Furlong P.,Parent Project Muscular Dystrophy |
Charnas L.,Shire Plc |
Fallon J.R.,Brown University |
Fischer R.,Parent Project Muscular Dystrophy |
And 6 more authors.
Orphanet Journal of Rare Diseases | Year: 2015
Among the challenges confronting patients with rare diseases is a dearth of treatment options. The development of safe and effective new therapies is hampered by challenges associated with conducting clinical trials in small populations. In this article, we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration. This unprecedented undertaking involved a broad coalition of more than 80 stakeholders collaborating across nine time zones to produce a document in only 6 months. We hope that other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents. © 2015 Furlong et al.
Van Buskirk G.A.,Nonclinical Drug Development Consulting Services LLC |
Yacobi A.,DOLE Pharma LLC |
Bolger M.B.,Simulations Plus Inc. |
Chittenden J.,Pharsight |
And 17 more authors.
Dissolution Technologies | Year: 2014
This article summarizes the proceeding of the September 2012 Workshop on Application of In vitro-In vivo cor- relation (IVIVC) in Formulation Development. The workshop brought together international experts with the goal of establishing common concepts that could be utilized to facilitate the development and validation of IVIVCs in the registration of and post-approval changes to oral solid dosage forms. The workshop was organized by the Product Quality Research Institute (PQRI) and cosponsored by AAPS, FDA, FIP, and USP. Open access of this information is available to all interested parties.
Schafermeyer R.G.,YourEncore |
Hoffman R.R.,Institute for Human and Machine Cognition
IEEE Intelligent Systems | Year: 2016
This essay hearkens to the origins of IEEE Intelligent Systems in the era of 'expert systems,' and it revisits a recurring theme of using intelligent systems technology to help preserve and share individual and corporate expertise. The article recounts the experiences of a director of research at Procter & Gamble, who encountered issues of knowledge management. Using intelligent systems, a methodology was developed to capture and share expert knowledge. This methodology can be seen in an instance of independent and simultaneous discovery, and thereby reinforces arguments about the potential value of knowledge management that have been expressed in previous essays in this department. © 2001-2011 IEEE.