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S130: Analysis of Quality of Life Patient-Reported Outcomes in Clinical Trials for Parkinson’s Disease from 2010-2021

Poster Presenter

James Kim

Undergraduate Student Researcher
University of Southern California
United States

Objectives

Examine the use of disease-specific Quality of Life (QoL) Patient-Reported Outcomes (PROs) in Parkinson’s Disease (PD) clinical trials (CT) conducted from 2010 to 2021.

Method

Examined FDA-approved drugs for PD using drugs@fda.gov to determine what PROs were used in approval. Searched clinicaltrials.gov to assess these and other measures in interventional trials from 2016-2021. Enumerated disease-specific QoL PROs and extended search for trials from 2010-2015.

Results

The majority of PD clinical trials that were successful in obtaining drug approval from the FDA implemented outcome measures MDS-UPDRS II and III, and PDQ-39. There were also many other outcome measures that were specific for a condition in PD such as dyskinesia and depression. Data gathered from clinicaltrials.gov show that there was a rise in the use of PDQ-39 and its short form (PDQ-8), an increase from 17.6% of trials in 2010-2015 to 25.4% of trials in 2016-2021. UPDRS II and III and PDQ-39 were found to be the most used outcome measures in both 2010-2015 and 2016-2021, representing 70.5% of all outcome measures in 2010-2015 (n=197) and 65.2% of all outcome measures in 2016-2021 (n=135), which reflects the main outcome measures used in the approval trial for PD FDA-approved drugs. The frequency of PROs used in trials from the years 2010-2015 was 52.2% and from the years 2016-2021 was 53.8%. Using a chi-square test it was further determined that there was no significant increase in the use of outcome measures MDS-UDPRS II & III, and PDQ-39 or PDQ-8 in clinical trials from 2010-2015 to 2016-2021 (P=0.26).

Conclusion

While there were other symptom-specific outcome measures used, depending on each clinical trial’s needs, the overall use of outcome measures did not seem to become more standardized. Following the Patient-Focused Drug Development (PFDD) meeting for PD held by the FDA, there was no significant increase in frequency of trials that use UPDRS II and III and PDQ-39. The implementation of UPDRS II, a ClinRO, alongside PROs like UPDRS III and PDQ-39 reflects the importance of lessening the number of tools used to gather patient-focused data to avoid burdening patients with multiple outcome measures. There is no standardized set of guidelines by the FDA that lists PROs to be used for PD CTs. A select few PROs that are standardized will ensure that patient voices are being included in CTs. Composite outcome measures (those that included a PRO and a ClinRO) are a way to show both the clinical perspective as well as the patient perspective. Because it is difficult to see what is happening in the brain with non-invasive measures, other signs of disease such as mobility are observed to measure the degree of PD. The ClinRO MDS-UPDRS II provides a meaningful way to look at treatment effects and disease progression in patients, while the PROs highlight outcomes that are important to the patients. This knowledge provides different aspects of PD that allows clinicians and researchers to better understand the impact of PD, improve the QoL of patients, and illuminate patient experiences with this condition. This study shows the necessity and implementation of a harmonized set of outcome measures for PD CTs.