OBJECTIVES: The study aim is to investigate the parallel qualification procedure established between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) for the development of novel patient reported outcome instruments (PROs).

METHODS: A comprehensive literature review was conducted in EMA and US FDA websites pertaining to the EMA/FDA parallel qualification procedure and in PubMed®. The regulatory framework for the EMA/FDA Parallel qualification procedure was identified and the published qualification decisions were extracted.

RESULTS: The parallel qualification is a stand-alone procedure, intended to provide simultaneously advice from the EMA and FDA for the development of novel PRO instruments applicable for multiple clinical development programs. Although each Agency follows its internal rules during the qualification assessment, this procedure reduces the administrative burden, supports the regulatory bodies information scientific sharing to reach a convergence during the decision-making process to optimize the development of novel PRO instruments.

Both Agencies publish independently their final qualification decisions on their websites. The use of the PRO instruments is specified in respect to the disease, trial population, type of the trials, phase of the clinical development program, endpoints positioning, target claims and can support label claims and so the direct advertising to patients in US, irrespective of the drug prescription status.

So far 3 tools were submitted for parallel qualification: the Exacerbations of Chronic Pulmonary Disease Tool (EXACT), EXACT respiratory symptoms tool (E-RS: COPD), the Modified Pediatric Ulcerative Colitis Activity Index (PUCAI) / TUMMY Scale.

CONCLUSIONS:The EMA/FDA dialog offers the option to obtain simultaneously advice for the development of novel PRO instruments applicable for multiple clinical development programs. The procedure allows dialogue and alignment between FDA and EMA for the development of novel PRO instruments. It will likely become the preferred route for PRO development intended to inform drug development.