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.

Patient Engagement at a Tipping Point-The Need for Cultural Change Across Patient, Sponsor, and Regulator Stakeholders: Insights From the DIA Conference, "Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle of Medical Products".

Benefit-risk assessment is the foundation for decision making throughout the life cycle of medical products. Because patients are the beneficiaries of the efficacy of medical treatments and also bear their possible risks, their perspectives and judgments about value and the relative importance of benefits and risks should be at the heart of the medical decision-making process. Patient engagement is now at a tipping point; there have been a growing number of patient engagement initiatives over the past several years, but there remains the need for a common language, alignment on engagement approaches and best practices, and a shared vision regarding a desired future state. This article discusses insights gleaned from the DIA conference, "Patient Engagement in Benefit-Risk Assessment throughout the Life Cycle of Medical Products" (September 2015). It highlights the changes that will need to occur within the patient, medical-product sponsor, and regulatory cultures in order for patient engagement to become integrated into the medical-product development process and life cycle maintenance. Furthermore, it emphasizes that reaching the desired future state will require a conscious commitment from all stakeholders to work collaboratively to develop shared solutions and to map a common path forward.

Medicines in Pregnancy Forum: Proceedings on Ethical and Legal Considerations.

To raise awareness and promote dialogue leading to action, this article provides proceedings on ethical and legal considerations associated with medicine use during pregnancy discussed during the 2014 DIA Medicines and Pregnancy Forum. A key focus of discussion at the forum was "When is it ethically appropriate to include or unethical not to include pregnant patients in clinical studies, and how can ethical barriers be addressed?" Also debated was the question "What are the most appropriate methods to collect and share data on medication use in pregnancy, and what is the best process for sharing such information?" Goals of the forum were to gain participant alignment on answers to these ethical questions, offer rationale for the answers, and provide insight into which stakeholders might be needed to facilitate discussion and action. Participants felt that under the right circumstances, drug research in pregnant women is justified and necessary. Multiple ideas and opinions on the handling of pregnant patients in clinical research, treating pregnant women in clinical practice, and communicating data to physicians and patients are presented.

A Proposed Framework to Address Needs of Clinical Data for Informed Medication Use in Pregnancy.

The objective of this paper is to communicate a proposed framework for addressing research limitations and communication barriers that contribute to a lack of data for making clinical treatment decisions about medication use in pregnancy. To address this global public health concern, a cross-stakeholder coalition composed of several workstreams is proposed. The intent is to foster collaborative discussion regarding potential solutions to address gaps in communication, engagement, and data generation and collection. Topic areas that require focus include development of awareness initiatives, cultural transformation efforts, collaboration initiatives, research standards, data compilation projects, and new data capture methods. Objectives to aid these efforts are outlined, and collaboration among researchers, regulators, health care providers, and patients is emphasized.

Clinical Data for Informed Medication Use in Pregnancy: Strengths, Limitations, Gaps, and a Need to Continue Moving Forward.

The objective of this paper is to explore the strengths, weaknesses, gaps, and needs in research on medication use in pregnancy, where opportunities have been bypassed to develop standards and collaborations for collecting data to better understand how medications can impact clinical outcomes in pregnant women and developing fetuses. The availability of existing data and the methods of its capture are reviewed, including registries, claims and health record databases, and meta-analyses. The paper focuses on why these efforts have not fundamentally provided benefit-risk information and clinical treatment algorithms for medication use in pregnant women. Methodological issues, such as lack of standardization and central data collection, are discussed. Common barriers are examined, including a lack of awareness and education, cultural hurdles, collaboration deficiency, and an insufficient development of new data collection methods.