OBJECTIVES: In the biopharmaceutical industry, site-based studies are the norm for evaluating drug safety and efficacy.  Recently patient-centered studies (PCSs) have gained popularity. These studies offer advantages over site-based studies, but present unique challenges. This abstract seeks to describe the patient-centered approach and associated challenges and opportunities. METHODS:  This descriptive study reviews operational and design issues of PCSs and discusses utility, advantages, and challenges. RESULTS:  PCSs revolve around patients. They use an open-enrollment model whereby all eligible patients can self-enroll directly, thereby maximizing enrollment. Enrollment is not limited to patients treated at particular study sites. These studies typically employ a single site and “virtual” study coordinating center to screen/enroll patients and collect data, thus reducing site costs. PCSs are used extensively for pregnancy registries and other rare-exposure/disease registries, and more recently for clinical trials. This unique approach requires buy-in from regulatory agencies and IRBs. Recruitment activities should cast a wide net using various means targeted to the population (e.g., internet and social media recruitment sources for younger patients; television, radio, or print media for older patients). Streamlined patient screening, informed consent (e.g., on-line consent or waivers of written consent), and data collection processes are essential. Retention is facilitated by engaging participants in the reporting process, minimizing reporter burden, and providing ongoing support using various means targeted to the population (e.g., internet or mobile apps for younger patients and human support through coordinating centers for older patients). It may also be critical to obtain medical release to verify patient-reported medical data from treating physicians. However, discrepancies between patient-reported and physician-reported data may pose challenges in analyses and reporting. CONCLUSIONS:  PCSs offer advantages such as maximizing enrollment and reducing site costs, but pose regulatory, operational, and analytic challenges. Researchers opting to employ PCSs must weigh challenges versus opportunities in the design phase.