Donnelly A, Christopher S, Chaudhuri S, Hauber AB, Mange B, Benz HL, Caldwell B, Gwinn K, Saha A, Ho M, Sheehan M, McLaughlin L, Sheldon M, Lo A. Expedited access to therapies: how measuring and incorporating patient preferences can make clinical trials more efficient and more effective. Poster presented at the 5th World Parkinson Congress; June 5, 2019. Kyoto, Japan.

TOPIC AREA: Clinical Sciences - Clinical trials: design, outcomes, recruiting, PwP involvement, communications Medical innovators and regulators have increasingly recognized the importance of working with patients to design medical therapies and clinical trials that meet the needs of specific patient populations. For diseases such as Parkinson’s Disease (PD), a progressive, degenerative disease with few effective treatment options, traditional randomized clinical trials with a fixed statistical threshold may not reflect patients’ perspectives on the trade-off between the risk of endorsing an ineffective therapy (false positive) and the risk of rejecting an effective therapy (false negative). This collaborative project, which involved academia, industry, FDA, patient-scientists and MJFF, developed and tested methods for incorporating patient preference information as explicit means to set significance levels in clinical trial design.

With direct input from patients with PD, we developed a patient preference survey and deployed it online through Fox Insight for 6-weeks and received 2,752 complete responses (24.4%), allowing us to analyze differences in outcome priorities among various demographic groups. We then assigned weights to the consequences of errors based on identified patient preferences, and proposed a hypothetical clinical trial design optimized 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. Differences emerged from different groups within the patient population, depending upon how the disease manifested itself. Preferences from respondents with mild PD symptoms and no prior experience with deep brains stimulation (DBS) resulted in larger, more conservative trials, with acceptable significance level less than 5%. Preferences from respondents with severe PD symptoms and history of DBS resulted in smaller, less conservative trials, with acceptable significance level greater than 5%.

CONCLUSION: This method has the potential to remove barriers to access to innovative technologies by giving patients a pathway to breakthrough, lifesaving technologies based on their treatment priorities and risk tolerance and the resulting potential for reduced clinical trial size. Though this test case was in Parkinson’s Disease, we envision that this method may have dramatic implications for the design of clinical trials for many diseases.

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