learned From Chenodeoxycholic Acid and Cholic Acid.Frontiers in pharmacology · 2021Article
- Orphan Drugs, Compounded Medication and Pharmaceutical Commons.Frontiers in pharmacology · 2021Review
- Global drug shortages due to COVID-19: Impact on patient care and mitigation strategies.Research in social & administrative pharmacy : RSAP · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundWhen there is no authorized on- or in absence even no off-label treatment for patients with rare diseases, pharmacists have to compound medicinal products to meet the patients special needs. However it is important that there is evidence in the medical and/or pharmaceutical literature for such compounded medications. POSITION STATEMENT: Pharmaceutical compounding must be performed in the best possible circumstances by certified practitioners (pharmacists) using validated standard operating procedures (standardized formulations) in order to obtain medicinal products of the highest quality to assure patient safety. More than 60 compounding procedures were identified in 17 on-line pharmaceutical compounding reference sources worldwide but more operating procedures still need to be validated. All ingredients used in the preparation of the compounded medication must be accompanied by a certificate of analysis and full records of the pharmaceutical production process need to be kept for full traceability and accountability.
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.