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Mapping the Landscape of Patient-centric Activities Within Clinical Research

      Abstract

      Purpose

      Although there has been more involvement by patients in the drug-development process, there are not a lot of published data that quantify patient-centric activities or that document these activities across a large scale. In order to examine the patient-centricity landscape and to quantify the adoption and implementation of these initiatives, the Tufts Center for the Study of Drug Development and the Drug Information Association collaborated on a research study. The study examined patient-centric activities implemented by pharmaceutical, biotechnology, and contract research organizations, as well as activities being piloted or in the planning stages.

      Methods

      A global industry survey was conducted across pharmaceutical, biotechnology, and contract research organizations, assessing 25 patient-centric activities within clinical research. Some of these initiatives involve the use of social media to engage with patients, or the use of social listening to monitor study activity. Initiatives being implemented, planned, or piloted in addition to those not being considered were evaluated by respondents. Twenty-two unique companies responded to the survey, representing a mix of large, mid-sized, and small organizations.

      Findings

      The most widely adopted patient-centric initiatives, including activities both implemented and piloted across organizations, were patient advisory boards (17/22 companies), professional panels (16), lay-language clinical trial results summaries (13), assessment of the patient–organization landscape (10), and the use of home nursing networks (9).

      Implications

      The results of the study suggest that organizations have a varied approach to the adoption and implementation of patient-centric initiatives, with more activities occurring in the planning stages than are being piloted or implemented. Many factors affect implementation and adoption, including buy-in by senior management, organizational vision, resources, and level of investment.

      Key words

      Introduction

      A recent focus of the pharmaceutical industry is assessing the role of patient-centricity in clinical research, as reflected in the numerous publications, reports, and conferences examining the role of the patient within the drug-development process. Although there has been more patient involvement in drug development compared with a few years ago, there remains a lack of published data that quantify patient-centric activities or that document these activities across a large scale. Patient involvement in the clinical research process is significant, as it can potentially improve the research process, minimize the burden of participation, and improve clinical trial design and execution. Patients may also provide input into clinical trial protocols and study design that could lead to improved recruitment.
      • Lowe M.M.
      • Blaser D.A.
      • Cone L.
      • et al.
      Increasing patient involvement in drug development.
      Recent findings from studies suggest that best practices for patient involvement in drug development have not yet been established.
      • Lowe M.M.
      • Blaser D.A.
      • Cone L.
      • et al.
      Increasing patient involvement in drug development.
      • Hoos A.
      • Anderson J.
      • Boutin M.
      • et al.
      Partnering with patients in the development and lifecycle of medicines: A call for action.
      • Dewulf L.
      Patient engagement by pharma-why and how? A framework for compliant patient engagement.
      This research also reveals that the present frameworks are not sufficient to support more widespread patient involvement.
      • Lowe M.M.
      • Blaser D.A.
      • Cone L.
      • et al.
      Increasing patient involvement in drug development.
      • Dewulf L.
      Patient engagement by pharma-why and how? A framework for compliant patient engagement.
      Data were gathered in these studies through various methodologies, including interviews with health care and patient thought leaders, and through case examples in the literature. Additional research indicates that no “master framework” for systematic patient involvement in drug development can be identified and that patient involvement is inconsistent and fragmented (although in some therapeutic areas it may be productive).
      • Hoos A.
      • Anderson J.
      • Boutin M.
      • et al.
      Partnering with patients in the development and lifecycle of medicines: A call for action.
      Other researchers acknowledge that guidance on patient engagement does not exist but that there is a need for it.
      • Dewulf L.
      Patient engagement by pharma-why and how? A framework for compliant patient engagement.
      Various definitions of patient-centricity are also offered in the published literature. Robbins et al
      • Robbins D.A.
      • Curro F.A.
      • Fox C.H.
      Defining patient-centricity: opportunities, challenges, and implications for clinical care and research.
      define patient-centricity as "a dynamic process through which the patient regulates the flow of information to and from him/her via multiple pathways to exercise choices consistent with his/her preferences, values and beliefs (p. 350)." Getz
      • Getz K.A.
      Establishing return on investment expectations for patient centered initiatives.
      described 4 core principles of patient-centricity, including relevance, pragmatism, feasibility, and interactivity. Through these principles, a methodology for measuring return on investment of patient-centricity initiatives is proposed. The return on investment of these initiatives can be measured in pharmaceutical, biotechnology, and contract research organizations through quantitative (internal and external reach) and qualitative measures. One example of an internal reach measure is the total number of initiatives being piloted or implemented within a particular time frame, while external reach may be the total number of patients or study volunteers reached by all patient-centric initiatives within a particular time frame.
      Patient-centric initiatives are numerous and vary in their levels of uptake and adoption by the pharmaceutical industry. The range of initiatives include patient advisory boards, professional panels, advocacy group support and involvement, real-time data collection, direct-to-patient clinical trials, home nursing networks, telemedicine, wearables (patient-reported outcomes, which are patient self-reported data), and eConsent. Mobile nursing services in clinical trials have been broadening their reach, especially in North America and Western Europe.
      • Stergiopoulos S.
      • Eustace C.
      • Stem K.
      • Getz K.A.
      Mobile Nurse Services in Clinical Trials: Usage and Industry Perceptions.
      eConsent manages patient consent using technology, but has not had much utilization.

      Delente E. Improve Patient Comprehension and Retention With eConsent. http://www.appliedclinicaltrialsonline.com/improve-patient-comprehension-and-retention-econsent. Accessed May 23, 2017.

      Other uses of patient-centric initiatives can be seen in crowdsourcing protocols, defined as gathering input from patients, physicians, and other stakeholders via the World Wide Web, especially within oncology clinical trials.
      • Leiter A.
      • Sablinski T.
      • Diefenbach M.
      • et al.
      Use of crowdsourcing for cancer clinical trial development.
      Another patient-centric initiative is social listening which aggregates social media data such as patient posts or comments to gather information on patient insights or to identify unmet needs during treatment. Telemedicine, the use of electronic communication and remote technologies to treat patients, also has potential in patients within clinical research. However, its use can be costly and challenging both in terms of technology requirements and training or usability from the perspective of a patient.
      • Kidholm K.
      Renewing health-regions of Europe working together for health.
      In the United States, there have also been increases in patient wearables and other monitoring devices that transmit patient data to health care professionals. The use of remote monitoring technologies is expected to reach 4 million patients by 2020.

      Morton N., Blackman D. The growing availability of wearable devices: A perspective on current applications in clinical trials. http://www.appliedclinicaltrialsonline.com/growing-availability-wearable-devices-perspective-current-applications-clinical-trials. Accessed May 22, 2017.

      The use of electronic patient-reported outcomes in clinical trials is an application of wearables. There are a number of challenges with the use of mobile health applications, including data security and privacy, data qualification, and validation and regulatory acceptance.

      Morton N., Blackman D. The growing availability of wearable devices: A perspective on current applications in clinical trials. http://www.appliedclinicaltrialsonline.com/growing-availability-wearable-devices-perspective-current-applications-clinical-trials. Accessed May 22, 2017.

      Smith et al
      • Smith M.Y.
      • Hammad T.A.
      • Metcalf M.
      • et al.
      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.".
      discussed the need for a common language, alignment, and best practices around patient engagement. The Patient-Centered Outcomes Research Institute (Washington, DC) has developed general guidance through their Engagement Rubric, which describes how input from patients and other stakeholders can be gathered throughout the research cycle.

      PCORI Engagement Rubric. PCORI (Patient-Centered Outcomes Research Institute) website. http://www.pcori.org/sites/default/files/Engagement-Rubric.pdf. Published February 4, 2014. Updated October 12, 2015. Accessed May 11, 2017.

      The Clinical Trial Transformation Initiative has also developed recommendations and best practices for effective engagement with patients in clinical trials.

      CTTI’s Patient Groups & Clinical Trials Project. Engage early. Engage often. Best practices for effective engagement with patient groups around clinical trials. https://www.ctti-clinicaltrials.org/projects/patient-groups-clinical-trials Link accessed September 14, 2017.

      The European Patients Academy on Therapeutic Innovation (Brussels, Belgium) is a public–private partnership and consortium of 33 organizations that focuses on facilitating patient involvement in research and development. In addition, the US Food and Drug Administration recently hosted meetings that focused heavily on patient involvement, including panels representing different stakeholder groups such as patient advocacy groups, consumer advocacy groups, health care professionals, and other industry professionals.

      US Food and Drug Administration. Prescription Drug User Fee Act; Public Meeting; Request for Comments. https://www.federalregister.gov/articles/2016/07/19/2016-16916/prescription-drug-user-fee-act-public-meeting-request-for-comments. Accessed May 18, 2017.

      Despite many efforts to integrate various stakeholder input from patients, pharmaceutical companies, academic, and others, published data examining the adoption and implementation of specific patient-centric initiatives across pharmaceutical and biotechnology companies have been lacking. Some research highlights specific initiatives but is limited and does not evaluate activities being piloted or in the planning stages.

      Quintiles IMS: Reinventing biopharma: strategies for an evolving marketplace the patient-led R&D strategy. Economist intelligence unit report. http://www.quintiles.com/library/white-papers/reinventing-biopharma-strategies-for-an-evolving-marketplace--the-patient-led-rd-strategy. Accessed May 31, 2017.

      To date, no published data exist that quantify the adoption of specific patient-centric initiatives that can provide value and guide decision making among biopharmaceutical companies and other industry stakeholders.

      Materials and Methods

      In order to examine the patient-centricity landscape and quantify the adoption and implementation of this patient-centric activity, the Drug Information Association (DIA; Horsham, Pennsylvania) and the Tufts Center for the Study of Drug Development (CSDD; Boston, Massachusetts) collaborated on a research study with key objectives that included quantifying the adoption and usage of specific patient-centric initiatives by pharmaceutical and biotechnology companies and examining management models that support patient-centricity. Other major objectives included defining evidence-based metrics to illustrate and demonstrate return on engagement from specific patient-involvement or -engagement initiatives and a review of the guidance and frameworks to inform implementation. This article focuses on one research objective of the study: examining the adoption and usage of specific patient-centric initiatives across the industry through mapping the landscape of these initiatives or assessing their prevalence and utilization by gathering data from organizations through a survey. The survey focused on evaluating patient-centric initiatives and their implementation within clinical research.
      The study used a working-group model composed of participating companies that collaborated with Tufts CSDD and DIA to define the study focus and analysis frame. In total, 20 companies joined the working group, including pharmaceutical and biotechnology companies and contract research organizations. Tufts CSDD and DIA, in collaboration with a subcommittee of the working group, developed a Web-based survey to gather metrics on specific patient initiatives that organizations were implementing, planning, piloting, or not considering within clinical research. Other areas of focus of the survey were assessment of the challenges to implementation, specific management approaches or models used, types of organizational structure and function that companies established, and corporate drivers supporting a patient-engagement function. The survey was conducted across the working group and also to a broader cross-section of industry contacts. The survey was sent out using Qualtrics, a survey software, through a link in an e-mail invitation. Participating companies gathered data for the survey based on their recent experiences at their organizations with patient-centric initiatives.

      Results

      The survey evaluated 25 patient-centric initiatives within clinical research across organizations that were in various stages: those presently being implemented and those in the planning or piloting stages. Also, data were gathered on activities not being considered. Survey completion required respondents to collaborate cross-functionally in order to gather a complete set of data. Responses were gathered from 22 unique companies. The organizations represented were 19 pharmaceutical and biotech companies and 3 contract research organizations comprising 11 large and 11 mid-sized and small companies based on research-and-development spending. Respondents' roles varied, but most were director level or above and occupied senior-level management positions within their organizations.
      The initiatives were categorized into 4 groups: innovative partnerships, technology advancements, protocol design, and study-volunteer ease (Figure 1). Overall, the results revealed that there are more organizational patient-centric initiatives in the planning stages than are being implemented or piloted. In general, the majority of organizations (13 of 22) described their organization as "somewhat patient-centric"; 3 organizations, "very patient-centric"; and 4 as "not very patient-centric." In open-ended responses, those who characterized their organization as "somewhat patient-centric" indicated that they were using a fragmented and inconsistent approach at their organizations. Activities were not uniformly conducted across the organization and were implemented on a limited basis, for example, in one therapeutic area or for a particular study.
      Figure Au: Are the legends for Figures 1-5 ok as written? Please expand as necessary.1
      Figure 1Four categories of patient-centric initiatives.
      The most widely adopted initiatives, including implemented and piloted activities combined, were identified as patient advisory boards (17/22 companies), professional panels (16), lay-language clinical trial results summaries (13), assessment of patient organization landscape (10), and the use of home nursing networks (9). On examination of each category separately, the top implemented initiatives were patient organization landscape tools, patient advisory boards, and professional panels (all, 10 companies) (Figure 2). The top piloted initiatives included end-of-study surveys (9 companies) and patient wearables (8) (Figure 3). The top planned initiatives were eConsent (11 companies), adaptive trial designs and adaptive licensing (10), and establishing patient communities during and after clinical trials (10) (Figure 4).
      The initiatives that companies were not considering were medicine co-development partnerships with patient associations (16 companies), open design and crowdsourcing (16), and telemedicine (12) (Figure 5). On examination of the size of companies by patient-centric activities, no notable differences were observed.
      The primary barriers to the adoption of patient-centric initiatives were gaining internal company buy-in (6 companies) and the authority to implement them (5). Other factors reported that present challenges are sponsor readiness or risk tolerance, lack of resources (including budget and staff), and perceived lack of time. Despite the budget challenges noted for implementation, the majority (13/20 companies) reported having a budget for patient-engagement activities.
      The most prevalent initiatives being implemented were patient–organization landscape analysis tools specific to disease areas, and patient advisory boards and professional panels. The landscape analysis tools are developed internally to understand patients and patient organizations to target for a specific disease area. The advisory boards and professional panels are used for engaging patients, physicians, and other clinical research stakeholders. The top piloted initiatives were end-of-study surveys and patient wearable devices.
      eConsent was mentioned as the top planned initiative (11 companies). Other planned initiatives mentioned by less than half of the group (10 companies) were adaptive trial designs and adaptive licensing; patient communities during and after clinical trials were also reported to be a planned initiative (11). Medicine co-development partnerships were reported by many companies as not being considered. These partnerships comprise a sharing of resources or risk with patient advocacy organizations to development new treatments. Open design and crowdsourcing were also not among those initiatives presently being evaluated by organizations.

      Discussion

      The results suggest that organizations are not adopting a uniform approach to patient-centric initiatives. In addition, initiatives are occurring across all functions of the organization and in different stages of development and implementation. It was also found that there were more organizational patient-centric initiatives in the planning stages than being piloted, and only a few areas in which there were more activities being implemented compared with those being planned or piloted. Those activities being implemented, such as professional panels and patient advisory boards, can be done so easily by organizations, provide rapid feedback, and can be used across multiple protocols.
      Another key finding of the survey is that company commitment and buy-in were crucial to adoption and implementation. Buy-in by senior management and having a patient-centric vision are crucial to whether initiatives are achievable and sustainable and differentiate organizations, rather than amount of budget dollars, since most companies in our survey reported having a budget for these activities. Prioritizing activities as well as having the resources and time to implement them also play a role. The Tufts CSDD–DIA study found wide variation in patient-centric activities, although the most prevalent management approach to the patient-engagement function was centralized. Many companies, however, do not yet have standard metrics or do not use metrics to evaluate the approach or role.

      Tufts CSDD DIA patient engagement study. Capturing the value of patient engagement: summary of the results of the 2016 study of patient-centric initiatives in drug development. http://engage.diaglobal.org/DIAPatientEngagementStudy.html?_ga=2.73245781.778125997.1494960937-965062505.1485202980. Accessed May 30, 2017.

      Some of the more commonly implemented activities, such as professional panels and patient advisory boards, offer quicker return for organizations, and the costs are minimal. It has become standard procedure for some organizations in order to get patient input early on in the clinical trials process. In addition, these panels and boards can be used across all disease states, and the data gathered can provide valuable insight into clinical trial protocol and other materials. The Tufts CSDD–DIA study found that data and insights gathered from patient advisory boards across >50 case studies can yield changes to protocols in terms of the schedule of visits, procedures, the informed-consent form, study positioning, and communications material.

      Tufts CSDD DIA patient engagement study. Capturing the value of patient engagement: summary of the results of the 2016 study of patient-centric initiatives in drug development. http://engage.diaglobal.org/DIAPatientEngagementStudy.html?_ga=2.73245781.778125997.1494960937-965062505.1485202980. Accessed May 30, 2017.

      In contrast, using telemedicine or developing patient apps or wearables can potentially be more costly, and results can be mixed. Wearables can also be difficult to implement, vary in functionality, and require technical support. Also, patient wearable devices may not be suitable for all disease indications, and patient privacy could be difficult to manage. Telemedicine poses challenges in use of technology and costs involved and may be limited and not seen as a substitute for standard care.

      Conclusions

      Organizations have a varied approach to the adoption and implementation of patient-centric initiatives due to many factors, including buy-in by senior management, organizational vision, staff resources, and prioritization of activities. In addition, many of the activities presently being implemented can be done relatively quickly, provide rapid feedback, and are less costly. Some of the other initiatives being piloted or planned pose challenges for organizations. There are often initiatives requiring investment in much time and effort, where return is questionable, and they are therefore typically not being widely adopted by organizations. As the survey was conducted as a baseline assessment, follow-up is needed to evaluate whether uptake by organizations is gaining momentum and how the challenges of implementation can be met.

      Conflicts of Interest

      The authors have indicated that they have no conflicts of interest with regard to the content of this article. All authors contributed equally.

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