title: Clinical trials in pulmonary hypertension: Time for a consortium.
abstract: Current and past clinical trials in pulmonary hypertension, while valuable, are limited by the absence of mechanistic aims, by dissatisfaction with endpoints and the inability to share data. Clinical studies in pulmonary hypertension might be enhanced by a consortium approach that utilizes the expertise of academic medicine, the treatment initiatives of the pharmaceutical industry and study design from funding agencies interested in biological mechanisms. A meeting of interested parties, the Pulmonary Hypertension Academic Research Consortium (PHARC), was held from 30 April to 1 May 2012 in Bethesda, Maryland. Members at the conference were from the USA Federal Drug Administration (FDA); pharmaceutical industry (Pfizer, Novartis, Bayer and Gilead); USA National Institutes of Health (NHLBI); the Pulmonary Vascular Research Institute (PVRI), a non-governmental organization (NGO); and research and clinical members of pulmonary hypertension programs of international scope. A recommendation to develop a clinical trials consortium was the product of the working group on academic standards in clinical trials. The working group concluded that clinical trials hold immense promise to move the field of pulmonary hypertension forward if the trials are designed by a consortium with input from multiple groups. This would result in study design, conduct and analysis determined by consortium members with a high degree of independent function. The components of a well-balanced consortium that give it scientific effectiveness are: (1) the consortium can work with multiple companies simultaneously; (2) sponsors with special interests, such as testing biological mechanisms, can add investigations to a study at lower cost than with present granting strategies; (3) data handling including archiving, analysis and future sharing would be improved; (4) ancillary studies supported by the collection and dissemination of tissues and fluids would generate a broader approach to discovery than is now possible; and (5) development of improved endpoints in consultation with regulatory agencies, industry and academia would be possible.
authors: Department of Medicine, Vanderbilt University School of Medicine, Nashville, Tennessee, USA.
authors: John H
authors: JH
authors: Newman
authors: Gregory C
authors: GC
authors: Elliott
authors: Glennis S
authors: GS
authors: Haworth
authors: Edio
authors: E
authors: Zampaglione
authors: Satjit
authors: S
authors: Brar
authors: Simon J
authors: SJ
authors: Gibbs
authors: Julio
authors: J
authors: Sandoval
keywords: Federal Drug Administration; National Institutes of Health; Non-Governmental Organization; Pulmonary Vascular Research Institute; clinical trial; consortium; industry; international; pharmaceutical; pulmonary hypertension
references: JAMA. 2012 Feb 22;307(8):795-803
references: 22307571
references: J Am Coll Cardiol. 2009 Apr 28;53(17):1573-619
references: 19389575
references: Circulation. 2011 Jun 28;123(25):2919-21
references: 21709071
references: Am J Respir Crit Care Med. 2012 Aug 1;186(3):261-72
references: 22679007
references: Sci Transl Med. 2011 Jun 29;3(89):89cm16
references: 21715677