Key Considerations for Patient-Reported Outcome Measures
By Sharona Hoffman
Patient-reported outcome measures (PROMs) are questionnaires that patients fill out on tablets or other computers or devices. They ask patients to check boxes in answer to questions about their symptoms, treatment effects, and ability to function physically, emotionally, and socially. They thus may solicit very sensitive information about matters such as anxiety, depression, and sexual satisfaction. To illustrate, a query might be “in the past month, how often did you have a lot of trouble falling asleep,” and the patient is asked to check “never,” “rarely,” “sometimes,” “often,” or “always.”
PROM responses can be used for purposes of clinical care, research, quality improvement, Food and Drug Administration (FDA) approval of drugs and devices, and even insurance reimbursement. For example, insurers hypothetically could decide to decline coverage of particular treatments based on PROM responses indicating that many patients find them to be unhelpful.
I first became interested in patient-reported outcome measures because of an experience my husband had. Andy has Parkinson’s disease, and one of the neurologists he saw asked him to fill out a long questionnaire on a tablet computer before each appointment. This task was difficult for Andy because he had a hand tremor, and it was stressful because Andy worried that he would not have time to complete the survey before his appointment began. Moreover, Andy’s physician never referred to his responses and appeared never to look at them. Upon investigation, I found little to no analysis of PROMs in the legal literature, so Andy and I recently published a law review article about them.
PROM Benefits
PROM advocates are enthusiastic about their many potential benefits. Chief among them is filling data gaps. Because contemporary medical appointments are often brief, rushed, and in short supply, PROMs can provide valuable information that clinicians would otherwise not obtain. This includes not only details about symptoms and disease course, but also reports about treatment success or failure, since many physicians do not have the luxury of inviting patients for follow-up appointments unless further interventions are needed. PROMs can be particularly informative because the data come directly from patients. By contrast, clinician notes often constitute an interpretation of what the patient has said.
Other benefits are touted as well. PROMs can save clinicians time, allowing them to scan forms rather than have lengthy conversations with patients during office visits. They can also save costs because shorter visits generally cost less. Further, based on PROMs, clinicians may be able to discontinue expensive treatments that patients score poorly or withhold expensive treatments from patients who indicate they are doing well without them.
PROM Concerns
But PROMs have several shortcomings and raise important concerns as well. First, the questions must be well formed, and the tool must be characterized by reliability, responsiveness, and validity. Also, it may be challenging for clinicians to select the appropriate PROM from among the hundreds or thousands that are available. The National Institutes of Health have a database called PROMIS (PROM Information System) that contains over 570 PROMs that it endorses. Yet, even within this helpful resource, many different questionnaires address each subject matter, such as sleep, anxiety, and depression.
Patient responses may be skewed for a variety of reasons. First, they may be affected by response shift. Patients who initially indicated their symptoms are mild may later indicate that they are more severe not because there has been an objective change, but because of impatience or frustration that the illness persists. Response bias is another concern and occurs when patients’ answers are influenced by nonmedical factors such as a desire to please the physician or a personal tolerance for discomfort. Two people may experience identical levels of pain, but one may indicate that it is severe while the other indicates it is mild because she tends to avoid complaining or does not want to disappoint the doctor by relating that treatment is unsuccessful.
Missing data may also diminish PROM usefulness. First, not everyone who is asked to complete PROMs will be willing to do so. Some patients will not be comfortable with technology or will have difficulty clicking on responses because of disabilities such as Parkinson’s disease. Others may have reading comprehension limitations or decline to answer for other reasons. If the population of patients completing PROMs is not representative of all patients of interest, research outcomes could be adversely affected.
Further, even patients who do fill out surveys may skip certain questions or stop before completion because they find the survey to be too long. According to one source, to avoid survey fatigue, questionnaires should not exceed thirty queries in length.
PROM shortcomings are not limited to data quality concerns. In recent years, there is significant discussion of physician burnout, a very troubling and increasingly common phenomenon. Adding the additional task of reviewing dozens of PROMs for each patient may well exacerbate burnout. PROM review during appointments may also further reduce the time that physicians have to interact with patients, which is already often limited to 15-20 minutes. In addition, administering PROMs can be costly for medical practices. Offices may need to purchase tablets to distribute to patients in waiting rooms or pay staff to process PROM responses and prepare them for clinician use.
In the legal realm, both privacy and medical malpractice vulnerabilities are of concern. PROMs often solicit very private information from patients, and if the data are not stored securely, hackers and other unauthorized personnel may have access to more sensitive details than would otherwise be present in electronic health records.
Medical malpractice could occur for two reasons. First, clinicians may ignore important data that patients provided in PROMs and did not repeat verbally during visits. Second, physicians may over-rely on PROMs, discontinuing needed treatment or failing to conduct necessary tests because patients indicate (too optimistically) that they are doing well in PROM responses. Both disregarding PROM responses and over-relying on them can lead to medical mistakes.
Recommendations
Addressing PROM concerns will require both technical and legal measures. Clinicians, researchers, and regulators who use PROMs must recognize their existing shortcomings and carefully consider the extent to which they rely upon them. Pilot programs should be conducted prior to implementation to determine whether particular PROMs are useful and manageable for clinicians and patients. Every effort should be made to minimize the burden of collecting and reviewing PROM responses. Computer adaptive testing can shorten questionnaire length by adjusting the questions posed based on prior answers. Thus, patients who indicate they do not have particular symptoms could have relevant follow-up questions eliminated. To ease the burden on clinicians, staff members could review PROM answers and provide clinicians with summaries and highlights of matters that require attention. Better yet, artificial intelligence could be harnessed for the same purpose.
The HIPAA Privacy Rule should address PROMs as well. Because of the sensitive nature of much of the data, HIPAA should provide that as a default, PROM responses should not be disclosed to third parties (even when HIPAA permits disclosures) without specific requests for them along with justifications as to why they are needed. For example, researchers may well need PROM responses in some circumstances. But employers who are trying to determine if someone is physically qualified for a job will not need information about matters such as anxiety or sexual functioning.
Litigants should consider PROMs as well and ask about them in discovery. As indicated above, medical errors may potentially occur because clinicians ignore data provided in PROMs or inappropriately rely on PROM responses to the exclusion of other forms of medical examination.
When medical practices use PROMs, they should educate patients about them. Patients should be told whether providers will rely heavily on their responses so that they know the questionnaires must be completed fully and thoughtfully. By contrast, if surveys will be used only for research or quality improvement, patients should know that they must repeat important data when speaking with the provider during the appointment.
PROMs are likely to become more commonly implemented in the future as an efficient way to gather and process medical data. It is thus important to analyze their strengths and weaknesses and make every effort to minimize their deficiencies and maximize their benefits.
This post is based on Sharona Hoffman & Andy Podgurski, The Patient’s Voice: Legal Implications of Patient- Reported Outcome Measures, 22 Yale J. Health Pol’y, Law, and Ethics 1 (2023).
Sharona Hoffman is the Edgar A. Hahn Professor of Law, Professor of Bioethics, and Co-Director of Law-Medicine Center, Case Western Reserve University School of Law.
