The NICE Diagnostics Assessment Programme

1. The NICE Diagnostics Assessment Programme UK Genetic Testing Network Conference: Transformation – Changes to Improve Patient Care November 2012, London Nick Crabb Associate Director, Diagnostics Assessment Programme, NICE

2. Overview • Introduction to NICE • Medical technologies programmes • Medical Technologies Evaluation Programme (MTEP) • Diagnostics Assessment Programme (DAP) • Guidance recommendations • Evidence considerations • Companion diagnostics • Support for implementation

3. NICE The National Institute for Health and Clinical Excellence (NICE) is the independent organisation responsible for providing national guidance on the promotion of good health and the prevention and treatment of ill health. It was established in 1999 as a Special Health Authority and in 2005 it was expanded to include the functions of Health Development Agency. In 2013 NICE will be re-established as a non departmental public body and will expand to incorporate social care.

4. Medical technologies programmes • Programmes established in 2010 driven by notification of technologies by manufacturers / sponsors • Two new programmes representing major increase in capacity for medtech evaluation • Programmes aim to improve the timeliness and consistency of adoption of technologies with the potential to: • Improve patient outcomes • Reduce costs • Provide system benefits (e.g. facilitate service redesign)

5. The two programmes • Medical Technologies Evaluation Programme (MTEP) and the Medical Technologies Advisory Committee (MTAC) • Undertakes topic selection and routing for all medtech products, including diagnostics • Produces guidance on topics routed to itself • Supported by an infrastructure of independent external assessment centres Note: Some diagnostic technologies are assessed within MTEP • Diagnostics Assessment Programme (DAP) and the Diagnostics Advisory Committee (DAC) • Specialist programme for complex assessments of diagnostic technologies • Appropriate topics routed to DAP by MTAC • Supported by an infrastructure of independent external assessment groups

6. Assessment of diagnostic technologies within the MTEP • Programme methodology includes the evaluation of clinical effectiveness and cost consequences – but not cost effectiveness (no evaluation of patient outcome benefits) • 38 week process from topic selection to guidance publication • Assessments limited to the specific technology notified to the programme by the sponsor • Evidence submission by the sponsor, including economic model to support the case for adoption • Applicable to diagnostics technologies offering both: • Equivalent or superior clinical performance compared to current practice • Potential for cost savings or no net increase to NHS costs • Dependence on the evidence submission means that sponsors will normally need to be commercial sponsors (manufacturers or distributors)

7. Diagnostics Assessment Programme (DAP) • Specialist programme to undertake complex assessments of diagnostic technologies • Diagnostics advisory committee structure includes standing members and specialist members to ensure deep expertise on each topic • Assessments include cost effectiveness analysis (requires the evaluation of outcome benefits – QALYs) • Assessments may involve single or multiple related diagnostic technologies (scope may be expanded beyond the notified technology that was selected and routed by MTAC) • 62 week process from topic selection to guidance publication • Manufacturers encouraged to provide relevant information through a structured evidence request but no formal submission is required • Modelling to estimate outcome benefits and cost effectiveness is undertaken as part of the assessment • Sponsors may be commercial sponsors or senior members of the clinical community

8. Characteristics of topics for the DAP • Cost effectiveness analysis needed to provide a meaningful assessment • Expensive new technology with the potential to significantly improve patient outcomes • Care pathway(s) not well understood requiring access to highly specialist clinical expertise • Significant advantages of assessing multiple technologies

9. Guidance recommendations • The NICE guidance from the DAP or MTEP may include: • Adoption recommendations • Research recommendations • Research recommendations expected to be important for DAP and MTEP • The programmes include capacity for research commissioning to: • Clearly capture the key research questions resulting from evidence gaps • Facilitate the efficient generation of research data

10. Evidence considerations - DAP • DAP methodology includes cost effectiveness analysis • Patient outcome benefits – length and quality of life, are estimated (in comparison to current standard clinical care – the comparator) • Measured in quality adjusted life years (QALY) • Costs throughout the care pathway are also determined in comparison to costs for the comparator • Cost effectiveness calculated – cost (£) per QALY • This requires evidence throughout the care pathway Impact on treatment decisions Diagnostic accuracy Impact on outcomes

11. Generating the patient outcome estimates • End to end clinical studies that follow patients from diagnosis through care to outcomes • Typically not available for diagnostics • In some cases may be the only way to demonstrate the value of a diagnostic • Linked evidence modelling • In some cases there may be existing evidence for parts of the care pathway that can be used to develop models for estimating outcome benefits and cost effectiveness • In some cases it may also be possible to use existing models (directly or with modification) • It may be possible to link to the existing evidence from diagnostic accuracy data (robust diagnostic accuracy data is probably the minimum evidence needed)

12. Companion diagnostics(CDx) • CDx are used to identify sub-populations of patients where treatment is likely to be more effective • Increasingly, licensed indications for pharmaceuticals require the use of CDx • The health technology assessment (HTA) of CDx may be simpler than for diagnostics generally • Health outcomes from treatment informed by the CDx are available from the clinical trials of the treatment • Less dependence on modelling approaches to estimate the patient outcome benefits • European regulation of CDx does not require that the diagnostic used in clinical trials be used in clinical practice • There may be several proprietary or “in-house” tests that could be considered as consistent with the licensed indications even though the diagnostic accuracy of the various options and potential impact on the patient population for treatment may not be understood

13. CDx in NICE Technology Appraisals (TA) • Pharmaceuticals, including those dependent on the use of CDx are evaluated in the NICE TA programme • Updated methods guide developed in 2012 (publication expected Q4 2012/13) includes consideration of companion diagnostics: • Within a TA the CDx used in the pivotal clinical trials is likely to be considered • Cost effectiveness determination accounts for the cost of the drug and CDx (with the sensitivity analysis including a scenario where the CDx costs are excluded) • The guidance may include commentary on the companion diagnostic used in the pivotal trials • The guidance may include consideration of the potential impact of using alternative tests • The appraisal is unlikely to evaluate alternative CDx options

14. CDx in the NICE DAP • Diagnostic technologies for use as CDx products may be notified to NICE by sponsors (manufacturers or senior members of the clinical community) • Where appropriate these may be selected for evaluation • The DAP process allows for the various CDx options (e.g. other proprietary and “in-house” tests) to be included in the assessment • The assessment may include separate cost effectiveness determinations for each pharmaceutical / CDx combination • Current DAP CDx topics: • Epidermal growth factor receptor tyrosine kinase (EGFR-TK) mutation testing in adults with locally advanced or metastatic non-small-cell lung cancer • KRAS mutation testing in adults with metastatic colorectal cancer

15. Support for implementation • NICE implementation support activities • NICE is committed to bespoke and innovative implementation products to support medical technologies and diagnostics guidance - so far, these have included audit criteria, podcasts, webinars, technical/usability reports and costing tools • Promotion of guidance through regional implementation consultants • NHS technology adoption centre (NTAC) • Increased focus on technologies approved by NICE • New activities from the NHS Innovation Health and Wealth report including establishment of NICE implementation collaborative

16. Further information • Please visit http://www.nice.org.uk/aboutnice/whatwedo/aboutdiagnosticsassessment/diagnosticsassessmentprogramme.jsp for: • Further background to the DAP • Published diagnostics guidance (6 topics completed to date) and supporting documents • Information on DAP assessments in development • Programme manual detailing DAP processes and methodology