PCORI In Practice: Highlighting Opportunities for ... · research, public health, and prevention...

PCORI In Practice: Highlighting Opportunities for Infectious Disease ResearchDecember 3, 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.

http://www.pcori.org/get-involved/pcori-in-practice/

Today’s Presenters

Greg Frank, PhDProgram Officer for Science & Research Policy, IDSA

David Hickam, MD, MPHProgram Director, Clinical Effectiveness Research, PCORI

Jeff Gerber, MD, PhDPCORI Researcher, Attending Physician, Division of Infectious Diseases, Children’s Hospital of Philadelphia (CHOP)

Agenda

2:00 - 2:05 p.m. Welcome and Introductions Greg Frank, PhD, Program Officer for Science & Research Policy, IDSA

2:05 p.m. – 2:10 p.m.

IDSA Introduction Greg Frank, PhD, Program Officer for Science & Research Policy, IDSA

2:10 – 2:25 p.m. PCORI Introduction and Overview Dave Hickam, MD, MPH, Program Director, Clinical Effectiveness Research, PCORI

2:25 – 2:45 p.m. IDSA Researcher Perspective

Jeff Gerber, MD, PhD, PCORI Researcher, Attending Physician, Division of Infectious Diseases, Children’s Hospital of Philadelphia (CHOP)

2:45 – 3:00 p.m Question and Answer Session Dave Hickam, MD, MPH, Program Director, Clinical Effectiveness Research, PCORI

3:00 p.m. Adjourn

Goals for Today

• Familiarize the infectious diseases community and researchers with PCORI, what PCORI funds, and future use of PCORI funded research findings

• Engage IDSA members and the broader infectious diseases community more actively in PCORI’s work and funding

• Encourage the community of infectious disease physicians and researchers to activate their research capacity for conducting patient-centered comparative effectiveness research

• Explore what other players in the infectious diseases community are doing in the way of patient and other stakeholder engagement

Copyright © 2015 Infectious Diseases Society of America All Rights Reserved.

TitleSub-caption

Greg Frank, PhDProgram Officer for Science & Research Policy

[email protected]


What is the Infectious Diseases Society of America?

IDSA represents over 10,000 physicians and scientists specializing in infectious disease (ID) patient care, research & prevention

5%8%

3%

14%

3%54%

4%

7%2%

Administration

Basic Research

Clinical Microbiology

Clinical Research

Hospital Epidemiology

Patient Care

Public Hlth

Teaching/Education

Other

Primary Professional Activity


What does IDSA do?

• IDSA’s purpose is to improve the health of individuals, communities, and society by promoting excellence in patient care, education, research, public health, and prevention relating to ID

• IDSA’s strategic priorities:• To promote the value of the ID specialist and attract the best and brightest to ID

• To promote ID leadership in antimicrobial resistance and antimicrobial stewardship

• To continue to produce useful, timely, and relevant clinical guidelines

• To promote ID/HIV research and its clinical translation

• To advocate for essential funding for critical prevention and public health programs in

ID/HIV


Promoting ID and HIV Research

• Advocacy for increased funding to ID research (NIAID, CDC, FDA,

BARDA, etc.)

• Reducing unneeded regulatory burden in research

• Supporting trainees interested in pursuing ID research careers

• Outreach program with APSA, the American Physician Scientists

Association

• Annual Research Careers Meeting with NIAID

• Medical scholars program

• High Impact Journals to disseminate research:

• CID (8.886), JID (5.997), and OFID

• IDWeek: 2016 Meeting in New Orleans


IDSA and PCORI

• IDSA firmly believes PCORI can help address major issues in ID patient needs, and

is working to increase ID representation in PCORI’s funding portfolio

• 2013: Held a conference call with Dr. Joe Selby, executive director of PCORI, to

discuss how PCORI can address ID patient research questions

• 2014: IDSA submitted a letter to Dr. Selby highlighting ID patient issues for PCORI

to consider as it develops its funding portfolio

• 2015: IDSA nominee Dr. Robert Bonomo is selected to the PCORI Advisory Panel

on Assessment of Prevention, Diagnosis, and Treatment Options

• 2015: Joint PCORI-IDSA Webinar on PCORI ID funding opportunities

• Next Steps: Develop an educational document for IDSA members with tips when

pursuing PCORI funding.


Visit Idsociety.org for more information

Greg Frank: [email protected]

www.hivma.orgwww.Idweek.org

mailto:[email protected]

http://www.idsociety.org/

http://www.hivma.org/

http://www.idweek.org/

The Patient-Centered Outcomes Research Institute—Studying What Works for Whom

David H. Hickam, MD, MPH

Director, Clinical Effectiveness Program

• Review PCORI’s model of patient-centered outcomes research• Introduce PCORI’s funding programs• Describe our approach for ensuring that research projects

provide results that are useful for decision makers

Goals for this Presentation

Our Research Focus

How is Our Work Different?

• We fund research on which care options work, for whom, under which circumstances.

• We aim to produce evidence that can be easily applied in real-world settings.

• We engage patients, caregivers, clinicians, insurers, employers and other stakeholders throughout the research process.

• This makes it more likely we’ll get the research questions right and that the study results will be useful and taken up in practice.

Research that….

• Generates evidence comparing benefits and harms of at least two different methods to prevent, diagnose, treat, and monitor a clinical condition or improve care delivery

• Measures benefits in real-world populations

• Describes results in subgroups of people

• Informs a specific clinical or policy decision

Note: We do not fund cost-effectiveness research

We Fund Comparative Clinical Effectiveness Research

Clinical evidence: Valid data about the outcomes experienced by patients who receive specific medical interventions• The population is well defined• The clinical interventions are well defined• We have information about the most important outcomes

(both benefits and harms) associated with specific clinical interventions

What is Evidence-based Information?

• Examine the choices people make about the options for managing a disease

• Consider how compelling it is to make a choice among these options

• Consider how the need to compare these options could inform the focus of new research

• The research should compare the benefits and harms associated with each option

What is the Starting Point of Comparative Effectiveness?

• Assess the evidence about available options and their important outcomes• Systematic reviews• Evidence gaps that are important to decision makers

• Design a study that can feasibly close the evidence gap• If the gap is not important, the research will not be

useful

New Comparative Effectiveness Research Must Address Important Evidence Gaps

Our National Priorities for Research

Assessment of Prevention, Diagnosis, and Treatment Options

Research that:

• Compares the effectiveness and safety of alternative prevention, diagnosis, and treatment options

• Determines which ones work best for different people with a particular health problem



Improving Healthcare Systems

Research that:

• Compares health system–level approaches to improving access

• Supports patient self-care, innovative use of health information technology, care coordination for complex conditions, and effective workforce deployment




Communication and Dissemination Research

Research on:

• Providing information produced by CER

• Empowering people to ask for and use the information

• Supporting shared decision making between patients and their providers





Addressing Disparities

Research on:

• Prevention, diagnosis, or treatment effectiveness

• Preferred clinical outcomes across patient populations

• Health care required to achieve best outcomes in each population





Addressing Disparities

Accelerating Patient-Centered Outcomes Research and Methodological Research

Research on:

• Building data infrastructure

• Improving analytic methods

• Training researchers, patients, and other stakeholders to participate in this research

We Also Work to Improve Research Methodology

In any study, methods matter. That’s why we’ve developed methodology standards that patient-centered CER should follow, at a minimum.

Methodology Standards: 11 Broad Categories

• Formulating Research Questions

• Patient-Centeredness

• Data Integrity and Rigorous Analyses

• Preventing/Handling Missing Data

• Heterogeneity of Treatment Effects

• Data Networks

• Data Registries

• Adaptive and Bayesian Trial Designs

• Causal Inference

• Studies of Diagnostic Tests

• Systematic Reviews

Pragmatic Clinical Studies

Overview

• Anticipated Awards per Funding Cycle: Six to Nine

• Funds Available per Cycle: Up to $90 Million

• Maximum Project Duration: 5 Years

• Maximum Direct Costs per Project: $10 Million

Seeks to produce information that can be directly adopted by providers:

• Compares two of more options for prevention, diagnosis, treatment, or management of a disease or symptom

• Addresses critical clinical choices faced by patients, caregivers, clinicians, and systems

• Often conducted in routine clinical settings• Though often large, usually less complex

protocols than traditional trials• Topics of special interest from stakeholders,

Institute of Medicine, Agency for Healthcare Research and Quality

Thank You

David Hickam, MD, MPHDirector, Clinical Effectiveness Program

[email protected]

Engaging Patients in Research

Jeffrey S Gerber, MD, PhDDivision of Infectious Diseases

The Children’s Hospital of Philadelphia

Overview

• key features of what PCORI seems to want

• how we approached this

What is patient centered research?

• evaluation of questions and outcomes meaningful and important to patients and caregivers

• assumes that patients have unique perspectives that can change and improve the pursuit of clinical questions

Frank et al, JAMA October 15, 2014 Volume 312, Number 15


• including the perspectives of end users of the research, which include patients, physicians, and other health care stakeholders, will enhance the relevance of research to actual health decisions these end users face

• increased relevance is hypothesized to:– improve uptake of the evidence– improve the likelihood that patients will achieve the

health outcomes they desire



• PCORI directs applicants to engage patients and other stakeholders or their organizations throughout study planning, conduct, analysis, and dissemination

Selby et al, JAMA December 1, 2015, Volume 314, Number 21

The Patient Perspective• patient input, guided by researchers, informs standard

research design components(inclusion/exclusion criteria, comparators, outcomes)

• practical reasons to incorporate patient-centeredness in research:– improved choice of research questions– improved selection and refinement of outcomes

through elicitation of stakeholder perspectives– enhanced accrual and participant retention strategies– more appropriate dissemination and implementation

strategies for findings


PCORI Review Criteria

• impact of the condition on the health of individuals and populations

• potential for the study to improve health care and outcomes

• technical merit• patient-centeredness• patient and stakeholder engagement


Assessment of Merit Review• 1 patient, 1 other stakeholder, and 2-3 scientists• PCORI convenes all reviewers to discuss higher-scoring

applications.• quantitative analyses of scores:

– participation of stakeholders leads to a different overall ranking of proposals than ranking by scientist scores alone (scores change after discussion)

• strongest predictors of final overall scores:– scientists’ preliminary scores for technical merit– all reviewers’ preliminary scores on the potential to

improve health care and outcomes

Selby et al, JAMA December 1, 2015, Volume 314, Number 21

COMPARATIVE EFFECTIVENESS OF ANTIBIOTICS FOR RESPIRATORY INFECTIONS

CEARI

• Family Advisory CouncilKathryn Conaboy, Darlene Barkman

• Primary Care PediatricsLou Bell, MD Alex Fiks, MD Mort Wasserman, MD

• Pediatric Infectious DiseasesTheo Zaoutis, MD, MSCE Jeff Gerber, MD, PhD Julie Szymczak, PhDBiostatistics/data managementRussell Localio, PhD, Matt Bryan, PhD

• Pediatric Research Consortium LiasonJim Massey, RN

Study Team

The Problem• antibiotics are the most common medicines prescribed to

children, usually for acute respiratory tract infections (ARTIs)

• experts recommend narrow-spectrum drugs as first-line (penicillin, amoxicillin)

• but … broad spectrum drugs used 50% of the time (amoxicillin-clavulanate, azithromycin, cefdinir)

• so, which are better?• and how do you assess this?

Study Setting: CHOP Care Network

29 practices-urban, suburban, rural

2009 data:>1 million encounters>200 clinicians>200,000 children

Broad Antibiotic Prescribing

Excluding: preventive visits, CCC, antibiotic allergy, prior antibioticsStandardized by: age, sex, race, Medicaid

CEARI: Project AimsSpecific Aim 1• identify outcomes most important to parents of children

receiving antibiotics for ARTIs– conduct in-depth, semi-structured interviews with

parents of children at acute office visits to identify, refine, and prioritize outcomes most important to caregivers for future CE study

CEARI: Project Aims

Specific Aim 2• compare rates of Tx failure and ADEs between

narrow-spectrum and broad-spectrum antibiotics for common ARTIs (ear infections, sinus infections, strep throat)

• outcomes informed by AIM 1 interviews• assessed by phone interviews 10 and 30 days

after antibiotic use

CEARI: Aim 1

• both open-ended and closed-ended (a priori generated outcomes) questions about outcomes important to parents

• 109 subjects from 4 different practices

CEARI: Aim 1

• open-ended questions:– what concerns you the most about your child’s

symptoms?– what are your expectations for this visit?– how do you feel when your child is prescribed

an antibiotic? – when your child is ill, how does it impact your

family’s daily life?

Sleep“[My expectations for this visit] is to get her better to actually let her get some sleep.” [P]

“Oh yeah, sleep. Sleep is the big one. Sleep, them eating, not feeling well, that is the concern I have.” [SP]

“We don’t get enough sleep and everybody is kinda cranky because if one of them is sick then they are cranky which makes every around them cranky. But mostly the no sleep you don’t get a lot sleep when they are sick.” [H]

Child’s Suffering

“It disturbs the child’s routine and psychologically it impacts the child too.” [K]

“My expectation is for him to be fine and a happy kid again…When he is not happy, the family cannot be happy.” [P]

“It just makes you feel bad when you’ve got a sick child at home. You can see it all in their face. You just wanna save them. So thank god for antibiotics and doctors.” [SP]

Missed School, Work“The fact that she is sick and she is not able to do her school work. Falling behind in school. There is so much schoolwork and especially at this time of year, she is a senior and to miss any time, you know, and when she isn’t feeling good she can’t do her homework. It all snowballs, it’s horrible.” [P]

“[When my children are ill it impacts our family’s daily life] tremendously so I would say because I have to take time off of work, I don’t have any more sick days because of my maternity leave and so I don’t have any sick days technically so taking a day off like today is unpaid and it doesn’t look good for me.” [H]

“I have to take off work to be with them because I don’t like to push them onto other people when they are sick.” [K]

Speed of Symptom Resolution

“I am a fan of antibiotics just because of the fact that it does heal them pretty quickly.” [H]

“I think [antibiotics] are wonderful. They clear up everything quickly. As long as you take them how the doctor prescribes, the infection is gone.” [K]

“All I can say is, antibiotics work. That is the only thing I can say. When we use it right it works. It helps them get better quickly.” [SP]

Closed-Ended Questions: Treatment Failure

0

20

40

60

80

Worried Somewhat Worried Not at all Worried

Obs

erva

tions

Q1: The antibiotic won't work and you'll need to come back to your child's doctor to get a new

antibiotic prescription?

01020304050607080

Worried SomewhatWorried

Not at allWorried

Don't know

Obs

erva

tions

Q2: The antibiotic won't work and your child will need to be hospitalized?

0

10

20

30

40

50

60

Worried Somewhat Worried Not at all Worried

Obs

erva

tions

Q3: The antibiotic won't work and your child's infection will get much worse?

Closed-Ended Questions: Impact on Life

0

20

40

60

80

100

Very Important Somewhat Important Not Important

Obs

erva

tions

Q9: That his/her symptoms resolve quickly?

010203040506070

Very Important SomewhatImportant

Not Important Don't know

Obs

erva

tions

Q10: That he/she gets back into school/daycare quickly?

01020304050

Very Important SomewhatImportant

Not Important

Obs

erva

tions

Q11: That you/your child's caregiver don't/doesn't have to take too much time off of

work?

Final Thoughts• operationalized these findings by incorporating these

concepts into parent interviews• as with all other steps, the FAC helped to shape the

content and format of data collection• engaging primary care clinicians to help refine outcomes

for EHR cohort• partner with FAC reps and clinician stakeholders to

interpret the analyses and disseminate results

Thank YouJeffrey S Gerber, MD, PhDDivision of Infectious DiseasesThe Children’s Hospital of Philadelphia

[email protected]

Question and Answer Session

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 [email protected]

An archive of this webinar will be posted to http://www.pcori.org/get-involved/pcori-in-practice/ following this event.

Accessing this Webinar:


http://www.pcori.org/get-involved/pcori-in-practice/

Have More Questions?

General [email protected] | (202) 827-7200

Research/Programmatic [email protected] | (202) 627-1884

Administrative/Financial/Technical Questions

[email protected]

Find us Online at:www.pcori.org

54


http://www.pcori.org/

PCORI In Practice: Highlighting Opportunities for ... · research, public health, and prevention...

Documents

Transcript of PCORI In Practice: Highlighting Opportunities for ... · research, public health, and prevention...