HPM 794:

Patient-Reported Outcomes Measurement and Application in Healthcare Research and Practice

(Credit Hours: 3)

Department of Health Policy and Management Gillings School of Global Public Health

Spring 2017 Syllabus

Faculty: Bryce B. Reeve, Ph.D.

Email: bbreeve@email.unc.edu Phone: 919-962-5434

Office Hours: By appointment Course Time & Location: Tuesdays from 2:00pm – 4:45pm in McGavran-Greenberg Hall 1304

Course Overview

Patient-reported outcomes (PROs) are any report of health status that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else. PROs include, but not limited to, health-related quality of life, symptom burden, functional status, and satisfaction with care. PROs are becoming increasingly important as a primary or secondary outcome to measure in clinical trials and comparative effectiveness research studies. In addition, PROs are starting to be routinely collected in healthcare delivery settings to enhance patient-doctor communication and decision making. This course will provide a comprehensive overview of the PRO measurement and research field. This includes an in-depth review of how to select a high quality measure of a PRO and how to design and evaluate a PRO questionnaire. The course will provide best practices and real case examples for including PROs in clinical research and healthcare delivery settings. The course is open to doctoral and masters students, including students from the Schools of: Public Health, Medicine, Nursing, Pharmacy, Psychology (within Arts and Sciences), Dentistry, and Information and Library Science. Students will develop and leave the course with a tangible product that will serve as a springboard to the next stage in their careers, be it additional class work, dissertation, or entering (or returning to) the professional job market. Students will lead class discussions of current issues in PRO research, meet and interact with care providers and researchers, and develop and present a term paper focusing on a major issue in PRO measurement and research that may serve as the basis of a future work report, manuscript, or grant application.

Learning Objectives and Competencies

Course Learning Objective Doctoral-level Competencies

Masters-level Competencies

1 Build understanding of the fundamental principles,

concepts and models of PROs including health-related quality of life.

1,2,3,4,8,9,10 2,3,6,9,11

2 Develop and apply skills using qualitative methods such as focus groups and cognitive interviewing to evaluating a PRO measure.

1,2,3,4,6,8,9,10 2,12

3 Develop and apply skills using quantitative methods for evaluating the psychometric properties of a PRO measure.

1,2,3,4,6,7,8,9,10 2,3

4 Build knowledge on the principles and best practices for integrating PROs in observational studies, population surveillance, and clinical trials.

1,2,3,4,5,6,8,9,10 2,3,9,10,12,14,23

5 Build knowledge on the principles and best practices for integrating PROs in healthcare delivery settings.

1,2,3,4,5,6,7,8,9,10 2,3,4,9,10,12, 14,15,22,23

6 Understand the role of patient engagement as both co-investigators and research participants.

1,2,3,4,8,9,10 2,9,14

7 Develop and practice skills in research by reviewing and synthesizing literature, leading topic-focused discussion, developing a research proposal or manuscript draft, and presenting it in a small group forum.

3,5,6,7,8,9,10,11,1213,15

1,2,3,5,11,12, 13,18,19,21,25

HPM Core Competencies (Doctoral Level)

1. Understand critical issues 2. Develop expertise in a substantive area 3. Review and synthesize a body of research literature 4. Identify, apply theoretical knowledge / conceptual models 5. Develop hypotheses that can be tested in research 6. Select appropriate research designs and methodologies 7. Understand and apply analytical strategies 8. Identify ethical implications of research methods

9. Interpret and explain the results of research 10. Critically evaluate articles from scholarly journals and

research presentations 11. Write articles for submission to scholarly journals 12. Understand grant writing process / write proposals 13. Make oral presentations to scientific audiences 14. Participate in teaching a course 15. Explain research to various audiences.

HPM Core Competencies (Masters Level)

1. Accountability 2. Achievement

Orientation 3. Analytical Thinking 4. Change Management 5. Communication Skills 6. Community and Public

Health Orientation

7. Financial Skills 8. Human Resource

Management 9. Information Seeking 10. Information Technology 11. Initiative 12. Innovative Thinking 13. Interpersonal Awareness

and Emotional Intelligence

14. Organizational Awareness 15. Performance Measurement 16. Political Savvy 17. Process Management and

Organization Design 18. Professionalism 19. Project Management 20. Reputation Management

21. Self-Confidence 22. Strategic Orientation 23. Systems Thinking 24. Talent Development 25. Team Dynamics

Resources

Articles: Most assigned readings are available electronically via Health Sciences Library website:

http://www.hsl.unc.edu/

Books: There is no required book for class, however there are a few good books out there to reinforce understanding of the topics covered in class:

Cappelleri JC, Zou KH, Bushmakin AG, et al. Patient-Reported Outcomes: Measurement, Implementation and Interpretation. New York: Chapman & Hall/CRC Biostatistics Series, 2014.

Fairclough DL. Design and Analysis of Quality of Life Studies in Clinical Trials. Boca Raton, FL: Chapman & Hall/CRC Press Company, 2002.

Fayers PM, Machin D. Quality of Life Assessment, Analysis and Interpretation. Chichester: John Wiley & Sons, LTD, 2000.

Lenderking WR, Revicki DA. Advancing Health Outcomes Research Methods and Clinical Applications. McLean, VA: Degnon Associates, 2005.

Cella D, Hahn EA, Jensen SE, Butt Z, Nowinski CJ, Rothrock N, Lohr KN. Patient-Reported Outcomes in Performance Measurement. RTI Press. 2015: assessable at: http://www.rti.org/pubs/patient-reported_outcomes_in_performance_measurement.pdf

Websites: Measures and Measure Databases MAPI’s Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID): http://www.proqolid.org/ NCI’s Grid-Enabled Measures Database: https://www.gem-beta.org/ AHRQ’s Consumer Assessment of Healthcare Providers and Systems (CAHPS) http://cahps.ahrq.gov/ AHRQ’s CAHPS Item Set for Children with Chronic Conditions https://cahps.ahrq.gov/surveys-guidance/item-sets/children-chronic/index.html

NIH’s HealthMeasures website (PROMIS, NIH Toolbox, Neuro-QOL, ASCQ-Me: http://www.healthmeasures.net/ Rehabilitation Measures Database http://www.rehabmeasures.org/ Medication Non-adherence Scale (by Corrine Voils) http://sites.duke.edu/corrinevoils/ RTI & UNC’s Patient-Centered Communication in Cancer Care (PCC-Ca) Measures https://www.rti.org/impact/patient-centered-communication-cancer-care-instrument PRO Organizations Patient-Centered Outcomes Research Institute (PCORI): http://www.pcori.org/ International Society for Quality of Life Research (ISOQOL): http://www.isoqol.org/ International Society for Pharmacoeconomics and Outcomes Research (ISPOR): http://www.ispor.org/ Research Projects / Consortiums GeneQoL Consortium: Genetic Research into Quality of Life: http://www.geneqol-consortium.org/ Critical Path Institute’s Patient-Reported Outcome (PRO) Consortium: http://c-path.org/PRO.cfm Center for Disease Control and Prevention: Health-Related Quality of Life (HRQOL)

http://www.cdc.gov/hrqol/ http://www.cdc.gov/hrqol/HRQOLdata/index.html

Allows visitors to view HRQOL trend data for the entire United States and individual states overall, by gender, age group, race/ethnicity, and by measure and year.

PRO Guidance CMTP’s Effectiveness Guidance Document on Recommendations for Incorporating Patient-Reported Outcomes in Clinical Comparative Effectiveness Research (CER) in Adult Oncology http://www.cmtpnet.org/wp-content/plugins/download-monitor/download.php?id=62 COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) http://www.cosmin.nl/index.php Institute of Medicine (IOM) report on Capturing Social and Behavioral Domains and Measures in Electronic Health Records: Phase 2 http://iom.edu/Reports/2014/EHRdomains2.aspx ISOQOL-NCI Sponsored Webinars: Best Practices for Integrating Patient Reported Outcomes in Oncology Clinical Trials http://prevention.cancer.gov/news-events/events/videos/pro_symptommanagementwebinars PlainLanguage.gov: to promote the use of plain language for all government communications. http://www.plainlanguage.gov/ Webinar on Item Response Theory modeling (by Dr. Karon Cook) – 5-part series starting with: https://www.youtube.com/watch?v=SrdbllMYq8M&list=PLJNUIJnElUzDmrIPunMyF3tTvIHb65wNb Publicly Available Datasets: CMS’ Medicare Health Outcomes Survey (MHOS) http://www.hosonline.org/ NCI & CMS’ SEER-MHOS Linked Database https://healthcaredelivery.cancer.gov/seer-mhos/ NCI & CMS’ SEER-CAHPS Linked Database https://healthcaredelivery.cancer.gov/seer-cahps/ CDC’s National Health Interview Survey (NHIS) http://www.cdc.gov/nchs/nhis.htm

CDC’s Behavioral Risk Factor Surveillance System (BRFSS) http://www.cdc.gov/brfss/ NCI’s Health Information National Trends Survey (HINTS) http://hints.cancer.gov/ Key Regulatory Documents: FDA’s Guidance for Industry: Patient-Reported Outcomes Measures: Use in Medical Product Development to Support Labeling Claims: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf FDA’s Guidance for Industry: Qualification Process for Drug Development Tools: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf Clinical Outcome Assessment Qualification Program http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm284077.htm

Requirements and Expectations

Class preparation and participation:

Attendance in class is mandatory for each class session. In the event of a truly extraordinary circumstance, attendance may be excused if brought to the instructor’s attention in advance. All students are expected to do the pre-reading required for each class and to come to class prepared to actively participate in class discussions. Class participation is worth 25% of the final grade, and will be evaluated in a summary measure based on the Evaluation Method noted below. Beyond attending class, participating fully in the discussions will count towards achieving the full score for this requirement. Phones, Laptops, tablets: Please turn off cell phones in class. Laptops may be used in class only for taking notes and for looking up information relevant to the topic being discussed – email programs, facebook, games, twitter, instagram, etc. will not be accessed during class. Violations of this rule will affect your participation grade. Percentage of final grade: 25%.

Group Assignment

Students will work in groups of 4 or less on an evaluation of an existing PRO measure of uncertainty in medical care. Students will be expected to work on the project both in and outside class. The project will reinforce the student’s skills in evaluating and revising a questionnaire. More information will be provided in class. Each group will present their results on Feb 28 (subject to change).

Percentage of final grade: 10% (peer grading)

Individual Assignments: Article Discussion.

Each student will identify an article from the literature related to PRO measurement and research, and lead a twenty to twenty-five minute class discussion of the article during the semester. The article should be relatively recent (e.g., past 5 years), but I would prefer you to select a good quality (i.e., interesting, controversial) article over one that is just recent. Approximately 10-15 minutes of the time should be left to moderating a discussion of the article with the class, including providing lead questions for discussion. Presentation date: Students will notify Bryce of their top 3 dates to present an article in priority order. I will do my best but can’t guarantee a spot. If you need to switch a day, please arrange on your own with another student to switch; however, please notify Bryce of the change. Please submit the full citation and pdf of article to Bryce at least 2 weeks prior to your presentation date. **Make sure you read the article before sending the information to Bryce. All students are expected to read and be prepared to discuss the student article on day of class presentation. Presenters should treat this presentation as if they were presenting at a scientific conference.

Guidance for structuring the article discussion. The following is a widely-used format for structuring journal article discussions, and is encouraged for discussion of articles in this class. Please note that all students will have read your paper before class; thus, you don’t need to go into deep detail on methods. 1. Overview. (Consider one PP slide per bullet point below)

What is the overall study question/issue? i. There may be multiple questions/issues, though there is typically a

primary one. ii. A brief background to the issue is important for understanding the context

of this article, its relevance, and implications.

What methods were used to answer the question / address the issue?

What are the major results, as the author reported them?

What are the conclusions and implications of findings?

2. Evaluation and Discussion Note: The evaluation and discussion is both a presentation and an interactive discussion – it is appropriate to solicit the feedback of others in the class; for example, if they agree or disagree, or have particular perspectives on the matter, particularly with regard to the conclusions, implications, and future research points.

Address the strengths and limitations of the study, including (as appropriate): i. Study design appropriate for the study question?

1. Strengths/weaknesses of study design (e.g., qualitative and/or quantitative approach; randomized double blind control trial; case-control; etc.)

2. Groups treated equally/appropriately during study ii. Results

1. Significant? (In the statistical or clinically-relevant sense) 2. Important? (In the substantial or meaningful sense)

iii. Conclusions / Implications 1. Justified? 2. Actionable (e.g., can inform the "Why?" of an issue [vs. just being

descriptive or oriented toward "What?"], or contribute to an intervention to change research or outcomes?)

Take home message (****very important to address this) i. This is the focal point of the discussion. What do you think about the

article? What is the big take home message? What are next steps or research gaps? Are the authors on-track or off-base? What do we learn from this, in terms of what was right/good, what was wrong/off-target?

Guidance for future research. (****very important to address this) i. This may come through discussion, above, but I would like to emphasize it

as a meaningful take-away from the article in and of itself. As researchers and practitioners, how is our research (or our colleagues' research, if this isn't exactly our personal research focus area) informed by this work?

Percentage of final grade: 20%.

Term Paper.

The purpose of the term paper is three-fold: 1. To provide you a forum to focus on an element of PRO measurement and/or

research that is of greatest interest to you, 2. To help cement what you have learned in the class through (near) real-world

application, and 3. To allow you to develop a tangible product that may serve as the basis for the next

steps in your career, such as a. A manuscript for publication, b. Basis for your dissertation research. c. A draft PRO questionnaire for a study. d. A grant proposal for a K-award or other investigator-initiated funding, e. An interview and job talk for an academic or practice-related position in cancer

care quality, or f. All of the above.

There are three components to the term paper assignment:

a. The term paper topic and overview (1 page) will be proposed and submitted at the beginning of class 4 (please make sure your name is on the page). This will insure that students are actively engaged in thoughtful preparation and work on the term paper, and allow timely feedback to shape the completion of the term paper. You must respond to any questions or comments I raise and seek comment or changes. You must get final confirmation from me before grade can be determined.

b. The final term paper will be in the format of a manuscript (1. Introduction, 2. Methods, 3. Results, 4. Conclusions) or portions of a grant application (A. Overview and specific Aims, B. Background, C. Preliminary studies [if appropriate], and D. Proposed methods.) The term paper is due on the First Day of Exams for the University, according to the University Registrar’s calendar. It will be at maximum 15 pages in length excluding citations (line-spacing exactly 2.0 (double-spaced), 1” margins, 11 point TNR or Arial font). Citations are required, but do not fall within the page constraints.

c. The term paper presentation will be approximately 10 minutes in length plus approximately 5 minutes for Q&A, and will occur on one of the last two days of the class. It will be an oral, PowerPoint-aided presentation, in which the student stands to present (as if in a scientific conference). The presentation format will be variable, but should generally be structured as a professional presentation of research, or the synthesized motivation for a research agenda (e.g., literature review or grant proposal development). Given the short length, hit on the major issues (Overall study questions/objectives/goals; methods used to address question/objective/goals; results; overall conclusions**; take home message**)

A goal of the class is for you to complete the class with momentum toward your next set of academic/professional activities, be it publishing a paper or submitting a grant application. Accordingly, you are encouraged to select a topic that you may be interested in developing further, or may be working on in another class, in order to gain synergy and develop products that may be of a higher caliber than if you were to address them independently.

For example, if you are taking a research methods class and are working on data, you may approach this as an opportunity to review and synthesize the literature supporting your analysis. An idea may be to approach the data as a pilot analysis for a grant application, in which your methods/data would be presented in Section C of a grant proposal, and this class could provide the forum for an extensive literature review and synthesis to comprise Sections A and B. It is important that a topic or issue be explicitly identified and evaluated. That is, this is not to be a "book report," rather, your topic should be developed following the guidelines outlined in the Evaluation section, below. Guidance for the presentation of the article discussion (above) may also be informative in terms of providing a structure, and insight regarding emphasis. While this synergy is encouraged, it is not acceptable to turn in the exact same product for credit in two separate classes (or one you submitted to a prior class). To minimize uncertainty, it is encouraged to work with the instructor to select a topic and touch base periodically regarding progress, to obtain guidance for an effective end-product. It is important that the central focus of this paper is to be on patient-reported outcomes measurement and/or research. In other words, it is incumbent upon the student to successfully make the case for how or why their topic is related to the course topic. Also, please make sure you are not duplicating what may have been recently published in the field. Percentage of final grade: Term paper topic and overview: 5%. Percentage of final grade: Final Term Paper: 30%. Percentage of final grade: Term Paper Presentation: 10%.

Exams:

There are no written mid-term or final examinations for this course. There may be a pop-quiz if it is felt by the instructor that students are not doing the readings. Grades on the pop-quizzes will count in the participation grade.

Student Evaluation

Work will be evaluated, given a letter grade, and returned to you as soon as possible. Your assignments will be graded on the following criteria: organization, responsiveness, use of readings, and synthesis of ideas, as follows:

CRITERION RANGE OF PERFORMANCE

ORGANIZATION Marginal (L) Good (P) Excellent (H)

Organization and presentation of ideas.

Difficult to follow. Sequence hard to see.

Clear, crisp logical response.

Innovative organization. Use of charts, diagrams, and other materials where appropriate.

RESPONSIVENESS

Responsiveness to questions/issues.

Tendency to take a tangent.

Generally on track. Understands key principles and interprets appropriately within the assignment.

Interprets questions, innovates and explicitly responds to them. Clearly states assumptions under-lying position.

USE OF READINGS

Evidence of awareness of key ideas or facts brought out in readings

Little or no mention of ideas from readings.

Mentions key ideas or thoughts from core readings.

Discusses and critically analyzes ideas and theories as applied to assignment. Integrates optional/ recommended readings. (Uses appropriate outside citations to support analysis.)

INTEGRATION

Integration of ideas; models, thought based on readings and/or assignment; evidence of synthesis, new perspective, unifying concepts.

Repetition or summarization of articles with little or no application.

Straightforward, factual response.

Builds, innovative, and relates concepts from several articles to make a point or establish a position.

Letter grades will be assigned for all assignments, and numerically averaged using the values here:

Grades

Letter Number

H 98

H- 93

P+ 90

P 85

P- 80

L+ 75

L 70

L- 65

F <65

The expectation is that most students will receive a “P” grade. Truly exceptional work may result in a grade of “H”.

UNC Honor Code

The principles of academic honesty, integrity, and responsible citizenship govern the performance of all academic work and student conduct at the University as they have during the long life of this institution. Your acceptance of enrollment in the University presupposes a commitment to the principles embodied in the Code of Student Conduct and a respect for this most significant Carolina tradition. Your reward is in the practice of these principles. Your participation in this course comes with the expectation that your work will be completed in full observance of the Honor Code. Academic dishonesty in any form is unacceptable, because any breach in academic integrity, however small, strikes destructively at the University's life and work. If you have any questions about your responsibility or the responsibility of faculty members under the Honor Code, please consult with someone in either the Office of the Student Attorney General (966-4084) or the Office of the Dean of Students (966-4042). Read “The Instrument of Student Judicial Governance” (http://instrument.unc.edu).

Recognizing, Valuing, and Encouraging Diversity

Promoting and valuing diversity in the classroom enriches learning and broadens everyone’s perspectives. Inclusion and tolerance can lead to respect for others and their opinions and is critical to maximizing the learning that we expect in this program. This may challenge our own closely held ideas and personal comfort zones. The results, however, create a sense of community and promote excellence in the learning environment. Diversity includes consideration of (1) the variety of life experiences others have had, and (2) factors related to “diversity of presence,” including, among others, age, economic circumstances, ethnic identification, disability, gender, geographic origin, race, religion, sexual orientation, social position. This class will follow principles of inclusion, respect, tolerance, and acceptance that support the values of diversity.

Disability Accommodation

UNC-CH supports all reasonable accommodations, including resources and services, for students with disabilities, chronic medical conditions, a temporary disability, or a pregnancy complication resulting in difficulties with accessing learning opportunities. All accommodations are coordinated through the UNC Office of Accessibility Resources & Services (ARS), http://accessibility.unc.edu; phone 919-962-8300 or email accessibility@unc.edu. Students must document/register their need for accommodations with ARS before any accommodations can be implemented.”

Course Evaluation

HPM participates in the UNC-CH’s online course evaluation system. Your responses will be anonymous, with feedback provided in the aggregate. Open-ended comments will be shared with instructors, but not identified with individual students. Your participation in course evaluation is an expectation, since providing constructive feedback is a professional obligation. Feedback is critical, moreover, to improving the quality of our courses, as well as for instructor assessment.

Course Schedule

Week

1

Jan. 17 Topic:

Course Overview and Introduction to “Patient-Reported Outcomes Measurement and Application in Healthcare Research and Practice”

Readings

Patrick DL, Chiang YP. Measurement of health outcomes in treatment effectiveness evaluations: conceptual and methodological challenges. Medical Care 2000;38(9, Supp II):II-14-II-25.

Eton DT, Ridgeway JL, Egginson JS et al Finalizing a measurement framework for the burden of treatment in complex patients with chronic conditions. Patient Related Outcome Measures 2015:6 117-126.

Module: Design and Evaluation of a PRO Measure

2

Jan. 24 Topic: Steps to Design and Evaluate a PRO Measure

Assignment due before class:

Submit to Bryce via email (bbreeve@email.unc.edu) prior to start of class a ½ page (in Word) narrative bio (program, education, background, research interests, favorite fun things/hobby) and a picture (head shot) of yourself. Please put your picture in the Word document.

Registered students: Submit to Bryce your top 3 preferred dates for student article presentation

Go to website: http://www.sf-36.org/demos/SF-36.html ,complete survey and see your scores. Think about what you learned.

Readings: Scientific Advisory Committee of the Medical Outcomes Trust. Assessing health status and quality-of-life instruments: Attributes and review criteria. Quality of Life Research 2002;11:193-205.

McCormack LA, Treiman K, Rupert D, Williams-Piehota P, Nadler Eric, Arora NK, Lawrence W, Street RL. Measuring patient-centered communication in cancer care: A literature review and the development of a systematic approach. Social Science & Medicine 2011;72:1085-1095.

3

Jan 31 Topic: Qualitative Methods to Develop and Evaluate a PRO Measure

Guest Speaker:

Antonia Bennett, PhD Research Assistant Professor, HPM

Readings: Patrick, D. L., et al. Content Validity-Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 1-Eliciting Concepts for a New PRO Instrument. Value Health 2011; 14(8): 967-977.

Patrick, D. L., et al. (2011). Content Validity-Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 2-Assessing Respondent Understanding. Value Health 2011; 14(8): 978-988.

Students Presenting

Jordy Laks o Temel JS, Greer JA, El-Jawahri A, Pirl WF, Park ER, Jackson VA,

Back AL, Kamdar M, Jacobsen J, Chittenden EH, Rinaldi SP, Gallagher ER, Eusebio JR, Li Z, Muzikansky A, Ryan DP. Effects of Early Integrated Palliative Care in Patients With Lung and GI Cancer: A Randomized Clinical Trial. J Clin Oncol. 2016 Dec 28:JCO2016705046. [Epub ahead of print]

Jenny Spencer o Souza, Jonas A., et al. "The development of a financial toxicity

patient‐reported outcome in cancer: The COST measure." Cancer 120.20 (2014): 3245-3253.

PRO Measurement project

Bryce give overview

Meet in teams

4

Feb 7 Topic: Quantitative Methods to Evaluate a PRO Measure (Part 1)

Assignment due:

Submit to Bryce by email (bbreeve@email.unc.edu) before start of class your one page term paper topic overview.

Readings: Fabrigar LR, Wegener DT, MacCallum RC, Strahan EJ. Evaluating the use of exploratory factor analysis in psychological research. Psychological Methods 1999;4(3):272-299.

Reeve BB, Stover AM, Alfano CM et al. The Piper Fatigue Scale-12 (PFS-12): psychometric findings and item reduction in a cohort of breast cancer survivors. Breast Cancer Research and Treatment 2012;136:9-20.

PRO Measurement project

Meet in teams

5

Feb 14 Topic: Quantitative Methods to Evaluate a PRO Measure (Part 2)

Readings: Edelen MO, Reeve BB. Applying item response theory (IRT) modeling to

questionnaire development, evaluation, and refinement. Quality of Life

Research 2007;16 Suppl 1:5-18.

Petrillo J, Cano SJ, McLeod LD, Coon CD. Using classical test theory, item response theory, and Rasch measurement theory to evaluate patient-reported outcome measures: a comparison of worked examples. Value in Health 2015;18(1):25-34.

o In Table 1, the order of the first row for the IRT and RMT were switched by mistake.

View IRT webinars by Karon Cook (see page 5 of syllabus for link)

Assignment due:

Go to PROMIS website (http://www.NIHpromis.org), click on tab “PROMIS For You” at top, then click on menu item, “PROMIS Demo”. Click on “Try a demonstration of the PROMIS CAT” and then select 3 or more domains and complete questionnaire. What do you think of measure and summary?

Students Presenting

Will Bayliss

Gracelyn Cruden

PRO Measurement project

Meet in teams

6

Feb 21 Topic: Methodological Issues in PRO Measurement

Guest Speaker:

Antonia Bennett, PhD Research Assistant Professor, HPM

Readings: Stull, D. E., et al. Optimal recall periods for patient-reported outcomes: challenges and potential solutions. Curr Med Res Opin 2009; 25(4): 929-942.

Barclay-Goddard R, Epstein JD, Mayo NE. Response shift: a brief overview and proposed research priorities. Quality of Life Research 2009 18:335-346.

Eremenco SL, Cella D, Arnold BJ. A comprehensive method for the translation and cross-cultural validation of health status questionnaires. Evaluation & the Health Professions 2005;28(2):212-232.

Online 18 minute video: http://www.isuma.tv/en/arnaitvideo/charlie-pisuk (and read the comments section)

Students Presenting

Effie Wei

Everett Young

Module: PROs in Clinical Research (Observational Studies, Comparative Effectiveness Research, Clinical Trials, Population Surveillance)

7

Feb 28 Topic: Best practices for integrating PROs in clinical research (Part 1)

Readings: Jensen RE, Moinpour CM, Fairclough DL. Assessing health-related quality of life in cancer trials. Clinical Investigation 2012;2(6):563-577.

CMTP’s Effectiveness Guidance Document on Recommendations for Incorporating Patient-Reported Outcomes in Clinical Comparative Effectiveness Research (CER) in Adult Oncology http://www.cmtpnet.org/wp-content/plugins/download-monitor/download.php?id=62

PRO Measurement project

Present findings

8

Mar 7 Topic: Best practices for integrating PROs in clinical research (Part 2)

Readings: Mullins CD, Abdulhalim AM, Lavallee DC. Continuous patient engagement in comparative effectiveness research. JAMA 2012;307(15):1587-1588.

Calvert M, Blazeby J, Altman DG, Revicki DA, Moher D, Brundage MD. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA. 2013;309(8):814-822.

Students Presenting

Anagha Gogate

Herodes Guzman

9

Mar 21 Topic: PRO Research in a Regulatory Setting

Guest Speaker:

Wen-Hung Chen, PhD Social Science Analyst, Food and Drug Administration (FDA) Lori McLeod, PhD Director of Psychometrics, RTI Health Solutions

Readings: FDA’s Guidance for Industry: Patient-Reported Outcomes Measures: Use in Medical Product Development to Support Labeling Claims: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf

FDA’s Guidance for Industry: Qualification Process for Drug Development Tools: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf

Fehnel S, DeMuro C, McLeod L, Coon C, Gnanasakthy A. US FDA patient-reported outcome guidance: great expectations and unintended consequences. Expert Reviews in Pharmacoeconomics and Outcomes Research. 2013;13(4):441-446.

Students Presenting

Alecia Slade

Chris Iskander

Module: PROs for Improving Quality of Care

10

Mar 28 Topic: Best practices for integrating PROs in healthcare delivery settings

Readings: Snyder CF, Aaronson NK, Choucair AK, Elliott TE, Greenhalgh J, Halyard MY, Hess R, Miller DM, Reeve BB, Santana M. Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations. Quality of Life Research. 2012;21:1305-1314.

Abernethy AP, Etheredge LM, Ganz PA, Wallace P, German RR, Neti C, Bach PB, Murphy SB. Rapid-learning system for cancer care. JCO 2010;28(27):4268-4274.

Students Presenting

Eric Tran

Alex Gertner

11

Apr 4

Topic: Using PROs as performance measures for assessing quality of care.

Guest Speaker: 3:15-4:45pm

Ethan Basch, MD, MSc Director, Cancer Outcomes Research Program Professor, School of Medicine and School of Public Health, UNC Angela Stover, PhD Post-Doc, Cancer Outcomes Research Program

Readings: Basch E, Torda P, Adams K. Standards for patient-reported outcome-based performance measures. JAMA 2013;310(2):139-140.

Stover AM, Basch E. Using Patient-Reported Outcome Measures as Quality Indicators in Routine Cancer Care. Cancer 2016; 122(3): 355-357.

Casalino LP, Gans D, Weber R, et al. US Physician Practices Spend More Than $15.4 Billion Annually To Report Quality Measures. Health Affairs. 2016 35(3): 401–406. doi: 10.1377/hlthaff.2015.1258

Watch TED talk: Stefan Larsson: What doctors can learn from each other: http://www.ted.com/talks/stefan_larsson_what_doctors_can_learn_from_each_other.html?utm_source=newsletter_daily&utm_campaign=daily&utm_medium=email&utm_content=button__2013-11-14

Students Presenting (2-3:15)

Neela Kumar

Garrett Thompson

12

Apr 11 Topic: Best practices for integrating PROs in healthcare delivery settings (II)

Readings: Cleeland CS, Wang XS, Shi Q, Mendoza TR, Wright SL, Berry MD, Malveaux D, Shah PK, Gning I, Hofstetter WL, Putnam JB, Vaporciyan AA. Automated symptom alerts reduce postoperative symptom severity after cancer surgery: a randomized controlled clinical trial. Journal of Clinical Oncology 2011;29(8):994-1000.

Basch E, Deal AM, Kris MG, et al. Symptom monitoring with patient-reported outcomes during routine cancer treatment: a randomized controlled trial. Journal of Clinical Oncology 2016;34(6):557-567.

Student Project Presentations

13

Apr 18

Student Project Presentations (15 minutes total per person)

1. 2. 3. 4. 5. 6. 7.

14

Apr 25

Student Project Presentations (15 minutes total per person)

1. 2. 3. 4. 5. 6. 7.