Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra...

Tumori rari

Lisa Licitralisa.licitra@istitutotumori.mi.it

▪ Pediatric cancers

▪ Haematologic rare neoplasms

▪ Sarcomas

▪ Rare thoracic cancers

▪ Neuroendocrine tumours

▪ Head & neck cancers

▪ Central nervous system tumours

▪ Rare female genital cancers

▪ Rare urological and male genital

tumours

▪ Endocrine gland tumours

▪ Digestive rare cancers

▪ Rare skin cancers & non-cutaneous

melanoma

Eur J Cancer 2011;47:2493

Ann Oncol 2012;23:2442

▪ Referral to expert rare cancer pathologists

is crucial for appropriateness

▪ Networks are the best tool for proper

referral

▪ Multidisciplinarity is the best environment

for rare cancer patient healthcare

Treatment «phases»

localized metastases terminal

disease (1st CT) (2st CT) disease

cancer

M-C

T-N-M

PS

▪ Clinical decision-making

▪ Methods to combine evidence

▪ New study designs

▪ Surrogate end points

▪ Organization of studies

London, October 3rd 2014

London, June 30th 2016

Martinalbo J et alESMO 2016

Pros/retrospective clinical DBs

Networking

www.jointactionrarecancers.eu

Innovative trial designs

◼ Biomarker led designs (umbrella, basket): predictive, prognosticenrichment

◼ Adaptive design: trial strategymodification pre-defined rules

◼ Cohort led design & use of RWD: pragmatic trials, hybrid, observational

efficacy

effectiveness

CLINICAL RESEARCH

OUTCOME RESEARCH

Pro & Cons of generating RWE

from RWD

◼ Low validity

◼ Quality control

◼ Collection bias

◼ Multiple sources > closer to causality

◼ Closer but unstructured

Dec 2018

Pharmacoepidemiology and Drug Safety and

Value in Health. Berger

2017

«Big data»…

R p<0.05

Examples of issues that real world data (RWD) can address at

the different stages of the drug development lifecycle

R p<0.05

…is a patient-physician shared decisionin conditions of uncertainty!

The good clinical decision…

N Engl J Med 1987;316:250

lisa.licitra@istitutotumori.mi.it