Patient Preferences Regarding Informed Consent Models for Participation in a Learning Health Care System for Oncology

PURPOSE:

The expansion of learning health care systems (LHSs) promises to bolster research and quality improvement endeavors. Stewards of patient data have a duty to respect the preferences of the patients from whom, and for whom, these data are being collected and consolidated.

METHODS:

We conducted democratic deliberations with a diverse sample of 217 patients treated at 4 sites to assess views about LHSs, using the example of CancerLinQ, a real-world LHS, to stimulate discussion. In small group discussions, participants deliberated about different policies for how to provide information and to seek consent regarding the inclusion of patient data. These discussions were recorded, transcribed, and de-identified for thematic analysis.

RESULTS:

Of participants, 67% were female, 61% were non-Hispanic Whites, and the mean age was 60 years. Patients' opinions about sharing their data illuminated 2 spectra: trust/distrust and individualism/collectivism. Positions on these spectra influenced the weight placed on 3 priorities: promoting societal altruism, ensuring respect for persons, and protecting themselves. In turn, consideration of these priorities seemed to inform preferences regarding patient choices and system transparency. Most advocated for a policy whereby patients would receive notification and have the opportunity to opt out of including their medical records in the LHS. Participants reasoned that such a policy would balance personal protections and societal welfare.

CONCLUSION:

System transparency and patient choice are vital if patients are to feel respected and to trust LHS endeavors. Those responsible for LHS implementation should ensure that all patients receive an explanation of their options, together with standardized, understandable, comprehensive materials.

INTRODUCTION

Health care professionals anticipate that learning health care systems (LHSs), wherein routinely collected patient data are compiled for secondary uses, will be valuable assets in medical research and quality improvement.¹ The convergence of these systems with advances in precision medicine,² big data analytic techniques,^3,4 genetic testing,⁵ and other novel technologies stimulates interest regarding the promise of such systems.

However, the patient voice must not be lost in this well-intentioned, rapidly expanding approach. All stewards of patient data have an ethical and professional duty to respect the views of the patients from whom, and for whom, these data are being collected,^6-8 and to design the LHS in a manner that will ensure its security, stability, and medical usefulness.^9-11 Both efforts require that patients be included as partners in this mutually beneficial endeavor, rather than as unaware or unwilling subjects.

Although prior studies have investigated patient opinions about this matter, most have been limited to surveys, interviews, or focus groups,^6-8 which may not allow for adequate information and reflection on complexities. Democratic deliberation methods engage citizens in dialogue and solicit their informed and considered opinions through education by experts and facilitated group discussion.^12-14 Therefore, we conducted democratic deliberations with patients to assess their views about CancerLinQ,^15,16 a real-world LHS developed by ASCO.

METHODS

We conducted a qualitative study of discussions during democratic deliberation sessions involving 217 patients with cancer that focused on ethical considerations of LHSs. Four deliberation sessions were held between June 2017 and May 2018. This qualitative study was part of a larger research project, and the full methods are reported elsewhere.¹⁷ This research was determined to be exempt by the University of Michigan Institutional Review Board.

In the 2 main sessions at each deliberation, expert presentations were followed by discussions (recorded and transcribed) at tables of 4 to 8 participants. The first considered “Disclosure and Consent.” The second, which was about potential users and uses of data, will be the topic of a future article.

Experts provided an overview of LHS goals and benefits, ethical and practical considerations, and policy options for information and consent regarding the inclusion of patients’ health data. The policy options were as follows: opt-in, opt-out, notification with no option to opt out, and no notification (see Appendix Fig A1, online only, for policy descriptions). Before and after the discussions, participants voted for the policy that they thought should be implemented. Presenters and facilitators emphasized that participants should explain the reasoning behind their votes and think like policy makers to determine which policy they believed would be best for society.

Three analysts (R.D.J., C.K., and M.G.) developed a coding scheme after reading transcripts and conferring about initial impressions. Each transcript was independently coded by 2 analysts, who then met to reconcile any discrepancies (with the third serving as tie breaker when necessary). If warranted, updates to the coding scheme were made in response to new insights generated during this process. Coding was conducted using Dedoose version 8.0.35. After coding was finalized, the analysts, together with the principal investigator (R.J.), collated the codes into potential themes using thematic analysis.¹⁸

RESULTS

Participant characteristics are summarized in Table 1; 67% were female, 61% were non-Hispanic Whites, and the mean age was 60 years.

TABLE 1.

Participant Characteristics (N = 217)

Patients’ opinions varied along 3 dimensions: their positions on data sharing, their priorities for implementation, and their policy preferences. Patients' positions about sharing data fell along 2 spectra: (1a) trust/distrust and (1b) individualism/collectivism. Their positions on these spectra seemed to influence the level of weight put on 3 priorities they identified as important for ensuring ethical LHS implementation: (2a) promoting societal altruism, (2b) ensuring respect for persons, and (2c) protecting patients. In turn, consideration of these priorities seemed to inform their preferences regarding the policies and procedures that should constitute LHS governance of the disclosure and consent process. Patients commonly discussed (3a) patient choices and (3b) system transparency when voicing their preferences. A thematic map is displayed in Fig 1 (Table 2 offers exemplar quotes).

Fig 1.

Thematic map of patients’ positions, priorities, and policy preferences. Patients' opinions about sharing their data illuminated their positions along 2 spectra: trust/distrust and individualism/collectivism. Positions on the spectra seemed to influence the level of weight put on 3 priorities identified as important for ensuring ethical learning health care system (LHS) implementation: promoting societal altruism, ensuring respect for persons, and protecting patients. Consideration of priorities seemed to inform preferences regarding policies and procedures that should constitute LHS governance of the disclosure and consent process. Patients commonly discussed patient choices and system transparency when voicing their preferences.

TABLE 2.

Major Themes and Exemplar Quotes

Theme 1A: Patients' Positions: Trust/Distrust

A primary theme reflected the varying levels of trust that patients have in different entities that might have interest in LHSs. Generally speaking, patients trusted medical professionals and distrusted both insurance and pharmaceutical companies. The main reasons articulated related to personal connections with physicians and suspicions of companies, which were perceived to be driven by profit.

In addition, participants expressed varying levels of trust for LHS security measures, both with respect to protocols for determining who is allowed access, as well as concerns about external threats such as hackers.

Theme 1B: Patients' Positions: Individualism/Collectivism

Participants also positioned themselves along a continuum from individualism to collectivism. Those with a more individualistic mindset emphasized the importance of individual control over their data; others with a more collectivist disposition focused more on their obligation to contribute to, or at least not detract from, the common good.

The intersection of trust/distrust and individualism/collectivism seemed to influence how participants weighted the patient priorities further described in the following sections: (1) societal altruism, (2) respect for persons, and (3) protection of patients. At 1 end of the spectrum, participants who voiced a relatively high degree of trust and a collectivist mindset tended to focus more on the altruistic benefits to society and less on the need for personal protections. At the other end of the spectrum, those whose comments seemed more distrustful of the system or certain potential users of the data and whose remarks displayed a more individualistic point of view generally had the greatest concerns about privacy and harms that might arise from their data being released. Other patients fell between these 2 poles.

Theme 2A: Patients' Priorities: Societal Altruism

Patients who prioritized societal benefit expressed 3 interconnected motivations for sharing their data: a desire to help other patients, a sense of reciprocity or “paying it forward,” and a desire to contribute to collective knowledge. Most participants expressed a desire to help others; some participants, such as those quoted in Table 2, placed a greater emphasis on societal benefits than on potential harms.

Theme 2B: Patients' Priorities: Respect for Persons

A second priority that patients discussed was respect for persons, which manifested in 2 forms. Comments associated with the concept of human dignity had to do with respecting people as human beings with innate worth (sometimes described as God-given worth). Discussion of human rights involved a sense of fundamental rights that entitle personal control over one’s data (sometimes described as one’s property) and the freedom to choose. Violation of these rights was generally perceived as obtrusive and unjust. Participants who emphasized the importance of respect were concerned primarily with preserving some amount of control.

Theme 2C: Patients' Priorities: Protection of Patients

Many participants were also concerned with protecting themselves and their fellow patients from direct or indirect harms. They expressed a desire for protection from a variety of harms that could result from LHS data being used in certain ways or being leaked (see Table 2 for exemplar quotes). These potential harms included:

• Stigma: negative treatment or damage to one’s reputation because of the disclosure of certain medical conditions or other sensitive information.

• Financial hardship (eg, insurance companies denying coverage).

• Exploitation: pharmaceutical companies profiting from patients’ data, or price gouging.

• Violations of personal space (eg, when marketers contact individuals without permission, perceived as unwanted intrusion or harassment).

• Threats to personal identity: identity theft, especially if patients’ social security numbers could be linked to their data.

• Unknown risks: recognition of the fact that this is largely uncharted territory and, as such, new harms may emerge that we cannot foresee.

Most participants expressed some level of concern about these issues; some patients, such as those quoted in Table 2, placed a greater emphasis on potential harms than on potential benefits. How patients balanced the competing priorities of societal altruism versus respect for persons and protection of patients informed their preferences for LHS governance, specifically the levels of patient choice and system transparency that should be incorporated into the disclosure and consent process.

Theme 3A: Patients’ Preferences: Patient Choices

Participants’ desire for patients to have control over their own data was informed by 2 areas of concern:

• Extent of participation (whether to allow data sharing and for how long).

• Sensitivity and use (the type of information shared and with whom).

Many participants felt it was important to have choices in these areas to ensure respect for persons and protection of patients. Nevertheless, some were willing to forgo all or at least some choices if they perceived it would benefit society as a whole. Although preferences varied, most participants wanted at least some degree of control over their data.

Theme 3B: Patients’ Preferences: System Transparency

Another major preference for LHS governance related to transparency during the disclosure and consent process, specifically with respect to:

• Notification: whether patients are told that their data will be released to an LHS (which includes whether patients are aware that they are being told, because some types of notification, such as e-mail, may be overlooked).

• Information: whether patients are given sufficient information to make decisions about their data, such as the nature/sensitivity of the data and the potential users and uses of that data.

• Comprehension and retention: whether the information is presented in such a way that patients will be able to understand and remember what they are told (which may be challenging for patients with cancer who are already dealing with a great deal of information and emotions regarding their diagnosis and treatment).

Finding a Balance

The results from the final vote (taken after the group discussion) were as follows: 38 (17.5%) for policy 1 (“opt-in”), 119 (54.8%) for policy 2 (“opt-out”), 28 (12.9%) for policy 3 (“notification with no opt-out”), 31 (14.3%) for policy 4 (“no notification”), and 1 (0.5%) did not vote. Ultimately, patients’ preferences regarding system transparency and patient choices seemed to inform the disclosure and consent policies they deemed best. In general, participants who wanted greater transparency and control to protect themselves and other patients from potential harms were more likely to vote for policy 1 (opt-in) or policy 2 (opt-out). Notably, a few who expressed a desire for options to choose what happens with their data also emphasized that they might still allow their own personal data to be released, but they wanted the ability to make these decisions for themselves.

Some participants emphasized potential societal benefits and less desire for system transparency or patient choices; they wanted to maximize the data contributed and minimize the administrative costs associated with monitoring and managing patients’ individual preferences. In these cases, they generally tended to vote for either policy 3 (notification with no opt-out) or policy 4 (no notification).

When comparing those voting for policy 1 (opt-in) with all others, demographics for the voters were similar by age, sex, and education. However, Hispanic voters were significantly (P = .003) more likely to vote for policy 1 (7 of 13 [53.8%]) than were non-Hispanics (31 of 193 [16.1%]; 11 respondents did not provide information at his/her Hispanic ethnicity). Nonwhites voted for policy 1 (17 of 69 [24.6%]) more often than did whites (21 of 142 [14.2%]; 6 respondents did not provide information at his/her Hispanic ethnicity), although not significantly so (P = .089).

Although participants’ preferred policies varied, a majority voted for policy 2 (opt-out). Overall, many thought this struck a reasonable balance between 2 extremes. Of note, the mental process of balancing factors when considering the appropriateness of each policy option was a concept that was explicitly discussed by a number of participants, that is:

We're talking about what might be best for society as a whole versus the individual. It needs to be balanced. If it was society as a whole, we would always give all our medical information all the time. Completely. On an individual basis though, there may be some information we may not want to present to others for various reasons.…There has to be a balance between the two [opt-out policy, White, non-Hispanic, more than high school].

Quality of Deliberation

Part of the value of democratic deliberation is the opportunity for participants, much like policymakers, to consider various viewpoints and to explore the reasoning behind their opinions. The typical deliberation in this study exemplified a breadth of perspectives and quality of discourse.

When educated on the topic and given the opportunity to engage other patients in dialogue, most participants exhibited a willingness to discuss complex and controversial issues. Many participants changed their policy votes after considering others’ perspectives. A separate article presents quantitative analyses of patient perspectives.¹⁷

DISCUSSION

Our deliberations offer several insights that are important to inform the design of CancerLinQ and similar systems. Although prior research has investigated patient preferences using standard survey, interview, and focus group techniques,^6-8,19-22 concluding that many desire to be asked permission before secondary use of their clinical data, this study is innovative in its approach. To our knowledge, it is the first to engage patients with cancer in ethical discussions that are based on a real-world LHS while using democratic deliberation specifically to develop their informed and considered judgments. Democratic deliberation intentionally seeks to generate proxies for understanding what other patients would conclude, and the current report analyzing their reasoning is particularly important to inform policy.

One key finding was that participants’ positions on the spectra of trust/distrust and individualism/collectivism provided a foundation on which they weighed their priorities and built their policy preferences. Although it may be difficult to affect participants’ degree of individualism/collectivism, their level of trust in the system and those who use it has the potential to be either cultivated or degraded. In previous survey and interview studies, patients who trusted the health care system were more likely to be comfortable with the use of LHS data, and doctors tended to be perceived as more trustworthy users of data than insurers or the pharmaceutical industry^.6,7 During prior democratic deliberations, patients with high levels of trust tended to be more accepting of a less painstaking informed consent process to allow researchers access to their medical records.²³ Similarly, the findings of our study suggest that patients’ trust in the medical community might be leveraged to explain the purpose and value of such systems while mitigating concerns about profit-driven companies. If patients’ priorities and policy preferences are downstream of their level of trust, significant investments in cultivating that trust should be established early in the process. Clearly articulating to patients the security measures that are in place, as well as who will have access to LHS data and why, may alleviate unease about data breaches and bad actors.

Our findings also indicate that many participants are apprehensive about how their data may be used in an LHS. The potential harms they identified range from breaches of security to unknown risks that cannot yet be identified. These concerns are in tension with the competing motivation of helping fellow patients. Other researchers have similarly observed that patients weigh the altruistic benefits of sharing their de-identified data against the possible risks.²⁴ Thus, in addition to building trust, making societal benefits apparent to activate patients’ altruistic motivations may offset some concerns about potential harms. If patients consider the meaningful benefits to future patients like themselves, they may be more open to sharing their data and supporting LHS endeavors. Furthermore, identifying how data gathered thus far has benefited them as individuals could activate a sense of reciprocity and duty to future patients, as advocated by theorists.²⁵

When educated and given a chance to reflect, most participants favored an opt-out model, which is the model currently used by CancerLinQ.²⁶ Encouragingly, some who advocated for even opt-in consent indicated they would allow their data to be released but wanted to control that decision. Given that a nontrivial proportion desired opt-in consent, and evidence that this was most common in minority groups, the optimal format of consent remains unclear. What this study convincingly demonstrates is that some degree of consent (at least opt-out) is necessary to demonstrate respect for patients; this preference held even after extensive deliberation, suggesting that systems of notification alone or those that allow secondary data use without notification are not advisable in this context, even if legally permissible.¹⁰

One limitation of this study is that the time and resource-intensive nature of democratic deliberation limits the number of sites and participants, which can affect generalizability. It is possible that our sample was biased by a selection effect, as participation required attendance for a full Saturday. Patients who were too sick, busy, or uninterested in research might not have made this commitment, and the demographics of participants do not perfectly overlap those of patients with cancer in the United States, suggesting that our sample may have differed from those whom they were intended to represent. In addition, with deliberative approaches, presentation content or discussion structure may affect the reproducibility of findings. We attempted to minimize bias by engaging and training facilitators experienced in qualitative methods and developing study materials by incorporating diverse perspectives, including those of practicing clinicians, professional society staff, ethicists, and patients. Ultimately, our participants’ intensive discussions conform with the 4 dimensions of high-quality deliberation: (1) equal participation, (2) respect for others’ opinions, (3) adhering to a societal perspective (rather than focusing on what is best for the individual), and (4) thoughtful reasoning and justification of one’s ideas.²⁷ Finally, we focused participants specifically on CancerLinQ because of the usefulness of a concrete example. However, CancerLinQ’s current reliance on an opt-out approach might have influenced participants’ opinions; future research could engage patients in deliberation after focusing on an alternative example, with a different notification and consent approach.

Given that federal regulations²⁸ are subject to confusion and debate among experts,²⁹ the considered perspectives of patients generated in the current study merit attention. As legislators consider policies to protect individuals’ rights with respect to collection of their personal information, including health data,³⁰ understanding patients’ priorities and reasoning becomes particularly important. Our findings suggest that if system transparency and patient choice are established, patients will be more likely to feel respected and to trust and support LHS endeavors. Policy makers and system administrators must ensure that all patients receive an explanation of their options, together with standardized materials that are understandable and comprehensive. This is essential to minimize disillusionment with the system while appropriately encouraging patient contributions.

Appendix

Fig A1.

Policy options for information and consent regarding the inclusion of patients’ health data.

AUTHOR CONTRIBUTIONS

Conception and design: Kent A. Griffith, Rebecca Spence, Angela R. Bradbury, Raymond De Vries, Rodney A. Hayward, Richard L. Schilsky, Reshma Jagsi

Financial support: Reshma Jagsi

Administrative support: Reshma Jagsi

Provision of study material or patients: Robin Zon, Sage Bolte, Reshma Jagsi

Collection and assembly of data: Rochelle D. Jones, Chris Krenz, Rebecca Spence, Raymond De Vries, Robin Zon, Navid Sadeghi, Reshma Jagsi

Data analysis and interpretation: Rochelle D. Jones, Chris Krenz, Michele Gornick, Kent A. Griffith, Rebecca Spence, Angela R. Bradbury, Raymond De Vries, Sarah T. Hawley, Rodney A. Hayward, Sage Bolte, Reshma Jagsi

Manuscript writing: All authors

Final approval of manuscript: All authors

Accountable for all aspects of the work: All authors

AUTHORS' DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST

Patient Preferences Regarding Informed Consent Models for Participation in a Learning Health Care System for Oncology

The following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO's conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/op/authors/author-center.

Open Payments is a public database containing information reported by companies about payments made to US-licensed physicians (Open Payments).

Angela R. Bradbury

Consulting or Advisory Role: AstraZeneca, Merck

Rodney A. Hayward

Honoraria: Up-To-Date

Robin Zon

Stock and Other Ownership Interests: AC3

Consulting or Advisory Role: MedPro Specialty Advisory Board

Richard L. Schilsky

Research Funding: AstraZeneca (Inst), Bayer (Inst), Bristol-Myers Squibb (Inst), Genentech/Roche (Inst), Eli Lilly (Inst), Merck (Inst), Pfizer (Inst), Boehringer Ingelheim (Inst)

Travel, Accommodations, Expenses: Varian

Open Payments Link: https://openpaymentsdata.cms.gov/physician/1138818/summary

Reshma Jagsi

Employment: University of Michigan

Stock and Other Ownership Interests: Equity Quotient

Consulting or Advisory Role: Amgen, Vizient

Research Funding: AbbVie (Inst)

Travel, Accommodations, Expenses: Amgen

Other Relationship: JAMA Oncology Editorial Board

Open Payments Link: https://openpaymentsdata.cms.gov/physician/373670/summary

No other potential conflicts of interest were reported.