Phased Large Awards for Comparative Effectiveness Research PCORI Funding Announcement (PFA) Applicant Town Hall July 14, 2020 12:00-1:30 PM EDT
Agenda • About PCORI • Funding Announcement Overview • Patient and Stakeholder Engagement • Administrative Overview • Merit Review Process • Key Dates and Resources • Questions and Answers Submit questions via the Question box in GoToWebinar 2
Today’s Presenters Anne Trontell Carly Parry Donna Gentry Carolyn Mohan Julie Kennedy Lesch Associate Director, Senior Advisor, Care Assistant Director, Assistant Director, Senior Engagement Clinical Effectiveness Coordination & Contract Program Support, Officer, and Decision Science Transitions, Management Merit Review Public and Patient Healthcare Delivery Engagement and Disparities Research 3
About PCORI
Why PCORI? For all the advances it produces, research still has not answered many • questions patients face. People want to know which treatment is best for them. • Patients and their clinicians need information they can understand and use. • 5
Our Mission PCORI helps people make informed health care decisions, and improves health care delivery and outcomes, by producing and promoting high integrity, evidence-based information that comes from research guided by patients, caregivers and the broader health care community. 6
Our Work Answers Patients’ Questions Given my personal characteristics, conditions and preferences… 7
Focus on Clinical Comparative Effectiveness Research (CER) Comparative Effectiveness Research (CER) includes: Studies that compare health outcomes and the clinical effectiveness, risks, and benefits of • two or more approaches to healthcare • Clinical effectiveness research • Improving healthcare delivery CER • Communications and dissemination CER • CER to reduce/eliminate health and healthcare disparities All applicants should: • • Explain how the research is comparative • Name the comparators • State why the comparisons are important 8
Research We Do Not Fund PCORI does not fund research that will produce… • clinical practice guidelines • coverage recommendations • payment or policy recommendations In general, PCORI will not cover costs for study interventions that constitute the procedures, treatments, interventions, or other standard clinical care (“patient care”) that are being proposed for comparison in the research project (“patient care costs”). 9
Research We Do Not Fund PCORI does not fund studies of cost-effectiveness analysis (CEA). Examples of Cost-Effectiveness Analysis: • Research that conducts a formal CEA in the form of dollar-cost per quality-adjusted life-year to compare two or more alternatives • Research that conducts a formal CEA in the form of dollar-cost per non- adjusted life-year to compare two or more alternatives PCORI may fund studies that capture data that reflect economic impacts — for example, out-of-pocket costs to patients. 10
PCORI Methodology Standards Research funded by PCORI must adhere to the PCORI Methodology Standards, which represent minimal requirements for the design, conduct, analysis, and reporting of patient-centered outcomes research. The 65 standards can be grouped into 2 broad categories and 16 topic areas: Cross-Cutting Standards Design-Specific Standards • Formulating Research Questions • Data Registries • Data Networks • Patient Centeredness • Causal Inference Methods* • Data Integrity & Rigorous Analyses • Adaptive & Bayesian Trial Designs • Preventing/Handling Missing Data • Studies of Medical Tests • Heterogeneity of Treatment Effects • Systematic Reviews • Research Designs Using Clusters • Studies of Complex Interventions • Qualitative Methods • Mixed Methods Research *The first standard for Causal Inference Methods (CI-1) • Individual Participant-Level Data Meta-Analysis is considered cross-cutting and applicable to all (IPD-MA) PCOR/CER studies. 11
Funding Announcement Overview 12
PLACER PFA Overview Objective of this PFA : Support large-scale, high-impact randomized trials in CER with attendant risks of achieving their research aims and thus merit an initial phase of testing and refinement to determine their feasibility and viability • maximize the likelihood of full-scale trial success. • Total Direct Costs: Up to $22 million per award Project Duration: Up to 6.5 years per award • Up to $2 million (feasibility phase) • Up to 1.5 years (feasibility phase) • Up to $20 million (full-scale study phase) • Up to 5 years (full-scale study phase) Available funds : $150 million 13
PLACER PFA Highlights Requirements • Scope: Proposed studies of significant scale and scope, requiring funding in excess of $10 million in direct costs • Design: Individual-level or cluster randomized controlled trial • Data Coordinating Center: Required to be used and to operate with scientific independence Opportunities • Development of strong stakeholder engagement: enabled during the initial phase of award • Use of PCORnet clinical research infrastructure & data resources: PCORnet is the National Patient Centered Clinical Research Network developed by PCORI 14
PLACER PFA : Distinctive Requirements Relative to Prior PCORI PFAs • Two well-integrated trial phases: an initial feasibility phase, followed by full- scale study and conduct of clinical trial • Data coordinating center : Applicants must include a data coordinating center (DCC) with an independent scientific leadership role in study decision-making about the analytical, statistical, and data management aspects of both study phases • Stakeholder engagement : • Applicants should include experienced leadership in stakeholder engagement as part of overall trial leadership in both study phases • PLACER applicants are not required to demonstrate that patients and other stakeholders are already engaged as research team members at the time of application submission 15
PLACER PFA Feasibility Phase Activities in the first phase may include, but are not limited to: Development and initiation of stakeholder engagement plan • Protocol development • Finalizing clinically relevant and patient-centered outcome measures • Piloting of data collection methods • Refinement of recruitment methods • Establishment of agreements with study sites and the DCC • Submission of plans for all aspects of ethical and regulatory oversight and • protection of human subjects 16
PLACER PFA Full-Scale Study Phase • Prior to the planned initiation of the full-scale study, PCORI and an external advisory panel (EAP) will evaluate feasibility phase progress to assess • Accomplishments and findings relative to pre-specified plans • Likelihood that the planned full-scale study remains feasible with high probability of success • If continued conduct of the full-scale trial is approved, PCORI will continue funding via modification of the original contract • Full-scale trial milestones may be revised based on feasibility phase findings 17
PLACER PFA: Data Coordinating Center Review and Expected Functions PCORI Review of DCC Letters of Intent (LOIs) may be submitted without a named DCC, but a DCC must be included if • you are invited to submit a full application • Applications submitted without a DCC will be removed from consideration for review Applications must provide sufficient assurances for review to determine if the proposed DCC • meets all necessary standards for data integrity, security, and protection Expected DCC Functions Feasibility phase: Contribute substantively to refining the final trial design and protocol and • planning of data collection, management, and the statistical analysis plan Full-scale study phase: Management/monitoring of data reporting, completeness, and quality, • study enrollment and retention, reporting & analyses to the Data Safety and Monitoring Board (DSMB), and conduct of statistical analyses of trial data 18
PLACER PFA: Data Coordinating Center Relationship to Clinical Leadership of the Study PCORI advocates separation and independence of clinical and data/statistical leadership of the • study and strongly discourages the use of a combined DCC and clinical coordinating center (CCC). • If such an arrangement is proposed, it must provide evidence of distinct lines of institutional reporting, accountability, and financing between the DCC and CCC. Applicants must certify or otherwise document the existence of adequate policies, procedures, and • standards to ensure independence, autonomy, absence of conflicts, and firewalls between clinical care and study data PCORI recommends that the PI of the DCC be named as a dual-PI to promote DCC parity, scientific • independence, and autonomy in study decisions. If the DCC is subcontracted to the prime applicant, the prime institution bears responsibility for • ensuring that all relevant regulatory and certification requirements of the data coordinating centers are met to guarantee data integrity, safety, confidentiality, etc. 19
PLACER PFA Invited Research Areas • Investigator-initiated research in one of five PCORI priority areas: • Addressing disparities • Assessment of prevention, diagnosis, and treatment options • Improving healthcare systems • Intellectual and developmental disabilities • Prevention of maternal morbidity and mortality 20
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