Developing drugs for treatment of atopic dermatitis in children (≥3 months to <18 years of age): Draft guidance for industry

Abstract

Atopic dermatitis is the most common chronic skin disease, and it primarily affects children. Although atopic dermatitis (AD) has the highest effect on burden of skin disease, no high-level studies have defined optimal therapy for severe disease. Corticosteroids have been used to treat AD since the 1950s and remain the only systemic medication with Food and Drug Administration approval for this indication in children, despite published guidelines of care that recommend against this option. Several clinical trials with level 1 evidence have supported the use of topical treatments for mild to moderate atopic dermatitis in adults and children, but these trials have had little consistency in protocol design. Consensus recommendations will help standardize clinical development and trial design for children. The Food and Drug Administration issues guidance documents for industry as a source for "the Agency's current thinking on a particular subject." Although they are nonbinding, industry considers these documents to be the standard for clinical development and trial design. Our consensus group is the first to specifically address clinical trial design in this population. We developed a draft guidance document for industry, Developing Drugs for Treatment of Atopic Dermatitis in Children (≥3 months to <18 years of age). This draft guidance has been submitted to the Food and Drug Administration based on a provision in the Federal Register (Good Guidance Practices).

Optimization of placebo use in clinical trials with systemic treatments for atopic dermatitis: an International Eczema Council survey‐based position statement

“…Patients randomized to placebo may not receive standard‐of‐care, effective treatment . Consequently, AD patients with intractable itch, sleep loss and reduced quality of life would be left untreated for extended periods of time . The World Medical Association Declaration of Helsinki promotes ACT as the design of choice, with PCT considered only with a robust scientific methodological justification and when patients do not face severe risks, as most would consider is the case of AD…”

Use of dupilimab in pediatric atopic dermatitis: Access, dosing, and implications for managing severe atopic dermatitis

“…Until optimal pediatric dupilumab dosing and administration have been defined, clinicians caring for children with severe AD that require systemic treatment are between a rock and a hard place. Fortunately, the pipeline for AD is robust and clinical trials are including pediatric patients earlier in development . But until other options are available, dupilumab may be the best choice, despite its limitations.…”

Pediatric melanoma—The whole (conflicts of interest) story

“…They do not address conflicts of interest of regulatory authorities and some pediatric researchers. They need an update to prevent authors from regulatory authorities from not revealing their conflicts of interest (Mentzer, 2014, Saint-Raymond and Brasseur, 2005, Tomasi et al, 2017, Tsukamoto et al, 2016, Wharton et al, 2014) and pediatric researchers from omitting FDA/EMA decisions that trigger payment by companies (Casanova et al, 2016, Falkner, 2017, Fraser et al, 2014, Gaspar et al, 2018, Geoerger et al, 2012, Geoerger et al, 2017, Hoppu et al, 2012, Jeha et al, 2006, Merchant et al, 2016, Moreno et al, 2018, Pearson et al, 2017, Ruperto et al, 2013, Siegfried et al, 2018, Snyder et al, 2013, Torok et al, 2018, Turner et al, 2014, Turner et al, 2017, Ward et al, 2018).…”