Roberta Joppi - Horizon Scanning

Health Technology Assessment
Drugs in development…
development…
According
to
the
Pharmaceutical
Research
and
Manufacturers of America (PhRMA
(PhRMA)) more than 2700
new molecules for 4600 indications are currently
under development.
[http://www.phrma.org/files/2008%20Profile.pdf. Accessed Dec. 2008]
Italian Horizon Scanning Project
Roberta Joppi, Luca Demattè,
Demattè, Anna Michela Menti,
Menti,
Daniela Pase,
Pase, Chiara Poggiani,
Poggiani, Luigi Mezzalira
27%
Phase III
26%
Phase I
40%
Phase III
17%
Phase I
HTA is an important tool for information support
to decision makers.
– Pharmaceutical Department – Verona LHU
THE MANAGED INTRODUCTION OF NEW MEDICINES
PIPERSKA course Glasgow 9 – 11 July 2009
Health Technology Assessment
As HTA as an activity became more common
practice around the world, it was increasingly
recognized that timeliness of the assessment was
key in the support of healthcare decision makers.
47%
Phase II
EU
World
43%
Phase II
[http://clinicaltrials.gov; last access 17 June 2009]
Henshall c et al. Priority setting for Health Technology Assessment. Theoretical considerations
and practical approaches. Int J Technol Assess Health Care, 1997; 13:144-185
Early Warning System
Banta and Gelijns were the first to conclude that it is not
satisfactory to react to technological developments only
when confronted with their consequences. Their study
for the Dutch government in the 1980s called for a
systematic approach to identification and early
assessment of new health technologies to provide early
notice to decision makers in health care.
An Early Warning System was subsequently established
at the Dutch Health Council
Mowatt G et al. Implementing early warning messages on emerging health technologies.
Int J Technol Assess Health Care. 1998;14:663-70
HTA is a form of policy research that studies the
short- and long-term consequences of the
introduction of health technologies in a healthcare
system, in terms of safety, clinical benefit, costs
effects on organization of health services, and
legal, social, and ethical consequences.
Host organization:
organization: Local Health Unit,
Unit, ULSS 20 in Verona
Banta HD, Gelijns AC. An early system for the identification and assessment of future health care
technology: The Dutch STG project: Int J Technol Assess Health Care. 1998;4:607-12
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Aims
TO ORGANIZE and EVALUATE available information on emerging drugs
BEFORE SUBMISSION of a MAA to Regulatory Agency and before any
decision on COSTS and POSSIBLE CLASS OF REIMBURSEMENT
IHSP-SC
Methods
Organization Structure
IHSP
Scientific Committee (SC)
Database Team (DT)
Evaluation Team (ET)
- Representatives of Ministry of Health
- Representatives of the Veneto Region
- IHSP promoters belonging to the Verona Local Health Unit
- Experts in drug evaluation concerning innovation and healthcare impact
IHSP-SC RESPONSABILITIES:
 to
Specific aims:
aims:
 to produce periodical lists of emerging drugs for which a MA will be
expected within 12-36 months
Data Sources
 to evaluate potential clinical impact and cost effectiveness in terms
of healthcare and cost for National Health Service
 to give well-timed information to improve regulatory decisions about
emerging drugs
IHSP Database
 to identify further research fields needed to be investigated
IHSP-DT
The IHSP Database Team (IHSP-DT) includes three part-time and three full time
pharmacists, three part-time IT people, one part-time administrative employee.
Regulatory Agencies
Medical-scientific literature
Scientific databases
Medical websites/Press-releases
Pharmaceutical Bulletins
Data Collection
Check
Reports
Discussion Forum
IHSP-ET
- Panel of 50 clinicians with expertise in different medical and surgical fields
- Scientific Secretary including six pharmacists (three part-time and three full-time
IHSP-DT TASKS:

to set up, maintain and update the database

to guarantee the confidentiality of the stored data

to collect information


to produce the - 36 months lists and -18/ -12 months reports of
emerging medicines
to produce any possible additional document useful to the IHSP-SC
and/or to the Evaluation Team.
people) and a part-time administrative employee.
IHSP-ET TASK : to produce the New Product Information Report
establish and approve Standard Operating Procedures to handle the
collected information
 to
prioritise drugs
 to
sign up experts to be involved in the assessment of prioritised drugs
 to
review and approve New Product Information Reports
 to
establish the publication policy on IHSP material
 to
identify therapeutic needs and priority research areas of interest to
the NHS
to establish connections with NHS institutions, with scientific associations
and with international groups

Data Sources
 EMEA - European Medicines Agency
 FDA - Food and Drug Administration
Medical-scientific
literature






Regulatory Agencies
the Lancet
the New England Journal of Medicine
British Medical Journal
JAMA – the Journal of the American Medical Ass.
Annals of Internal Medicine
Others…
 PubMed - US National Library of Medicine
 International Clinical Trials Registry
 ClinicalTrials.gov
 R&D and Clinical Trials Insight
 NeLM – UK National electronic library for medicines
Medical websites/
Press-release




Scientific
databases
BioSpace (www.biospace.com)
WalGreens Health Service
News Release from Drug Companies
Others…
 Scrip World Pharmaceutical News
Pharmaceutical bolletin
2
The IHSP Database
The IHSP Database
Database Stratified Access
Information Input
Database IHSP
Italian Horizon Scanning Unit
(LHU of Verona)
Project Management
Italian Horizon
Scanning Project
CTS-IHSP
IHSP Workflow
Rules for access
Access stratified by profile
Experts
S
E
C
U
R
E
Product classification and description
Brand information
IHSP Database
Regions
MinSal
Licensing information
Efficacy and safety
W
E
B
Burden of disease
Emerging drugs
- 36 months report
S
E
C
U
R
E
PRELIMINARY SELECTION (SC-IHSP)
Selected Drugs
-18 months report
IHSP Database
New Product Information
Report - NPIR
(-12 months M.A.)
Evaluation
Team
PRIORITIZATION
SC-IHSP
W
E
B
Possible price
…
DRUGS
ATC
ICD IX
…
MinSal
REGIONS
Integrated Information
Percentage of drugs grouped by
stage of development in EU
6.74%
Unknown
8.99%
Phase I
34.35%
Phase II
49.92%
Phase III
Drugs in development grouped by ATC
ATC code
(I level)
US+EU
n
EU
n
EU phase II
n (%)
EU phase III
n (%)
L
Antineoplastic and immunomodulating agents
661
228
92 (40,35)
108 (47,37)
N
Nervous system
258
87
33 (37,93)
40 (45,98)
A
Alimentary tract and metabolism
182
64
15 (23,44)
37 (57,81)
J
Anti-infectives for systemic use
167
60
14 (23,33)
33 (55,00)
C
Drug Group
Cardiovascular system
97
49
19 (38,78)
24 (48,98)
B
Blood and blood forming organs
103
46
15 (32,61)
23 (50,00)
M
Musculo-skeletal system
72
25
6 (24,00)
11 (44,00)
R
Respiratory system
56
22
8 (36,36)
9 (40,91)
G
Genito-urinary system and sex hormones
44
16
6 (37,50)
8 (50,00)
D
Dermatologicals
32
13
5 (38,46)
7 (53.85)
V
Various
22
5
0
5 (100)
S
Sensory organs
30
4
1 (25,00)
3 (75,00)
10
3
0
2 (66.6)
H
P
Total
Systemic hormonal preparations, excluding
sex hormones and insulins
Antiparasitic products, insecticides and
repellents
8
1
0
1 (100)
1,742
623
214
311
72.78%
Source:: IHSP database, Update on May 2009
Source
Eur J Clin Pharmacol 2009 DOI 10.1007/s00228-009-0666-z
3
IHSP Workflow
IHSP Workflow
Database IHSP
Emerging drugs
- 36 months report

PRELIMINARY SELECTION (SC-IHSP)
New Product Information
Report - NPIR
(-12 months M.A.)
Selected Drugs
-18 months report
Evaluation
Team
PRIORITIZATION
-36 MONTHS
REPORT
Produced annually
SC-IHSP
Emerging drugs
- 36 months report
company
ATC Group
...

licensee

stage of development

possible submission date of the MAA

main proposed indication(s)

ongoing studies
PRELIMINARY SELECTION (SC-IHSP)
Selected Drugs
-18 months report


general information
company
ATC Group
route of administration
……
possible submission date of the MAA
proposed indication(s)

summary of the available data on clinical
efficacy and safety
PRIORITIZATION
IHSP Workflow

overview of all ongoing trials
completed studies not published

possible price and economic impact (if available)
alternative(s) alrealdy on the market

possible competitors in development
REGIONS
Priority-setting criteria used by SC-IHSP
AREA to INVESTIGATE
Database IHSP
PARAMETERS
EVALUATION
Burden of disease
Emerging drugs
- 36 months report
Selected Drugs
-18 months report
Evaluation
Team
SC-IHSP
PRELIMINARY SELECTION (SC-IHSP)


New Product Information
Report - NPIR
(-12 months M.A.)
MinSal
drug/brand name
active substance
Produced every 6
months
general information
REGIONS
MinSal
-18 MONTHS
REPORT
Database IHSP
drug/brand name
active substance
Epidemiology
Rare
Not rare
Severity
Severe
Not severe
Duration
Acute
Chronic
Treatment
A v a i l a b l e Absent
Patient impact
New Product Information
Report - NPIR
(-12 months M.A.)
Evaluation
Team
PRIORITIZATION
and
NHS
Efficacy vs. current treatments
(mortality, morbidity, quality of life,
H i g hetc.)
er
Equal or Lower
Safety vs. current treatments
Higher
Equal or Lower
Compliance vs. current treatments
Higher
Equal or Lower
Social impact (Media, patients associations, lobbies ...)
YES
NO
Service reorganization and/or staff training required
YES
NO
Economic impact on the NHS
High
Low
Possible launch date
< 18 months> 18 months
Drug in development for other indications of interest
YES
NO
Other drugs in development for the same indication
YES
NO
Pressures
SC-IHSP
Others
MinSal
REGIONS
4
New Product Information Report
(-12 months to M.A.)
IHSP Workflow
active substance
brand name
company
ATC Group
dosage
development state
...
Database IHSP
 General Information
Emerging drugs
- 36 months report
PRELIMINARY SELECTION (SC-IHSP)
Selected Drugs
-18 months report
New Product Information
Report - NPIR
(-12 months M.A.)
Evaluation
Team
 Clinical need and burden of disease
NPIR
PRIORITIZATION
 Summary of efficacy/safety data
from available clinical trials
“drug name”
“drug indication”
SC-IHSP
 Ongoing Clinical trials for the same
or other indication(s)
 Clinical critical assessment
MinSal
REGIONS

Social/economic impact
Source:: IHSP
Source
Prasugrel transferability model
Clopidogrel-based
Clopidogrel-based
estimation of
prasugrel utilization
2007
EUROSCAN
August 2006
IHSP kickoff
IHSP chronology
2008
EuroScan
2009
Today
Database set up
- 36 reports
-
20
33
- 18 reports
-
39
23
NPIR
-
9
8
Total IHSP documents produced (n): 132
Eur J Clin Pharmacol 2009
DOI 10.1007/s00228-009-0666-z
In 1999 several Horizon Scanning Systems (HSS)
established EuroScan, an information network on new and
changing health technologies.
The network currently consists of 21 representatives
(Canada, Denmark, Norway, Sweden, Australia, New
Zealand, The Netherlands, The United Kingdom, Israel,
Spain, France, Switzerland, Germany, Ireland, Austria,
Italy, Finland).
Any HSS is a non-profit organization with at least 50%
funding from public sources
Douw K, Vondeling H. Selection of new health technologies for assessment aimed at informing decision
making: A survey among horizon scanning systems. Int J Technol Assess Health Care. 2006;22 (2):177-83
5
Since November 2008, IHSP is a member of:
EuroScan vs IHSP Database Activity
to evaluate and exchange
information
100%
to share methodology
technologies
80%
to develop the sources of
information
60%
191
166
1909
320
40%
616
20%
0%
1
EUROSCAN
to disseminate the information
IHSP – Working Group
2
HORIZON
Other
Settings
Programmes
Procedures
Diagnostics
Devices
Drugs
Pharmaceutical Department
Verona LHU
Cineca IT-Group
Bologna
Roberta Joppi
Anna Michela Menti
Luigi Mezzalira
Chiara Poggiani
Daniela Pase
Laura Agnoletto
Michela Franceschi
Chiara Roni
Alessandro Azzini
Luca Demattè
Elisa Rinieri
Enrico Aiello
Maria Teresa Marano
Eugenia Rinaldi
Elisa Rossi
database
Thank you for your attention!
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