The Scottish Medicines Consortium (SMC)

- assessment of relative effectiveness and cost-effectiveness

Jan Jones

NDC Chairman

Apr 2009

Overview

• Scottish healthcare system• SMC assessment process

– relative effectiveness• SMC acceptance rate• Challenges faced• Key factors for success?

Scotland

Healthcare system• National Health Service

– funded from taxation• Branded drug prices set by the Pharmaceutical

Price Regulation Scheme (PPRS) • Full cost of medicines reimbursed by the NHS• Value for money assessed by the SMC• Health Boards issue local recommendations for use

What is the SMC?• A consortium of Area Drug and Therapeutics

Committees (ADTCs) from NHS Boards in Scotland– ownership – common purpose

• to reduce duplication• to increase standardisation

– end ‘postcode prescribing’• Established October 2001• First guidance issued May 2002 (imatinib for CML)• Issued 626 recommendations to date (Apr 09)

• To make judgements on relative clinical effectiveness and cost-effectiveness

• Provide advice to NHS Boards and ADTCs on:– all new medicines (NCEs)– new formulations of medicines– major new indications for medicines– 80-100 products per annum

• Provide advice as close to product launch as possible (within 3-6 months)

SMC remit

• SMC – multidisciplinary (n = 30)– physicians, pharmacists, nurse, health economist– NHS executives/finance managers– pharmaceutical industry nominees (ABPI)– public partners (n=3)– lawyer, Scottish Government representatives

• NDC - clinical/scientific (n = 18)– physicians, pharmacists, nurse, health

economists, statistician, academics, industry nominees

– Pharmacy Assessment Team – Health Economics Team

Membership

Assessment process SMC Assessment Process

Scottish Medicines Consortium

Submission of new product assessment form

Economic Assessors

Assessment review

Assessment team

New Drugs Committee

Final SMC detailed advice document

Applicant company

Patient interest group submission

Scottish Medicines Consortium NDC detailed advice

Assessment & draft detailed advice document

Clinical Assessors

Company comments to SMC

NHS Boards

Area Drug & Therapeutic Committees

Applicant Company

Advice made public

8 weeks

6 weeks: NDC – last Tues/month; SMC

4 weeks

Competitor Company

Appeals process

• Resubmission – new clinical data– new economic data– repositioning of product

– high resubmission rate

Clinical effectiveness• Meaningful clinical benefit in the real world

compared to existing practice– issues:

• study design• selection of appropriate comparator• methodology of indirect comparison

• If clinical effectiveness is unclear then cost-effectiveness is uncertain

Study design• Translating efficacy into effectiveness requires:

– robust trial methodology• blinding, randomisation, study power,

missing data– clinically relevant outcomes

• mortality, morbidity, quality-of-life, length of follow-up

– applicability to local clinical practice• demographics, clinical management

strategy eg comparator, positioning

• Licensing authorities dictate design of studies

Selection of comparator• Critical to the economic evaluation

– ratio of additional costs to additional benefits relative to current practice

• Interested in the treatment most likely to be replaced in Scottish practice

• Potential difficulties in selecting comparators:– current practice inconsistent– comparator unlicensed for indication– comparator not best practice

• 12% SMC submissions used inappropriate comparator

Indirect comparisons• Naïve (unadjusted) or adjusted (network meta-

analysis)– naïve comparisons

• no stronger than observational data• results unpredictable

– but sometimes the best comparison we have…

– adjusted comparisons• combine trials with common treatment arm• preferable to naïve comparisons• becoming increasingly complex

– transparency becomes as issue

• 34% SMC submissions used indirect comparison

SMC submission types• Full submission

– new chemical entities– new indications

• Abbreviated submission– minimal cost implications

• Non-submission– absence of a submission

Outcome of assessments – all submissions (to Dec 2008 n=597)

• Accepted for use – 30%• Accepted for restricted use – 32%

• sub-group of patients• prescriber type

• Not recommended – 38%

• Overall acceptance rate for all new medicines (full submissions) – 72%

Outcome of assessments – all submissions (to Dec 2008)

0%

5%

10%

15%

20%

25%

30%

35%

40%

45%

50%

2002 2003 2004 2005 2006 2007 2008

Accepted

Restricted

Notrecommended

Why do we say no?

• 2% due to clinical reasons alone• 51% due to economic reasons alone• 47% due to both clinical and economic

reasons– value for money not demonstrated– justification of cost in relation to health

benefits not sufficient

Thresholds for assessing value• Benefits measured in QALYs

– allows comparison across therapeutic areas• Similar approach to NICE (UK)

– under £20k/QALY (€22-€30k/QALY)– £20-£30k/QALY– over £30k/QALY

– level of certainty is important– not just cost/QALY point estimate

Acceptance rate by cost per QALY

0%

10%

20%

30%

40%

50%

60%

70%

80%

<£10k £10k - £20k £20k - 30K >£30k

£/QALY

% a

ccep

ted

Challenges?• Uncertainty caused by study design

– relatively few controlled studies versus current practice

– use of proxy outcomes – short follow-up in clinical studies– lack of quality-of-life measurement in clinical

studies

Challenges?• Orphan medicines

– limited clinical data– processed the same as other medicines– ‘modifiers’

• life threatening disease• substantial increase in life expectancy or

quality of life• reverse rather than stabilise a condition• provide a bridge to a definitive therapy

Challenges?

• Non submissions– small market?– high cost product?

• patient access schemes• work in progress

Key factors for success?• Local ADTC ownership essential

– implementation of decisions• Engagement with key stakeholders

– Area Drug & Therapeutics Committees– clinicians– industry– patients

• Transparent robust process• Credibility• Drive, enthusiasm and commitment

– shared purpose

www.scottishmedicines.org