FORUM

PATIENT ENGAGEMENT IN HEALTH ECONOMIC AND OUTCOMES RESEARCH:

CURRENT AND FUTURE ISPOR INITIATIVES

Moderator FORUM

Todd Berner, MD Co-Chair Patient Engagement In Research Working Group ISPOR PATIENT CENTERED SPECIAL INTEREST GROUP

Speakers

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Rob Camp, Communications Manager, EUPATI, Barcelona, Spain

Amie Scott, MPH, Business Health Research Analyst, SEAS Capital Partners, LLC, Plymouth, MI, USA

Don Husereau, MSc, Senior Associate, Institute of Health Economics Adjunct Professor, Epidemiology and Community Medicine, University of Ottawa, Ottawa, Canada

Emil Chiauzzi, PhD, Research Director, PatientsLikeMe, Cambridge, MA, USA

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ISPOR Involvement with Patients PATIENT CENTERED SPECIAL INTEREST GROUP Patient Engagement In Research Working Group

PATIENT REPRESENTATIVE ROUNDTABLE European North American

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PATIENT ENGAGEMENT IN RESEARCH WORKING GROUP

Goal: To determine how best to involve patients and their representatives in the research process by identifying: The stages at which patients should be involved The level of their involvement in each stage The challenges that will face the researchers Recommendations

FORUM

Goal: provide an opportunity for patient representatives to learn and discuss how they can participate in the assessment of a new health technology (drug, medical device, diagnostics, biopharmaceutical, vaccines)

increase their involvement in health policy decision making.

Patient involvement in health technologies research Are they involved enough?

ISPOR 20th International Meeting Philadelphia PA 18 May 2015

The project is receiving support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n° 115334, resources of which are composed of financial contribution from the European Union's Seventh Framework

Programme (FP7/2007-2013) and EFPIA companies.

Patient involvement is patient engagement is not recruitment! (recruitment is recruitment) It is a process of utilizing the expertise we have to improve the research process and shape it together

What do I mean by patient involvement?

Why patient involvement/engagement?

We make the research better

A wide range of stakeholders can give research teams advice about research questions, procedures and conduct Stakeholders, especially community stakeholders (patients), have critical knowledge about local cultures and dynamics of the disease that trial sponsors/designers may lack. Their feedback can help ensure that the research and procedures are culturally sensitive and appropriate This advice can lead to better (and faster) recruitment, better retention, better adherence, better data, and better likelihood of uptake of interventions should they be safe and effective (ie, better research) It is their right

Are there Guidelines?

Standard practices for stakeholder engagement. They provide trial funders, sponsors, and implementers with systematic guidance on how to effectively engage with stakeholders in the design and conduct of clinical trials. YES, they exist and you can adapt any of them!

Community Stakeholders are: Patients and individuals and groups who are ultimately representing the interests of people who would participate in a trial. patient advocates, representatives ex-patients (cured, etc. with experience!) family, caregivers

Who is a Community Stakeholder?

Examples of Research Stakeholders

• Respect • Mutual Understanding • Integrity • Transparency • Accountability • Community Stakeholder Autonomy

Guiding principles

Where can it be applied?

Formative Research Activities Stakeholder Advisory Mechanisms Stakeholder Engagement Plan Stakeholder Education Plan Communications Plan Issues Management Plan Site Selection Protocol Development Informed Consent Process Current standards Access Co-morbidities and Care Trial-related harm Accrual, follow-up and exit Trial closure and dissemination Post-trial access

Stakeholder Advisory Mechanisms

Community Advisory Boards (CABs) are one of many ways (formal or informal) that research teams can engage with stakeholders

Ex. about Protocol Development

• involvement in defining the priorities in research activities

• involvement in better protocol design, e.g. defining trial endpoints

• involvement in benefit-risk assessment, • involvement in data monitoring committees

• A reminder of multidisciplinarity

Concrete involvement

Implementation, monitoring, and evaluation

• Impact – Did the engagement improve the research? How?

– Did stakeholders provide useful feedback on the design of the study and the protocol?

– How do various stakeholders feel about the quality of the engagement process and the relationships?

– Did community stakeholders feel their inputs and feedback were listened to and addressed?

– All stakeholders can be involved in the evaluation process and give their perspectives

Application/implementation

• It is a learning process! • It will be different in every setting. There

isn’t one answer. And there won’t be one answer.

• It will depend on country, institution, sponsor, etc.

EUPATI Training and Information • By 2017, there will be a searchable education website with

teachable and downloadable information in 7 languages – providing user-friendly information and education resources on the

research development process that will further community understanding of these issues

– create the leading public library of patient-friendly information on research development and research in the seven most common languages in Europe

– establish a sustainable website and information service to provide up-to-date information

– to show you how to get involved in research and development, to support ongoing and future research and clinical trials

– Supported by 4 patient umbrella org’s, 3 universities, 7 int’l NGOs and some 30 pharmaceutical companies; non- disease-specific, non-treatment specific

Our future

There are very competent and willing science-oriented partners in the patient community • EUPATI is working on increasing the number of

well-educated advocates • Education, education, education • Implementation, implementation,

implementation

Thank you

• To all of you

• www.patientsacademy.eu

• rob.camp@patientsacademy.eu

PATIENT ENGAGEMENT IN HEALTH ECONOMIC AND OUTCOMES RESEARCH: CURRENT AND FUTURE ISPOR INITIATIVES Emil Chiauzzi, Ph.D. Research Director PatientsLikeMe May 18, 2015

A patient network that improves lives & a real-time research platform that advances medicine

About PLM

patients data insights

• 25+ million structured data points

• 3+ million free-text posts • 10+ PROs • Increasing third-party data

integration

• 300,000+ patients • 2,000+ conditions • Ongoing

engagement • Deep trust

• 60+ peer-reviewed papers • Patient-generated

taxonomy • Safety monitoring platform • Endpoints in clinical trials • Dozens of biotech/pharma

relationships

What Members Do

• Document changes to symptoms and new treatments, triggers and side effects

• Generate a real-world outcomes research data

Learn about living with and treating life-changing

conditions

• Answer the question “Is this normal?”

• Learn from aggregated patient treatment and symptom data

Connect with others sharing those

experiences

• Search patients based on age, gender, disease, treatment, symptoms, interests, etc.

• Day-to-day support

• Live better, together

Track their history and progress

over time

Structured Data Overview

Patient Experience & QoL

PatientsLikeMe Data Fields Patient Reporters Stakeholders Data Utilization

Care Management

Access & Reimbursement

Research Registries

CER, PCOR, HEOR

• Industry • Regulators • Payers/Plans • Researchers • Providers • Patients

Conditions

Age

Gender

Race

Ethnicity

Location

Payer type

Active Safety Surveillance

Diagnostic Journey

Symptoms

Primary / Secondary

Hospitalizations

Comorbidities

Treatments

Effectiveness

Dates of Therapy Indication for Use

Adherence History

Side Effects

Stop Reasons

Advice / Tips

Related Data Labs, Tests, BMI

Free Text Narrative

Engagement Over Time

Member Communications

• 89% of logins are triggered by an engagement message

• These communications include monthly newsletters, blog posts, social media, etc.

• Dedicated team of Community moderators

Tracking and Social

• Patients can signup for reminders to update their profile

• Patients can view other member profiles and connect

• “Data for Good” campaign

Research Opportunities

• Patients can participate in longitudinal health studies with PLM partners

• PLM supplements traditional study recruitment by sending targeted outreach to potential subjects

Givebacks

• Funded by RWJF • World’s first open-participation

research platform for creating patient-reported health outcome measurements

• A hub where researchers, clinicians and patients can work together to increase the understanding of disease – faster than ever before

• Year 1 (2013): researcher-led measure development

• Year 2 (2014): patient-led measure development

www.openresearchexchange.com

Open Research Exchange

Wearables

“Out of box” experience

Tailored communications

Informed consent

IRB review

Research protocol design

Device procurement

Data architecture design

Product enhancements

Study participant selection

Study execution

Insight generation

Trial Access: Leveraging the PLM Platform

Attitudes towards clinical trial aspects (randomization, open access)

Opinions on pt involvement in CT

Triggers for interest in clinical trials

Exposure to media and offline marketing channels

Past experiences in clinical trials

Ability to participate in future trials

Concerns / factors in deciding to participate

Key domains relevant to trial design

X

Age Sex Geography Primary condition Quality of life Disease

progression

=

Ability to quickly identify trial issues before beginning trial

Design a more specific / targeted survey around a specific clinical program

Understand patient perspectives on clinical trials across conditions

Trial design effects

Stratification

PatientsLikeMe Publications

Contact Information Dr. Emil Chiauzzi, PhD PatientsLikeMe Research Director echiauzzi@patientslikeme.com 617-674-1846

As a newly formed SIG, to guide our direction and provide materials that are in line with the needs of the ISPOR community, we conducted an informal survey.

The objective of conducting this informal survey was to better understand how aspects of patient

engagement in outcomes research was perceived and implemented among the ISPOR community.

Setting

ISPOR Annual Meeting (Montreal, June 2-4, 2014)

ISPOR-EU Meeting (Amsterdam, November 8-12, 2014)

Structured Interview conducted by SIG members

Interviews guided by an survey tool consisting of

29 questions (closed and open-ended).

Purposive sample

Requested conversation regarding patient engagement with ISPOR members attending outcome sessions

Analysis of informal survey

Patient Engagement along Outcomes Research Continuum2

Preparation: Agenda setting

Execution: Study design, recruitment; Data collection and analysis

Translation: Dissemination, implementation and evaluation

1Carman, 2013; Exhibit 1: A multidimensional Framework for Patient and Family Engagement in Health and Health Care 2Domecq, 2014; Figure 3 Methods and Phases of Engagement

Framework: Preparation, Execution, Translation

Domecq, 2014

n = 142 studies

Primary Work Areas N=39 N=7443

Industry 22 (56.4%) 3385 (45.5%)

Health Research 7 (17.9%) 1871 (25.1%)

HealthCare Communications 0 (0.0%) 334 (4.5%)

Academia 7 (17.9%) 1101 (14.8%)

Managed Care 2 (5.1%) 116 (1.6%)

Government 1 (2.6%) 502 (6.7%)

Clinical Practice 1 (2.6%) 134 (1.8%)

Montreal meeting (n=21, 53.8%)

Amsterdam meeting

(n=18,46.2%)

* Primary work areas reported by ISPOR membership between September 15, 2013 –September 15, 2014

Respondent Characteristics

N=39 Yes No

Are you aware of any patient engagement activities, initiatives in your organization/industry/academia? 32 (84.1%) 4 (10.3%)

Awareness of Patient Engagement at current organization

N=39 Yes No Don’t Know

Brought patients/advocacy groups in to your organization to meet with researchers? 18 (46.2%) 7 (17.9%) ---

Involve patients in discussion with approval or review agencies? 11 (28.2%) 16 (41.0%) 2 (5.1%)

Personal Experience: Patient Engagement in Preparation Phase

N=39 Yes No Don’t Know

Involve patients or patient community in study design? 16 (41.0%) 10 (25.6%) ---

In research methods or clinical trials? 2 (5.1%) 25 (64.1%) ---

In write up of patient information leaflets? 4 (10.3%) 23 (59.0%) ---

In informed consent document creation? 4 (10.3%) 22 (56.4%) 1 (2.6%)

Collecting Data from patients? 29 (74.4%) 3 (7.7%) ---

Qualitative Data Collection ? 24 (61.5%) 8 (20.5%) ---

Quantitative Data Collection? 15 (38.5%) 17 (43.6%) ---

Involve patients in recruitment plans for studies? 3 (7.7%) 24 (61.5%) ---

Personal Experience: Patient Engagement in Execution Phase

N=39 Yes No

Involve patients in discussion of results to facilitate interpretation? 12 (30.8%) 16 (41.0%)

Personal Experience: Patient Engagement in Translation Phase

Challenges: Patient Engagement in Research ‘

It’s a bad idea’ ‘Not helpful if not complete information from patients’

‘Concerns about social media, challenges about regulation, approach may be seen

as commercial’ ‘Access to participants without all kinds of approvals, how to make interactive and a

win-win situation’

‘Costly, so should be need based’ ‘Patient advocacy groups feeling alienated due to budget constraints’

‘Keeping the underserved community engaged’

‘How prepared are patients to engage?’

‘Motivating clinicians to involve patients in clinical routine, patient follow-up, missing data after the intervention/engagement’

Conclusions

No shared notion of consistent structure of methodology for patient engagement in OR among the ISPOR community; No one mentioned any frameworks used.

Currently no standard checklist or structure in methodology that meets the needs of the ISPOR outcomes research community

Although our sample may be skewed, affirmed the need for a toolkit of practical methodology for use in health outcomes research

Update on Patient Representatives Roundtable Europe & CASE REPORT on patient involvement in payer healthcare decisions to access new therapies

FORUM

First European Roundtable - 5 November 2013, ISPOR 16th Annual European Congress in Dublin, Ireland

Second European Roundtable – 1- November 2014 - ISPOR 17th Annual European Congress, in Amsterdam, The Netherlands

Promote opportunities for collaboration

How to improve Ensure representation and understanding of patient perspectives in research? HTA?

How to help researchers understand patients and patients understand researchers and create linkages

1 page template developed by the European Roundtable similar to EUPATI Case Report for clinical research

Gather descriptions of HTA interactions with patients to

Understand good practices/successes

Understand continued challenges/barriers

Sign up as Review Group Member • Business card to Kelly • Sign-up sheet • Website: Interest Groups • Join ISPOR Special Interest Groups Select ISPOR Patient Centered Special Interest Group

FORUM SLIDES are AVAILABLE

Go to the ISPOR PHL MEETING home page and click on the orange Released Presentations menu OR via this link to ISPOR’s Released Presentations webpage:

http://www.ispor.org/Event/ReleasedPresentations/2015Philadelphia