Including Patient Preferences to Assess the Balance of Benefits and Harms of Treatment Options for People with Multiple Chronic Conditions

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What was the research about?

Clinical practice guidelines are recommendations for doctors about when and how to treat health problems. Guidelines are often based on research that compares the benefits and harms of different tests or treatments for one health problem. But this research doesn’t always consider that people may have other health problems or different preferences. Guideline developers need to know what is important to patients.

In this study, the research team developed a process to inform development of clinical guidelines. The team wanted to learn how the balance of benefits and harms of treatment options changes when it includes patient preferences. In this new process, the team

• Defined questions comparing treatment options based on input from patients with three or more long-term health problems
• Used data from prior research studies to answer these questions and assess the balance of benefits and harms of treatment options
• Used results from a patient survey, looking to see if the balance of benefits and harms could change when patients have different preferences.

What were the results?

Using their process, the research team identified two research questions that looked at

1. Comparing different blood pressure goals for patients with high blood pressure
2. Comparing two medicines to treat diabetes if the most common medicine, metformin, is not enough

The research team found that the balance of benefits and harms for treatment options changed when looking at how much patients worried about different outcomes. For example, the balance of benefits and harms was about the same for the two medicines to treat diabetes. But this balance changed when taking into account that some people want to avoid shots.

What did the research team do?

To identify research questions, the research team had group discussions with 27 patients and their caregivers who had three or more long-term health problems. Next, the team reviewed data from prior studies related to these research questions and looked at the harms and benefits of treatment options. The team also received survey data from 434 patients with two or more long-term health problems. The surveys asked patients about their preferences, with questions about the importance of benefits and harms relative to one another.

The research team looked at whether the balance of benefits and harms is different for some groups of people, and whether preferences matter.

A total of four patients with long-term health problems and caregivers of people with long-term health problems, or both, were part of the research team. They helped plan and conduct the study. Researchers who create guidelines gave feedback on the usefulness of the study results.

What were the limits of the study?

Studies don’t exist for some treatment harms that were important to patients. The balance of benefits and harms may differ when this information becomes available.

Future research could study treatment harms that are important to patients with more than one long-term health problem.

How can people use the results?

Organizations that create guidelines can consider using the new process to include patient preferences when making guidelines.

Objective

To develop methods that incorporate patient preferences in assessing the benefit-harm balance of treatment options to inform care of people with multiple chronic conditions (MCC).

Study Design

Clinical practice guidelines make recommendations for treatments based on whether the benefits of a treatment outweigh the harms. However, guidelines often do not incorporate patient preferences in this benefit-harm balance.

In this study, researchers adapted a five-step process to understand how the benefit-harm balance for treatment options for patients with MCC can change when incorporating patient preferences for treatment outcomes. Throughout this process, a group of four patient and caregiver investigators provided input to the research team. The steps included

1. Defining research questions. Researchers conducted focus groups and a prioritization process with 27 patients or caregivers of patients with MCC, to identify comparative effectiveness research questions that were important to patients.
2. Data gathering. Based on systematic reviews, researchers selected the most valid and applicable data sources to answer the questions identified in the focus groups. They also reviewed survey data from 434 patients with MCC to understand which potential harms from treatment patients worry most about.
3. Determining the benefit-harm balance of treatment options across patient subgroups. With data from the systematic reviews, researchers used the Gail Index, a measure that uses the expected number of events for different benefits (e.g., avoiding death) and harms (e.g., increased risk of stroke) to estimate the benefit-harm balance of interventions for each research question for different patient subgroups, defined based on demographics, condition, or other characteristics.
4. Assessing how patient preferences and changes in assumptions change benefit-harm balances. Researchers used survey responses to explore whether the benefit-harm balance depends on individual preferences.
5. Dissemination. Researchers presented the results to guideline developers and other medical professionals to see what additional information would be needed to implement the results in guideline development.

Results

From the focus groups, researchers identified two research questions, which included assessing the balance of harms and benefits of

• Different blood pressure targets to treat hypertension in different subgroups of patients
• Treating type 2 diabetes with basal insulin versus sulfonylurea, in addition to metformin, in different subgroups of patients

For the hypertension question, lower blood pressure targets in individuals without a history of prior stroke had a better benefit-harm balance for many patient subgroups, particularly for patients aged 75–84. For almost all patient subgroups, the benefit-harm balance depended on the individual’s preferences, such as worries about kidney injury versus heart attack.

For the diabetes question, the research team found that sulfonylurea and basal insulin had a similar benefit-harm balance among patient subgroups. However, evidence was lacking for many of the outcomes. After accounting for patient preferences, sulfonylurea was better for most patients except for those who do not mind insulin injections.

Limitations

Published literature on some harms that were important to patients was not available. The benefit-harm balance may differ if researchers had this information.

Conclusions and Relevance

The five-step process detected how preference-sensitive benefit-harm balances are and how much they vary across subgroups of patients. Organizations may consider using the process developed in this study when developing clinical practice guidelines.

Future Research Needs

Future research could fill information gaps on outcomes that are important to patients and on approaches to bring estimates of benefit-harm balance to clinical decision making.