Introduction

PCORI’s mission is to produce knowledge that allows individuals to make informed health care decisions and that improves health care delivery and outcomes. The 2019 law reauthorizing PCORI’s funding includes a provision clarifying that PCORI-funded research should consider, as appropriate, the full range of outcomes data, including potential burdens and economic impacts. Consistent with this mandate, PCORI adopted Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research (PCORI Principles).

PCORI seeks investigator-initiated requests to build on existing, PCORI-funded comparative clinical effectiveness research (CER) studies to improve the understanding of appropriate costs and other economic burdens that are meaningful to and inclusive of patients, caregivers and other stakeholders.

Only PCORI-funded principal investigators/institutions with active CER project funding contracts are eligible to submit a request in response to this opportunity.

Purpose

The aim of this supplemental funding opportunity is to produce knowledge on potential burdens and economic impacts that will support patients and other stakeholders in making informed health care decisions that account for such impacts. PCORI is seeking requests from awardees engaged in advancing patient-centered care who can demonstrate capability of collecting and analyzing credible data on costs and other economic burdens, using rigorous, established methodologies. Requests must include plans to collect and analyze data from the following required area of interest:

• Costs and other economic burdens patients and/or caregivers incur (e.g., time away from usual activities, out-of-pocket costs, time spent caregiving, changes in health care utilization, perceived financial toxicity)

In addition to the above required area of interest, PCORI encourages awardees to also include plans to collect and analyze data on:

• Costs incurred by stakeholders besides patients or caregivers such as clinicians, health systems, employers, purchasers and payers (e.g., reimbursements to patients from their insurer, absenteeism and loss of workplace productivity, changes in health care utilization)

Analysis should synthesize findings of costs and other economic burdens from the perspectives of the different stakeholders for whom such data are proposed to be collected as part of the supplement. PCORI is particularly interested in the consideration of the impact of social risk factors and/or social determinants of health on costs and other economic burdens incurred by patients, caregivers and other stakeholders. Requests must include information on the proposed teams and their experience and qualifications in the collection and analysis of the data being proposed for this supplement. Requests must also include plans to produce meaningful knowledge on costs and other economic burdens that will support informed health care decision-making that accounts for such impacts.

Please note that PCORI’s authorizing law, as amended by the 2019 legislation reauthorizing PCORI’s funding, includes the following prohibitions: (1) development or employment of dollars-per-quality-adjusted life year (QALY) thresholds; (2) mandating coverage, reimbursement or other policies for any public or private payer, and prohibits PCORI-funded research from including practice guidelines, coverage recommendations, payment or policy recommendations; (3) funding the conduct of total cost comparisons, cost-effectiveness analysis (CEA) or related types of economic evaluation. PCORI does not fund studies for which cost and economic impacts are the primary outcome.

Requests focused on QALY methods development or employment, development of practice guidelines, coverage recommendations, payment or policy recommendations or the conduct of total cost comparison, CEA or related types of economic evaluation will be considered nonresponsive to this opportunity and will be declined. Analyses that focus on a cost comparison to determine a preferred intervention will also be considered nonresponsive.

Recently, PCORI engaged patients and the broader health care community to identify attributes of patient-centered value. The following attributes, are intended to help inform PCORI investigators and the broader community, and should not be construed as prescribed research areas or outcomes; rather, investigators can consider them when proposing work under this supplement:

• Financial impacts and barriers can reflect the cost data to be collected.
• Clinical factors and patient characteristics can be incorporated to describe heterogeneity in patient-centered economic outcomes.
• Access and social impacts can be incorporated to include social and structural determinants of health and access to care.

Awardees are encouraged to think about analysis that explores these factors in some depth while avoiding aggregating or comparing total costs of care.

Eligibility

• Awarded contracts that were funded under PCORI Funding Announcements (PFAs) issued in Cycle 1 2019 or later.
• Projects must have submitted at least one Interim Progress Report (IPR) by June 1, 2023, and have the Final Progress Report (FPR) due on October 1, 2024, or later.
• The current CER project must be a multi-site study.
• Project is deemed by PCORI staff to be in good standing. Metrics for assessment include:
  • On-time completion of project milestones to date
  • Cumulative enrollment progress (if the project includes prospective enrollment of participants and is anticipated to have started enrollment by the supplement application receipt date)
• Project has not received a cumulative extension of greater than 18 months to the original executed contract’s research period of performance end date (i.e., the FPR due date).
• Phased Large Awards for Comparative Effectiveness Research (PLACER) currently in the Feasibility Phase are not eligible.
• Eligible awardees may apply for either this supplemental funding opportunity or the Supplemental Funding for Existing PCORI-funded Comparative Clinical Effectiveness Research to Increase Diversity and/or Advance Health Equity opportunity, not both.
• Awardees that received a prior award under the PCORI COVID-19 enhancement mechanism are eligible to apply.

Specifications

Supplements for all projects should seek to leverage existing project personnel expertise and infrastructure when possible. At least one core member of the research team must have demonstrated experience with cost data and analysis. Additional personnel may be necessary to ensure there is appropriate expertise on the research team. Requests for supplement funding should provide evidence that existing project stakeholders support the proposed supplement. Proposed supplements must adhere to the PCORI Methodology Standards. A detailed report of all work completed as part of the supplement will be included in the original award’s Draft Final Research Report (DFRR).

Review and Approval Process

Following submission, PCORI will complete review of each request. You may be contacted by your assigned Program Officer (PO) for additional information as part of PCORI’s review.

Determinations will be made using the review criteria below:

1. Demonstrated usefulness of the acquired economic knowledge to assist patients and stakeholders in making informed health care decisions
2. Methodological rigor of proposed costs and other economic burden data collection and analyses, e.g., use of established methods
3. Appropriate expertise on the research team
4. Demonstrated patient and stakeholder interest through engagement in selection, and affirmation of importance, of patient-centered economic outcomes to be collected
5. Requested budget amount commensurate with scope of proposed supplement
6. Feasibility of collection and analysis of proposed additional measures in the current study
7. Currently funded award is in good standing and no anticipated negative impact of the supplement on success of the original award

Key Dates