CONTENT DEVELOPMENT FOR A NEW INSTRUMENT TO ASSESS PATIENT AND PARENT PREFERENCE FOR GROWTH HORMONE REPLACEMENT THERAPY DELIVERY DEVICES

Author(s)

Jennifer M Stephens, PharmD, Clinical Director1, Kimbach Tran Carpiuc, MPH, MS, Scientist1, Karen F Gold, PhD, Adjunct Professor2, Pablo Altman, MD, MBA, Clinical Scientific Director3, John Germak, MD, Clinical Director, Growth Hormone3, Ashish V. Joshi, PhD, Senior Manager, Health Economics31Pharmerit North America LLC, Bethesda, MD, USA; 2 Harvard University, Cambridge, MA, USA; 3 Novo Nordisk Inc, Princeton, NJ, USA

Objective: Few patient-reported outcome measures for growth hormone (GH) replacement therapy have been formally validated and none address preference for a particular delivery method. Our objective was to formally develop a new instrument to assess child and parent satisfaction with and preference for GH therapy delivery devices. Methods: An instrument item pool was generated by combining the following four steps: 1) thorough literature review to identify relevant domains and questions, 2) consultation with clinicians knowledgeable about treatment of GH deficiency, 3) identification of indicators specific to GH delivery devices, and 4) input from families with children aged 10-16 years receiving GH therapy, including both new (2 to 12 weeks) and established (= 12 weeks) users. Structured feedback forms were completed during cognitive interviews with focus groups on relevance of questions, missing topics, understandability, and feasibility of use. Based on the above steps, a preliminary instrument was created for validation testing. Results: Key themes generated from literature, clinicians, and families included various aspects of pain, difficulty using the device, embarrassment, and time involved affecting productivity, convenience, and compliance. Two versions of the preference instrument were created to reflect the child and parent perspectives. A 4-point Likert scale was used for most questions. The child version included 38 survey items on clarity of instructions (2), preparation (7), administration (4), convenience (3), pain (5), embarrassment (2), anxiety (2), productivity (4), compliance (3), mood (1), and overall satisfaction (5). All items from the child questionnaire were included in the parent version, supplemented with five questions regarding administration, parent productivity, and parent overall satisfaction. Conclusion: Areas of concern for families using GH delivery devices include pain, productivity, and convenience. This new instrument, which may offer clinicians and researchers an opportunity to evaluate different device alternatives for GH replacement therapy, will soon undergo formal validation testing.

Conference/Value in Health Info

2008-05, ISPOR 2008, Toronto, Ontario, Canada

Value in Health, Vol. 11, No. 3 (May/June 2008)

Code

PDB57

Topic

Patient-Centered Research

Topic Subcategory

Stated Preference & Patient Satisfaction

Disease

Diabetes/Endocrine/Metabolic Disorders

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