The NordiNet® International Outcome Study and NovoNet® ANSWER Program®: rationale, design, and methodology of two international pharmacoepidemiological registry-based studies monitoring long-term clinical and safety outcomes of growth hormone therapy (Norditropin®)
Authors Höybye C, Sävendahl L, Christesen HT, Lee P, Pedersen BT, Schlumpf M, Germak J, Ross J
Received 10 January 2013
Accepted for publication 13 February 2013
Published 26 April 2013 Volume 2013:5(1) Pages 119—127
Checked for plagiarism Yes
Review by Single-blind
Peer reviewer comments 3
Charlotte Höybye,1 Lars Sävendahl,2 Henrik Thybo Christesen,3 Peter Lee,4 Birgitte Tønnes Pedersen,5 Michael Schlumpf,6 John Germak,7 Judith Ross8
1Department of Molecular Medicine and Surgery, Karolinska Institute and Department of Endocrinology, Metabolism and Diabetes, 2Department of Women’s and Children’s Health, Karolinska Institute and Division of Pediatrics, Karolinska University Hospital, Stockholm, Sweden; 3Hans Christian Andersen Children’s Hospital, Odense University Hospital, Odense, Denmark; 4Department of Pediatrics, Penn State College of Medicine, Hershey, PA, USA; 5Global Development, Novo Nordisk A/S, Søborg, Denmark; 6Global Medical Affairs Biopharm, Novo Nordisk Health Care AG, Zurich, Switzerland; 7Clinical Development and Medical Affairs, Novo Nordisk Inc, Princeton, NJ, USA; 8Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA, USA
Objective: Randomized controlled trials have shown that growth hormone (GH) therapy has effects on growth, metabolism, and body composition. GH therapy is prescribed for children with growth failure and adults with GH deficiency. Carefully conducted observational study of GH treatment affords the opportunity to assess long-term treatment outcomes and the clinical factors and variables affecting those outcomes, in patients receiving GH therapy in routine clinical practice.
Design: The NordiNet® International Outcome Study (IOS) and the American Norditropin® Studies: Web Enabled Research (ANSWER Program®) are two complementary, non-interventional, observational studies that adhere to current guidelines for pharmacoepidemiological data.
Patients: The studies include pediatric and adult patients receiving Norditropin®, as prescribed by their physicians.
Measurements: The studies gather long-term data on the safety and effectiveness of real-life treatment with the recombinant human GH, Norditropin®. We describe the origins, aims, objectives, and design methodology of the studies, as well as their governance and validity, strengths, and limitations.
Conclusion: The NordiNet® IOS and ANSWER Program® studies will provide valid insights into the effectiveness and safety of GH treatment across a diverse and large patient population treated in accordance with real-world clinical practice and following the Good Pharmacoepidemiological Practice and STrengthening the Reporting of OBservational studies in Epidemiology (STROBE) guidelines.
Keywords: growth hormone replacement therapy, treatment outcome, pharmacoepidemiology, survey
This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.Download Article [PDF] View Full Text [HTML]
Readers of this article also read:
Effects of a pain self-management intervention combining written and video elements on health-related quality of life among people with different levels of education
Stalker C, Elander J
Published Date: 20 August 2015
George LA, Camire RM
Published Date: 24 April 2015
Patient preference and ease of use for different coagulation factor VIII reconstitution device scenarios: a cross-sectional survey in five European countries
Cimino E, Linari S, Malerba M, Halimeh S, Biondo F, Westfeld M
Published Date: 12 December 2014
Published Date: 13 October 2014
Second case report of successful electroconvulsive therapy for a patient with schizophrenia and severe hemophilia A
Saito N, Shioda K, Nisijima K, Kobayashi T, Kato S
Published Date: 16 May 2014
Khair K, Holland M
Published Date: 6 May 2014
Published Date: 16 May 2013
The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia
Rivka Yatuv, Micah Robinson, Inbal Dayan-Tarshish, et al
Published Date: 6 August 2010
Published Date: 28 June 2010