ArticleJACC. Basic to translational science2018
Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".
Article in JACC. Basic to translational science, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Systematic review of the registered clinical trials for coronavirus disease 2019 (COVID-19).Journal of translational medicine · 2020Pooled it
- Phage therapy for recurrent urinary tract infections: A qualitative study using the theoretical framework of acceptability.PloS one · 2026Article
- Optimized preparation pipeline for emergency phage therapy against Pseudomonas aeruginosa at Yale University.Scientific reports · 2024Article
- Standardized bacteriophage purification for personalized phage therapy.Nature protocols · 2020Article
- Update to Drugs, Devices, and the FDA: How Recent Legislative Changes Have Impacted Approval of New Therapies.JACC. Basic to translational science · 2020Article
- The Role of the Food and Drug Administration in Drug Development: On the Subject of Proarrhythmia Risk.The Journal of innovations in cardiac rhythm management · 2020Review
- Application of the New Process for Unapproved Drug Use: Dilemma of Universal Health Care Coverage in Japan.Journal of global oncology · 2019Article
- 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
- Expanding Patient Access to Investigational New Drugs: Overview of Intermediate and Widespread Treatment Investigational New Drugs, and Emergency Authorization in Public Health Emergencies.JACC. Basic to translational science · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
With drug approval times taking an average of 8 years from entry into clinical trials to full U.S. Food and Drug Administration (FDA) approval, patients with life-threatening and severely debilitating disease and no reasonable therapeutic options are advocating for expanded access (EA) to investigational drugs prior to approval. Special investigational new drug (IND) application categories allow patients who meet specific criteria to receive treatment with non-approved drugs. The FDA approves over 99% of all single-patient INDs, providing emergency approval within hours, and non-emergency approval within an average of 4 days. "Right-to-try" laws passed in 38 states would allow patients to bypass FDA processes altogether, but contain controversial provisions that some claim risk more harm than benefit to desperate and vulnerable patients. This review focuses on FDA EA to non-approved drugs through a special category of IND-the single-patient IND-and "right-to-try" (R2T) access outside of the FDA.
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.