Resources
(2023) National Landscape Assessment of Academic Medical Center Support for Expanded Access to Investigational Products
Gravelin M, Adamo J, Ellison S, Segear E, Parrish AB, Deeter C, Hamill J, Rigan L, Mashour GA, Weatherwax K. National Landscape Assessment of Academic Medical Center Support for Expanded Access to Investigational Products. J Clin Transl Sci. 2023. 7(1), E4. doi:10.1017/cts.2022.494.
(2023) Successful Treatment of Prolonged Severe Acute Respiratory Syndrome Coronavirus 2 Infection in Patients With Immunodeficiency With Extended Nirmatrelvir/Ritonavir: Case Series
Breeden M, Aitken SL, Baang JH, Gravelin M, Kaul DR, Lauring AS, Petty LA, Gregg KS. Successful Treatment of Prolonged Severe Acute Respiratory Syndrome Coronavirus 2 Infection in Patients With Immunodeficiency With Extended Nirmatrelvir/Ritonavir: Case Series. Open Forum Infect Dis. 2023 Apr 6;10(4):ofad189. doi: 10.1093/ofid/ofad189. PMID: 37089775; PMCID: PMC10114524.
(2022) Adapting an Expanded Access program to enable investigational treatments for COVID-19
Samuels E, Champagne E, Gravelin M, Racklyeft J, Weatherwax K. Adapting an Expanded Access program to enable investigational treatments for COVID-19. J Clin Transl Sci. 2022 May 16;6(1):e77. doi: 10.1017/cts.2022.403. PMID: 35836783; PMCID: PMC9274384.
(2021) Role of CTSA Institutes and Academic Medical Centers in Facilitating Preapproval Access to Investigational Agents and Devices During the COVID-19 Pandemic
Gravelin M, Wright J, Holbein M, Berro M, Brown J, Mashour G, Weatherwax K. Role of CTSA Institutes and Academic Medical Centers in Facilitating Preapproval Access to Investigational Agents and Devices During the COVID-19 Pandemic. J Clin Transl Sci. 2021:1-28. doi:10.1017/cts.2021.15
(2020) Navigating the Informed Consent Process When Using Innovative Surgery
Wehrmann D, Green GE, Weatherwax KJ, Shuman AG1. Navigating the Informed Consent Process When Using Innovative Surgery. Otolaryngol Head Neck Surg. 2020 Feb;162(2):177-180. doi: 10.1177/0194599819897067. Epub 2019 Dec 24.
(2020) The Inherent Unfairness of COVID-19 Drug Access Pathways
Spector-Bagdady K, Gravelin M, Weatherwax KJ, Shuman AG. The Inherent Unfairness of COVID-19 Drug Access Pathways. Am J Bioeth. 2020;20(9):18-20. doi: 10.1080/15265161.2020.1795546. Epub 2020 Aug 25. PMID: 33505223; PMCID: PMC7837294.
(2019) The Critical Role of Medical Institutions in Expanding Access to Investigational Interventions
Spector-Bagdady K, Weatherwax KJ, Gravelin M, Shuman AG. The Critical Role of Medical Institutions in Expanding Access to Investigational Interventions. The Hastings Center report. 2019 March;49(2):36-39. PubMed PMID: 30998277; PubMed Central PMCID: PMC6853061; DOI: 10.1002/hast.991.
(2019) 3D-printed, externally-implanted, bioresorbable airway splints for severe tracheobronchomalacia
Les AS, Ohye RG, Filbrun AG, Ghadimi Mahani M, Flanagan CL, Daniels RC, Kidwell KM, Zopf DA, Hollister SJ, Green GE. 3D-printed, externally-implanted, bioresorbable airway splints for severe tracheobronchomalacia. Laryngoscope. 2019 Aug;129(8):1763-1771. doi: 10.1002/lary.27863. Epub 2019 Feb 22. PMID: 30794335; PMCID: PMC6662576.
(2018) Right now, in the right way: U. S. Food and Drug Administration's expanded access program and patient rights
Holbein MEB, Weatherwax KJ, Gravelin M, Hutchinson R, Mashour GA. Right now, in the right way: U. S. Food and Drug Administration’s expanded access program and patient rights. J Clin Transl Sci. 2018 Jun;2(3):115-117. doi: 10.1017/cts.2018.318.
Determining Eligibility
The purpose of this document is to define criteria for use of FDA’s Expanded Access Program.
Academic Considerations
Committee and/or processes for tracking expanded access requests via a central data portal.
Manufacturer Approval
Gaining approval from the manufacturer to provide the product or device to treat the patient.
Contracts & Agreements
Ensuring that appropriate contracts and/or agreements have been put in place to protect the manufacturer, patient, and institution.
Templates
Confidential Disclosure Agreement Single Patient IND Template
Clinical Treatment Plan Agreement Single Patient IND Template
FDA Submission
Submitting the request to the appropriate FDA division.
Templates
Non-Emergency Individual Patient Expanded Access IND Cover Letter Template
Individual Patient Expanded Access IND Letter of Authorization (LOA) Template
Individual Patient Expanded Access IND Annual Report Template
Other Resources
FDA Example of Wording for Letter of Authorization (LOA) For Individual Patient Expanded Access IND
IRB Review
Submitting a regulatory application to IRB for approval or chair concurrence for patient protection.
Templates
IRB Sample Application Pathway for Individual Patient IND submission
Sample Single Patient IND Consent
Other Resources
FDA Guidance for individual patient IRB chair concurrence
Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers
Product Handling
Receiving the product or device, storing it under the appropriate conditions, and preparing to document administration to the patient.
Treating the Patient
Taking the appropriate measures to treat the patient and document in the medical record.
Reporting Responsibilities
Understanding the reporting responsibilities of a sponsor and investigator under expanded access.
Templates
Email Template for Physician Training
Other Resources
Duke University Sponsor and Investigator Training Modules
ReGARDD Educational Videos
Adverse Events and Safety Reporting - Single Patient Expanded Access
There are risks to every treatment, and sometimes a patient will experience negative effects – an adverse event – from an investigational product through Expanded Access. It is important for physicians to report these events to the appropriate authorities. This video describes the classifications of adverse events and when reporting is required.
Physician Responsibilities - Single Patient Expanded Access
Physicians who request Expanded Access for their patients are considered “sponsor-investigators” by the FDA. Sponsor-Investigators have certain responsibilities, the most important of which are described in this video.
Submission Tips - Single Patient Expanded Access
The process for non emergency Expanded Access, including FDA and IRB (institutional review board) submissions, can be daunting. This video provides tips for these submissions from the experience of TEAMSS collaborators.
Emergency Use Process - Single Patient Expanded Access
No time to complete the FDA or IRB submission? This video describes the process to request emergency use of an investigational product.
Single Patient Expanded Access to Investigational New Drugs - Overview
Don’t know what to expect when requesting expanded access to an investigational drug for your patient? This video describes the steps to obtain approval for treatment under expanded access.