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

ABSTRACT

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. The 18^th birthday is an administrative limit that does not correspond to a physiological change. Separate drug approval before/after the 18^th birthday reflects the children-are-therapeutic-orphans concept that emerged after 1962. The Food and Drug Administration (FDA) has backed away from this concept for antiepileptic drugs, but sticks to it in other areas. In contrast, the European Medicines Agency (EMA) is continuously expanding its demand for ‘pediatric’ studies. Parents hesitate increasingly to let their children participate in questionable studies.

Areas covered

Neurologists challenge the children-are-therapeutic-orphans mantra. Young patients do not need separate proof of efficacy & safety, but appropriate dosing recommendations. Minors should be treated as human beings, instead of being abused in questionable studies.

Expert opinion

Young patients with multiple sclerosis and other neurological diseases deserve studies with therapeutic intentions. ‘Pediatric’ careers have emerged in academia, regulatory authorities, and pharmaceutical companies. Institutional Review Boards/ Ethics Committees should suspend questionable ‘pediatric’ studies and reject newly submitted ones. The medical professions should distance themselves from questionable ‘pediatric’ research that reflects massive conflicts of interest.

KEYWORDS:

• Multiple sclerosis
• pain
• pediatric drug development
• developmental pharmacology
• pediatric legislation
• children as therapeutic orphans

Confirmation

We confirm that all our tables are original and have not previously been published

Article highlights

• Separate adult and pediatric approval of drugs for epilepsy, multiple sclerosis, pain, cerebral palsy, & more attribute a physiological meaning to the 18th birthday that does not exist. Drugs treat patients, not their legal status.

• Placebo-controlled proof-of-efficacy studies in ‘children’ prove again that drugs work before and after the 18th birthday. Such trials are questionable.

• We welcome that the International Pediatric Multiple Sclerosis Study Group (IPMSSG) has challenged separate ‘pediatric’ studies in multiple sclerosis.

• Peer-reviewed journals should no longer accept flawed formulations such as ‘adult-onset multiple sclerosis’ (AOMS) vs. ‘pediatric-onset multiple sclerosis’ (POMS).

• Institutional Review Boards (IRBs)/ ethics committees (ECs) should suspend questionable ‘pediatric’ studies and should reject newly submitted ones.

Declaration of interests

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.