![Page 2: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/2.jpg)
▪ 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
![Page 3: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/3.jpg)
Ann Oncol 2012;23:2442
![Page 4: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/4.jpg)
▪ 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
![Page 5: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/5.jpg)
Treatment «phases»
localized metastases terminal
disease (1st CT) (2st CT) disease
cancer
M-C
T-N-M
PS
![Page 6: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/6.jpg)
![Page 7: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/7.jpg)
![Page 8: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/8.jpg)
▪ Clinical decision-making
▪ Methods to combine evidence
▪ New study designs
▪ Surrogate end points
▪ Organization of studies
![Page 9: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/9.jpg)
London, October 3rd 2014
![Page 10: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/10.jpg)
London, June 30th 2016
![Page 11: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/11.jpg)
Martinalbo J et alESMO 2016
![Page 12: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/12.jpg)
Pros/retrospective clinical DBs
![Page 13: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/13.jpg)
Networking
![Page 14: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/14.jpg)
![Page 15: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/15.jpg)
www.jointactionrarecancers.eu
![Page 16: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/16.jpg)
![Page 17: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/17.jpg)
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
![Page 18: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/18.jpg)
efficacy
effectiveness
CLINICAL RESEARCH
OUTCOME RESEARCH
![Page 19: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/19.jpg)
Pro & Cons of generating RWE
from RWD
◼ Low validity
◼ Quality control
◼ Collection bias
◼ Multiple sources > closer to causality
◼ Closer but unstructured
![Page 20: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/20.jpg)
Dec 2018
![Page 21: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/21.jpg)
![Page 22: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/22.jpg)
Pharmacoepidemiology and Drug Safety and
Value in Health. Berger
2017
![Page 23: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/23.jpg)
«Big data»…
R p<0.05
![Page 24: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/24.jpg)
Examples of issues that real world data (RWD) can address at
the different stages of the drug development lifecycle
![Page 25: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/25.jpg)
![Page 26: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/26.jpg)
R p<0.05
![Page 27: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/27.jpg)
…is a patient-physician shared decisionin conditions of uncertainty!
The good clinical decision…
![Page 28: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/28.jpg)
N Engl J Med 1987;316:250
![Page 29: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/29.jpg)
![Page 30: Tumori rari - Healthcare Research & Pharmacoepidemiology · Tumori rari Lisa Licitra lisa.licitra@istitutotumori.mi.it Pediatric cancers Haematologic rare neoplasms Sarcomas Rare](https://reader033.vdocument.in/reader033/viewer/2022042803/5f45abf046480712417c3f0b/html5/thumbnails/30.jpg)