Changes in patient organisations - and how this changes the medical world - presented by Jan Geissler at EBMT Annual Meeting, 24 March 2019

1. Changes in patient
organisations - and how this
changes the medical world
Jan Geissler @jangeissler
CEO Patvocates; Co-founder CML Advocates Network;
WP co-leader IMI HARMONY; ePAG member EuroBloodNet; Advisor ESMO, ECCO, EHA
1

2. A common perception of what patient organisations are…

3. ...and how they operate…

4. Patient activism from 1968

5. This is today’s reality of patient advocacy
Provide
patient support
Shape
health & regulatory
policy
Support and shape
research

6. Patient advocacy operates on
3 “horizontal” and 3 “vertical” levels
6
Patient support
Inform, support, navigate
Health policy
Influence health policy,
patient care
Research
Contribute in partnership with
clinicians, networks and industry
Local/
national
Information and support, national policy, awareness, campaigning, access (reimbursement)
Largest and (locally) most influential organisations operate within the national level.
European
EU policy / frameworks / systems, research collaboration,
access (systems, inequalities), evidence generation, inequalities
Strengthen, share, disseeminate, amplify work of national member organisations.
Global Some European-based organisations are Europe-resident global coalitions,
or members of global umbrellas or alliances

7. European patient advocacy community
(cancer, rare diseases, chronic diseases)
EUROPEAN
CANCER
PATIENT
COALITION:
>400 mostly national
patient organisations
WECAN:
Workgroup of leaders
of 22 European
Cancer patient
Advocacy
Networks
ALL CANCERS
RARE
DISEASES
RARE
CANCERS
HEMA-
TOLOGY
PAEDI-
ATRIC

8. European hematology patient community:
14 networks, >270 national organisations in Europe

9. Key activities of the umbrella organisations
• Developing patient information – most provide at least some basic disease
information, but some more than others. Often this is a domain/specialism of the
national organisations working in their field.
• Sharing best practices/facilitating networking – all do this.
• Provide a knowledge base/sharing educational and research resources – most do
this in varying degrees.
• Advocacy resources/toolkits – most do this.
• Involvement in research/regulatory projects, guideline/clinical practice
development – some do this more than others.
• Global/regional surveys – most carry out some form of patient experience
research.
• Healthcare professional education programmes – a small number provide such
learning.
• Awareness raising/public campaigns – most/all involved in this area of work.

10. European hematology patient community is
collaborating with key hematology societies and
initiatives

11. Successful collaboration with EHA
• EHA Fellowship Programme:
55 patient advocate leaders at 2018 congress
• EHA Capacity Building Programme
• Patient Advocacy Track sessions
2 until 2018, 3 from 2019
• EHA PO Workgroup (14 umbrellas)
• Patient Advocacy Booth
• Speaker at EHA Education Sessions 2017
on „Fertility preservation“
• Member of EHA European Affairs Committee
• EHA Patient Organisations Workgroup
11

12. Focus on unmet needs: Avoidable waste in the
production and reporting of research evidence
Chalmers I, Glasziou P. Lancet 2009;374:86–9
Over 30% of trial
interventions not sufficiently
described
Over 50% of planned study
outcomes not reported
Most new research not
interpreted in the context of
systematic assessment of
other relevant evidence
Unbiased and
usable report?
85% research waste = over $85 billion / year
Low priority questions
addressed
Important outcomes not
assessed
Clinicians and patients not
involved in setting research
agendas
Questions relevant
to clinicians &
patients?
Over 50% studies designed
without reference to
systematic reviews of
existing evidence
Over 50% of studies fail to
take adequate steps to
reduce biases, e.g.
unconcealed treatment
allocation
Appropriate
design and
methods?
Over 50% of studies never
published in full
Biased under-reporting of
studies with disappointing
results
Accessible
full
publication?
1 2 3 4

13. Data, not just
opinions:
Evidence-based
advocacy

14. Patient involvement in clinical trials in practice
Improving Patient Involvement in Medicines Research and Development: A Practical Roadmap. Geissler, Ryll, Leto, Uhlenhopp, Therapeutic Innovation & Regulatory Science (2017),
doi: 10.1177/2168479017706405, and at www.eupati.eu
Setting
research
priorities
Protocol
synopsis
Protocol
design
Trial
Steering
Committee
Information to
participants
Data & Safety
Monitoring
Committee
Investigator
meetings
Fundraising for
research
Practical
Considerations
Patient
Information
Informed
Consent
Ethics
Review Study
Reporting
Post-Study
Communication
Regulatory
Affairs
Health
Technology
Assessment
Research Design
and Planning
Research
Priorities
Research Conduct and
Operations
Market Authorization and
Post-approval

15. Contributions to IMI HARMONY
• One of Europe’s largest Big Data projects
• Builds a high-quality Big Data platform for AML, NHL, CLL,
Myeloma, pediatric leukemias, MDS
Currently >4.500 AML cases being uploaded
• Speed up drug development, access pathways and bench-to-
bedside process
• Harmonization of outcome measures and endpoint definitions
for HMs at European level
• Increase the application of omics data in clinical practice
Our role as patient organisations:
• Work package co-lead to involve patient orgs, regulators, HTA
• Input into research projects and legal/ethics framework
• Core outcome set defition (Delphi)

16. Community Advisory Boards, e.g. CML, Hematology, Myeloma
• Patient-run community advisory boards where patient organisations set the
agenda and invite stakeholders
• Two-way dialogue with researchers, academics, authorities and
pharmaceutical industry, to improve patients’ outcomes
• Address & tackle challenges that patients face
in accessing diagnosis, monitoring, treatment
and care
• Help develop patient-focused trials CAB
1st Hematology CAB

17. European Patients‘ Academy (EUPATI):
Patient Expert Training Course & web-based training:
www.eupati.eu
1. Discovery of Medicines
2. Pre-clinical Development
3. Clinical Development
4. Clinical Trials
5. Regulatory Affairs, Drug
Safety,
Pharmacovigilance
6. Health Technology
Assessment

18. What’s next?
• Patient advocacy is not a new fashion, it is becoming an essential part of medicine.
• While patient support is at the core of what we do,
we are increasingly shaping healthcare policy, regulation and research
• We are moving from anecdotes to data:
Evidence-based advocacy underlines our opinions on patients’ needs
• Successful studies will involve patient advocates in design and conduct to deliver
patient-relevant outcomes, e.g. in choice of endpoints, QoL instruments, feasibility,
patient information
• Set your priorities and partner with us! The hematology patient community is
strong, well-trained, and will make your work more impactful for patients

19. Involve us!
Jan Geissler
@jangeissler
jan@patvocates.net