Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?

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.

Therapeutic Innovations in Neuroscience: What’s New on the Horizon?

“…Stephenson et al 2 discuss whether and how innovative trial designs and technologies, including multiarm adaptive platform designs and digital health tools to monitor progression in rare diseases like Duchenne muscular dystrophy and amyotrophic lateral sclerosis (ALS), can drive the advancement of treatments for common neurological diseases like Parkinson's disease. At the same time, the authors provide an excellent insight into the importance of collaborative efforts: Novel platforms containing data from completed clinical trials, registries, natural history studies, and preclinical data do not only foster data sharing but also provide a means of connectivity to sophisticated tools like disease progression models and clinical trial simulation applications.…”

“…Stephenson et al 2 . discuss whether and how innovative trial designs and technologies, including multiarm adaptive platform designs and digital health tools to monitor progression in rare diseases like Duchenne muscular dystrophy and amyotrophic lateral sclerosis (ALS), can drive the advancement of treatments for common neurological diseases like Parkinson’s disease.…”

Analysis of research topics and trends in investigator-initiated research/trials (IIRs/IITs): A topic modeling study

“…IST primarily focuses on obtaining drug approval from regulatory authorities, whereas IIRs/IITs aim to explore scientific value and address specific clinical questions, [ 1 ] including rare diseases, comparative diagnostics or treatments, novel uses for approved drugs, new indications, or innovative administration methods. [ 2 , 3 ] Clinical studies can employ noninterventional (prospective or retrospective) or interventional research methods. One key advantage of IIRs/IITs is its origin from real clinical problems encountered in daily practice, allowing investigators to have greater autonomy and a closer connection to practical aspects of medicine.…”