The Patient Perspective on Clinical Trials

For the past several years, there has been increasing discussion of what patient centricity means in clinical trials. The premise is that better study designs, approaches, and services that are focused on the needs and preferences of patients can improve clinical research participation and, therefore, help advance the development of medicine and medical devices. Yet, despite the focus on this concept, the general consensus is that much work remains to realize its potential.

Previous research by the Center for Information and Study on Clinical Research Participation (CISCRP) has shown that patients typically partake in clinical research for both altruistic reasons and with hopes of benefiting their personal health. Other research has also begun to identify what patients want when participating in a trial.

While this is valuable research, the industry hasn’t addressed some important questions: Do patient needs and preferences vary by condition and other patient demographics? Could gaining a better understanding of patients’ lives and the factors that affect their decision to participate in the trial help to create a more attractive patient experience? Is it time to think about making the studies fit the patients’ needs better? What would it mean to move away from viewing patients as subjects in medical research, and engaging them as the stakeholders they are?

Overview of survey and methods

During the summer of 2018, Antidote Technologies and SCORR Marketing partnered on a survey to gain a deeper understanding of patient perceptions of clinical trials and their motivations to participate. The survey also was designed to identify differences related to condition, household income, education, ethnicity, and gender.

Survey participants were recruited to participate by Antidote’s partners: American Kidney Fund, Allergy & Asthma Network, Healthline, JDRF, Lung Cancer Alliance, Lupus Research Alliance, Melanoma Research Alliance, and Multiple Sclerosis Association of America. Each partner organization distributed the survey to its membership through a combination of emails, website posts, and social media posts. Some worked with other partner organizations in their disease area to generate responses from caregivers and patients.

About the sample

Of the nearly 4,000 survey respondents, the majority (89%) identified as a patient. Twenty-seven percent of respondents have multiple sclerosis (MS), 15% have asthma and/or allergies, 13% have kidney disease, 12% have melanoma, 12% have type 1 diabetes, 10% have lupus, 6% have lung cancer, and 5% have gastrointestinal disease. Twenty-six percent of respondents had participated in a clinical trial at the time of data collection.

Most survey respondents (84%) had at least some college or a college degree, and more than half (53%) reported an annual household income of less than $75,000. The sample skewed older (74% of respondents were over the age of 45), white (90% of respondents), and female (80%).

Patient motivators for participating in medical research

Providing prospective patients with the right information to make a decision starts with understanding what drives patients to join a trial in the first place (see Table 1).

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Of the 26% of the respondents who had previously joined a clinical trial:

• 75% reported that the major reason or one of the major reasons they joined the clinical trial was to help future patients.

• 69% said they participated to improve their quality of life.

• 63% indicated they were highly motivated to participate in order to receive the best care possible.

All survey participants rated the importance of 16 potential motivators to take part in a trial. Each motivator was placed in one of the following categories: safety concerns, health benefits, logistical concerns, institutional support, and financial benefits. The percentage of the survey population rating the type of consideration as very important and the average ordinal ranking (where a lower number indicates a higher ranking; see Table 2).

Safety

When we asked respondents about the possible motivators to participate in a clinical trial, unsurprisingly, safety topped the list of priorities. Seventy-three percent said it was very important that the research not interfere with their current treatment or make their conditions worse. This was the chief safety concern expressed. Of the eight condition categories included in the survey, patients with kidney disease assigned the highest importance to the idea that the clinical trial should do no harm.

Institutional support

Having someone available to answer questions throughout the study (66%) and their doctor’s support for their decision to participate (50%) were important to respondents as well. Lung cancer and melanoma patients were most likely to deem it “very important” that their doctor supports their decision to join a clinical trial. Women are more likely to value the importance of having their questions answered throughout the study.

Health benefits

The potential to benefit their personal health was also important to the respondents. About two-thirds (67%) of respondents identified this statement as the top health benefit: “The trial provides me with a drug, therapy, treatment, or medical device that potentially could extend or improve the quality of my life.”

Logistics

Logistics play a role in decision-making as well. Respondents felt it very important that they could complete the entire trial. More than half (59%) of the respondents deemed this as very important and the top logistical concern. Patients with kidney disease, MS, or allergy/asthma place a higher priority on logistical factors than patients with other conditions. Overall, only a minority of respondents said it is very important that a trial doesn’t take time away from their obligations and, similarly, only a minority considered it very important to have clinical researchers make home visits.

Financial benefits

Financial benefits, while very important to some, are generally viewed as less important than other considerations. Oncology and type 1 diabetes patients were the least inclined to be concerned with financial considerations. Less than half (46%) of the respondents said being compensated to participate would be a motivator; however, younger respondents or those with lower household incomes were much more inclined to believe compensation would be a motivator to participate. 

Patients as partners

A key element of patient centricity may relate to the industry’s ability to truly embrace patients as partners. And this effort revolves around medical professionals:

• 90% said that talking with doctors, clinical trial coordinators, and nurses involved in the research would either “very likely” or “likely” help them feel more like a partner in the research.

• Older patients have a stronger preference for talking with not only medical professionals, but also other patients like them or the hospital or company responsible for the project.

However, despite the influential role medical professionals have, only one-third of these respondents have ever talked about research with their doctor.

Similarly, having the necessary information to make well-informed decisions about participation is a key theme that runs through many of the patient responses:

• 77% want the industry to make it easier to learn about clinical trials.

• 70% would like information about findings from clinical trials to be more readily available.

• 66% desire clearer information about the costs that they will incur (time, financial, etc.).

• 56% want healthcare professionals (HCPs) to speak with them about  clinical research before discussing participation in a specific trial.

Patient obstacles

The results of the survey raise important points about clinical research:

• Patients with life-threatening conditions are most concerned about gaining access to treatments.

• Individuals whose condition limits their mobility are concerned about logistical factors.

• People with asthma or allergies-a condition that impacts low-income people more-are concerned about the financial obstacles to participating in medical research.

More important than these findings is the confirmation that there is not a one-size-fits-all solution for engaging individuals in medical research. Rather, it is important to consider the particular obstacles facing patients when they are making decisions about whether or not to partake.

Providing information is a start. While survey respondents showed a clear preference for receiving information about clinical trials from their doctor or other HCP, 49% want to receive information from patients who have previously participated in a clinical trial (especially true for minority patients who haven’t joined a trial before), and almost twice as many preferred to receive information from an advocacy/nonprofit organization or health and wellness website (42%) than from a drug company or advertisement.

In addition to the survey findings, issues like low health literacy and the growing use of the internet for health information point to the need to better engage patient advocacy groups and medical professionals not only in recruiting participants for clinical trials, but also in raising awareness of clinical research as a care option and dispelling common myths that may discourage participation.  

Patient communities and advocacy groups can help bridge the knowledge gap to improve health literacy, give patients access to former clinical trial participants, provide information about clinical trial opportunities, and direct patients to the best places where they already are searching for information-online.

To that end, we also need to be smarter about our methods of communicating. Digital solutions provide one avenue to reach patients, but we need to think about how we leverage digital tools to facilitate two-way conversations. While we understand that interpersonal communication with medical professionals is an essential part of the patient recruitment, screening, and consent process, too often we limit important conversations by failing to use all the tools available, like message boards and online communities.

These avenues are important: Our survey revealed that about three in five respondents use message boards and health-based online communities to learn more about their condition and the experiences of other patients. And about two-thirds of melanoma, gastrointestinal disease, lung cancer, lupus, and type 1 diabetes patients use message boards and health-based online communities to learn more about their condition and the experiences of other patients. This is particularly true among younger digital natives.

Yet, as any behavior change specialist can attest, information is not enough. As an industry, we must also remove barriers to participation by considering the burden placed on participants as the trials are designed. The industry needs to include better and more robust mechanisms for patients to participate at early stages of research planning from protocol design to endpoint selection.

This collaborative approach has been supported by the FDA as well. Under PDUFA V, the agency is conducting disease-specific patient-focused drug development (PFDD) meetings with key stakeholders to obtain patient perspectives on specific diseases and treatments. The FDA also supports externally led PFDD meetings such as the Lupus Patient-Focused Drug Development (PFDD) meeting. These meetings and subsequent reports seek to give patients a voice in what researchers are investigating in addition to how the research is conducted.

Conclusions

At the start of the survey project, we set out to answer the question: How can researchers engage patients in a way that makes them feel like stakeholders, rather than subjects, in research? What we learned is that while different patient populations have different specific desires, the underlying theme is that patients want more information from medical professionals, advocacy groups, and their peers.

The key takeaways from our efforts include:

1. Patients want to be well-informed and empowered consumers of health information.

• Industry has a role to play here, especially with regard to health literacy. Reallocating marketing spend from advertisements to educational materials or grants for health-focused nonprofit organizations may aid in developing the confidence healthcare consumers need to consider clinical trials as a care option.

2. We need a more collaborative planning process that incorporates all stakeholders, especially patients.

• Patients want a say in what new treatments are researched and how they are tested, and they are willing to share their ideas and thoughts through participatory design processes. Having the right trial design for the right patient population may significantly reduce recruitment timelines and get new treatments to market faster.

• There is a benefit to leveraging patient communities and advocacy groups since they are patient-focused and understand the nuances of specific conditions. Our research found what motivates patients to participate in a clinical study varies with condition and other demographic data, and patient organizations have valuable insight into engaging these patient groups. Patient advocacy groups are also expert communicators and can convey information about clinical trials and answer questions and concerns from patients and caregivers when they are seeking that information.

3. Data and technology companies and collaborations are increasingly important, but a human touch is still necessary. 

• The addition of electronic health records (EHRs) to identify sites with potentially eligible patients can further add to the recruitment funnel, but without an understanding of which patients are interested in participating in medical research, these initiatives can fall flat.

• It is important to have mechanisms to quickly engage patients once they express interest in a clinical trial, and our data clearly show that the majority of patients would prefer that follow-up include interpersonal communication.

• Sites should ask patients who have already participated in a clinical trial to share their experiences with other patients considering joining a trial either by arranging phone or online conversations or by helping them create online videos. The ability to speak with or learn from other patients was an important factor with study-naïve respondents.

• Make data sharing and technology easy for patients and sites to use by standardizing processes and improving data sharing. Technology should reduce the burden of on-site staff and trial participants and make it easier for them to communicate and share information.

4. Build lasting relationships with patients for improved retention, repeat study consideration, and continuous process improvement initiatives.

• Treat patients as the research stakeholders they are and establish an ongoing flow of easy-to-understand information about the study progress and results to all participants during and after the trial.

• Utilize surveys and focus groups. Data collected from patient surveys and/or focus groups can inform future protocol design and clinical endpoints selection and facilitate investigator quality management.

• Create long-term relationships with patients before, during, and after the study with ongoing engagement programs that are specific to their preferences and needs.

As an industry, we need to do a better job of two-way communication from the start of trial design through to sharing the results. Patients are stakeholders in the research process with an equally strong interest in seeing new treatment options come to market. Yet, the barriers we present to their ability to engage with the research process in a meaningful way outside of clinical trial participation limit this collaboration and may reinforce feelings of unease and distrust. By improving our mechanisms for listening to and engaging patients at all stages of research design in a way that is meaningful to them, we can finally begin to move the needle on patient centricity and truly understand the power the concept holds.

Lindsey Wahlstrom-Edwards, MPH, CPH, is Head of Partnerships, Antidote Technologies Inc.; Anne-Marie Hess is Senior Strategic Advisor and Director of Market Intelligence, SCORR Marketing