Designing clinical trials on lifestyle interventions using patient reported outcomes

1
Designing clinical trials on lifestyle
interventions using patient
reported outcomes
Jim Roldan
CEO
Linkcare Health Services

2
Traditional clinical trial goals
The traditional purposes of controlled clinical trials have
been to, on scientific evidence basis:
• Prove effectiveness of new drugs
• Determine optimal dosage
• State undesired side effects
• Seek for drug authority approvals.
© 2014 DIA, Inc. All rights reserved.

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New challenges clinical trials need to address (I)
Patients, pharmaceutical companies and health
professionals are demanding new approaches to:
• Measure and improve lifestyle habits
• Understand the effect of comorbidity conditions
• Discover long term drug interactions
• Age-related dosage adjustment
• Systems medicine based variability.

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New challenges clinical trials need to address (II)
Personalized medicine requirements also increase the difficulties
to find the right case candidates. Phenotype variation, omics
diversity and lifestyle implications are becoming an increased
source of interest and concern.
Long term monitoring of drug effects and environmental
interaction with disease are also important topics requiring new
trial approaches.
Some recent episodes, such as the recent ones related Ebola
treatment has shown the ethic limitations of controlled trials to
face extremely adverse prognosis situations.

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Lifestyle interventions
A number of recently published studies have shown the important
role of lifestyle behaviors such as dietary habits, drug abuse,
daily activity, sleep disorders and social behavior to play a
much important role on health and disease personal record.
Theories imported from psychology such as the "perceived
behavioral control theory” and “theory of planned behavior”
are changing the way we understand lifestyle interventions.
Such behaviors not only influence treatment adherence but has
a paramount impact on treatment effectiveness and disease
progression.

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Lifesytle interventions research papers
Evolution of
Lifestyle
intervention
peered reviewed
scientific
publications
(Source: Scopus
Elsevier)

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A lifestyle intervention generic model
HEALTH PROFILE
ASSESTMENT
ELEGIBILITY
STRATIFICATION
RANDOMIZATION
DAILY GOAL
ACTIVITY
UPLOAD
DAILY FEEDBACK
WEEKLY
FEEDBACK
GOAL
TRADEOFF
ADMISSIONENROLLMENT DAILY WEEKLY
BASELINE
ASSESTMENT
DEVICE
CONFIGURATION

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A three level motivation strategy
Compliance: The patient performs the assigned activities
Adherence: The patient achieves the agreed level of activities
Outcome: The patient’s health improves as expected
LEVEL INDICATOR
COMPLIANCE “Take a picture of each meal”
ADHERENCE “Reduce meat down to two takes a week”
OUTCOME “Cholesterol reduction” or “BMI improvement”

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Compliance and adherence short term effectiveness…
Troosters, Thierry. “Proactive-COPD CCT” KU Leuven et al.

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…but motivation drops over time…

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…on long term health improvement consolidates behaviour

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The role of patient reported outcomes
As chronic disease prevalence has increased due to life
expectancy increase, new end points have been identified to be
added to the traditional ones based on laboratory and
functional findings, exacerbation records and survival rates.
Today, the patients perception of his own quality of life has
become a central focus, as life quality and self perceived
happiness become sometimes more relevant for patients than
purely objective clinical data.
The availability of smart phones and the lower cost of
telecommunication infrastructures have become enabling
technologies making patient self report simple and inexpensive.

13
Patient reported outcomes research papers
Evolution of
patient reported
outcomes in
peered reviewed
scientific
publications
(Source: Scopus
Elsevier)

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The raising interest on observational trials
All the above is having a tremendous impact in many aspects of
clinical trial design and goals.
The possibility of following up a controlled set on patients
world wide during a long period of time as part of their daily
care is making possible to design observational trials that allow
long term longitudinal data to be collected an analyzed.
The establishment of a strong networks of researchers and
practitioners around health-profile-oriented panels will allow
unprecedented treatment benchmarking.
A new wave of treatment efficacy improvements is expected to
outcome in the next years.

15
Observational trial research papers
Evolution of
patient reported
outcomes in
peered reviewed
scientific
publications
(Source: Scopus
Elsevier)

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Dynamic patient clustering: Learning from practice
Lowie E. G. W. Vanfleteren, Martijn A. Spruit,
Miriam Groenen, Swetlana Gaffron, Vanessa P.
M. van Empel, Piet L. B. Bruijnzeel, Erica P. A.
Rutten, Jos Opt Roodt, Emiel F. M. Wouters,
and Frits M. E. Franssen "Clusters of
Comorbidities Based on Validated Objective
Measurements and Systemic Inflammation in
Patients with Chronic Obstructive Pulmonary
Disease", American Journal of Respiratory and
Critical Care Medicine, Vol. 187, No. 7 (2013),
pp. 728-735.

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The era of clinical shared knowledge
A wider range of therapeutic options available, a much better
understanding of personal and disease profile implications and an
accelerated growth of the knowledge generated by big data
sources and exponential research put translational medicine under
reform.
New health care professional profiles specifically trained for case
management require a different set of skills and will use new tools for
their daily care to address the increased demand of care, derived from
population ageing.
Despite they will lack of the expertise that only many years of practice
can provide, new forms of knowledge exchange and clinical
decision support services facilitate the access to knowledge.© 2014 DIA, Inc. All rights reserved.

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What is big data adding to the picture?
KNOWLEDGE
INFORMATION
DATA

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Available knowledge is growing at an unprecedented pace

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Taking clinical decisions may be though!

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Knowledge exchange versus knowledge sharing

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From a classical to collaborative learning
RESEARCH
PUBLICATION
EDUCATION
CLINICAL PRACTICE
SHARED
KNOWLEDGE
RESEARCH
EDUCATION
CLINICAL
PRACTICE

23
Collaborative learning research papers
Evolution of
collaborative
education and
collaborative
learning in
peered reviewed
scientific
publications
(Source: Scopus
Elsevier)

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Challenge-based collaborative education
® Medscape Continued Medical Education

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Disclaimer
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affiliated.
These PowerPoint slides are the intellectual property of the individual
presenter and are protected under the copyright laws of the United States of
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trademarks of Drug Information Association Inc. All other trademarks are the
property of their respective owners.

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Disclosure Statement
Type of Financial Interest within last 12 months Name of Commercial Interest
 Grants/Research Funding Synergy-COPD – Modelling and simulation
environment for systems medicine (Chronic
obstructive pulmonary disease -COPD- as a use
case) - FP7-ICT-270086
PROactive project – European project funded by
the Innovative Medicines Initiative (IMI). IMI is a
Joint Initiative (JI) funded by both the EC (via the
FP7 research programme) and EFPIA.
 Stock Shareholder Linkcare Health Services, SL
☐ Consulting Fees
 Employee Linkcare Health Services, SL
☐ Other (Receipt of Intellectual Property
Rights/Patent Holder, Speaker’s Bureau)

Designing clinical trials on lifestyle interventions using patient reported outcomes

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