- Pilot Grant
CPCE is proud to receive two PCORI awards for patient-centered comparative effectiveness research
The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors has selected two CPCE investigators to lead patient-centered research to inform critical choices by patients, caregivers, clinicians and stakeholders in health care. These projects will provide insight to the comparative benefits and harms of the options and provide information about outcomes that are important to patients and their caregivers.
Pediatric IntraVenous Versus Oral antibiotic Therapy – PIVVOT project
When a child who has been hospitalized with a serious infection is discharged home to complete a prolonged course of antibiotics, there are two ways the child can receive the medicine by mouth (oral antibiotics) or intravenously (via a peripherally inserted central catheter (PICC line) placed prior to discharge). Though PICC lines can be scary to pediatric patients, and require caregivers to be trained in their use and care, many clinicians often prefer them to oral medicines for long-term antibiotic treatments.
Ron Keren, MD, MPH, Professor of Pediatrics and Epidemiology and director of the Center for Pediatric Clinical Effectiveness, was awarded nearly two million dollars from PCORI in January 2013 to lead a study examining whether oral antibiotics are as effective at treating infection over an extended period as are peripherally inserted central catheter (PICC) lines. "These two antibiotic treatment options have major implications for the overall experience of the child, families and caregivers, but there is a lack of real-world evidence on their benefits and drawbacks to help clinicians and patient families make an informed choice," said Dr. Keren.
A type of intravenous catheter, a PICC line is a long, flexible tube that is inserted in a peripheral vein, often in the arm or neck, and advanced until its tip rests near the heart. Because they tap directly into the circulatory system, PICC lines offer maximum drug delivery. Unlike regular IV catheters, PICC lines can stay in the body for weeks to months, but they require regular maintenance. PICC lines must be flushed daily, their dressings have to be inspected and changed, and patients with PICC lines must avoid getting them wet or dirty — a tall order for some active pediatric patients and their families. In addition, a variety of medical equipment is required to use and maintain PICC lines, including infusion pumps, IV antibiotic solutions, dressings, and portable IV poles. PICC lines also have some risks. They can clot, break, or become dislodged. And because they sit in large blood vessels directly above the heart, any bacteria that are inadvertently introduced into the catheter go directly to the heart and are pumped throughout the body, which can lead to a dangerous infection called sepsis.
Oral antibiotics, on the other hand, are much easier for patients to take and caregivers to manage. However, because oral medications must pass through the digestive system, to have the same efficacy as IV medications oral antibiotics must have high "bioavailability" – the percentage of the drug that reaches the blood. Drugs administered via PICC lines have, by definition, 100 percent bioavailability.
"If we find that the prolonged IV option is no better than the oral route, we think that most families would prefer for their child to take oral antibiotics," Dr. Keren noted. "However, if IV antibiotics are marginally better than oral antibiotics, then that benefit will need to be weighed against any reduction in quality of life and complications that we anticipate with the PICC lines."
Dr. Keren's project was among the 25 awards issued in PCORI's first full funding cycle.
Broad vs. Narrow Spectrum Antibiotics for Acute Respiratory Tract Infections in Children
On September 10, 2013, CHOP was approved to receive another research award from PCORI to study the comparative effectiveness of broad vs. narrow spectrum antibiotics for treating acute respiratory tract infections in children.
Jeffrey S. Gerber, MD, PhD, Assistant Professor of Pediatrics at the University Of Pennsylvania School Of Medicine and CPCE faculty member, will lead the research project. The project will focus on determining the most effective and appropriate antibiotic choices for children with ear infections, sinus infection, and strep throat, focusing on the outcomes most important to the parents and caregivers of children with these illnesses.
"Although most national guidelines recommend narrow-spectrum antibiotics for these extremely common infections, many children receive broad-spectrum agents. Using parent- and patient-centered outcomes, this study is designed to identity which antibiotic choices best optimize clinical outcomes while minimizing side effects" said Dr. Gerber.
"This project was selected for PCORI funding not only for its scientific merit but also for its potential to fill an important gap in our health knowledge and ultimately help patients and those who care for them make more fully informed decisions about their care," said PCORI Executive Director Joe Selby, MD, MPH. "The project reflects PCORI's commitment to support patient-centered comparative effectiveness research, a new approach to health research that emphasizes the inclusion of patients and caregivers at all stages of the study process. We look forward to following the study's progress and working with CHOP to share the results."
Dr. Gerber's study is one of 71 projects totaling more than $114 million approved for funding by PCORI's Board of Governors. This round of awards were selected through a highly competitive review process in which scientists, patients, caregivers, and other stakeholders helped to evaluate more than 570 proposals that responded to five PCORI funding announcements. Proposals were evaluated on the basis of scientific merit, how well they engage patients and other stakeholders, their methodological rigor, and how well they fit within PCORI's national research priorities. All awards were approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
This award is part of PCORI's latest round of primary research funding. Through previous funding cycles, including Dr. Keren's and a round of pilot projects, PCORI has committed a total of $304 million since 2012 to support patient-centered comparative effectiveness research.
The Patient-Centered Outcomes Research Institute (PCORI) was authorized by the Patient Protection and Affordable Care Act of 2010 as an independent, non-profit, non-governmental organization. PCORI's purpose, as defined by the law, is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions by "advancing the quality and relevance of evidence about how to prevent, diagnose, treat, monitor, and manage diseases, disorders, and other health conditions." PCORI is committed to continuously seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.
CPCE Works-in-Progress sessions bring together clinical effectiveness researchers across the hospital to discuss research projects that are in various stages of development, from protocol development all the way to data analysis, interpretation, and publication. Faculty as well as fellows interested in receiving feedback on their research are welcome to present. These sessions also provide a venue to discuss potential collaborative responses to requests for applications and/or program project grants. Works-in-progress Sessions are held one to two times per month from September through June. If you have a research project you would like to present, please email firstname.lastname@example.org.
CPCE Works-in-progress Sessions qualify for Category 2 CME credit. Use the self-reporting log to record your attendance. You can also obtain documentation of attendance at CPCE Works-in-progress Sessions by contacting Debra Hillman at email@example.com.
For a schedule of upcoming Works-in-progress Sessions, please check the CPCE calendar.
Pilot Grant Program
CPCE offers awards twice each year through its Pilot Grant Program. The purpose of this program is to promote and support CHOP investigators in clinical effectiveness pilot research studies that will attract external support for large-scale studies. Investigators from all CHOP departments and divisions, including fellows in their final year of fellowship transitioning to a faculty position at CHOP, are encouraged to apply. Selected proposals will be supported for up to a maximum of $10,000 for one year. Projects should be able to be completed within one year.
Application and Submission Process:
- Please submit proposals in NIH format to include project summary/abstract, specific aims, and a research strategy section which includes significance, innovation and approach. (5 page maximum)
- Proposals must also include a cover page, an investigator biosketch in NIH format and project budget, budget justification, and references. (not included in 5 page maximum)
- Budgets must be reviewed by your business manager prior to submission.
- Funding may not be used for investigator salary support. Staff salaries are allowable budget items.
- There are two submission deadlines per year. The 2013 deadlines are Monday, April 1 (awards announced Wednesday, May 1) and Tuesday, October 1 (awards announced Friday, November 1).
- Selected applications must show documentation of IRB submission within 30 days of award notification.
- Proposals submitted for other awards (e.g., Foerderer) are not eligible for CPCE pilot grant consideration.
- Please submit the proposal as a single word document via email to Linda Scott, Sponsored Projects Officer, at firstname.lastname@example.org no later than 4:00 p.m. on the deadline date.
For a submission template, please click here.
Review Process and Selection Criteria
As detailed above, the review process will consist of two rounds. Applications meeting the definitions (above) for pilot clinical effectiveness research studies, and judged by the Pilot Grant Steering Committee to be of sufficient quality for further review, will be assigned to a reviewer, critiqued and scored. Reviewers will meet in a study section to discuss the merits and limitations of the competing proposals and to determine the awardee(s). All applicants whose proposals qualify for round two will receive a copy of reviewers' comments and score. Those applications that do not qualify for round two will be returned to the applicant without further review, critique or score.
The following criteria will be used to score the proposals. They are adapted from NIH Study Section Criteria.
- Significance of Study: Does the project address an important problem or a critical barrier to progress in the field?
- Approach: Are the overall strategy, methodology, and analyses well-reasoned and appropriate to accomplish the specific aims of the project? Are potential problems, alternative strategies, and benchmarks for success presented?
- Likelihood of Impact of Clinical Effectiveness: If the aims of the project are achieved, how will scientific knowledge, technical capability, and/or clinical practice be improved? How will successful completion of the aims change the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field?
- Appropriateness of Budget: Is the proposed budget and period of support appropriate in relation to the proposed research?
- Likelihood of Future Research: If the aims of the project are achieved, will the results lend themselves to future research?
- Innovation: Does the application challenge and seek to shift current research or clinical practice paradigms by utilizing novel theoretical concepts, approaches or methodologies, instrumentation, or interventions? Are the concepts, approaches or methodologies, instrumentation, or interventions novel to one field of research or novel in a broad sense? Is a refinement, improvement, or new application of theoretical concepts, approaches or methodologies, instrumentation, or interventions proposed?
The above criteria will be scored on a scale from 1 (high impact) – 9 (low impact).
Common Pitfalls of Past CPCE Pilot Grant Applications:
- Failure to anticipate difficulties or address limitations
- Uncertainty regarding future direction or funding
- Incomplete significance section; failure to adequately describe rationale
- Overambitious for pilot study; unrealistic timeline
- (Budget) Underestimate of biostatistical support needed to complete aims
Although projects may be funded up to a maximum of $10,000 priority will be given to those projects that include a prudent spending plan. CPCE will attempt to fund all requested budgets in full, but may elect to partially fund certain protocols in order to increase the number of funded proposals.
- Principal investigators must submit a report at the end of the grant period for review by the Pilot Grant Program steering committee. The reports should include progress, expenditures and plans for disseminating results. Subsequent reports will be requested to include publications and extramural funding resulting from the research.
- Funds unspent at the end of the one-year grant period will be returned to CPCE. However, requests for rollover of funds for an additional year will be considered with adequate justification. We are very excited about the opportunity to support fellows and junior faculty who are interested in advancing CPCE's goals.
Contact Megan Foley at email@example.com with any questions about the CPCE Pilot Grant Program. The Office of Sponsored Projects will coordinate the submission process; questions regarding this process can be directed to Linda Scott at firstname.lastname@example.org.
The Healthcare Analytics Unit (HAU) is a service unit of two centers at CHOP: CPCE and The Policy Lab. Visit the Policy Lab website at: http://policylab.us/index.php
- Serves as a resource for investigators who want to use administrative or other existing data to answer research questions;
- Offers programmer/analysts services to pull, clean, manage, model and analyze data;
- Provides expertise of center faculty members with advanced training in clinical epidemiology and public health.
Examples of databases to which the HAU provides access and expertise:
- Pediatric Health Information System (PHIS)
- Kids' Inpatient Database (KID)
- NAMCS (National Ambulatory Medical Care Survey)
- NHDS (National Hospital Discharge Survey)
- Premier Perspective Database
To see samples of articles published using PHIS data, click here
To see samples of poster presentations using PHIS data, click here
For PHIS Data Use Forms, click here
Xianqun Luan MS, Director
3535 Market Street, Rm 1545
Mr. Luan has expertise in clinical trial studies (Phase I-III), biomedical research, and outcomes research. As a biostatistician, he has experience in clinical trial design and analysis, sample size estimation, implementation of statistical methods, statistical modeling for longitudinal, categorical, time to event and outcome data, and analysis of measurement agreement. Mr. Luan has extensive experience in statistical programming, statistical graphics, and statistical report presentation (SAS, Splus, and R). He has comprehensive knowledge about utilization and analysis of regional and national survey datasets, and he has strong database (Oracle, SQL server, Access, Filemaker, and Excel) integration, research data modeling, and analysis data generation skills.
Dingwei Dai, MSc, PhD, Programmer/Analyst
3535 Market Street, Rm 1544
Dr. Dai has expertise in healthcare database management and data analysis, statistical modeling, clinical epidemiology, and health outcomes research. His skills include statistical programming (SAS, SQL, Matlab, SPSS), data linkage, data sampling, propensity score matching, and statistical methods in epidemiology. Dr. Dai has extensive experiences with large medical, pharmacy, and patient registries; national health surveys; and clinical trials databases.
Vera Huang, MS, Programmer/Analyst
3535 Market Street, Suite 1536
Ms. Huang has expertise in statistical design/analysis, structural equation modeling, multivariate regression, and sampling methodologies. She is skilled with PC SAS, Unix SAS, PC SPSS, and SQL. Ms. Huang has extensive knowledge of PHIS, Medicaid, and NSCAW databases.
Lihai Song, MS, Programmer/Analyst
3535 Market Street, Rm 1536
Mr. Song has expertise in statistical modeling, experimental design, and analysis of large databases. He is skilled in designing, developing, and testing applications. As an SAS Certified Advanced Programmer, Mr. Song is experienced developing reports and models using SAS (SAS/BASE, SAS/ACCESS, SAS/MACRO, SAS/STAT, SAS/GRAPH). He has expertise with programming for clinical trials and for biological, pharmaceutical, and healthcare industries.
Requesting Data Services from the HAU
Complete and submit the HAU Data Services Request Form
We will contact you to schedule an appointment to discuss your project's feasibility and cost.
IRB APPROVAL FOR STUDIES THAT UTILIZE HAU SERVICES
The IRB has increasingly judged large datasets, such as those obtained from PHIS, to contain data that is not readily identifiable and therefore, outside the requirements for prospective IRB review and approval. Regulatory statements have made clear that "readily identifiable" does not mean possibly or potentially identifiable. This lowers the barriers for receipt and use of these datasets.
HIPAA does apply to the receipt/use of these datasets.
- If the dataset is a limited dataset, then all that is required to obtain and use it would be a data use agreement (no IRB approval required) with the data source (e.g. CHCA for PHIS data).
For research that utilizes datasets containing identifiable PHI, or for which data will be prospectively collected, IRB review/approval will continue to be needed.
It is the responsibility of the investigator to obtain IRB review and approval for those studies that require it, and to complete any data use agreements required by the data source.
For more complete information regarding what must be reviewed by the IRB, the use of registries/repositories for research, and other related topics, please see these sections of the research intranet site (authentication may be required):
SUBMITTING MANUSCRIPTS BASED ON RESEARCH UTILIZING DE-IDENTIFIED OR LIMITED DATASETS
When submitting a manuscript for publication, for a study that utilized PHIS or a comparable large and not readily identifiable dataset, and which therefore did not require IRB review/approval, the following boilerplate text can be used when needed during the journal's submission process. There are two versions, please choose the appropriate one, depending on the nature of your dataset.
1) In accordance with the Common Rule (45 CFR 46.102(f)) and the policies of the The Children's Hospital of Philadelphia IRB, this research, which utilized a de-identified dataset, was determined to not meet the definition of human subjects research.
2) In accordance with the Common Rule (45 CFR 46.102(f)) and the policies of the The Children's Hospital of Philadelphia IRB, this research, which utilized a limited dataset according to the terms of a valid data use agreement, was determined to not meet the definition of human subjects research.
Sample Table To help define your population and measures, inclusion/exclusion criteria, etc
ICD 9 codes -- Use ICD 9 codes in your table to define population of interest
CTC codes (©2008 Thomson Healthcare) please use the appropriate PDF from the list below:
- Medical Supplies
- Laboratory Services
- Imaging Services
- Clinical Services
- Other Transacted Services
If you have questions about HAU services, please contact:
Xianqun Luan, MS
CPCE Grants Database
In order to further support our goal of facilitating the performance of clinical research at CHOP, CPCE aims to support researchers in their grant writing endeavors by providing good examples of successfully funded grants. These samples help all to benefit from good ideas about grant formatting, methods, content and organization. We have created a central repository of grants by and for center members. The searchable listing is categorized and includes a complete document library for each grant.
Access to the Grants Database
Faculty who are center members and have a USER ID and password can click here to access the CPCE grants database.
Center members who do not have a USER ID/password for the database can request one by emailing email@example.com.
Those interested in becoming center members can also contact firstname.lastname@example.org.
Contribute to the Grants Database
We hope you will find this to be a useful tool. If so, we would greatly appreciate if you can support it by contributing one or more of your grants. This will give CPCE members a greater variety of examples of successfully funded grants. To submit a grant for uploading, simply e-mail the grant (no need to include the budget or budget justification) to center manager, Deb Hillman at: email@example.com.
Word documents are preferred, but other formats (e.g. pdfs) are also accepted.
Center for Simulation, Advanced Education and Innovation
CPCE members utilize the resources provided by The Center for Simulation, Advanced Education and Innovation at CHOP, which operates within the division of Anesthesiology and Critical Care Medicine. The Center facilitates the translation of scientific discoveries into practical implementation for both research and clinical care. The center is directed by Vinay Nadkarni, MD, and Evelyn Lengetti, RN, MSN., and is administered by Stephanie Tuttle, MBA.
Centralized classroom and skills lab training, satellite skills labs, and unit-based "virtual" lab exercises adjacent to patient care settings are routinely conducted. More than 250 teaching sessions are offered with at least 4,500 attendees, including:
- education encompassing recognition of shock and/or cardiac/respiratory failure
- vascular access, chest tube care and hemodynamic monitoring
- advanced and difficult airway assessment and intervention
- pacemaker evaluation
- EKG interpretation
- critical incident debriefing
- crew resource management
The education scope is expanding and includes basic and advanced life support training, simulation "boot camps" for orientation and training. This Center is supported by CHOP, and additional funding is supplemented with a federal Agency for Healthcare Research Quality (AHRQ) grant to study "training to excellence" using "just-in-time" and "just-in-place" simulation resuscitation education techniques for sharp end providers. The center staff includes multidisciplinary center directors, full-time administrative staff, a full-time clinical educator, as well as additional volunteer faculty (e.g. physicians, nurse, therapists, educators) to conduct training. The center also has non-overlapping research coordinator and research assistant support through funding from the AHRQ and Laerdal Foundation for Acute Medicine.
The is equipped with more than $500,000 of equipment in the form of:
- flat screen simulators
- virtual reality technology
- simulation mannequins with software
- task trainers
- video monitoring and programming equipment
- 10-12 PCs and monitors
- a network server with secure storage
- video editing hardware and software
- disposable supplies
In addition, there are collaborative links with the 7,000 square foot Brunner Technology Center at the Penn School of Nursing, which trains more than 8,000 simulation encounters annually, and the Measey Medical Simulation Center at the Penn School of Medicine. Both of these centers provide adjunctive expertise, equipment and personnel to accomplish training and research objectives. These resources have been mobilized and committed to support the evolving Laerdal-funded CHOP Center of Excellence for Resuscitation Research.