Therapeutic orphans, off-label, pediatric drug development: towards reasonable pharmacotherapy for minors.

Introduction: The concept that children are therapeutic orphans emerged in the 1960s, triggering eventually worldwide legislation to facilitate pediatric studies, called 'Pediatric Drug Development (PDD).' However, PDD's true aim is not better medicines for children but labels in minors; minors are not another species.

Areas Covered: Absorption, distribution, metabolism, and excretion (ADME) differ in preterm newborns, but babies mature.

Pharmacogenomics and pediatric drug development: science and political power. A narrative review.

Introduction: Pharmacogenomics (PGx) investigates how genomes control enzyme expression. Developmental pharmacology (DP) describes the temporal sequence of enzymes impacting absorption, distribution, metabolism, and excretion (ADME) of food and drugs.

Areas Covered: US and European Union (EU) legislation facilitate and/or enforce pediatric studies for all new drugs, called overall 'pediatric drug development' (PDD).

"Pediatric" Drug Studies Might Be the Largest Abuse in Medical Research in History. It Is Time for Lawyers to Step In.

A new type of research has emerged with United States and European Union pediatric laws that request/demand separate clinical studies for vaccines and drugs in minors less than 18 years of age. Physiologically, minors mature before their 18th birthday. Medicine treats the body, not the administrative status.

Pharmacokinetic considerations surrounding the use of levetiracetam for seizure prophylaxis in neurocritical care - an overview.

Introduction: Levetiracetam (LEV) is one of the most widely used anti-seizure medications (ASMs) in clinical practice. This is due both to a different mechanism of action when compared to other ASMs and its easy handling. Indeed, because of its interesting pharmacokinetic properties, it is often used outside of the labeled indications, notably in the neurocritical setting as prophylaxis of epileptic seizures.

Neurology's vital role in preventing unnecessary and potentially harmful pediatric studies.

Introduction: Regulatory authorities recognize two human populations: adults and children defined as <18 years. For drug approval, they demand separate studies. But humans mature slowly during puberty.

COVID-19 and Treatment and Immunization of Children-The Time to Redefine Pediatric Age Groups is Here.

Children are infected with coronavirus disease 2019 (COVID-19) as often as adults, but with fewer symptoms. During the first wave of the COVID-19 pandemic, multisystem inflammatory syndrome (MIS) in children (MIS-C), with symptoms similar to Kawasaki syndrome, was described in young minors testing positive for COVID-19. The United States (US) Centers for Disease Control and Prevention (CDC) defined MIS-C as occurring in <21-year-olds, triggering hundreds of PubMed-listed papers.

The Challenges of Pediatric Drug Development.

Introduction And Background: "Pediatric Drug Development" is being used to describe not the development of drugs for children, but rather the planning & conducting separate efficacy and safety (E&S) studies requested/demanded by regulatory authorities designed to produce pediatric labels. Pediatric studies required for drug approval enroll "children"; defined as <17 years of age (US Food and Drug Administration [FDA])/ <18 years (European Union [EU]). The medical rationale for study designs was examined.

Questionable Industry-Sponsored Postneonatal Pediatric Studies in Slovenia.

Background: US and EU pediatric laws promote industry-sponsored pediatric studies, based on the therapeutic orphans concept that claims discrimination of children in drug treatment and drug development.

Objective: We investigated the medical validity of international pediatric studies with centers in Slovenia, an EU member state, and challenge their medical utility.

Methods: We analyzed international industry-sponsored pediatric studies with centers in Slovenia, listed in www.

Rational Use of Medicine in Children-The Conflict of Interests Story. A Review.

Background: United States (US) and European Union (EU) legislation attempts to counterbalance the presumed discrimination in pediatric drug treatment and development.

Methods: We analyzed the history of drug development, US/EU pediatric laws, and pediatric studies required by US/EU regulatory authorities and reviewed relevant literature.

Results: The US and EU definitions of a child are defined administratively (rather than physiologically) as being aged <17 years and <18 years, respectively.

The Meanings of "Pediatric Drug Development".

Pediatric drug development (PDD) became an industry goal when the Food and Drug Administration (FDA) granted patent extensions. This was later expanded to obligations for pediatric studies and to the European Medicines Agency's (EMA's) strict pediatric investigation plans (PIPs). Industry now sponsors many often international studies in young patients that are difficult or impossible to recruit.

Pediatric Melanoma and Drug Development.

Importance-Pediatric melanoma occurs, albeit rarely. Should patients be treated by today's medical standards, or be subjected to medically unnecessary clinical studies? Observations-We identified international, industry-sponsored pediatric melanoma studies triggered by regulatory demands in www.clinicaltrials.

Current state and future of pediatric allergology in Europe: A road map.

The history of pediatric allergology (PA) in Europe is relatively youthful, dating back to 1984, when a small group of pediatricians founded the European Working Group on Pediatric Allergy and Immunology-later giving rise to ESPACI (European Society on Pediatric Allergology and Clinical Immunology). In 1990, the first dedicated journal, Pediatric Allergy and Immunology (PAI), was founded. There are striking differences across Europe, and even within European countries, in relation to the training pathways for doctors seeing children with allergic disease(s).