1
Treaty establishing the European Community (EU-6)
Signing Ceremony in Rome, Campidoglio - March 25, 1957
2
AIFA – The Italian Medicines Agency
Marketing
Authorisation
GMP-GCP
Inspections
Pharmacovigilance
Pricing &
Reimbursemnt
Governanance and maintenance
Clinical trials
of the economic ceiling
Independent
research
Drug
Communication
3
The key (common) issues
• The assessment process: how safe and
effective a medicine is
• Pricing & Reimbursement: which
patients will benefit most
• The transferability of the b/r assessment
into in the real life
• Public funding of drug research for a
better decision making process: an
impossible task for National Agencies?
4
Problems coming from
the European MA Process
• Place in therapy of newly licensed drugs
• ATV (Added Therapeutic Value) assessment
for new drugs
5
Source: J. Avorn, Circulation 2006
Innovation and cost-effectiveness
The case of new anticancer drugs
• Poor predictivity of “responders”
• Patients with disease progression
failures”) identifiable only at follow-up
• Reimbursement of innovative drugs as
a function of their “real” effectiveness
Sharing Innovation – Risks - Costs
6
National Registry of new anticancer drugs
Distribution of patients treated
by drug and gender
N. of Patients: 1586
Drug
AVASTIN
Female
N.
Male
N.
Total (F+M)
N.
Total (F+M)
%
77
99
176
11.1
221
290
511
32.2
7
7
14
0.9
ERBITUX
149
203
352
22.2
FASLODEX
434
0
434
27.4
FOSCAN
0
2
2
0.1
GLIADEL
6
9
15
0.9
TARCEVA
24
37
61
3.8
ZEVALIN
12
9
21
1.3
930
656
1586
100.0
ELOXATIN
EMEND
Total
8
http://oss-sper-clin.agenziafarmaco.it/
9
Ethics Committes in Italy:
309
4
2*
10
24
61*
OSSERVATORIO
NAZIONALE
Ministero della Salute
8*
18
12
14
13
1*
5
4
35
18
26
4
10
14
30
10
Clinical Trials per Phase
Phase
Phase III
Phase II
Phase IV
Bioeq / Biod
Phase I
Totale
Nr. CTs
%
1.899
1.198
306
70
54
3.158
53.8
34.0
8.7
2.0
1.5
100
11
CTs per Year and Phase
Year
2000
2001
2002
2003
2004
2005
Total
Nr. CTs
%
%
%
%
%
Phase III
Phase II
Phase IV
Bioeq
/
Bioav
Phase I
560
610
565
565
607
620
61,6
53,8
52,4
53,7
53,2
49.5
27,5
33,5
36,4
35,4
35,1
36.0
7,9
8,8
7
7,7
8,1
9,8
2,1
3,1
2,3
1,3
2
1,1
0,9
0,8
2
2
1,5
2.3
3.158
54,5
33,9
8,1
2,1
1,5
12
Clinical Trials per Therapeutic Classification
Therapeutic branch
CT
%
1,055
27.5
Cardiology/Vascular diseases
443
11.5
Immunology and infectious diseases
363
9.5
Neurology
311
8.1
Gastroenterology
272
7.1
Endocrinology
224
5.8
Respiratory system diseases
163
4.2
Nephrology/Urology
129
3.4
Hematology
128
3.3
Psychiatry/Psychology
108
2.8
Musculo-skeletal system diseases
105
2.7
Ophthalmology
98
2.6
Dermatology/Plastic surgery
85
2.2
Rheumatology
82
2.1
Gynecology and obstetrics
70
1.8
Genito-urinary system
66
1.7
Anesthesiology
55
1.4
Pharmacology/Toxicology
32
0.8
Pediatrics/Neonatology
23
0.6
Otolaryngology
14
0.4
Traumatology and emergency medicine
7
0.2
Odontology and maxillofacial surgery
6
0.2
3,839
100.0
Oncology
Total
13
The Italian Clinical Trials Registry
The key issues
• The general sense and direction of research
• Which diseases are investigated by which
drug(s) and strategy
• The degree of innovative research in real life
14
Independent Research
AREAs
• Orphan drugs and rare desease
• Head to head clinical trials
• Pharmacovigilance and appropriateness
Promoted and funded by AIFA
15
Aims of the conference
To discuss
The best way to implement evidence in
regulatory decisions
To consider
Licensing, pricing, reimbursement and impact
on population health
To share
Experiences and points of view
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17
18
19
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