1. The Patient Journey Planner: an integrated approach to trials of targeted therapies with application to cancer and haematological malignancies Robert Hills1, Nigel Russell2, Kerry Hood1, Girish Patel3, Richard Adams1 1 Centre for Trials Research, Cardiff University, Cardiff UK; 2 Nottingham University Hospitals NHS Trust, Nottingham UK; 3 Cardiff and Vale UHB, Cardiff UK

2. Background Large simple trials have advantages
Wide eligibility
Real-life
Easy to participate in
Allow detection of moderate treatment effects
But increasingly, conditions are becoming more delineated
Smaller groups of patients
Disease doesn’t stay the same (clonal evolution)
Requires identification of what targeted treatment may be helpful
What is best for a patient may change as time goes on

3. Challenges of an all-comers trial

4. AML: a suitable case for treatment?

5. NCRI AML17 Trial
Pacritinib on relapse
viii

6. NCRI AML17 Trial viii Pacritinib on relapse Monitor Marker identifiedix
No monitor

7. Breaking this down… What is the best induction treatment?
Based upon emergent risk factors …
Is a suitably targeted therapy beneficial?
Is treatment intensification worthwhile in high risk patients?
Does monitoring disease levels allow for early intervention for relapse – and is it beneficial?
In patients who relapse
What is the best therapy?
Does the targeted therapy Pacritinib show promise in patients with a FLT3 mutation?

8. In other words… What is best at a given stage in the patient journey?
Trial presents the information relevant to a patient at that stage in their journey – just as in a clinical consultation
70% of all younger patients enter this protocol

9. Dealing with targeted therapies
Prompt identification of targets (see poster by Betteridge)
Umbrella trial
But groups may be very small
Master protocol
“Treatment” Trial 1
“Treatment” Trial 2
“Treatment” Trial 3
“Treatment” Trial 4
“Treatment” Trial 5

10. Marquee trials
Interim review for futility of new therapies – rotation of therapies in the trial (see Thomas – next talk)

11. Patient Journey Planner
Available options depend on patient stage; previous treatments; biomarkers
Option
Option
Option
Option
Induction
Option
Option
Option
Option
Consolidation
Option
Option
Option
Option
Transplant
Option
Option
Option
Option
Maintenance
Option
Option
Option
Option
Relapse/ Refractory

12. Patient Journey Planner
Option
Option
Option
Option
Induction
Option
Option
Option
Option
Consolidation
Option
Option
Option
Option
Transplant
Option
Option
Option
Option
Maintenance
Option
Option
Option
Option
Relapse/ Refractory

13. Patient Journey Planner
Option 
Option
Option
Option
Induction
Option
Option
Option
Option
Consolidation
Option
Option
Option
Option
Transplant
Option
Option
Option
Option
Maintenance
Option
Option
Option
Option
Relapse/ Refractory

14. Patient Journey Planner
Option 
Option
Option
Option
Induction
Option
Option
Option
Option
Consolidation
Option
Option
Option
Option
Transplant
Option
Option
Option
Option
Maintenance
Option
Option
Option
Option
Relapse/ Refractory

15. Patient Journey Planner
Option 
Option
Option
Option
Induction
Option
Option
Option 
Option
Consolidation
Option
Option
Option
Option
Transplant
Option
Option
Option
Option
Maintenance
Option
Option
Option
Option
Relapse/ Refractory

16. Patient Journey Planner
Option 
Option
Option
Option
Induction
Option
Option
Option 
Option
Consolidation
Option
Option
Option
Option
Transplant
Option
Option
Option
Option
Maintenance
Option
Option 
Option
Option
Relapse/ Refractory

17. Skin Cancer Patient Register
Beyond AML
Skin Cancer Patient Register
Prevention
Tenovus sun avoidance
British Skin Foundation Sun awareness
Screening
CDNK2a
MCR1+
Ocular melanomas
Others
Adjuvant treatment (high risk)
Alpha interferon
Immune checkpoint inhibitors (node +ve)
Immune checkpoint inhibitors (thick melanomas)
Oncogene targeted therapy
BRAF +
NRAS
c-KIT
NF-1
GNAQ
Immune checkpoint inhibitor
ipilimumab
pembrolizumab
novilumab
others
combos
Tumour vaccine studies
CARS
Melanoma Antigen Immunotherapy
Supportive Care/QoL

18. Conclusions
NCRI AML Working Group trials already use the Patient Journey Planner approach
Requires joined up information
Real time marker identification
Centralised IT to determine eligibility
Allows cross trial group collaboration
Different treatments at different stages of development at different points on the journey
Adaptable to other conditions
@CTRCardiffUni