This document discusses strategies for optimizing patient recruitment in clinical trials, especially for rare diseases. It notes that patient recruitment is critical for drug and device approval but can be challenging for rare diseases where patients are spread out globally. New technologies like social media and mobile devices have helped increase awareness of studies. Trial designs are evolving to be more accessible and reduce patient burden, such as through multi-site trials. Collaboration between clinical research organizations, sites, and patient groups can also enhance recruitment. The future of recruitment looks encouraging as awareness and access continue to improve through technological advances.

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2. Optimizing Patient Recruitment
to Accelerate Research Today –
and Trends for Tomorrow
John Lynn Jefferies, MD, MPH,
FACC, FAHA, FHFSA
Director, Advanced Heart Failure
and Cardiomyopathy Programs,
Cincinnati Children’s Hospital
Medical Center

3. o Studies for drug and
device approval by
the Food and Drug
Administration (FDA)
rely on patient data
for safety and
efficacy
Optimizing Patient Recruitment
Therapeutically-Focused | Scientifically-Driven3
o Patient recruitment is critical for clinical research
o Many adult trials involve thousands of participants

4. o Rare disease studies have an advantage that
smaller numbers of patients are required by the
FDA for approval
Patient Recruitment: Rare Disease
Therapeutically-Focused | Scientifically-Driven4
o However, rare disease
patients are typically
spread out all over the
globe making access
to them for trials
challenging

5. o Many rare diseases
have epicenters of
care
o Institutions that have
a reputation for
being national or
international leaders
in the care of the
specific condition
Rare Disease Awareness
Therapeutically-Focused | Scientifically-Driven5
o However, many patients that may want to
participate in research are not aware of clinical
studies

6. o In the past, patients not seen at centers
performing the research would not have access to
participation
Increasing Awareness with Technology
Therapeutically-Focused | Scientifically-Driven6
o Developments in
technology and
changes in trial
designs now allow for
larger numbers of
patients and families
to be made aware of
clinical investigations

7. o Use of the internet and other mechanisms of
communication have greatly enhanced awareness
of studies
Increasing Awareness with Technology
Therapeutically-Focused | Scientifically-Driven7
 Facebook and
other social media
 Hand held
technologies
 Patient advocacy
groups

8. o Patient self-referral has also added to trial
participation
Participants Take Control
Therapeutically-Focused | Scientifically-Driven8
o Many families will find
out about clinical
trials through various
mechanisms and go
to the centers doing
the research to
participate

9. o Prior research in rare diseases has typically been
done at single institutions
o This is challenging as it may be hard for families
to get to that center
Increasing Access
Therapeutically-Focused | Scientifically-Driven9
o Recent years have
seen the development
of multicenter trials
and collaborative
groups which facilitate
more options for
patients and families

10. o The future of trials and recruitment is very open
and encouraging
The Future of Recruitment
Therapeutically-Focused | Scientifically-Driven10
o As technologies continue to
improve and become more
available, the awareness of
clinical studies will be more
widespread allowing families
to prioritize which studies they
would like to participate in
moving forward

11. Bespoke Trial Design
and Patient Centricity
Michelle Petersen, MS
Associate Director, Clinical
Trial Management

12. o Rare disease requires strategic trial design as the
number of subjects to enroll are limited
 Studies require patient input to ensure the design is
appropriate and includes what is meaningful to the patient
 Collect samples at different time points in different patients
 Crossover trial design is one way to increase data
collection in a limited patient population but only works in
ideal circumstances
Bespoke Trial Design
Therapeutically-Focused | Scientifically-Driven12

13. Therapeutically-Focused | Scientifically-Driven
o Hub and spoke trial design
for a hub site for complex
visits and spoke sites for
minor procedures or
dosing
 Commit to opening
spoke sites in extension
studies
o Home healthcare
compliant visits and home
delivery of IP
o Increase time between
visits as study duration
increases
o Visit window flexibility
o Request site to extend
hours of service and offer
weekend appointments
o Travel/reimbursement
support
 Concierge level service
to address challenges of
study participation and
remove financial burden
 Support family (i.e.
considering childcare or
schooling of young
patients)
Retention Key: Burden Reduction
13
Complex endpoints can reduce the number of centers able to
participate which often increases subject burden
To Reduce This Impact Consider:

14. Therapeutically-Focused | Scientifically-Driven
o Get patient input of trial design, informed consent
process and what would improve the trial
o Avenues for patient insight:
 Advocacy groups
 Patient panels
 International and regional patient meetings
Patient Input
14
o Keep updating the
community on progress
 Webinar
 Press releases
 Sending results overview
in layman’s terms

15. CRO and Site
Collaboration
Michelle Petersen, MS
John Lynn Jefferies, MD, MPH,
FACC, FAHA, FHFSA

16. o The increased use of CROs in rare disease
clinical research has enhanced participation
o CROs have numerous touchpoints across the
globe at leading institutions
 Repeated collaboration can lead to enhanced
startup
o CROs also work with leaders in the field of the
particular rare disease to gain additional insight
into trial design and patient/family perspectives
CRO and Site Collaboration
Therapeutically-Focused | Scientifically-Driven16

17. A Family’s Story
Beth Woelfel Harvey
Executive Director, JB’s Keys to DMD
Mother of child with Duchenne
Muscular Dystrophy

18. JB Harvey
Diagnosed in 2009 at 16 months with DMD
JB one month before diagnosis
JB age 2JB walking on the beach age 3

19. Therapeutically-Focused | Scientifically-Driven
o No standards of care
o 3 clinical trials
o 3 companies
developing drugs
o Recently updated
standards of care
o Over 30 current
trials
o Over 40 companies
developing drugs
2009 vs. 2018
19
2009 2018

20. Therapeutically-Focused | Scientifically-Driven
o Trial #1
 1 visit Age 2
 DXA scan and function testing
o Trial #2
 10 visits over 2 years, Age 6-9
 Imaging and functional testing
o Trial #3
 8 visits over 2 years from Age
7-9
 Thigh MRI, labs, functional
testing, Echo, PFT, eye exam
o Trial #4
 Over 20 visits in first year Age
9-10
 Thigh MRI, Cardiac MRI, Liver
MRI, X-rays, labs, Echo,
Functional Testing, DXA
JB’s Clinical Trial Experience
20
Trial #3, 2015
Trial #4, 2016

21. Therapeutically-Focused | Scientifically-Driven
For children to participate in clinical trials they
must have stable mental health and be happy!
Mental Health
21
JB after skiing 20 runs
JB after his musical
performance
JB sailing

22. Rare Disease Day
Therapeutically-Focused | Scientifically-Driven22
#ShowYourRare

23. Questions?
Contact us at
info@medpace.com