The Implementation and Evaluation of a Promortora Led Cervical Cancer 1.22.09
Tylosky_S_Trk17_Session_121_DIA_22Jun2016
1. 1
Solving Enrollment Challenges for Pediatric Rare
Disease Global Clinical Trial in Latin America
Sara Tylosky
President & CEO
Farmacon
@Sarafarma
2. 2
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4. 4
1. Learning Objectives
2. Enrollment Challenges in Pediatric Rare Diseases Clinical Trials
3. Latin America Landscape – Trial Management & Potential Patients
4. Pediatric Rare Disease Case Study
5. Polling Question/Questions/Comments
Agenda
5/17/16 4
5. 5
Learning Objectives
1. To review the enrollment challenges of pediatric rare disease trials
2. To understands the challenges and opportunities within the Latin
American environment and culture
3. To apply best practices to meet enrollment targets in a rare
disease pediatric study in Latin America
5/17/16 5
6. 6
Pool of
patients is
limited
Complexity
of disease &
trial can be
limiting
Logistics,
timelines &
care
coordination
can be
complicated
Culture
needs to be
understood
& managed
well
Enrollment Challenges in
Pediatric Rare Diseases Clinical Trials
7. 7
STATISTICS
26 countries > 626.7 million-2X of US & 8.63% of world
Growing middle class
US Latino population represents 17%
CLINICAL RESEARCH IN LATIN AMERICA
Big 3 - Brazil, Argentina, Mexico = 55 million,
70% of countries’ population
Many rare disease patients in LatAm haven’t
been in a clinical trial before
# of pediatric rare disease clinical trials in
Latin America = 25, only 7 countries
Latin America Environment
8. 8
Regulatory can be longer – 3-9 months (EC, MOH) but enrollment
high
More standard of care available now
PI with a strong network of Sub-Is are more successful
Perception of inability of having quality data
Monitoring is closer to US time zone
Understanding the cultural context is vital, e.g.,
– Working with PI and/or a Sub-I vs. study coordinator normal
– F2F interactions or phone preferable, What’s App popular with relationship
– Confirmation of proper diagnosis, e.g., medical histories from large institutions
difficult to obtain
Strong on the ground presence for logistical details and ad-hoc
problems help execution
Latin America Landscape – Trial Management
9. 9
Family is key – Spanish mostly universal minus Brazil & indigenous
– All decisions are always family based
– Population less educated regarding clinical trials
– Growing middle class helps level of understanding
– People tend not to travel within the country as much
– Frequent communication to patient family = smoother decision making
– Only reimbursement for travel & food per Latin American laws
Simple understanding of ICF is critical in order for family to sign
Physician-patient relationship very strong
Advocacy groups help pinpoint potential patients
Limited ability to directly advertise, social media is growing (FB)
Concierge approach is needed
Latin America Landscape – the patients
11. 11
Who
– PIs familiar disease-estimated incidence ≈ 1 per 155,000 male live
births
– Sites identified in Colombia, Peru, Uruguay, Argentina & Mexico to
contribute potential patients & estimate recruitment rates
– Patients had to be on currently approved treatment, so only went
into countries where current treatment was in place
– Identification of KOLs using current treatments considered
What was needed
– Site experience with procedures & cognitive scales; competition for
patients and resources; and acceptability of referrals
Number of patients needed
– 42, but was increased to 48 after statistical analysis was completed
Feasibility Conduct and Methodology
12. 12
Investigators
42 investigators in Latin America contacted,18 responses received (43% responded)
8 investigators interested in study participation (44% interest rate)
6 investigators (75%) managing at least 1 rare disease patient aged ≥3 to ≤8 years
Only 3 investigators (38%) managing at least one rare disease patient of any age
Argentina, Colombia and Mexico chosen 1 site/country
Regulatory
Planned time to bring up a site (from submission to SIV)
– Argentina 4-6.5 months, took 12 months
– Colombia 3 months, took 13 months
– Mexico 6-7 months took 11 months
Prolonged regulatory approval timelines impacted ability to open sites &
actively screen
Feasibility Conduct and Methodology
14. 14
Enrollment Challenges
Cultural and
Communication Issues
• Patients’ families didn’t want to
sign ICF
• PI not reaching out to referring
drs. w/ patients
• Patients’ families felt travel was
too far
Logistical Issues
• Sites & travel agents handled patients’
travel poorly (Mexico)
• Reimbursements for patients in Mexico
not being processed easily
• CRO not scoped to problem solve in a
timely manner
15. 15
An on-the-
ground team
was
identified to
problem
solve
Provide
feedback to
sponsor as 3rd
party
Meet F2F with
referring drs.
to connect to
protocol & PI
Establish
relationships
with local rare
disease
advocates Act as liaison
between site,
travel agency &
patient family
Provide tech
solutions to
increase
communication
Enrollment Solutions
16. 16
Session #121
Poll Question #1:
What has been your main enrollment challenge in your current
clinical trial?
Poll Question
17. 17
Mexico – Increased enrollment by 50%
Argentina – No patients qualified due to lower incidence
of rare disease
Colombia – Selected PI was overwhelmed with other
priorities, therefore didn’t enroll
Additional sites in Mexico & Colombia added (still in process)
Sub-I model implemented for Mexico & Colombia
Coordinated Mexico patient travel & patient reimbursements and
retention was 100%
Technology applied:
– What’s App
– Local 800# and 24/7 logistical help line
Results
