Pharmaceutical Medical Affairs
Real World Evidence (RWE):
Experiences & Lessons Learned
Medical Affairs Manager
Clinical Trial Workshop ELEFI CongressAthens 21 June 2018
CHC Medical Head
Bill Giannakopoulos
Objectives of this presentation
To describe what is RWE & what it provides
To understand the Value of RWE
To highlight present state & obstacles
To envision the future of RWE
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Real-World Evidence
Clinical Evidence
Οι τυχαιοποιημένες κλινικές μελέτες
αποκλείουν συστηματικά ειδικούς
πληθυσμούς - ασθενείς με άλλα νοσήματα,
παράλληλη χρήση φαρμάκων, σε
προχωρημένη ηλικία ή ασθενείς που
διατρέχουν κίνδυνο μη συμμόρφωσης
Η μελέτη πληθυσμών σε πραγματικές
συνθήκες παρέχει πρόσθετες
πληροφορίες για ετερογενείς
πληθυσμούς.
http://www.fda.gov/Safety/SafetyofSpecificProducts/ucm180547.htm. (Προσπελάστηκε τον Απρίλιο του 2010).
Γιατί Real World Evidence?
Real-world patients are diverse and complex
FOR MEDICAL AND SCIENTIFIC PURPOSES ONLY
DO NOT DISTRIBUTE OR USE IN PROMOTIONSAGLB.TJO.17.06.0585 June 2017
Typical features of different studies
Randomized, controlled studies (RCTs)
• Double blind/open label• Strict inclusion/exclusion criteria• Adherence encouraged/monitored• Frequent visit• Drugs provided• Traditional efficacy endpoints
Real-life studies• Open label• Broad inclusion/exclusion criteria• Set in normal care• No extra visits• Drugs prescribed and collected in usual way• Adherence, health outcome and resources
utilisation endpoints
RWE versus RWD
Real world data (RWD)
Data used for decision making that are not collected in conventional
controlled randomized trials (supplements to RCTs, QoL, registries,
Claims databases/administrative data; health surveys; electronic
health records and medical chart reviews).
https://www.ispor.org/terminology/ last accessed June 2017
Real world evidence (RWE)
Evidence derived from the analysis and/or synthesis of real-world data
(RWD).
RCTs determine whether an
intervention CAN work
Randomized real-life studies show
whether an intervention DOES work
Sources of Real World Data
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Χρειάζονται δεδομένα από τις μελέτες σε
πραγματικές συνθήκες για να ληφθούν οι
σωστές θεραπευτικές αποφάσεις;;;;
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The HCP experience
The lessons are being taught. ………….We need to learn.
RWE in LTPM: Patient characteristics change over a lifetime, influencing treatment choices
Age 20 50 70+4030 60
Initiation of treatment treatment = Patients growing old on treatment
AlcoholInfectionSmokingIVDU
CVD riskComorbidities
Concomitant medicationsAlcohol
More severe disease
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FOR MEDICAL AND SCIENTIFIC PURPOSES ONLY
DO NOT DISTRIBUTE OR USE IN PROMOTIONSAGLB.TJO.17.06.0585 June 2017
Real-world evidence can inform our understanding to enhance clinical practice
1. Cziraky M, Pollock M. Applied Clinical Trials 2015. Available at: http://www.appliedclinicaltrialsonline.com/real-world-evidence-studies. Last accessed June 2017.
Improve quality and delivery of
care1
Improve outcomes1
Reduce overall cost1
FOR MEDICAL AND SCIENTIFIC PURPOSES ONLY
DO NOT DISTRIBUTE OR USE IN PROMOTIONSAGLB.TJO.17.06.0585 June 2017
Patient Centric Approach: The Value of
RWE……
FOR MEDICAL AND SCIENTIFIC PURPOSES ONLY
DO NOT DISTRIBUTE OR USE IN PROMOTIONSAGLB.TJO.17.06.0585 June 2017
The Industry Experience:……….What’s in it for Pharma??
Registration
RWE is one step towards market access
The evolving role of Medical across the industry
FROM
Medical as a supporting role
TO
Medical as a core strategic partner
Medical
Commercial
R&D
MedicalR&DCommercial
What role should Medical Affairs play
in Real World Evidence?
To generate
compelling evidence
which can transform
clinical outcomes and
enhance access for
patients to products in
each business unit.
RWE can be generated by MedAffairs, Clinical and/or Access teams within Pharmaor through external CRO partnerships
Potential REAL WORLD Obstacles for RWE
RWE has not reached its maturity curve, yet it is accelerating.
Obstacle 1: ACCEPTANCE
Willingness to use RWE compared to RCTs.
Obstacle 2: ACCESS TO DATA
Ability to obtain data that meet quality, volume, risk
Obstacle 3: REGULATORY SUPPORT
Standards and guidelines that govern access.
RWE Governance in Greece
RWE Governance: What we know
Κατάθεση των Mη-Παρεμβατικών μελετων PASS μόνο στον ΕΟΦ για έγκριση.
Καταχώρηση των Mη-Παρεμβατικών μελετών στο www.dilon.sfee.gr
ΕΡΩΤΗΣΗ:
1) ΠΩΣ ΘΑ ΕΦΑΡΜΟΣΤΕΙ?
2) ΘΕ ΕΧΕΙ ΑΝΑΔΡΟΜΙΚΗ ΙΣΧΥ?
RWE Governance by EOF: What we do not know
7 Mar 2018 νέο ΦΕΚ (άρθρο 4 & 5) :
-σύσταση επιτροπής εγκρίσεων των Μη-Παρεμβατικών μελετών. - ηλεκτρονικό Μητρώο Μη-Παρεμβατικών μελετών αναρτημένη στον ιστότοπο τουΕΟΦ .
ΦΕΚ Α 41 7-3-2018_Μη παρεμβατικές μελέτες.pdf
Conclusion: The Future of RWE?
• MA should have a leadership role in in RWE from developing the evidence strategy through to supporting scientific exchange.
• There will be increased use of ‘hybrid roles’ that merge capabilities from HEOR, Clinical Development and Market Access.
• To design and execute pragmatic trials, fully understand clinical impact and the financial implications of a therapy on the health care system.
• Regulators/Payors will use RWE to compare treatments and to approve label revisions or expansions.
Where Pharma Needs to Progress.....
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Did you know??
The growth of RWE also raises questions about data access and patient privacy.
Who’s data is it?
In 2017, British hospital
trust was sued for misusing
data, after it passed on
personal information of
around 1.6 million patients
to artificial-intelligence
firm Google.
Ευχαριστώ!
Ερωτήσεις;
Bill Giannakopoulos