title: Recommendations to Facilitate Expanded Access to Investigational Therapies for Seriously Ill Patients.
abstract: When clinical trial enrollment is not an option for seriously ill patients whose illnesses have not responded to approved treatment options, those patients and their physicians may consider gaining access to investigational therapies through a pathway established by the Food and Drug Administration (FDA) called expanded access. However, recent events have highlighted the challenging dynamics involved in accessing investigational therapies through expanded access that include a complex interplay of factors involving the patient, physician, drug company, FDA, and, increasingly, social media. The authors offer several potential strategies to streamline what is otherwise an arduous process for all involved. (1) The drug company should prospectively determine whether it will establish an expanded access program for specific drugs. (2) A central clearinghouse for companies should support registration of expanded access drugs for suitable patients. (3) The determination of whether a patient fits criteria would be made by an independent review board of clinicians. (4) An independent coordinating center is needed; academic health centers are ideally suited for that role. (5) Adequate financing of the costs of therapy need to be in place to make expanded access a reality, given frequent lack of payer coverage for therapies. (6) Further enhancement of regulatory pathways, approaches, or rules would promote expanded access. (7) Patients should explicitly acknowledge the limited data available. (8) There should be a shared, secure, technical platform to facilitate expanded access. All the authors' strategies present important prospects for improving treatment options for the most seriously ill patients.
authors.affiliation: R.N. Jerome is research services consultant, Research Support Services, Vanderbilt Institute for Clinical and Translational Research, Vanderbilt University Medical Center, Nashville, Tennessee. T.L. Edwards is associate director, Research Support Services, Vanderbilt Institute for Clinical and Translational Research, Vanderbilt University Medical Center, Nashville, Tennessee. H.C. Boswell is research services consultant, Research Support Services, Vanderbilt Institute for Clinical and Translational Research, Vanderbilt University Medical Center, Nashville, Tennessee. G.R. Bernard is associate vice chancellor for research, senior associate dean for clinical science, and principal investigator, Vanderbilt Clinical and Translational Award and Vanderbilt Institute for Clinical and Translational Research, and professor of medicine, Department of Medicine, Vanderbilt University Medical Center, Nashville, Tennessee. P.A. Harris is director, Office of Research Informatics, operations director of biomedical informatics, Vanderbilt Institute for Clinical and Translational Research, and associate professor, Departments of Biomedical Informatics and Biomedical Engineering, Vanderbilt University Medical Center, Nashville, Tennessee. J.M. Pulley is director, Research Support Services, operational director, Vanderbilt Institute for Clinical and Translational Research, and research associate professor, Medical Education and Administration, Vanderbilt University Medical Center, Nashville, Tennessee.
authors.forename: Rebecca N
authors.initials: RN
authors.lastname: Jerome
authors.forename: Terri L
authors.initials: TL
authors.lastname: Edwards
authors.forename: Haley C
authors.initials: HC
authors.lastname: Boswell
authors.forename: Gordon R
authors.initials: GR
authors.lastname: Bernard
authors.forename: Paul A
authors.initials: PA
authors.lastname: Harris
authors.forename: Jill M
authors.initials: JM
authors.lastname: Pulley
mesh_terms: D057176:Compassionate Use Trials / Q000331:legislation & jurisprudence*; D015507:Drugs, Investigational / Q000627:therapeutic use*; D006297:Health Services Accessibility / Q000458:organization & administration*; D006801:Humans; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015507:Drugs, Investigational