Orphan Drugs &
Personalised Medicine:
The Patient Perspective
Dr. Cees Smit,
Inspire2Live/VSOP/EGAN
DCTF, Ede, October 8, 2014
a Patient Advocacy Platform, bringing together
patients, doctors, scientists and others to work on the
challenge to get cancer under control.
Content
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Fundamental Research (Animal Research)
ALS Ice Bucket Challenge
Unmet medical needs
Innovative Power of Patients
Personalised Medicine
EU New Clinical Trial Regulation
Conclusion
The other side of the coin
• 2004: Foundation Informatie Dierproeven
(SID), founded by
• Patients: VSOP
• Scientists: Federa (FMWV)
• Animal lab scientists (NPV)
• www.understandinganimalresearch.org.uk
• www.eara.eu
www.informatiedierproeven.nl
You Tube movie
‘Dierproeven doe je niet zomaar’
3 minuten
De patiënt in 40 jaar
Patiënt 1.0
Informatie, voorlichting
Lotgenotencontact
Belangenbehartiging
Patiënt 2.0
Internet, social media, Wiki’s,
Patientslikeme, Mijn Zorgnet
Patiënt 3.0
Samenwerking onderzoekers,
patiënten en bedrijfsleven
Drijvende kracht:
‘Unmet medical needs’
Dutch ALS campaign
‘I’m now deceased’
treeway.nl & projectmine.com
The ALS Catch-22 Challenge
Every year in Europe 22.000 new cases
Every year in Europe 22.000 deaths of ALS
This is why we need as patients a
fast and effective clinical trial
regulation in Europe
for unmet medical needs
Adaptive licensing
• ‘New medicines for serious conditions: how
patients would weigh benefits and risks’
• Report form Genetic Alliance UK with
Welsh Institute for Health and Social Care
• April 2014, www.geneticalliance.org.uk
Unmet medical needs
Unmet medical needs
are the driving force
for patient groups
to become active in medical research
Proportion of patients
Hemophilia, age-distribution (n=338)
100%
90%
80%
70%
60%
>40 years
30-40 years
50%
40%
30%
20%
10%
0%
1975
20-30 years
10-20 years
0-10 years
1985
1995
Year
2000
Mauser et al, Van Creveldclinic, Utrecht, 2010, personal communication
2009
The patient in 40 years
Patiënt 1.0
Information, education, peer support
lobbying for their interests
Patiënt 2.0
Internet, social media, Wiki’s,
Patientslikeme, Mijn Zorgnet
Patiënt 3.0
Cooperation patients, physicians,
researchers and industry
Driving force:
‘Unmet medical needs’
‘Trek’ or ‘Pull’ model
Innovative power patients
• To bring together patients and their body
material (DNA, tissue, etc.)
• Sharing of data for research and the
organization of cooperation (EuroBiobank)
• Fundraising for biobanks and research (gene
therapy), also for the long term (Téléthon)
(Ref.: Van der Valk & Smit, NTvG, mei 2011)
Genethon BioProd → Genopole
Genopole, Paris
• The Genopole project was conceived in the late
1980s by Bernard Barataud (the then President of
the French Muscular Dystrophy Association,
organizer of France’s first Telethon fund-raising
event and the founder of Genethon, a hi-tech
genomics and genetics research lab) and Daniel
Cohen (Scientific Director of the Foundation)
• Now, 15 years on, the biopark is home to 71
innovative companies and 21 academic research
laboratories.
Artritis research
• Patient reported outcome measures
• So far from scientists: pain
• Addition from patients: Fatigue
Sleep disturbances
• Maarten de Wit (EULAR), thesis 2014
Six minute walking test
• Additions from the Duchenne community
• Small movies on relevant patient outcome
measures for EMA (very well received)
Movie Duchenne
Personalised Medicine (1)
For patient registries/biobanks as a
research infrastructure tool we need
appropriate long-term finance structures
Good examples: HIV, HOVON, Parelsnoer
For example: by a percentage of a DOT/DBC !
Personalised Medicine (2)
• The new EU Data Protection Regulation tends to
ignore the negative effect the new Regulation
could have on scientific and medical research
• A viable alternative could be to have a separate
legislation specific to data protection in the
areas of health and medical research.
• A precedent: Directive 2006/123/EC
(the ‘Bolkestein directive’ on services)
Personalised Medicine (3)
• Registration & Reimbursement needs of n = 1
products instead of n = 10.000 needs
rethinking (a paradigm shift)
• The same for therapeutics & diagnostics
• The rare disease example: the need for EUwide databases and procedures (legislation)
NOS Journaal, Juli 2012
Meaningful Patient Involvement
• Value +, European Patient Forum (EPF)
• Patient Partner, EFGCP, EGAN, ECRIN, EPF
• Summer School Eurordis, LSE
• EUPATI, the EU Patient Academy
EU Regulations
• EU Clinical Trial Directive (2000)
• EU Clinical Trial Regulation (2014 – 2016)
EU CTrR Art. 2, point 11
• ‘Ethics committee’ means an independent
body established in a Member State in
accordance with the law of that Member State
and empowered to give opinions for the
purposes of this Regulation, taking into
account the views of laypersons, in particular
patients or patients’ organisations
EPF opinion www.eu-patient.eu
• ‘We believe the review of the legislative
framework presents an opportunity for
reform towards more patients’
involvement throughout the research
process; greater trust and public
confidence in medical research; and
improved participation rates’.
Input from patient groups (1)
• Nationally, groups of 10-20 patients could be
trained and assist CA/EC in giving their views
(disease-specific or disease-exceeding)
• Before application however, timely
consultation of relevant patient groups is
essential to discuss pros and cons of proposal
• There are not so many cultural differences for
patients within the EU MS (access)
• Number of EC’s in EU is too big to be effective
Input from patient groups (2)
• In some EU MS there is already experience
with pat reprs in EC’s, like in the UK
• National Policy of NHS: Involve
• In some EU MS patient participation in EC’s
is forbidden by law (The Netherlands, Germany)
• But in an optimal ethical review in the next
decade, there is no space for paternalism !
We are positive, but
• Patients wants faster access to medicines
& therefore a well balanced administrative burden
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New EU regulation animal research
New EU clinical trial regulation
New EMA procedures on transparency
New EU Data Protection Directive
Growth in HTA calculations
Difficult ICT mechanism for CTrR
• EU strong in procedures/processes, but weak in vision !
(ref.: Jonathan Holslag, The Power of Paradise, 2014)
be more competitive !
• My estimation is that all the requirements to
fullfill all clinical trial requirements and
reimbursement procedures in all EU Member
States make up 50 – 80 percent of the
developments costs of new medicines.
• That’s too much to make the EU competitive
& that was the reason for the revision of CTrR
UK discussion: merge Part 1 & 2
In conclusion
• European patient groups are in principle
positive about the new regulation, but we
hope we will be taken serious and really
involved in the implementation at the level
of the CA/EC of national Member States
• In the past decade EU Patient Groups have
proven to be serious partners in the
development of therapies and medicines
For more information
Dr. Cees Smit
info@smitvisch.nl
www.smitvisch.nl
www.inspire2live.org
www.vsop.nl
www.egan.eu