ReviewIntractable & rare diseases research2016
An overview of Compassionate Use Programs in the European Union member states.
Review in Intractable & rare diseases research, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers, 1 of them a synthesis 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
44 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.Frontiers in public health · 2019Pooled it
- Phage therapy: from basic biology to clinical application.Nature reviews. Microbiology · 2026Review
- An Industry Perspective on Compassionate Use in Europe: A Call for Change.Therapeutic innovation & regulatory science · 2026Review
- Early and accelerated access programs for medical devices in the European Union: mapping regulatory derogations and national schemes.Frontiers in medical technology · 2026Review
- Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.ESMO open · 2025Article
- Characteristics and availability of medicine early access programs and donations in Slovenia.The oncologist · 2025Article
- Early access disparities in innovative therapies across the US, EU, China, and Japan.Frontiers in medicine · 2025Article
- Implementing psychedelic-assisted therapy: History and characteristics of the Swiss limited medical use program.Neuroscience applied · 2025Review
- The impact of expanded access programs for systemic anticancer therapy in an Irish cancer centre.Irish journal of medical science · 2024Article
- The Early Access and the Potential Cost Savings by the Compassionate Use of Onco-haematological Drugs: Results from the Italian Study Compass-O.Clinical drug investigation · 2024Observational
- Optimising oncology drug expenditure in Ireland.Irish journal of medical science · 2024Review
- A Qualitative Interview Study on Expanded Access Clinical Trials for Compassionate Use in Japan.Patient preference and adherence · 2024Article
- Regulatory Approval, Reimbursement, and Clinical Use of Cyclin-Dependent Kinase 4/6 Inhibitors in Metastatic Breast Cancer in the Netherlands.JAMA network open · 2023Article
- Do Physicians Have a Duty to Discuss Expanded Access to Investigational Drugs with their Patients? A Normative Analysis.The Journal of law, medicine & ethics : a journal of the American Society of Law, Medicine & Ethics · 2023Article
- Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study.Orphanet journal of rare diseases · 2022Article
- Characteristics of the Compassionate Use Program in Japan: An Analysis of Expanded Access Clinical Trials from 2016 to 2021.Clinical pharmacology and therapeutics · 2022Article
- The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency.Journal of pharmaceutical policy and practice · 2022Article
- International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions.JAMA health forum · 2022Article
- [Efficacy and safety of Polatuzumab Vedotin in combination with rituximab plus bendamustine in patients with relapsed/refractory diffuse large B-cell lymphoma: a single center analysis].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022Article
- Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals.BMJ open · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The past decade witnessed rapid development of novel drugs and therapeutic biological agents. The marketing authorization for novel therapies is often time consuming and distressing for patients. Earlier clinical trials were the only way to access new drugs under development. However, not every patient meets the enrolment criteria, and participation is difficult for patients with life-threatening, long-lasting or seriously debilitating diseases like rare diseases. Early access programs like "Compassionate Use Program (CUP)" have generated alternative channels for such patients. The European Medical Agency provides regulations and recommendations for compassionate use, upon which every European Union (EU) member state has developed its own rules and regulations. Despite previous reviews and studies, the available information is limited and gaps exist. This literature review explores CUP in 28 EU member states. Data was collected through literature review and use of country-specific search terms from the healthcare domain. Data sources were not limited to databases and articles published in journals, but also included grey literature. The results implied that CUP was present in 20 EU member states (71%). Of 28 EU states, 18 (∼64%) had nationalized regulations and processes were well-defined. Overall, this review identified CUP and its current status and legislation in 28 EU member states. The established legislation for CUP in the EU member states suggest their willingness to adopt processes that facilitate earlier and better access to new medicines. Further research and periodic reviews are warranted to understand the contemporary and future regulatory trends in early access programs.
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.