Patient Recruitment

Brief overview by InnoAction Advisory Services

(www.innoaction.co)

• The clinical trial recruitment market is estimated to be between $1.2-$1.8B

• Since 2000, the number of studies being conducted has gone up from ~5K to nearly

~150K, globally, increasing complexity, as well as site & patient recruitment demand

• At the same time regulatory requirements have increased the size of trials, requiring

on average 3.5-4K patients per trial (and increasing shift towards PMx)

• Patient recruitment remains the biggest factor in enrollment and trial delays – now

taking an average of ~8 years from the start of a trial to approval

• Patient recruitment costs average at ~6% of trial costs, going up to 10%, while

doubling the timelines for Phase II-IV trials to meet desired enrollment levels

• Further, 11% of sites end up enrolling no patients, while an additional 37% of sites

chronically under enroll patients for trials

• Little has changed by way of operating model or technology impact to create step-

wise change in the patient recruitment model over the decades

Confidential – Do not copy or distribute | 1

Executive Summary

• On average, less than 5% of patients in a therapeutic category are aware about

clinical trials, and even fewer raise their hand to participate in a trial

• Of patients that are identified, 2-3% actually make it past screening, into randomized

selection, and trial completion

• However, of those that do participate in trials, most have a positive experience, and

~90% would participate in trials again

• There is a significant opportunity take a patient-centric approach, combined with

technology, data, and analytics to grow the size of the patient recruitment pie

• However, this will require a rethink to traditional processes and operating models

– Leveraging data and analytics for practical protocol design

– Early input from key investigators on protocol design

– Use of technology and data to identify patient concentrations by geography

– Selecting appropriate sites based on patient availability

– Working closely with sites to attract and maintain patients throughout the trials

– Patient-centric design e.g. using local infrastructure & mobile/ remote tools

Confidential – Do not copy or distribute | 2

Executive Summary

There is significant opportunity to redefine patient recruitment

Patient Recruitment Challenges Market Opportunity

Very low awareness of trials by patients Digital awareness & education

High fear & trust issues by patients Medical education

Low patient convenience & access Patient-centric design

Low awareness of trials by physicians Targeted physician education

Complex (exclusionary) trial protocols Protocol feasibility assessment & investigator feedback

Poor use of data & automation Digital, mobile, & big data investments

Inefficient trial/ recruitment processes Redesigning some processes

Privacy rules and concerns Allowing for dynamic patient consent

Industry will need to enhance its connectivity & engagement assets to drive more efficient & patient-centered trials

Portal a. Develop patient consent-based portal

b. Allow patients ability to upload personal data

c. Create linkage between providers-patients

d. Provide effective education & communication

e. Allow for patient & provider referrals

f. Provide visualization & planning tools for

protocol feasibility & patient recruitment

g. Enable researchers to conduct privacy/

consent-based searches

h. Enable near-patient collection & virtualization

of trials (smart phones, devices, data mgmt.)

i. Link Lab, AP, Gx & external data sources over

time for precision medicine services

j. Create deeper engagement (incentives,

gamification, social networks …)

Industry will carefully have to navigate privacy rules & regulations as well as physician and patient concerns to establish trust

Key Privacy Use Cases Regulatory Feasibility Considerations

Link to general clinical trials site on

portal Follow communication SOP

Link to specific clinical trial based on

patient lab values on portal Allowed under HIPAA research provision

Outreach to health volunteers Follow communication SOP

Outreach to patients who fit trial criteria Allowed under HIPAA research provision

Sharing identifiable patient & trial info

with patient’s physician Only data for physician’s patients

Sharing identifiable patient info with

third party Requires patient consent

Outreach to patient’s physician within

network No restrictions

Sharing physician information with

investigators (for trial recruitment) Follow communication SOP

Sharing de-identified patient info with

third party Compliance with de-identification SOP

The monetization model will evolve as the solution matures, leveraging various potential revenue streams

Base License Fee

Patient ID + Recruitment Fee

Value-added Services Fee

• Traditional software/ data

licensing fees

• Annual license based on

users/ seats

• Per patient fee for identified

patients that meet criteria

• Per patient fee for patients

recruited into trial

• Project fees for using “near

patient” infrastructure

• Potential “freemium” model

for patients and providers

• Potential advertising

revenues on portal(s)

• While clinical trial protocol design and recruitment services are highly inefficient and

cost sponsors billions of dollars, change to traditional models has been very slow

• Historically, few data and technology driven players have been successful in shifting

the clinical trials paradigm

– Lack of appropriate and statistically representative data

– Inability to create patient and provider scale on their platforms

– Long cycle times selling to Pharma & CRO’s

– Lack of pharma R&D and healthcare knowledge

• One size does not fit all – the solution(s) have to be carefully designed and curated

by disease area, and should align with Pharma R&D spend

• No one organization can “build” the entire solution – partnerships are a critical

success factor e.g. data, technology, advocacy groups, media partners, etc.

• Traditional top-down models for solutions development have generally failed

– Iterative approaches that use human-centered design & agile development can de-risk

– Patient & provider-centricity should sit at the heart of the approach

Confidential – Do not copy or distribute | 7

Risks & Challenges

Industry overview & challenges

Confidential – Do not copy or distribute | 8

A 2012 Tuft’s estimate put the US patient recruitment market at $1B

37% of sites under-enroll, while 11% don’t enroll at all

Recruitment is the biggest contributor to enrollment & trial delays

Lack of awareness is a significant issue in patient recruitment

Source: Harris Interactive, Centerwatch

16%

84%Unaware or

unsure that trials

was an option

Aware but

unwilling

Top 5 Reasons Physicians Don’t Refer Patients

to Trials

n = 6,000

Reasons patients did not participate in trials

Of these, 75%

would have enrolled

had they known it

was possible

Patient recruitment can account for up to 10% of a trial’s budget, while comprising a significant amount of economic risk

Reasons why patients don’t participate in clinical trial (based on physician survey)

(8*, 0%)(6, 2%) (7, 1%)

(1, 44%)

(4, 18%)

(3, 10%)

(2, 17%)

(5, 8%)

1. Lack of knowledge on where to look for information on

clinical trials

2. Lack of knowledge about what clinical trials actually

are (e.g., objectives and processes)

3. Lack of trust in the purpose of clinical trials

4. Fear of clinical trial processes, potential adverse

effects, or receiving a placebo

5. Lack of patient referrals from physicians

6. Extensive inclusion / exclusion criteria that prohibit

many patients from qualifying

7. Barriers to accessing clinical trials

8. Other*

A total of 169 respondents chose one of the above as

the most important reason that so few patients choose

clinical trials as a treatment option

The following three accounted for 79% of the total

responses Lack of knowledge on where to look for information on

clinical trials (74)

Fear of clinical trial processes, potential adverse effects,

or receiving a placebo (31)

Lack of knowledge of what clinical trials actually are (e.g.,

objectives and processes) (28)* “Other” includes lack of motivation, patient demographics, and insurance issues

Total Number of Respondents 169

Appendix (additional industry/ market insights)

Confidential – Do not copy or distribute | 15

Over the last 13 years, the number of registered studies has grown by 25x, with increasing complexity of patient recruitment

While US & EU still account for ~70% of global trials, other regions continue to make headway, especially East Asia

High level study management process

PI reviews

protocol

synopsis

If PI wishes to

proceed,

confidentiality

agreement

signed

Full protocol with

site

questionnaires

sent to site

Questionnaires

returned &

reviewed, site

visit conducted

Final docs

submitted – IRB

approval,

consent forms

Contract

negotiation and

signing

Patient

enrollment

preparations

Enrollment

initiated

Enrollment

completed

Data analysis &

reporting

Study lock-up

Site challenges in patient recruitment

Develop IIP

and CSA

Site SelectionRecruitment

Outreach/

Advertising

Patient

Follow-Up

Recruitment

Monitoring

• Site selection strategy

and patient recruitment

strategies not coupled

• Site evaluation and

selection process not

driven by a

standardized set of

performance metrics

• Site assessment

evaluation only

focused on recruitment

#’s and does not

include assessment of

other factors such as

geographic location or

local demographics

• No clear accountability

or decision rights for

patient recruitment

strategy & effectiveness

• Lack of standardized

processes for

development of patient

recruitment strategy

• Minimal enforcement

and encouragement of

study teams to leverage

in-house patient

recruitment expertise

• Lack of metrics and

data used to drive

outreach/ advertising

strategy

Key Factors Affecting Recruitment

Key Issues in Patient Recruitment Process

• Lack of metrics defined

to capture effectiveness

of recruitment practices

• Lack of standardized

metrics and process to

track patient follow-up

for all sites

Site challenges in patient recruitment

Unfavorable perception of industry

Competition for limited # patients

Lack of awareness by patients

Lack of awareness & support by

physicians

Competition for limited # sites

Practical barriers facing patients

Complex protocols

ExplanationIndustry Challenges

• 7% of public believes Rx companies care about public good

• 14% of Americans believe drug companies “honest” (similar

ratings for tobacco, oil and used car sales)

Source: Decision Resources, ResearchAmerica!, Harris Interactive; Tufts CSDD

• Even in TA’s with more “involved” patient base (e.g., breast

cancer in US), inadequate number of patients to fully assess

all drugs in pipeline (over 750 breast cancer trials currently

recruiting, over 160 in NY alone)

• Nearly 85% of patients in a survey stated they were

unaware that trials were an option

• 31% of physicians surveyed did not refer patients to trials

due to lack of information

• Also report unwillingness to “lose control” of patient care,

concerns of admin burdens, concerns of patient reactions to

referrals

• From 2001-2005, only 8,600 new investigators compared to

over 35,000 new studies

• Language barriers, transportation issues, family obligations,

financial barriers all cited as reasons why qualified patients do

not enroll

• From 2002-2006, mean number of inclusion criteria rose

from 10 to 26; for exclusion criteria from 21 to 23

Key insights & feedback from sponsors

Top mentions of clinical trial recruitment services companies

Quintiles patient networks rank high with sponsors

Social and mobile trends are highly favorable in the healthcare industry and can drive innovation in clinical research

Myths about social and mobile solutions in healthcare …

Top reasons consumers chose to participate in clinical research

Most people who participate in studies would do so again …

Research volunteers by phase of research

Study participant experiences

Illustrative trial process & timelines

1. Limited marketing expertise

and planning insight put sites

at risk of failure

2. Challenges in patient recruiting

require greater investment in more

personalized emerging channels

Illustrative benefits of improving trial process & timelines