Considerations for clinical trials in CML: Patient perspective, presented in the "Meet the Expert" Session of the Global Oncology Leadership Summit in Berlin, 28 Feb 2014

1. Meet-the-Expert session:
Considerations for clinical trials in CML
Patient Perspective
Jan Geissler
Co-founder, Leukemia Patient Advocates Foundation
& CML Advocates Network & LeukaNET
Director, European Patients’ Academy on Therapeutic Innovation
(EUPATI)

2.  Spreads news,
builds skills and tools,
builds capacity,
collaborates and builds partnerships.
 Established 2007
 Connecting 95 CML patient
organisations in 72 countries today
 Low budget, high impact

3. Current research on CML,
and future patients’ needs
 Today’s CML trials assume all treatments and good PCR are available,
focus on optimal use
• What drug and dose gives best efficacy and “MR4.5 rates”?
• When is the best time to switch TKI (ELN, NCCN guidelines, milestones,
mutations)?
• When and who can stop treatment (treatment-free remission)?
• How to improve speed and depth of response
 Gaps: Cure, Quality of life, best use of existing therapies!
• We need a cure - new therapies that truly eradicate CML
• Impact of additional diseases (“co-morbidities”) on CML therapy
• Efficacy might be not very different but quality of life may:
How to optimize quality of life on low-grade side effects of TKIs?
Lower dose trials?
• Pregnancy & fertility in young(er) patients
• Psychology of TFR

4. Challenges in CML research,
and room for additional research
 Recruitment in new trials on a “high comfort level”
• Will patients accept additional tox of combo treatments, given QoL
of existing 5 TKIs is relatively good?
• This needs very good justification, patient information, informed
consent, retention strategy.
 TFR
• very important but only feasible for ~30% of CML patients that will
get into MR4.5 and maintain MMR…
 Perfect environment vs. reality:
• Comorbidities in the “true 65+ population” (ENEST, DASISION,
IRIS: median age ~47 years)
• Imatinib generics – no comparative clinical data of generics and
copy drugs. Lack of data when healthcare decides to do IM-first or
IM-switchback.

5.  Before a trial starts
• Identify therapy gaps, target patient population
• Identify ethical or risk/benefit dilemmas
• Identify patient needs, barriers for recruitment
• Define relevant endpoints and PRO (PFS, OS, QoL)
• Make informed consent a meaningful tool
 While trial is in progress
• Managing expectations: hope/hype
• Inform (potential) participants
• Support patient recruitment, retention
 After conclusion of trial
• Dissemination of research results
• Improving adherence
• Reimbursement, health technology ass and
(cost-)effectiveness assessment
Patient Advocates’ potential
contribution to clinical trials

6. Our community action to increase
collaboration in CML research
 “CML Advocates Network Research Workgroup” set up
 European Patients’ Academy on Therapeutic Innovation
develops training material on medicines R&D
 CML Horizons conference
for capacity building
 Working with study groups,
ELN summary, etc.
Greatest impact of patient
input created in design
phase!

7. Practical “Roadmap” of examples
unique insights of “expert patients” along the whole R&D development life cycle
Trial design phase
Design of Protocol:
• relevant end points (e.g. PFS vs. OS)
• benefit/risk balance
• inclusion / exclusion criteria,
target population
• quality of life and choice of QoL tool
• patient reported outcomes
• ethical issues, data protection
• cross-over
• frequency of visit, remote monitoring
• Regulatory Protocol Assistance
Informed Consent:
• content
• visual design
• readability, language
Study reporting:
• summary of publication
findings (interim findings),
dissemination in patient
community
Post-study
communication:
• Information to participants,
“thank you letter”
• synopsis of results /
dissemination of results
• Communication of results
to practice (professional
community)
Patient Information
Leaflet:
• content
• visual design
• readability / language
• dissemination strategy
Trial steering
committee:
• follow up the
protocol
Investigators Meeting:
• patient perspective on trial,
recruitment, challenges,
opportunities
• can trigger amendments
After end of trial
Level of expertise in the disease area required:
mediumhigh
Data Monitoring Committee:
• impact of the results on patients
• recruitment issues
• assessment of side effects
• assessing adherence issues, study
retention / drop-outs
Practical con-
siderations:
• e.g. travel
expenses, support
for family members,
mobility
Health Technology
Assessment:
• assessment of value
Protocol
Synopsis:
• design,
comparators
Research
Priorities
Setting
research
priorities:
• Gap analysis
• Early horizon
scanning
• Matching unmet
patients’ needs
with intended
research
outcomes
• Defining patient-
relevant added
value
Fundraising
Information to trial participants
• When protocol amendments need to be made
• New safety information
Implementation/
While the trial runs
Source: Geissler, Ryll,
EPALCO (2014, unpublished)
Regulatory affairs:
• Application evaluation
• EPAR Summaries
• Package leaflets
• Updated safety comm.

8. Collaborate!
Jan Geissler
jan@cmladvocates.net
Twitter @jangeissler