3 research outputs found
Access to Investigational Drugs: FDA Expanded Access Programs or âRightâtoâTryâ Legislation?
PurposeThe Food and Drug Administration Expanded Access (EA) program and âRightâtoâTryâ 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 PatientâInvestigational New Drug (SPâIND), Emergency SPâIND, 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. âRightâtoâTryâ 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 SPâIND 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.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/115991/1/cts12255.pd