Transcript The aims of the registry

Negoziazione e rimborsabilità:
strumenti per la sostenibilità?
Francesco Perrone
Director Clinical Trials Unit
National cancer Institute of Naples
Italy
Preamble
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I’m a medical oncologist
External advisor for AIFA from 2005 to 2012
…
I’m not delegated by AIFA
The things I’m going to say represent my
personal views
AIFA
• Was born in 2004
• General directors
– Nello Martini (2004-2008)
• I thanks him for some slides…
– Guido Rasi (2008-2011), current EMA director
– Luca Pani (from 2011)
The Italian scenario, few years ago
• The context
– An advisory group focused on cancer drugs was created by
Dr. Martini in 2005
– The only available infrastructure was the “price
commission”
– Many new drugs were in the “waiting list” and their cost
was very high…
• The needs
– To make drugs available shortly and without discrimination
– To build a link between benefit size and reimbursment
– To look for “easy to establish” and “not expensive”
solutions
The proposal was to build an
impressive machinary …
The Italian Registry…
• In 2005 the IT partner was CINECA
– the major Italian computer center, a non-profit consortium including
70 Universities, 5 research bodies and the Ministry of Instruction,
University and Research
• In 2012, the technological partner changed
– ACCENTURE a multinational management consulting services private
company, the world's largest consulting firm as measured by revenues
and a Fortune Global 500 company
• In 2015, the technological partner changed again…
– ALMAVIVA an Italian company based in Rome, 13,000 employees in
Italy and 32,000 abroad, the 6th private Italian Group in terms of
employees worldwide and the 3rd based on business management
The Italian Registry…
• The first version of the Italian Registry
(working well) was closed on Dec 31, 2012
• And I will show you some data downloaded on
Dec 30, the “day before”…
1.507
units treating cancer
35 “new” drugs
215.464
patients
The aims of the registry
• To introduce new drugs being sure that their
use was appropriate (tutorial function)
• To provide an informatic platform to build a
bridge between clinical practice and
reimbursement strategy
• To provide the scientific community (and the
patients) with solid evidences on drug
consumption in clinical practice
The aims of the registry
• To introduce new drugs being sure that their
use was appropriate (tutorial function)
• To provide an informatic platform to build a
bridge between clinical practice and
reimbursement strategy
• To provide the scientific community (and the
patients) with solid evidences on drug
consumption in clinical practice
227.792216.897=
----------10.895 (5%)
patients who were avoided
an inappropriate treatment
The aims of the registry
• To introduce new drugs being sure that their
use was appropriate (tutorial function)
• To provide an informatic platform to build a
bridge between clinical practice and
reimbursement strategy
• To provide the scientific community (and the
patients) with solid evidences on drug
consumption in clinical practice
The bridge
• In clinical practice we are able to measure
success/failure
• Reimbursement strategies that link payment
to the success (lack of failure) of the
treatment can be put in place …
– Payment by result (based on individual outcome)
– Risk sharing (based on individual outcome)
– Cost sharing (based on trial results and checked in
real world)
MEAs : Payment by results - Cost Sharing
Risk sharing – Success Fee
Registries
MEAs
34
PbR- Payment by Results
19
CS - Cost Sharing
2
RS- Risk Sharing
1
SF – Success Fee
40 drugs x 56 indicazioni
(1) Cost sharing = payback of 50% treatment for all patients at the follow up
(2) Payment by results = payback of 100 % treatment for all patients non responders
(3) Risk sharing = payback of 50% treatment for all patients non responders
(4) Success Fee = PbR reversed
Nello Martini
Not only cancer
• Registries have been expanded in Italy to
other disciplines
– Orphan drugs
– Antidiabetics
– Antirheumatics
– Cardiovascular
– Psoriasis
• End of 2014, 76 drugs for 58 indications
• 505.000 patients
The trajectory of imatinib price
Price/volume means…
Price
Volume
Time (eg years)
The aims of the registry
• To introduce new drugs being sure that their
use was appropriate (tutorial function)
• To provide an informatic platform to build a
bridge between clinical practice and
reimbursement strategy
• To provide the scientific community (and the
patients) with solid evidences on drug
consumption in clinical practice
This was (and still is) a failure
Today
• No publication in scientific journals with data
from the cancer registries
• Few official reports
• I believe there were (are) interesting data and
many questions might be addressd
• Unofficially, I prepared one slide with data
from the baseline form of the first generation
registry
• …
Age of patients
Drug/indication
Registry
Registration trial
N
median
N
median
Avastin/CRC
27.343
64
2792 (3)
60
Avastin/NSCLC
1.771
61
878
43%≥65
Avastin/breast
4.786
56
722
56
Tarceva/NSCLC
25.567
67
731
61.4
Mabthera/lymphoma
23.976
67
671 (2)
59
Erbitux/CRC
14.773
66
599
61
Alimta/NSCLC_1°_line
10.326
64
1725
61
Alimta/NSCLC_2°_line
3.851
65
571
58
Nexavar/HCC
10.046
70
602
65
Nexavar/kidney
3.639
66
903
58
Sutent/kidney
9.578
66
750
60.5
Thanks to the registry…
• This is the strongest evidence and one of the few
empirical demonstrations that the population
receiving cancer drugs after registration is almost
always older than that enrolled in registrative
trials
• There are many implications…
– Generalizability of registrative trials
– Need for elderly-specific studies
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Safety
Comorbidities
Poly-pharmacotherapy
Possible (but mostly unknown) drug-drug interactions
Actually
• AIFA has established a program of analysis and
publication of registry data in scientific peerreviewed journals
• They have started from non-cancer drug
• The model envisages cooperation with
scientific societies or major stakeholders in
the field
Conclusion… I believe…
• The AIFA experience with MEAS
– began very early
– (in my view) was “useful”
• Successful for tutorial and access aims
• MEAS are “one tool” but not enough with
skyrocketing drug price
• Strategies linking initial price to true drug value
(eg cost-efficacy) need to be implemented at the
EU level to make easier the challenge in single EU
contries
Thank you for your attention
[email protected]