PCORI Funding Opportunity

Pragmatic Clinical Studies (PCS) to Evaluate Patient-Centered Outcomes – Cycle 2 2021

Letter of Intent (LOI) due June 1 – $10 million

Highlights

• $90 million in total available funding for clinical trials comprising comparative effectiveness research
• Up to $10 million in direct costs available per application, including a reasonable budget for coverage of patient care costs
• Total project period of up to five years
• The full PCORI Funding Announcement (PFA) will be posted on May 4
• Town Hall webinar on May 21, from 11:30am-1:00pm ET
• Letters of Intent (LOI) are due Tuesday, June 1, by 5:00pm ET
• Full applications will be due Tuesday, August 31, by 5:00pm ET

Overview

The Patient-Centered Outcomes Research Institute (PCORI) has announced funding for Pragmatic Clinical Studies to Evaluate Patient-Centered Outcomes. This PFA emphasizes relevant Methodology Standards and the PCORI Guidance on the Design and Conduct of Trials in Real-World Settings. Full details about the PFA will be available on the website May 4.

A total of $90 million in funding is available through this PFA. Investigators may request up to $10 million in direct costs for a clinical trial lasting up to five years.

Below are details on applying for PCORI funding, which may differ in some respects from applying to NIH or other funding sources. Interested potential applicants can request additional information from Leslie MacGregor, PhD, VMD, JD, Harvard Catalyst PCORI funding specialist.

The opportunity will be repeated once more in 2021. Cycle 3 opens September 7.

Funding Opportunity Details

PCORI seeks to fund clinical trials, large simple trials, or large-scale observational studies that compare two or more alternatives for addressing prevention, diagnosis, treatment, or management of a disease or symptom; improving healthcare system-level approaches to managing care; or eliminating health or healthcare disparities. Randomized study designs are strongly encouraged but not required.

Proposed studies must address critical clinical choices faced by patients, their caregivers, clinicians, or delivery systems. They must involve broadly representative patient populations and be large enough to provide precise estimates of hypothesized effectiveness differences and to support evaluation of potential differences in treatment effectiveness in patient subgroups.

How to Apply

To apply for this funding opportunity, there are four steps you must follow:

1. Read the full PFA for the Cycle 2 announcement on May 4
2. Follow the process outlined in the submission instructions
3. Submit a Letter of Intent, using the template to be posted for this PFA on May 4
4. Submit an application (if invited)

To prepare prior to the funding announcement on May 4:

• Read the full funding announcement for the Cycle 1 2021 PCS PFA
• Review the LOI template for Cycle 1 2021 PCS PFA, under “Applicant Resources” and review resources on PCORI policies, including patient engagement
• Watch the Cycle 1 2021 PCS PFA Town Hall and review the slides
• Review resources to guide applicants through the LOI and full application process from Cycle 1 2021 PCS PFA

The Letter of Intent (LOI) is typically three pages long (in addition to references) and utilizes a template that requires short paragraph responses to headers. It is essentially an abstract of the full research plan. The LOI deadline for this PFA is June 1, 2021 by 5:00pm ET. All templates and instructions will be provided with the funding announcement link on and after May 4, 2021.

About three weeks later, PCORI will invite applicants who have submitted LOIs deemed fully responsive to submit full applications. Those invited to proceed have until 5:00pm ET on August 31, 2021 to file full applications. The full application must be consistent with the plan set out in the LOI.

Assistance with your LOI

PCORI staff are accessible and extremely helpful. Contact them by emailing pfa@PCORI.org for administrative questions.

They strongly encourage applicants to make an appointment to speak to the program officer about the proposed research questions and approach prior to submitting their LOI and application. Ask to make a phone appointment with the program officer for this PCS PFA via email to science@pcori.org. It is highly advisable to do this soon sooner rather than later and not to wait until near the LOI deadline. If possible, email the program officer before your phone appointment and send a summary including information about the proposed research plan, team, and plan for engaging patients. The program officer will coach you about exactly what they seek in the LOI and plan and make suggestions as to the formation of your team.

Lastly, there is a Town Hall webinar on May 21, 2021 from 11:30am-1:00pm ET, when PCORI will provide an overview and information that applicants may need to submit a responsive application. Since that is less than two weeks before the LOI deadline, you might want to watch the Town Hall webinar and review the slides from the cycle 1 PFA.

Constructing a Successful LOI

It is critical to assemble a research team with expertise in conducting large clinical trials as intended for funding under this PFA. There can be a co-PI in addition to the lead PI. While junior investigators can apply for this PFA as lead PI, they would strengthen their application by having a co-PI who is a senior clinical researcher with significant experience in conducting past large multisite trials focused on the condition being studied. PCORI values large data sets that allow for power in subgroup analysis, such as by ethnicity, education, age, income, urban/rural, etc. Multisite studies frequently are required to ensure diversity within the patient subject population. In such cases, the study team includes a co-investigator at each of the sites other than that of the lead PI. The study team usually includes statistician(s) and others with appropriate expertise. At the time of submitting the LOI, you do not have to have the entire research team in place.

To be considered for PCORI funding, researchers must partner with patients and other stakeholders, beginning during the planning stages and continuing through the final dissemination of findings. Evidence of including patient partners must be demonstrated in the research plan of the LOI for it to be considered responsive. While applicants are not required to demonstrate that patient partners and stakeholders are already engaged at the time the LOI is submitted, it would be beneficial. Applicants should briefly describe in the LOI how patients and stakeholders will participate as partners in the various phases of the proposed research, once funded. Applicants should describe their plan to form a Patient and Stakeholder Advisory Board (PSAB), or other appropriate engagement body, to ensure that patients with the condition being studied and stakeholders, as defined below, advise and assist the research team with refining the study questions, outcomes, and protocols.

Ideally the PSAB would be convened in the early stages of preparing the LOI, especially for getting patient input in selection of study outcomes and outcome measurement instruments. Typically, a PSAB (or equivalent engagement body) is a group of about 10 to 15, of which at least half are “patients.” In referring to “patients,” PCORI includes not just individuals with the condition, but also patient family members and caregivers, as well as members of patient advocacy groups. The rest of the advisory board are “stakeholders,” which means people with professional expertise and interest related to this condition. This group includes non-researcher clinicians (physicians, nurses, mental health providers, PTs, social workers, pharmacists, etc.), as well as others, such as representatives from pharmaceutical companies, health insurers, and policy makers, as appropriate for the studied condition and patient population.

Please note: The PCORI Cost Principles state that PCORI will not cover costs for study interventions that constitute the procedures, treatments, interventions, or other standard clinical care (“patient care”) that is being proposed for comparison in the research project (“patient care costs”).

However, for this funding cycle of Pragmatic Clinical Studies, PCORI will pilot consideration of funding support for meritorious applications providing well-supported and justified requests to cover patient care costs defined to include the intervention being studied as well as clinical personnel costs for those providing the care. For additional information on the conditions for such coverage, see the Coverage of Intervention Costs section in the funding announcement. Applicants should submit a realistic budget and timeline reflecting the proposed study’s scope and requirements.

The PCORI website will provide resources to guide applicants through the LOI and full application process for the Cycle 2 2021 PFA on May 4. Prior to that date, you may review the resources provided for Cycle 1 2021 PFA.

Contact Information

For questions about the PCORI application process or PCORI in general that are not addressed above or on the PCORI website (www.pcori.org), please contact Leslie MacGregor, PhD, VMD, JD, leslie.macgregor@hms.harvard.edu, PCORI Funding Specialist.

Harvard Catalyst would appreciate your informing us when you apply for PCORI funding, as well as their response, so we can better develop helpful resources to support future applications by HMS affiliated researchers.

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) was authorized by Congress in 2010 as a nonprofit, nongovernmental organization. PCORI’s purpose, as defined by its authorizing legislation, is to help patients, caregivers, clinicians, policy makers, and other healthcare system stakeholders make better-informed health decisions by “advancing the quality and relevance of evidence about how to prevent, diagnose, treat, monitor, and manage diseases, disorders, and other health conditions” and by promoting the dissemination and uptake of this evidence.