Primary Biliary Cholangitis — Detection Program for Patients With Primary Biliary Cholangitis Lost in the System
Citation(s)
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European Association for the Study of the Liver Electronic address: easloffice@easloffice.eu; European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol. 2017 Jul;67(1):145-172. doi: 10.1016/j.jhep.2017.03.022. Epub 2017 Apr 18.
Garrido I, Liberal R, Cardoso MJ, Macedo G The impact of undiagnosed primary biliary cholangitis. Eur J Gastroenterol Hepatol. 2021 Dec 1;33(1S Suppl 1):e1027-e1031. doi: 10.1097/MEG.0000000000002268.
Kaplan MM, Gershwin ME Primary biliary cirrhosis. N Engl J Med. 2005 Sep 22;353(12):1261-73. doi: 10.1056/NEJMra043898. No abstract available. Erratum In: N Engl J Med. 2006 Jan 19;354(3):313.
Mayo MJ Mechanisms and molecules: What are the treatment targets for primary biliary cholangitis? Hepatology. 2022 Aug;76(2):518-531. doi: 10.1002/hep.32405. Epub 2022 Mar 10.
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.
