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
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
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
Long‐term efficacy and safety of cannabidiol in patients with treatment‐resistant epilepsies: Four‐year results from the expanded access program
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DOI: 10.1111/epi.17496
Abstract: Cannabidiol (CBD) expanded access program, initiated in 2014, provided add‐on CBD to patients with treatment‐resistant epilepsies (TREs) at 35 US epilepsy centers. Prior publications reported results through December 2016; herein, we present efficacy and safety… read more here.
Keywords: resistant epilepsies; treatment resistant; expanded access; access program ... See more keywords
Abstract CT127: Phase I and expanded access experience of LOXO-195 (BAY 2731954), a selective next-generation TRK inhibitor (TRKi)
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DOI: 10.1158/1538-7445.am2019-ct127
Abstract: Background: Larotrectinib, a selective TRKi, is now FDA approved for pediatric and adult TRK-fusion solid tumors, regardless of tumor origin. Emergent TRK kinase mutations are a common mechanism of resistance to TRKis. LOXO-195, a selective… read more here.
Keywords: trk kinase; trk; expanded access; loxo 195 ... See more keywords
Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study
Sign Up to like & getrecommendations! Published in 2020 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-020-01477-7
Abstract: Background Expanded access programs (EAPs) allow patients with serious, life-threatening conditions access to drugs prior to their formal approval. Despite the possible benefits for patients, EAPs present several challenges, including uncertainty regarding a drug’s efficacy… read more here.
Keywords: spinal muscular; treatment; muscular atrophy; atrophy ... See more keywords
Reassessing existing expanded access programs and planning forward in the time of Right to Try.
Sign Up to like & getrecommendations! Published in 2019 at "Journal of Clinical Oncology"
DOI: 10.1200/jco.2019.37.15_suppl.e18172
Abstract: e18172 Background: Desperately ill patients who have exhausted treatment options often seek access to therapy through investigational therapies. Participation in a clinical trial may be one option. When a clinical trial is not an option,… read more here.
Keywords: right try; access; expanded access; policy ... See more keywords