European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials.

Development of novel therapies for Duchenne muscular dystrophy (DMD) are driving the need for more efficient ways of detecting changes in disease- progression in DMD [1]. However, medicines' approval must be based on outcome measures that are acceptable from a regulatory perspective. In this article, European regulators provide an update on the recent regulatory consideration of a new endpoint (Stride Velocity 95th Centile (SV95C)) that could be used in therapeutic DMD trials.

[Future Perspective of Pharmacoepidemiology in the "Big Data Era" and the Growth of Information Sources].

The arrival of new drug into the market requires many years of previous research along with the need of continuous evaluation throughout the lifetime of the drug. This warrants pharmacoepidemiological research which may be defined as the study of the use and the effects of drugs in large populations. Nowadays this type of research seems more feasible thanks to the massive expansion of the information sources and data (e.

[Clinical-Epidemiological Profile of Patients Initiating Intensive Statin Therapy for the Secondary Prevention of Vascular Disease in Spain].

Background: The new recommendations regarding the utilization of high potency statins (intensive therapy) for the treatment of cardiovascular disease have been based on the extrapolation of data coming from clinical trials. The objective is to describe the clinical-epidemiological profile of statin therapy users for the secondary prevention of cardiovascular disease in Spain and to examine the predictors for intensive therapy initiation.

Methods: Cross-sectional study from a sample of 88,751 patients aged ≥45 years-old with previous cardiovascular disease which initiated statin therapy between 1st January 2007 to 31st December 2011.

The placebo arm in clinical studies for treatment of psychiatric disorders: a regulatory dilemma.

Background: The use of placebo in clinical trials, and, related to this, ethical and feasibility aspects, are often debated. However, regulatory authorities must ensure that only new drugs with a positive benefit/risk would be granted a marketing authorization. It is therefore not surprising that they often put forward the need for placebo control in clinical trials in an area where many trials fail, and assay sensitivity is not self-evident.

Common definition for categories of clinical research: a prerequisite for a survey on regulatory requirements by the European Clinical Research Infrastructures Network (ECRIN).

Background: Thorough knowledge of the regulatory requirements is a challenging prerequisite for conducting multinational clinical studies in Europe given their complexity and heterogeneity in regulation and perception across the EU member states.

Methods: In order to summarise the current situation in relation to the wide spectrum of clinical research, the European Clinical Research Infrastructures Network (ECRIN) developed a multinational survey in ten European countries. However a lack of common classification framework for major categories of clinical research was identified, and therefore reaching an agreement on a common classification was the initial step in the development of the survey.

Albuminuria as a surrogate marker for drug development: a European Regulatory perspective.

Clinical trials with surrogate end points generally require smaller sample sizes and shorter duration than those with clinical outcomes. However, in practice, surrogate end points are likely to result in less comprehensive conclusions than 'outcome' end points as they sometimes reflect only partial aspects of complex phenomena. A number of conditions help to establish the validity of a particular variable as predictive of clinical outcome.