KCR Presentation on Cost Effective Execution of Bio-similar and Bioequivalent Clinical Trials Applying New Technologies and Processes

1. Cost Effective Execution of
Bio-similar and Bioequivalent
Clinical Trials Applying New
Technologies and Processes
May 28th 2014
Mike Jagielski / President & CEO KCR S.A.

2. agenda
CRO Industrie Trends
Clinical Trial Cost Driver
What are the latest trends towards decreasing Clinical Trial
Costs?
– Risk Based Monitoring – True driver for cost reduction?
– Technology advances
Patient Retention – Particular subject in Bio-equivalent
projects
Future – Direct Patient Engagement Solutions

3. Disclaimer
BioForum 2014PAGE / 3
Every aspect about cost reduction is discussed with the
mindset of maintaining the high execution quality, GCP
and FDA compliance.
Quality matters!

4. BioForum 2014PAGE / 4
CRO Industrie TRENDS
CRO Value Creation starts sooner and broadens in scope
– In the actual project development – CRO input expected
– CRO are involved sooner in actual drug development process –
especially for Biotech companies
Core CRO services become a COMMODITY
– Monitoring Services
– Project Management
– Conclusion: Price matters
Technology plays a larger role to achieve efficiencies
– Integrated DM solutions
– Risk Based Monitoring
– Recruitment support technologies

5. BioForum 2014PAGE / 5
CRO Industrie TRENDS
CRO Value Creation starts sooner and broadens in scope
– In the actual project development – CRO input expected
– CRO are involved sooner in actual drug development process –
especially for Biotech companies
Core CRO services become a COMMODITY
– Monitoring Services
– Project Management
– Conclusion: Price matters
Technology plays a larger role to achieve efficiencies
– Integrated DM solutions
– Risk Based Monitoring
– Recruitment support technologies
Decreases Cost
Increases Cost

6. BioForum 2014PAGE / 6
Clinical Trial Cost Driver
2 Main Cost Driver for Clinical Trials
– 1. CRO/Service fees
– 2. Investigator fees
Monitoring, DM,
Drug Supply,
Central Lab
Estimated
50-60%
CRO/Service
Fees Investigator
Payments
Out Patient
Clinical
Estimated
40-50%
Investigator
Fees

7. BioForum 2014PAGE / 7
Clinical Trial Cost Driver
CRO cost Split (500 patients, 6 visits)
– 20% Project Management
– 40% Site Monitoring and site management
– 40% Other – DM, Statistics, Regulatory, QA……
Site Monitoring – logical target for efficiency gains
Monitoring,
DM, Drug
Supply, Central
Lab
Estimated 50-
60%
CRO/Service
Fees

8. CRO Services – Risk based Monitoring
BioForum 2014PAGE / 8
Risk based Monitoring - true option to reduce Monitoring
costs?
– CRO Industry is promising returns
– FDA and EMA issue guidelines
Bio-similar and Bioequivalent Clinical Trials seem to the
ideal candidates for this solution
– Studies are considerable in scale as far as number of patients
– Safety Profiles are known

9. Guidance - Risk based Monitoring
BioForum 2014
1. Guidance for Industry: Oversight of Clinical Investigations—A
Risk-Based Approach to Monitoring, August, 2013
2. Reflection paper on risk based quality management in clinical
trials, November, 2013
Page / 9

10. RBM – What Does the Future Hold?
BioForum 2014Page / 10

11. RBM – Only One Model out of Many
Alternative SDV Solutions
BioForum 2014
Adaptative monitoring
Targeted monitoring
Remote/Centralized monitoring
Risk-based monitoring
Page / 11

12. Risk Based Monitoring – RBM (1)
Tailored Risk-Based Monitoring Plan
Page / 12
Risk-based monitoring approach focuses on the high risk data
points
data points which are prone to mistakes
or difference in interpretation or transcription and which have a high
impact on the outcome of the study)
BioForum 2014

13. TRIGERS
Protocol–based
(e.g. in case of death,
critical outliers etc.)
Volume–based
(achived collection of
estimated amount of
data)
Time treshold
(certian amount of
time elapsed)
ON-SITE MONITORING VISIT
Page / 13
Risk Based Monitoring – RBM (2)
BioForum 2014

14. What does the future hold for Clinical Monitoring?, Applied Clinical Trails, September 2013: 3-7.
Page / 14
Risk Based Monitoring – RBM (3)
BioForum 2014

15. Page / 15
PROSPROS PROSCONS
 Analyse data in real-time
(use of statistical methods
to trace discrepancies,
outliers, alert signals etc.).
 Focuses on critical data
points.
 Allows to identify
issues/critical areas faster.
 Better utilization of time
spent on-site and decrease
of monitoring visit hours.
 Triggers action in results
of situation at the site.
 Risk of improperly
allocating monitoring
resources.
 Risk that algorithms
predict high-level risk
across all sites
(negligible impact on
saving costs).
 Risk of not revealing
critical findings.
PROSTechnology needs
 Possiblity of signifficant reduction of number
of MV and associated costs.
 Focuses on key variables.
 Robust predictive modelling tool using sophisticated statistical algorithms that
take into account variables/risks such as historical data and the statistical
distribution of patients.
 An electronic system – capable of extracting data from multiple sources (EDC,
IVRS, CTMS etc.) – being able to translate the event into trigger.
 Scheduling tool.
Risk Based Monitoring – RBM (4)
BioForum 2014

16. Study Characteristics vs Monitoring Approach
Page / 16
Clear and focused objectives (valid
endpoints).
Multidisciplinary team (Biostatisticians,
Data Managers, CPLs, Medical Monitors)
involved in all phases of the study.
Tailored Monitoring Plan.
Well designed eCRF that collects „adequate
case histories”.
Clear training strategy- for both internal
and site staff.
Implementation Conditions
BioForum 2014

17. Risk-based monitoring. Reduce clinical trial costs while protecting safety and quality. Pwc, March 2013
Page / 17
Costs of implementation (2)
BioForum 2014

18. RBM - Costs of implementation (1)
Risk-based Source Data Verification Approaches: Pros and Cons, Drug Informatin Journal, Vol. 44, pp. 745-756, 2010.
Page / 18
Reductions of costs are unlikely in small (phase 1/2a) studies and studies with high safety
risk profiles (e.g. Oncology). Significant savings can be gained in large phase 2b/3 studies.
BioForum 2014

19. BioForum 2014PAGE / 19
Technology Advances – Does it reduce costs?
Standard Clinical Trial technologies are moving towards a
true single platform service
– Reduces costs and increases quality
• e -
Diary
• Drug
Supply
• IWRS• EDC
Single
technology
/ Module 1
Single
technology
/ Module 2
Single
technology
/ Module 4
Single
technology
/ Module 3

20. Page / 20
Patient Retention – Particular subject in
Bio-equivalent projects
Challenges
– 1. Product on the Market
– 2. Often study design requires placebo arm in Bioequivalent
studies
Simple formula – less drop outs – less patients to be
randomized = lower cost
Solutions – there is no easy solution
– 1. Site training and education to pass on to patient about
importance to complete the trial – absolutely critical
– 2. Study design has to have the patient in mind as far as visit
frequency and types of tests to be performed

21. Future – Direct Patient Engagement
Solutions
BioForum 2014PAGE / 21
Patient Recruitment Portals
Solutions for Investigator and Patients to communicate
– Collections of different type of data via patient sensors
Sensors for Patients

22. Conclusions
BioForum 2014PAGE / 22
1. Yes – there are ways to optimize costs especially for
Bio-similar and Bio-equivalent clinical Trials
2. Yes – Integration between process and technology plays
a major factor in achieving efficiencies
3. Yes – Effective implementation of new trends such as
risk based monitoring requires careful preparation - Still
4. Solutions to safe money in study execution phase
require increased investments in Start-up phase
Cost reduction via Technology and New
Processes is possible - TODAY - BUT

23. About KCR
KCR is a European Contract Research Organization (CRO) with a dynamic team of nearly
300 professionals operating across 18 countries in Europe as well as the U.S.
With 17 years of experience, more than 350 trials executed, over 30,000 patients recruited
and almost 3,000 sites contracted, KCR is a strategic solutions provider and a reliable
alternative to global CROs, delivering the all-important flexibility.
We provide services on long standing global or local contracts to 12 out of the Top 20
Global Pharma companies, and have been granted by 3 of them with the Preferred
Provider certification.
KCR offers clinical development support via 3 types of professional services:
- Full Service Model for Clinical Development Services (Phase I-IV)
- Functional Service Provider (FSP)
- Post-Marketing Clinical Services
For more information about the KCR offer, please visit www.kcrcro.com or contact us at
info@kcrocro.com.
CORPORATE
HEADQUARTERS KCR S.A.
6 Postepu str.
02-676 Warsaw, Poland
Phone: +48 22 313 13 13
Fax: +48 22 313 13 14
Email: info@kcrcro.com
www.kcrcro.com