Rare Diseases

A Clinical Trial Readiness Study of Patient Reported Outcome Measures in TTP (THINK-TTP)

Posted on by Jodi Snare

Page last updated May 11, 2026

Study Design: Prospective Observational Study
PCORnet Infrastructure: Single IRB, Patient partners or engagement
Principal Investigator: Toyosi Onwuemene and Theresa Coles
Institution: Duke University
PCORnet® Network Partner: STAR
Funder: Federal (NIH/NCATS)
Funding Date: 2024
Study Duration: 2024 – 2026
Participating PCORnet® Clinical Research Networks: GPC, STAR
Therapeutic Area: Rare Diseases
Status: Active, not recruiting

Research Question(s): Can we develop a consensus-based strategy that comprehensively measures TTP-associated cognitive function impacts from the patient perspective?

Primary Publication(s):

Coles T, Mkumba L, Thompson D, et al. The THINK-TTP Study for Assessing Cognitive Function in Clinical Trials: A Qualitative Research Protocol. International Journal of Qualitative Methods. 2026;25:16094069261429876.
doi:10.1177/16094069261429876

PANDA – MSD: Predictive Analytics via Networked Distributed Algorithms for Multi – System Diseases

Posted on by Jodi Snare

Page last updated May 11, 2026

Study Design: Retrospective Observational Study
PCORnet Infrastructure: Common Data Model (CDM), Patient partners or engagement
Principal Investigator: Jiang Bian
Institution: University of Florida and University of Florida Health
PCORnet® Network Partner: OneFlorida+
Funder: Federal (NIH)
Funding Date: 2023
Study Duration: 2023 – 2026
Participating PCORnet® Clinical Research Networks: OneFlorida+
Therapeutic Area: Rare Diseases
Condition: Granulomatosis with Polyangiitis (GPA); Psoriatic Arthritis (PsA)
Age Range: 18 Years and older (Adult, Older Adult )
Status: Active, not recruiting

Research Question(s):
Can the use of EHR data develop predictive tools to assist healthcare providers in reaching earlier diagnoses and interventions, and improve the diagnostic journey and clinical outcomes of patients with rare diseases?

Primary Publication(s):

Jian X, Zhang D, Yu Z, et al. Leveraging undecided cases in chart-reviewed phenotypes to enhance EHR-based association studies. J Biomed Inform. 2025;166:104839. doi:10.1016/j.jbi.2025.104839

Tong J, Li L, Reps JM, et al. Advancing Interpretable Regression Analysis for Binary Data: A Novel Distributed Algorithm Approach. Stat Med. 2024;43(29):5573-5582. doi:10.1002/sim.10250

Wu Q, Tong J, Zhang B, et al. Real-World Effectiveness of BNT162b2 Against Infection and Severe Diseases in Children and Adolescents. Ann Intern Med. 2024;177(2):165-176. doi:10.7326/M23-1754