Scenari per la determinazione del valore di un farmaco oncologico (ASCO ESMO). Perché Ni Giovanni L. Pappagallo
The wide array of treatment options, their attendant clinical impact, and cost to the patient suggested the importance of developing a framework with which to assess new therapies. Two frameworks have been developed: one for advanced disease and the other for potentially curative disease. Essential domains in the assessment of an agent s value were clinical benefit, toxicity, and cost.
ASCO Framework for Assessing Value in Cancer Care Will serve as a tool in the process of shared decisionmaking between physician and patient: designed to have the relative weights (importance) be modifiable according to patient preference
ASCO Framework for Assessing Value in Cancer Care Will serve as a tool in the process of shared decisionmaking between physician and patient: designed to have the relative weights (importance) be modifiable according to patient preference The composite of the clinical benefit, bonus points, and toxicity scores is tallied to generate a net health benefit (NHB): a measure of the relative improvement a new regimen has yielded when compared with the control therapy against which it was compared in the clinical trial
ASCO Framework for Assessing Value in Cancer Care
This tool uses a rational, structured and consistent approach to derive a relative ranking of the magnitude of clinically meaningful benefit that can be expected from a new anti-cancer treatment.
ESMO-MCBS To assign the highest grade to trials having adequate power for a relevant magnitude of benefit, and to make appropriate grade adjustment to reflect the observed magnitude of benefit. does not address the issue from the perspective of communication between physician and patient a policy tool, not a tool to be deployed at the clinical interface
ESMO-MCBS To assign the highest grade to trials having adequate power for a relevant magnitude of benefit, and to make appropriate grade adjustment to reflect the observed magnitude of benefit. Clinical benefit in either the advanced or potentially curative settings is judged by both the hazard ratios achieved and absolute improvement in the prespecified clinical endpoint defined for the trial in question
ESMO-MCBS What if non proportional hazards? (metastatic disease, no long-survivors; plateau of longsurvivors; etc.) Therapeutic alternative Vs control arm Incomplete efficacy estimator; correlation with HR?
ESMO-MCBS as I thought M.I.D. or P<0.05? M.I.D. or P<0.05? Clinical relevance or P<0.05? What if grade 2 diarrhea?
ESMO-MCBS To assign the highest grade to trials having adequate power for a relevant magnitude of benefit, and to make appropriate grade adjustment to reflect the observed magnitude of benefit. Clinical benefit in either the advanced or potentially curative settings is judged by both the hazard ratios achieved and absolute improvement in the prespecified clinical endpoint defined for the trial in question To provide a clear, well-structured and validated mechanism to indicate the magnitude of benefit in addition to the level of evidence in ESMO guidelines
ESMO-MCBS To assign the highest grade to trials having adequate power for a relevant magnitude of benefit, and to make appropriate grade adjustment to reflect the observed magnitude of benefit. Clinical benefit in either the advanced or potentially curative settings is judged by both the hazard ratios achieved and absolute improvement in the prespecified clinical endpoint defined for the trial in question To provide a clear, well-structured and validated mechanism to indicate the magnitude of benefit in addition to the level of evidence in ESMO guidelines
ESMO-MCBS
Il percorso verso la proposta terapeutica Una volta definito con chiarezza il quesito clinico sarà necessario verificare: l affidabilità delle evidenze (confidence) la diretta (o meno) trasferibilità delle evidenze disponibili alla tipologia di paziente oggetto del quesito clinico (directness) la rilevanza clinica degli effetti osservati (relevance)
Strutturazione del Quesito Clinico sec. modello P.I.C.O. P I C O Nei Pazienti con l Intervento (è suscettibile di impiego) in Confronto con riguardo agli Outcome di beneficio/danno Specifiche caratteristiche di malattia (stadio, classe di rischio, ecc.) Intervento terapeutico oggetto del quesito clinico Trattamento altrimenti considerabile in alternativa all intervento in esame Parametri clinico-laboratoristici ritenuti essenziali per la decisione terapeutica
Il percorso verso la proposta terapeutica Una volta definito con chiarezza il quesito clinico sarà necessario ESMO-MCBS verificare: Guidelines: l affidabilità non strutturazione delle evidenze sec. (confidence) P.I.C.O. la diretta (o meno) trasferibilità delle evidenze disponibili alla tipologia di paziente oggetto del quesito clinico (directness) la rilevanza clinica degli effetti osservati (relevance)
Il percorso verso la proposta terapeutica Una volta definito con chiarezza il quesito clinico sarà necessario verificare: l affidabilità delle evidenze (confidence) la diretta (o meno) trasferibilità delle evidenze ESMO-MCBS disponibili Guidelines: alla tipologia di paziente Study oggetto Design, del Allocation quesito clinico Concealment (Attrition? (directness) Detection? Performance? la rilevanza Imprecision? clinica Inconsistency?) degli effetti osservati (relevance)
Il percorso verso la proposta terapeutica Una volta definito con chiarezza il quesito clinico sarà necessario verificare: l affidabilità delle evidenze (confidence) la diretta (o meno) trasferibilità delle evidenze disponibili alla tipologia di paziente oggetto del quesito clinico (directness) ESMO-MCBS Guidelines: non strutturazione sec. P.I.C.O. la rilevanza clinica degli effetti osservati (relevance)
Il percorso verso la proposta terapeutica Una volta definito con chiarezza il quesito clinico sarà necessario verificare: l affidabilità delle evidenze (confidence) la diretta ESMO-MCBS (o meno) Guidelines: trasferibilità delle evidenze Soglie disponibili rilevanza alla clinica tipologia di paziente per oggetto QoL e reazioni del quesito averse? clinico (directness) la rilevanza clinica degli effetti osservati (relevance)
For Against
Relevance of outcomes (benefit and harm) Quality of Evidence (confidence, directness) Base Risk (related to disease state) Benefit to Harm ratio Relevance of Effects (benefits and harms) ESMO-MCBS
Scenari per la determinazione del valore di un farmaco oncologico (ASCO ESMO). Ni, perché Giovanni L. Pappagallo