ReviewClinical and translational science2015
Access to Investigational Drugs: FDA Expanded Access Programs or "Right-to-Try" Legislation?
Review in Clinical and translational science, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 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
18 citing papers in PubMed.
- 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
- Characteristics of the Compassionate Use Program in Japan: An Analysis of Expanded Access Clinical Trials from 2016 to 2021.Clinical pharmacology and therapeutics · 2022Article
- Great Expectations: recommendations for improving the methodological rigor of psychedelic clinical trials.Psychopharmacology · 2022Review
- Adapting an Expanded Access program to enable investigational treatments for COVID-19.Journal of clinical and translational science · 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
- "I Think It's Been Met With a Shrug:" Oncologists' Views Toward and Experiences With Right-to-Try.Journal of the National Cancer Institute · 2021Article
- Early Access Provision for Innovative Medicinal Products in Oncology: Challenges and Opportunities.Frontiers in oncology · 2020Article
- What do patients with unmet medical needs want? A qualitative study of patients' views and experiences with expanded access to unapproved, investigational treatments in the Netherlands.BMC medical ethics · 2019Article
- Application of the New Process for Unapproved Drug Use: Dilemma of Universal Health Care Coverage in Japan.Journal of global oncology · 2019Article
- Current State of Compassionate Phage Therapy.Viruses · 2019Review
- 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
- Use, Safety, and Efficacy of Single-Patient Use of the US Food and Drug Administration Expanded Access Program.JAMA oncology · 2019Article
- The Critical Role of Medical Institutions in Expanding Access to Investigational Interventions.The Hastings Center report · 2019Article
- Right now, in the right way: U. S. Food and Drug Administration's expanded access program and patient rights.Journal of clinical and translational science · 2018Article
- Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".JACC. Basic to translational science · 2018Article
- Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits.Expert opinion on investigational drugs · 2018Review
- Chimpanzee adenoviral vectors as vaccines for outbreak pathogens.Human vaccines & immunotherapeutics · 2017Review
- Going "social" to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access.BMC medicine · 2016Article
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
Abstract
purposeThe Food and Drug Administration Expanded Access (EA) program and "Right-to-Try" legislation aim to provide seriously ill patients who have no other comparable treatment options to gain access to investigational drugs and biological agents. Physicians and institutions need to understand these programs to respond to questions and requests for access.
methodsFDA EA programs and state and federal legislative efforts to provide investigational products to patients by circumventing FDA regulations were summarized and compared.
resultsThe FDA EA program includes Single Patient-Investigational New Drug (SP-IND), Emergency SP-IND, Intermediate Sized Population IND, and Treatment IND. Approval rates for all categories exceed 99%. Approval requires FDA and Institutional Review Board (IRB) approval, and cooperation of the pharmaceutical partner is essential. "Right-to-Try" legislation bypasses some of these steps, but provides no regulatory or safety oversight.
conclusionThe FDA EA program is a reasonable option for patients for whom all other therapeutic interventions have failed. The SP-IND not only provides patient access to new drugs, but also maintains a balance between immediacy and necessary patient protection. Rather than circumventing existing FDA regulations through proposed legislation, it seems more judicious to provide the knowledge and means to meet the EA requirements.
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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.