PCORI Funding for Rare Disease Research: Spring 2015 Broad PFAs Thursday, February 12, 2015
Welcome and Introductions We welcome your questions and comments via the chat function on the right side of your screen We welcome your comments via Twitter to @PCORI and #PCORI An archive of this webinar will be posted to http://www.pcori.org/get-involved/pcori-in-practice/ following this event. 2
Agenda for today’s webinar Time Agenda Item Welcome and Overview of PCORI 3:00 - 3:10 p.m. PCORI’s Rare Disease Portfolio 3:10 - 3:25 p.m. New Funding Opportunity for Rare 3:25 – 3:45 p.m. Disease Research David Hickam, MD, MPH Program Director, 3:45 – 4:00 p.m. Question and Answer Clinical Effectiveness Research Adjourn 4:00 p.m. 3
Goals for Today PCORI has designated a special pool To provide an overview of PCORI of $12 million to fund rare disease research as part of the Spring 2015 Broad PCORI Funding To explore PCORI’s current Announcements (PFAs). investments in rare disease This funding will be available through research the broad national priorities areas: • Assessment of Prevention, Diagnosis, and Treatment Options, To provide a background behind • Improving Health Care Systems, this special pool of funds for rare • Communication and Dissemination Research, and disease research • Addressing Disparities. To prepare potential applicants for this rare disease research funding with the information needed to submit a responsive letter of intent (LOI) 4
About PCORI 5
About PCORI An independent research institute authorized by Congress in 2010. Governed by a 21-member Board representing the entire healthcare community. Funds comparative clinical effectiveness research (CER) that engages patients and other stakeholders throughout the research process. Seeks answers to real-world questions about what works best for patients based on their circumstances and concerns. 6
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. 7
What is Comparative Effectiveness Research? Compares two or more options for prevention, diagnosis, or treatment (can include “usual care”) Considers the range of clinical outcomes relevant to patients Is conducted in real-world populations and real-world settings Attends to differences in effectiveness and preferences across patient subgroups Often requires randomized trial design 8
What is Patient-Centered Outcomes Research? Comparative effectiveness research that has the goal of helping people make better-informed healthcare decisions. Actively engages patients and key stakeholders throughout the research process. Examines important clinical management options. Evaluates the outcomes that are the most important to patients. Addresses implementation of findings in clinical care environments. 9
Research We Support 10
The Research We Fund is Guided by Our National Priorities for Research Assessment of Communication & Prevention, Improving Dissemination Diagnosis, and Healthcare Systems Research Treatment Options Accelerating PCOR Addressing and Methodological Disparities Research
Snapshot of Funded Projects Number of projects: 360 Amount awarded: $671 million Number of states where we are funding research: 39 (plus the District of Columbia and Quebec) As of September 30, 2014
Current Status of Funded Studies in Rare Disease PCORI uses the federal government’s definition of rare diseases. Conditions that affect fewer than 200,000 people in the United States (i.e., less than 1 in 1,500 persons) Special efforts, such as combining data across large populations, may be needed to address these conditions. PCOR has appointed the RDAP. Current portfolio: 17 RD research projects 9 RD PPRNs 2 pipeline to proposal awards 13
PCORI’s Current Portfolio of RD Projects Pediatric neurologic disorders (5 projects) Immunologic disorders (4 projects) Sickle cell disease (3 projects) Genetic testing (2 projects) Nephrotic syndrome Cystic fibrosis Management of symptoms 14
Highlighted Projects A Randomized Controlled Trial of Anterior Versus Posterior Entry Site for Cerebrospinal Fluid Shunt Insertion Principal Investigator: William Whitehead, MD, MPH Comparative Efficacy of Therapies for Eosinophilic Esophagitis Principal Investigator: Marc Rothenberg, MD, PhD 15
A Randomized Controlled Trial of Anterior Versus Posterior Entry Site for Cerebrospinal Fluid Shunt Insertion Methods • Randomized controlled trial Aims to determine if the choice of entry • Head to head comparison site (anterior vs. posterior) results in a Engagement significant improvement in shunt survival for pediatric patients, if quality of life • The Hydrocephalus Association differs due to shunt entry site, and to Patient Partner Committee will compare shunt infection rate, length of monitor the project. surgery, number of catheter passes, and • Input on study design and conduct length of stay associated with anterior and coming from pediatric posterior shunt entry site techniques. neurosurgeons Potential Impact William Whitehead, MD, MPH • Could change practice if one Baylor College of Medicine technique found to be superior for Houston, TX shunt survival that results in fewer Assessment of Prevention, Diagnosis, and Treatment hospitalizations, diagnostic tests, Options, awarded September 2014 and days off from school and work
Comparative Efficacy of Therapies for Eosinophilic Esophagitis Methods • Randomized controlled trial of Aims to determine whether simpler, dietary regimens. markedly less restricted diets might Engagement effectively treat eosinophilic esophagitis • The stakeholders’ advisory board (EoE) in addition to current standard will guide all aspects of the project. therapies that either eliminate all foods triggering a reaction during allergy testing or Potential Impact eliminate the six most commonly allergenic • Could change practice by foods. identifying simpler and easier diet- management protocols that will require fewer endoscopies and improve quality of life. Marc Rothenberg, MD, PhD Cincinnati Children’s Hospital Medical Center Cincinnati, OH Assessment of Prevention, Diagnosis, and Treatment Options, awarded September 2014
PCORnet: A Network of Networks This map depicts the number of PCORI-funded Patient-Powered or Clinical Data Research Networks that have coverage in 18 each state.
PCORnet’s goal PCORnet seeks to improve the nation’s capacity to conduct clinical research by creating a large, highly representative, national patient- centered network that supports more efficient clinical trials and observational studies. 19
Rare Disease Cohorts in CDRNs CDRN Rare Disease Mid-South CDRN Sickle Cell Disease LACDRN Sickle Cell Disease; rare cancers CAPriCORN Sickle Cell Disease; recurrent C. Difficile colitis SCIHLS Pulmonary arterial hypertension PORTAL Severe congenital heart disease PEDSNet Hypoplastic left heart syndrome GPC ALS NYC-CDRN Cystic fibrosis ADVANCE Alpha 1 Antitrypsin deficiency PaTH Idiopathic pulmonary fibrosis pSCANNER Kawasaki disease 20
Rare Disease Cohorts in PPRNs . PPRN Disease Pediatric Crohn’s disease & ulcerative colitis ImproveCareNow PARTNERS Consortium Juvenile Rheumatic Disease ALD Connect Adrenoleukodystrophy PMS_PPRN Phelan-McDermid syndrome PI-CONNECT Primary immunodeficiency diseases The Vasculitis PPRN Vasculitis DuchenneConnect Duchenne & Becker muscular dystrophy NephCure Primary Nephrotic syndrome REN Aicardi, Lennox-Gastaut, Phelan-McDermid, Dravet Syndromes; Hypothalamic Hamartoma; Tuberors Sclerosis CENA Alström, Joubert, Klienfelter Syndromes; Gaucher Disease, PXE, etc. 21
Our Application Process 22
Who Can Apply? Private Sector • Nonprofit and for-profit research organizations Public Sector • Universities and colleges; Hospitals and healthcare systems; Laboratories and manufacturers; Units of state, local, or federal government. US Organizations • Must be recognized by the Internal Revenue Service Foreign Organizations and Nondomestic Components of US Organizations • May apply if here is demonstrable benefit to the US healthcare system; US efforts in the area of patient-centered research can be clearly shown Individuals • Not permitted to apply 23
Annual Funding Cycles Funding Application Application Closes Cycle Opens Winter November February Spring February May Summer May August Fall August November 24
Assessment of Prevention, Diagnosis, and Treatment Options • Comparative studies in prevention, screening, diagnosis, treatment, and long-term management that have not been adequately addressed previously. • Comparative studies that also reported on treatment effect modifiers including demographic, biological, clinical, socioeconomic, and other factors. • Comparative studies addressing rare diseases. Available funds: Up T o $32 Million Budget: $2 million in direct costs Project Period: 3 years Assessment of Prevention, Diagnosis, and Treatment Options 25
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