December 9, 2014
An article published in the Journal of Huntington’s Disease details the outcome measures commonly used in Huntington Disease (HD) trials. Identification of the gene mutation causing HD symptoms has raised hopes for new treatments. Designing efficient clinical trials and identifying optimal outcome measures are therefore essential to develop innovative therapies. While the National Institute of Neurological Disorders and Stroke (NINDS) has published recommendations for measurement selection in HD, data published on many of the recommended measures is still very scarce. According to the authors, clinical outcomes assessments generally fall into one of three categories: patient-reported outcome (PRO) assessment, observer reported outcome assessment (ObsRO) and clinical-reported outcome (ClinRO) assessment. The authors conducted a systematic review of the literature to identify the frequency of usage of these outcome measures. The authors found that ClinRO measures are used most commonly in HD drug studies. Few studies use PROs and almost no studies use ObsRO. The authors believe that much work is still needed before specific recommendations for HD PROs and ObsRO measures are made.