An analysis of common ethical justifications for compassionate use programs for experimental drugs [PDF]
, 2016 BACKGROUND: When a new intervention or drug is developed, this has to pass through various phases of clinical testing before it achieves market approval, which can take many years.
Raus, Kasper
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Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022
Orphanet Journal of Rare Diseases, 2023 Background Compassionate use is a system that provides patients with expedited access to drugs which has not yet been approved, but currently in clinical trials.
Jiayu Wu +5 more
doaj +1 more source
Investigational drugs for visceral leishmaniasis. [PDF]
Expert Opin Investig Drugs, 2015 Sundar S, Chakravarty J.
europepmc +2 more sources
Investigational new drugs for focal epilepsy. [PDF]
, 2015 For more than 30 years, antiepileptic drug development has been based on specific assumptions regarding the neurobiology of epilepsy but all marketed drugs have not changed the proportion of drug refractory patients.
Mula, M
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From bench to bedside and to health policies (and back): ethics in translational research
Annali dell'Istituto Superiore di Sanità, 2014 INTRODUCTION: The medical aim of translational research is to smooth the transition of discoveries made through basic research from the laboratory bench to their diagnostic or therapeutic applications for patients.
Carlo Petrini
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Drug Repurposing Screen for Compounds Inhibiting the Cytopathic Effect of SARS-CoV-2
Frontiers in Pharmacology, 2021 Drug repurposing is a rapid approach to identify therapeutics for the treatment of emerging infectious diseases such as COVID-19. To address the urgent need for treatment options, we carried out a quantitative high-throughput screen using a SARS-CoV-2 ...
Catherine Z. Chen +18 more
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Consensus on the Clinical Approach to Moderate-to-Severe Atopic Dermatitis in Spain: A Delphi Survey [PDF]
, 2020 Background. The purpose of this study was to gather information on the current assessment and management of patients with moderate-to-severe AD in routine daily practice. Methods.
Armario Hita, José Carlos +9 more
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BMC Medical Ethics, 2019 Background Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs.
Eline M. Bunnik, Nikkie Aarts
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Effects of the anti-RANKL antibody denosumab on joint structural damage in patients with rheumatoid arthritis treated with conventional synthetic disease-modifying antirheumatic drugs (DESIRABLE study): a randomised, double-blind, placebo-controlled phase 3 trial. [PDF]
, 2019 ObjectiveTo evaluate the efficacy of denosumab in suppressing joint destruction when added to conventional synthetic disease-modifying antirheumatic drug (csDMARD) therapy in patients with rheumatoid arthritis (RA).MethodsThis was a multi-centre ...
Genant, Harry K +9 more
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Medicine Access @ Point of Care, 2021 Introduction: Physicians in the United States play an essential role guiding patients through single patient pre-approval access (PAA) to investigational medical products via either the Food and Drug Administration (FDA)’s Expanded Access (EA) or the ...
Carolyn Riley Chapman +4 more
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