1. Life beyond the Cancer Drug Fund: an update on the CDF and a view on the sustainable commissioning of cancer drugs in England
Professor Peter Clark
Medical Oncologist
Chair of the NHS England Chemotherapy Reference Group and of the Cancer Drug Fund
Presentation 15 January 2015

2. Background to English healthcare
The state directs almost all of health service supply (private health insurance held by 9%); total NHS England budget £97bn in 2014/15
Commissioning of systemic therapy (‘chemotherapy’ but not hormone therapies prescribed by GPs for breast cancer and prostate cancer) in NHS England is central and in Specialised Commissioning (latter’s total budget is £13bn)
Spend on chemotherapy in NHS England is £1.7bn
Drugs £1450m; tariff £250m
Of £1450m, generic £175m; NICE approved on patent £875m; CDF £390m
Baseline commissioning thus £1050m for drugs
Reimbursement via baseline: via NICE approval (legally binding for funding) or NHS England policy (has to compete with all other spec comm cost pressures)
Reimbursement via Cancer Drugs Fund
Presentation 15 January 2015

3. Background to NICE
Drug regulatory framework has 3 common hurdles: safety, quality and efficacy
NICE: clinical effectiveness – how well does something work in comparison with what we already use? NICE: cost effectiveness – how much more life or quality of life do we get for the extra money we have to pay?
NICE thus added cost effectiveness as the 4th hurdle for reimbursement
A positive NICE appraisal has to be funded by the NHS: as the budget is fixed, some other planned improvement has to be axed or delayed
A negative NICE appraisal is rarely routinely funded
Appraisals: yes in 75%, no in 25%; cancer drugs: yes in 60%, no in 40% but recently yes 30%, no 70% (many reasons: is one the presence of the CDF?)
New cancer drugs: prices and costs rising and expensive vs other diseases
Binary outcome of appraisal: cost reduction mechanisms (Patient Access Schemes) determined by and offered by most pharmaceutical companies
Presentation15 January 2015

4. NICE technology appraisal Impact on baseline commissioning (BC):
Commissioning of new cancer drug/indication:
Timeline pre Cancer Drugs Fund
Local decision making
NICE technology appraisal
Evidence of benefit
Drug licensing
Impact on baseline commissioning (BC):
NICE yes - into BC
NICE no - rarely funded
Presentation to NHSE | 15 January 2015

5. Cancer Drug Fund:
From 2007 onwards, increasing patient, patient group, clinical, pharma and media pressure re growing issues of NICE blight and NICE non-approval
CDF a Conservative Party election pledge and coalition agreement commitment
Originally CDF was labelled as a ‘bridge to value’ and a bridge to Value Based Pricing ie was an interim manoeuvre prior to a long term solution of solving access
Clinically driven implementation: allocation £200m/year until 2013/14; first overspend in 2013/14 and was £30m.
Activity and expenditure rising esp in 2014/15; budget increased to £280m
Potential increased overspend prevented by some NICE approvals and thus transfer of funding of these drugs from CDF into baseline
Until now, entry into CDF on clinical benefit using simple scoring system evaluating impact on PFS, OS, QOL, toxicity & unmet need plus use of wisdom & judgement
Since NHSE took over CDF in 2013, 65 cohort applications: 26 approved (40%)
CDF extended by government to April 2016
Presentation 15 January 2015

6. Late 2014 Cancer Drugs Fund: the drugs
84 different approved drug indications: 4 different types of drugs
NICE appraisal negatives: clinically effective but not cost effective despite common use of Patient Access Schemes – 37 indications, 62% applications
Pre-NICE outcome: covers variable gap again between licensing & NICE guidance – 14 indications, 22% applications
Drugs below NICE’s radar (rarer cancers) – 26 indications, 12% applications
Off label drugs – 7 indications, 3% applications
Thus 85% of CDF drug applications in NICE’s remit, 15% beyond
Open prioritisation meetings: clinicians, pharma, patient groups, CDF panel
NB Cancer Drug Fund list on NHS England website: approved drugs, not approved drugs, plus all the prioritisation tool scores of clinical benefit and decision summaries for drugs considered since 1 April 2013
Currently 2000 new applications per month for cohort applications
CDF: access, speed of access, simple application process, open
Presentation 15 January 2015

7. Current Cancer Drugs Fund: the drugs
Increasing access (23000 in 14/15) and increasing expenditure (£390m in 14/15)
Some drugs offer substantial clinical benefit
Some drugs offer very modest clinical benefit
Some drugs cost <£10K per patient, some >£50k per patient, some >£100k
What about value? It is very variable (see slide)
Greatest value: those drugs which become NICE ‘yes’ and hence CDF offers bridging between licensing and baseline commissioning
Greatest value: rare cancers or rare indications for more common cancers – often not commissioned or variably so
Greatest value: off label use for rare cancers NB growing with molecularly targeted agents
Poorest value: expensive drugs offering low clinical benefit
Poorest value: drugs offering modest/greater benefit but very highly priced
CDF process and decisions: clinicians and patient representatives central
Presentation 15 January 2015

8. Cost per patient divided by CDF clinical score: one estimation of value

9. Current commissioning of cancer drugs
NICE continues to determine clinical and cost effectiveness but its role in guiding the NHS as to value of cancer drugs has been affected: outcomes of appraisals have been affected by drug pricing but also by necessary assumptions in economic modelling because of: short follow-up (survival, resource use, impact on patient pathway), cross-over design etc
In ever greater number of appraisals, there are now more assumptions in the economic modelling, thus uncertainty is greater….and more negative appraisals
C has resulted in less incentive to offer Patient Access Schemes to NICE; some pharma still strive hard for NICE approval, others not
NICE/CDF assess evidence from clinical trial populations of patients more likely to over estimate gains to patients in every day practice
There is no feedback loop of outcomes in the NHS back to NICE appraisal
The CDF is overspent at a time of great financial pressure in the CDF
Presentation 15 January 2015

10. Timeline at present: the Cancer Drugs Fund
CDF funding
CDF depending on NICE decision
NICE technology appraisal
Evidence of benefit
Drug licensing
Impact on baseline commissioning or CDF
Presentation 15 January 2015

11. Commissioning of cancer drugs in England
Commissioning is central ie one policy for all England
Cost of cancer drugs is by far the single greatest cost pressure in Specialised Commissioning
Power of a positive NICE appraisal: must be funded by NHS England
Baseline commissioning: the only safe place for commissioning – either via NICE approval or NHSE policy (but latter is a very competitive process within specialised commissioning)
Political creation of the Cancer Drugs Fund
CDF: stacking drug indications with no long term decisions as to availability or not
Presentation 15 January 2015|

12. Current commissioning of cancer drugs
Undermining of NICE guidance with less incentive to gain NICE approval
NICE negative decisions: drug cost and uncertainty as to benefit
Overspent CDF: does not offer a long term solution
Great financial pressure in NHS England
Pharma offered solution which kept all indications in CDF and linked level of reimbursement to degree of clinical benefit: rejected
ABPI were offered a different mechanism to retain all indications in CDF and link level of reimbursement to degree of clinical benefit: rejected
Maximal emphasis on maximising value in NHS England for healthcare
1: NHS England changed its CDF prioritisation procedures to combine scores of clinical benefit and the median costs of treatment into overall scores of value and prioritise accordingly to keep the CDF within budget
2: NHS England wishes to produce a sustainable way of commissioning the best value cancer drugs
Presentation 15 January 2015

13. New CDF prioritisation procedure
Each drug indication scored as before for impact on median progression-free survival, median overall survival, quality of life, toxicity and CDF definition of unmet need (is it the only systemic therapy for that disease?)
Process is open to patient groups, pharma, clinicians
CDF panel assesses clinical impact and scores accordingly
There is a confidential scoring system for assessing the median drug cost which may reflect list price or a price determined by the manufacturer
CDF panel combines clinical and cost score and decides whether to be retained in the CDF for existing indication or enter the CDF for new indication
NHS England meets with the manufacturers and informs them of the overall scores. For indications of sufficient clinical benefit to stay in the CDF but with cost scores that bring the overall scores below the overall threshold, the manufacturers have the option if they so choose to reduce their median costs
NHS England publishes the clinical scores and reasoning as to inclusion and exclusion on its website but any cost discounts remain confidential
Presentation 15 January 2015

14. New CDF prioritisation procedure outcomes
14/15 expenditure on November list £390m and £420m in 15/16
84 separate drug indications in the CDF in November 14 and lowest scoring 45 indications assessed in this recent prioritisation process
Current best evidence assessed by CDF panel
Some new very effective treatments being licensed
Of 45 indications, 25 are due for removal in March 15, 18 have been retained and 2 require further consideration
Of 25 to be removed, 17 are in effect NICE negatives; 8 never assessed by NICE
Of 25 to be removed, 92% of applications are for NICE negative indications
Of 25 to be removed: breast cancer, bowel cancer, lung cancer, prostate cancer, sarcoma, meloma, lymphoma
Constructive engagement in whole process by some manufacturers
Presentation 15 January 2015

15. Commissioning of new cancer drug/indication:
Timeline at present with the Cancer Drugs Fund
Prioritisation by Cancer Drugs Fund
CDF funding if prioritised
Continued funding by CDF if NICE not approved. Re-evaluation process incorporating cost from November 2014 and potential removal from CDF
NICE technology appraisal
Evidence of benefit
Drug licensing
Impact on baseline commissioning if NICE approved
Presentation to NHSE | 15 January 2015

16. Commissioning of cancer drugs
Baseline commissioning. The NHS must pay for NICE positive appraisals, within a fixed allocation funding and is at a cost of some other commissioned service. Most pharma still want NICE approval for cancer drugs but now have market access route if negative. NICE is here to stay but no value-based pricing. Baseline commissioning also possible via NHS England policy but cancer drug policy has to compete with all other policy developments in specialised commissioning
Cancer Drugs Fund - increasing new drugs, some exciting drugs, ever increasing drug costs to treat patients, £340m allocated for 2015/16 but permanent decisions re commissioning are required
New CDF working to remove drugs of lowest clinical benefit but also introduce assessment of cost into prioritisation process alongside clinical benefit
Longer term solution is for the fund to be used for ‘Commissioning through Evaluation’ to provide evidence to both NHS and NICE of mature ‘real life’ benefits for drugs that NHSE would like to see in baseline commissioning whether going to NICE or not

17. Commissioning of new drug/indication in proposed CDF-CtE: cancer drug referred to NICE & positive CDF prioritisation
Positive prioritisation by Cancer Drugs Fund & NICE
If mature outcome data will not change NICE re-appraisal: removed from the CDF
Cancer Drugs Fund
Cancer Drugs Fund
funding
If mature outcome data might change NICE re-appraisal, drug into CDF Commissioning through Evaluation
Continued funding by CDF
Removed from the CDF
If no
If no
NICE technology appraisal
NICE TA review
NICE
Timeline
If yes
If yes
Clinical trial results on 1o end points
Drug licensing
Baseline commissioning
Baseline commissioning
Baseline

18. Commissioning of new drug/indication in proposed CDF-CtE: cancer drug referred to NICE & negative CDF prioritisation
No prioritisation by CDF & NICE
No CDF funding as not prioritised
NICE technology appraisal
Evidence of benefit
Drug licensing
Impact on baseline commissioning only if NICE approved
Presentation to NHSE | 15 January 2015

19. Commissioning of new drug/indication in proposed CDF-CtE: cancer drug not referred to NICE & positive CDF prioritisation
Cancer Drug Fund
Positive prioritisation by Cancer Drugs Fund
CDF Commissioning through Evaluation
Evaluation by CDF for baseline commissioning
Removed from CDF
If no
Timeline
Clinical trial results on 1o end points
Drug licensing
If yes
Baseline commissioning
Baseline

20. Commissioning of new drug/indication in proposed CDF-CtE: cancer drug not referred to NICE & negative CDF prioritisation
No prioritisation by Cancer Drug Fund
No CDF funding as not prioritised
Evidence of benefit
Drug licensing
Presentation to NHSE | 15 January 2015

21. The Cancer Drug Fund and Commissioning through Evaluation
The process: the opportunity for the more highly valued cancer drugs assessed by the CDF (whether in NICE appraisal process or not to be appraised) to be audited for key outcomes and then be re-assessed: outcomes could be as predicted from clinical trials, better or worse (NB survival, utilities, resource use, effect on the patient pathway)
Then options: return to NICE with mature data and the opportunity for a new Patient Access Scheme if necessary and then continued funding depends on NICE approval or not; for non-NICE drugs, assess outcomes and either into baseline commissioning or out of the CDF
The consequence: NHS England has to fund the baseline commissioning of those effective drugs which do not to go to NICE ie those for rare cancers
The NHS gains key outcome information to guide assessment of value for consideration of baseline commissioning but which also feeds back into NICE appraisals and guidelines: a huge contribution to England and the world

22. The Cancer Drug Fund: the upside
Increased access to cancer drugs for many patients
Best value for drugs which subsequently gain NICE approval
Best value for rarer cancers including off label indications
Prioritisation process, though very simple, works well in practice for drugs with large and small evidence bases
Open and transparent process: run by clinicians and lay representatives; engagement with clinicians, patients, pharma
Quick decisions and rapid release of rationales in decision summaries on NHSE website
Presentation 15 January 2015

23. The Cancer Drug Fund: the downside
Initial regional implementation: variation in process, variation in cohort policies, pressure to spend allocation
As a consequence, some drugs in national CDF approved list which included drugs of poor clinical value
Undermined impact and reputation of NICE in cancer drugs
Its planned successor, Value Based Pricing, too ambitious in terms of time scale and implementation
CDF has reduced the pressure on drug pricing in NICE appraisals
Only information on which to assess value comes from clinical trials
A political creation and one that has not been integrated into the longer term sustainability of chemotherapy commissioning
Presentation 15 January 2015

24. The Cancer Drug Fund and beyond
Economic hardship generally: for individuals, for governments
Ever greater pressure on healthcare budgets
Ever greater realisation of need for greatest value in healthcare
Relentless rise in pricing and costs of cancer drugs
More patients, more lines of treatment
The NHS cannot afford everything in cancer let alone other diseases
Re-prioritisation of the drugs on the CDF list and removal was inevitable
NHS focussing much more on outcomes and value as it looks for savings
By providing funding for CDF, there can be use of this money to fund a sustainable process which helps the more effective cancer drugs that have uncertainty as to longer term outcomes seek the safety of baseline commissioning via NICE or by NHS England policy
Presentation 15 January 2015