1. Current Challenges and Future Developments in HTA in the UK Frances Macdonald, 23 rd September 2008 (A personal, Industry View)

2. Agenda Office of Health Economics (OHE) review of SMC Decisions : 2002 - 2008 Pharmaceutical Price Regulation Scheme

3. SMC Decisions – OHE Project AIMs Overview of the trend in SMC decisions, over time In-depth analysis of the restricted decisions (normally counted as accepted)

4. All SMC submissions by submission type* * ‘2008’ includes only the fist six months (January-June)

5. Count and annual share of SMC decisions ( excluding abbreviated submissions)

6. Count and annual share of SMC decisions, (excluding abbreviated and non-submissions)

7. Counting ‘Yes-equivalent’ as yes

8. Conclusions Excl. abbreviated submissions, the rate of ‘Not recommended’ (NR) increased from 26- 38% (2002-2005) to approx 50% in 2006-7 Explanation? Excl. non-submission, the proportion of NR decisions stabilises and appears to be decreasing from the 2006 peak The proportion of non-submissions has grown, from 8% of decisions (excl. Abbrev. Subs) in 2006 to 29% in the first half of 2008. If the ‘yes-equivalent’ decisions are counted as ‘yes’ decisions, the overall trend for ‘acceptance’ has not changed significantly over recent years, averaging 37% of all decisions during the period 2005 to H1 2008. (excl. abbreviated submissions and non-submissions) Further work is needed to fully understand the trends eg Analysis of resubmissions Implications of incr. restricted submissions/approvals

9. PPRS- a significant factor now and future Pharmaceutical Price Regulation Scheme Covers all 4 Nations, to a large extent Currently under renegotiation, to be effective from January 2009

10. Principle Objectives of the PPRS Government and Industry agree on the following aims: Deliver value for money Encourage and Reward Innovation Assist the Uptake of new Medicines Provide Stability, sustainability and predictability Details still under discussion SMC has a role in 3, possibly 4, of these aims Ultimate aims – to ensure availability of cost-effective medicines to patients - to supportive innovation, uptake and competition Price cut on medicines likely from January 2009

11. Assessing ‘Value’ in HTA Health Select Committee recommended that NICE take into account a wider definition of ‘value’. What does this mean, and how can it be measured systematically? Cost per QALY – doesn’t address everything What are societal preferences on ‘value’? Rarity? Severity of disease?…. How to assess the value of long-term benefits to the NHS? Value of Innovation, where this adds demonstrable benefits? How broad should the perspective be eg NHS costs only or carer costs? What would happen to the ‘threshold’ if any such changes made?

12. Uptake of Innovation Innovation is only sustainable if there is uptake HTA is only of benefit if CE medicines are subsequently available, equitably & in good time So……… England – DH is investigating options to ensure HTA recommendations are implemented, and access is faster – to be written into new PPRS Scotland – the Evaluation project is already underway Some of the challenges: Ascertaining when variation is acceptable, and when not Quality of the data sources, although perfection not required

13. Supporting Uptake Single UK horizon scanning tool by end 2009 Will support consistent forward planning and budget planning Market Access Schemes are being discussed for potential inclusion within the new PPRS This aspect of the PPRS is relevant to England and Wales…but…. Scottish Government has already expressed interest in evaluating such options Ideal opportunity for Scotland to embrace such schemes for the benefit of Scottish patients

14. Early Assessment of new Medicines is a Challenge The SMC assesses all new products and indications close to launch, and NICE is moving in the same direction..understandable need, but…… By definition, at this point uncertainty is high and all the answers don’t exist It increases the risk that a medicine may be assessed as not CE, due to uncertainty, and thus ‘not recommended’ Result - increased pressure from all sides to consider Market Access Schemes for high need products

15. Market Access Schemes May be considered where a medicine might otherwise be evaluated as not cost-effective….and medical need is high eg Financial – eg total cost capped Clinical – eg outcome guarantee Challenges exist eg Suitable database to evaluate outcomes Suitable standardisation to allow evaluation ………but… Some schemes are relatively simple The potential value to patients is considerable Post-code variability can be targeted and reduced

16. Summary HTA methods are constantly developing – current focus is on assessing ‘value’ The Scottish Evaluation project and the PPRS both indicate that there is a significant shift beyond HTA methods, to the next decision points in the chain, eg to supporting timely & equitable uptake of cost- effective (and valued) medicines ….. Scottish patients will benefit