technologies only in the context of an appropriately designed - PowerPoint PPT Presentation

Informing a decision framework for when NICE should recommend the use of health technologies only in the context of an appropriately designed programme of evidence development MRC NIHR Methods programme, Jan 2010 to July 2011 Claxton K, Palmer S, Longworth L, Bojke L, Griffin S, McKenna C, Soares M, Spackman E, and Youn J. Centre for Health Economics, University of York Health Economics Research Group, Brunel University .

What assessments are needed • Is it expected to be cost-effective? – What impact on overall population health • Are there significant investment or reversal costs? – Capital costs and initially negative net health effects (NHE) • Is additional evidence needed – Does further research seem worthwhile • What type of evidence is needed – Type of research required, is it possible with approval • Will other sources of uncertainty resolve over time? – Changes in prices, technologies and evidence • Do the benefits of research exceed the costs – Will it be conducted, when will it be available, how much will be resolved • Are the benefits of approval greater than the costs

Sequence of assessment and decision (judgements) • Start where NICE appraisal stops – Assessment of expected cost-effectiveness is not sufficient – Categories of guidance • Approve and Reject • OIR = approval restricted to use only in research • AWR = approval but only with research, i.e., those not participating in the research can also have access – Different types of OIR, AWR (different considerations) • Represented as an algorithm – How different categories of guidance might be arrived at – Different consideration lead to the same category of guidance – Order of the assessments required – How guidance might change (price, evidence and technologies

Assess cost-effectiveness and Key Technologies population net health effects No Assessment Go to Figure 2.2 Is it cost-effective? expected to be Yes Decision Assess irrecoverable costs cost-effective # No Guidance Are there significant irrecoverable costs? Yes Assess need for evidence No Does more research seem worthwhile? Go to Yes Appendix A Part II What type of evidence is needed? No Is the research possible with Approval? Yes Assess other sources of uncertainty Assess other sources of uncertainty Will this No Will this No Uncertainty be resolved Uncertainty be resolved over time? over time? Yes Yes Will research be conducted? Will research be conducted? When will it be available? When will it be available? How much will be resolved? How much will be resolved? Re-assess the benefits and costs Re-assess the benefits and costs of further research of further research No No Are the Are the benefits of research greater benefits of research greater than the costs? than the costs? Yes Yes Assess the benefits and costs of early approval No Are the benefits of approval greater than the costs? Yes 1 1 2 1 3 4 AWR Approve Approve OIR Approve Approve

Technologies not expected to be Assess cost-effectiveness and population net health effects No Is it cost-effective? cost-effective Yes Assess irrecoverable costs No Are there significant irrecoverable costs? Yes Go to Figure 2.1 No Does more research seem worthwhile? Yes Go to Appendix A What type of evidence is needed? Part III No Is the research possible without Approval? Yes Assess other sources of uncertainty Assess other sources of uncertainty No No Will this Will this Uncertainty be resolved Uncertainty be resolved over time? over time? Yes Yes Will research be conducted? Will research be conducted? When will it be available? When will it be available? How much will be resolved? How much will be resolved? Re-assess the benefits and costs Re-assess the benefits and costs of further research of further research No No Are the Are the benefits of research greater benefits of research greater than the costs? than the costs? Yes Yes Assess the benefits and costs of early approval No Are the benefits of approval greater than the costs? Yes 2 1 2 2 4 OIR Reject AWR Reject Reject 3

Technologies with investment and reversal costs • Costs which are irrecoverable – Capital costs of long lived equipment (training and learning) – Initial losses (negative NHE) offset by later gains • Guidance might change – Research reports – Prices change, new technologies, other evidence • Expected to be cost-effective – OIR rather than AWR even if research is possible – OIR rather than Approve more likely to be appropriate – Reject rather than Approve is possible • Not expected to be cost-effective – Reject rather than OIR is more likely

Different types of guidance No significant irrecoverable costs Significant irrecoverable costs Research Not Possible with approval Not possible with Not Possible with approval Not possible with needed approval needed approval Benefits > Benefits Benefits > Benefits < Benefits > Benefits Benefits > Benefits < costs < costs costs costs costs < costs costs costs Approve (12) 4 1 2 3 11, 12 5,6 7, 9 8, 10 AWR (3) 1 3,4 OIR (5) 1 3,4 5,6 Reject (3) 7 5 6 No significant irrecoverable costs Significant irrecoverable costs Research Not Possible without Not possible without Not Possible without Not possible without needed approval approval needed approval approval Benefits > Benefits Benefits > Benefits < Benefits > Benefits Benefits > Benefits < costs < costs costs costs costs < costs costs costs Approve (0) AWR (2) 2 5 OIR (2) 2 7 Reject (8) 4 1 2 3 11 8 9 10

Change in effective prices (PAS VBP) and evidence • Price influences the benefits of early approval and the benefits of research – Threshold price (p*) for Reject to Approve (no uncertainty) • Reject to OIR > p* > OIR to Approve • Reject to OIR > OIR to AWR > AWR to Approve • Incentives for evaluative research • Prospects of research – Type of research needed – Will it be feasible and regarded as ethical – When likely to report – Priority for public funding or for manufacturers to undertake

A checklist of assessments Technologies not expected to be cost-effective Technologies expected to be cost-effective Point Assessment Judgement Point Assessment Judgement Yes No Yes No 1 1 Is it cost-effective? Is it cost-effective? Yes No 2 Are there significant irrecoverable costs? 2 Are there significant irrecoverable costs? 3 Does more research seem worthwhile? 3 Does more research seem worthwhile? 4 Is the research possible with approval? 4 Is the research possible without approval? 5 5 Will other sources of uncertainty resolve over time? Will other sources of uncertainty resolve over time? 6 6 Are the benefits of research greater than the costs? Are the benefits of research greater than the costs? 7 Are the benefits of approval greater than the costs? 7 Are the benefits of approval greater than the costs?

A checklist of assessments Technologies not expected to be cost-effective Point Assessment Judgement Yes No 1 Is it cost-effective? No 2 Are there significant irrecoverable costs? 3 Does more research seem worthwhile? 4 Is the research possible without approval? 5 Will other sources of uncertainty resolve over time? 6 Are the benefits of research greater than the costs? 7 Are the benefits of approval greater than the costs?

Assessment 1 2 3 4 5 6 7 Guidance 1 Yes No Yes Yes Yes/No Yes - AWR 1 2 Yes No Yes Yes Yes/No No - Approve 1 3 Yes No Yes No Yes/No Yes Yes Approve 2 4 Yes No Yes No Yes/No Yes No OIR 1 Part I of the algorithm 5 Yes No Yes No Yes/No No - Approve3 6 Yes No No - - - - Approve 4 7 No No Yes Yes Yes/No Yes - OIR 2 8 No No Yes Yes Yes/No No - Reject 1 9 No No Yes No Yes/No Yes Yes AWR 2 Possible pathways 10 No No Yes No Yes/No Yes No Reject 2 11 No No Yes No Yes/No No - Reject 3 12 No No No - - - - Reject 4 13 Yes Yes Yes Yes Yes Yes Yes AWR 3 14 Yes Yes Yes Yes Yes Yes No OIR 3 15 Yes Yes Yes Yes Yes No Yes Approve 5 16 Yes Yes Yes Yes Yes No No Reject 5 17 Yes Yes Yes Yes No Yes Yes AWR 4 Part II of the algorithm 18 Yes Yes Yes Yes No Yes No OIR 4 19 Yes Yes Yes Yes No No - Approve 6 20 Yes Yes Yes No Yes Yes Yes Approve 7 21 Yes Yes Yes No Yes Yes No OIR 5 22 Yes Yes Yes No Yes No Yes Approve 8 23 Yes Yes Yes No Yes No No Reject 6 24 Yes Yes Yes No No Yes Yes Approve 9 25 Yes Yes Yes No No Yes No OIR 6 26 Yes Yes Yes No No No - Approve 10 27 Yes Yes No n/a Yes n/a Yes Approve 11 28 Yes Yes No n/a Yes n/a No Reject 7 Part III of the algorithm 29 Yes Yes No n/a No - - Approve 12 30 No Yes Yes Yes Yes/No Yes - OIR 7 31 No Yes Yes Yes Yes/No No - Reject 8 32 No Yes Yes No Yes/No Yes Yes AWR 5 33 No Yes Yes No Yes/No Yes No Reject 9 34 No Yes Yes No Yes/No No - Reject 10 35 No Yes No - - - - Reject 11