Assessing effectiveness of pulmonary arterial hypertension therapies in daily practice

In clinical trials of pulmonary arterial hypertension, disease progression is measured using the composite endpoint, time to clinical worsening, which may provide the best indication of clinical status and disease progression. However, as is the case for all therapeutic areas, the great challenge is to translate results from these studies into day-to-day clinical practice. Ideally, the goal of targeted therapy is to normalize patients, but this is not possible in the vast majority of patients. Treatment goals need to be carefully defined in order to correctly measure whether a treatment is successful and indicate when additional treatment is required.

Goal-orientated therapy is now becoming an accepted treatment strategy, whereby goals and follow-up procedures are predefined, and may be more appropriate than the traditional approach used in randomized controlled trials for demonstrating efficacy and for translating this into clinical practice. It is important to tailor such therapy to individual patients and to take all measured parameters into account.

Currently, there is a wide gap between how clinical worsening is measured during clinical trials, and the indicators used in day-to-day clinical practice. Whichever indicators of disease severity are selected should correlate closely with clinical outcome (i.e. survival) for all patients.