Tom Macek, Takeda Global Research and Development - Speaker at the marcus evans Evolution Summit 2012, held in Wheeling, IL, April 30-May 2, 2012, delivered his presentation entitled Site Identification and Patient Recruitment and Retention – A Perfect Union

1. Site Identification and Patient Recruitment and Retention –
a perfect union
f t i
Tom Macek, PharmD, PhD
Senior Fellow, Clinical Sciences
Takeda Global Research and Development

2. Factors that determine study success
• Assuming a drug or product is safe and effective, the
following are critical
▫ Appropriate p
pp p patient recruitment
▫ Appropriate site selection
▫ Appropriate subject participation (retention)

3. Determinants of quality
q y
sites
Quality
patients participation

4. Factors that determine study success
• Interdependence of these factors
▫ Negative impact of speed on quality
• Difficult challenge by sponsors to determine
performance of each
▫ Especially during conduct of ongoing studies that
may be double blind
▫ Often – quality only known or assessed at end of
study
▫ U ili i metrics or methodologies to asses quality
Utilizing i h d l i li
in ongoing study

5. Sponsor pressures impact quality
• Internal pressures of sponsor
▫ Expectations of management
▫ Realistic timelines
▫ Unknown negative
• External pressures of sponsor
p p
▫ Expectations of investors
▫ Expectations of patients and advocacy groups
▫ Competing competitor programs or products

6. Managing internal and external pressures
• Common methods to manage these pressures
within a clinical study
▫ Add more sites
▫ Loosen Inclusion/Exclusion criteria
▫ Conduct more studies
With limited investment prior to Phase 3
▫ Enhanced recruitment and retention measures
▫ Others?

7. General issues for consideration
• What constitutes quality within a study?
• Is a quality study only defined by positive
results?
• How to define quality of these factors?
• What subjective or objective measures do we
utilize for each factor

8. General issues for consideration
• What ongoing (during study) measures of
performance can be utilized
• How to utilize novel tools of patient recruitment
and retention
• Are measures of quality dependent upon
therapeutic area?
p
▫ Example - is the use of central monitoring of
patient ratings by PI (in psychiatry/neurology)
enhancing ability t d t t signal?
h i bilit to detect i l?

9. Selecting appropriate sites for a study
• What are subjective measures that are utilized in
identifying appropriate sites?
▫ Are positive sponsor-CRO-PI relationships
sponsor CRO PI
important to ensuring quality?

10. Selecting appropriate sites for a study
• What objective measures are utilized for site
selection?
▫ Past performance – how to quantify quality
Is rate of enrollment appropriate measure of q
pp p quality?
y
What measures of quality could be utilized to asses site
performance?
Metrics of site training
Placebo versus active control
▫ Operational measures of performance
Are number of queries or other operational measures a
true measure of site performance?
▫ Other methods?

11. Utilizing appropriate recruitment measures
• What common methods do we utilize for patient
recruitment?
▫ How do these methods ensuring that the patients
recruited are appropriate for the study?
pp p y
▫ What are the pros/cons of advertising or using
referral networks for study recruitment?
▫ How can social networking or other technologies
be utilized to enhance patient recruitment?

12. Utilizing appropriate retention measures
• Is patient retention a surrogate of quality?
▫ Are there any instances in which patient retention may
be a hindrance to quality?
Is it possible to encourage patient retention to a point in
which it has a negative impact on quality?
▫ What are the motivating factors to patients to remain
in study?
Can financial or other incentives be detrimental to study
quality?
▫ What methods or techniques can be utilized to help
ensure that the appropriate patients are participating
in your study?

13. Determinants of quality
q y
sites
Quality
patients participation