Advancing Patient Centered Outcomes Research (PCOR): Implications for Research Methods and Standard Disclosures Terminology Dr. Robin Newhouse has disclosed a relevant financial interest Second International Conference on Research Methods with Patient Centered Outcomes Research Institute (PCORI) and for Standardized Technologies Getwell Network to the learners of this activity. There are no conflicts of interest or relevant financial interests Robin P. Newhouse, PhD, RN, NEA-BC, FAAN that have been disclosed by the remaining presenters or planners University of Maryland Baltimore, School of Nursing of this activity that apply to this learning session. April 15, 2015 This conference is sponsored by the University of Minnesota Center for Nursing Informatics Outline Objective Emerging Trends Infrastructure Engagement Discuss patient-centered outcomes research and Training Data Harmonization (PCOR)and implications for standardized PROs PROEM PCORI terminologies PATIENTS PCORI Patients Standards PCORnet Key Points • Standard terminology is essential to for learning health care systems • Patient and stakeholder engagement are PCORI Methodology Standards central to developing standard terminology for patient-centered outcomes research Robin Newhouse is Chair of the Methodology Committee of the • Patient reported outcomes should be Patient Centered Outcomes Research Institute (PCORI). The views incorporated into Electronic Health Records expressed in this presentation are those of the authors and not necessarily those of PCORI. • Standard terminology should be disseminated and used in health systems 1
We Fund Research That… We Focus Attention On… Conditions Studies the benefits and harms of • Conditions that affect large numbers of interventions and strategies delivered in real- people across a range of populations world settings • Conditions that place a heavy burden on individuals, families, specific populations, and Compares at least two alternative approaches society Adheres to PCORI’s Methodology Standards • Rare diseases, which are difficult to study Is based on health outcomes that are Populations meaningful to the patient population • Racial and ethnic minorities Engages patients and other stakeholders at • Older adults every stage • Low-income and rural populations Is likely to improve current clinical practices • Children The Research We Fund is Guided by Our We Target Specific, High-Priority Topics National Priorities for Research Assessment of Prevention, Diagnosis and Treatment Options • PCOR Treatment Options in Uterine Fibroids* Improving Healthcare Systems Assessment of Communication & • Clinical Trial of a Multifactorial Fall Injury Prevention Strategy in Older Improving Prevention, Diagnosis, Dissemination and Treatment Options Healthcare Systems Persons** Research • Effectiveness of Transitional Care Addressing Disparities • Treatment Options for African Americans and Hispanics/Latinos with Uncontrolled Asthma • Obesity Treatment Options Set in Primary Care for Underserved Accelerating PCOR Populations Addressing and Methodological Disparities • Clinical Interventions to Address Hypertension Disparities Research * Administered by AHRQ ** Administered by the National Institute on Aging We Follow a Unique Proposal Review We Work to Improve Research Methodology Process In any study, methods matter. That’s why we’ve developed Applications are reviewed against five criteria: methodology standards that all research should follow, at a minimum. Impact of the condition on the • Applications are reviewed Methodology Standards: 11 Broad Categories health of individuals/populations by a panel of two • Data Networks scientists, one patient, • Formulating Research Questions Potential for the study to and one other • Data Registries • Patient-Centeredness improve healthcare and stakeholder. • Adaptive and Bayesian • Data Integrity and Rigorous outcomes • PCORI’s Board of Trial Designs Analyses Governors makes funding Technical merit • Causal Inference decisions based on merit • Preventing/Handling Missing review and staff Patient-centeredness Data • Studies of Diagnostic recommendations . Tests • Heterogeneity of Treatment Patient and stakeholder • Systematic Reviews engagement Effects 2
Role of the Standards in PCORI Applications Methodology Standards and Proposals The Methodology Standards and report are tools “In the Research Strategy section of the Research Plan for applicants in preparing their applications. Template, you should describe in detail your research strategy and plan, and you should demonstrate how Applications must demonstrate adherence to your proposed study is responsive to the PFA. Include PCORI’s Methodology Standards. the relevant PCORI Methodology Standard citations Not all standards apply to all studies. (e.g., “PC - 3”) as identified in the Methodology Report; The standards do NOT dictate specific study you should refer to this report for an explanation on designs. each standard. You do not need to list every standard, The Methodology Standards map to PCORI’s merit only standards that are relevant to your proposal .” review criteria. PCORI technical reviewers assess the See http://www.pcori.org/content/faqs- methodological rigor of each study’s methods. applicants#WHAT%20TO%20INCLUDE The Standards Help to Craft a Research The Standards Guide Development of Question Patient-Centered Projects RQ-1 Identify gaps in evidence Gap analysis and systematic reviews should be used to support the need for a proposed study. If PC-2 Identify, select, recruit, and retain study a systematic review is not available, a systematic review should be performed using accepted participants representative of the spectrum of the standards in the field (see standard SR-1), or a strong rationale should be presented for proceeding without a systematic review. In the case where a systematic review is not possible, population of interest and ensure that data are the methods used to review the literature should be explained and justified. collected thoroughly and systematically from all RQ-6 Measure outcomes that people representing the population of study participants interest notice and care about Identify and include outcomes the population of interest notices and cares about (e.g., survival, PC-3 Use patient-reported outcomes when function, symptoms, health-related quality of life) and that inform an identified health decision. Define outcomes clearly, especially for complex conditions or outcomes that may not have patients or people at risk of a condition are the best established clinical criteria. Provide information that supports the selection of outcomes as source of information meeting the criteria of “patient - centered,” and “relevant to decision makers” such as patient and decision maker input from meetings, surveys, or published studies. Select outcomes based on input directly elicited from patient informants, people representative of the population of interest, either in previous studies or in the proposed research. Stories Highlight Important Methods Issues The Standards Address Missing Data MD-1 Describe methods to prevent and The stories are not monitor missing data intended to endorse specific research MD-3 Use validated methods to deal with approaches; they missing data that properly account for demonstrate that good statistical uncertainty due to missingness methods make a difference. 18 3
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