Unique Burdens of Pediatric Clinical Trials in Duchenne Muscular Dystrophy, April 20-21, 2017, Bethesda, Maryland, USA

Franson T, Kinnett K, Cripe TP. Unique Burdens of Pediatric Clinical Trials in Duchenne Muscular Dystrophy, April 20-21, 2017, Bethesda, Maryland, USA. Therapeutic innovation & regulatory science 2018:2168479018764650.

Franson_2018_Ther Innov Regul Sci. Jan 1;2168479018764650

The number of clinical trials in DMD is increasing. Participation in clinical trials has a burden on children and their families. A workshop was organised addressing the main burdens (study protocol, financial psychosocial and emotional, and informed consent) and gave recommendation to address and minimizeĀ these burdens in clinical trials.

 

Abstract

Recent increases in the number and breadth of clinical trials for patients with Duchenne muscular dystrophy (DMD) have engendered hope for a better future. Despite the overall enthusiasm by the DMD community for these trials, however, the burdens and pressures that they place on children with Duchenne muscular dystrophy and their families have become painfully apparent. In order to identify, and mediate, these challenges, Parent Project Muscular Dystrophy (PPMD) sponsored a meeting to examine some of these issues more closely in Bethesda, Maryland, on April 20-21, 2017. The meeting focused on key burdens for patients participating in clinical trials including technical (protocol complexity), financial, psychosocial and emotional issues, and informed consent. Participants recommended mitigation strategies falling into clinical, operations, regulatory, and ethical domains. The development of consensus action plans for short- and long-term enhancements in trials should facilitate discovery and development research for DMD patients.

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