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HTA and Regulatory Perspectives and Interactions:
bridging the gap
For Colleagues at the
2014 CADTH Symposium
April 7, 2014
Gatineau, Canada
Lawrence Liberti, MS, RPh, RAC
Executive Director
Photo credit: http://fineartamerica.com/featured/grandfather-mountain-heavy-
fog-bridge-to-nowhere-dave-allen.html
2025 - Landscape – A Viewpoint
• Complimentary evidence requirements from regulators and HTAs
• Tiered but convergent landscape between high, middle and low income countries
Convergent
•Trust: Acceptance of a Collaborative/work sharing environment –between agencies/regions
•Managing uncertainty: Innovative technologies for disease identification, treatment, management, adherence and vigilance monitoring
Collaborative
• Priorities: Patient and HCPs- facilitating access to “safe”, high quality and effective medicines globally
• Adoption of Risk-Based Facilitated Regulatory Pathways – development, approval, inspections, life cycle management, vigilance
Enabling
CIRS- Project 2020 3
Differences in
priorities seen
between functions
In this example only two
scenarios overlap between
Regulatory and
HEOR/Access
SC04 Evidentiary standards used by HTA agencies will be integrated into licensing body requirements with separate, but parallel reviews
SC19 Interaction between licensing bodies and HTA agencies will increase
HTA and Regulatory Alignment
Bruening L: Regulatory and HTA Alignment. Presented at CIRS Workshop Is there a commonality across the structured decision frameworks used by HTA and
regulatory agencies? which took place 1-2 October in Surrey UK.
How could the expectations of
diverse stakeholders, including
sponsors, regulators and HTA,
be aligned in an international
environment?
Through
• Joint Scientific Advice
• Common or aligned evidentiary requirements
• Consistency in review package evidence
Timing of HTA-related scientific advice at a global level N
um
be
r o
f c
on
su
lta
tio
ns
Timing of scientific advice by the type of advice
(Number of products, Number of companies)
Timing of development when advice was sought
7
1
2
1
2
2
1
0
2
4
6
8
10
12
Ph II (7,5) Ph III (4,3)
Formal advice from multiple HTA agenciespresented in the same meeting
Formal advice from a single HTA or payeragency
KOL Panel with primary purpose of payerinformation
Company sponsored payer advisory board
0 5 10 15 20
Extra patient visits requiredNeed for companion diagnostic
Societal benefitBudget impact / affordability issues
Cost effectiveness
Unmet needPlace in treatment pathway
Use of PRO measurePRO methodology
Patient enrichment (biomarkers)Sub-group analysis methodology
Patient selection
Use of adaptive trial designUse or applicability of patient crossover
External validity of trialTrial follow-up and longer-term outcomes
Analysis methodologyTrial durationStudy design
Benefit risk evaluationSafety evidence
Biomarkers used as a measure of efficacySpecification of non-inferiority margin
Health-related quality of lifeSpecification of superiority margin
Relative / Comparative efficacy evidenceUse of surrogate outcomes
Comparator choiceInclusion of a comparator arm in the trial
Secondary endpoint(s)HTA acceptable primary endpoint
The number of licensed products that incorporated HTA requirements into Phase 3 trials, by requirement type
Number of products
Efficacy
Safety
Trial design
PROs
Technical requirement
Patient selection
Cost
Value to healthcare system
Non-technical requirement
Non-price barriers to market
(CIRS HTA Industry Survey 2013)
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
Australia Canada England France Germany Italy Spain USA -Wellpoint
Phase III pivotal study,Phase IIIb pivotalstudy,Real-world evidence
Phase II study,Phase III pivotalstudy,Phase IV study,Real-worldevidencePhase II study,Phase III pivotalstudy,Real-world evidence
Phase III pivotal study,Phase IIIb pivotalstudy
Phase IIIb pivotal study
Phase III pivotal study
Phase II study,Phase IV study
Phase II study,Phase III pivotalstudy,Phase IIIb pivotal study
Phase II study,Phase III pivotal study
Phase II study
Pe
rce
nta
ge
of
Pro
du
cts
HTA review package by jurisdiction (an Overlap with Regulatory Dossiers?)
(CIRS HTA Industry Survey 2013)
Evidence used for recommendation by HTA agency
[Q17]
Total responses
5 agencies provided data overall Total reviews = 18 (CIRS HTA Agency Survey 2013)
“Input from Clinical Professionals” combines data from the
fields: “Written submissions from clinical professionals” and
“Personal statements from clinical experts presented at committee
meeting”
“Input from Patients” combines data from the fields:“Patient group
submitted information”, “Written submissions from patient experts”
and “Personal statements from patient experts presented at
committee meeting”
The association between European Medicines Agency approval and Health
Technology Assessment recommendations: HTA Assessment Outcomes (n=
86)
10
•The longer the approval time at EMA, the less beneficial
the HTA recommendation was*
•There were negative correlations between approval time
in
• HTA recommendations in England
• HTA recommendations in Netherlands
• total indicator of HTA recommendations
* All of the correlations had marginally significant tendencies
(Based on work conducted by Iga Lipska, Utrecht University 2014)
Regulatory and HTA interactions: considerations
• The independent regulatory and HTA systems are grounded
in science and focused on meeting stakeholder needs.
• Scientific advice: provides insights into what is expected,
practical, and achievable especially when provided jointly
• Opportunities to align regulatory and HTA requirements
►maximise process efficiency, consistency of outcomes.
• Sponsors: should address both regulatory and HTA data
requirements from the earliest stages of development
• Heterogeneous HTA processes are moving towards
alignment: integrating diverse needs (ie comparators, study
designs) will be challenging
• Convergent-Collaborative-Enabling: How can industry,
regulators and HTAs align to facilitate the evolution to these
goals?
HTA and Regulatory Perspectives and Interactions:
bridging the gap
For Colleagues at the
2014 CADTH Symposium
April 7, 2014
Gatineau, Canada
Lawrence Liberti, MS, RPh, RAC
Executive Director
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