Sarah Karlsberg Schaffer, Martina Garau, Phill O’Neill and Simona Bianchi ISPOR 19th Annual European Congress Vienna - 1 November 2016

1. OHE Consulting Ltd: Results reported here are preliminary, and subject to
change as a result of journal peer review. The content of these slides is not
to be quoted or used without the consent of the authors.
Modelling the Impact of Reform to the
Cancer Drugs Fund
Sarah Karlsberg Schaffer, Martina Garau, Phill O’Neill
and Simona Bianchi
ISPOR 19th Annual European Congress
Vienna • 1 November 2016
Work funded by the Association of the British Pharmaceutical Industry (ABPI)

2. Modelling the Impact of Reform to the Cancer Drugs Fund
01/11/2016 2
Background: HTA in England
and Wales
• NICE assesses technologies against cost per
QALY threshold of £20-30k/QALY
• Additional threshold = £50k for technologies
that meet end-of-life (EoL) criteria
• Life expectancy without treatment ≤ 24 months
• Life extension from treatment ≥ 3 months
• ‘Small’ patient population (<7000 patients) [now
changed]

3. 2010
2011
2012
2013
2014
2015
Due to end in 2014, when
system of Value Based Pricing
(VBP) was to begin
CDF timeline
Consultation
document = basis of
OHE analysis

4. Modelling the Impact of Reform to the Cancer Drugs Fund
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Summary of changes to
HTA/reimbursement policy
• All new cancer therapies to be appraised by NICE
• NICE can make 1 of 3 possible recommendations
1. Recommended for routine commissioning – ‘yes’
2. Not recommended for routine commissioning – ‘no’
3. Recommended for use within CDF (new)
– For when NICE considers plausible potential for drug to
satisfy criteria for routine commissioning but when there
is significant remaining clinical uncertainty
• ‘Patient population’ criterion dropped from EoL
criteria

5. Modelling the Impact of Reform to the Cancer Drugs Fund
01/11/2016 5
Details of changes to CDF
funding
• Drug recommended for CDF will receive interim
funding (2 years) while the uncertainty is
resolved
• At end of managed access period  drug
reappraised by NICE
• From March 31st 2016: transition period
• All drugs on ‘old’ CDF list (‘transition medicines’) are to
be (re)appraised by NICE over 18 month period

6. Modelling the Impact of Reform to the Cancer Drugs Fund
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Study aim and objectives
• Aim: understand the implications of the new CDF on those
technologies transitioning
• Objective: estimate number of transition
medicines/indications that will be recommended (or not)
for use within new CDF
• Note that the CDF consultation document contained little
detail about definition of uncertainty and emphasised
flexibility:
• Proposed: ‘… amendments to emphasise the discretion for
NICE Appraisal Committee to interpret the uncertainty criteria’
• Disclaimer: analysis based on CDF proposal (consultation
document) – does not reflect most recent decisions [discuss this
later]

7. Methods
• Interpretation of ‘flexibility’ for inclusion on CDF = ICER (point
estimate or range) below or including ‘acceptable’ threshold
point (£50k/QALY for non-EoL; £70k/QALY for EoL)
• Thresholds higher than those in NICE Methods Guide to reflect
possibility for managed entry agreements between DH and
manufacturers
• No available ICER  assumed funded on CDF = “optimistic”
EoL ICER Funding decision
x ≤£30k Baseline commissioning
x £30k≤ ICER ≤£50k New CDF
x >£50k Not funded
 ≤£50k Baseline commissioning
 £50k≤ICER≤£70k New CDF
 >£70k Not funded
Decision rules
used in analysis

8. Modelling the Impact of Reform to the Cancer Drugs Fund
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Data
• NICE technology appraisals (TAs), published
October 2007-December 2015
• NHS England CDF List version 6
• Combined with:
• NICE pipeline information
• ICERs from Scottish Medicines Consortium (SMC)
assessments (where NICE ICERs not available)

9. Results
• Of 48 transition CDF medicines/indications:
• 13 (27%) predicted to be funded by new CDF
• 3 (6%) likely to receive positive NICE appraisal
• 32 (67%) judged to fall into ‘not recommended’ category
Of those predicted to be not
recommended:
High ICER beyond
plausible range of
acceptability
Not reviewed by NICE
but high SMC ICER
Off-label
Suspended or
terminated NICE TA

10. Medicines/indications predicted to
be funded by new CDF (n=13/48)
Molecule Indication NICE TA
ICER
(£/QALY)
Meets
EoL?
Bevacizumab
Persistent, recurrent or metastatic
carcinoma of the cervix
No TA
Bevacizumab
Advanced and/or metastatic renal cell
carcinoma
178a > 53,800
Yes (NEW
EoL)
Brentuximab
vedotin
Relapsed or refractory systemic
anaplastic large cell lymphoma
No TA
Clofarabine
Acute lymphoblastic leukaemia in
paediatric patients
No TA
Ibrutinib Chronic lymphocytic leukaemia
Pipeline
(ID749)
Ibrutinib
Relapsed or refractory mantle cell
lymphoma
Pipeline
(ID753)
Idelalisib Chronic lymphocytic leukaemia 359a No
Nelarabine
T-cell acute lymphoblastic leukaemia
and T-cell lymphoblastic lymphoma
No TA
Ponatinib
Philadelphia chromosome positive acute
lymphoblastic leukaemia
No TA
Radium 223 Castration-resistant prostate cancer
Pipeline
(ID576)
>50,000 Yes
Ruxolitinib Chronic idiopathic myelofibrosis 289b 45,000 No
Sorafenib
Progressive, locally advanced or
metastatic, differentiated thyroid
carcinoma
No TA
Temsirolimus Advanced renal cell carcinoma 178e No
Only 1 subgroup
affected by
changing EoL
criteria
No ICER available for
10/13  optimistic

11. Modelling the Impact of Reform to the Cancer Drugs Fund
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Concluding thoughts
• Key conclusion: CDF reform is likely to reduce use
of new cancer medicines in England and Wales (at
least temporarily)
• Potential improvements to efficiency/equity
– Reduced opportunity cost of funding cost-ineffective
medicines
– Cancer treated less like a ‘special’ disease
• Potential improvements to manufacturer incentives
– Increased incentives to provide satisfactory clinical/cost-
effectiveness evidence for HTA
• NICE TA pipeline full of cancer drugs…

12. Modelling the Impact of Reform to the Cancer Drugs Fund
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Postscript
• At time of writing*, of 48 transition indications:
• 8 indications received positive (or ‘optimised’) appraisal 
baseline commissioning (bosutinib was first)
• 11 indications currently ‘not recommended’
• 18 indications scheduled for NICE appraisal
• 11 indications not yet scheduled
• 0 indications funded by new CDF
• Of other indications:
• 1 indication funded by new CDF (Osimertinib for NSCLC)
• 2 indications ‘invited’ to submit to CDF at ACD/FAD stage
• Further NICE/NHS England consultations
• HTA is an evolving space…
*Based on separate OHE analysis

13. Modelling the Impact of Reform to the Cancer Drugs Fund
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Thank you
To enquire about additional information and analyses, please contact
Sarah Britton (née Karlsberg Schaffer) – sbritton@ohe.org
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