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PCORI funded the Pilot Projects to explore how to conduct and use patient-centered outcomes research in ways that can better serve patients and the healthcare community. Learn more.
Before a patient can join a research study, the researchers doing the study need to get informed consent. Informed consent is a process in which a doctor or researcher explains what the research study is about and what the benefits and risks of joining the study are. The patient then chooses whether or not to join the study.
Getting informed consent can take a long time and a lot of paperwork. Researchers wanted to know if they could use a simpler informed consent process for studies that compare two or more available treatments. For example, a researcher may compare two headache medicines to find out which one works better for people who have severe headaches. Such studies are called comparative effectiveness research studies. They are different from studies in which one of the treatments studied is experimental or one group of patients receives no treatment.
The research team wanted to know how patients, doctors, and researchers felt about three ways to get informed consent in comparative effectiveness research studies:
The research team wanted to find out which informed consent option participants preferred for two types of studies on blood pressure medicine.
Specifically, the researchers wanted to learn from patients, doctors, researchers, and others
The researchers held two day-long meetings with patients. One meeting occurred at Geisinger Health System in Pennsylvania and included 62 patients. The other meeting took place at Johns Hopkins Community Physicians in Maryland and included 75 patients. The researchers randomly called patients and asked them to participate in one of the meetings. All of the patients were age 40 or older. Patients either lived less than 35 miles from Geisinger Health System or got care from Johns Hopkins Community Physicians. The researchers asked different groups of people to participate in the study: men and women, people with and without high blood pressure, and people of different races.
The research team also held a day-long meeting with 58 doctors, representatives from insurance companies, hospital staff, research experts, and patient advocates. This group is called a stakeholder group.
Before each meeting, participants filled out a survey that asked them about their attitudes toward research. It also asked what they thought about the three informed consent options. During the meetings, the research team explained comparative effectiveness research. Then they described two examples of comparative effectiveness research studies. The examples were from a hospital or clinic that regularly did research studies to learn from patients about how to improve health care as quickly as possible. Both examples included an ethics board that reviewed all research studies.
The research team then explained the three ways to get informed consent. Participants talked about the options in small groups. After the meeting, participants filled out another survey asking them how they felt about research and the informed consent options. The research team reviewed the surveys and notes from the meetings to see which informed consent option the participants liked best and why.
At the end of the day-long meetings, patients preferred the Opt In and Opt Out informed consent options. Seventy percent of patients liked Opt In, 65 percent liked Opt Out, and 40 percent liked General Approval.
Patients described the factors they cared about most in informed consent. Ranked from most common to least common, these factors included: choice, information, privacy and confidentiality, the quality of the research, how the study might change the care that patients get, trust, and respect.
The stakeholder group gave different answers. At the end of the day-long meeting, 80 percent of stakeholders liked Opt In, 55 percent liked Opt Out, and only 11 percent liked General Approval.
The research team also found that patients and stakeholders changed how they answered some survey questions after the meeting. At the end of the day, more participants liked General Approval than at the beginning of the day, and fewer liked Opt In than at the beginning of the day.
The study was small. The research team doesn’t know if the results would be the same with patients from other health systems. However, the results were the same in the two health systems the researchers worked with, even though they were in different parts of the country and served patients with somewhat different needs. Also, participants went to only one meeting, so the researchers don’t know whether their opinions about informed consent might stay the same or change over time.
Participants were open to informed consent options that make the process simpler and faster than standard methods. Study participants also said that it’s important for patients to choose whether they participate in research. Because of this, participants would support a simpler informed consent process only if it’s clear that patients are making a choice about whether to participate in a research study.
The research team has presented this work at research meetings and in professional research journals. The research team is developing more papers on the findings of this work.
Informed consent requirements generally necessitate a lengthy process and signed documentation for patients to participate in clinical research. With growing interest in comparative effectiveness research (CER), whereby patients receive approved (nonexperimental) medicines for their medical condition, questions have been raised as to whether the same consent requirements should apply. To advance the debate, more information about the views of informed and engaged patients is needed.
The purpose of this study was to investigate whether patients from two different health systems and other key stakeholders find alternative models of consent, disclosure, and authorization (i.e., opt-in, opt-out, and general approval approaches) acceptable in the context of (a) observational and (b) randomized studies comparing widely used antihypertensive medications.
Researchers conducted three day-long deliberative engagement sessions: two sessions with patients from Johns Hopkins Community Physicians (JHCP) and Geisinger Health System (GHS) and one session with stakeholders, including clinicians, insurers, health system administrators, research oversight experts, and patient advocates.
Participants included 75 patients from JHCP, 62 patients at GHS, and 58 participants from the multi-stakeholder session. For sampling, 500 patients were identified randomly from JHCP, and 1,191 patients were identified randomly from GHS through clinic databases, split evenly by gender, history of hypertension (yes/no), and race (at JHCP only). Patients >40 years old who received care at one specific site (JHCP) or lived within 35 miles of the health system (GHS) were called and invited to attend a deliberative engagement session. For stakeholder sampling, the research team identified seven broad stakeholder categories and invited roughly equal numbers of participants to the deliberative engagement session from existing lists and networks, oversampling for patient advocates.
JHCP is part of a large academic health system in Baltimore, Maryland. GHS is a private, integrated delivery health system in rural Pennsylvania. Multi-stakeholders were invited from across the United States.
The researchers used a day-long, in-person deliberative engagement session to introduce participants to two different CER designs (observational versus randomized) comparing two antihypertensive medications, and three disclosure or consent approaches: opt-in, opt-out, and general approval. The CER designs were described as taking place in a learning healthcare system where participants were informed that their hospital or clinic regularly engaged in research to learn from patients in order to improve health care as quickly as possible, and that a multi-stakeholder ethics board would review all research studies. Sessions consisted of presentations and extensive discussion at small-group tables. Pre- and post-test surveys were completed by participants before and after all-day discussions, measuring attitudes about research and about each of the three disclosure and consent options.
Outcome measures were pre- and post-session attitudes about research and about each of the three disclosure and consent options.
Quantitative data were gathered in the form of a pre- and post-test survey about attitudes toward three different consent and disclosure models for the two CER hypertension case studies. Qualitative data were gathered through small-group discussions moderated by facilitators during the deliberative engagement session to engage participants in conversation about what they heard and understood in the presentations. Facilitators encouraged all participants to share their opinions.
For quantitative data, responses regarding attitudes toward the disclosure and consent model were divided into dichotomous categories, grouping those participants who expressed that they liked a model (“somewhat” or “very much”) versus those participants who did not express liking a model (“neutral” “somewhat” or “really” disliked a model). Chi-square or Fisher’s exact tests were used to analyze survey data and test for differences between patients from the two sites= with respect to demographics, health status, attitudes regarding research, and the patients’ health systems, and attitudes toward the disclosure and consent models. McNemar’s test was used to compare paired pre- and post-test survey responses.
For qualitative data, researchers identified emergent themes from small-group discussions through systematic coding of qualitative transcripts. Members of the research team identified themes in the transcripts and created a standard codebook that was applied to all transcripts by two team members independently. Team members and the study investigators discussed and reconciled any discrepancies to ensure that the codebook was employed consistently.
Attitudes were similar between JHCP and GHS patients. Participants strongly preferred opt-in or opt-out consent options to general approval for both observational and randomized designs. For the randomized CER study, 70 percent of patient participants liked opt-in, 65 percent of patient participants liked opt-out, and 40 percent of patient participants liked general approval after the deliberative engagement session. In discussing disclosure and consent options, patients cared most about choice, information, privacy and confidentiality, quality of the research, trust, respect, and impact of the study on patient care. Multi-stakeholder preferences for consent options for the randomized CER study were different from patients’ preferences: 80 percent of stakeholders liked opt-in, 55 percent liked opt-out, and 11 percent liked general approval.
This pilot study had a limited number of patients who participated in a single deliberative engagement session. Multiple sessions would have allowed the researchers to see if changes in attitudes were sustained over time. It is encouraging that there were no differences in patient attitudes between JHCP and GHS, which were selected because of marked differences in demographic make-up and health care institutional structure. Still, inclusion of patients from other health systems would have increased the robustness of the findings.
The majority of participants from two different types of health systems liked both opt-in and opt-out approaches for observational and randomized designs for low-risk CER. The proportion of participants who liked opt-in and opt-out approaches was the same at post-test. Patients in this study wanted to be told about research and to have a choice about whether to participate, but they were open to such disclosures being streamlined. Findings revealed that major stakeholder groups have strong concerns and preferences about individual autonomy. Researchers found that attitudes changed as a result of the deliberative engagement sessions, where participants had the opportunity to discuss their opinions and engage with other participants. The prioritization of autonomy, despite clear recognition of the value of learning, may be necessary to garner support for streamlined consent processes in a future learning healthcare system in the United States.