D. Patient-Centered Outcomes Research and HTA

HTA is directly and indirectly related to certain patient-oriented concepts, including patient-centered care, patient-centered outcomes, patient-reported outcomes, and patient-centered outcomes research.

The term “patient-centered medicine” was introduced more than 40 years ago by Balint and colleagues (Balint 1970) in contrast to what they called “illness-centered medicine.” Whereas “traditional diagnosis” was based on disease-centered thinking, an “overall diagnosis” involved an understanding of the patient’s complaints based on patient-centered thinking (Stewart 2013). A contemporary definition of patient-centered care is:

The experience (to the extent the informed, individual patient desires it) of transparency, individualization, recognition, respect, dignity, and choice in all matters, without exception, related to one’s person, circumstances, and relationships in health care (Berwick 2009).

The main dimensions of patient-centered care include:

• Disease and illness experience (patient-as-person)
• Whole person (biopsychosocial perspective)
• Common ground (sharing power and responsibility)
• Patient-doctor relationship (therapeutic alliance) (Hudon 2011; Mead 2000; Stewart 2013)

As a particular form or variant of health care, patient-centered care can have a favorable impact on safety, effectiveness, and related health care outcomes, although the evidence to date of its impact is mixed (Street 2012; Zandbelt 2007). As such, patient-centered care can be a topic of HTA.

Patient-centered care can be evaluated using such measures as:

• Patient Perception of Patient-Centeredness (PPPC): patient perceptions of patient-centered care during the last visit with a family physician; uses 14 items
• Consultation Care Measure (CCM): patients’ perceptions of patient-centered care during the last visit with a family physician; 5 subscales: communication and partnership, personal relationship, health promotion, positive and clear approach to the problem, interest in effect on life
• CAHPS (Consumer Assessment of Healthcare Providers and Systems): brief general measure comparing overall quality of interpersonal care across health care settings; includes some patient-centered care domains: access (getting care quickly, getting needed care), provider communication (Epstein 2011; Hudon 2011)

In addition, patient-centeredness may be considered a dimension of health care quality (Berwick 2009), i.e., a standard attribute of practice.

Patient-centered outcomes (or patient-oriented outcomes) comprise a subset of the broad array of health outcomes noted above (mortality, morbidity, adverse events, quality of life, etc.). Patient-centered outcomes refer to outcomes that patients experience across the variety of real-world settings, including: survival, functional status, quality of life, quality of death, symptoms, pain, nausea, psychosocial well-being, health utility (patient-perceived value of particular states of health), and patient satisfaction. Excluded are outcomes or other endpoints that patients do not experience directly, e.g., blood pressure, lipid levels, bone density, viral load, or cardiac output. Patient-centered outcomes can be assessed at a generic level or a disease/condition-specific level. Examples of generic instruments for assessing patient-centered outcomes include:

• CAHPS
• EuroQol (EQ-5D)
• Functional Status Questionnaire (FSQ; 34 items)
• Health Utilities Index
• Nottingham Health Profile
• Quality of Well-Being Scale
• Short Form (12) Health Survey (SF-12)
• Short Form (36) Health Survey (SF-36)
• Sickness Impact Profile (SIP; 136 items)

There are patient-centered outcomes instruments for such diseases and conditions as angina, asthma, epilepsy, kidney disease, migraine, multiple sclerosis (MS), and vision. For example, standard outcomes for assessing MS activity and severity include annualized relapse rate and the Expanded Disability Status Scale. Patient-centered outcomes instruments that may better reflect patient experience with MS include MS-specific instruments and hybrid instruments (i.e., generic instruments with additional MS items). Examples of MS-specific patient centered outcome instruments are: the Multiple Sclerosis Quality of Life Inventory (MSQLI), Multiple Sclerosis Quality of Life-54 scale (MSQoL-54), Functional Assessment of Multiple Sclerosis (FAMS), Multiple Sclerosis Impact Scale (MSIS-29), and Leeds Multiple Sclerosis Quality of Life scale (LMSQoL) (Miller 2010).

Patient-reported outcomes (PROs) are those patient-centered outcomes that are self-reported by patients (or reported on their behalf by their caregivers or surrogates) or obtained from patients by an interviewer without interpretation or modification of the patient’s response by other people, including clinicians (Rothrock 2010; US FDA 2009). Patient ratings of health status, adverse events, functional status, quality of life, and other outcomes may differ from measures recorded by clinicians (see, e.g., Ebell 2004; Nakamura 2012), and patients may weigh benefits and risks differently among themselves as well as differently than clinicians when considering treatment alternatives (Wong 2012). Non-patient reported outcomes are those that matter to patients but are not reported directly by them.

Patient-centered outcomes research (PCOR) generates evidence comparing the impact of health care on patient-centered outcomes. PCOR can draw on a wide variety of methods, including, e.g., practical or pragmatic RCTs, cluster randomized trials, and other trial designs; registries, insurance claims data, systematic reviews, and others (Methodology Committee 2012). As described by the recently established Patient-Centered Outcomes Research Institute (PCORI) in the US, PCOR answers these patient-focused questions:

• Given my personal characteristics, conditions and preferences, what should I expect will happen to me?
• What are my options and what are the benefits and harms of those options?
• What can I do to improve the outcomes that are most important to me?
• How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare? (Patient-Centered Outcomes Research Institute 2013)

In summary, HTA can assess patient-centered care as a type of intervention. HTA can assess the impact of health care interventions on patient-centered outcomes, including patient-reported outcomes, of the full range of health technologies. Using a variety of primary and secondary data methods, patient-centered outcomes research generates data and evidence that can be used in HTA.