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www.jointactionrarecancers.eu
www.jointactionrarecancers.eu
rare
diseases
cancerrare
cancers
rarecancers
Eur J Cancer 2011;47:2493
1. Pediatric cancers
2. Haematologic rare neoplasms
3. Sarcomas4. Rare thoracic cancers5. Neuroendocrine tumours6. Head & neck cancers7. Central nervous system tumours 8. Rare female genital cancers9. Rare urological and male genital tumours10. Endocrine gland tumours11. Digestive rare cancers12. Rare skin cancers & non-cutaneous melanoma
Rare cancers…
childhood
Rare cancers…
haematologicalchildhood
Rare cancers…
childhoodhaematological
solid of the adult
“Orphan drugs”
“unlikely that marketing would generate sufficient returns to justify the investment needed for development”
EU «Orphan Regulation»
10-year marketing exclusivity fee reductions and exemptions specific scientific advice protocol assistance national incentives EU-funded research
Registration & reimbursementrisk…
p
t
p
t
* in 2017 RCE is also supported by Lilly and AbbVie
17.
Call for increased integration of local, national and European centres of expertise into European reference networks, based on 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 organisational structures for more efficient clinical research and early transfer of research data into clinical practice, thus improving the clinical management of rare cancers.
“will providehighly specialised healthcare for rare or low prevalence complex diseases or conditions”
promote good quality and safe care to patients by fostering properdiagnosis, treatment, follow-up and management of patientsacross the Network
empower and involve patients offer and promote multi-disciplinary advice for complex cases develop and implement clinical guidelines and cross-border patient
pathways exchange, gather and disseminate knowledge, evidence and
expertise within and outside the Network promote collaborative research within the Network reinforce research and epidemiological surveillance, through
setting up of shared registries exchange and disseminate knowledge and best practices, in
particular by supporting national centres and networks
Objectives
With regard to RCs in the EU, to improve:1. Epidemiological surveillance2. Quality of care through ERNs3. Clinical practice guidelines4. Innovation5. Medical and Patient education6. Health policy measures7. Patient empowerement
Work packages
WP
1 Coordination INT, IT2 Dissemination NKUA, GR3 Evaluation CSF, FI4 Epidemiology INT, IT
5 Assuring Quality of Care OECI
6 Clinical practice guidelines DKG, DE
7 Innovation and access to innovation WIV-ISP, BE
8 Medical education UP, HU
9 Childhood Cancers SIOPE10 Rare Cancer Policy ICO, ES
18 MSs 34 ass. partners
Distance case sharing
Big data & machine learning…
PLoS One 2015;10:e0141357
«Big data» & knowledge generation…
R p<0.05
Milan, 2018
Methodological implications(vs clinical trials!)
Technological windows ofopportunity
Added values for rare cancers
“BIG DATA, AI & RCs”
Martinalbo J et alESMO 2016
Martinalbo J et alESMO 2016
London, June 30th 2016
Adaptive licensing
“a prospectively planned processby which an early authorizationin a restricted patient populationis followed by iterative phasesof evidence-gathering and adaption of the marketing authorization to broader patient populations”
16 April 2018
Oye KA et al. Clin Pharmacol Ther 2016;100:626
The Bayesian probabilityin clinical research…
the probabilitythat a treatment is effective…
The frequentist probabilityin clinical trials…
the probabilityyou had to find this differenceif there were no difference(null effect)…
p<0.05
Mr. Bayes & Mr. Price. Phil Trans 1763;53:370
P[A|B] = P[A] x P[B|A]P[B]
The Bayes theorem…
Mr. Bayes & Mr. Price. Phil Trans 1763;53:370
J Natl Cancer Inst 2011;103:2
Clinical decision-making Methods to combine evidence New study designs Surrogate end points Organization of studies
London, October 3rd 2014
London, April 30th 2015
Pharma
Regulators
Pharma
RegulatorsResearchers
Pharma
RegulatorsResearchersPatients
…….
Frankfurt, 18 April 2017
SARCOMAS
Approval vs reimbursement…
Pandora’s boxJ.W. Waterhouse - 1896
Eur J Cancer 2011;47:2493
Precision oncology…
Precision medicine
