Copyright © 2013 Quintiles

Advance Research by Working Directly with Patients

ISPOR US 2013

New Orleans

23 May 2013

John Reites

Director of Operations

Digital Patient Unit

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#012-064

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Patient Involvement in Research

We are being asked to do more with less

Decrease in availability of funds for research

Increase in stakeholder demand for real-world data post-approval

Timelines are being cut

Why we need it…

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Patients Are Active & Eager to Participate

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Patient Involvement in Research

Innovation potential for research

Why we need it…

Faster

Enrollment

Increased

Retention

Decreased

Costs

Upfront study design more patient-friendly

Recruitment and retention activities relevant to patients

Patient outreach to supplement recruitment

Patient engagement during study (and beyond)

Remote visits (hybrid virtual study designs)

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Direct-to-Patient Innovation

* Patient protocol assessment for feedback on:

• I/E criteria

•Barriers & facilitating messages

* Supplemental patient recruitment

* Retention

* Pre-registration

* Alumni communities

* Direct recruitment of patients (no sites)

* On-line patient consent & screening

* Capture of ePRO data

* Direct outreach to patients

* Online consent and ePRO

* Patient consent for medical record access

* Patient provision of DNA or blood sample

* Device integration

(all without sites)

* Direct patient outreach & pre-qualification

* Physician visit for screening & randomization

* Remote visits with ePRO; in-person visits for physician endpoints or risk

* Would work well for pragmatic trials

Clinical Study Support

Observational PRO Data

Observational PRO+EMR+Lab

+Device

Hybrid Virtual Clinical Trials

Now Next

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Innovation Thru Technology

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Balance to Direct-to-Patient

Benefits

> More rapid study launch and

shortened timelines

> Decreased costs compared to

the physician-centric model

> Strong patient interest in method

- Helping others

- Alignment of patient incentives

- Comprehensive condition

monitoring and tracking

> Patient perspective directly

provided

Limitations

> Questions about data quality

- Verification of patient diagnosis

- Self-reported data

- Length of recall

- Lack of randomization

> No physician involvement

> Regulatory requirements in

process of being defined in some

countries

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Future: Virtual Research Driving greater efficiencies through direct patient engagement

• Rapid and Efficient Enrollment

> Direct-to-patient recruitment

> On-line screening and consent

• Continued patient engagement

> Retention activities

> Remote lost to follow-up tracking

• Next stage in patient involvement = virtual consent and/or remote patient visits

> Reduces site and patient burden

> Reduces investigator grant fees

> Reduces site monitoring visits

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Direct-to-Patient Research Case Studies

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Case Study Example:

Burden of Illness in MS Walking Ability

• Walking impairment was associated with

increased falls and severe falls led to

hospitalization/ER use and doctor’s visits

• Objective:

• To assess the clinical,

psychological and economic

impact of differences in walking

ability in patients with MS

• Approach:

• 346 US patients with MS

completed an on-line study in 2

weeks

– PDDS=0: 106 (31%)

– PDDS of >0: 240 (69%)

• Walking ability was significantly

linked to increased:

– Falls

– Depression

– Absenteeism

– Activity restriction

Conclusions

Background Findings Related to Falls

Compliance with Daily Diary (n=74 Subjects)

14%

62% 50%

0%

50%

100%

PDDS 0 PDDS 3 PDDS 4

% of Patients that “Sometimes” Fall Because of MS by PDDS Score

2% 2% 11% 16%

32% 27%

0%

50%

100%

Hospital / ER Doctor's Office

Resource Utilization for Severe Falls by PDDS Score

PDDS 0 PDDS 3 PDDS 4

Source: Sidovar M, Horowicz-Mehler N, Hawryluk E, Cascade E, McCarthy S: Patient-Reported Burden of Walking

Impairment in Multiple Sclerosis. ISPOR June 2012.

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Case Study Example:

PRO + Chart Data in US Gout Patients

• Although concerns exist regarding the

validity of self-reported diagnosis, this

PRO+MR pilot shows nearly all (37 of

38) charts confirm patient data

• Objective: To collect patient-reported

outcomes (PRO) and medical record

(MR) data

• Approach: MediGuard.org members

were invited to participate based upon

treatment or diagnosis in their profile

• Interested members screened based

on self-reported diagnosis and

willingness to release medical records

(electronic and paper signature)

• OHIS contacted physicians and

obtained participant charts

• Results:

• 42 of 50 paper release forms returned

• 38 of 50 charts retrieved (28

electronic, 10 paper)

Conclusions

Background Findings

35 2 1

0 5 10 15 20 25 30 35

Diagnosis Treatment Missing

% Match from PRO to MR

Medical Records

Source: Cascade E, Marr P; Tuttle D, Winslow M: Patient-Reported Outcomes (PRO) and Medical Record Data (MR) in

Observational Study Designs: Results from a Direct-to-Patient Pilot Study in Gout. ISPOR June 2012.

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Case Study Example:

Measuring UK Real-World Outcomes

• Quintiles patient consent & connectivity

provide opportunity for intervention

• PRO+EMR environment serves as

foundation for intervention evaluation

• Objective: Build a UK data-rich

environment to measure outcomes

• Approach: Conducted study to

demonstrate PRO+EMR link with

SAIL data warehouse in Wales, UK

• In 6 weeks, recruited 240

cholesterol patients who

completed PRO assessments and

consented to share identifiers

• Identifiers provided to NHS Wales

to create pseudo-identifier bridge

into SAIL data warehouse

o PRO data combined with

electronic data stored in SAIL at

Swansea Univ. for all Wales

Conclusions

93% 98%

0%

25%

50%

75%

100%

Patients (224 of 240)

Diagnosis (89 of 91)

Background Findings

% Match from PRO to EMR Wales, UK

Source: Cascade E, Nixon M, Ford D, Brooks C, Heaven M: Combining Electronic Health Records and Patient-Reported

Information in the UK: Preliminary Results from the WASPS (Wales SAIL + PRO Study) . ISPOR June 2012.

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Case Study Example:

Evidence-based Program Design

• Data-driven patient framework

serves as the foundation for

program design

• Design includes continuous

program evaluation component

• Objective: Build HTN engagement

program around deep

understanding of patient drivers

• Approach: Surveyed >350 patients

in UK, DE, ES, IT to support design:

• Behavioral segments

o Control: External / Internal

o Emotion: Positive / Negative

o Agency/Action: High / Low

• Validated patient-reported

outcomes instruments used to

evaluate adherence, treatment

satisfaction, and other drivers of

engagement

• Communication preferences:

Content, Message, Frequency

Results

51%

39% 39%

24% 23% 21%

13% 8%

0%

20%

40%

60%

IPH EPL EPH IPL INH ENH ENL INL

% Perfect Adherence by Segment

Background Findings

Source: Cascade E, Cousins F, Connor U, Sandy R: Variance in Medication Adherence by Patient Behavioral Segment: A Multi-

Country Study in Hypertension . ISPOR June 2012.