Embed Size (px)
Citation preview
Personalised Medicine - the Future of Drug Development
Payer’s Perspective
Anouk Waeytens
National Institute for Health and Disability Insurance Belgium
1
the views and opinions expressed in this presentation are thoseof the author
2
disclaimer
improved health outcomes
avoid unnecessary toxicities (treatment, hospitalization)
improve compliance and persistence
avoid loss of precious treatment time
more clear benefit/risk profile
avoid needless expenses (treatment, toxicities)
3
opportunities
re-use in new populations
smaller and shorter clinical studies
higher cost-effectiveness, easier to demonstrate
high market share within select patient population
extension of select patient populations
4
opportunities
opportunities…
or “great expectations”?
5
6
‘personalised medicines’ reimbursed in Belgium
0
1
2
3
4
5
6
2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018
7
relevant biomarkers
Vermaelen et al (2018) Sem Canc Biol 52: 166
possibility of response vs.
absence of response
Vermaelen et al (2018) Sem Canc Biol 52: 1668
relevant biomarkerscomplexity -->
standardization
exact quantification
reduce bias
centralization of expertise
automation
digitalization
9
relevant biomarkers
Loyola University Health System 2017
10
relevant biomarkers
Rugo et al (2017) JAMA 317: 37
11
personalised medicine
ADAM SIMPSON, PERSONALISED MEDICINE
personalised dosingde-escalation
unmet need
early dialogue
12
NEW EU regulation on in vitro diagnostics medical
devices (IVDR)
Entry into force: spring 2022
new risk classification system (A-D)
clinical evidence, including an EU-wide coordinated procedure for authorisation of multi-centre clinical investigations
Specific rules CDx, LDTs, genetic tests etc.
13
relevant biomarkers
proof of clinical utility
retrospectively or without co-development
when evidence is of sufficent methodological quality
original vs.
“me too” cfr. biosimilar
LDT cfr. pharmaceutical compounding
14
cost-effectiveness
Van den Bulcke et al (2015) KCE report 240
€ 24.500 € 24.700 € 24.900 € 25.100 € 25.300 € 25.500 € 25.700 € 25.900 € 26.100 € 26.300 € 26.500
sensitivity
specificity
prevalence biomarker
cost of diagnostic test
cost of therapy/cycle
Tornado diagram +/-1%
15
cost-effectiveness
San Miguel & Hulstaert, J. Comp. Eff. Res. 2015
Eichler et al (2011) Nat Rev Drug Discovery (10): 49516
17
budget impact drugs vs. tests
Vlerick HMC report 2015-1 oncology horizon scanning based on NIHDI data
Pact for the Future:
improved access to medication
fostering innovation
new deontological framework
medicines
MEA BENELUXAIR
UMN, MOCA
patent cliff
biosimilars
tests
revision of current fees ( savings)
extra input
18
market access
19
market accessreimbursement of medicines (CRM)
versus
reimbursements of acts/diagnostic tests (TMC)
Challenge:
2 different technologies/processes/expertise
2 different commissions/timelines/budgets/… (TMC vs. CRM)
quick technological & scientific evolution
GCP
20
market accessAS IS:
Medicines:
6 months + suspensions
Tests: first: WG Clinical Biology, then:
minimum 18 months
TMCMedico-
mutCBC Ins Com Minister RD
CRM Minister MD
NIHDI vision on
access to personalized medicine:
health care reimbursement procedures should allow
a simultaneous reimbursement start for treatment and test
21
market access
22
market accessTO BE:
Medicines + companion tests:
6 months + suspensions
Tests: first: WG Clinical Biology, then:
minimum 18 months
TMCMedico-
mutCBC Ins Com Minister RD
CRM Minister MD
Concept: 1 joint HTA to assess both drug + test
– diagnostic tests: • new “generic” nomenclature codes for molecular biology tests linked to a drug
• 3 levels of complexity and reimbursement
• defined by TMC, published by royal decree
• list with biomarkers, updated monthly by CRM
– medicines:• “personalised” drugs with “companion list”
23
market access
24
market access
implementation of NGS in routine analysis in Belgium
• create a reimbursement for NGS
• ensure uniform high-quality NGS analysis in Belgium
– creation of NGS expertise platforms
– monitoring and validation of quality and results
• within the limits of the available resources and within an ethical framework
– start in controlled setting
– limited budget impact
Van Valckenborgh et al (2018) Archives of Public Health (76): 49
25
market access
PITTER and NGS registries @ Healthdata.be26
real world evidence
