Agenzia sanitaria e sociale regionale Methods in HTA – Health outcomes Bringing patients’ outcome at the forefront in HTA of diagnostic tests using the GRADE approach Luciana Ballini Luca Vignatelli, Susanna Maltoni, Antonella Negro, Fabio Trimaglio, Roberto Grilli Bilbao 26th June 2012 http://asr.regione.emilia-romagna.it
Appropriateness ofAgenzia sanitaria e sociale regionale FDG-PET in oncology: 3rd update Since 2001 a permanent research activity alongside the development and diffusion of the technology An obsession ? … or an opportunity • To reason on diagnostic-therapeutic strategies, rather than simply on diagnostic test • To address research gaps (clinical effectiveness) • To test a new methods for working groups developing diagnostic recommendations
Appropriateness of a diagnostic testAgenzia sanitaria e sociale regionale The value of any medical test is ultimately measured by whether the information it provides affects patient-relevant outcomes (Bossuyt 2010) • Initial diagnostic assessment with subsequent therapeutic approach • The potential of the new test to modify initial diagnostic Rationale assessment (e.g. stage of disease) • The change in management following change of diagnostic assessment • The diagnostic accuracy of the new test Available evidence • The resulting improvement in patients’ outcome due to change in therapeutic ? approach induced by new test’s results Schunemann et al BMJ 2008;336:1106-1110.
Agenzia sanitaria e sociale regionale Consequentialist Approach NOT MODIFIABLE MODIFIABLE Initial diagnostic assessment Treatment options Change in Management effectiveness SIGNIFICANT NEGLIGIBLE CURATIVE PALLIATIVE Diagnostic Impact on accuracy patients’ outcomes Diagnostic Outcomes efficacy trade off TEST POSITION harms versus benefits APPROPRIATENESS CRITERIA
Agenzia sanitaria e sociale regionale The consequentialist approach at work : information provided to the panel
NOT MODIFIABLE MODIFIABLE Initial diagnostic assessmentAgenzia sanitaria e sociale regionale Change in Management Treatment options effectiveness SIGNIFICANT NEGLIGIBLE CURATIVE PALLIATIVE CLINICAL QUESTION: Role of FDG-PET in early response to pre-operative chemoradiation of patients treated for locally advanced esophageal cancer Treatment effectiveness : Preoperative chemo/radio therapy improves 2 year survival by 13% (absolute difference) compared to surgical treatment only (Gebski Lancet Oncology 2007) Rationale: Preoperative chemo/radio therapy reduces tumour mass but increases risk of post-operative mortality (ESMO 2010). Early identification of non responders could avoid futile treatment and unnecessary risks RESEARCH QUESTION: FDG-PET as replacement Is FDG-PET accurate in evaluating early response to pre-operative chemoradiation of patients treated for locally advanced esophageal cancer ?
Diagnostic Outcomes accuracy importanceAgenzia sanitaria e sociale regionale LEVEL OF EVIDENCE PATIENT IMPORTANT OUTCOMES Schunemann et al BMJ 2008;336:1106-1110.
Diagnostic Outcomes efficacy trade off harms versus benefits TEST POSITIONAgenzia sanitaria e sociale regionale APPROPRIATENESS CRITERIA Median scores of importance 7 8 7 5 Not good enough INAPPROPRIATE
RAND/UCLA Appropriateness Method: ResultsAgenzia sanitaria e sociale regionale 7 panels (60 experts) – 7 tumours Lung Breast Head & Neck 55 Esophageal questions Colo-rectal H Lymphoma 12 11 4 indeterminate 13 inappropriate 27 inappropriate NH Lymphoma appropriate Disagreement No rationale 5 9 11 appropriate inappropriate 2 uncertain disagreement 28/55 (50.9%) resolved at first voting round 9 light Disagreement 16/55 (29.1%) resolved at second voting round 11/55 (20%) persistent disagreement 2 Strong Disagreement
Was the approach followed by the panels ?Agenzia sanitaria e sociale regionale 21 20 18 N° of questions 16 14 YES (40 questions) 14 12 NO (4 questions) 10 8 6 4 4 2 1 2 1 1 0 in te ria de in p in te pr o rta ap rm e te un ap nc ri a pr in u ce p te op pro ap ina at rt ria e ap pr ai Observed (RAND) in rm et e te n op ind ria Expected(GRADE) te Yes in 73% of questions (considering also the 11 unresolved questions)
Agenzia sanitaria e sociale regionale The panelists Thank you firstname.lastname@example.org http://asr.regione.emilia-romagna.it