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

Charlotte Höybye, Department of Molecular Medicine and Surgery, Karolinska Institute and Department of Endocrinology, Metabolism and Diabetes, Stockholm, Sweden
Lars Sävendahl, Department of Women’s and Children’s Health, Karolinska Institute and Division of Pediatrics, Karolinska University Hospital, Stockholm, SwedenFollow
Henrik Thybo Christesen, Hans Christian Andersen Children’s Hospital, Odense University Hospital, Odense, DenmarkFollow
Peter Lee, Department of Pediatrics, Penn State College of Medicine, Hershey, PA, USAFollow
Birgitte Tønnes Pedersen, Global Development, Novo Nordisk A/S, Søborg, DenmarkFollow
Michael Schlumpf, Global Medical Affairs Biopharm, Novo Nordisk Health Care AG, Zurich, Switzerland
John Germak, Clinical Development and Medical Affairs, Novo Nordisk Inc, Princeton, NJ, USAFollow
Judith Ross, Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA, USAFollow

Document Type

Article

Publication Date

4-24-2013

Comments

This article has been peer reviewed. It was published in: Clinical Epidemiology.

Volume 5, Issue 1, 24 April 2013, Pages 119-127.

The published version is available at http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3641810/

DOI: 10.2147/CLEP.S42602

Copyright © 2013 Höybye et al, publisher and licensee Dove Medical Press Ltd.

Abstract

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 reallife 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.

Recommended Citation

Höybye, Charlotte; Sävendahl, Lars; Christesen, Henrik Thybo; Lee, Peter; Pedersen, Birgitte Tønnes; Schlumpf, Michael; Germak, John; and Ross, Judith, "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®)." (2013). Department of Pediatrics Faculty Papers. Paper 50.
https://jdc.jefferson.edu/pedsfp/50

PubMed ID

23658497