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www.rarecancerseurope.org Paolo G. Casali paolo.casali@istitutotumori.mi.it Rare Tumours in Europe Rare Tumours in Europe CHALLENGES AND SOLUTIONS CHALLENGES AND SOLUTIONS 6 November 2008 - Brussels Rare Tumours in Europe Rare Tumours in

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Paolo G. Casali

paolo.casali@istitutotumori.mi.it www.rarecancerseurope.org

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Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

6 November 2008 - Brussels

Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

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Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

6 November 2008 - Brussels

Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

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Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

6 November 2008 - Brussels

Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

11. Encourage clinical studies in rare cancers that would provide data that supports rational, patient-shared, clinical decision-making, whilst acknowledging this as an essential means to overcome uncertainty surrounding patients’ immediate clinical needs.

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Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

6 November 2008 - Brussels

Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

17. Call for increased integration of local, national and European centres of expertise into European reference networks, based

  • n specific criteria as set out in the Commission’s proposed

Directive on the application of patients’ rights in cross-border healthcare , in order to provide the necessary sound

  • rganisational structures for more efficient clinical research and

early transfer of research data into clinical practice, thus improving the clinical management of rare cancers.

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From the reference center...

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…to the Reference network

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Reference center

" "

! expertise ! multidisciplinarity ! research

" "

! health migration ! implicit rationing ! failures in routine care

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! health migration ! implicit rationing ! failures in routine care

Reference network

! expertise ! multidisciplinarity ! research

" "

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Collaborative networks

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Extra medical time!

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Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

6 November 2008 - Brussels

Rare Tumours in Europe

CHALLENGES AND SOLUTIONS

7. 7. Call upon the research community to consider using a Bayesian approach for the design of clinical trials whenever well- powered randomised trials are not feasible due to the low incidence of the cancer entity and granted that sufficient information is available on the specific disease entity to empower such statistics (e.g. other clinical studies, biological evidence, analogies with more frequent diseases, the natural history of the disease, etc.). A mechanism for consensus development for definition of prior probability distributions should be devised.

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  • Mr. Bayes & Mr. Price. Phil Trans 1763;53:370
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P[A|B] = P[A] x P[B|A] P[B]

The Bayes theorem…

  • Mr. Bayes & Mr. Price. Phil Trans 1763;53:370
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The preclinical rationale

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Prior probability

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! clinical decision-making ! methods to combine evidence ! new study designs ! surrogate end points ! organization of studies

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Eur ur J J Cancer ancer 2011;47:2493 2011;47:2493

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Cas asali ali PG G et et al, al, 2012, 2012, submit ubmitted ed

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«Families» of rare cancers

! NON NON CUT UTANE NEOUS OUS MELA LANOM NOMA ! SKI KIN N -

  • Rar

are ! THOR HORACIC -

  • Rar

are ! UR UROGE OGENI NITAL L -

  • Rar

are ! FE FEMALE LE GE GENI NITAL L -

  • Rar

are ! MALE LE GE GENI NITAL ! NE NEUR UROE OEND NDOC OCRINE NE ! END NDOC OCRINE NE OR ORGA GAN N ! CNS NS ! SARCOM OMAS ! DIGE GESTIVE -

  • Rar

are ! HE HEAD & NE NECK K – – Rar are e ! HE HEMATOL OLOGI OGICAL L -

  • Rar

are ! PEDIA PEDIATRIC TRIC

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Nat Med 2010;9:363

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Malakoff D, Science 2011;331:1545

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0,1 0,2 0,3 0,4 0,5 0,6 0,7 0,8 0,9 1 3 6 9 12 15 18 21 24

Δ" Δ"

p <0.05

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Paolo G. Casali paolo.casali@istitutotumori.mi.it www.rarecancerseurope.org