Evaluating Highly Specialised Technologies -- An Economist's View

2,036 views

Published on

This presentation addressed core issues in the challenge of evaluating technologies that treat rare diseases. It draws together information about current economic thinking and practices that most affect decisions, offering suggestions as to where and how the patient's input might be most important and influential.

Published in: Health & Medicine
0 Comments
0 Likes
Statistics
Notes
  • Be the first to comment

  • Be the first to like this

No Downloads
Views
Total views
2,036
On SlideShare
0
From Embeds
0
Number of Embeds
71
Actions
Shares
0
Downloads
0
Comments
0
Likes
0
Embeds 0
No embeds

No notes for slide

Evaluating Highly Specialised Technologies -- An Economist's View

  1. 1. Evaluating Highly Specialised Technologies – An Economist’s View Jon Sussex Genetic Alliance Patient Group Workshop London • 28 November 2013
  2. 2. Agenda 1. Cost effectiveness and outcomes 2. Problems evaluating HSTs 3. Factors driving yes/no decisions 4. A value framework for rare disease treatments? 5. Maximising patient / carer impact HST Evaluation – An Economist’s View 28/11/2013 2
  3. 3. Cost Effectiveness + cost x x  - effect x  - cost + effect  HST Evaluation – An Economist’s View 28/11/2013 3
  4. 4. Patient-reported Outcome Measures • Patients’ own assessment of their health – purposefully subjective • Many well-validated instruments exist that are reliable, sensitive and widely used • Simple to complete; quick to analyse • Repeated “snap shots” of health (e.g. before and after treatment) can provide a clear picture of changes in health • Disease specific PROMs: more question items/response options; focussed on a specific aspect of health • Generic PROMs: measure health-related quality of life generally. Enable comparisons of health across conditions/health services, e.g. EQ-5D and SF-36 HST Evaluation – An Economist’s View 28/11/2013 4
  5. 5. EQ-5D Please indicate which statements best describe your own health state today. Tick one box for each group of statements. Mobility I have no problems in walking about I have some problems in walking about I am confined to bed    Self-Care I have no problems with self-care I have some problems washing or dressing myself I am unable to wash or dress myself    Usual Activities I have no problems with performing my usual activities (e.g. work, study, housework, family or leisure activities) I have some problems with performing my usual activities I am unable to perform my usual activities    Pain/Discomfort I have no pain or discomfort I have moderate pain or discomfort I have extreme pain or discomfort    Anxiety/Depression I am not anxious or depressed I am moderately anxious or depressed I am extremely anxious or depressed © 2013 EuroQol Group   
  6. 6. Measuring quality of life using EQ-5D © 2013 EuroQol Group
  7. 7. Problems Evaluating Medicines for Rare Diseases • Costs not much lower than for other medicines • Patents last no longer than for other medicines – but OMP* legislation helps offset that • Small patient numbers mean low sales volumes and hence high prices • Small patient numbers mean longer and more complex trials yielding more uncertain evidence *orphan medicinal products HST Evaluation – An Economist’s View 28/11/2013 7
  8. 8. Mean R&D Costs per Successful New Molecular Entity by Year of Study Publication (US$2011m)  2,000  1,500  1,000  500 2012 2011 2010 2008 2006 2004 2003 2001 1999 1997 1995 1993 1991 1989 1987 1985 1983 1981  ‐ 1979 Estimates of mean costs per approved drug (US$m, in 2011prices)  2,500 Source: Mestre-Ferrandiz et al. 2012 HST Evaluation – An Economist’s View 28/11/2013 8
  9. 9. The Commercial Imperative € Revenue minus production costs Launch R&D costs Patent expiry HST Evaluation – An Economist’s View 28/11/2013 9 Years
  10. 10. Accepting Uncertain Evidence • If payer is risk neutral, and the state should be, then no problem in principle • But in practice regulators are risk averse • And are much more risk averse than sufferers from severe diseases and their carers HST Evaluation – An Economist’s View 28/11/2013 10
  11. 11. NICE’s Standard Criteria (weights not revealed) • Incremental cost effectiveness ratio (ICER) £/QALY • Severity of illness (Rawlins et al, 2010) • Stakeholder insights (Rawlins et al, 2010; NICE, 2008) • End of life treatments (Rawlins et al, 2010; NICE, 2008) • Disadvantaged populations (Rawlins et al, 2010; NICE, • Children given “benefit of the doubt” (Rawlins et al, 2010) 2008) HST Evaluation – An Economist’s View 28/11/2013 11
  12. 12. Impact of ICER Ranking on NICE Recommendations (Dakin et al, 2013) HST Evaluation – An Economist’s View 28/11/2013 12 £500,000 £100,000 £70,000 £45,500 £50,000 £60,000 £37,500 £40,000 £45,000 £35,000 £32,500 £30,000 £27,500 £20,000 £22,500 £25,000 £17,500 £15,000 £12,500 £10,000 £7,500 £5,000 £2,500 £0 Decisions with high ICERs are more likely to be rejected, but there are many exceptions
  13. 13. Predicted Probability of NICE Rejection at Different ICER Values – Holding All Other Variables at Mean Levels (Dakin et al, 2013) HST Evaluation – An Economist’s View 28/11/2013 13
  14. 14. NICE HST Interim Process and Methods HST Evaluation – An Economist’s View 28/11/2013 14
  15. 15. Who Gives Special Consideration to Medicines for Rare Diseases? NICE – England and Wales Yes – Interim HST process (previously AGNSS). Also Cancer Drugs Fund SMC – Scotland No – But Rare Conditions Fund PBAC - Australia Life Saving Drugs Program and “rule of rescue” though not about rarity per se GB-A - Germany < €1million exempt from economic evaluation Other countries with formal No evaluation systems (Canada, Finland, France, Netherlands, New Zealand, Norway, Sweden) HST Evaluation – An Economist’s View 28/11/2013 15
  16. 16. A Pilot Study of MCDA for Valuing Orphan Medicines http://www.valueinhealthjournal.com/article/ S1098-3015(13)04356-8/abstract http://www.ohe.org/publications/article/valuing -orphan-medicines-using-multi-criteriadecision-analysis-129.cfm HST Evaluation – An Economist’s View 28/11/2013 16
  17. 17. Possible Value Attributes and Weights (Sussex et al, 2013) Per cent ‘Experts’ ‘Patients’ workshop workshop 19.5 11 Disease survival prognosis with current soc 14 11.5 Disease morbidity and patient clinical disability with current soc 12 15 Social impact of disease on patients’ and carers’ daily lives with current soc 8 15 53.5 52.5 0 5 27.5 17.5 Treatment safety 8 7.5 Social impact of treatment on patients’ and carers’ daily lives 11 17.5 Sub-total weight for impact of new medicine 46.5 47.5 Total 100 100 Availability of existing treatments Sub-total weight for impact of disease / extent of unmet need Treatment innovation: scientific advance + contribution to patient outcome Evidence of treatment clinical efficacy and patient clinical outcome HST Evaluation – An Economist’s View 28/11/2013 17
  18. 18. Compared with NICE Criteria Among NICE criteria? ‘Patients’ workshop workshop 19.5 11 14 11.5 Disease morbidity and patient clinical disability with current soc 12 15 Social impact of disease on patients’ and carers’ daily lives with current soc ≈ Severity ‘Experts’ Disease survival prognosis with current soc Standard NO, HST YES Per cent 8 15 53.5 52.5 0 5 27.5 17.5 Treatment safety 8 7.5 Social impact of treatment on patients’ and carers’ daily lives 11 17.5 Sub-total weight for impact of new medicine 46.5 47.5 Total 100 100 Availability of existing treatments { Sub-total weight for impact of disease / extent of unmet need No Treatment innovation: scientific advance + contribution to patient outcome { Evidence of treatment clinical efficacy and patient clinical outcome ≈ QALY HST Evaluation – An Economist’s View 28/11/2013 18
  19. 19. Who Listens to “Patient’s Voice”? (Shah et al, 2013) • NICE (England & Wales) – Yes, sometimes cited in explanations of recommendations • SMC (Scotland) – No evidence • CED (Ontario) – No evidence • HAS (France) – No evidence • PBAC (Australia) – No evidence HST Evaluation – An Economist’s View 28/11/2013 19
  20. 20. Getting in Early – Where Patient Inputs Can Achieve Most • Value greater from patients / carers: • • Aiding better measurement of quality of life • Informing better valuation of different qualities of life (“health states”) and other dimensions of value • • Enabling better understanding of what kinds of quality of life impacts diseases and treatments have Changing attitudes to uncertainty Rather than advocacy / special pleading HST Evaluation – An Economist’s View 28/11/2013 20
  21. 21. For Example… • Supporting development of quality of life instrument for rare diseases (as a group) • Supporting research to produce a rare-disease value framework • Inputting to design of clinical trials – what is measured and how • Filling gaps in evidence – by surveying patients/carers HST Evaluation – An Economist’s View 28/11/2013 21
  22. 22. About OHE To enquire about additional information and analyses, please contact Jon Sussex at jsussex@ohe.org To keep up with the latest news and research, subscribe to our blog, OHE News. Follow us on Twitter @OHENews, LinkedIn and SlideShare. The Office of Health Economics is a research and consulting organisation that has been providing specialised research, analysis and expertise on a range of health care and life sciences issues and topics for more than 50 years. OHE’s publications may be downloaded free of charge for registered users of its website. Office of Health Economics Southside, 7th Floor 105 Victoria Street London SW1E 6QT United Kingdom+44 20 7747 8850 www.ohe.org ©2013 OHE HST Evaluation – An Economist’s View 28/11/2013 22

×