Summary of recommendations for the design of clinical trials and the registration of drugs used in the treatment of asthma

Holgate, S.T., Bousquet, J., Chung, K.F., Bisgaard, H., Pauwels, R., Fabbri, L., Rabe, K., Doherty, M., Snell, N.J.C., Cuss, F., D'Amato, M. and Reginster, J.Y. , Group for the Respect of Ethics and Excellence in Science (GREES): Asthma section (2004) Summary of recommendations for the design of clinical trials and the registration of drugs used in the treatment of asthma. Respiratory Medicine, 98 (6), 479-487. (doi:10.1016/j.rmed.2003.12.005).

Abstract

With new drugs being introduced to treat asthma it is timely to review criteria that can be used to assess efficacy in clinical trials. Anti-asthma drugs are classified into symptoms-modifying, symptom preventers and disease modifying agents. Attention is drawn to the types of experimental evidence required in preclinical studies to support further clinical development of a new therapy. Clinical trials demand careful selection of patients to maximise the strength of the efficacy signal according to the type of trial being designed. While provocation tests are useful in suggesting efficacy, negative tests do not necessarily indicate lack of anti-asthma activity. Therapeutic trial designs need to take account of duration of treatment, dose–response relationships and confirmatory trials. Outcome measures include symptoms, lung function, reduction in concomitant medication, exacerbations, quality of life and measures of inflammation. Interpretation of results need to include the clinical relevance of any changes as well as statistical significance. Special consideration needs to be given to the evaluation of drugs for acute severe asthma, asthma in children and older people, co-morbidity such as rhinitis, and inhaler devices. As with all drugs introduced into practice, careful attention needs to be paid to both short- and long-term safety.

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