1. Comparative Effectiveness Research (CER) and Patient- Centered Outcomes Research (PCOR) Presentation Developed for the Academy of Managed Care Pharmacy Updated: February 2016

2. Topics Covered Background Definitions Goals of CER and PCOR Stakeholders Funding sources Challenges CER study design and user tools

3. Background Early draft of the Affordable Care Act (ACA) included provisions for comparative effectiveness research (CER) – “CER” changed to “Patient-Centered Outcomes Research” (PCOR) in legislation based on voter input – Many entities view terms as interchangeable; however, the definitions do differ ACA established a Patient-Centered Outcomes Research Institute (PCORI) – Funded through the general fund of the US treasury and in small part by assessments on Medicare, private health insurance, and self-insured plans

4. Definitions: CER CER Includes any type of medical or pharmaceutical intervention (e.g. lab tests, procedures, medications, etc.) Institutes of Medicine (IOM) definition: generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care Kaiser Family Foundation definition: compares two or more different methods for preventing, diagnosing, and treating health conditions. Such research is performed using methods such as practical clinical trials, analyses of claims records, computer modeling, and systematic reviews of existing literature CER and PCOR are not synonymous

5. Definitions: PCOR Assesses the benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions to inform decision making, highlighting comparisons and outcomes that matter to people; Is inclusive of an individual’s preferences, autonomy and needs, focusing on outcomes that people notice and care about such as survival, function, symptoms, and health related quality of life; Incorporates a wide variety of settings and diversity of participants to address individual differences and barriers to implementation and dissemination; and Investigates (or may investigate) optimizing outcomes while addressing burden to individuals, availability of services, technology, and personnel, and other stakeholder perspectives ACA established a Patient-Centered Outcomes Research Institute (PCORI) Funded through the general fund of the US treasury and in small part by assessments on Medicare, private health insurance, and self-insured plans PCORI’s working definition states that PCOR:

6. Goals of CER and PCOR CER: IOM: assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels Kaiser Family Foundation: improve health outcomes by developing and disseminating evidence based information to patients, providers, and health care decision-makers about the effectiveness of treatments relative to other options. Identifying the most effective and efficient interventions has the potential to reduce unnecessary treatments, which in turn, may help lower costs PCOR: PCORI: help people and their caregivers communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options CER and PCOR definitions differ, but goals are similar

7. Stakeholders Patients: Understand risks/benefits of treatment options Make informed healthcare decisions Providers: Make informed treatment recommendations Facilitate patient- shared decision- making Managed care organizations: Support benefit/formulary changes Educate members and providers United States Healthcare System Improve health of U.S. population Decrease variability in clinical practice across the country Decrease healthcare costs

8. Funding Sources – To date, most CER and PCOR projects have been funded through grants PCORI – Awards research grants for PCOR from federal funds US Department of Health & Human Services (HHS) – Agency for Healthcare Research and Quality (AHRQ) – Funding to train researchers to conduct CER and PCOR has been provided by The PhRMA Foundation

9. Challenges CER and PCOR require special skill sets: – Researchers Focus on new approaches to research to inform decision making by patients and caregivers – Educators Training researches to conduct CER and PCOR – Clinical decision-makers and Assessing the validity and applicability of CER and PCOR output Interpreting and applying the resulting body of evidence – Policy-makers Establish policy based on a body of evidence that is valid and applicable to their population – Payers Make payment decisions based on the most effective treatment options based on individual patient characteristics

10. CER Study Design and User Tools Study design: – Individual studies: Prospective randomized or observational trials Retrospective observational trials Indirect treatment comparisons Network meta-analyses Modeling studies – Evaluate the overall body of evidence User tools: – AMCP/ISPOR/NPC collaborative: www.cercollaborative.orgwww.cercollaborative.org – AMCP CER Certificate Program

11. CER Certificate Program Advance evidence assessment expertise with new research designs and study data Learn about tools to help assess current questions in real-world settings Developed by the CER Collaborative Certificate Program Details – 19 Contact Hours – Five - Online Modules – One – 4 hour workshop – with live presentations at one of AMCP’s national meetings – Student Fee $400 – Additional information: http://www.amcp.org/CERCertificate/ http://www.amcp.org/CERCertificate/ Modules Introduction to Comparative Effectiveness Research Value of Prospective & Retrospective Observational Studies in Comparative Effectiveness Research Modeling Studies Indirect Treatment Comparisons Synthesizing a Body of Evidence in Comparative Effectiveness Demonstrating Skills in Assessing CER Studies and Synthesizing CER Literature

12. References 1.Patient-Centered Outcomes Research Institute (website). Accessed on: February 23, 2016. Available at: http://www.pcori.org/ http://www.pcori.org/ 2.Selby JV, Beal AC, Frank L. The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda. JAMA. 2012 Apr;307(15):1583-4. 3.Institutes of Medicine. Initial national priorities for comparative effectiveness research. June 30, 2009. Accessed on: February 23, 2016. Available at: http://www.iom.edu/Reports/2009/ComparativeEffectivenes sResearchPriorities.aspx http://www.iom.edu/Reports/2009/ComparativeEffectivenes sResearchPriorities.aspx 4.The Henry J. Kaiser Family foundation. Explaining health reform: what is comparative effectiveness research? September 29, 2009. Accessed on: February 23, 2016. Available at: http://kff.org/health-costs/issue- brief/explaining-health-care-reform-what-is-comparative/http://kff.org/health-costs/issue- brief/explaining-health-care-reform-what-is-comparative/

13. Thank you to AMCP members Carly Rodriguez and Cheryl Kaltz for creating this slide deck. Thank you to AMCP member Melissa McCart for updating this presentation for 2016.