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®)
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 by Dove Medical Press Limited, and licensed under Creative Commons Attribution - Non Commercial (unported, v3.0) License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php
Readers of this article also read:
Noninvasive detection of macrophages in atherosclerotic lesions by computed tomography enhanced with PEGylated gold nanoparticles
Qin J, Peng C, Zhao B, Ye K, Yuan F, Peng Z, Yang X, Huang L, Jiang M, Zhao Q, Tang G, Lu X
Published Date: 2 December 2014
Mesenchymal stromal cell labeling by new uncoated superparamagnetic maghemite nanoparticles in comparison with commercial Resovist – an initial in vitro study
Skopalik J, Polakova K, Havrdova M, Justan I, Magro M, Milde D, Knopfova L, Smarda J, Polakova H, Gabrielova E, Vianello F, Michalek J, Zboril R
Published Date: 20 November 2014
Self-assembled nanoparticles based on the c(RGDfk) peptide for the delivery of siRNA targeting the VEGFR2 gene for tumor therapy
Liu L, Liu X, Xu Q, Wu P, Zuo X, Zhang J, Deng H, Wu Z, Ji A
Published Date: 29 July 2014
Xia S, Li P, Chen Q, Armah M, Ying X, Wu J, Lai J
Published Date: 23 May 2014
Efficiently engineered cell sheet using a complex of polyethylenimine–alginate nanocomposites plus bone morphogenetic protein 2 gene to promote new bone formation
Jin H, Zhang K, Qiao C, Yuan A, Li D, Zhao L, Shi C, Xu X, Ni S, Zheng C, Liu X, Yang B, Sun H
Published Date: 7 May 2014
Preparation and characterization of polylactide/poly(ε-caprolactone)-poly(ethylene glycol)-poly(ε-caprolactone) hybrid fibers for potential application in bone tissue engineering
Wang YL, Guo G, Chen HF, Gao X, Fan RR, Zhang DM, Zhou LX
Published Date: 17 April 2014
Astigmatism induced by conventional spherical ablation after PRK and LASIK in myopia with astigmatism < 1.00 D
Christiansen SM, Mifflin MD, Edmonds JN, Simpson RG, Moshirfar M
Published Date: 20 December 2012
Mannan-modified Ad5-PTEN treatment combined with docetaxel improves the therapeutic effect in H22 tumor-bearing mice
Liu Z, Li J, Li J, Huang J, Ke F, Qi Q, Jiang X, Zhong Z
Published Date: 17 September 2012
Cellular interaction of folic acid conjugated superparamagnetic iron oxide nanoparticles and its use as contrast agent for targeted magnetic imaging of tumor cells
Kumar M, Singh G, Arora V, Mewar S, Sharma U, Jagannathan NR, Sapra S, Dinda AK, Kharbanda S, Singh H
Published Date: 6 July 2012
Sun S, Liang N, Kawashima Y, Xia D, Cui F
Published Date: 28 November 2011