Abstract Details

Title Patient-Centered Clinical Trial Design: A Case Study in Parkinson’s Disease

Topic Movement Disorders

Presentation(s) P2 - Poster Session 2 (5:30 PM-6:30 PM)

Poster/Presentation
Number 10-008

Objective

To develop an approach to identifying patient-centered outcomes and designing patient-centered clinical trials and demonstrate this approach in a case study on clinical trials for Parkinson’s disease (PD).

Background

Clinical trial design has begun to incorporate patient-centered considerations, such as measuring outcomes that matter to patients. Because PD is chronic, debilitating, and has few treatment options, patient-centered trial design for PD should also incorporate patients’ urgency for new treatments to determinate acceptable type I and type II statistical error in a clinical trial.

Design/Methods

This project developed a method for using patient preferences to set significance levels in clinical trial design. A panel of patient scientists with PD prioritized outcomes for a survey. Clinical and FDA perspectives on using these considerations for regulatory decision-making refined the list. The resulting patient preference survey was administered to 2,740 respondents. We assigned weights to type I and type II errors based on the identified patient preferences and developed a hypothetical clinical trial design to maximize the values identified by patients.

Results

Movement symptoms, which are common endpoints in PD clinical trials, were ranked as most important, and psychological and cognitive symptoms, which are less commonly studied, were ranked as the next most important. Preferences from respondents with mild PD symptoms and no prior experience with deep brains stimulation (DBS) resulted in larger, more conservative DBS trials, with acceptable significance level less than 5%. Preferences from respondents with severe PD symptoms and history of DBS resulted in smaller, less conservative DBS trials, with acceptable significance level greater than 5%.

Conclusions

By optimizing clinical trial size based on patient preferences, we aim to develop efficient clinical trials that expedite patient access to new therapies. Although PD was well-suited to this case study, the methodology can be used for other disease-treatment areas.

Authors/Disclosures
Katrina A. Gwinn, MD, F�鶹��ýӳ�� PRESENTER	No disclosure on file
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A. Hauber	No disclosure on file
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