Presentation of Jan Geissler at EFGCP Annual Conference on 29 January 2014 about "Harms and Benefits in Health Research: The patients' perspective".

1. Considering
Harms and Benefits:
A patient perspective
29 January 2014, EFGCP Annual Conference
Jan Geissler, CML Advocates Network / EUPATI

2. Willingness of patients to take risk for
potential benefits differs
Survival probability
CML IV
2013, 10 years survival 83%
Mortality of co-morbidities > CML
CML IIIA
2000, 10 years
survival 50%
CML III
CML I/II
CML I/II
CML I
Year after diagnosis
German CML Study Group (2013)
1990s

3. Quality of life (%)
Cure
Un
tre
ate
Effective
palliation,
increased
survival
d
Effective
palliation,
same
survival
Overtreatment
decreased
survival
Overtreatment,
same survival
Overall survival
Start of therapy
Lifetime gained
3
Senn. H. J.. Z. Allg. Med. 55. (1979) 284-295.

4. It is not just about clinical efficacy:
Different patients want different things
Being part of decision-making?
Ownership 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)

5. There is no „magic bullet“
for most patients yet


“Success stories” available only to
small numbers of cancers,
rare diseases, patients at “best age”
Research is crucial to address unmet
needs
RareCare (Gatta et al, 2012)

6. What patients need to know
about BENEFITS in clinical trials

Knowing that trials exist at all

Benefits of participation in trials
• Personal benefit:
Access to innovative treatment,
“hope” or “last resort”,
hope for response, closer monitoring,
better quality of life
• Altruistic reasons: Contributing to progress
beyond their own case

7. Quality measures of study sites are
improving overall quality of care & outcome

Example quality assurance programme QIII 2001
German Working Group Gynecological Oncology Ovarian
Cancer (AGO OVAR)

Standard therapy FIGO I-IIA
Site without clinical trials (n=68)
Study center (n=56 patients)
52%
48%
25%
Standard
therapy
Suboptimal
therapy
75%
Jalid Sehouli (Charité), on: QS-Programm der AGO Organkommission OVAR, Zentralbl Gynakol 2005; A. du Bois DOI 10.1055/s-2005-836289

8. What patients want to know
about RISKS in clinical trials







Uncertainty/risk of therapy,
prior results of studies
Impact on quality of life,
unpleasant diagnostics
Risks about fertility
Pediatric trials: administration
Influence on family life
(intensity of care) and financial impact (travel)
Influence on ability to work (both patient & carers)
Protection from unauthorized and unwarranted use
of data and tissue: stigma and discrimination

9. Barriers to trial participation are well
documented 1





1
Uncertainty: Proven vs. experimental
Lack of information and understanding
(no real „informed consent “)
Therapy preferences, fear of randomization
Impact of quality of life,
inappropriate burden of diagnostics
Time of recruitment conflicts with patients‘ life
(e.g. family planning, children, work, mobility)
Fayter, D. et al. (2006): Systematic Review of barriers, modifiers and benefits involved in participation in cancer clinical trials.

10. Five suggestions
to improve
patients’
understanding
of harms/benefits

11. 1. Make „informed consent“
and patient info more accessible
§

12. 2. Involve patient
organisations as navigators






Average consultation: 8-12 minutes
Medical language barrier
Different perceptions on what matters
to patients
Poor Adherence
Patient organisations: experts on patient
information, quality of life, anxiety,
expectation management, “navigation” to
clinical excellence, compliance
Help understanding benefits and risks

13. 3. Educate the public on “basics of
research” to improve understanding of risk
and benefit in research
?

14. 4. Increase transparency of research
(results, harms and benefits) to patients
Research only helps if it reaches out to patients!

„Had I only known about this study / had I understood“
(Most patients are not even informed about the existence of studies, or there is no time for
consultation. Patient organisations often act as navigators.)

„What was the result of ‚my‘ study?“
(90% of study participants want to know results of the study1, 93% never receive study
results from doctors or sponsors2, 98% of study doctors would like to share results3)

Only what is being published is also being picked up
(32% of industry studies, 18% of academic studies have not been published 5 years after
conclusion of trial, BMJ @ 585 studies with >500 participants 4)
1. Shalowitz, D. and Miller, F. 2008. PLoS Medicine. 5: 714-720. 2. Sood, A. et al. 2009. Mayo Clinic Proceedings. 84(3): 243-247.
3. Dixon-Woods, M. et al. 2006. BMJ. 332: 206-210.
4. Jones, C et al, BMJ 2013;347:f6104

15. 5. Seek input from patient organisations in
regulatory risk/benefit assessment
Example EMA:
Considering refusal, withdrawal, suspension, revocation of
a medicine in an area where there remains unmet medical
need
Advice on package leaflet,
risk management plan
Acceptability of
comparators and
endpoints
Ethical issues
Drug shortages

16. No research
No research
about us
about us
without us!
without us!
Jan Geissler
Jan Geissler
jan@patientsacademy.eu
jan@patientsacademy.eu
Twitter @jangeissler
Twitter @jangeissler