Optimizing Exclusion Criteria For Clinical Trials of Persistent Lyme Disease Using Real-World Data

January 7, 2025

https://www.mdpi.com/2227-9032/13/1/20

Optimizing Exclusion Criteria for Clinical Trials of Persistent Lyme Disease Using Real-World Data

by Lorraine Johnson1, Mira Shapiro2, Deanna Needell3 and Raphael B. Stricker4,*
1LymeDisease.org, Los Angeles, CA 91040, USA
2Analytic Designers LLC, Bethesda, MD 20817, USA
3Department of Mathematics, University of California, Los Angeles, CA 90025, USA
4Union Square Medical Associates, 595 Buckingham Way, Suite 350, San Francisco, CA 94132, USA
*Author to whom correspondence should be addressed.
Healthcare 2025, 13(1), 20; https://doi.org/10.3390/healthcare13010020
Submission received: 15 October 2024 / Revised: 19 December 2024 / Accepted: 21 December 2024 / Published: 25 December 2024
(This article belongs to the Topic Public Health and Healthcare in the Context of Big Data)

Abstract

Background/Objectives: Although eligibility criteria for clinical trials significantly impact study outcomes, these criteria are often established without scientific justification, leading to delayed recruitment, small sample sizes, and limited study generalizability. Persistent Lyme disease (PLD) presents unique challenges due to symptom variability, inconsistent treatment responses, and the lack of reliable biomarkers, underscoring the need for scientifically justified eligibility criteria.
Objective: This study examines the effects of commonly used enrollment criteria on sample yield in PLD clinical trials using real-world data (RWD) from the MyLymeData patient registry. The study also compares the effects of these criteria on enrollment for PLD versus acute Lyme disease (ALD) trials and evaluates the scientific rationale for each criterion.
Methods: Data from 4183 Lyme disease patients enrolled in the MyLymeData registry were analyzed to assess the prevalence and cumulative impact of various criteria on sample yield. A comparative analysis of cohorts with PLD (n = 3589) versus ALD (n = 594) was conducted to identify differences in sample attrition.
Results: In a large PLD cohort study, we found that current commonly used eligibility criteria would exclude approximately 90% of patients, significantly limiting study generalizability. Substantial differences in sample attrition between PLD and ALD cohorts highlight the need for tailored criteria. The strength of scientific justification varied widely among criteria.
Conclusions: This study demonstrates the importance of using RWD to optimize eligibility criteria in PLD clinical trials. By providing insights into the balance between sample attrition and scientific justification, researchers can enhance trial feasibility, generalizability, and robustness. Our RWD sample demonstrates that researchers could substantially increase the sample yield from 10% to 64% by loosening restrictions on coinfections and misdiagnoses of chronic fatigue syndrome, fibromyalgia syndrome, and psychiatric conditions.

Category:

Lyme, research

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