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Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?
Stephenson, Diane; Ollivier, Cecile; Brinton, Roberta; Barrett, Jeffrey.
  • Stephenson D; Critical Path Institute, Tucson, Arizona, USA.
  • Ollivier C; Critical Path Institute, Tucson, Arizona, USA.
  • Brinton R; Center for Innovation in Brain Sciences, University of Arizona Health Sciences, Tucson, Arizona, USA.
  • Barrett J; Critical Path Institute, Tucson, Arizona, USA.
Clin Pharmacol Ther ; 111(4): 799-806, 2022 04.
Article in English | MEDLINE | ID: covidwho-1626126
ABSTRACT
Global regulatory agencies have transformed their approach to approvals in their processes for formal review of the safety and efficacy of new drugs. Opportunities for innovation have expanded because of the coronavirus disease 2019 (COVID-19) pandemic. Several regulatory-led initiatives have progressed rapidly during the past year, including patient-focused drug development, model-informed drug development, real-world evidence, and complex innovative trial designs. Collectively, these initiatives have accelerated the rate of approvals. Despite demands to focus on urgent needs imposed by the COVID-19 pandemic, the number of new drug approvals over the past year, particularly for rare diseases, has outpaced expectations. Advancing therapeutics for nervous system disorders requires adaptive strategies that align with rapid developments in the field. Three relentlessly progressive diseases, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and Parkinson's disease are in urgent need of new treatments. Herein, we propose new regulatory initiatives, including innovative trial designs and patient-focused drug development that accelerate clinical trial conduct while meeting critical regulatory requirements for therapeutic approval.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: Muscular Dystrophy, Duchenne / COVID-19 Drug Treatment Type of study: Prognostic study / Randomized controlled trials Limits: Humans Language: English Journal: Clin Pharmacol Ther Year: 2022 Document Type: Article Affiliation country: Cpt.2528

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Muscular Dystrophy, Duchenne / COVID-19 Drug Treatment Type of study: Prognostic study / Randomized controlled trials Limits: Humans Language: English Journal: Clin Pharmacol Ther Year: 2022 Document Type: Article Affiliation country: Cpt.2528