A Novel Collaborative Approach to Building Better Clinical Trials: New Insights From a Patient Engagement Workshop to Propel Patient-Centricity Forward.

BACKGROUND: (1) A growing number of pharmaceutical and biotechnology organizations are engaging patients, their support networks, and clinical trial site staff at various touchpoints along the clinical research development spectrum to solicit feedback on how to reduce the burden of clinical trial participation and administration. (2) However, many organizations are still evaluating how to best implement such engagement initiatives in a manner that will evoke meaningful, sustainable results and change. METHODS: In an effort to support meaningful engagement in a novel way, Janssen organized a 2-day innovative workshop designed to promote collaboration and foster mutual understanding among a cross-functional group of clinical research stakeholders. Over the course of the workshop, patients, sponsor team members, and clinical trial site staff each leveraged their unique experiences to address the challenges of today's clinical trials, and collectively envision the ideal clinical trial of the future. RESULTS: The workshop design created a level playing field for the stakeholders to interact with one another as partners with the shared goal of building better clinical trials. A significant number of transformative ideas were generated as a result of the innovative workshop exercises. Participants agreed that future clinical trials must be convenient and customizable and truly put the patient at the center of research. CONCLUSION: Creating a comfortable atmosphere and engaging environment for patients, site staff, and pharmaceutical companies to discuss current challenges of clinical trial participation and potential solutions together as partners in real time is critical and has proven to be a valuable novel engagement option for other organizations to consider adopting.

Differences in Clinical Research Perceptions and Experiences by Age Subgroup.

BACKGROUND: (1) To assess the differing viewpoints of actual and potential adult clinical trial participants in 5 age cohorts, (2) identify misperceptions and opportunities to reduce unnecessary burdens on volunteers, and (3) provide age-specific feedback to study sponsors on their current engagement efforts. METHODS: Based on 12,427 responses to a global online survey collected between May and July 2017, we conducted a subgroup analysis by age of general clinical research impressions, the participation decision-making process, and experiences of study participants. RESULTS: Overall, younger people were the most misinformed and experienced the most difficulties, while older people had the most misgivings about studies and sponsoring pharmaceutical companies. In the middle years, the desire for compensation peaked. Social media and clinical participation conveniences were important at every life stage. CONCLUSIONS: Effectively engaging the public and patients in conversations about clinical research could be achieved via targeted efforts addressing age-specific knowledge gaps, trial-searching behaviors, and displeasing aspects of trial participation. The altruistic benefits of participation, and involvement of patients' personal physicians, resonate particularly well across multiple age cohorts. Social media and other technologies are underutilized, especially among older populations.

Global consensus building and prioritisation of fundamental lupus challenges: the ALPHA project.

OBJECTIVE: Lupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-term and long-term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress. METHODS: A Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic 'nodes' (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus. RESULTS: Expert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology. CONCLUSIONS: ALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.

Using Patient Advisory Boards to Solicit Input Into Clinical Trial Design and Execution.

Academic institutions, pharmaceutical and biotechnology companies, foundations, and associations are routinely implementing patient advisory boards (PABs) to solicit patients' voices and perspectives on a variety of clinical research-related areas, including protocol design, clinical trial execution, informed-consent form design, clinical trial medicine kit design, wearable devices and mobile technologies, and patient-communication materials. Based on experience conducting >50 PABs during the past several years, the authors provide insights into how to best plan and execute PABs and their value in informing improvement in patient engagement.

A Novel Collaborative Approach to Building Better Clinical Trials: New Insights From a Patient Engagement Workshop to Propel Patient-Centricity Forward.

BACKGROUND: (1) A growing number of pharmaceutical and biotechnology organizations are engaging patients, their support networks, and clinical trial site staff at various touchpoints along the clinical research development spectrum to solicit feedback on how to reduce the burden of clinical trial participation and administration. (2) However, many organizations are still evaluating how to best implement such engagement initiatives in a manner that will evoke meaningful, sustainable results and change. METHODS: In an effort to support meaningful engagement in a novel way, Janssen organized a 2-day innovative workshop designed to promote collaboration and foster mutual understanding among a cross-functional group of clinical research stakeholders. Over the course of the workshop, patients, sponsor team members, and clinical trial site staff each leveraged their unique experiences to address the challenges of today's clinical trials, and collectively envision the ideal clinical trial of the future. RESULTS: The workshop design created a level playing field for the stakeholders to interact with one another as partners with the shared goal of building better clinical trials. A significant number of transformative ideas were generated as a result of the innovative workshop exercises. Participants agreed that future clinical trials must be convenient and customizable and truly put the patient at the center of research. CONCLUSION: Creating a comfortable atmosphere and engaging environment for patients, site staff, and pharmaceutical companies to discuss current challenges of clinical trial participation and potential solutions together as partners in real time is critical and has proven to be a valuable novel engagement option for other organizations to consider adopting.

Insights and Best Practices for Planning and Implementing Patient Advisory Boards.

A growing number of organizations-including pharmaceutical and biotechnology companies, foundations and associations-are routinely implementing patient advisory boards (PAB) given their high reported value for minimal relative investment. Organizations are typically implementing PABs to solicit patient voices and perspectives on a variety of areas such as protocol designs, clinical trial medicine kit designs, informed consent form designs, technology solutions, and patient communication materials. The Center for Information and Study on Clinical Research Participation (CISCRP) has planned, executed, and facilitated more than 30 PABs. In this article, the authors share lessons learned and best practices with regard to structure, format, and process for organizations wishing to adopt and implement PABs. The authors also provide metrics on the adoption and impact of PABs.

Global Public Attitudes About Clinical Research and Patient Experiences With Clinical Trials.

Importance: Effective, continuous improvement in patient engagement depends on an intimate understanding of public and patient perceptions and experiences in clinical research. Objectives: To identify the views of clinical trial participants and nonparticipants and characterize trends in these views over time. Design, Setting, and Participants: In this survey study, a questionnaire was administered online from May 8 to July 24, 2017, by the Center for Information and Study on Clinical Research Participation (CISCRP), and findings were compared with previous studies conducted in 2013 and 2015. The 2017 sample included responses from 12 427 individuals from 68 countries and represents a 10% participation rate. Similar to international assessments conducted by the CISCRP and other organizations, this study drew responses from a convenience sample; any adult older than 18 years who received an email or had online access was eligible to participate. Main Outcomes and Measures: Significant changes were observed in the perceptions and clinical trial experiences of the public and study volunteers compared with past CISCRP studies. Results: A total of 12 427 individuals (mean [SD] age, 55 [15] years; 7355 [59.2%] female; 10 085 [81.2%] white), 2194 (17.7%) of whom had participated in previous clinical research studies, responded to the survey in 2017. Findings indicated a belief in the importance of clinical research, but limited understanding of the research process persists. In 2017, a total of 10 506 individuals (84.5%) perceived clinical research to be very important to the discovery and development of new medicines; however, 4079 of 6919 respondents (59.0%) were unable to name a place where studies were conducted. A total of 11 182 respondents (90.0%) believed that clinical research is generally safe; however, 5578 of 12 427 individuals (44.9%) reported that clinical trials are rarely considered as an option when discussing treatments or medications with their physician. Clinical trial participation was perceived as inconvenient and burdensome; in the latest survey, 1075 respondents (49.0%) expressed that their clinical trial participation disrupted their daily routine. Conclusions and Relevance: The results of this study may provide a foundation from which to build meaningful and effective engagement with the public and patients and revealed roadblocks, including knowledge gaps among the public, limited physician involvement in discussing clinical trials as treatment options, and the inconveniences that patients encounter after they volunteer to participate. These findings may inform patient engagement strategies and tactics and ultimately help accelerate the drug-development process.

Informed consent in clinical research: Consensus recommendations for reform identified by an expert interview panel.

BACKGROUND: Informed consent is the cornerstone for protection of human subjects in clinical trials. However, a growing body of evidence suggests that reform of the informed consent process in the United States is needed. METHODS: The Clinical Trials Transformation Initiative conducted interviews with 25 experienced observers of the informed consent process to identify limitations and actionable recommendations for change. RESULTS: There was broad consensus that current practices often fail to meet the ethical obligation to inform potential research participants during the informed consent process. The most frequent single recommendation, which would affect all participants in federally regulated clinical research, was reform of the informed consent document. The interviews also identified the need for reform of clinical research review by institutional review boards, including transitioning to a single institutional review board for multi-site trials. CONCLUSION: The consensus recommendations from the interviewees provide a framework for meaningful change in the informed consent process. Although some proposed changes are feasible for rapid implementation, others such as substantive reform of the informed consent document may require change in federal regulations.