"Patient-based evidence from the patient perspective", presented on 27 June 2014 by Jan Geissler, CML Advocates Network, at Novartis Global Patient Forum, Basel

1. Patient-based evidence
from the patient perspective
27 June 2014
Novartis Global Patient Forum, Basel
Jan Geissler, CML Advocates Network / EUPATI
jan@cmladvocates.net / jan@patientsacademy.eu

2. It is not just about clinical efficacy
Different patients want different things
 Being part of decision-making?
Master of your health vs “fix it for me”?
 A quick fix vs. long durable remissions?
 Maximum disease control?
 Fewer side-effects / better QoL?
 Oral outpatient treatment
or hospital based care?
 Ability to work? Have a social life?
 Impact on family / family planning?
 Financial impact (travel,
patient/carer’s ability to work)

3. Perception of doctors, nurses, patients
differs largely
 Balance of risks vs. benefits
 Balance of side-effects
vs. treatment efficacy
 Which treatment-related side-effects
have the greatest impact on QoL
Want some evidence?
Here it is.

4. Doctor/patient perception on impact on QoL differs
4
The Myeloma Euronet Study
Detecting Myeloma, ways to shortening an often painful and tedious patient odyssey: results from an international
survey. Myeloma Euronet (2009). 314 physicians & nurses, 260 patients & carers, 43 countries

5. Doctor/patient perception on impact on QoL differs
The CML Quality of Life Study
Efficace F, Rosti G, Aaronson , et al, Haematologica, 2013
Nausea
Headache
Fatigue
Diarrhea
Muscle cramps
Edema
Skin problems
Pain (Musculo-skeletal)
Abdominal discomfort
Health Status
Patient graded higher Physician graded higher
N=422

6. “This is cancer!
- of course my patients are taking their drugs!”

7. Truly global patient-led research in 12 languages:
2546 CML patients from 79 countries participated
Sample: Total of 2546 respondentsGlobal reach
Methodology
• Online - Recruited by patient associations online & via other methods
• Paper & Pen (France, Germany, Italy) – Recruited by physicians at consultations
• CML patients over 18 years old, currently taking oral medication for CML
• Fieldwork – Started on CML World Day, 22/9 2012 – 30th January 2013
2151
395
Paper
Online
Countries with >30
respondents
Countries with base <30
respondents
Sharf et al., Haematol 2013; 98(s1), EHA-Abstract [1104]; Geissler et al., Blood 2013;112:4023. ASH-Abstract [4023]

8. Valid data? We also used the validated Morisky Adherence
Scale to classify patients into adherence levels
Questions:
Forget medication
Miss for other reason
Stopped because felt
worse
Forget when travelling
Take yesterday?
Stop when under control
Inconvenience
How often difficulty
remembering
Low: 21 %
Medium: 47 %
High: 33 %
Adherence score
classifies patients into
adherence groups:
Sharf et al., Haematol 2013; 98(s1), EHA-Abstract [1104]; Geissler et al., Blood 2013;112:4023. ASH-Abstract [4023]

9. Patient Based Evidence from the Patient Organization Perspective
Our expectations for future patient based evidence reporting is …
 Who
• Patients should be part of the research team,
especially in terms of outcomes, side effects, QoL measurement
 How and when
• The patient is the only source of data for QoL, side effects, symptoms
• Involve patients at the design stage of research,
not when the protocol is already done.
 Reliability / validation
• Patient evidence can use validated tools,
will increase confidence when decisions are made
 Impact
• Solid PRO data based on true patients needs
might make or break trials, treatments, decisions
• It will influence the way doctors and patients manage the disease

10. No research
about us
without us!
Jan Geissler
jan@cmladvocates.net
jan@patientsacademy.eu
Twitter @jangeissler