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Table 4 Impact of PRO trial data: a practical example

From: The impact of patient-reported outcome data from clinical trials: perspectives from international stakeholders

Tocilizumab, a new treatment for rheumatoid arthritis (RA) in adults and juvenile idiopathic arthritis (JIA) in children showed significant improvements.
Patients improved 30% or more on at least three of the six variables in the American College of Rheumatology (ACR) core set for JIA, with no more than one variable worsening by more than 30%. Furthermore, patients showed improved symptoms such as absence of fever and rash, as measured with the Disability Index of the Childhood Health Assessment Questionnaire (CHAQ-DI) and; improved laboratory abnormalities (anemia, thrombocytosis, and hyperferritinemia) – primary outcome [39].
Drug approval: Tocilizumab was approved in 2009 by the EMA (European Medicines Agency) [40] and by the FDA in January 2010 [41] for use in RA based on clinical and PRO findings.
Health improvements: Tocilizumab has been supported for prescription by NICE in the UK for patients with severe RA. There is evidence that tocilizumab halts joint damage, improves function and increases quality of life [39]. Significantly more patients treated with tocilizumab showed improvements of ≥0.3 units in the HAQ-DI score compared to patients treated with placebo [39].
Informed cost-effectiveness: The National Institute for Health and Clinical Excellence (NICE) recommended the use of tocilizumab for systemic onset JIA, the most severe form of JIA, in 2011. The manufacturer’s submission mapped the CHAQ-DI scores to utilities, using a mapping formula derived in adults with rheumatoid arthritis that mapped Health Assessment Questionnaire [HAQ] results onto EQ-5D utilities to determine the effectiveness of the intervention [42].
Reduction in the number of work loss days: according to a case study presented by the National Rheumatoid Arthritis Society, patients under tocilizumab are able to return to work as the drug halts joint damage and improves function [43].
Income from the intellectual property:Roche, manufacturer of the drug, reported 496 m CHF (£335 m) in sales of the drug in just the first half of 2013 (up 33% on the previous year, due to increasing demand) [44].