Scottish Medicines Consortium -

Approach to Cancer Medicines

Dr Ken PatersonDr Ken Paterson

BOPA SymposiumBOPA Symposium

13 September 200713 September 2007

Scottish Medicines Consortium

Chairman: Professor David WebbChairman: Professor David Webb

Vice-Chairs:Vice-Chairs: Ms Angela Timoney, Ms Angela Timoney, Dr Ken PatersonDr Ken Paterson

Remit

Provide advice to NHS Boards and ADTCs Provide advice to NHS Boards and ADTCs on comparative and cost-effectiveness of:on comparative and cost-effectiveness of:

New MedicinesNew Medicines New Formulations of MedicinesNew Formulations of Medicines Major new indications for MedicinesMajor new indications for Medicines

80 products (approx) per annum80 products (approx) per annum

SMC Membership

Membership (30) Membership (30) - multi-disciplinary, - multi-disciplinary, geographically spreadgeographically spread

Physicians (1Physicians (1° and 2° care)° and 2° care) PharmacistsPharmacists Nurse, EconomistsNurse, Economists Board and Trust Executives, ABPIBoard and Trust Executives, ABPI Lay & Patient RepresentativesLay & Patient Representatives NHS QIS NHS QIS Full declarations of interestFull declarations of interest

New Drugs Committee

Membership: Total = 18Membership: Total = 18Physicians, Pharmacists,Physicians, Pharmacists,Health EconomistsHealth EconomistsNurse, Public Health ConsultantNurse, Public Health Consultant

Primarily an Evidence Review CommitteePrimarily an Evidence Review Committee

Chairman: Dr Ken Paterson (Glasgow)Chairman: Dr Ken Paterson (Glasgow)

SMC Advice to NHSScotland

3 Categories of advice 3 Categories of advice

Accepted for use in NHS ScotlandAccepted for use in NHS Scotland Accepted for restricted use in NHS ScotlandAccepted for restricted use in NHS Scotland Not recommended for use within NHS Not recommended for use within NHS

ScotlandScotland Some drugs may also be ‘unique!’Some drugs may also be ‘unique!’

The Aim of Product Assessments

Efficacy – does the drug have an effect?Efficacy – does the drug have an effect?

Effectiveness – does it work in normal Effectiveness – does it work in normal use?use?

Cost-effectiveness – how much bang for Cost-effectiveness – how much bang for the buck!the buck!

Submission Content

Standardised formStandardised form Summaries of efficacy/effectiveness and safetySummaries of efficacy/effectiveness and safety Detailed health economic caseDetailed health economic case

Cost-utility approach preferred (£ per QALY)Cost-utility approach preferred (£ per QALY) Budget impact for Scotland (or per 100,000)Budget impact for Scotland (or per 100,000) Full explanation of model assumptionsFull explanation of model assumptions

Linked to Scottish (or UK) data Linked to Scottish (or UK) data Full sensitivity analysisFull sensitivity analysis

Univariate Univariate ± probabilistic analyses± probabilistic analyses

Submission data

All referenced data to be includedAll referenced data to be included May include unpublished dataMay include unpublished data

...including ‘commercial in confidence’...including ‘commercial in confidence’ Economic data to be includedEconomic data to be included Supplementary data – SPC, draft protocols, etcSupplementary data – SPC, draft protocols, etc

Clinical Expert Panel

Important to inform the SMC processImportant to inform the SMC process Impact of diseaseImpact of disease Unmet therapeutic needUnmet therapeutic need Current therapeutic strategies in ScotlandCurrent therapeutic strategies in Scotland Test economic case assumptionsTest economic case assumptions

NOT asked “do you want this drug?”NOT asked “do you want this drug?” All interests declaredAll interests declared

Patient & Public Input

Patient & Public Involvement GroupPatient & Public Involvement Group Patient group submissions considered at Patient group submissions considered at

SMCSMC Only ~30% of medicines have a patient Only ~30% of medicines have a patient

group submissiongroup submission Can say things which pharma company Can say things which pharma company

cannot say!cannot say! Now actively seeking patient group Now actively seeking patient group

submissionssubmissions

Process timelines

SMC Publishes a dvice on w w w(4 w eeks a dvice to N H SScotla nd)

w w w .scottishm edicines.org.uk

SMC Advises N H SScotla ndN H S B oa rds & AD T C s& Applica nt C om pa ny

N D C R eport to SMCfirst T uesda y/m onth

R eport to N D Cla st T uesda y/m onth

SMC Assessm ent3/4 W eeks

Subm ission to SMC

10-12 weeks

4 weeks

2002 - 2007

382 submissions considered382 submissions considered 2002 – 292002 – 29 2003 – 622003 – 62 2004 – 742004 – 74 2005 – 872005 – 87 2006 – 130 (111)2006 – 130 (111)

~20% are ‘abbreviated’ subs~20% are ‘abbreviated’ subs Rising proportion of re-submissionsRising proportion of re-submissions

Outcome of Assessments

Accepted for Use – 34%Accepted for Use – 34% Accepted for Restricted Use – 36%Accepted for Restricted Use – 36% Not Recommended – 30%Not Recommended – 30% No real evidence of change over timeNo real evidence of change over time

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45

2002 2003 2004 2005 2006

AcceptRestrictNo

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Oncology Assessments

Fewer RCTs per drug (median 1 v 2)Fewer RCTs per drug (median 1 v 2) Longer follow-up (52 wks v 12 wks)Longer follow-up (52 wks v 12 wks) Acceptance rate - 67%Acceptance rate - 67%

About half with some restriction, usually to About half with some restriction, usually to specialist usespecialist use

Higher cost per QALY (£15K v £8.5K)Higher cost per QALY (£15K v £8.5K)

Driven by Budget Impact?

AcceptAccept £413K£413K (120-760K)(120-760K) RestrictRestrict £581K£581K (299-863K)(299-863K) NoNo £743K£743K (366-1100K)(366-1100K)

Large overlap suggests budget impact Large overlap suggests budget impact is not driving SMC decision-makingis not driving SMC decision-making

Obsessed by QALYs?

Cost per QALY < £20KCost per QALY < £20K ……20% not recommended!20% not recommended!

Cost per QALY £20-30KCost per QALY £20-30K ……58% not recommended58% not recommended

Cost per QALY plays (appropriately?) aCost per QALY plays (appropriately?) a large large role – but not the only consideration!role – but not the only consideration!

Special Cancer Issues - 1

Often scanty phase 3 clinical dataOften scanty phase 3 clinical data Complex regimens with poly-pharmacy make Complex regimens with poly-pharmacy make

comparators hard to definecomparators hard to define RCTs often use comparators different from RCTs often use comparators different from

current Scottish practicecurrent Scottish practice May require indirect comparisonMay require indirect comparison

Survival benefits often unclearSurvival benefits often unclear Overall v ‘progression-free’ survivalOverall v ‘progression-free’ survival Extrapolation not clear-cutExtrapolation not clear-cut

Extrapolation Possibilities

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1) Only benefit observed in RCT

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2) ‘Frozen’ at end of RCT

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3) Continuing divergence

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4) Limited divergence then ‘frozen’

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5) Limited divergence then tapering

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Special Cancer Issues - 2

Quality of life assessment difficultQuality of life assessment difficult Impact of adverse events a problemImpact of adverse events a problem ? revaluation of QoL near life’s end? revaluation of QoL near life’s end ? special benefit with low expectancy? special benefit with low expectancy

Increased niching by indicationIncreased niching by indication ……more (ultra-)orphan drugsmore (ultra-)orphan drugs

……with expectations of “special case”with expectations of “special case” Rule of Rescue - a rule??Rule of Rescue - a rule??

Quality of Life

Are the impacts of adverse events limited to Are the impacts of adverse events limited to when they occur?when they occur?

With 3 months to live, if you say your QoL is With 3 months to live, if you say your QoL is 90%, is that true?90%, is that true? Are time-trade off/standard gamble useful?Are time-trade off/standard gamble useful?

Is 3 months extra life worth more if you’ve Is 3 months extra life worth more if you’ve had the diagnosis for 3 months rather than 5 had the diagnosis for 3 months rather than 5 years?years? ? discriminates against certain cancers?? discriminates against certain cancers?

Early Technology Appraisal of Oncology Drugs

……is possibleis possible ……can be done within similar parameters to can be done within similar parameters to

other drugs and technologiesother drugs and technologies ……allows real breakthroughs even at allows real breakthroughs even at

considerable costconsiderable cost ……does not reward small incremental change does not reward small incremental change

at substantial costat substantial cost ……can avoid ‘decision blight’ and meet the can avoid ‘decision blight’ and meet the

timelines of specialists and patientstimelines of specialists and patients

To consider…

Is this whole process ethical?Is this whole process ethical? Would it be ethical NOT to do it?Would it be ethical NOT to do it?

What should the NHS pay for?What should the NHS pay for? Life, QoL, feeling better, carer time?Life, QoL, feeling better, carer time?

How should specialists react…?How should specialists react…? Is money not spent on drugs to manage Is money not spent on drugs to manage

cancercancer always money wasted?! always money wasted?! What should the threshold £ per QALY be?What should the threshold £ per QALY be? How should we handle orphan drugs?How should we handle orphan drugs?

Scottish Medicines Consortium

www.scottishmedicines.org.uk