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

1

2

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