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The role of basket trials in drug development for neurodegenerative disorders
Jeffrey Cummings1,2, Arturo Montes3, Sana Kamboj4
1Chambers-Grundy Center for Transformative Neuroscience, Pam Quirk Brain Health and Biomarker Laboratory, Department of Brain Health, School of Integrated Health Sciences, University of Nevada Las Vegas (UNLV), Las Vegas, NV, USA. jcummings@cnsinnovations.com.
Background:
Drug development for neurodegenerative disorders (NDDs) is a long, complex, and expensive enterprise. Methods to optimize drug development for NDDs are needed. Basket trials have been widely used in oncology and have been promoted by the Food and Drug Administration as a means of enhancing the efficiency of drug development.
Discussion:
We reviewed clinical trials for NDDs registered on clinicaltrials.gov in the past 10 years. We identified 59 basket trials assessing the impact of treatment on more than one NDD in the trial. Forty-one of the trials were for 25 agents addressing symptoms of NDD such as motor impairment, hypotension, or psychosis. Eighteen of the trials assessed 14 disease-modifying therapies; the principal targets were mitochondrial function, tau biology, or alpha-synuclein aggregation. Basket trials are most common in phase 2 but have been conducted in phase 1, phase 3, and phase 4. The duration and size of the basket trials are highly variable depending on their developmental phase and the intent of the trial. Parkinson's disease was the most common disorder included in basket trials of symptomatic agents, and Alzheimer's disease was the most common disorder included in basket trials of disease-modifying therapies. Most of the basket trials of symptomatic agents were sponsored by pharmaceutical companies (29 of 41 trials); similarly, most of the basket trials investigating DMTs in basket trials were sponsored by the biopharmaceutical industry (11/17 trials).
Conclusions:
Basket trials may increase drug development efficiency by reducing redundancy in trial implementation, enhancing recruitment, sharing placebo groups, and using biomarkers relevant to the mechanism of action of the treatment across NDDs. There have been relatively few basket trials including multiple NDDs in the same trial conducted over the past 10 years. The use of the basket trial strategy may represent an opportunity to increase the efficiency of development programs for agents to treat NDDs.
Insights
Basket trials show promise for improving neurodegenerative disorder drug development efficiency. This strategy may streamline recruitment and reduce redundancy for new NDD treatments.
Area of Science:
- Neuroscience
- Clinical Trials
- Drug Development
Background:
- Drug development for neurodegenerative disorders (NDDs) is lengthy, costly, and complex.
- Optimizing drug development for NDDs is crucial.
- Basket trials, successful in oncology, offer a potential solution for NDD drug development efficiency.
Purpose of the Study:
- To review clinical trials for neurodegenerative disorders registered on clinicaltrials.gov over the past 10 years.
- To identify and analyze the use of basket trials in NDD drug development.
- To assess the potential of basket trials to enhance the efficiency of NDD drug development.
Main Methods:
- A review of clinical trials for NDDs registered on clinicaltrials.gov from the past decade.
- Identification of trials assessing treatments for multiple NDDs within a single trial (basket trials).
- Categorization of trials by therapeutic intent (symptomatic vs. disease-modifying) and developmental phase.
Main Results:
- 59 basket trials were identified, evaluating treatments for multiple NDDs.
- 41 trials focused on symptomatic agents (e.g., for motor impairment, hypotension) and 18 on disease-modifying therapies (targeting mitochondrial function, tau, alpha-synuclein).
- Basket trials are most common in Phase 2, with Parkinson's and Alzheimer's diseases being the most frequently included NDDs.
Conclusions:
- Basket trials can enhance drug development efficiency by reducing redundancy, improving recruitment, and utilizing shared placebo groups.
- Despite their potential, relatively few basket trials involving multiple NDDs have been conducted.
- The basket trial strategy presents a significant opportunity to accelerate the development of effective treatments for neurodegenerative disorders.
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