2. eurordis.org
Triple objective
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Eyewitness of the regulatory process
• See regulators at work
• Report back to your community
Contribute to the scientific discussion
• Bring your own perspective as a patient
• No room for political stands, lobbying, confrontation
Propose other approaches to involve patients
• Evaluation: how to best organise the consultation?
• For you, and for future consultations
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3. eurordis.org
1992
3
“With a high quality
dialogue, patients and
regulators can only agree.”
Jean-Michel Alexandre
Agence du Médicament, France
Former CPMP chair, EMEA
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4. eurordis.org
The law. Reg. (EC) No 726/2004
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• Article 63.2 About the European Medicines Agency
• Members of the Management Board, members
of the committees, rapporteurs and experts shall
not have financial or other interests in the
pharmaceutical industry which could affect their
impartiality. They shall undertake to act in the
public interest and in an independent manner.
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5. eurordis.org
Case 1: how would you react?
• CHMP invites MAA,
clinicians and patients to
SAG meeting (questions
sent in advance)
• CHMP asks a specific
question to patients
• MAA representative
explains they had decided
the clinicians would
answer this question
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• EMA experts realised
patients, clinicians and
MAA had been meeting
before and coordinating
their responses
• Patients, clinicians were
clearly under influence
• SAG meeting could have
stopped there
• What happened • Impact
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6. eurordis.org
Case 2: how would you react?
• CHMP invited 2 patients to an
oral explanation with MAA
• Two hours before the meeting,
MAA, all 3 patients and
clinicians who had been
working on the product for
years all together met in a
hotel, prepared by MAA
• Mentor explained the issue
– asked MAA reps to leave the
room
– advised not to have the meeting
– finally left the room and informed
the EMA about the incident
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• Even if patient invited by MAA
claimed to be independent, this
could be questioned
• CHMP agreed to have the 2
patients they invited in the
room, as they were there
anyway
• CHMP chair explained the
situation before meeting started
• What happened • Impact
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7. eurordis.org
Case 3: how would you react?
• Company launched a first
clinical trial in 24 patients
• After 9 patients treated for 6
months, press release on
“promising results”
• Patient quoted "We welcome
the company update and
appreciate the efforts they are
making in developing a
treatment for this deadly ultra-
rare disease," said X, Chief
Exec, Disease Family
Association
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• What happened • Impact
• Press-release was clearly
investors oriented
• No anticipation of the impact
on patients (compassionate
use)
• Putting the quoted patient in
a difficult position vis-à-vis
other patients
• Linking an organisation to a
company with transparency
issues for future regulatory /
HTA consultations
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8. eurordis.org
Case 4: how would you react?
• Patients’ organisation (A)
co-sponsor of a clinical trial
with 2 pharmaceutical
companies
• Patients from other
organisations unsatisfied
with the information
provided on when a MAA
could be submitted
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• What happened • Impact
• Was there a financial
arrangement between A and
the 2 companies (royalties)?
• How would a delegation of
patients’ organisations lead
by (A) be looked at when
meeting with a regulatory
agency?
• Should (A) be considered as
a patients’ organisation or a
pharmaceutical company?
9. eurordis.org
Possible solutions
• Financial arrangements
– Compliance with EMA criteria for eligibility
– Compliance with EMA requirements on financial
information
• Influence in general
– Use the Code of Practices between Patients’ organisations
and the healthcare Industry
• Practical arrangements for patients’ organisations
– Those raising funds from industry should be different
people from those discussing R&D with industry,
regulators, HTA doers and price negotiators
– And preserve the independence of the latter
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10. eurordis.org
Code of Practices
• Written and endorsed by patients’ organisations
eligible to work with the EMA
• Code of practices
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Promotional activities
Patient organisations should develop a list of the types of activities
that can be considered promotional and therefore might cause a
conflict of interest and be against the law. The list should include
the following:
• Being quoted in the company’s corporate communication in
favour of, or against a product.
• …
Industry press releases
Patient organisations and their representatives must be vigilant
and refuse to be quoted in industry press releases that relate to a
marketed product or a product under development
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