November 13, 2020
Nakela L. Cook, MD, MPH
Patient-Centered Outcomes Research Institute (PCORI)
1828 L St NW, Suite 900
Washington, DC 20036
RE: Proposed Principles for the Consideration of the Full Range of Outcomes Data
Submitted electronically: https://www.pcori.org/webform/proposed-principles-consideration-full-range-outcomes-data-2020
Dear Dr. Cook,
The National Pharmaceutical Council (NPC) appreciates the opportunity to provide comments to PCORI on its Proposed Principles for the Consideration of the Full Range of Outcomes Data (Principles). 
NPC is a health policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. NPC is supported by the major U.S. research-based biopharmaceutical companies. We focus on research development, information dissemination, education and communication of the critical issues of evidence, innovation and the value of medicines for patients. Our research helps inform important health care policy debates and supports the achievement of the best patient outcomes in the most efficient way possible.
NPC supports PCORI’s new mandate to consider the full range of patient-centered outcomes in its research:
Research shall be designed, as appropriate, to take into account and capture the full range of clinical and patient-centered outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers in making informed health decisions. In addition to the relative health outcomes and clinical effectiveness, clinical and patient-centered outcomes shall include the potential burdens and economic impacts of the utilization of medical treatments, items, and services on different stakeholders and decision-makers respectively. These potential burdens and economic impacts include medical out-of-pocket costs, including health plan benefit and formulary design, nonmedical costs to the patient and family, including caregiving, effects on future costs of care, workplace productivity and absenteeism, and health care utilization. 
This new mandate further strengthens PCORI’s role in understanding and elucidating patient-centered value. The patient is at the epicenter of health value, yet many of the factors that patients care about are not meaningfully included in many value assessments. PCORI’s new mandate will help fill these gaps on behalf of patients, enabling a more comprehensive measurement of patient-centered value.
PCORI’s role is vital in a health system that seeks ever greater accountability for value demonstration and will lead to better-informed health care decision-making. With the new mandate, PCORI has the opportunity to make a broader range of patient-centered clinical- and economic- outcomes data available for use by researchers in value assessments and comparative effectiveness research studies. These data can include factors beyond clinical benefits and harms, such as work productivity, caregiver burden, and social determinants of health.
Please find our specific comments on the proposed Principles below.
Focus on holistically measuring what is missing and what matters to patients
PCORI should seek to fill data gaps to support the holistic measurement of patient-centered value. Robust data are frequently unavailable or limited for patient-centered factors such as workplace absenteeism and presenteeism, disability, caregiver burden, transportation, and many social determinants of health. PCORI has the opportunity to meaningfully fill in these gaps to form a complete picture of patient economic burden – including the impact of out-of-pocket expenses and deductibles – and outcomes.
Elevate the use of patient-reported measures
Patient-reported measures are tools to capture data directly from patients on their experiences and outcomes. They play an important role in integrating the patient voice into quality measurement and value-based payment programs. In an October 2020 manuscript, we recommended that payers engage in collaborative patient-centered measurement projects with PCORI.  Insight into caregiver-reported measures can also yield valuable research for integrating the patient voice and should be considered by PCORI in addition to patient-reported measures. We recommend that PCORI elevate data collection through the expansion of the sites and leverage PCORnet to include patient-reported measures that reflect patient experience in the real world.
Support the prohibition against cost-effectiveness analyses
NPC appreciates the explicit statement within the Principles of the prohibition against PCORI-funded research conducting specific economic analyses, including cost-effectiveness analyses, or aggregating findings on health outcomes with findings on economic impact. Although PCORI is unable to conduct such studies, the breadth of economic burden and outcomes data collected by PCORI will better inform non-PCORI analyses.
Clarify methodology standards
While the Principles will provide useful guidance to Spring 2021 PCORI research applicants seeking to interpret the new mandate and incorporate it into their research proposals, this is only a first step. Next, PCORI must establish clear methodologic standards, including detailed explanations and examples, to demonstrate what data and methods are in and out of bounds. We commend PCORI for planning this activity for the second phase of its Cost Data Implementation process.
We appreciate this opportunity to provide input on PCORI’s Proposed Principles for the Consideration of the Full Range of Outcomes Data. NPC’s continued engagement with PCORI signifies our shared commitment to using robust patient-centered evidence to help all stakeholders make informed decisions about health care treatments and services. We look forward to future opportunities to engage with PCORI on this and other significant topics.
Robert W. Dubois, MD, PhD
Interim President and CEO
Kimberly Westrich, MA
Vice President, Health Services Research
 Patient-Centered Outcomes Research Institute. 2020. Proposed Principles for the Consideration of the Full Range of Outcomes Data. Available at: https://www.pcori.org/sites/default/files/PCORI-Proposed-Principles-Consideration-Full-Range-Outcomes-Data.pdf
 Social Security Act. Section 1181 [42 U.S.C. 1320e] (d)(2)(F). Available at: https://www.ssa.gov/OP_Home/ssact/title11/1181.htm
 The National Pharmaceutical Council. (2020). Current Landscape: Value Assessment Frameworks. Available at: https://www.npcnow.org/publication/current-landscape-value-assessment-frameworks-0
 Schmidt T, Valuck T, Perkins B, et al. Improving patient-reported measures in oncology: a payer call to action. J Manag Care Pharm. Available at: https://www.jmcp.org/doi/pdf/10.18553/jmcp.2020.20313