PCORI Methodology Standards: Categories 3, 4, & 5 — Data Integrity, Preventing and Handling Missing Data, and Heterogeneity of Treatment Effects

This CME activity has expired. You may still view the program content, however, you will not be able to obtain CME/CE credit.

CME/CE Information

Course Overview

The Patient Protection and Affordable Care Act of 2010 provided the authorizing language to create the Patient-Centered Outcomes Research Institute (PCORI). PCORI is tasked with producing comparative effectiveness research (CER) that is guided by patients, caregivers, and the broader health care community to assist people in making informed healthcare decisions and to improve healthcare delivery and outcomes.1

In authorizing PCORI, Congress recognized that it was essential that PCORI base its research program on rigorous scientific methods. To that end Congress directed PCORI to develop methodology standards that “…provide specific criteria for internal validity, generalizability, feasibility, and timeliness of research and for health outcomes measures, risk adjustment, and other relevant aspects of research and assessment with respect to the design of research.”2

While PCORI’s methodology standards do not address all potential issues in clinical research, they are broad, span 11 categories with 47 standards, and were selected to target areas where: “…1) there were either substantial deficiencies or inconsistencies in how available methods were applied in practice, despite specialized knowledge in how best to conduct research; or 2) there were barriers to the effective dissemination of research results.” As a result these standards offer investigators guidance in the design and conduct of patient-centered outcomes research (PCOR) and for those seeking funding from PCORI provide the minimal quality benchmarks that must be met in responding to PCORI funding announcements.1

This educational activity is one of a series of continuing medical education (CME)/continuing education (CE) modules or activities designed to introduce PCORI’s Methodology Standards, review the development and types of standards, and provide context and guidance for adherence relevant to the use and application of these standards in research and in responding to PCORI funding announcements.

This module is designed to provide investigators, clinicians interested in CER investigation, and other learners with an understanding of three important cross-cutting standards for CER. The Standards for Data Integrity & Rigorous Analyses address the need for investigators (once they have selected their study design and analytical approach) to report on the data accuracy, availability, validity, and study decision. The Standards for Preventing and Handling Missing Data address the fact that virtually every study will have some missing data and investigators need to have plans to minimize the missing data, how to handle missing data in the subsequent analyses, and how to report those findings in the interpretation of the study results. And finally, the rationale for the Standards for Heterogeneity of Treatment Effects relate to the fact that the effectiveness of the treatment may actually vary depending on patient characteristics, and thus by definition each heterogeneity of treatment effect implies that different people do not respond the same way to the same treatment or exposure. These standards are important in helping to answer the patient-centered question, “What is likely to happen to patients like me?”

Other Modules in This Series

Module 1: PCORI Methodology Standards: Basic Context, Role, and Development of Standards in Comparative Effectiveness Research

Module 2: PCORI Methodology Standards: Role of Standards in PCORI Funding Announcements and the Application Process

Module 3: PCORI Methodology Standards: Standards Categories 1 & 2 — Formulating Research Questions and Patient Centeredness

Module 5: PCORI Methodology Standards: Categories 6, 7, and 8 — Data Registries, Data Networks, and Causal Inference

Module 6: Standards for Specific Study Designs: Standard Groups 9 and 10 — Adaptive and Bayesian Trials Designs and Studies of Diagnostic Tests

1 Research Methodology. (n.d.). Retrieved from Patient-Centered Outcomes Research Institute:

2 Extracted sections concerning Patient-Centered Outcomes Research and the Authorization of the Patient-Centered Outcomes Research Institute (PCORI). (n.d.). Retrieved from Patient-Centered Outcomes Research:

Educational Objectives

At the conclusion of this activity, the participant should be able to:

  • Identify the types of approaches for handling missing data in studies.
  • State the strengths and limitations of the approaches for handling missing data in studies.
  • Define heterogeneity of treatment effects.
  • Describe why heterogeneity of treatment effects is important in interpreting data from CER studies.

Target Audience

This online enduring material is designed to meet the educational needs of researchers and/or those clinicians and junior faculty interested in applying for PCORI funding, participating in research, or using PCORI research. This audience may include physicians, nurses, pharmacists, and physician assistants.

Method of Participation

This activity is in streaming video and in text/slide formats. References with links to online resources are also provided.

To obtain CME/CE credit you should:

  • View the presentations in this enduring material.
  • Complete the posttest.
  • Complete and submit the registration and evaluation forms.

The estimated time to complete this activity, including review of the materials, is 0.50 hours.

Fee: There is no fee for this activity.

Hardware/software requirements: A computer with broadband internet access and speakers is required. Activities should be run with recent versions of common browsers, including Internet Explorer, Firefox, and Google Chrome.

If you have questions about the participation process, please e-mail the Office of Continuing Medical Education, or phone 713-798-8237.

Accreditation/Credit Designation


Baylor College of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Baylor College of Medicine designates this enduring material activity for a maximum of 0.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

American Academy of Family Physicians (AAFP)

This Enduring Material activity, Contemporary Treatment Options for Prostate Cancer, has been reviewed and is acceptable for up to 0.50 Prescribed credit(s) by the American Academy of Family Physicians. AAFP certification begins 06/26/2018. Term of approval is for one year from this date. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physician Maintenance of Certification (MOC)

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.25 Medical Knowledge MOC points in the American Board of Internal Medicine’s (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider’s responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

Physician Assistants

This program has been reviewed and is approved for a maximum of 0.50 hours of AAPA Category 1 CME credit by the Physician Assistant Review Panel. Approval is valid for one year from the issue date of October 1, 2016. Participants may submit the posttest at any time during that period.

This program was planned in accordance with AAPA’s CME Standards for Enduring Material Programs and for Commercial Support of Enduring Material Programs.

Successful completion of the posttest is required to earn Category 1 CME credit. Successful completion is defined as a cumulative score of at least 70 percent correct.


The University of Texas Houston Health Science Center at Houston School of Nursing is an approved provider of continuing nursing education by the Texas Nurses Association, an accredited approver by the American Nurses Credentialing Center’s Commission on Accreditation.

This course is approved for 0.50 contact hour(s) of Nursing Continuing Education. The course is sponsored by the Office of Continuing Nursing Education at The University of Texas Health Science Center at Houston School of Nursing.


Houston Methodist Hospital is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. Participants documenting activity completion by viewing the slides, passing the learning assessment, and completing an online evaluation within 45 days will receive 0.50 contact hours (0.050 CEUs) of continuing pharmacy education credit as a knowledge-based activity (UAN: 0478-0000-15-017-H04-P).

Term of Approval

Release Date: September 30, 2015 | Expiration Date: September 30, 2017

Peer Review

In May 2015, this continuing medical education online enduring material was reviewed by Gillian Sanders-Schmidler, Ph.D., Associate Professor, Duke University, Durham, North Carolina. To ensure the continued scientific relevance of this enduring material, its content will be reviewed again in September 2017.

Disclosure: Nothing to disclose.

Program Director

Mark Helfand, M.D., M.S., M.P.H.
Professor of Medicine
Professor of Medical Informatics and Clinical Epidemiology
Oregon Health & Science University
Portland, Oregon
Baylor College of Medicine
Houston, Texas

Disclosure: Nothing to disclose.


Michael Fordis, M.D.
Sr. Associate Dean and Director, Center for Collaborative and Interactive Technologies
Director, The John M. Eisenberg Center for Clinical Decisions and Communications Science
Baylor College of Medicine
Houston, Texas

Disclosure: Nothing to disclose.


This CME/CE activity is designed for use by healthcare professionals for educational purposes only. Information and opinion offered by the contributors represent their viewpoints. Conclusions drawn by the participant should be derived from careful consideration of all available scientific information. Prescription information and use of medical devices should be undertaken only after confirmation of information by consulting the FDA-approved uses and information.

Baylor College of Medicine makes every effort to have accurate information presented, no warranty, expressed or implied, is offered. The participant should use his/her clinical judgment, knowledge, experience, and diagnostic decision-making before applying any information, whether provided here or by others, for any professional use.

Links are provided to other Internet sites solely for the convenience of users. Once you link to another site, you are subject to the site's terms and conditions of use including copyright and licensing restrictions.


The Office of Continuing Medical Education (OCME) makes every effort to develop CME/CE activities that are scientifically based, accurate, current, and objectively presented. In accordance with the Accreditation Council for Continuing Medical Education Standards for Commercial Support℠, Baylor College of Medicine (BCM) has implemented a mechanism requiring everyone in a position to control the content of an educational activity (e.g., directors, planning committee members, contributors, peer reviewers) to disclose any relevant financial relationships with commercial interests (drug/device companies) and manage/resolve any conflicts of interest prior to the activity. Individuals must disclose to participants the existence or non-existence of financial relationships: 1) at the time of the activity or within 12 months prior; and 2) of their spouses/partners.

Baylor College of Medicine does not view the existence of interests or relationships with commercial entities as implying bias or decreasing the value of a presentation. It is up to the participants to determine whether the interests or relationships influence the presenter with regard to exposition or conclusions.

If at any time during this activity you feel that there has been commercial or promotional bias, please inform us by using the commercial bias comments box in the evaluation form. Please answer the questions about balance in the CME/CE activity evaluation candidly.

The following individual(s) has/have reported no financial or other relationships with commercial entities whose products/services may relate to the educational content of this activity:

Nancy I. Bergstrom, Ph.D., Nursing CE Planning Member
Trumble Professor of Aging Research and Associate Dean for Research
The University of Texas Health Science Center at Houston School of Nursing
Houston, Texas

Disclosure: Nothing to disclose.

Carl E. Fasser, B.A., P.A., Physician Assistant CE Planning Member
Professor and Director, Physician Assistant Program
Baylor College of Medicine
Houston, Texas

Disclosure: Nothing to disclose.

Vaunette P. Fay, Ph.D., R.N., Nursing CE Planning Member
Professor of Clinical Nursing
The University of Texas Health Science Center at Houston School of Nursing
Houston, Texas

Disclosure: Nothing to disclose.

Sharon-Lise Normand, Ph.D., Contributor
Professor, Health Care Policy (Biostatistics)
Department of Health Care Policy
Harvard Medical School
Professor, Department of Biostatistics
Harvard School of Public Health
Boston, Massachusetts

Disclosure: Nothing to disclose.

Anne N. Perch, M.B.A., Physician CME Planning Member
Senior Associate Director, CME
Baylor College of Medicine
Houston, Texas

Disclosure: Nothing to disclose.

Gillian Sanders-Schmidler, Ph.D., Peer Reviewer
Associate Professor
Duke University
Durham, North Carolina

Disclosures: Nothing to disclose.

Julie T. Won, Pharm.D., BCPS, Pharmacy CPE Planning Member
CPE Board Chair
Houston Methodist Hospital
Houston, Texas

Disclosure: Nothing to disclose.

The following individual(s) has/have reported financial or other relationship(s) with commercial entities whose products/services may relate to the educational content of this activity:

Mark Helfand, M.D., M.S., M.P.H., Contributor
Professor of Medicine
Professor of Medical Informatics and Clinical Epidemiology
Oregon Health & Science University
Portland, Oregon

Disclosure: Consultant for Consumer Reports Best Buy Drugs.

Jose R. Murillo, Pharm.D., M.H.A., M.B.A., Pharmacy CPE Planning Member
CPE Board Vice Chair
Houston Methodist Hospital
Houston, Texas

Disclosure: Advisory Committee Member: Genentech.

Some drugs/devices identified during this activity may have United States Food and Drug Administration (FDA) clearance for specific purposes only or for use in restricted research settings. The FDA has stated that it is the responsibility of the individual physician to determine the FDA status of each drug or device that he/she wishes to use in clinical practice and to use the products in compliance with the applicable law.

Baylor College of Medicine requires that all contributors disclose an unlabeled use or investigational use (not yet approved for any purpose) of pharmaceutical and medical device products, and provide adequate scientific and clinical justification for such use. Physicians are urged to fully review all the available data on products or procedures before using them to treat patients.

Acknowledgement of Support

This CME/CE activity is supported by a contract from the Patient-Centered Outcomes Research Institute.