Patient-Centeredness and Engagement in Clinical Research ... · Clinical Research: Opportunities...

2/5/2014

1

Patient-Centeredness and Engagement in Clinical Research: Opportunities for Industry

February 5, 2014

The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be

Disclaimer

attributed to Drug Information Association, Inc. (“DIA”), its directors, officers, employees, volunteers, members, chapters, councils, Special Interest Area Communities or affiliates, or any organization with which the presenter is employed or affiliated.

These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners.

2www.diahome.orgDIA

2/5/2014

2

Our speakers

Freda Lewis-Hall, MD, DFAPA Chief Medical Officer, Pfizer Inc.

Janet Woodcock MD

PCORI Board Member

Janet Woodcock, MDDirector, Center for Drug Evaluation and Research, FDA

DIA www.diahome.org 3

Our speakers

Anne Beal, MD, MPH Deputy Executive Officer andDeputy Executive Officer and

Chief Officer for Engagement, PCORI

Susan Sheridan, MBA, MIM


, ,Director of Patient Engagement, PCORI

2/5/2014

3

1. Share how PCORI, industry, FDA, patients and other healthcare stakeholders are advancing the cause of

Our objectives for today

healthcare stakeholders are advancing the cause of “patient-centeredness” and engagement in clinical research;

2. Discuss how increased patient engagement will lead to changes in clinical trial processes;

3. Begin to understand how these paradigm changes will impact the pharmaceutical industry’s efforts in clinical research.

5www.diahome.orgDIA

We Are at an Inflection Point

Where Are We Today?

At an inflection point on patient engagement in the drug/device/ diagnostics development processdiagnostics development process

What Do We Have Now?

A heightened sensitivity to transform all aspects of healthcare to focus more acutely on the unmet needs of the patient

What Do We Need?

A clear framework to engage patients and incorporate their feedback into d l t d l

We Need?development and approval processes

What Might a Patient‐Centered Process Look

Like? Patient engagement fully “built-in”

2/5/2014

4

Patient Focused Drug DevelopmentDevelopment at FDA

Janet Woodcock, MD

• Patient-focused drug development ti

Agenda: FDA’s Recent Activities

meetings

• Patient reported outcomes


2/5/2014

5

• FDA’s drug benefit-risk assessment considers severity of disease condition and degree of unmet

Patient-Focused Drug Development under PDUFA V

severity of disease condition and degree of unmet medical need— clinical context

• Patient-Focused Drug Development is part of FDA commitments under PDUFA V– Convene at least 20 meetings on specific diseases – Patient perspective helps inform our understanding of

the context for the assessment of benefit-risk and decision making for new drugs

– Input can inform FDA analysis both during and outside of review

• Disease areas that are chronic, symptomatic, and affect functioning and activities of daily living

Which 20 Disease Areas?Criteria Used for Nomination

g y g

• Disease areas for which important aspects of that disease are not formally captured in clinical trials

• Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affects how a patients feels, functions, or survives

• Disease areas that reflect a range of severity

• Disease areas that have a severe impact on identifiable sub-populations (such as children or the elderly)

• Disease areas that represent a broad range in terms of size of the affected population

2/5/2014

6

• In September 2012, FDA announced a preliminary set of diseases as potential meeting candidates

P bli i t th i ti ll t d th h

Identifying Disease Areasfor the Patient-Focused Meetings

– Public input on these nominations was collected through an online docket and at a public meeting held in October 2012

– Over 4,500 comments were submitted, which addressed over 90 disease areas

– FDA carefully considered these public comments and the perspectives of our drug review divisions at FDA

• FDA selected a set of 16 diseases selected to be the focus of meetings for fiscal years 2013-2015g y– This set was published in the Federal Register in April 2013– Another public process will be initiated in 2015 to determine the

set for fiscal years 2016-2017

Disease symptoms and daily impactsthat matter most to patients (draft questions)

1 Of ll th t th t i b

What Questions to Ask?

1. Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact?

2. Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition?

3 H h diti d it t3. How has your condition and its symptoms changed over time?

4. What worries you most about your condition?

2/5/2014

7

1. What are you currently doing to help treat your condition or its symptoms?

Questions for Patients regarding current approaches to treatment

y p

2. How well does your current treatment regimen treat the most significant symptoms of your disease?

3. What are the most significant downsides to your current treatments, and how do they affect your daily life? y

4. Assuming there is no complete cure for your condition, what specific things would you look for in an ideal treatment?

13

• Myalgic encephalomyelitis/chronic fatigue d (ME/CFS) A il 25 2013

Patient-Focused Drug DevelopmentMeetings Held in FY 2013

syndrome (ME/CFS) - April 25, 2013

• Human immunodeficiency virus (HIV) -June 14, 2013

• Lung cancer - June 28, 2013

Narcolepsy September 24 2013• Narcolepsy - September 24, 2013

2/5/2014

8

FY 2014 – 2015

• Alpha-1 antitrypsin deficiency• Breast cancer

Disease areas to be the focus of meetings for FY 2014-2015

Breast cancer• Chronic Chagas disease• Female sexual dysfunction• Fibromyalgia (December 10, 2013)• Hemophilia A, Hemophilia B, von Willebrand disease, and other

heritable bleeding disorders• Idiopathic pulmonary fibrosis• Irritable bowel syndrome, gastroparesis, and gastroesophageal

reflux disease with persistent regurgitation symptoms on proton-pump inhibitors

• Neurological manifestations of inborn errors of metabolism• Parkinson’s disease and Huntington’s disease• Pulmonary arterial hypertension• Sickle cell disease (February 7, 2014)

• Each meeting will result in a meeting report that will be posted on the FDA website

Product of Patient-Focused Meetings

p– The patient perspectives captured in these reports will

provide helpful insights for FDA reviewers • The Voice of the Patient: A Series of Reports from

FDA's Patient-Focused Drug Development Initiative – http://www.fda.gov/ForIndustry/UserFees/Prescription

DrugUserFee/ucm368342 htmDrugUserFee/ucm368342.htm– The Voice of the Patient Report: Chronic Fatigue

Syndrome and Myalgic Encephalomyelitis (PDF -267KB)

2/5/2014

9

• Describe a process NOT evidentiary standards

Final DDT Guidance

evidentiary standards

• Qualification process described for Biomarkers, Animal Models, and Clinical

http://www.fda.gov/downloads/Drugs/GuidanceComplicanceRegulatoryInformationi/Guidances/

17

Outcome Assessments (COA)

UCM230597.pdf

• EXACTA PRO f h

First Clinical Outcome Assessment Qualified 1/14

– A PRO for the measurement of symptoms of acute bacterial exacerbation of chronic bronchitis in patients with chronic obstructive pulmonaryobstructive pulmonary disease

18

2/5/2014

10

COA Qualification Projects Status Report by Stage (January 24, 2014)

COA DDT Stage Number in Stage

Initiation Stage 20

Initiation – DDT # assigned 11

Initiation – Letter of Intent (LOI) received 5

Initiation – revised LOI requested 4

Consultation and Advice Stage (C&A) 26

C&A – Initial Briefing Package requested 9

C&A – Active 17

Review Stage 3

19

49 COA qualification projects including: 35 PROs, 4 ClinROs, 3 PerfOs, 1 containing multiple elements including, PRO, ClinRO, ObsRO components, and 6 TBD (appropriate reporter will be based on additional research)

• Qualification projects actively underway for a wide variety of conditions, including but not li it d t

Ongoing COA Qualification Efforts

limited to:

• Multiple sclerosis• Cancer fatigue• Mild cognitive

impairment• Irritable bowel syndrome

• Functional dyspepsia• Community-acquired

bacterial pneumonia• Acute bacterial skin and

skin structure infections

20

y• Asthma• Cystic fibrosis• Depression• Non-small cell lung

cancer

• Ulcerative colitis• Crohn’s disease• Esophagitis

2/5/2014

11

• CDER partnering with multiple consortia, patient groups, academics, researchers, and others on COA qualification projects, including:– FNIH Biomarkers Consortium

Ongoing COA Qualification

– Critical Path Institute PRO-Consortium (includes 7 distinct working groups: Functional Dyspepsia, Irritable Bowel Syndrome, Non-Small Cell Lung Cancer, Rheumatoid Arthritis, Depression, Cognition)

– Critical Path Institute Coalition against Major Diseases (CAMD) Consortium

– Critical Path Institute Multiple Sclerosis Outcomes Assessments Consortium (MSOAC)Consortium (MSOAC)

– PROOF-C Cancer Fatigue Consortium• CDER is collaborating with NIH to explore potential qualification of

selected PROMIS measures• CDER continues to encourage instrument development and

qualification, particularly for pediatric populations, rare diseases, and other areas of unmet need

21

• FDA is actively engaged in incorporating the patient’s voice into drug development

Summary

the patient s voice into drug development• We are holding a series of disease-

specific, patient focused meetings under PDUFA 5

• We also are involved in qualifying many PROPROs

• These will be utilized in our structured benefit-risk assessments


2/5/2014

12

Anne C. Beal, MD, MPH Deputy Executive Director and Chief Officer for Engagement

DIA/PCORI Webinar

Patient-Centeredness and Engagement in Clinical Research: Opportunities for Industry

DIA/PCORI WebinarFebruary 5, 2014

PCORI

An independent non-profit funder of clinicalAn independent non profit funder of clinical comparative effectiveness research.

Authorized by Congress as part of the 2010 Patient Protection and Affordable Care Act (ACA).

24

2/5/2014

13

Why PCORI?

Research has not answered many questions patients face

People want to know which treatment is right for them

Patients need information they

Research has not answered many questions patients face

People want to know which treatment is right for them

Patients need information they can understand and use

25

can understand and use

Our Mission

PCORI helps people make informed health care decisions and improves health care delivery anddecisions, and improves health care delivery and outcomes, by producing and promoting high integrity, evidence-based information that comes from researchguided by patients,caregivers and the gbroader health care community.

26

2/5/2014

14

Our Growing Research Portfolio (as of January 2014)

Total number of research projects awarded : 279

Total funds awarded: $464.2 million

Number of states where we are funding research:funding research: 39 states (plus the District of Columbia and

Quebec, Canada)

15 27

828

2/5/2014

15

How We Engage Patients and Others in PCORI’s Work

29

“Research Done Differently”Patient engagement as a path to rigorous research

Tell us what PCORI should study

Help determine what we fund

Engagement

Tell us how we are doing

Help us share research findings

30

2/5/2014

16

Tell us what PCORI should study

Engagement

How We Pick Research Questions to Study

31

Help Determine What We Fund Help determine what we fund

Help Determine What We Fund

Engagement

Advisory Panels & Working Groups PCORI Reviewers

32

2/5/2014

17

Patient-Centeredness vs. Patient Engagement

Patient‐ Centeredness is a component of what PCOR is looking for in research applications

Patient engagement is about having patients as partners in research as opposed to merely subjects

Active engagement between scientists, patients, and stakeholdersC i i d i i l l d i i ll h h

Patient engagement is about having patients as partners in research as opposed to merely subjects

Active engagement between scientists, patients, and stakeholdersC i i d i i l l d i i ll h h

applications Does the project aim to answer questions or examine outcomes that matter to patients

within the context of patient preferences? Research questions and outcomes should reflect what is important to patients and

caregivers

33

Community, patient, and caregiver involvement already in existence or a well‐thought out plan

Community, patient, and caregiver involvement already in existence or a well‐thought out plan

Dissemination & Implementation

Blueprint for the PCORI Dissemination and Implementation Action Plan:

Help Us Share Research Findings

Engagement

Implementation Action Plan: D&I roundtable and webinar (July, 2013)

RFP issued to develop D&I plan (Feb, 2014)

September 2014 workshop of experts and stakeholders in implementation science and quality improvement

34

2/5/2014

18

The Case for Addressing the Implementation Gap

O i l H l h D liOptimal Healthcare Delivery

PCORI’s Blueprint for Dissemination and Implementation Targets the Gap New Investments in Knowledge

Implementation Gap to Improve Practice

Research + Practice

Current Knowledge and Practice

35

Facilitating Patient Partnership in Research

Engagement Awards Pipeline to Proposals

36

2/5/2014

19

Purpose of Engagement Awards

Projects designed to provide “wrap-around” support and enhance impact of our major research awards

NOT meant to be research, but meant to: Support knowledge of PCORI’s work, and inform about our program efforts

Training and development of “non-usual suspects” and others

Disseminate the results of our research to promote implementation into practice

Smaller awards, up to $250,000 total, and less than two years in length

Other objectives:Other objectives: Engage new groups who have not previously been involved with PCORI

Develop new mechanisms for disseminating research findings

Promote research done differently by supporting the engagement and partnering

37

Pipeline to Proposals

The chart below summarizes the three tiers of the Pipeline to Proposal Awards Program.

38

2/5/2014

20

What is Patient Engagement in Research?

39

Find Us Online

www.pcori.org

40

Check out our webinar on the Engagement Awards on February 13, 2014

2/5/2014

21

Meaningful Patient Engagement in Clinical Research

S Sh id MIM MBASue Sheridan, MIM, MBA

Director of Patient Engagement

41

Getting to know the Patient and Family Engagement Rubric

2/5/2014

22

Why develop a rubric?

• The rubric is a response to frequent

What is the rubric?

• The rubric is a framework that provides

How will the rubric be used?

• The rubric will be used as a guide forfrequent

questions from the patient and research communities asking what we mean by

that provides a variety of options for incorporating engagement, where relevant, into the research

guide for applicants, merit reviewers, awardees and Engagement Officers.

43

we mean by “engagement in research.”

the research process.

Elements of the Rubric

Planning the Study

Conducting the Study

Disseminating the Study ResultsDisseminating the Study Results

PCOR Engagement Principles

44

2/5/2014

23


Planning the Study




45

Rubric: Planning the Study

Planning the StudyFormulating Research Questions and Study Design

Patient partners participate in: Identifying the topic and developing the

research question to be studied

Examples: Epilepsy study: the patients and parents of patients with epilepsy

pose the question: which anti epileptic drugs best preserve

Formulating Research Q S

research question to be studied.

Creating the intervention to be studied (if applicable) and identifying comparators.

In identifying the goals or outcomes of the interventions to be studied.

Defining essential characteristics of study participants.

Other study design and preparation.

pose the question: which anti-epileptic drugs best preserve sufficient cognition to go to work or school and function normally, while still preventing seizures adequately?

Asthma study: the patients and patients' parents help create the paper asthma tracker tool being compared to the e-asthma tracker tool.

Cancer study: patient partners determine that all women with breast cancer would be eligible versus only women who had completed active treatment.

How can you demonstrate this in your proposal? Provide letters of support from patient partners that clearly

describe the origin of the study topic, the role of the patient partners in defining the question outcomes comparators and

Questions and Study DesignPatient partners participate in:

• Identifying the topic and developing the research question to be studied.

• Creating the intervention to be studied (if applicable) and identifying comparators.I id tif i th l t f thpartners in defining the question, outcomes, comparators, and

goals/outcomes, etc.

Include the patient partners in all relevant sections of the application, such as the biosketches, the budget, and the dissemination and implementation assessment.

Avoid relying entirely on patient partners who have dual roles on the project, e.g., relying on stakeholders or researchers who also happen to be patients. Including one or more patient partners who have no other role on the project is important.

46

• In identifying the goals or outcomes of the interventions to be studied.

• Defining essential characteristics of study participants.

• Other study design and preparation.

2/5/2014

24


Planning the Study




47

Rubric: Conducting the StudyConducting the StudyParticipating in and monitoring the conduct of the project

Patient partners participate in and monitor the conduct of the research project.

Examples: Chronic pain study: the informed consent document is developed with patient partners to make it understandable to

study participants.

Epilepsy study: patient (and parents of patient) partners suggest that an adult survey tool be adapted for children to answer, and they help develop one.

Depression study: patient partners advise researchers to substitute the term, “emotional well-being,” for the term, “mental health,” to enhance the recruitment of study participants.

Participating in and monitoring the conduct of the project

, y p p

How can you demonstrate this in your proposal? Provide letters of support from patient partners that clearly describe the role of the patient partners in conducting and

monitoring the study.

Clearly articulate in the application the roles of the patient partners in each component of the study, (e.g., helping to draft survey tools and focus group questions, reviewing participant materials for readability, etc.), including the dissemination and implementation assessment.

Patient partners participate in the recruitment and data collection from the study participants, when appropriate.

Examples: Depression study: patient partners are trained to go out into the community to recruit study participants and to

conduct interviews with them.

Example:

Depression study: patient advocacy groups assist with recruitment through their patient networks—the “book club” model.

H d t t thi i l?

Patient partners participate in and monitor the conduct of the

research project.

48

How can you demonstrate this in your proposal? Provide letters of support from patient partners that clearly describe the role of the patient partners in interacting with

study participants, if appropriate.

Clearly articulate in the application the roles of the patient partners in interacting with study participants (e.g., recruiting participants, conducting interviews, leading focus groups, etc.).

The research team, including patient partners, participates in all potential evaluation activities of patient engagement.

Example: ER study: The PI regularly asks the patient partners if they feel that they are truly involved in the research and if they

think that their involvement is contributing to the research.

How can you demonstrate this in your proposal? Include in your application a plan for “check-ins” with patient partners to monitor their perceptions of the extent to

which a) they are meaningfully involved in the study and b) their participation is contributing to the study.

Also include a plan for “check-ins” with the other research team members to monitor their perceptions of the extent to which a) patient partners are meaningfully involved in the study and b) their participation is contributing to the study.

2/5/2014

25




study participants.




, y p p







Example:


H d t t thi i l?

Patient partners participate in the recruitment and data collection from the study

participants, when appropriate.

49













study participants.




, y p p







Example:


H d t t thi i l?

The research team, including patient partners, participates in all potential evaluation activities

of patient engagement.

50










2/5/2014

26


Planning the Study




51

Rubric: Disseminating the Study Results

Disseminating the Study ResultsHelping to plan the

dissemination of the study’s

l

Patient partners are involved in plans for

disseminating the study’s findings to patient,

k h ld d h di h h

Examples:

Chronic pain study: patient partners co‐author manuscripts,

i ifi d l f d h d

Helping to plan the dissemination of the study’s resultsresults. stakeholder, and research audiences so that the

findings are communicated in understandable,

usable ways.

present at scientific and lay conferences, and share study

findings through their networks.

Cardiac study: a Patient Dissemination Board is helping to craft

the dissemination plan and advise the research team on how to

best share study findings.

How can you demonstrate this in your proposal?

Provide letters of support from patient partners that clearly

describe the role of the patient partners in planning the

dissemination of the study’s results.

In the application, clearly identify the role of patient partners in

planning the dissemination of the study’s findings.

the study’s results.

Patient partners are involved in plans for disseminating the study’s findings to patient,

stakeholder, and research audiences so that the findings are communicated in understandable,

usable ways

52

usable ways.

2/5/2014

27


Planning the Study




53

Rubric: PCOR Engagement Principles

PCOR Engagement PrinciplesReciprocal Relationships The roles and decision-making authority of

all research partners, including patient t l l t t d

Examples: Many applications state that patient partners are co-

i ti t d th t d i i b t th t d dReciprocal Relationships

partners, are clearly stated. investigators, and that decisions about the study are made by consensus among all the research project partners.

Many applications describe patient partners as key personnel, and their biosketches illustrate how the skills and experiences of the patient partners prepare them to function effectively in this role.

p p

The roles and decision-making authority of all research partners,

including patient partners, are

54

clearly stated.

2/5/2014

28


PCOR Engagement PrinciplesCo-learning The application includes plans to ensure that

the patient partners will understand the h d th h ill

Examples: Training and educational opportunities are provided such

ti t t t i i i h bj t t tiCo-learning

research process and the researchers will understand patient centeredness and patient engagement.

as patient partner training in human subjects protection.

Training is provided by patient advocacy organizations, patient/survivor, and clinician/caregiver for the researchers providing the intervention (e.g., training in better communication with patients, led by patient instructors).

g

The application includes plans to ensure that the patient partners

will understand the research

55

process and the researchers will understand patient centeredness

and patient engagement.


PCOR Engagement PrinciplesPartnership Time and contributions of patient partners

are valued and demonstrated in fair financial ti ll bl d

Examples: Compensation for patient partners is included in the

b d t t k t t f lt tPartnership

compensation, as well as reasonable and thoughtful time commitment requests. When the patient partners represent unique populations, the research team proposes to accommodate their cultural diversity and/or disability.

budget at market rates for consultants. In a study focused on a Latina population, several

members of the research team are Hispanic and fluent in Spanish.

In a project with a patient partner with a disability, the research team selects sites for team meetings that are accessible.

p

Time and contributions of patient partners are valued and demonstrated in fair financial compensation, as well as reasonable and

thoughtful time commitment requests.

56

When the patient partners represent unique populations, the research team proposes to accommodate their cultural diversity and/or

disability.

2/5/2014

29


PCOR Engagement PrinciplesTrust, Transparency, Honesty

a) Major decisions are made inclusively and information is shared readily with all

h t

Example: Commitments to trust, transparency, and honesty are

t t d i li ti d t d bTrust, Transparency, Honesty

research partners, b) Patient partners and research partners express commitment to open and honest communication with one another. c) The study team commits to communicate the study’s findings back to the study community in a meaningful and usable way.

stated in many applications – and supported by descriptions of how the research team will communicate with each other frequently, and make decisions about the study by consensus.

p y y

• Major decisions are made inclusively and information is shared readily with all research partners,

• Patient partners and research partners express commitment to open and honest

57

p pcommunication with one another.

• The study team commits to communicate the study’s findings back to the study community in a meaningful and usable way.


Planning the Study




58

2/5/2014

30

Have Questions?

W l tiWe welcome your questions and comments at

[email protected]

Find Us Online

www.pcori.org

2/5/2014

31

An Industry PerspectivePerspective

Freda Lewis-Hall, MD, DFAPA

Where Are We Today?

Companies Are:

• Devoting new resources and staff roles to integrating patient engagement into product development lifecycle

• Seeking alignment with regulators and patients/patient advocates on the definitions of patient engagement and its fundamental activities

• Partnering with patient advocacy groups to develop new


treatments for rare diseases where patients/groups collaborate in research

– Sanofi/Michael J. Fox Foundation – Pfizer/Cystic Fibrosis Foundation

2/5/2014

32

1. A clearly articulated and transparent pathway to incorporate the patient’s voice consistently, including a clear pathway/guidance for:

I t t i t th ti t’ i i t th d l t l

What Actions by FDA will Help Industry be More Patient-centered?

– Innovators to incorporate the patient’s view into the development plan, – Regulators to incorporate results into product review and labeling– Clinicians and patients to incorporate new info into joint decision-

making

2. Regulatory consensus on both accepted methods for patient engagement and specific guidance on how to use gathered information in product development plans

3. Uniform standards accepted by all FDA divisions and other regulators -- and within those standards, flexibility to accommodate differences in patient populations, culture, geography, and diseases


1. A more holistic viewpoint on how to bring greater value to the healthcare system through patient

What Actions by PCORI will Help Industry be More Patient-centered?

a ue to t e ea t ca e syste t oug pat e tengagement;

2. The methods and tools to fully engage patients across the research spectrum, (“the leads to get to those needs”);

3. Examples of patient engagement models –resulting in substrate we can pick up and carry forward.


2/5/2014

33

• Identify existing gaps in processes

How Can Industry Help Itself Become More Patient-centered?

• Explore innovative models of patient engagement

• Increase focus on external collaborations to drive engagement


So What Is Our Plan of Action?


2/5/2014

34

Thank you


Date post:	17-Aug-2020
Category:	Documents
Upload:	others
View:	2 times
Download:	0 times

Patient-Centeredness and Engagement in Clinical Research ... · Clinical Research: Opportunities...

Documents