Improving Health Outcomes Through Equity and Access

Better access to healthcare equals better health outcomes. Oftentimes, health outcomes depend on factors beyond the control of patients and their doctors. Disparities across age, gender, race, ethnicity, and socioeconomic level are some examples. Biogen’s approach toward health equity involves researching and understanding these issues as well as their compounding factors, such as, inadequate representation in
clinical trials and limitations to accessing care. Overall, the goal is clear: improve health outcomes for Black, African American, Hispanic, Latino, and other underserved communities in Biogen’s main disease areas.

Issues of culture awareness and mistrust remain prevalent alongside a host of other considerations prior to enrollment in a clinical trial. To address these issues, Biogen uses a multi-channeled health equity strategy
which includes incorporating diverse community perspectives and insights into our drug development and
engaging with communities by partnering with organizations to educate, build awareness, establish trust, and drive health equity.

When recruiting for clinical trials, Biogen teams are building patient engagement strategies including setting race and ethnicity recruitment targets for their trials that reflect the patient populations they intend to treat.
These targets are supported by robust DE&I and patient-focused drug development strategies. The results are
clear with 2021 having 100% of studies in the U.S. including a plan to recruit participants from underrepresented communities to ensure the study population is representative of the intended treatment population.

Biogen has also fostered employee collaboration to increase awareness of health equity needs cross-functionally.
In 2021, they introduced a Health Equity Ideas Cafe Series to deepen organization-wide understanding of Biogen’s commitment, including representation in clinical trials and equitable access.

Biogen’s approach to health equity is guided by the motivation to help those who have historically been underserved. Concretely, this commitment to health equity looks like: clinical trial diversity, empowering underrepresented groups, engaging employees, and launching new tools to improve accessibility.

To learn more, visit, biogentriallink.com

Written by Biogen

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Volunteer opportunities with CISCRP, visit our Volunteer page.

Improving Health Outcomes through Equity and Access

Article from our 2022 October Patient Diversity Campaign

Better access to healthcare equals better health outcomes. Biogen strives to increase access and address the barriers that patient populations face. Oftentimes, health outcomes depend on factors beyond the control of patients and their doctors. Disparities across age, gender, race, ethnicity and socioeconomic level are some examples. One of the key parts to Biogen’s approach involves researching and understanding these issues as well as their compounding factors, such as, inadequate representation in clinical trials and limitations to accessing care. Overall, the goal is clear: improve health outcomes for Black, African American, Hispanic, Latino and other underserved communities in Biogen’s main disease areas.

Research and Collaboration

Issues of culture awareness and mistrust remain prevalent alongside a host of other considerations prior to enrollment in a clinical trial. To address these issues, Biogen uses a multi-channeled health equity strategy which includes incorporating diverse community perspectives and insights into our drug development and engaging with the community by partnering with organizations to educate, build awareness, establish trust, and drive health equity.

In partnership with the Center for Information and Study on Clinical Research Participation (CISCRP), Biogen formed a Community Advisory Board (CAB) of patient advocates from underserved and underrepresented communities who co-develop honest and transparent educational assets for patients, HCPs and clinical trial sites to discuss the importance of diversity in clinical trials. The CAB has also advised on various program and study designs as well as the development of Biogen Trial Link, a publicly facing website to learn more about and find clinical trials.

In collaboration with the National Minority Quality Forum (NMQF), Biogen launched the Clinical Trial Index and Clinical Trial Learning Community (CTLC) which uses U.S. heat maps of Medicare/Medicaid beneficiary data by patient demographics and maps them against clinical trial site locations. This information seeks to identify the right sites in the right locations to meet patient needs. The CTLC launched as a virtual space for local stakeholders and subject matter experts to integrate routines in local care networks to increase underrepresented and underserved population participation in clinical trials.

Clinical Recruitment and Specific Disease Areas

As the clinical study recruitment begins, Biogen teams track and report their progress toward the study targets that represent the epidemiology of their respective disease. Given this commitment, 2021 had 100% of Phase 1–4 studies in the U.S. including a plan to recruit participants from underrepresented communities to ensure the study population is representative of the intended treatment population. They also launched an Internal Participant Demographic Distribution Dashboard to track and measure the demographic distribution of participants across programs and studies, and to compare directly to epidemiology targets. Within certain disease areas, Biogen has built community and faith-based outreach and education programs for disease awareness and general clinical trial education with trusted organizations such as HEAL Collaborative and Proximity Health Solutions.

In Alzheimer’s specifically, they have sponsored the Bright Focus Foundation’s Virtual Community Outreach Series, bringing sustained Alzheimer’s disease and clinical trial education and access to communities with an underrepresented focus. Additionally, Biogen co-developed a paper with the NMQF titled “A Roadmap for Real-World Evidence Generation in Alzheimer’s Disease.” It highlights how real-world evidence can include larger patient populations that are historically underrepresented in randomized controlled trials.

In Lupus, Biogen collaborated with Saira Z. Sheikh, M.D., Director of University of North Carolina (UNC) Rheumatology Lupus Clinic and Director of the Clinical Trials Program at UNC’s Thurston Arthritis Research Center, to gather insights on barriers to clinical trial enrollment among underrepresented groups. These insights are being used to inform Biogen’s clinical development programs in lupus and address equity in study participation. For the Phase 3 studies in systemic lupus erythematosus (SLE), Biogen set enrollment targets that reflect the prevalence of SLE in Black or African American and Hispanic and/or Latino communities to achieve appropriate representation. Through partnerships with community-based Proximity Health Solutions and faith-based HEAL Collaborative, along with expert panels of community leaders, HCPs and patient advocates, they participated in seven events across the U.S. in 2021 to educate communities about lupus and clinical trial research.

A Holistic Approach to Inclusion and Equity

Beyond this commitment to the patients and their specific needs, Biogen also focuses on the workers involved in clinical trials. In 2021, they joined the Association of Clinical Research Professionals (ACRP) as part of ACRP Partners in Workforce Advancement™, which works to expand the diversity of the clinical research workforce and to set and support standards for workforce competence.

Additionally, Biogen has fostered employee collaboration to increase awareness of health equity needs cross-functionally. In 2021, they introduced a Health Equity Ideas Cafe Series to deepen organization-wide understanding of Biogen’s commitment, including representation in our clinical trials and equitable access. The three-part series engaged internal and external guest speakers on themes of lupus awareness, patient voices and community partnerships, and equitable access to healthcare. In addition, Biogen hosted an event with John Sawyer, Ph.D., ABPP-CN and a board-certified clinical neuropsychologist of the Ochsner Neuroscience Institute; and Donnie Batie, M.D., on successful approaches to caring for underrepresented patients.

From their research into healthcare disparities and patients’ needs, Biogen’s approach to health equity is guided by the motivation to help those who have historically been underserved. Concretely, this commitment to health equity looks like: clinical trial diversity, empowering underrepresented groups, engaging employees, and launching new tools to improve accessibility.

Visit Biogen Trial Link to learn more about clinical trials and how you can support equity and access efforts such as these.


To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Volunteer opportunities with CISCRP, visit our Volunteer page

Learn more about our Patient Diversity Campaign Here

The Clinical Trial Challenge: Boosting Clinical Trial Appeal in Patient Communities – Part 1

Part 1 of 2 Part Series

Clinical research is critical to progress towards new treatments and cures for all diseases. But engaging patients in clinical trial participation is often an uphill battle. Hear from Rare Patient Voice Senior Vice President Pam Cusick and patient advocate Grace Charrier about the results of a new RPV survey exploring factors that could make participation of greater interest to patients and family caregivers.

Read Webinar Highlights Here
Read Q&A Responses From the Webinar

Pam Cusick | Panelist
Senior Vice President, Rare Patient Voice

Grace Charrier | Panelist
Host, Cancer Convos with Grace B., and Cancer Advocate

To view the second part in this webinar series, click here.

Voices from Within: Humanizing Clinical Research Data: Episode 2- Conversations on DCTs: Data Privacy

3 PART FLASH WEBINAR SERIES

Join in on the growing topic of patient data protection and policies. In a time when healthcare has adopted technology and data collection software at an exponential rate, it is important to educate patients and stakeholders on what that standard for patient data collection should be. 

Join Curebase’s Vice President of Clinical Trial Innovation, Jane Myles, Vice President of Clinical Operations, Sean Lynch, and Type 1 Diabetes Clinical Trial Participant, Phyllis Kaplan for Episode Two of our Voices from Within: Humanizing Clinical Research Data series.

View the webinar below.

Watch Webinar Episodes 1 & 3

Voices From Within: Humanizing Clinical Research Data:
Episode 1 – Patient Data Collection 101 Flash Webinar
Watch Here

Voices from Within: Humanizing Clinical Research Data:
Episode 3 – Conversations on Clinical Trials
Watch Here

Panelists:

Jane Myles
VP, Clinical Trial Innovation, Curebase

Phyllis Kaplan
Type 1 Diabetes Clinical Trial Participant

Sean Lynch
VP of Clinical Operations, Curebase

DEI Series — Educating Clinical Research Practitioners Through Video

Recently, CISCRP partnered with WCG to create a video for researchers and study staff that emphasizes the importance of diversity in clinical trials. In addition to educating the public, raising awareness among research professionals about the importance of Diversity, Equity, and Inclusivity (DEI) is critical to increasing cultural competence and understanding of participants’ needs.

You may have read our post about how videos are becoming a key tool of health literacy, and how clinical research could benefit from more videos communicating information in an accessible way. In this post, we dive into our creative process for producing a video with that objective in mind.

We were delighted to collaborate with WCG on this video. WCG partnered with CISCRP to produce this video because of our expertise in educating the public and clinical research professionals about the importance of diversity in clinical research. Like CISCRP, WCG is committed to helping ensure that the public gets the most effective and safe treatment possible. 

Because of that urgent need, WCG were very excited to work with CISCRP on addressing this topic. We know this engaging video collaboration will help energize researchers, and especially site staff, to bring more diversity into their trials.

To see the video, click here.

Research and Creative Planning

As with any complex project, whether you’re writing a novel or baking a cake, it’s important to plan every step in advance. However, for this project, assessing our audience’s needs, perceptions, and general level of cultural competency would determine how we structured the tasks ahead – so we started there.

The purpose of this video was simple: to encourage clinical research professionals to take tangible actions to ensure their trials had diverse populations. Given our experience developing educational materials on diversity in clinical research by collaborating with patients, their communities, and the public to understand their concerns and needs, we knew the key messages researchers needed to see and hear. Delivering that message in a way that will have an impact was the challenge.

Clinical research professionals may already know about the importance of diversity in clinical research and may even know about the available tools and practices to improve their trial’s diversity. So, how could we convince them to make more of an effort to invest in and implement these tools and practices?

We certainly didn’t want to bore them with information they already knew. Instead, we decided to focus on an impactful narrative to give them that extra push and reinvigorated perspective. And we had to do it in under 3 minutes.

Script

We decided on an animated video that portrayed a realistic scenario: patients from a diverse background whose prescribed treatments were not as effective or tolerable as demonstrated in the less diverse study populations during clinical trials. The storyline focuses on one patient to begin with, then shifts to a larger patient population who all realize that the clinical trials for the treatment they needed did not include patients from their respective communities.

As each patient asks their doctor some tough questions, treatments disappear from the shelves of the pharmacy shown in the animation:

“Could my race affect how this drug works?”

“Was the drug tested on women like me?”

“Could my weight be why this drug didn’t work?”

Then in the final scene of this part of the video, the problem is summed up by a disappointed patient:

“I think I understand. They didn’t test this for people like us.”

After the scenario, the narrator addresses this problem directly to the audience. It was important that the messages were not received as a critique of researchers—as we say in the video, there’s already so much good work they’re doing. Rather, we wanted to bring this subject to the top of their minds and say that by taking steps to ensure diversity in their trials, we can avoid situations like the one they just watched.

The script went through several drafts which included reviews from CISCRP’s team, our WCG collaborators, and subject matter experts on DEI in clinical research.  Each round of feedback included more voices and perspectives from folks in the clinical research world. As a result, each draft became more engaging, appropriate, and relatable to our intended audience.

For example, the original plan was to follow one patient. In the final script, we decided to combine the stories of five diverse participants. Then we realized that our characters’ names were not diverse. At first, they were named common names, which likely wouldn’t represent the diverse groups that are underrepresented in clinical research. We also ensured we represented the doctor, a “Dr. Smith,” from a white man to a black woman.

Key Learnings

In early drafts of the video animations, while the characters had different skin tones, they didn’t have other aspects that represented diversity. Something important we all have to keep in mind is that diversity comes in many different forms—from some of the more obvious things, like race, ethnicity, sex, and gender, to more specific details like health conditions, weight, and even lifestyle. So, we made a few subtle tweaks to represent diverse bodies, appearances, and cultural appearances.

Also, in choosing the voice-over actor, we settled on someone who represented a diverse population to add credibility to the messaging.

Some of the considerations went beyond diversity. We wanted our video to feel real, because the scenario we crafted is something that unfortunately happens too often. Given the prevalence of this issue, we also wanted the video to demonstrate sensitivity towards the difficult situations participants often face.

For example, in early drafts of the video, the doctor was smiling throughout, even as she learned that her patients were suffering. Not only is this not realistic, it appeared insensitive and even offensive. Still, we had to find the balance between making character emotion powerful while making sure they didn’t act like children’s cartoon characters.

Finally, once we were done tweaking the thematic elements of the video, we polished the presentation to a fine, professional gloss. Over the course of three drafts, spread out over weeks of review, we gave feedback on the timing of edits, the tone and speed of the voice actors through specific sections, and the subtle connotations of the animated characters’ movements. All of this was to ensure that this video was produced to the highest standards.

Never call it a day

As we’ve mentioned in other posts, the health literacy process does not stop here. We want to learn how effective this video is, and what we can do to improve our communication and education in future projects. Whether we’re making content for a professional audience or for the general public, the insights we gain on any given project accumulate and inform the production of future projects.

Written by Scott Finger, Senior Editor – Health Literacy, CISCRP

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Volunteer opportunities with CISCRP, visit our Volunteer page

Voices from Within: Humanizing Clinical Research Data: Episode 3- Conversations on Clinical Trials

Jane Myles, Curebase’s VP of Clinical Trial Innovation, Sean Lynch, VP of Clinical Operations, and patient Qiana Alexander discuss the difference between clinical data and experiential data, focusing on the patient point of view. In their engaging conversation, they shared the importance of collecting patient experience data, how patient input/insights can be used to assist in trial design for decentralized clinical trials, and the difference in how DCTs enable gathering patient feedback.

View the webinar below.

Watch Webinar Episodes 1 & 2

Voices from Within: Humanizing Clinical Research Data:
Episode 1- Patient Data Collection 101
Watch Here

Voices from Within: Humanizing Clinical Research Data:
Episode 2 – Conversations on DCTs: Data Privacy
Watch Here

Panelists:

Jane Myles
VP, Clinical Trial Innovation, Curebase

Sean Lynch
VP of Clinical Operations, Curebase

Qiana Alexander
Lupus Clinical Trial Participant

Findings from a Long-Term Patient Engagement Model

Clinical Researcher: The Authority in Ethical, Responsible Clinical Research
August 2022 (Volume 36, Issue 4) | PEER REVIEWED

Government agencies, pharmaceutical companies, and patient advocacy groups increasingly involve patient and caregiver feedback at various checkpoints throughout the clinical trial process to develop research protocols with greater benefit and less burden to participants and their families.{1} Involving patients and their support network in the protocol development process can take several forms, including patient advisory boards, surveys, and interviews. However, best practices and standardized processes for developing patient-centric trials have not yet been widely established.{1,2}

In recent years, several organizations and initiatives have emerged with the intention of advancing patient engagement in drug development and/or research, including the Patient-Centered Outcomes Research Institute (PCORI),{3} the Clinical Trials Transformation Initiative (CTTI),{4} the U.S. Food and Drug Administration’s (FDA’s) Patient Engagement Collaborative with CTTI,{5} the FDA’s Patient-Focused Drug Development (PFDD) program,{6} and the not-for-profit Patient Focused Medicines Development (PFMD){7} organization. All seek to increase patient involvement across the lifecycle of medical product development, including through the phases of research, and to improve trials in terms of quality, efficiency, safety, and ethical conduct, as well as in engaging all stakeholders as equal partners. The current trajectory toward greater patient involvement represents an opportunity to develop and conduct more effective research via outcomes such as realistic inclusion and exclusion criteria, lower participant burden, and patient-relevant endpoints.{8}

To create meaningful impacts, patient engagement strategies need to be put into place that result in substantive feedback and turn communication into actionable change.{9} Numerous types of patient engagement approaches exist, including community advisory boards, patient advisory boards, individual interviews, and surveys (see Table 1). Tools for implementing these models have been provided by the FDA, PFMD, the PARADIGM (Patients Active in Research And Dialogues for an Improved Generation of Medicines){10} initiative in Europe, and other organizations committed to furthering patient engagement across the lifecycle of medical product development.{11-14} For example, PFMD has published a series of toolkits, resources, and “how-to” guides for quality and effective patient engagement.{12,13}

Among the wide range of available tools and engagement strategies, one approach Janssen utilized was to implement a long-term, recurring global community advisory board. While one-time market research activities such as interviews and surveys can and do yield actionable insights, single touchpoint projects can also limit the amount and type of data obtained and usually solicit patient feedback only on a single protocol. In contrast, the community advisory board model’s long-term focus provides the opportunity to establish a long-term partnership with patients based on trust, knowledge-building, and the reciprocity of true dialogue.

Through the Patient Voice in clinical trials program, Janssen routinely obtains patient and caregiver feedback into clinical trial design across all therapeutic areas.

 

Because many learnings apply to more than one clinical trial, and because of a desire to build on insights in an iterative manner, the Janssen Clinical Insights and Experience team collaborated with an independent third party—the nonprofit Center for Information and Study on Clinical Research Participation (CISCRP){15}—to create a standing Global Community Advisory Board (GCAB) for the immunology therapeutic area.

 

The GCAB consisted of patient advocates with a variety of immunological conditions. The main objectives of the GCAB were to obtain feedback on strategies and solutions intended for patients, with a primary focus on clinical trial design; establish a strong working relationship between patients and Janssen; establish lines of communication with the broader community of patients with immunologic conditions that Janssen could connect with, both regularly and on an ad hoc basis; and ensure that patient-centric practices and principles are incorporated into clinical trials sponsored by Janssen.

This approach to engaging with patients aligns with industry recommendations and frameworks. It was formed to be a standing, long-term advisory panel consisting mainly of patients from around the world, and was facilitated by an independent third party (both CISCRP and Janssen personnel were active panel members). This model focused on the broader therapeutic area of immunology to promote a long-term, more impactful engagement. Thus, the GCAB provided the opportunity to explore a host of questions about Janssen’s clinical trials development and engagement strategies with a group of patient advocates in an atmosphere intended to build openness, trust, and mutual respect. The transparency and sense of trust built over time through this model were found to benefit both sides of the patient-sponsor equation.

Patients and Methods

Formation of the Immunology GCAB

Through relationships with patient advocacy groups and its own participant community, CISCRP identified a group of 11 individuals (eight women and three men) with various immunological conditions who expressed a willingness to serve on the GCAB. Some, but not all patients were familiar with the clinical trial process. Because of their experience in community and patient support groups, even GCAB members who were mostly unfamiliar with clinical trials had some knowledge about clinical research. The decision to convene an international advisory board resulted in a population of patient-advocates from seven countries across three global regions: North America (United States), Europe (Czechia, Denmark, Estonia, United Kingdom), and Asia-Pacific (India, Taiwan). Each patient advocate had a keen understanding of the specific challenges of his or her country of origin. CISCRP and Janssen intended for the GCAB to be a standing, long-term advisory panel with greater flexibility than the traditional, single touchpoint patient advisory board model. The Janssen team consisted of members in clinical research and operations roles, as well as members in patient engagement roles. Additional personnel from across the business also joined certain GCAB meetings as needed.

GCAB Activities

Over the course of one year, two in-person GCAB meetings were held: a kick-off meeting in February 2019 and a year-end review meeting in February 2020. In between, there were four GCAB virtual meetings held via an online platform. GCAB members also engaged in ad hoc communications throughout the year, both to provide feedback on questions that arose between meetings and as they desired to support one another.

In addition to the six full-group meetings, a further six virtual condition/topic-specific meetings were held. Furthermore, one-on-one feedback was solicited from GCAB members during in-person meetings (see Figure 1).

Figure 1: Immunology Global Community Advisory Board (GCAB) Meeting Schedule.

GCAB members also completed surveys and reflection exercises for Janssen to address areas of interest. These included general questions, such as the perception of clinical trials among patient groups, and specific questions that assessed the effectiveness of trial information materials, receptivity to trial-related technologies, inclusion/exclusion criteria, and planned clinical trial assessments that could increase or decrease participation.

Results

Impact on Janssen Programs

For Janssen, the opportunity to seek patient feedback on various projects over time resulted in several operational and protocol changes to clinical trial study designs that fulfilled the original aim of the GCAB. The willingness of GCAB members to respond to study materials in conversation with staff and researchers has changed some of the baseline assumptions of the trial design process. This enabled a greater focus on patient-relevant outcomes, which procedures are truly necessary to achieve the desired results, and how to inform and empower patients using materials that respect their experience of living with a condition. The decision to involve patients with a broad range of immunologic conditions—rather than any specific one—presented the opportunity to gather feedback and discuss problems consistently, without the need to pause a trial or to complete work based on a specific protocol’s schedule. This efficiency enabled changes to be implemented as quickly as possible, which further built trust among GCAB members.

Seeking feedback from GCAB members on proposed trials resulted in several straightforward actions intended to increase enrollment and retention. For example, a proposed trial involved regular trial visits for bloodwork and medical photography, as well as required patients to wear an actigraphy device to monitor adherence and physical activity. GCAB members highlighted the time burden involved in short repeat visits to a study site would limit participation by those with unreliable transportation and/or a long travel distance. Numerous concerns also arose regarding the invasiveness of the actigraphy device, as well as regarding data security (the latter particularly from European members). Ultimately, investigators chose to remove these requirements from the study.

GCAB Feedback Additionally Supported and Informed Patient Engagement Strategies

In addition to gathering and acting on clinical trial design feedback, Janssen obtained insights from GCAB members to help shape MyTrialCommunity, a website for engaging with patients enrolled in Janssen clinical trials. Member feedback was integral to the development of this initiative. They recommended the site name and suggested changing logos and adding testimonial videos from patients to explain the trial process in a non-threatening environment. In one example, a GCAB member shared personal experiences with the life-altering diagnosis of inflammatory bowel disease (IBD), as well as a video from a patient advocacy group about the day-to-day challenges of dealing with IBD. Janssen staff were moved by these patient stories and experiences and appreciated the opportunity for open dialogue about the impact of an IBD diagnosis, as well as the impact of living with the condition on people’s lives. The Research and Development team disseminated the video throughout the research group, to drive home the real-world impacts of their work.

Trust, Awareness, and Advocacy Strengthened among GCAB Members

This integration of GCAB feedback led GCAB members to feel empowered and enhanced their engagement in the advisory board process.

GCAB members expressed surprise that their input affected the trial design, as some previous patient engagement experiences had not resulted in the same level of transparency and change. As a result, GCAB members reported a resulting sense of empowerment and engagement in the advisory board process, as they could observe the impact of their feedback and how it was genuinely valued.

Overall, GCAB members reported having a positive engagement experience and gaining a greater understanding of clinical trials as well as pharmaceutical companies in general. Before joining the GCAB, some believed clinical trials to be a last-resort option where patients were treated as “just a statistic.” Their perceptions evolved as members learned more about how clinical trials work, the level of effort and care pharmaceutical companies use to implement responsible trials, and the role of clinical trials in finding new treatments for their condition. Thus, GCAB members now understood that not only were clinical trials a viable treatment option for a wide range of patients, but they were an essential component in advancing the causes and objectives of their patient community.

These paradigm shifts among GCAB members highlight the success of the GCAB model in providing a collaborative, supportive environment where GCAB members could become more engaged in and educated about clinical trials. This enhanced the quality and actionability of insights, as well as greater understanding of things that could not be changed in clinical trial design.

Consistent Communication and Transparency are Important

For many patients with chronic inflammatory conditions, barriers to research can be present from the onset of their condition journey, which can ultimately influence patients’ perception and willingness to participate or engage with clinical research. GCAB members reported difficulties obtaining a diagnosis, and once diagnosed, still dealt with feelings of shame and isolation brought on by their symptoms and the knowledge that they may never “get better.”

Outside the United States, patients reported greater difficulty obtaining information about clinical trials. This was particularly the case for smaller European countries without a robust patient organization network. Patients were frustrated by a lack of information about available trials on the part of their physicians and/or a perceived lack of interest from physicians who would not personally participate in the trial. Even when they searched online, barriers to information included a lack of clarity about where trials are available, limited country-specific public information about ongoing clinical trials, and engagement materials being available only in English.

Further barriers included an overuse of technical language by researchers.

The era of molecular diagnostics (e.g., biomarkers) and treatment has added new layers of complexity to the research process, and this area of research remains too difficult for many patients to understand. GCAB members requested an in-depth, accessible explanation of what biomarkers are and how targeted therapy works.

A lack of transparency about what data will be collected from patients, how it will be used, and the risks and benefits of experimental interventions was also cited frequently. Long lists of potential adverse events can intimidate potential trial participants, especially when not accompanied by clear explanations of their true risk and prevalence. Further, patients consistently mentioned their discomfort with joining trials where they would never find out the results, whether their own or the overall trial outcomes, including not knowing whether they had been randomized to the active treatment or placebo arm of a study. The need for trial documents in plain language was another item identified by GCAB patients as an important barrier to overcome in building trust and ensuring patients are truly informed before they consent to clinical trial participation.

Meaningful Communication is a Key Component of True Patient Engagement

Meaningful communication, as identified by the GCAB, was that which either resulted in actionable strategies (e.g., adjusting inclusion/exclusion criteria, changing ads to increase diversity/representation) or clarified the parts of a clinical trial that remain difficult for most patients to understand. One such opportunity is the regulatory and informed consent process—giving patients information about the importance and function of regulatory requirements in place to protect participant safety was repeatedly reported by the patients as increasing their sense of trust as well as their willingness to engage with a lengthy consent process. Additionally, many patients were unaware of the many “moving parts” of a clinical trial that are not patient-facing, so educating patients about the time constraints for things such as data analysis and regulatory approval can increase trust in the timeline of therapeutic development. Ensuring that patients will have access to results of a study—and thus an understanding of how their time and effort contributed to the study—is also advantageous to building trust.

One unexpected outcome of this engagement model was that, by educating and empowering patients, they become more comfortable with and supportive of the clinical trial process in general.

Several GCAB members reported that their new understanding of safety measures for Phase I and II trials removed their fear of being “lab rats.”

On the sponsor side, in response to GCAB member feedback, Janssen took steps in several areas. For example, a patient brochure was substantially modified to provide more comprehensive data about the purpose and conduct of clinical trials. A patient testimonial video was also modified to increase a sense of inclusivity by starting with definitive statements instead of questions.

A meeting addressing topics of digital health brought up questions about data collection and security. There was a division between GCAB members on this topic, with European GCAB members expressing greater skepticism about providing digital information about themselves than Asian and U.S. members. In response, Janssen put in place measures to increase transparency about what trial data would be collected and how the data would be stored and used. Discussion from yet another meeting spurred the development of patient educational materials to more clearly describe the benefits and purpose of early-stage clinical trials and the potential long-term benefits of these trials to patient health.

Best Practices and Learnings for Future Engagement Models

To ensure that meetings were structured, had pre-planned agendas and moderator guides, and were facilitated by an independent third party, Janssen partnered with CISCRP to implement the GCAB model. As a neutral party and liaison between patients and the pharmaceutical company, CISCRP helped establish a baseline of trust for both advisory board members and sponsor personnel. Because of its extensive experience in patient engagement initiatives such as community advisory boards, CISCRP was also able to manage the logistical coordination of GCAB meetings, ensure that the project progressed according to determined timelines, and accommodate GCAB member needs and questions in a timely manner.

Despite favorable responses about the GCAB and the approach taken, there were some limitations reported by GCAB members across several broad categories. These included: respect for patients’ time, organization of activities, information overload, burdens to patients caused by holding several meetings, a focus on English language, and a general U.S.-centric focus on materials. Although non-U.S. GCAB members had excellent English-language skills, several non-native speakers expressed discomfort with the speed of presentations, calling for a greater level of comfort with written communication to ensure that non-English native speakers are not excluded or discriminated against. Non-U.S. patients remarked that, especially in smaller countries, pathways to information about clinical trials are lacking. This presents an opportunity to explore enrollment opportunities via non-U.S. patient advocacy and physician networks.

Regardless of language of origin, patients reiterated the need for materials and presentations to be given in plain language and in easily digestible amounts.

Adequate discussion was a key factor leading to patient engagement and trust in the process. Patients frequently noted the need to keep meetings—both online and in-person—organized and on a schedule that respected patients’ time constraints.

Discussion

A growing focus on patient-centered outcomes by sponsors and regulatory agencies is an opportunity to establish practices that mutually benefit patients and pharmaceutical companies and other stakeholders. The year-long experience with the GCAB provided rich, actionable insights that could not have been obtained from other stakeholders, and demonstrated that a key component tying the GCAB’s feedback together was the sense of trust built by consistent, two-way dialogue.

Two overall themes became clear during the data review of the GCAB’s first year in operation. First, patients want more information and transparency. Throughout meeting sessions, patients wanted to know more about the purpose of clinical trials, the thinking behind different trial procedures, data collected, and inclusion/exclusion criteria. They wanted to know that results would be communicated to trial participants, and how their personal information would be used for the purposes of the clinical trial. To this end, Janssen has taken steps, such as creating educational materials about drug-development trials, with the aim of lessening the stigma of these studies as a “last resort.”

GCAB feedback highlights the importance of engaging patients in the clinical trial design process. Effective communication about how patients are centered in the conduct of a study can increase retention through trust and transparency. Patient engagement can allow researchers and sponsors to ask questions such as:

  • Are there opportunities to reduce the number of procedures, especially those which are highly burdensome for patients (for example, invasive and painful procedures like biopsies, endoscopies, and blood draws)?
  • How can visits and data collection be grouped to minimize travel and time burdens on participants and their support networks?
  • Which interventions, either additive (such as patient comfort kits for procedure days) or subtractive (for example, requiring fewer blood draws), have the greatest impact on the participant’s trial experience?

These types of practical questions can help researchers design protocols focused on greater efficiency, minimized invasiveness, and respect for the people taking part, while still enabling the collection of necessary evidence. The actions Janssen took in response to GCAB feedback resulted in increased engagement by both research teams and GCAB members.

It is beyond the scope of this article to delve into the financial metrics of this type of partnership, but involving patients as partners is a pragmatic strategy to increase transparency of the research process and overcome disparities in health research.{18} Other groups have assessed the financial benefit to sponsors and found significant potential for savings in terms of efficiency and retention.{19} One such potential is to troubleshoot trials before they even start. Not only does this potentially increase enrollment and retention, but it can prevent costly protocol amendments. The investment in time and educational materials in this new type of engagement model was well worth the outcomes in terms of engagement and trust.

Conclusions

A meaningful partnership among the various stakeholders in any clinical trial depends on defining goals, choosing the right partners, and investing the time to ensure that all voices are heard.{20} By engaging an advisory board consisting of knowledgeable patient advocates, we were able to present and receive valuable feedback on numerous projects and scenarios, including educational materials, inclusion/exclusion criteria and other protocol elements, and regulatory and consent documents.

In doing so, the sponsor received actionable takeaways, which resulted in changes to different elements of several protocols.

Engaging a neutral third party to serve as primary contact for patients, ensure well-planned meeting objectives and agendas, and collate feedback helped achieve quality engagements and effectively summarize feedback.

Further, the opportunity to make changes and demonstrate them to the GCAB was a powerful motivator for Janssen staff and researchers, as was the increased understanding of the real-world, daily experiences of patients living with immunological conditions. Because the GCAB was convened as a long-term advisory body, we were able to demonstrate to GCAB members the actions Janssen took in response to their recommendations. This investment in time and evidence proved to be key in the trust-building process and was cited by all GCAB members as a major factor in their overall positive reaction to this initiative and their views on clinical research in general.

Acknowledgements

GCAB members Nandan Baruah, Alicia Aiello, Jackie Zimmerman, Janek Kapper, Lucie Lastikova, Candace Lerman, Simon Stones, Jenni Lock, and Charlotta Norgaard critically reviewed and contributed to the contents of this article. Thanks also to Cynthia Wise, retired Global Development as Portfolio Delivery Operations Program Leader, Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), for her contributions.

Funding/Support: This work was funded by Janssen Pharmaceuticals.

Role of the Funders/Sponsors: Janssen Pharmaceuticals worked collaboratively with CISCRP to design and conduct this work. CISCRP was responsible for the coordination, management, and analysis of the data for this research. Janssen worked collaboratively with CISCRP to interpret the data as well as to prepare, review, and approve this manuscript, and on the decision to submit the manuscript for publication. Those currently active at Janssen Pharmaceuticals who were directly involved with the preparation of this manuscript are listed as its authors.

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Volunteer opportunities with CISCRP, visit our Volunteer page.

Written by: Annick de Bruin, MBA; Shalome Sine, MPH; Lieven Van Vijnckt, PharmD; Alyson Gregg, MBA; Catherine Cole

Annick de Bruin, MBA
Senior Director of Research Services
CISCRP, Boston MA

Shalome Sine, MPH
Health Informatics and Reporting Analyst
The Center for Health Information and Analysis, Boston MA

Lieven Van Vijnckt 
PharmD Investigator and Patient Engagement as TA Head (IMM – ID&V)
Janssen Pharmaceutica NV, Beerse Belgium.

Alyson Gregg, MBA
Director of Patient Insights
Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), Titusville N.J.

Catherine Cole
PharmD Investigator and Patient Engagement as TA Head (IMM – ID&V)
Janssen Pharmaceutica NV, Beerse Belgium.

References:

1. Vat LE, Finlay T, Schuitmaker‐Warnaar TJ, et al. 2019. Evaluating the “Return on Patient Engagement Initiatives” in Medicines Research and Development: A Literature Review. Health Expectations 2019. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6978865/
2. Gregg A, Getz N, Benger J, Anderson A. 2019. A Novel Collaborative Approach to Building Better Clinical Trials: New Insights from a Patient Engagement Workshop to Propel Patient-Centricity Forward. Therapeutic Innovation & Regulatory Science 2168497019849875.
3. Fischer MA, Asch SM. 2019. The Future of the Patient-Centered Outcomes Research Institute (PCORI). Journal of General Internal Medicine 34(11):2291–2.
4. Bloom D, Beetsch J, Harker M, et al. 2018. The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials. Therapeutic Innovation & Regulatory Science 52(2):206–13.
5. https://www.fda.gov/patients/learn-about-fda-patient-engagement/patient-engagement-collaborative
6. https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development
7. https://patientfocusedmedicine.org/
8. Tenaerts P, Madre, L, Landray M. 2018. A Decade of the Clinical Trials Transformation Initiative: What Have We Accomplished? What Have We Learned? Clinical Trials 15(S1):5–12.
9. Boutin M, Dewulf L, Hoos A, Geissler J, Todaro V, Schneider RF, et al. 2017. Culture and Process Change as a Priority for Patient Engagement in Medicines Development. Therapeutic Innovation & Regulatory Science 51(1):29–38.
10. https://www.imi.europa.eu/projects-results/project-factsheets/paradigm
11. Dewulf L. 2014. Patient Engagement by Pharma—Why and How? A Framework for Compliant Patient Engagement. Therapeutic Innovation & Regulatory Science 49(1):9–16.
12. How-to Guide on Patient Engagement in Clinical Trial Protocol Design. https://pemsuite.org/How-to-Guides/Patient-engagement-in-clinical-trial-protocol-design.pdf
13. Practical How-to Guides for Patient Engagement. https://pemsuite.org/how-to-guides/
14. Patients Active in Research and Dialogues for an Improved Generation of Medicine. PARADIGM Patient EngaRgement Toolbox. https://imi-paradigm.eu/petoolbox/
15. https://www.ciscrp.org
16. Center for Drug Evaluation and Research. FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making. www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical
17. Jones CW, Braz VA, McBride SM, Roberts BW, Platts-Mills TF. 2016. Cross-Sectional Assessment of Patient Attitudes Towards Participation in Clinical Trials: Does Making Results Publicly Available Matter? BMJ Open 6:e013649.
18. Šolić I, Stipčić A, Pavličević I, Marušić A. 2017. Transparency and Public Accessibility of Clinical Trial Information in Croatia: How it Affects Patient Participation in Clinical Trials. Biochem Med (Zagreb) 27(2):259–69.
19. Fillon M. 2017. Strategies to Boost Minority Participation in Clinical Trials. J Natl Cancer Inst 109(4):10.1093/jnci/djx076.
20. Levitan B, Getz K, Eisenstein EL, Goldberg M, Harker M, Hesterlee S, Patrick-Lake B, Robers JN, DiMasi J. 2018. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project. Therapeutic Innovation & Regulatory Science 52(2):220–9.

The Importance of Telehealth for Rare Diseases

Healthcare must reflect all of the opportunities of telehealth, but especially so for those suffering from rare diseases.

 

Before the pandemic, telehealth made up less than 0.01% of healthcare visits across the United States. By mid-April of 2020, that number had skyrocketed to 69%. While the concept is not new, patients could not always access it. Then, as a result of patient advocacy efforts, the COVID-19 public health emergency declaration expanded access to telehealth. This was welcome news for patients, especially those living with rare diseases.

The importance of telehealth

Why is telehealth important for people with rare diseases? For the past year, the team at the National Organization for Rare Disorders (NORD) has been working to help the rare disease community safely navigate the pandemic. Patients and caregivers have joined us to advocate for expanded access to telehealth, and hundreds have told us about its positive effects.

 
These include helping them feel safer, eliminating days of travel to and from doctor visits, and moving forward with clinical trials that would have otherwise been delayed.

 

In the case of many rare diseases, there are only a handful of experts nationwide, or even worldwide, with expertise in certain conditions. In the absence of telehealth, patients travel long distances to access their treating provider, often experiencing long waits for appointments. Such travel can be costly, often results in missed work or school, and presents logistical challenges that can be overwhelming or insurmountable when there is severe burden of illness. Not least of all, travel can pose health risks because many patients with rare diseases are immune-compromised, putting them at greater risk for complications from COVID-19 and other illnesses.

 

Participation in clinical research is another important area to examine when looking at the role of telehealth. Clinical research is needed for rare diseases, as approximately 90% of the 7,000 rare diseases still do not yet have an FDA-approved treatment.

 

Clinical trials are essential to this process, yet many patients struggle to participate when the clinical trials take place far from home.

Knowing about the uphill challenges that exist, it is easy to understand how COVID-19 has had a significant impact for more than 25 million Americans living with rare diseases. During the pandemic, nearly 8 in 10 rare disease patients experienced canceled medical appointments.

A ray of hope

Despite disruptions to care, a ray of hope emerged with the rise of telehealth: 83% of rare disease patients were offered a telehealth visit by their provider, 92% of those who had a telehealth visit described it as a positive experience, and 70% would like the option for future medical appointments. These numbers show that the demand for telehealth among rare disease patients is strong. With continued information sharing and collaboration among experts, scientists, healthcare workers, and advocates, together we will come out stronger. 

Lisa P. Sarfaty, M.P.H., Director of Strategic Planning, National Organization for Rare Disorders (NORD)

Article from 2022 Clinical Trials Supplement, USA Today. View Supplement Here >

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Volunteer opportunities with CISCRP, visit our Volunteer page.

Addressing Barriers to Clinical Trial Enrollment

Clinical trials offer patients with difficult-to-treat forms of cancer a chance to receive the most up-to-date and promising care available with the prospects of improved health outcomes and the benefit of advancing medical research. Most patients express a willingness to participate in clinical research, yet only a small fraction ultimately end up enrolling in a trial due to barriers that make participation difficult or even impossible. Consequently, approximately 20% of cancer clinical trials fail due to insufficient patient enrollment.

Barriers to treatment
One of the most common barriers to trial participation is location. Most cancer trials are concentrated at large academic centers that have the resources to dedicate to research, yet most cancer patients receive their care at small, local oncology practices. According to a 2018 American Cancer Society Cancer Action Network (ACS CAN) report, Barriers to Patient Enrollment in Therapeutic Clinical Trials for Cancer, only about 1 in 4 patients has access to clinical trials where they are being treated. Yet, if asked to enroll in an available trial, more than half of eligible patients typically agree to do so.

Another barrier is cost. While private health insurance as well as Medicaid and Medicare are required to cover the routine medical costs of trial participation, there is no such coverage for other non-medical out-of-pocket expenses patients may incur.

Trial participants are often required to see their doctors more frequently, which can mean more money spent on things like gas, parking, food, and lodging. Those costs add up, especially for low-income patients. Research has shown financial burdens can lead to a nearly 30% lower trial participation rate among individuals with annual family income of less than $50,000.

Cancer care inequity
Unfortunately, these barriers and others often contribute to long-standing inequities in cancer clinical trials. Despite having an increased burden of disease, racial and ethnic minority groups, older adults, rural residents, and those with lower socioeconomic status are consistently underrepresented in cancer clinical trials.

This underrepresentation hampers research and, without deliberate efforts to rectify these disparities, research may miss why cancer outcomes are often worse for patients with limited access to care, lower incomes, and other factors.

No one should be disadvantaged in their fight against cancer because of how much money they make, the color of their skin, their sexual orientation, their gender identity, their disability status, or where they live.

ACS CAN has prioritized health equity to ensure that cancer patients are provided with equitable care based on social determinants of health. This is why ACS CAN is working hard to remove these barriers and ensure every patient has equitable access to clinical trials.

Reducing disparities
In 2018, ACS CAN began analyzing common barriers to patient enrollment and provided stakeholders in the research ecosystem with over 20 recommendations that should be taken to make trial enrollment easier for patients. Earlier this year, ACS CAN issued another report targeted specifically at reducing disparities in clinical trials.

Right now, ACS CAN is working to help pass the bipartisan DIVERSE Trials Act through both chambers of Congress. The legislation would help address health equity and disparities by allowing clinical trial sponsors to reimburse patients for non-medical costs associated with their trial participation — such as travel, parking, food, or lodging — and would allow trial sponsors to provide patients with the technology necessary to facilitate remote participation in clinical trials.

The goal of cancer research is to generate new knowledge that can be used to improve survival rates and quality of life for all patients with cancer. But to achieve that goal, it’s crucial that clinical trials reflect the broad diversity of cancer patients they’re hoping to treat. ACS CAN is dedicated to working with patients, survivors, and their loved ones to ensure that happens and to make cancer clinical trials accessible and available to all interested patients. Together, we can improve access and address healthcare disparities for a world with less cancer.

Lisa A. Lacasse, M.B.A., President, American Cancer Society Cancer Action Network (ACS CAN)

Article from 2022 Clinical Trials Supplement, USA Today. View Supplement Here >

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

The Extraordinary Gift of Clinical Trial Participation

Medical heroes can be found everywhere. They are mothers and fathers, siblings, children, friends, colleagues, and everyday people who have chosen to give the extraordinary gift of participation in clinical research.

Their decision to participate is an altruistic gift that always carries risk, usually offers no direct personal benefit, yet contributes profoundly to collective knowledge about the nature of disease, its progression, and how to better treat it. Ultimately, future generations are the direct recipients of this gift.

Most people know very little about clinical trials until they face the sudden and often unexpected prospect of a serious and debilitating illness for which no medication is available or adequate. Typically, patients, their families, their friends, and their healthcare providers must gather information quickly to make decisions about whether to participate. This rush to navigate the unfamiliar terrain of clinical trials invariably feels overwhelming and confusing.

Appreciating medical heroes
In 2004, the Center for Information and Study on Clinical Research Participation (CISCRP) was founded to provide outreach and education to those individuals and their support network considering participation in clinical trials. Based in the Boston area, this nonprofit organization focuses its energy and resources on raising general awareness, on educating patients and the public, and on enhancing study volunteer experiences during and after clinical trial participation.

Our many events and services are designed to improve public and patient literacy, to engender feelings of empowerment and control, to ensure more informed decision-making, and to recognize and appreciate medical heroes.

This special supplement also plays an important part in raising awareness and literacy. It is a reference resource
offering an introduction to clinical trials and thanking the millions of people and the clinical research professional community who, together, help advance medical knowledge.

At the present time, nearly 4,000 experimental drugs and therapies are in active clinical trials, and that number continues to grow as improvements are made in detecting disease, in discovering new medical innovations, and in understanding and addressing the root cause of acute and chronic illnesses.

 
At the very heart of all this promising clinical trial activity are medical heroes to whom we owe our deepest appreciation for their profound
gift of participation.

Written by: Ken Getz | Founder and Chairman, CISCRP

Article from 2022 Clinical Trials Supplement, USA Today. View Supplement Here >

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.