Cycle 1 2019: Treatment of Post- Traumatic Stress Disorder in Adults PFA Applicant Town Hall January 17, 2019
Agenda • About PCORI • Programmatic Overview • Patient and Stakeholder Engagement • Administrative Overview • Merit Review Criteria • Resources • Questions and Answers Submit questions via the Question box in GoToWebinar 2
Today’s Presenters Mary Gardner Carolyn Mohan, Kim Bailey, MS Denese Neu, PhD, Administrator, J illian Nowlin, MA DrPH, Senior Program MS Contract Program Associate, Merit Review Officer, Officer, Engagement Management Clinical Program Support Clinical Officer, Program Support Effectiveness and & Information Effectiveness and Engagement & Information Decision Science Management Decision Science Management 3
About PCORI pcori.org About PCORI pcori.org
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
What does PCORI look for? 8
Focus on Comparative Clinical Effectiveness Research (CER) CER includes: Studies that compare health outcomes and the clinical effectiveness, risks, and benefits of • two or more approaches to healthcare All applicants should: • • Name the comparators • Explain how the research is comparative • State why the comparisons are important 9
Research We Do Not Fund PCORI does not fund studies of cost-effectiveness analysis (CEA). Examples of CEA ▪ Research that conducts a formal CEA in the form of dollar-cost per quality-adjusted life-year (including non-adjusted life-years) to compare two or more alternatives ▪ Research that directly compares the overall costs of care between two or more alternative approaches as the criterion for choosing the preferred alternative NOTE: PCORI does fund studies that explore the burden of costs on patients — for example, out-of-pocket costs. 10
Research We Do Not Fund PCORI does not fund research whose findings will include • development of clinical practice guidelines • coverage recommendations • payment or policy recommendations 11
2018 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 54 standards can be grouped into 2 broad categories and 13 topic areas. Design-Specific Standards Cross-Cutting Standards Data Registries Formulating Research Questions • • 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 • *The first standard for Causal Inference Methods (CI-1) is considered cross-cutting and applicable to all PCOR/CER studies. 12
Treatment of Post-traumatic Stress Disorder (PTSD) in Adults: PFA Overview
PFA Overview Objective of this PFA: • Fund comparative clinical effectiveness research (CER) studies that aim to determine which specific, evidence-based treatments for adults with PTSD are most effective, and for whom • Proposed comparators may include psychological interventions, pharmacological interventions, or a combination of the two Available Funds: Up to $15 million Total Direct Cost/Maximum Project Period: $5 million/3.5 years for all awards 14
Treatment of PTSD in adults The need for more evidence In 2018, PCORI and AHRQ published a systematic review update that found insufficient evidence • to determine: • The comparative effectiveness of most psychological treatments, pharmacological treatments, and combination treatments • Whether any treatment approaches are more efficacious or effective for patients with specific characteristics such as comorbid conditions or trauma type Current guidelines from the VA/DoD (2017) and the APA (2017) also highlight the need for • research in the field and underscore the following priorities: • Head-to-head comparisons of treatments • Studies that assess the heterogeneity of treatment effect of interventions in subgroups of interest (e.g., military v. civilian, men v. women, type of trauma, etc.) • Studies that include patients with comorbid conditions that frequently co-occur with PTSD • Studies that evaluate patient centered outcomes (including functional outcomes and QoL) 15
Treatment of PTSD in Adults PFA: Goal • Fund high-quality comparative studies that will generate important findings about which specific treatments for adults with PTSD are most effective, and for whom 16
Design Considerations Population • Adults aged 18 years or older with a diagnosis of PTSD per DSM-5 criteria • Areas of interest include: • Studies that allow the inclusion of individuals with conditions and diagnoses commonly co-occurring with PTSD (e.g., substance use disorder) • Studies that examine the effectiveness of interventions in key subgroups (e.g., military v. civilian populations, men v. women) • Applicants should clearly specify the proposed patient population(s) and delineate key inclusion and exclusion criteria 17
Design Considerations Interventions • Proposed comparisons may include psychological interventions, pharmacological treatments, or a combination of the two • All proposed comparators must have demonstrated evidence of efficacy that is at least moderate strength documented via systematic reviews, prior empirical investigations, or other scientific documentation • Applicants must clearly define why the proposed research question and comparison are of clinical interest 18
Design Considerations Outcomes • All applications should include appropriate patient-centered and clinical outcomes • Namely, functional outcomes and quality of life should be ascertained, in addition to PTSD symptoms and remission • All proposed endpoints must be well-validated • Applicants should provide a clear rationale for the selection of proposed endpoints 19
Design Considerations Timing • Applicants should clearly specify the duration, format, and dosage of each intervention • Studies must include a minimum of six months of follow-up, but longer follow-up is preferred 20
Patient and Stakeholder About PCORI Engagement pcori.org
Patients and Other Stakeholders Patient/ Consumer Caregiver/ Family Purchaser Member of Patient Clinician Payer PCORI Community Patient/ Caregiver Industry Advocacy Org Hospital/ Policy Health Maker System Training Institution 22
Patient-Centeredness vs. Patient Engagement • Patient-Centeredness • Addresses outcomes (both benefits and harms) that important to patients. • The interventions proposed for comparison are currently available to patients. • Patient and Stakeholder Engagement • Demonstrates active engagement between scientists, patients, and stakeholders. • Involves relevant organizations, community, patients, and caregivers through existing relationships or presents a well-thought out plan to establish these partnerships. 23
Evidence of Appropriate Engagement of Relevant Patients and Other Stakeholders • Applicants are expected to consult with patients and other stakeholders on their decisional dilemma and evidence needs or reference previously documented decisional dilemmas and describe how this consultation informed the proposed research project. • Applications should identify patients and stakeholders consulted in determining that the proposed study addresses their evidentiary needs for decision-making and indicate how they will be actively engaged as partners throughout the conduct of the study. 24
The Engagement Rubric The rubric is intended to provide guidance to applicants, merit reviewers, awardees, and engagement/program officers (for creating milestones and monitoring projects) regarding engagement in the conduct of research. It is divided into three segments: Planning the Study Conducting the Study Disseminating the Study Results 25
Budgeting Things to Consider: • Financial compensation of partners • Expenses of partners (transportation, childcare, caregiver) • Budgeting for program staff dedicated to engagement tasks • Costs of engagement meetings and events (travel, food, audio visual) • Additional time and resource to incorporate partner feedback into various project process 26
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