Conducting Drug Treatment Trials in Children: Opportunities and Challenges.

Children were often referred to as "therapeutic orphans" in the past due to different reasons such as ethical, regulatory, economic, scientific, etc., ones. They were exposed to avoidable risks while missing out on therapeutic advances. Pediatric patients have suffered from a lack of scientific and regulatory standards (e.g., proper drug testing, authorization of medicines for their use, etc.), although the pharmaceutical legislative framework, which ensures the high standards of safety, quality, and efficacy of medicinal products for use in adults, was developed primarily in response to past "drug disasters," mainly involving children. The adoption of pediatric regulatory initiatives first in the USA and then in Europe and other countries and regions has significantly changed the worldwide frameworks and permanently changed pediatric drug research and development. This article tries to give various perspectives with historical context, a review of the different challenges and opportunities as well as important stakeholders in pediatric drug development. The pediatric trial networks are probably the most important stakeholder that enables efficient patient recruitment, access to better resource utilization, and global collaboration of different stakeholders necessary for performing quality and well-designed clinical trials.

How to Distinguish Idiopathic Achalasia from Pseudoachalasia?

Achalasia is a disorder characterized by insufficient relaxation of the lower esophageal sphincter and absent peristalsis. It is usually primary (idiopathic) achalasia, but it can also be secondary achalasia (pseudoachalasia). With a review of the literature, epidemiological data was gathered on achalasia in this paper, and followed up with presented etiopathogenetic mechanisms of achalasia. An optimal diagnostic algorithm along with the therapeutic possibilities in achalasia, from endoscopic to surgical methods of therapy, was analyzed. The most common causes of pseudoachalasia and the mechanisms of this disorder are reported. In patients with dysphagia and suspected achalasia, it is important to distinguish idiopathic achalasia from pseudoachalasia, since pseudoachalasia most often occurs due to tumor infiltrations of the lower esophageal sphincter. In this paper, the importance of a timely and accurate diagnosis of pseudoachalasia in relation to idiopathic achalasia was shown.