ReviewExpert opinion on investigational drugs2018
Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits.
Review in Expert opinion on investigational drugs, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 23 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
23 citing papers in PubMed.
- Responses to Commentaries on "Ethics as if Your Life Depended on It".Journal of empirical research on human research ethics : JERHRE · 2026Article
- Is Digital Healthcare Better Than None? The Digital Health Technology Dilemma in Low-Income Countries.Asian bioethics review · 2026Article
- Ag(e)nostic precision oncology therapy approvals across the years.Trends in cancer · 2025Review
- Neighborhood characteristics of expanded access patients at an academic referral center.Journal of clinical and translational science · 2025Article
- Measuring the multidimensional reputation of a medicines regulatory agency: development and validation of a public-oriented scale.Frontiers in medicine · 2025Article
- The impact of expanded access programs for systemic anticancer therapy in an Irish cancer centre.Irish journal of medical science · 2024Article
- Optimising oncology drug expenditure in Ireland.Irish journal of medical science · 2024Review
- Ethical considerations for nurses working in rare cancer care: Focus on sarcoma.Canadian oncology nursing journal = Revue canadienne de nursing oncologique · 2024Article
- Tumor Necrosis Alpha (TNF-α) Antagonists Used in Chronic Inflammatory Rheumatic Diseases: Risks and their Minimization Measures.Current drug safety · 2024Review
- Molecular tumour boards - current and future considerations for precision oncology.Nature reviews. Clinical oncology · 2023Review
- Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs.JAMA network open · 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
- CRAC (Clinical Relevance of Alterations in Cancer): a Knowledge Base for the Selection of Molecularly Matched Therapy for Solid Tumors.Sovremennye tekhnologii v meditsine · 2022Article
- ClinicalTrials.gov as a Source of Information About Expanded Access Programs: Cohort Study.Journal of medical Internet research · 2021Article
- 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
- The Equitable Implementation of Cystic Fibrosis Personalized Medicines in Canada.Journal of personalized medicine · 2021Review
- Factors Associated With Off-Label Oncology Prescriptions: The Role of Cost and Financing in a Universal Healthcare System.Frontiers in pharmacology · 2021Article
- Expanded Access as a source of real-world data: An overview of FDA and EMA approvals.British journal of clinical pharmacology · 2020Article
- 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
- Providing Patients with Critical or Life-Threatening Illnesses Access to Experimental Drug Therapy: A Guide to Clinical Trials and the US FDA Expanded Access Program.Pharmaceutical medicine · 2019Review
Corrections and comments
- Erratum issuedCorrigendum.2018
Authors and funding
3 authors.
Funding
Abstract
introductionExpanded access is the use of an investigational product by patients with serious medical conditions without participation in a clinical trial. It is a complicated process involving the collaboration of many parties and pharmaceutical companies. Ongoing efforts focus on accelerating expanded access procedures in the best interest of patients with cancer. AREAS COVERED: We review the regulatory and ethical challenges encountered in efforts to optimize expanded access. EXPERT OPINION: In the era of personalized medicine, patients may benefit from novel therapeutic agents that demonstrate encouraging results in early studies. However, drug approval is a lengthy and cumbersome procedure that might exceed the time frame of a life-threatening disease. Expanded access provides options to patients with unmet needs. It may provide informative safety and efficacy data to the manufacturers and the scientific and regulatory organizations. Ongoing efforts are being made by global governmental and scientific committees, regulatory agencies, and patient organizations to address the ethical and regulatory issues and to optimize the expanded access process. Their goal is to expand access to promising novel drugs for individual patients and to accelerate the necessary procedures while preserving patient safety.
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.