It Is Time to Ask Patients What Outcomes Are Important to Them

ABSTRACT

Objectives: To identify the outcomes desired by patients (and their family members) with abdominal or back pain and to compare patient and physician opinions regarding the importance of each outcome.

Study Design: Mixed methods.

Methods: After identifying 21 potentially important outcomes from the lit­erature and telephone interviews with patients and family members, we asked 40 patients, 11 family members, and 11 primary care physicians in telephone interviews to rate the importance of each outcome to patients on a scale of 1 to 5 scale (5 = most important), stratified by pain location.

Results: Mean patient ratings of the 21 outcomes ranged from 3.3 to 5, with the average rating across all items higher for patients with back pain than those with abdominal pain (4.50 vs 4.09; P = .049). Physicians rated the importance of these outcomes to patients significantly lower than the patients did for both abdominal pain (4.1 vs 3.5; P = .04) and back pain (4.5 vs 3.6; P = .0003). Family member ratings were similar to those of the patients (4.3 vs 4.2; P = .8), where­as physicians rated the importance to patients to be an average of 0.6 points lower than the ratings of patients for abdominal pain and 0.8 points lower for back pain.

Conclusions: Many outcomes are important to patients and their family mem­bers, but they mostly represent quality-of-life events rather than the symptom and function measures heretofore focused on by researchers. Physicians appear to rate most of these outcomes somewhat lower in importance.

Patient outcomes and their measurement for comparative effectiveness research, performance measurement, and patient care are increasingly important. Early measures of patient outcomes were usually selected from the perspective of a clinician, but there was a shift to a more patient-centric perspec­tive 15 years ago as measures of health status and quality of life.¹ By 2010, patient outcomes had attained such priority nationwide that the Patient-Centered Outcomes Research Institute (PCORI) was established to emphasize incorporating patient viewpoints and outcomes in every aspect of healthcare research.²

This large shift has required the development of patient-re­ported outcomes measures (PROMs). Subsequently, the National Institutes of Health established the Patient-Reported Outcomes Measurement Information System (PROMIS) in 2004 with the goal of “providing clinicians and researchers access to efficient, precise, valid, and responsive…measures of health and well-be­ing.”³ Although this system was primarily created for clinical re­search, it is rapidly becoming the standard source of PROMs for all purposes. The PROMIS Assessment Center currently contains measure sets that are publicly available in 19 domains for assessing the physical, mental, and social health of both adults and children.

However, the PROMIS tools largely measure specific patient functions and symptoms and seem to assume that such specific measures are the only outcomes that matter to patients and their families. The PROMIS website does not describe any involve­ment of patients in the selection of its outcome measures, and no publications describe attempts to learn from patients what outcomes they care most about. Although patients probably do care about relief from symptoms and improvement in function, there may also be other outcomes that are equal or more import­ant to them.

Therefore, we developed this study to discover what outcomes patients and their family members cared about among those who had experienced back or abdominal pain problems that were se­rious enough to require advanced diagnostic imaging studies. We focused on patients with actual experiences, thinking they should be most aware of outcomes important to them. After identifying potentially important outcomes through preliminary open-ended interviews, we surveyed patients, their family members, and pri­mary care physicians to learn their rating of the importance of each outcome and then compared these ratings among patients, family members, and physicians.

METHODS

Setting

We conducted this study among the patients receiving care from an 800-physician multi-specialty medical group in the Minneap­olis-St. Paul metropolitan area. In order to facilitate access to health plan claims data for these patients for a later phase of the study, patient recruitment was limited to the 60% with insurance from the affiliated health plan; this population includes patients on prepaid medical assistance and a racial and ethnic profile sim­ilar to that of the Twin Cities metropolitan area.

Pilot Interviews

In order to create a list of potentially important patient outcomes, we randomly identified adults who had undergone either a mag­netic resonance imaging (MRI) or computed tomography (CT) scan for abdominal or back pain about 1 year prior. We first sent these individuals a letter explaining the study and providing an opportunity to opt out of further recruitment contacts. If they did not opt out, trained interviewers called them to assess their willingness to participate in a telephone interview, during which they were asked about all of the outcomes they had wanted from the care of their pain problem. Interviewers next read them a list of other possible outcomes that had been generated by the re­search team (which included a patient co-investigator) and asked them to rate the importance of each outcome on a scale of 1 to 5 (5 = extremely important, 1 = not at all important). Finally, they were asked again about any other outcomes they might have thought of. We gave completed interviewees a $40 gift card—this incentive was mentioned in both the pre-notification letter and introductory script. Out of 7 patients contacted within 3 tele­phone attempts, 6 agreed to participate and completed the inter­view. Two of those 6 said there was a family member who was very familiar with their pain problem and would be willing to be contacted, so we also recruited them and completed interviews. The responses from these 8 people were so consistent that no further pilot interviews seemed needed.

Patient/Family Interviews

Following the pilot, in hopes of completing 40 patient telephone interviews, we identified an additional random pool of 83 adult patients who had experienced a first CT or MRI scan of the back or abdomen for pain about 1 year prior. We followed the same protocol used in the pilot; the interview script—revised after the pilot calls—confirmed eligibility, obtained demographic infor­mation, and asked about the most important outcomes desired from the medical care of their problem. Then we asked subjects to rate a revised list of 21 outcomes (see Table 1 for the list in the order in which items were asked) on an importance scale of 1 to 5 (again, 5 = extremely important, 1 = not at all important). Finally, we asked them whether there was a family member who was familiar with their problem who might be willing to complete a similar interview. If so, we made contact arrangements and the family member interview followed the same process. Interviews lasted an average of 15 minutes and all respondents were sent a $40 gift card.

Physician Interviews

We identified and recruited practicing adult primary care physi­cians through the help of a co-investigator (CV) at the Institute for Clinical Systems Improvement for interviews by our practic­ing primary care physician co-investigator (JB). Because the phy­sicians had not had a specific personal experience to reference, we provided them with common case scenarios for patients with back or abdominal pain as context for their answers. We asked what outcomes they thought would be most important to the scenario patients; then they were asked to rate each of the 21 out­comes (see Table 1) identified from patients on the same 1-to-5 scale from the perspective of their patients. They were also asked about the most important outcomes from their perspective as a clinician.

Analysis

Associations between type of pain and patient attributes were tested using contingency tables and Pearson’s χ² and Fisher’s ex­act tests. Independent samples t tests were used to test differenc­es in outcome importance ratings between abdominal and back pain patients, as well as differences in outcome importance rat­ings between patients and physicians. The study has 80% power (α = 0.05, 2-sided test) to detect a difference of 0.9 standard deviations (SDs) in the mean outcome rating from abdominal and back pain patients. An α of 0.05 was considered statistically significant, 2-tailed tests were used, and there was no correction for multiple testing. Analyses were conducted using SAS version 9.1 (SAS Institute Inc, Cary, North Carolina).

RESULTS

Of 83 patients with call attempts, we could not reach 33, and 1 of those contacted was ineligible as they reported not having the scan (see Figure 1). Of the 49 eligible patients who could be contacted, 41 completed interviews—but the data from 1 was inadvertently lost&mdash;for a participation rate of 84% of eligible pa&shy;tients contacted, or 49% of all those attempted. Twelve patients nominated a family member who had been actively involved in the care of their pain problem for interviews, and 11 of those 12 completed them. Responding patients were similar to nonre&shy;sponders by age, race, Medicare insurance, pain type, and scan type. The major difference was that responders were much more likely to be females (92.5% vs 46.2%; P <.001) and to be on Med&shy;icaid (7.5% vs 0%; P = .045).

The characteristics of participants are listed by pain type in Table 2. The majority were female and white. Aside from the type of scan, none of the other characteristics differed signifi&shy;cantly by pain type. Ten of the family members were spouses and 1 was a daughter. Family member characteristics were similar to those of the patients except that family members were more likely to be male (7 of 11 [63%]) and married (10 of 11 [91%]) than patients as a whole.

Seventeen primary care physicians were identified as potential participants in the second-stage interview, but 4 of them referred us to colleagues who agreed to participate and completed inter&shy;views along with 7 others. Each physician was from a different clinic: 4 were from the Twin Cities metro area and 7 were from the rest of the state; 2 were internists and 9 were family physi&shy;cians; 3 were female and 8 male; and 6 had been in practice for 26 to 43 years, 2 for 12 to 15 years, and 3 for 4 to 6 years.

In the initial open-ended phase of the pilot patient inter&shy;views, the main outcome identified by most patients and family members was to learn the cause of the pain. Seven of the other outcomes on the list were also mentioned spontaneously before respondents were given the list to rate. Only 1 (“minimize dis&shy;comfort from the tests used to assess the pain”) was raised that was not on the list.

Table 3 contains the mean importance ratings by patients for the 21 outcomes and the difference between those ratings by pa&shy;tients with abdominal pain versus those with back pain. There was considerable overlap between the 2 groups, although patients with abdominal pain gave lower importance ratings for nearly all outcomes than did patients with back pain; the overall mean rat&shy;ing was 0.4 points lower than those with back pain (4.09 vs 4.50; P = .049). Higher ratings were given by patients with abdomi&shy;nal pain for only 2 outcomes: to find the cause and to get rapid and complete relief. The overall mean rating of the 21 outcomes from family and patients was similar (4.32 for family, 4.28 for pa&shy;tients; P = .86). Most (8 of 11) family members were connected with patients who had experienced abdominal pain. Importance ratings by family members of abdominal pain patients were the same or directionally higher than those of abdominal pain pa&shy;tients for 16 of 21 outcomes, and there were no statistically sig&shy;nificant differences.

Figures 2 and 3 provide graphic comparisons between patient and physician importance ratings for each of the outcomes, or&shy;dered by the magnitude of the difference between them. For ab&shy;dominal pain, physicians rated the importance to patients to be an average of 0.6 points lower than the ratings of patients (3.53 vs 4.09; P = .04); for back pain, that difference was 0.85 points lower (3.65 vs 4.50; P = .0003). Each of the 4 individual out&shy;comes with statistically significantly lower physician ratings for abdominal pain was also significantly lower for back pain. There were also another 9 of the outcomes for back pain with statis&shy;tically significant physician-patient differences. Moreover, all of the items with P <.01 or less were in the back pain list. The out&shy;comes with greatest agreement between patients and physicians were finding the cause, getting rapid and complete relief, and re&shy;turning to work and productivity as soon as possible. For most outcomes, the 3 younger physicians provided lower ratings than the 8 with longer practice experience. Patient ratings of abdomi&shy;nal pain outcomes tended to show more variability than physician ratings, with 15 of the 21 SDs for abdominal pain outcomes be&shy;ing higher for patients than physicians. However, for back pain, the pattern was reversed, with 7 of the 21 SDs for back pain outcomes being higher for patients than physicians.

When the physicians were asked about the most important outcomes from their perspective, 5 highlighted making a diagno&shy;sis, 6 thought it was needed to guide treatment, and 4 wanted to rule out something serious. Four physicians also noted a reduc&shy;tion in symptoms and 3 identified restoration of normal function as soon as possible. Single physicians mentioned humane care, prognosis, and good access to care.

DISCUSSION

These patients and their families rated a wide variety of out&shy;comes from their care as important. Although there were differ&shy;ences between the average importance ratings by type of pain, the differences were mostly small and all of the outcomes we asked about had relatively high ratings that were similar between patients and family members. Finally, the primary care physician interviewees tended to rate each of these outcomes as having lower importance than did patients or their families, even though the physicians were asked to rate the importance of each out&shy;come from the patients’ perspective rather than from their own. The similar shape of the importance difference curves in Figures 2 and 3 across all of these outcomes suggests that these dif&shy;ferences are real. Such differences highlight the importance of asking patients—both individually and collectively&mdash;about what outcomes are important to them.

The results of this study raise important questions about what approach should be used to identify PROMs. The current fo&shy;cus on using measures that have been identified by researchers and those limited to specific functions and symptoms will likely not capture the same outcomes that patients themselves might identify as important. McClimans, in his study, has objected to the whole idea of standardized questions, instead proposing a theoretical framework for thinking about PROMs that under&shy;stands them “as posing genuine questions to patients—questions that are open to reinterpretation [and context].”⁴ He particularly objects to measures that break down the outcome into a series of sub-questions and that assume their sum fully captures the overall outcome. McKenna raises a similar concern in his study, while emphasizing the importance of a hard science approach to measure development.⁵

Only one of the outcomes that these patients thought were im&shy;portant (ie, to get rapid and complete relief from pain and other symptoms) would be partially measurable by the PROMIS mea&shy;surement sets established by the National Institutes of Health to measure patient outcomes.⁶ PROMIS has developed question&shy;naires to ask about pain, fatigue, gastrointestinal symptoms, arm and leg function, sexual function, sleep, physical activity, affect, cognition, self-efficacy, substance use, social support, peer and family relationships, and social roles and activities. There are also global health status questions.^7.8 The individual question sets (short forms) for each of these domains varies from 4 to 10 questions selected from banked sets that contain as many as 121 questions. Their psychometric qualities are mostly well stud&shy;ied, and they are a wonderful resource for researchers, but there has been no patient involvement in selecting or reviewing these measures.^3,7,8

The other main source for patient outcome measures is the National Quality Forum. It has endorsed nearly 700 measures created by other organizations, 208 of which are identified as outcome measures.⁹ Although most of the latter are clinical out&shy;comes related to specific conditions, some do address patient function, usually in relation to a specific procedure—few are as generic as the outcomes considered important by our patient subjects. A similar resource in Britain, the Health and Social Care Information Centre, began work in 2009 on PROMs and current&shy;ly has measurement sets in place for hip and knee replacement, hernia repair, and varicose vein treatment&mdash;again, mostly clinical and specific for a procedure and condition.¹⁰ There is no indica&shy;tion on its website that patients had any role in identifying these measures.

PCORI has prioritized developing patient-centered outcome measures and is becoming a resource for PROMs. It is devel&shy;oping standards for such measures, but so far has not created or endorsed any specific measures. Its Methodology Committee has recommended that patients be included in the peer review process for grant proposals, and it has a standard requiring that researchers “engage people representing the population of inter&shy;est and other relevant stakeholders in ways that are appropriate and necessary in a given research context.”¹¹

Developing outcome measures that are meaningful to patients is a new endeavor for healthcare, and accordingly, it will take some time to get it right. Once we know what patients want mea&shy;sured, learning where to most efficiently and accurately obtain that information is another challenge. Although it might seem obvious that the best source of data is directly from patients, this may not always be feasible because of the cost and difficulty tracking and obtaining responses. Thus, we need to learn whether some of those outcomes can be obtained from medical records or insurance claims data. Yet another challenge will be learn&shy;ing how to incorporate those measures into the daily practice of medicine. Black suggests in his study that outcome measures could be transformative for healthcare, but admits that their use in routine practice is still uncommon, and identifies a series of challenges.¹² As if to demonstrate that, his group recently pub&shy;lished a study using PROMs to compare surgeons and conclud&shy;ed that the choice of outcome measure can substantially alter a surgeon’s rating.¹³ Valderas et al performed a systematic review of the literature on the impact of measuring PROMs in clinical practice.¹⁴ They confirmed that “contexts and interventions that will yield important benefits remain to be clearly defined.”

Limitations

Our findings in this study are limited to patients with 2 types of clinical situations: back pain that tends to be chronic or recur&shy;rent and abdominal pain that tends to be acute. The underlying disease processes and patient impacts of these 2 situations are both heterogeneous and different, which highlights how import&shy;ant it is to survey patients across a broad range of contexts if the goal is to fully understand patient preferences. The advantage of surveying patients as we did was that having their own recent experience with these potentially serious and disabling problems should have made their responses less abstract. All of the sample sizes in this study were small, and the subjects were not especially diverse or representative nationally, so these findings require con&shy;firmation in larger more representative, diverse samples. It is also likely that we have not identified all of the important outcomes.

Nevertheless, the findings are especially important in suggest&shy;ing that there are a wide variety of outcomes that are important to patients and their families, outcomes that are not necessarily limited to relief of particular symptoms or recovery of certain functions. Thus far, most of the attention to PROMs has focused on the psychometric and feasibility aspects of measures that are limited to specific symptoms and functions. It might be wise to first obtain more input from patients before once again learning that the outcomes we measure so well have little relevance to what patients want to know before they make decisions about their care.

Acknowledgments

The authors are grateful to the skilled interviewers who conduct&shy;ed all of these interviews so well, and to Cally Vinz, vice president at Institute for Clinical Systems Improvement, who has been an active participant in our meetings and helped identify and recruit physicians for interviews. The authors are also extremely grate&shy;ful to the patients, family members, and physicians who have provided them with so much helpful input; this includes Aimee Vang, Carol Dexter, Katharine Pittman, Jamie Gudknecht, Jen&shy;ny Zakoski, David Freedland, and Gail Soens from the Patient/ Family Advisory Board, who have been provided with a modest honorarium for their time. As advisers, they have each provided very helpful feedback on our survey protocol and pilot tested and provided suggestions to improve the survey described in this paper. In addition, John Wilkinson, MD; Ian Kenning, MD; and Deepti Pandita, MD, from the Physician Advisory Board have reviewed the authors’ plans and findings and provided practicing physician perspectives that have helped us to remain focused on that part of their targeted audience, even though we were unable to provide them with any recompense.

The funding agency, PCORI, had no role in the design and conduct of the study; collection, management, analysis, and in&shy;terpretation of the data; and preparation, review, or approval of the manuscript project.

Author Affiliations: HealthPartners Institute for Education and Research (LIS, SEA, JOT, RRW, JYZ), Minneapolis, MN; Health&shy;Partners Medical Group (JB), Minneapolis, MN; Group Health Research Institute (DC), Seattle, WA; University of Washington (JGJ), Seattle, WA; University of California (RS-B), San Francis&shy;co, CA; Saint Paul, MN (CN).

Funding Source: This work was supported through a Patient-Cen&shy;tered Outcomes Research Institute (PCORI) Pilot Project Pro&shy;gram Award. All statements in this report, including its findings and conclusions, are solely those of the authors and do not nec&shy;essarily represent the views of PCORI, its Board of Governors, or Methodology Committee.

Author Disclosures: Dr Solberg is a member of the ICSI regional board (Q1 collaborative), an employee of HealthPartners health system, and has past grants received from CMS, National Insti&shy;tute of Health, AHRQ, and PCORI. Dr Jarvik is a member of the General Electric-Association of University Radiologists Re&shy;search Academy Fellowship Advisory Board, has received a grant from PhysioSonics, an ultrasound-based diagnostic company, and also receives royalties from PhysioSonics. Dr Smith-Bindman has previously received a grant from PCORI for an unrelated study. The remaining authors report no relationship or financial interest with any entity that would pose a conflict of interest with the subject matter of this article.

Authorship Information: Concept and design (LIS, CKN, RS-B, JB, RRW); acquisition of data (LIS, DSC, JB, JOT, RRW, JYZ); analysis and interpretation of data (LIS, SEA, DSC, CKN, JGJ, RS-B, JB, JOT, RRW, JYZ); drafting of the manuscript (LIS, SEA, RS-B, JB); critical revision of the manuscript for important intel&shy;lectual content (LIS, SEA, DSC, CKN, JGJ, RS-B, JB, JOT, RRW, JYZ); statistical analysis (SEA, RRW); obtaining funding (LIS, RS-B); supervision (JOT); and providing content expertise on back pain (JGJ).

Address correspondence to: Leif I. Solberg, MD, HealthPartners In&shy;stitute for Education and Research, PO Box 1524, MS#21111R, Minneapolis, MN 55440-1524. E-mail leif.i.solberg@healthpart&shy;ners.com.

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