Utilizing Community Engagement Studios to Inform Patient Experience in a Multicenter Randomized Control Trial

Abstract

Objective:

To determine whether a community engagement approach can provide feedback for implementation of valuable measures to improve the outcome of a clinical trial.

Methods:

Review of the results from a Community Engagement Studio (CE Studio) for an NIH-funded, multi-institutional study designed to research the efficacy of valganciclovir in the treatment of congenital cytomegalovirus (cCMV)-induced hearing loss. Participants were given information about the trial then asked a series of questions to assess their input on the merits or weaknesses affecting their participation in the trial.

Results:

Thirteen parents whose children have congenital CMV infection were recruited for the CE Studio. The overall theme from the responses was a desire to advance the field but a need to clearly understand the risks and benefits of participation. Many requested more educational resources, more printed materials or greater access to researchers if questions arose. Many welcomed having patient stories and information displayed in a dedicated website or through social media.

Conclusion:

This community engagement approach provided useful feedback from families similar to those expected to be potential enrollees in the CMV ValEAR trial. In response to parental comments, two educational videos were created: one on the general topic of cCMV and the other specific to the CMV ValEAR trial. Researchers who wish to optimize their clinical trial’s success should consider incorporating a CE Studio into their study design.

Introduction

Increasing participation in clinical trials is a challenging, yet necessary component of clinical research^1,2. Having low participant numbers in clinical trials can have detrimental effects by prolonging study duration and increasing costs³. Many efforts are being made to identify important factors in increasing patient participation in trials^1,4,5. One identified factor to improve patient participation and retention is development of a patient support system⁶. Support systems help minimize physician-patient miscommunication by encouraging shared decision making and help in successful adherence to drug protocols through the duration of the study⁶. The role and involvement of patient support systems are both amplified and complicated in pediatric clinical research. Trial participation is significantly lower in pediatric clinical research compared to that in adults⁷. When working with children, it is often left to the parents to decide and provide consent for trial participation on behalf of the children⁸. The practice of consent by proxy increases the threshold for gaining consent in pediatric clinical trials and often leads to physicians and parents deciding to forego trial participation⁹. The amplified role of parents in the decision-making processes demands increased physician-parent communication, further complicating pediatric involvement in clinical trials¹⁰.

These complicating factors require novel approaches in order to overcome barriers to patient involvement in clinical trials. In order to understand the factors that prevent pediatric participation in clinical trials, we adopted a Community Engagement Studio (CE Studio) model that was developed by the Meharry-Vanderbilt Community Engaged Research Core (CERC) to enhance community engagement in research¹¹. CE Studios involve in-person meetings between researchers and a panel of prospective participants to receive project-specific input¹¹. Since these patients have life experience with the target of the study, they are deemed experts¹¹. These experts provide essential information and insights into the target study based on their experience. This first-hand feedback makes CE Studios an excellent model for developing patient support systems and enhancing patient participation, study design and implementation^6,11–13. Panel participants from past CE Studios have expressed an overwhelming positive response to the experience and 98% of the participants indicated willingness in participating again¹¹.

CE Studios may be carried out at any stage of a research project in order to identify important concerns and difficulties that may be hindering the progress of a given study. Our literature search has not demonstrated evidence that CE Studios have been used in the field of Otolaryngology-Head and Neck Surgery. The aim of our study was to determine the utility of CE Studios in Pediatric Otolaryngology clinical research and gain insight from our expert panel about how we could enhance patient experience and participation in a current research study. Our CE Studio was specifically tailored to a double-blinded, NIH-funded study being carried out across thirty institutions titled ValEAR. The objective of the trial is to test the efficacy and safety of valganciclovir in the treatment of congenital cytomegalovirus (CMV) infected patients who are otherwise asymptomatic.

Materials and Methods

Studio Design

The concept of CE Studio has been conceived and described by the Meharry-Vanderbilt Community-Engaged Research Core¹¹. After our CE Studio happened, the same group has recently produced the Community Engagement Studio Toolkit 2.0 which contains guidelines for conducting a CE Studio that approximate the steps contained in previous publications¹⁴. We adapted these steps to conduct a CE Studio with experts representing the CMV community on November 13, 2017.

For our study, the Collaboration & Engagement Team from Primary Children’s hospital recruited parents of children born with CMV and subsequent sensorineural hearing loss. The panel of experts was composed of 6 males and 7 females. Participants were given access to a website and Facebook page with more information on the ValEar trial prior to the night of the CE Studio.

Each of the participants was also provided with a pamphlet outlining the goal and importance of this study, possible drug adverse effects and dosing regimen of either valganciclovir or a placebo administered for 6 months (Figure 1). They were also informed that each child would be monitored for two years. The back page of the pamphlet listed four questions that participants would be asked during the studio meeting. These four questions were: 1) Would you be willing to have your child participate in this clinical trial? Why or why not? 2) What could the research team provide you to make participation more attractive? 3) How can the research team best communicate with parents before, during, and after participation? 4) Please provide feedback on the social media ad and website.

Figure 1:

Informational flyer given to panel participants regarding the study regimen, valganciclovir, and CMV infection

We invited our group of experts to join us for our studio meeting on November 13, 2017. A meal was provided for panel participants prior to the start of the meeting. A brief presentation was given by the primary investigator about CMV and the ValEar trial followed by discussion led by a neutral moderator. Panel experts were asked the above listed questions and their responses were recorded. All community experts were together in one open room during the CE Studio and gave verbal responses to the questions. In total, the event lasted for 90 minutes, and the experts were provided with with contact information should they have other feedback that they would like to give after they had left the meeting.

IRB Approval

This study was approved by the Institutional Review Board at the University of Utah.

Results

Panel responses

Question 1: Would you be willing to have your child participate in this clinical trial? Why or why not?

Many of the panel responses did not involve a direct yes or no answer to the question, but most participants discussed reasons why they would hesitate to participate in the study. The most frequently stated concern involved participant understanding of the risks involved in CMV infection versus the risks with taking valganciclovir:

“Known risks would not cause us to hesitate.”

“Need to have all education about CMV to participate.”

“A couple of years ago, there was nothing to Google about CMV. Most people don’t know what CMV is; it’s not something the general population knows about.”

“Need equal treatment of risks and benefits.”

“Would want to know the success rate for the new drug.”

“List of things science knows and doesn’t know about CMV would be helpful.”

One participant of the panel responded with a direct “yes,” to trial participation: “Yes, gives us the best chance to improve hearing.”

Three of the participants responded negatively to participation in the study, stating that the benefits did not outweigh the risks of taking valganciclovir:

“Talking with other families who have received the treatment gave us the ability to weigh risks and go ahead with CMV.”

“Children with CMV have good outcomes. A child with complete hearing loss can go on to live a full life, rather than risk having a child with infertility issues. A child can live a normal life with hearing loss. It’s not up to me to decide his fertility- that’s too high a risk.”

“No, the risks of the medication are too great to participate in the study (cancer, infertility).”

Other responses to our first question highlighted other potential concerns with patient participation in the study:

“Might not participate because don’t want placebo – want the real treatment; this is a concern since it may stop hearing loss in a child.”

“Expenses covered by the trial are a huge factor when making a decision.”

The vast majority of responses highlight the importance of participant education and the use of clear data when recruiting patients for study participation. The most strongly cited concern voiced by parents who ultimately decided against trial participation was the lack of a clearly stated benefit to risk ratio. Other concerns involved the potential of receiving placebo treatment and additional compensation for trial participation.

Question 2: What could the research team provide to you to make participation more attractive?

As with the previous question, many participants expressed that they would be more attracted to the research if they understood the risk-benefit balance of participating in the study. Some of the participants simply wanted more information, responding with, “[We] need resources.” or “Give us information to look over - risks, education; [We] want to make an informed choice.” Others asked for more time with the physician to aid them in their decision.

Participant responses also referenced specific concerns dealing with the risks and benefits of the trial drug that could be addressed in order to make participation in the study more attractive:

“Provide education about CMV beyond just its impact on ears and hearing.”

“Concrete numbers help, chances [and] percentages on how many might develop cerebral palsy, or complete hearing loss.”

Other responses referenced our flyer that we distributed to the panel participants. One participant suggested that the handout contain more information about the potential of valganciclovir to treat additional symptoms of CMV. Another participant also questioned a statement on our flyer which mentioned the effects of valganciclovir on fertility. All of these responses suggest that prospective trial participants want direct, complete, and clear information prior to making their decision.

One participant addressed the concern that participants may have receiving the placebo in the drug study by helping participants understand CMV infection:

“Let parents know that there are good outcomes for kids with CMV, even with the placebo. Kids can get early intervention, even in loss of hearing, and kids can learn to communicate. Teach parents not to rely on the drug to stop hearing loss. If science cannot fix it, you can still do other things to alleviate symptoms.”

In trying to understand how to make trial participation more attractive to trial candidates, the need for education about the new therapy and the disease itself can be important factors in participant recruitment. While most of the responses highlighted the need for education, others highlighted the need for proper communication of information, and others demonstrated that researchers also need to educate participants about the disease in question.

Question 3: How can the research team best communicate with parents before, during, and after participation?

The panel participants responded with a variety of ideas to improve communication during the different stages of trial participation. Before the study, participants recommended a candid conversation with their physician. As previously stated, many wanted clear statistics and graphics regarding the success of the trial drug and the effects of CMV infection, while others simply requested a list of pros and cons to trial participation. Some of the participants asked for this information to be presented in print for them to look over after their doctor visits. One participant also recommended that all information be presented by their provider.

Participants asked for quick methods to contact their physician during the study. Some suggested a hotline to contact the physician directly, while others requested quick responses via email. One participant suggested holding information sessions for participating families to gather and discuss the trial.

One participant requested some form of follow up after the conclusion of the study.

Question 4: Please provide feedback on the social media ad and website

Participants generally liked the idea of an additional resource for information and a sense of community. One panel member stated, “For future outcomes, it is important to participate in a clinical trial, which helps solve the unknowns; this is appealing to parents.” Participants stated that the “graphics on the website were easier to read” and that they “liked the statistics” on the website. Many participants saw the website as an additional opportunity to provide more information, suggesting that more links and stories be posted to the website for parents to learn more. They also suggested that the website should be properly maintained and contain current information. One participant also gave the interesting suggestion that the website could be used as a recruitment tool by demonstrating the wide-reaching effects of trial participation, suggesting that we “include stories; appeal to people’s altruism.”

Panel responses to this question also uncovered some participants’ feelings about ways that they would prefer that their conditions be phrased. One participant did not like the use of the word “preventable” and “serious” when describing CMV infection. Another participant did not like the use of the phrase “hearing impaired,” instead suggesting that researchers use the words “deaf” and “hard of hearing.”

Overall, the use of the social media ad and website was viewed as a positive tool. The panel’s feedback demonstrated that the web pages can be used as a great tool for education and recruitment, while also informing us about proper communication when discussing conditions and symptoms.

Research Team Actions Based on Expert Responses

To address requests for more information on cCMV, two videos were created: one providing more detailed information about cCMV titled “What is CMV?” and the other about the CMV ValEAR trial titled “What is the ValEAR Trial?”. These videos are included in the CMV ValEAR website and were produced since participants generally liked the idea of an additional resource for information (https://valear.org/videos/). The first video provides data on the incidence of infection, the signs and symptoms when contracting the infection, the nature of viral transmission and the consequences to unborn babies. Consequences of cCMV are presented which include no symptoms, isolated hearing loss to severe disseminated infection. Strategies to prevent transmission are also provided. The second video reiterates the range of signs and symptoms from cCMV and presents the rationale for the ValEAR trial for cCMV infected children with isolated SNHL. The video also provides information about clinical trials, the study versus placebo design, potential benefits and risks associated with the antiviral drug and with being enrolled into the study. A link to the videos is listed below: https://vimeopro.com/learngenetics/cmv-valear-trial-videos

As of February 26, 2020, 39 people have viewed our video, “What is CMV?” and 16 people have viewed the video, “What is the ValEAR Trial?” Across all institutions, we have successfully enrolled 5 patients in the trial at a rate of .9 enrollees per month with a target sample of 52 by July 2022. One important aspect of our recruited patients is that we have none have dropped from the study, a common detriment to clinical trials. One study demonstrated an attrition rate of 32% at extended in randomized control trials for pediatric chronic conditions¹⁵.

Discussion

From the studio panel, parents gave valuable input regarding the ValEar trial to enhance patient experience and participation. A primary concern from most parents was the lack of clear information on cCMV and on valganciclovir in the provided pamphlet. Parents expressed that clearly illustrated facts and statistics for cCMV and valganciclovir would not only help them make an informed decision, but also make trial participation more attractive. Our panel also suggested that more information can be added regarding not treating cCMV to help them understand the risks of foregoing treatment and to overcome fears of receiving placebo treatment. These recommendations and concerns are consistent with previous CE Studios that have been conducted to understand barriers to trial participation for patients. For example, in a study conducted on long-term effects of opioid analgesics, panel participants recommended that researchers provide more education to providers and patients on the topic¹¹. In another study on biomarker for early lung cancer detection, panel participants also recommended giving potential participants more information on the implications of the study including side effects and risks¹¹.

These combined results demonstrate the importance of clearly stated facts and appropriate statistics to make trial participation more appealing to candidates.

Communication throughout the trial is another important factor to improve the patient experience, whether directly with the provider or indirectly through social media. Some parents requested easy access to the primary investigator. Other parents requested clearer printed materials or an easier to read website. Similar to our discussion about clearly explaining facts, previous CE Studios have received feedback to alter recruitment materials to include more relevant images, cultural sensitivity, and layman wordings¹¹. Each of these results further demonstrates the utility of CE Studios to gain important feedback about stakeholder concerns in order to enhance participant experience and recruitment.

Our studio also revealed that parents preferred to have quicker access to and more engagement with the primary investigator. Convenient access to a physician via telephone has been identified as a key contributor to patients participating in shared decision making, which could potentially help in patient recruitment and adherence to study protocols¹⁶. Our research team did not take any specific steps to address this feedback, although methods allowing physician access is worth investigating.

Since our CE Studio was completed at the beginning of our trial, it is difficult to compare the impact of the meeting on patient recruitment. As demonstrated by the number of views, our videos have been a useful source of information for prospective and current trial participants. Future analysis is warranted to determine whether our improved educational videos contributed to an overall higher retention rate of study participants.

Conclusion

Community Engagement Studios are a valuable tool for understanding parent concerns and hesitations regarding enrollment of their child in clinical research. Our CE Studio specifically revealed that parents wanted clear information about the effects of untreated CMV and the short- and long-term risks of valganciclovir use. In order to address these concerns, we created two informational videos to educate parents about CMV and participation in clinical trials. These videos have provided a convenient space for parents to understand our trial in order to make informed decisions about trial participation. The results of our study indicate that CE Studios can inform specific steps that the research team can take in order to improve patient experience in clinical trials.