This document discusses the benefits and risks of patient involvement in pharmacovigilance based on a case study. It notes that proactively involving patient groups and representatives in assessments helped manage the crisis. Lessons included being available to patients and dealing with scientific complexity. The document outlines objectives to better understand patient information sharing online and develop apps to facilitate two-way communication between patients and regulators on medicine safety. It acknowledges challenges like varying awareness of regulators across Europe and finding trusted information sources. Finally, it recommends designating a "Vigil" contact in patient groups to liaise with regulators on pharmacovigilance issues.

1. François Houÿez
EURORDIS Summer School 2018, Barcelona
Which benefits for which risks? 1

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“Never trust anyone who doesn’t
have skin in the game. Without it,
fools and crooks will benefit, and
their mistakes will never come back
to haunt them.”
Nassim Nicolas Taleb

3. A case study
Thanks to: Frédéric Bouder, Maastricht University, David Haerry, EATG
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4. A case study (2)
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5. A case study (3)
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6. A case study (4)
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7. A case study - epilogue
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8. EMA proactively involved a patient representative
in all assessments / recommendations it made
MAH proactively involved patient groups
Local regulators proactively involved patients
Patients got alternative advice and maintained
separate channels of contact with MAH
Key to crisis management: engagement
of patients. Are you ready?
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9. Lessons learned
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Time: be available on short notice for calls, communication review & trips to
emergency meetings in London, for free
Dealing with complex matter while having little scientific understanding about it
Being in the sandwich between Agency and your group (inevitably)
Long uncertainty between detection and final assessments (here 13 months)
Sometimes conflicting voices; national & intl. dynamics
Difficult to communicate credibly between initial high risk alerts and final low risk
outcomes

11. Objectives
Patients exchange information on social media
• Can we access and understand what they say? Data mining/analysis
• Starting with analysing posts on medicines and their safety
Internet users use apps, no desktops anymore
• Can an app facilitate two-way communication between patients and
regulators?
• Starting with reporting side effects, signal detection
How regulators or industry can “mine” social media
searching for data: data protection, privacy, policy

13. To come (new consortium FDA, EMA, MHRA, UMC-
WHO, EFPIA, EURORDIS)
Non-
interventional
studies
PASS
Compassionate
use
programmes
Bar-code
reading
Batch
number
(biologicals)
Counterfeited
Recognition
Size
Colour
Shape
Motif
Pattern
Scheme
Leaflet
Educational
materials
Other apps
Adherence
Support
programme

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Reporting ADRs:
Sharing information is key
But for you: why does it matter?

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Challenges
Where do patients look for information?
Q5(a): How easy is it for you
to find information about
medicines from each of the
following sources?
Q5(b): How trustworthy do
you believe the following
sources are in providing you
with advice on the side effects
associated with specific
medicines
Source: Dominic Way’s (King’s College London) and
Frederic Bouder’s (Maastricht University) research (2012-
2015)
Juan Garcia Burgos
Head of Medical and Health Information
Communication Department

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Challenges
Awareness of regulatory authorities
Variation across Europe (general public). Hopefully greater awareness
among patients’ groups
Have you heard of [relevant national authority]?
a all superscript letters that differ between nations denote a statistically significant difference (p < 0.05) in the percentage of respondents answering “yes” between
those nations (determined by pairwise comparisons in a generalized linear model with logit link, binomial distribution, and Bonferroni corrections for multiple
comparisons)
Source: Dominic Way’s (King’s College London) and Frederic
Bouder’s (Maastricht University) research (2012-2015)
Juan Garcia Burgos
Head of Medical and Health Information
Communication Department

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Risks common to all anti-PD1: immune response reaction 10-15%
Endocrino
Hypo or hyperthyroidism
Diabetes
Hypophysitis
Neuro-muscular
Myositis
Epilepsies
Inflammation of nervous system
Ophtalmo
Uveitis
Renal
Nephrititis
Digestive
Colitis
Pancreatitis
Hepatitis
Pulmonary
Pneumopathies
Transplant rejection Cardiac
Myocarditis
The anti-PD1
Melanoma Patient Network Europe, Bettina Ryll
2,000+ melanoma patients discussing their
experience with anti-PD1 online

18. To conclude: think of a contact person for
pharmacovigilance in your group. the “Vigil”
• Each agency appoints a contact person among organisations: the Vigil
– Equivalent of the “QPPV” for industry
– France: 77 accredited organisations / 7 in the ANSM committee, for a total of 2 200
patients’ organisations
• Trained on what pharmacovigilance is and how it works
• Receives safety alerts and DHCP letters, share them with members
• Informed about international initiatives
• Aware of communication campaigns and tools (Take & Tell, SCOPE)
• Consulted when documents for the public are prepared
• Receives questions from patients, follows posts on social networks
• Organises two-way communication
→ CIOMS working group (Council for International Organizations of Medical
Sciences, by WHO and UNESCO)
28/06/201
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Thank you!
francois.houyez@eurordis.org