1. New medicines, new challenges: the SMC approach
Keynote Address:
New medicines, new challenges: the SMC approach
Anne Lee, Principal Pharmacist and Horizon Scanning, Scottish Medicines Consortium

2. New medicines, new challenges: the SMC approach
Anne Lee
Principal Pharmacist
Scottish Medicines Consortium
NPPG Conference
November 2009

4. Overview SMC – historical context A birds eye view of SMC
-new medicines assessment
(medicines for children)
-horizon scanning
Current challenges with new medicines for pharmacy

5. From EBM to cost effectiveness…….
UK Cochrane Centre established
First SIGN guideline published
1999 NICE established
First SMC advice issued

6. The solution: SMC in 2001

8. The Form of the Advice Accepted for use in NHS Scotland
Accepted for restricted use in NHS Scotland
Restricted by type of prescriber
Restricted by patient group
Not recommended for use in NHS Scotland
Detailed Advice Document (~ 8 pages)
- provides rationale for the advice
Resubmissions encouraged
- independent appeals process in place

9. SMC Assessment Process

10. Outcome of Assessments (n=458)
Accepted for Use – 30%
Accepted for Restricted Use – 33%
Not Recommended – 37% % 10

11. Obsessed by QALYs? Cost per QALY < £10K – 79% ‘yes’
Cost per QALY plays (appropriately?) a large role – but not the only consideration! 11

12. Stakeholders
Clinicians in the NHS – database of 450 experts advice from ~ 5 per medicine
Pharma Industry – product submission, clarification through assessment teams, SMC meeting if seek to resubmit, ABPI nominations on NDC and SMC, User Group Forum
Patients and public – 3 lay representatives on SMC, submissions from patient charities and organisations
NHS managers – 3 CEOs, DoFs on SMC

13. Current challenges with the assessment process
Challenges with the evidence
MA does not match evidence
Evidence not supportive of company’s requested positioning
Statistical significance / marginal benefit
Surrogate endpoints
‘Salami slicing’ of MA
Challenges with HTA - comparators
Indirect comparisons
Inappropriate comparators

14. SMC – key strengths
SMC applies a two stage process to decision making:
-NDC make recommendations on basis of evidence presented
- SMC have deliberative process, engage patient groups and apply pragmatism to develop final advice
Judgments made early in product life cycle
Consortium approach supports ‘buy in’

15. What has the SMC ever done for us?

16. SMC advice on medicines in children (2002 – 2007)
15 medicines assessed via full submission
(includes two resubmissions, 7 orphans)
- 3 Accepted
- 7 Accepted with restrictions
- 5 not recommended
8 abbreviated submissions (minimal dataset)
1 piece of NR advice due to non-submission

17. SMC & paediatrics
Impact of EU regulation (medicines in children) - SMC process working against the principle?
New product tracking – submissions requested (? Supporting data available)
Negative advice issued if no company submission (unhelpful)
Opportunity to assess more products via abbreviated process?

18. Could we introduce a new approach?
Abbreviated submission for paediatric licence extensions where medicine assessed as clinically and cost effective in adults
‘Modified abbreviated’ process proposed
Scoping exercise (of 15 previous full submissions only 4 would have qualified for abbreviated submission)
Advice from expert networks critical in setting up process (SNAPP, ScotMCN)

19. For medicines previously accepted by SMC for the relevant indication in adults (or products predating SMC) – abbreviated submission
Advice from clinicians in both networks is sought to inform the decision on whether CE in children expected to be similar to that in adults

20. Experience in the first year
8 abbreviated submissions accepted
3 full submissions for paediatric medicines
(stiripentol, caffeine, mecasermin)
Network advice invaluable
Some concern at NDC/SMC re no requirement for health economic case
Report to SMC on first year experience

22. Audit Scotland 2005

23. SMC horizon scanning
Support Health Board financial and service planning for new medicines
Reduces duplication of effort
Predicted NET budget impact for NHS Scotland (at yr 1 and yr 5)
Improve access to clinical & cost-effective new medicines

24. Estimated Budget Impact
Forward Look Content
Report
Total entries
(pre-launch
products in all
categories)
New
Medicines
indications/
formulations
Orphan
Products
Estimated Budget Impact H M L*
2005  47 31 16 26
12 (26%)
13 (28%)
22 (47%)
2006 36 24 12 14
9 (25%)
14 (24%)
13 (36%)
2007 29 21 8
1 (13%)
17 (58%)
11 (38%)
2008 22 13 9 15
13 (59%)
8 (36%)
2009 10
5 (20%)
7 (29%)
12 (50%)
 report covered 18 month period
* Orphan drugs are included despite low predicted impact

25. NHS board’s views on and use of Forward Look

26. “The NHS has better information for planning medicines budgets, but boards still identified gaps. The Scottish Medicines Consortium (SMC) has made good progress in providing boards with information to help them plan. It now produces annual reports that include an estimate of the budget impact of medicines that should become available in the coming year.”
Audit Scotland. Managing the use of medicines in hospitals: a follow-up review

27. New medicines: new challenges

28. New medicines on the horizon
Expect more…..
biopharmaceuticals / biosimilars
rare diseases - more orphan drugs
Advanced therapy medicinal products (ATMPs)
- gene therapy
- somatic cell therapy products
- tissue engineered products
EU Advanced Therapies Regulation
EMEA Committee on Advanced Therapies

29. Current challenges with new medicines: SMC
Pharmaceutical Price Regulation Scheme (PPRS) 2009
measures to promote innovation
Patient access schemes
Flexible pricing
UK-wide horizon scanning database
Metrics of uptake on new medicines

30. New medicines: challenges for pharmacy
Cost-effectiveness vs innovation
Disinvestment in medicines not cost-effective
Biotechnology products - safety
Advanced therapy medicinal products
Personalised medicine

31. Thank you!

32. SMC and NICE STA decisions
Both No 4
Both Yes
NICE No, SMC yes 3
NICE Yes, SMC no 6
TOTAL