Trends in collaboration with patient organizations on cancer-related clinical trials: Collaborative relationship with existing stakeholders and the characteristics of their co-sponsored studies in the United States

Abstract

Background In recent years, the importance of patient centricity in drug development has been recognized, affecting the stakeholders conducting clinical trials. In this context, end users, patients and patient organizations are becoming increasingly involved in the development process to address their unmet medical needs. However, there is a lack of research on environmental trends in which patient organizations collaborate with incumbent stakeholders in drug development. This study aimed to understand the current status of clinical trials regarding patient organization involvement as sponsors and collaboration with different types of sponsors in cancer-related clinical trials. This cross-sectional study also examined the characteristics of clinical trials involving patient organizations as new stakeholders by analyzing the environmental factors that promote collaboration with existing stakeholders. Methods A total of 14,830 interventional clinical trials registered on ClinicalTrials.gov between January 1, 2010, and December 31, 2020, were categorized and analyzed by target cancer types and five different sponsor types: 1. patient organization co-sponsored (PO-co), 2. academia (ACD), 3. governmental (GOV), and 4. industry co-sponsored (IND-co) and 5. industry only (IND). Results PO-Co studies accounted for 2% (289 of 14,830) of the total studies, ACD 5%, IND-Co 21%, GOV and IND 36%. When the 289 PO-Co studies were analyzed by cancer type and eligible study population, we found that the proportion of pediatric studies was significantly higher for PO-Co studies (30% (88 of 289)) than for total studies (9% (1,378 of 14,830); P < .001), and we also found that collaboration with PO was more common for certain cancer types compared with the total study. Regarding collaboration with POs, more than half of the PO-GOV collaborative studies were conducted at the Nation cancer center comprehensive cancer centers, and even particular investigators actively engaged in collaboration with POs. Conclusions A cross-sectional analysis of cancer-related clinical trials revealed that the involvement of patient organizations as sponsors in clinical trials did not increase significantly over time. However, some organizations have become actively engaged in certain cancers and pediatric populations that are less prioritized by incumbent sponsors. We also found that governmental sponsors and selected principal investigators played an important role in collaborating with patient organizations. These findings provide a basis for understanding the current status of patient organizations and for considering further contributions.