Study Design in Osteoporosis: A European Perspective

Authors

  • Ja Kanis MD,

    Corresponding author
    1. WHO Collaborating Centre for Metabolic Bone Diseases, University of Sheffield Medical School, Sheffield, United Kingdom
    • WHO Collaborating Centre for Metabolic Bone Diseases, University of Sheffield Medical School, Sheffield S10 2RX, UK
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    • Dr Kanis serves as a consultant and/or receives research funding from the following companies: Abiogen, Italy; Aventis, France; Bayer, Germany; Besins Iscovesco, France; Biosintetica, Brazil; Boehringer Ingelheim, UK; Boehringer Mannheim, Germany; Catalyst, UK; Celtrix, USA; Eli Lilly, USA; Gador, Argentina; Glaxo, UK; Health Technology Assessment, UK; Hoechst Marion Roussel, France; Hologic, Belgium; Hologic, USA; IGEA, Italy; Kissei, Japan; Lehman Brothers, UK; Leiras, Finland; Leo Pharma, Denmark; Lilly, UK; Merck Research Labs, USA; Merlin Ventures, UK; MRL, China; Novartis, Switzerland; Novo Nordisk, Denmark; Nycomed, Norway; Organon, Holland; OsteoMeter, Denmark; Parke-Davis, USA; Pfizer, USA; Procter and Gamble, USA; Roche, Germany; Roche, Switzerland; Rhone-Poulenc Rorer, France; Rotta Research, Italy; Schering, Berlin; Shire, UK, Strakan, UK, Strathmann, Germany; Teijin, Japan; Teva, Israel; Theramex, Monaco; Trent Health, UK; Unigene, USA; Warburg-Pincus, UK; Warner-Lambert, USA; Warner-Lambert, USA, WHO, Geneva. Dr Kanis has equity interest in: British Biotek, UK; Celtrix, USA; Glaxo, UK; Lilly, USA; Merck, USA, Novo Nordisk, Denmark; Procter and Gamble, USA, Shire, UK, Strakan, UK and Unigene USA

  • J-M Alexandre,

    1. Hopital Europeen George Pompidou, Paris, France
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  • Hg Bone,

    1. Michigan Bone and Mineral Clinic, Detroit, Michigan, USA
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  • E Abadie,

    1. AFFSAPS, St Denis, France
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  • D Brasseur,

    1. Belgium Ministry of Public Health, Brussels, Belgium
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  • O Chassany,

    1. DRRC Saint Louis Hospital, Paris, France
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  • S Durrleman,

    1. Sanofi Laboratories, Paris, France
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  • Jff Lekkerkerker,

    1. Medical Evaluation Board, The Hague, The Netherlands
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  • F Caulin

    1. FC Consulting SA, Paris, France
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  • See footnote after references for the conflict of interest statement for Dr Kanis. Dr Bone has received recent research support and/or has acted as a scientific consultant to the following companies: Amgen, GlaxoSmithKline, Merck, NPS Pharmaceuticals, Nordic Bioscience, Novartis, Pharmacia, Pfizer, Roche, and Schering Plough. All other authors have no conflict of interest

Abstract

The advent of effective agents for the treatment of osteoporosis has led to the view that placebo-controlled trials to test new agents for efficacy are no longer appropriate. Rather, studies of superiority, equivalence, or non-inferiority have been recommended. Such studies require very large sample sizes, and the burden of osteoporotic fracture in a trial setting is substantially increased. Studies of equivalence cannot be unambiguously interpreted because the variance in effect of active comparator agents is too large in osteoporosis. If fracture studies are required by regulatory agencies, there is still a requirement for placebo-controlled studies, although perhaps of shorter duration than demanded at present.

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