Availability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs
Sign Up to like & getrecommendations! Published in 2018 at "JAMA Network Open"
DOI: 10.1001/jamanetworkopen.2018.0283
Abstract: Key Points Question What is the timing and duration of investigational drug availability through the US Food and Drug Administration’s (FDA’s) expanded access program? Findings In this cross-sectional study of 92 FDA-approved drugs with associated… read more here.
Keywords: investigational medicines; drug administration; food drug; access ... See more keywords
Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs
Sign Up to like & getrecommendations! Published in 2022 at "JAMA Network Open"
DOI: 10.1001/jamanetworkopen.2022.39766
Abstract: Key Points Question How do oncologists at large academic medical centers with experience pursuing expanded access (EA) to investigational medicines perceive this regulatory pathway and their role in it? Findings This qualitative study involved semistructured… read more here.
Keywords: academic oncologists; oncologists offering; expanded access; perspectives academic ... See more keywords
The Role of Physicians in Expanded Access to Investigational Drugs: A Mixed-Methods Study of Physicians’ Views and Experiences in The Netherlands
Sign Up to like & getrecommendations! Published in 2021 at "Journal of Bioethical Inquiry"
DOI: 10.1007/s11673-021-10090-7
Abstract: Treating physicians have key roles to play in expanded access to investigational drugs, by identifying investigational treatment options, assessing the balance of risks and potential benefits, informing their patients, and applying to the regulatory authorities.… read more here.
Keywords: mixed methods; methods study; investigational drugs; access investigational ... See more keywords
The impact of expanded access programs for systemic anticancer therapy in an Irish cancer centre
Sign Up to like & getrecommendations! Published in 2024 at "Irish Journal of Medical Science"
DOI: 10.1007/s11845-024-03701-w
Abstract: Expanded access programs (EAPs) allow cancer patients with unmet clinical need to obtain access to pre-authorisation treatments. There is no standardised process for implementing these programs nationally, and real-world data on their impact is lacking.… read more here.
Keywords: cancer; cancer centre; access programs; access ... See more keywords
Little to lose and no other options: Ethical issues in efforts to facilitate expanded access to investigational drugs.
Sign Up to like & getrecommendations! Published in 2018 at "Health policy"
DOI: 10.1016/j.healthpol.2018.06.005
Abstract: BACKGROUND Today, public and private bodies around the world are trying to facilitate and increase expanded access to unapproved, investigational drugs for patients with unmet medical needs. METHODS This paper discusses three major shifts in… read more here.
Keywords: facilitate expanded; investigational drugs; efforts facilitate; ethical issues ... See more keywords
Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study
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DOI: 10.1038/s41598-025-99526-3
Abstract: Acute hepatic porphyria (AHP), a rare genetic disorder, causes life-threatening porphyria attacks and chronic pain and impairs daily functioning and quality of life. Recently, a new siRNA therapy, givosiran, became available for AHP. This open-label,… read more here.
Keywords: japanese patients; acute hepatic; hepatic porphyria; expanded access ... See more keywords
Cost-efficiency and budget-neutral expanded access modeling of the novel PD-1 inhibitor toripalimab versus pembrolizumab in recurrent or metastatic nasopharyngeal carcinoma.
Sign Up to like & getrecommendations! Published in 2024 at "Journal of medical economics"
DOI: 10.1080/13696998.2024.2331905
Abstract: Aims: To estimate, in the setting of recurrent or metastatic nasopharyngeal carcinoma (R/M NPC) for an assumed 1,207 incident US cases in 2024, (1) the cost-efficiency of a toripalimab-gemcitabine-cisplatin regimen compared to a similar pembrolizumab… read more here.
Keywords: cost; rate; budget neutral; expanded access ... See more keywords
Avelumab maintenance in patients with metastatic urothelial carcinoma in a real-life expanded-access program.
Sign Up to like & getrecommendations! Published in 2025 at "Future oncology"
DOI: 10.1080/14796694.2025.2570637
Abstract: BACKGROUND This study evaluates the real-world efficacy and safety of avelumab maintenance therapy in patients with metastatic urothelial carcinoma (mUC) who did not progress following first-line platinum-based chemotherapy, using data from the Expanded Access Program… read more here.
Keywords: urothelial carcinoma; metastatic urothelial; maintenance; patients metastatic ... See more keywords
Prescribing unproven cancer drugs: physician perspectives on expanded access and right to try
Sign Up to like & getrecommendations! Published in 2022 at "Journal of Law and the Biosciences"
DOI: 10.1093/jlb/lsac031
Abstract: Abstract Background For gravely ill patients who have no treatment options and who are ineligible for clinical trials, the US Food and Drug Administration (FDA) established the Expanded Access Program (EAP). Motivated by efforts to… read more here.
Keywords: right try; prescribing unproven; expanded access; unproven cancer ... See more keywords
135: Expanded Access and Unanticipated Benefits of Tele-ICU in the COVID-19 Pandemic
Sign Up to like & getrecommendations! Published in 2020 at "Critical Care Medicine"
DOI: 10.1097/01.ccm.0000726428.32372.bf
Abstract: INTRODUCTION: In the COVID-19 pandemic, novel solutions were desperately needed to protect healthcare workers while continuing essential bedside care, multiprofessional rounds, specialist consultation, and family visitation A strategy was implemented to harness the power of… read more here.
Keywords: covid pandemic; tele icu; family; access ... See more keywords
Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States: An Expanded Access Program
Sign Up to like & getrecommendations! Published in 2017 at "Journal of Immunotherapy (Hagerstown, Md. : 1997)"
DOI: 10.1097/cji.0000000000000186
Abstract: KEYNOTE-030 (ClinicalTrials.gov ID, NCT02083484) was a global expanded access program that allowed access to pembrolizumab, an antiprogrammed death 1 antibody, for patients with advanced melanoma before its regulatory approval. Patients with unresectable stage III/IV melanoma… read more here.
Keywords: response; access; access program; pembrolizumab ... See more keywords