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

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.

Measuring the Impact of Patient Engagement and Patient Centricity in Clinical Research and Development.

BACKGROUND: Recently, drug development companies have sought out patient feedback to improve overall drug development. However, characterization of the overall impact and return on engaging with patients have not been determined. METHODS: The Drug Information Association (DIA), the Tufts Center for the Study of Drug Development (Tufts CSDD), and 17 other stakeholder organizations collaborated on a study to (1) quantify and define patient-centric initiatives (PCIs) utilized in clinical research and development and (2) to define evidence-based metrics and performance indicators that demonstrate return on engagement (ROE) of specific PCIs. We conducted a literature review, industry surveys, and in-depth interviews to determine and measure the impact of adopted PCIs. RESULTS: We identified and defined 30 PCIs used to engage with patients. We analyzed 121 case studies and created a comprehensive list of metrics assessing overall return to the organization and to patients. Advocacy Group Support and Involvement, Conducting Patient Advisory Panels, and Focus Groups were examples of PCIs with the lowest cost and largest impact with respect to quality, speed, and impact on the patient relative to other PCIs. CONCLUSION: The results from the literature review and use cases provide drug development teams with evidence and insights to help facilitate the adoption of specific PCIs within their organization and to help select those initiatives that would provide the highest impact to patients and development organizations. It is also hoped that the biopharmaceutical industry will apply the standardized metrics in the toolkit to systematically assess the overall return on engagement.

Assessing Biopharmaceutical Company Experience with Patient-centric Initiatives.

PURPOSE: A growing number of biopharmaceutical companies have been implementing patient-centric initiatives (PCIs). The Drug Information Association (DIA) and the Tufts Center for the Study of Drug Development (CSDD) collaborated on a study to gather data on the usage and impact of these PCIs to characterize company experience and impact. METHODS: DIA and Tufts CSDD collaborated with 17 organizations to define PCIs used in clinical research and development and to quantify their use, and to define metrics in use to document impact and return on engagement (ROE) for these PCIs. The study used a mixed methods approach that consisted of an online survey, in-depth interviews, and literature review. FINDINGS: Twenty-two unique companies responded to an on-line survey on the use of 23 PCIs identified by the study working group. PCIs most frequently implemented included patient organization landscape analysis, support of patient advocacy groups, use of patient advisory boards, and use of home nursing networks. Seven additional PCIs were found through a literature search and included in the group of PCIs for which impact measures were assessed. A total of 121 cases of use of the 30 PCIs and associated impact measures and impact data were gathered through literature review, in-depth interviews with the study companies, and in-depth interviews with organizations identified in the literature as having experience with patient engagement in clinical research as well as with patients who had participated in clinical trials. Analysis of the 121 case studies resulted in a list of 666 measures of impact (metrics) in use for 13 of the PCIs. Assessment of overall ROE for these PCIs found that PCIs such as support of patient advocacy groups and use of patient advisory boards indicated the greatest ROE, whereas costlier, more complex PCIs such as digital medicine and gaming indicated relatively low ROE. IMPLICATIONS: Activity around PCIs among the companies studied was widespread, with initiatives more frequently planned and piloted than implemented at the time of this study. Measures of impact have been identified and can be used to assess ROE, providing insights to facilitate the adoption of PCIs of highest impact for patients and biopharmaceutical research organizations.

The Cardiac Safety Research Consortium enters its second decade: An invitation to participate.

The Cardiac Safety Research Consortium (CSRC), a transparent, public-private partnership established in 2005 as a Critical Path Program and formalized in 2006 under a Memorandum of Understanding between the United States Food and Drug Administration and Duke University, is entering its second decade. Our continuing goal is to advance paradigms for more efficient regulatory science related to the cardiovascular safety of new therapeutics, both in the United States and globally, particularly where such safety questions add burden to innovative research and development. Operationally, CSRC brings together a broad base of stakeholders from academia, industry, and government agencies in a collaborative forum focused on identifying barriers and then creating novel solutions through shared data, expertise, and collaborative research. This white paper provides a brief overview of the Consortium's activities in its first decade and a context for some of our current activities and future directions. The growth and success of the CSRC have been primarily driven by members' active participation and the development of goodwill and trust throughout our membership, which have facilitated novel collaborations across traditionally competitive or contentious stakeholder boundaries. The continued expansion of our base of participating academicians, industry experts, and regulators will define the Consortium's success in our second decade. It is our hope that sharing our endeavors to date will stimulate additional participation in the CSRC and also provide a model for other groups starting to develop similar collaborative forums.