Missing trials in drug regulatory dossiers may have good reasons, but should be predefined and transparent

Hubert G Leufkens

doi:10.1016/j.jclinepi.2021.10.026

Abstract

Boesen and colleagues have done a tremendous job in addressing the question: when I have a list of clinical trials of a class of long-acting medicinal products containing methylphenidate, and I want to see whether these trials also pop-up in the regulatory dossiers that are assumed to deal with the licensing process of these products for use in adult patients with ADHD, what do I see? [1]. At first glance a very simple and logical question for everybody interested in how regulatory processes function and whether these processes contribute to public health and patient safety.

Highlights

The case selection for the study of long-acting methylphenidate indicated for adult patients with ADHD
More importantly there has been a whole array of different views in the medical community of psychiatrists and patient organizations about the therapeutic value that methylphenidate indicated for adult patients with ADHD could bring
Not to forget, in Europe long-acting methylphenidate products have been regulated by National Competent Authorities (e.g. MHRA, Bfarm, MEB) with only a limited role of the EMA in the primary licensing process

Summary

Introduction

The case selection for the study of long-acting methylphenidate indicated for adult patients with ADHD. We have shown for oncology products for instance that the regulators respond to this with restrictions in the label, i.e. more narrow indication or request for extra data and evidence to justify the claim [6]. The labels for OROS methylphenidate products authorized by Health Canada, TGA, FDA and MRHA imply for the indication ADHD in adults the presence of hyperactive-impulsive or inattentive symptoms that were present before age 7 years.

Results

Conclusion