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Presentation 15 January 2015 1

• 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 2

• 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 4 th 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 3

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 4

• 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 5

• 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 6

• 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 7

Average Cost v Score
140000
120000
100000
80000
60000
40000
20000
0
-1 2 5 8
Score
10 13 16

• 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 9

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 10

• 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|
11

• 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 12

• 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 13

• 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
14
Presentation 15 January 2015

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 15

• 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 16

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

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 18

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

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 20

• 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
21

• 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 22

• 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 23

• 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 24