The time is now: making the case for compassionate use/pre-approval access to new TB drugs [PDF]
IJTLD OpenSUMMARY: Although the majority of people with multidrug-resistant/rifampicin-resistant TB (MDR/RR-TB) can now be successfully treated with all-oral shorter regimens, rising rates of resistance to the components in these regimens are a growing global ...
A. Reuter +12 more
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A Qualitative Interview Study on Expanded Access Clinical Trials for Compassionate Use in Japan
Patient Preference and AdherenceHaruto Hayashi,1 Takehide Nogita,2,3 Hideki Maeda1,2 1Department of Regulatory Science, Faculty of Pharmacy, Meiji Pharmaceutical University, Tokyo, Japan; 2Department of Regulatory Science, Graduate School of Pharmaceutical Science, Meiji Pharmaceutical
Hayashi H, Nogita T, Maeda H
doaj +3 more sources
A brief note on randomized controlled trials and compassionate/off-label use of drugs in the early phases of the COVID-19 pandemic [PDF]
Drugs in Context, 2020 Randomized controlled trials (RCTs) are the best way to find effective and acceptable safe treatments for COVID-19 and any possible future outbreak. However, caution is needed when comparing the number of participants in RCTs with that of patients with ...
Matteo Bassetti +4 more
doaj +4 more sources
Compassionate Drug Use – Time Arising for a New Law in Bulgaria in the Era of COVID-19 [PDF]
Folia Medica, 2020 Introduction: Despite clinical trials, there are still no approved specific therapies or any vaccine against COVID-19. The only option available is using investigational drugs for compassionate use.
Nigyar Dzhafer, Jannis V. Papathanasiou
doaj +3 more sources
Siblings and Discordant Eligibility for Gene Therapy Research: Considering Parental Requests for Non-Trial "Compassionate Use” [PDF]
Clinical Ethics, 2021 Deciding whether to grant an expanded access request for a child whose sibling is enrolled in a gene therapy trial involves a number of complex factors: considering the best interests of the child, the psychosocial and economic impact on the family, and the concerns and obligations of researchers.
Jamie Webb +2 more
openaire +2 more sources
COVID‐19, children, clinical trials and compassion: The ethical case for using innovative or compassionate treatments [PDF]
Acta Paediatrica, 2021 AbstractAimSafe, effective SARS‐CoV‐2 treatment has not yet been determined, though some drugs have favourable mortality and morbidity benefits in specific situations. No treatments have been explicitly tested in children, who are, therefore, once again therapeutic orphans.MethodWe echo calls to enrol patients, including children, into trials but those
Vic Larcher, Art Caplan, Joe Brierley
openaire +2 more sources
Cost-effectiveness of drug therapy prescribed in special situations at hospital [PDF]
Farmacia Hospitalaria, 2020 Objective: The Pharmacy and Therapeutics Committee is an advisory body to the medical management of our hospital. Following Royal Decree 86/2015, which regulates the Pharmacy and Therapeutics Committee of the Balearic Islands, this committee prepared ...
Gonzalo González-Morcillo +2 more
doaj +1 more source
Off-label prescription of drugs at hospital [PDF]
Farmacia Hospitalaria, 2016 Objectives: To develop a procedure for management of off-label medications, and to analyze the treatments, indications, and hospital units which will request them more frequently, as well as which variables will have an impact on the authorization ...
Vicente Arocas Casañ +6 more
doaj +1 more source
Progress and Pitfalls of Bacteriophage Therapy in Critical Care: A Concise Definitive Review
Critical Care Explorations, 2021 Objective:. Bacterial infections caused by antibiotic-resistant pathogens are a major problem for patients requiring critical care. An approach to combat resistance is the use of bacterial viruses known as “phage therapy.” This review provides a brief ...
Luca Valente, MD +3 more
doaj +1 more source
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