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
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Considerations for clinical trials in CML: Patient perspective
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.