1. NICE Decision Making Dr Katherine Payne North West Genetics Knowledge Park The University of Manchester katherine.payne@manchester.ac.uk

2. National Institute for Health and Clinical Excellence (NICE) Est. April 1999 ‘Health’ added April 2005 To provide guidance on the clinical and cost-effectiveness of new and existing health technologies in the NHS in England and Wales –medicines –medical devices –diagnostic techniques –surgical procedures –health promotion activities

3. Why do we need national guidance? NHS must be provided with a fixed budget → choices Health technology assessment, medicines evaluation and the NHS Local versus national decision-making –Medicines Management Committees –NICE Variation in decisions A centralised decision-making body

4. COX-2 selective NSAIDs: RCTs agent compared with placebo or traditional NSAIDs Outcomes used: endoscopic ulcers Patients with GI and CV risk excluded No trial > 6 months In practice: Used instead of PPIs, misoprostol or H 2 RAs Outcomes needed: symptomatic ulcers, GI bleeds, QoL, costs Patients with GI and CV risk Used for many years Medicines regulation in the UK MHRA EMEA Use quality, safety, efficacy data but no economic evidence

5. NICE guidance Technology appraisals new and existing medicines and treatments (94) Clinical guidelines specific diseases and conditions (42) Interventional procedures for diagnosis or treatment (from Feb 02) safety and efficacy of surgical procedures (145) Public health interventions and programmes Also …… Clinical Audit and Referral Advice for the NHS

6. Selecting technologies for appraisal Is the technology likely to result in a: –significant health benefit –relates to NHS clinical priority areas or government health-related policies –condition has significant disability, morbidity or mortality –significant impact on NHS resources (financial or other) –added value by issuing national guidance

7. Preparation of the ‘scope’ Consultees (patient/carers groups, healthcare professionals, manufacturers) input Commentators (manufacturers of comparator technology, research groups) input Prepare ‘assessment report’ (academic centre) Comment on assessment report (consultees/commentators) Produce evaluation report Overview of the NICE appraisal process (1)

8. Appraisal Committee meet 1: Evaluation report plus verbal evidence Appraisal Consultation Document (ACD) Appraisal Committee meet 2: Comments submitted on ACD Final Appraisal Determination (FAD) Consultees can appeal against FAD If no appeals: FAD forms basis of NICE guidance Start to end of appeal period: minimum 54 weeks Overview of the NICE appraisal process (2)

9. Timing of the NICE appraisal process Coronary heart disease – statins ScopeScope published Invited submissions 1 st AC Meet 2 nd AC Meet 3 rd AC Meet Expected completion Dec 03Apr 04Aug 04Mar 05May 05Jul 05Nov 05 ScopeScope published Invited submissions 1st AC Meet 2 nd AC Meet Expected completion Apr 05Jun 05Sep 05Mar 06May 06Oct 06 Inhaled insulin for types 1 and 2 diabetes

10. Timing of the NICE appraisal process (2) 2 nd AC Meet 3 rd AC Meet 4 th AC Meet Expected completion Jan 05Jun 05Dec 05tbc Alzheimer’s disease (review) – donepezil, rivastigimine, galantamine & memantine ScopeScope published Invited submissions 1st AC Meet Dec 03Jan 04Jun 04Oct 04

11. Transparency in decision-making Epidemiological: how many patients? Clinical evaluations (RCTs, meta-analyses) Economic evaluations Expert clinician and patient views Manufacturers submissions (commercial in confidence data) Evidence used at an Appraisal Committee Quality of evidence Strength of effect Risk of adverse events Evidence of patient value Availability of alternative treatment

12. Economic evaluations Provide evidence about ‘efficiency’ The comparative analysis of alternative courses of action in terms of both their costs and benefits. INPUTS Process of health care OUTPUTS Resources: staff drugs training etc Outcomes: effectiveness QALY/utility WTP Options: 1) Drug A 2) Drug B

13. The cost-effectiveness plane NE SWSE NW Increased cost Difference in cost = £A – £B Difference in QALYs = QALYs A – QALYs B ICER = difference in cost / difference in QALYs Decreased cost Increased QALYsDecreased QALYs Most NICE appraisals

14. Issues in NICE decision-making (1) Transparency in appraisal and evidence base The scope: individual medicines or class/groups? Evidence appropriate to the patient population Generalisability from setting to setting –Eg. glycoprotein 3b/2a inhibitors in heart disease Long-term follow-up data

15. Issues in NICE decision-making (2) Relevant end-points (QALYs) –Eg. Parent training for conduct disorders Level of uncertainty in the decision –Bias in data –Poor data –No data –Eg. risk-sharing and beta interferon NICE does not have a ‘cost per QALY’ threshold NICE and its value judgements –Scientific –Social