Benchmarking Patient Engagement Capabilities and Preparedness of Drug Development Sponsors.

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 &lt; .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. 

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

Phase I healthy volunteer trials test the safety and tolerability of investigational pharmaceuticals. In them, participants are exposed to study-drug risks without the possibility of direct medical benefit and typically must spend days or weeks in a residential research facility. Monetary payments are used to incentivize enrollment and compensate participants for their time. Together, these features of phase I healthy volunteer trials create a research context that differs markedly from most other clinical research, including by enrolling disproportionate numbers of economically disadvantaged people of color as participants. Due to these unique trial features and participation patterns, traditional biomedical research oversight offers inadequate ethical and policy guidance for phase I healthy volunteer research. This article details five ethical criteria crafted to be responsive to the particularities of this type of research: translational science value, fair opportunity and burden sharing, fair compensation for service, experiential welfare, and enhanced voice and recourse.

Ethical Criteria for Improved Human Subject Protections in Phase I Healthy Volunteer Trials.

Interest in patient-centric initiatives to engage patients as partners in clinical research and inform drug development strategy, planning and execution has increased exponentially during the past decade. Adoption, use, organizational approach and infrastructure supporting patient-centric initiatives, however, varies widely from company to company. The Drug Information Association (DIA) in collaboration with the Tufts Center for the Study of Drug Development (Tufts CSDD) at the Tufts University School of Medicine developed and validated an assessment tool that companies can use to evaluate their organization’s patient engagement preparedness and capabilities within the context of industry-wide practices. This paper discusses the development of the tool, the assessment experience, and implications for further refinement of the assessment process. Specifically, the team conducted an extensive literature review, compiled and analyzed case studies and gathered input from a working group of 18 biopharmaceutical companies. To validate the assessment tool and demonstrate its feasibility, the DIA-CSDD Tufts team conducted a pilot implementation involving onsite and virtual in-depth interviews among 14 biopharmaceutical companies. A subsequent paper will report on the findings from the 14 companies assessed.

Evaluating the Feasibility and Validity of a New Tool to Assess Organizational Preparedness and Capabilities to Support Patient Engagement in Drug Development.

Yaritza Peña

Papers

Evaluating the Feasibility and Validity of a New Tool to Assess Organizational Preparedness and Capabilities to Support Patient Engagement in Drug Development.