Research: Spring 2015 Broad PFAs Thursday, February 12, 2015 - - PowerPoint PPT Presentation
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
Thursday, February 12, 2015
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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.
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Time Agenda Item 3:00 - 3:10 p.m.
Welcome and Overview of PCORI
3:10 - 3:25 p.m.
PCORI’s Rare Disease Portfolio
3:25 – 3:45 p.m.
New Funding Opportunity for Rare Disease Research
3:45 – 4:00 p.m.
Question and Answer
4:00 p.m.
Adjourn
David Hickam, MD, MPH Program Director, Clinical Effectiveness Research
To provide an overview of PCORI To explore PCORI’s current investments in rare disease research To provide a background behind this special pool of funds for rare disease research To prepare potential applicants for this rare disease research funding with the information needed to submit a responsive letter of intent (LOI)
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PCORI has designated a special pool
research as part of the Spring 2015 Broad PCORI Funding Announcements (PFAs). This funding will be available through the broad national priorities areas:
Diagnosis, and Treatment Options,
Research, and
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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
process. Seeks answers to real-world questions about what works best for patients based on their circumstances and concerns.
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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.
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Compares two or more options for prevention, diagnosis,
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
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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.
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Assessment of Prevention, Diagnosis, and Treatment Options Improving Healthcare Systems Communication & Dissemination Research Addressing Disparities Accelerating PCOR and Methodological Research
and Quebec)
As of September 30, 2014
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.
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A Randomized Controlled Trial of Anterior Versus Posterior Entry Site for Cerebrospinal Fluid Shunt Insertion
William Whitehead, MD, MPH Baylor College of Medicine Houston, TX Methods
Engagement
Patient Partner Committee will monitor the project.
coming from pediatric neurosurgeons Potential Impact
technique found to be superior for shunt survival that results in fewer hospitalizations, diagnostic tests, and days off from school and work Aims to determine if the choice of entry site (anterior vs. posterior) results in a significant improvement in shunt survival for pediatric patients, if quality of life differs due to shunt entry site, and to compare shunt infection rate, length of surgery, number of catheter passes, and length of stay associated with anterior and posterior shunt entry site techniques.
Assessment of Prevention, Diagnosis, and Treatment Options, awarded September 2014
Comparative Efficacy of Therapies for Eosinophilic Esophagitis
Marc Rothenberg, MD, PhD Cincinnati Children’s Hospital Medical Center Cincinnati, OH Methods
dietary regimens. Engagement
will guide all aspects of the project. Potential Impact
identifying simpler and easier diet- management protocols that will require fewer endoscopies and improve quality of life.
Aims to determine whether simpler, markedly less restricted diets might effectively treat eosinophilic esophagitis (EoE) in addition to current standard therapies that either eliminate all foods triggering a reaction during allergy testing or eliminate the six most commonly allergenic foods.
Assessment of Prevention, Diagnosis, and Treatment Options, awarded September 2014
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This map depicts the number of PCORI-funded Patient-Powered
Research Networks that have coverage in each state.
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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.
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
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PPRN Disease ImproveCareNow Pediatric Crohn’s disease & ulcerative colitis 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. .
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Private Sector
Public Sector
and manufacturers; Units of state, local, or federal government.
US Organizations
Foreign Organizations and Nondomestic Components of US Organizations
US efforts in the area of patient-centered research can be clearly shown
Individuals
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Funding Cycle Application Opens Application Closes Winter
November February
Spring
February May
Summer
May August
Fall
August November
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and long-term management that have not been adequately addressed previously.
including demographic, biological, clinical, socioeconomic, and other factors.
Available funds: Up T
Budget: $2 million in direct costs Project Period: 3 years
Assessment of Prevention, Diagnosis, and Treatment Options
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electronic technologies
Available funds: Up T
Improving Healthcare Systems
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patients, their caregivers and clinicians
caregivers, and clinicians
choosing among available options for care and treatment
Available funds:Up T
Budget: $1.5 million in direct costs Project Period: 3 years
Communication & Dissemination Research
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disadvantage members of at least one of the program’s target population groups and limit their ability to achieve optimal, patient- centered outcomes
want studies that identify best options for eliminating disparities Available funds: Up T
Budget: $1.5 Million in direct costs Project Period: 3 years
Addressing Disparities
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PCORI is especially interested in the investigation of strategies that address care for patients with life- threatening or chronically debilitating rare diseases.
PCORI has designated a special pool of $12 million to fund rare disease research as part
Announcements (PFAs). This funding will be available through the broad national priorities areas:
Treatment Options,
and
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Application Deadline Final Decision
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Submitting Questions: Submit questions via the chat function in on the right of your screen If we are unable to address your question during this time, please e-mail your question to us at getinvolved@pcori.org An archive of this webinar will be posted to http://www.pcori.org/get- involved/pcori-in-practice/ following this event. Accessing this Webinar:
General Inquiries info@pcori.org | (202) 827-7200 Research/Programmatic Questions sciencequestions@pcori.org | (202) 627-1884 Administrative/Financial/Technical Questions pfa@pcori.org Find us Online at: www.pcori.org
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