Rethinking Patient Engagement: Mild vs. Severe Illness

Clinical research professionals have long held the view that patients with severe medical conditions have very different attitudes and perceptions about clinical research than do those facing milder conditions. Individuals managing severe and life-threatening conditions for which there are few if any treatment options, professionals believe are more desperate and willing to gamble with their health to gain access to investigational interventions. 

By extension, many research professionals have noted that patients with severe medical conditions are more motivated to gather information, to be more informed about their treatment options and more familiar with the clinical research enterprise.

The results of a recent 2015 study completed by the Center for Information and Study on Clinical Research Participation (CISCRP) suggests that the differences in attitudes and perceptions are far more nuanced between patients facing mild and moderate disease conditions and those facing severe conditions.

Among patients with self-reported severe and mild disease conditions, CISCRP’s 2015 Perceptions & Insights study results found:

• No significant difference was observed in the level of importance that they attribute to clinical research.

• Nearly the same proportion feels they are very informed about clinical research.

• An equal percentage considers clinical research to be a treatment option.

• A relatively low proportion in both groups is very confident that they can find an appropriate clinical research study.

• No significant difference was observed in their willingness to participate in a clinical research study.

 The study also found that among patients with severe medical conditions:

• A significantly higher percentage would prefer to learn about potential clinical research studies from their physician.

• A higher proportion said that they would talk to their physician and the research center staff prior to choosing to participate in a clinical research study.

• Although a much higher proportion report that they chose to participate in a clinical trial to gain access to an investigational treatment, a significantly higher percentage also indicated that they chose to participate for altruistic reasons.

2015 P&I study methods

CISCRP conducted the most recent Perceptions and Insights study online between April and July 2015 among a global community of patients and the public. The questionnaire was developed with input from a variety of stakeholders, including representatives from patient groups, pharmaceutical and biotechnology companies, contract research organizations (CROs), and investigative sites. An ethical review committee reviewed the final survey instrument.

CISCRP collaborated with a number of organizations, including Acurian, Clariness, Quintiles, and PMG Research, for help in reaching and engaging survey respondents. Acurian, for example, maintains a large proprietary database of people who have explicitly opted-in-via online and offline consumer health surveys-to receive healthcare information on specific diseases and clinical trial notifications.

A total of 12,009 international respondents completed the survey with the highest concentration (63% or n=6,665) based in North America; 15% (n= 2,618) based in Europe, and 12% (n=1,302) in Asia-Pacific. To our knowledge, this is the largest global assessment of public and patient attitudes and perceptions about clinical research ever conducted.

 Slightly more than half (54%) of respondents are female. Approximately one out of four respondents had participated in a clinical trial prior to completing the online survey. A similar proportion of respondents (n=2,811) reported that they suffered from a mild-to-severe illness, with 44% rating their condition “Very Mild” and “Mild” and 20% rating their condition “Severe” and “Very Severe.”

Shared views and perceptions

Patients share comparable general views and perceptions about clinical research regardless of the severity of their medical condition. Half of patients with severe conditions and half of those with mild conditions, for example, rate themselves as “Very Informed” about clinical research.  Approximately two-thirds of patients with mild conditions and 70% of those with severe conditions believe that they understand what is meant by the term “Clinical Research.” Nearly 90% of patients in both groups consider clinical research “Very Important” to the discovery of new medical treatments.

Although we expected to see a higher proportion of patients with severe and life-threatening conditions who consider clinical research as a treatment option, less than one-third (29%) do so. Almost the same percentage (28%) was observed among patients with mild conditions.

No significant differences were observed in one’s confidence level in finding, and willingness to participate in, a clinical research study. Forty-five percent of patients in both the severe and mild medical condition groups feel “Very Confident” in finding an appropriate clinical research study. And 73% of patients with severe and 65% with mild medical conditions say that they are “Very Willing” to participate.

Divergent areas

Attitudes and perceptions significantly diverge when looking at the role of the healthcare provider in influencing one’s decision to participate. And patients with severe medical conditions display higher relative motivation to not only gain access to investigational treatments but also to contribute to advancing science and to helping others who may be suffering.

Nearly 60% of patients with severe medical conditions say that would prefer to learn about clinical research studies from their primary and specialty care physicians. This compares with 45% of patients with mild conditions. No significant difference was observed, however, in the percentage of each group who actually learned about a clinical research study from their healthcare provider. Less than one-third (30%) of patients in each group reported that their primary and specialty care provider was the main source for information about clinical research studies. No significant differences were observed between the two cohorts across a wide variety of actual sources used to obtain clinical research study information, including printed and digital material, mass media, social media and health professionals.

A higher percentage of patients with severe illnesses (75%) say that they would speak with their physicians and the study staff prior to choosing to participate in a clinical research study. This compares with six out of 10 (61%) patients with mild conditions who would do so.

More than half of patients with severe medical conditions (52%) said that they chose to participate in a clinical trial to gain access to an investigational treatment, while 57% said it was to advance medical science and 50% attributed the reason to helping other patients who may be suffering from illness. These results are significantly higher than the 28%, 42%, and 35% respectively, observed among patients with mild medical conditions.

Common and unique opportunities

The high level of perceived importance of, and the high willingness to participate in, clinical research among patients with mild and severe medical conditions suggests a strong common foundation from which to build engagement. However, the low percentage of patients with severe medical conditions that consider clinical research as a treatment option and that are confident in finding relevant clinical studies-and the fact that these proportions are similar to those observed among patients with mild conditions-is particularly acute.

With 20 of the top 25 most active areas in drug development today targeting severe and life-threatening illnesses (e.g., breast, colorectal, non-small cell lung and pancreatic cancer), it is critical that pharmaceutical and biotechnology companies and their research collaborators develop and consistently execute more customized patient engagement strategies and tactics to increase awareness and higher levels of clinical research literacy among this subgroup. These strategies and tactics might include more effective use of communication technologies and online and live community forums.

But the results of the 2015 Perceptions and Insights study also point to healthcare providers as a particularly important and missed partner in engaging prospective study volunteers with severe medical conditions.   There are many anecdotal examples of highly engaged patient communities (e.g., Duchenne muscular dystrophy, cystic fibrosis, Parkinson’s disease) who would not have reached this level of engagement without the involvement of the healthcare provider community. Key to this involvement is working closely with patients, their families, caregivers, and the healthcare community during the early stages of development planning to understand and reach consensus around unmet medical need, the research agenda, and meaningful endpoints. Another key is offering higher levels of transparency-in the amount and frequency of R&D information before and after clinical trials are conducted-to inform patient and healthcare professional communities and to establish trust and credibility.

The opportunity to engage the healthcare provider community fits well within the broader scope of the patient-centric drug development movement-the ultimate goal of which is to engender a sense of ownership and a vested interest in the success of clinical research activity among patients and the healthcare provider community. The historical practice of advertising and promoting industry-centric clinical trials to draw volunteers into the research enterprise must be replaced by the new practice of bringing patient-centric clinical trials to the patients and professionals who requested them.

Kenneth A. Getz, MBA, is the Director of Sponsored Research at the Tufts CSDD and Chairman of CISCRP, both in Boston, MA, e-mail: kenneth.getz@tufts.edu