Spring 2015 Cycle: Large Pragmatic Studies to Evaluate Patient-Centered Outcomes Applicant LOI Town Hall April 14, 2015
Agenda Welcome Overview Programmatic Requirements Administrative Requirements Resources Questions? Submit questions via the chat function in Meeting Bridge. Ask a question via phone (an operator will standby to take your questions).
Today’s Presenters David Hickam, MD, MPH Iris Giggetts, MSW, CRA Program Director Contracts Associate, Pre Award Clinical Effectiveness Research Contracts Management and Administration
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.
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.
Our Focus at PCORI Comparative Clinical Effectiveness Research • Patient-centered • Answering questions important to patients and other clinical decision makers • Comparisons of choices that matter to patients • Attention to possible heterogeneity of treatment effects
Overview
PFA Overview: Large Pragmatic Studies to Evaluate Patient-Centered Outcomes Objective of this PFA: • Address critical clinical and health-related Available Funds and comparative effectiveness questions faced by Duration: patients, their caregivers, and their clinicians • A total of $90 million In this PFA we seek to fund or co-fund: (direct and indirect) for • Large pragmatic trials this cycle • Up to $10 million in total • Large simple trials direct costs per project • Large scale observational studies • Projects should be completed within 5 years
Two Types of Applications for the Large Pragmatic Studies PFA • Applications proposing “clinical comparative effectiveness research (CER)” • Applications proposing “improving healthcare systems (IHS) CER” • Both types of projects are patient centered outcomes research (PCOR) • CER model: compare 2 or more options • Examine outcomes that are important to patients • Assess the balance of benefits and harms
What is a Pragmatic CER Study? • Answers a practical, real world comparative effectiveness research question. • Assesses whether two or more options differ in effectiveness when administered as they are in real life, and it is conducted in a clinical setting that is as close as possible to a real world setting. • The methodological approach (including study design, outcome measures, and follow-up) is as simple as possible without sacrificing scientific rigor.
Programmatic Requirements
Essential Characteristics of Studies • Involve broadly representative patient populations in typical clinical care and community settings • Have strong endorsement and study participation by relevant national or regional patient organizations, professional organizations, and/or payer or purchaser organizations • Aim to • Address prevention, diagnosis, treatment, or management of a disease or symptom • Improve the performance of healthcare systems • Eliminate health or healthcare disparities • Have a sample large enough to allow precise estimates of effect sizes and support evaluation of differences in treatment effectiveness in patient subgroups • Measure health outcomes that are meaningful to the patients
Comparators of Interest • Specific drugs, devices, and procedures • Medical and assistive devices and technologies • Techniques for behavioral modification • Complementary and alternative medicine • Delivery-system interventions
The Case of Usual Care • “Usual care” is not an appropriate comparator for CER studies . • It is ill-defined, difficult to quantify, and subject to considerable geographic and temporal variations, thus limiting interpretability, applicability, and reproducibility. • If the applicant proposes “usual care” as a rational and important comparator in the proposed study, then it must be described in detail, coherent as a clinical alternative, and properly justified as a legitimate comparator (e.g., usual care is guidelines-based). • Additionally, it must be accompanied by an explanation of how the care given in the usual care group will be measured in each individual patient and how appropriate inferences will be drawn from its inclusion.
Sources for Topics of Interest • PCORI Priority topics (updated 3/2015; refer to the PFA) • AHRQ Future Research Needs Projects • IOM 100 priority topics for CER • Investigator initiated topics will also be considered. Researchers must make a strong case for the importance of the proposed research. Describe clearly the evidence gap that the study will fill.
PCORI Priority Topics • Medical vs. invasive procedures for asymptomatic carotid artery stenosis • Surgical options for hip fracture in the elderly • Pelvic floor mesh implants • Treatment strategies for patients with autism spectrum disorder • Treatment options for patients with multiple sclerosis • Benefits and harms of continuous ambulatory peritoneal dialysis compared with hemodialysis (daily or intermittent home, or conventional in-center) in patients with end-stage renal disease and in important patient subgroups (e.g., by age, race, ethnicity, cardiovascular risk, other comorbidities) • Multi-component interventions to reduce initiation of tobacco use and promote cessation of tobacco use among high-risk populations with known disparities • Integration of mental and behavioral health services into the primary care of the general population
PCORI Priority Topics • Integration of mental and behavioral health services into the primary care of persons at risk for disparities in health care and outcomes • Management of breast ductal carcinoma in situ (DCIS) • Reduction of cardiovascular disease (CVD) risk in underserved populations, such as racial and ethnic minorities and those living in rural communities • Strategies for preventing the progression of episodic acute back pain into chronic back pain • Treatment strategies for adult patients with migraine headache • Treatment strategies for symptomatic osteoarthritis (OA), including joint replacement • Treatment options for people with opioid substance abuse • Particle beam therapy for patients with lung, and prostate cancer
PCORI Priority Topics • Compare the effectiveness of multi-component systems interventions, such as evidence-based models of perinatal care, aimed at improving outcomes, such as pre-term birth and low birth weight, for mothers and babies at risk for health disparities • Clinical Interventions to reduce nontraumatic lower extremity amputations in racial/ethnic minorities and low income populations with diabetes: Does expert protocol-driven, team-based care reduce the risk of nontraumatic lower extremity amputations compared with existing and established guideline-based care for racial/ethnic minorities and low-income populations with diabetes? • Compare the effectiveness of diverse models of comprehensive support services (e.g., incorporation of wraparound services, alternative providers, and technology) for infants and their families/caregivers following discharge from neonatal intensive care unit.
PCORI Priority Topics • Compare the effectiveness of multidisciplinary rehabilitation programs (e.g., Community Integrated Rehabilitation: neurobehavioral; residential community; comprehensive holistic and home-based services) for moderate to severe traumatic brain injury in non-military or veteran adults. • Compare the effectiveness of alternative models of coordinated pain management (such as specialized, integrated pain centers, coordinated care models that emphasize cross-provider data- and communication-sharing, or those that include risk triage systems with care coordination) for treatment of chronic nonspecific, musculoskeletal pain on improving functional status and other patient-centered outcomes. • Compare the effectiveness of the various alternative delivery models (e.g., primary care, schools, mobile vans) versus the dental office in preventing dental caries in children in medically underserved areas.
PCORI Priority Topics • Compare the effectiveness of various strategies aimed at integrating pharmacists or pharmacy services into patient care (e.g., primary/acute care and pharmacy integration, pharmacist-provided preventive care, pharmacist- provided medication management or reconciliation services, other pharmacy- specific collaborative care models) on patient-centered outcomes (e.g., reduction in inappropriate medication use and polypharmacy, access to preventive vaccines, reduction in adverse events and hospital re-admissions, improved disease- or condition specific outcomes). • Compare the effectiveness of evidence based screening and primary prevention approaches, including different modes and settings (e.g., universal screening vs. targeting at-risk individuals, virtual vs. face-to-face screening, within primary care setting vs. school-based), at minimizing suicidality among adolescents and improving other patient centered outcomes.
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