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  1. S. J. Bartlett1,
  2. C. Bingham2,
  3. Z. Predmore3,
  4. T. Concannon3,
  5. E. Chen4,
  6. S. Schrandt5,
  7. R. Xie6,
  8. R. Chapman6,
  9. L. Frank4
  1. 1McGill University, Divisions of Clinical Epidemiology, Rheumatology and Respiratory Epidemiology and Clinical Trials Unit, Montreal, Canada
  2. 2Johns Hopkins University, Division of Rheumatology, Baltimore, United States of America
  3. 3RAND Corporation, RAND Health Care, Boston, United States of America
  4. 4RAND Corporation, RAND Health Care, Arlington, United States of America
  5. 5ExPPect, n/a, Arlington, United States of America
  6. 6IVI - Innovation & Value Initiative, n/a, Bethesda, United States of America


Background Common approaches to valuing health technologies often fail to capture outcomes that matter to patients and families. The treatment goals of people living with rheumatoid arthritis (RA) include common trial endpoints but also include other facets of disease impact. Identifying a feasible and rigorous approach to inclusion of the patient perspective is needed as trialists increasingly seek to incorporate patient-important outcomes in trial design and as varied patient-centered value assessment frameworks emerge. No standard approach is available to systemically identify and quantify patient-important outcomes, nor to include those outcomes in deliberative decision-making. We developed the Patient-Engaged Healthcare Valuation strategy, using principles of goal attainment scaling to frame survey-based goal collection directly from adults.

Objectives To develop and test a goal-based method for collecting RA patient input for use in clinical trials and value assessment and evaluating the feasibility of this approach in people with RA.

Methods Patient goals and domains were identified from (1) a literature review (2010-2020) of patient outcomes, goals, and preferences in RA, and (2) discussions with patients and clinicians during two meetings with a steering committee (SC) consisting of clinicians, outcomes researchers, patients/advocates, and health economists. These goals informed the development of a draft survey. Adults with RA were recruited from online patient networks to rate goal importance and suggest additional goals. SC members reviewed the survey findings and assessed feasibility of scaling up goal collection for HTA.

Results Of 135 articles identified, 17 were retained. An inductive and iterative approach was used to identify and thematically group the final set of 36 goals into 4 domains. The draft survey was cognitively debriefed with 4 adults with RA. The first survey was administered to 20 participants; results informed item revisions and additions for the second round of data collection (n=27).

The 47 respondents were mostly White (87%), college-educated (72%) women (93%) living with RA for an average of 15 years; 75% rated their RA as moderate to severe. Free-text goals added in round 1 include: 1) finding specialists who listen to patient input on symptoms; 2) addressing loneliness or isolation; and 3) finding support from or helping others with RA. All Symptom and Life Impact goals were rated as Important or Very Important by ≥85% of participants; endorsement for Management and Treatment goals was somewhat more variable, with ≥85% endorsing these as Somewhat to Very Important. Results suggested that domains match key goals. Steering committee ratings supported the feasibility of this method.

Conclusion Goals relevant for RA treatment evaluation can be efficiently identified and rated for importance by patients. Patient-important goals can be incorporated into deliberative healthcare valuation using this method to permit “crowd-sourced” input from people living with RA and to capture heterogeneous patient perspectives in healthcare valuation.

Table 1.

Top Goals based on rating as “Very Important” by >70% of subjects, from set of 36. “My goals for living with RA are to…”

Acknowledgements I have no acknowledgements to declare.

Disclosure of Interests None declared

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