ArticleBMC research notes2017
Characterizing expanded access and compassionate use programs for experimental drugs.
Article in BMC research notes, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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
15 citing papers in PubMed.
- [Pharmaceutical services in expanded access, compassionate use, and post-trial access: the perspective of clinical researchers].Cadernos de saude publica · 2025Article
- Advances in RNA-based therapeutics: current breakthroughs, clinical translation, and future perspectives.Frontiers in genetics · 2025Review
- Conceptualisation and Role of Market Access in Pharmaceutical Industry: A Scoping Review.Journal of market access & health policy · 2024Article
- Article
- The 'false hope' argument in discussions on expanded access to investigational drugs: a critical assessment.Medicine, health care, and philosophy · 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
- Regulating clinical trials in a resource-limited setting during the Ebola public health emergency in Sierra Leone.Trials · 2022Article
- The Role of Physicians in Expanded Access to Investigational Drugs: A Mixed-Methods Study of Physicians' Views and Experiences in The Netherlands.Journal of bioethical inquiry · 2021Article
- A comparative analysis of important public clinical trial registries, and a proposal for an interim ideal one.PloS one · 2021Article
- Expanded Access as a source of real-world data: An overview of FDA and EMA approvals.British journal of clinical pharmacology · 2020Article
- Systematic review of extracellular vesicle-based treatments for lung injury: are EVs a potential therapy for COVID-19?Journal of extracellular vesicles · 2020Review
- Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study.Orphanet journal of rare diseases · 2020Article
- Article
- Availability of Investigational Medicines Through the US Food and Drug Administration's Expanded Access and Compassionate Use Programs.JAMA network open · 2018Article
- Taking Psychedelics Seriously.Journal of palliative medicine · 2018Review
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
Abstract
objectiveWe sought to determine the characteristics of "expanded access" and "compassionate use" programs registered in ClinicalTrials.gov and to determine the percentage of drugs provided through these programs that ultimately received FDA marketing approval.
resultsWe identified 398 expanded access and compassionate use programs (hereafter referred to as expanded access programs) registered on ClinicalTrials.gov. Industry funded 61% (n = 241) of programs individually or collaboratively, while NIH and the US Federal Government rarely funded programs (3% [n = 11] and 2% [n = 6], respectively). Most programs provided access to drugs (71% [n = 282]), 11% to biologics (n = 43), and 10% to medical devices (n = 40). These programs covered 460 unique conditions, the most common being HIV (n = 26), leukemia (22), and multiple myeloma (n = 14). Only 2% of programs reported results in ClinicalTrials.gov. Most programs (82%) were open to enrolling adults and seniors (n = 326). These programs provided access to 210 unique experimental drugs, of which 76% have received FDA approval.
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.