Pulmonary Arterial Hypertension of Congenital Heart Disease — Observatory of Pulmonary Arterial Hypertension of Congenital Heart Disease
Citation(s)
Barst RJ, Ivy DD, Foreman AJ, McGoon MD, Rosenzweig EB Four- and seven-year outcomes of patients with congenital heart disease-associated pulmonary arterial hypertension (from the REVEAL Registry). Am J Cardiol. 2014 Jan 1;113(1):147-55. doi: 10.1016/j.amjcard.2013.09.032. Epub 2013 Oct 4.
Barst RJ, McGoon MD, Elliott CG, Foreman AJ, Miller DP, Ivy DD Survival in childhood pulmonary arterial hypertension: insights from the registry to evaluate early and long-term pulmonary arterial hypertension disease management. Circulation. 2012 Jan 3;125(1):113-22. doi: 10.1161/CIRCULATIONAHA.111.026591. Epub 2011 Nov 15.
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.
