"Reaching a public audience on medicines development", presented by Anna Zawada, Agency for Health Technology Assessment at EUPATI 2014 Workshop on 2 April 2014 in Warsaw

1. Anna Zawada
Agency for Health Technology Assessment (AOTMPol), Poland
EUPATI 2014 Workshop, 2 April 2014, Warsaw
Welcome
The project is receiving support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n 115334, resources of which are composed
of financial contribution from the European Union's Seventh Framework Programme (FP7/2007-2013) and EFPIA companies.

2. EUPATI 2014 WORKSHOP
Reaching a public audience
on medicines development
Anna Zawada a.zawada@aotm.gov.pl
Agency for Health Technology Assessment (AOTM)
Warsaw, April 2nd, 2014

3. 3
Welcome
In the name of the President of Agency for Health Technology
Assessment in Poland (AOTM, www.aotm.eu),
dr Wojciech Matusewicz,
I would like to warmly welcome all respectable guests gaethered
here for EUPATI 2014 Workshop
AOTM recognizes patients awareness of therapeutic innovations as
real added value and appreciates patients involvement in the process
of development of innovative drugs as well as the process of making
decisions on their reimbursement of public funds.
We believe that incorporationg well informed patients into the process
of health technology assessment is crutial.
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

4. 4
AOTM has been active in Polish health care
system till 2005/6, first recommendations being
provided in 2007
2005
(in line with
Dir 89/105/EEC)
Launching
AOTM by
ordinance
of MoH
coverage/
desinvestment
rules
June 2009
Act on Health
Care Benefits
Procedure of
public
consultations
01 Jan 2012
Reimburse-
ment Act
in the process
2014
Reimbursement
Act
update
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014
First opinion from patient organization being taken
into account in process of drug recommendation –
in January 2008

5. What are the possibilities of patients
involvement in AOTM works?
• AOTM HTA Guidelines as well as binding law enforce taking
into account „patient perspective” in HTA analysis
• In the analytical process the opinions of patient organizations
on drug assessed are sought
• Representatives of patients organizations may actively ask for
the invitation to Transparency Council meeting to provide their
opinion on drug assessed
• Patients Right Ombudsman representative is a member of
Transparency Council, present at every meeting
• Patients, among other stakeholders, may provide their
opinions on specific drug in the process of public
consultations
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014 5

6. So, if it is so good, why it is so bad?
• In public consultations huge part of analysis is coverd
black as containing (manufacturer) confidental
information
• Patients opinions are not relevant to HTA process. In
general patients – as well as general public – know little
about HTA and the role of AOTM
• Few patients are well prepared to the discussion, eg. can
provide characteristic of patients which may benefit the
most of the treatment
• Important: statements above don’t mean blaming
patients! It is rather the list of tasks to be done, by AOTM
among others, to improve patients role in the process of
making reimbursement decisions
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014 6

7. Place of AOTM in Polish system 2013
7
Patients
Healthcare providers (eg.
hospitals, GP practices)
NHF
Contracts with providers
Payer
Reimbursement decisions
MoH
Decision-maker
AOTM
MAH
submission
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014
Transp
Council

8. Current means of funding drugs
in Polish healthcare system
1. On the reimbursement list – drugs to be distributed by pharmacy on
the basis of registered indications
2. On the list of drugs to be funded under „regimen (drug) programs”
(designed for defined group of patients, tightly defined inclusion/
/exclusion criteria, careful monitoring; drug programs cover new,
expensive therapies)
3. On the catalogue of chemiotherapeutics delivered in hospital care
in oncology
4. On the reimbursement list of drugs funded in specific off-label
indictions
5. A few specific MoH therapeutic programs (eg. clotting factors for
haemophilia; in-vitro insemination for infertility)
6. On dedicated demand for individual patients (special cases of
chemiotherapy or even drugs not approved for Polish market)
8EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

9. Who applyes for coverage?
• In case of reimbursement on list, under drug program or
in the catalogue of chemiotherapy – MAH (Marketing
Authorisation Holder = manufacturer) should initiate the
process
• In case of the list of off-label use – MoH (Minister of
Health) initiates process by asking National Consultants
in specific medical domains to indicate drugs and their
off-label indications
• As for individual approval – MoH may ask AOTM
to assess specific technology in case the number of
demands exceeds the limit; negative recommendation
causes refusal
9EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

10. Proceeding with MAH submission
in Ministry of Health
• Checking formal completness of the submission
• If reimbursement under „drug program” – program
inclusion/exclusion criteria to be agreed
• If active substance not currently reimbursed, HTA
analysis compulsory; they should be provided to AOTM
(together with the assessment fee)
• After AOTM recommendation delivered – price and risk
sharing agreement to be negotiated with Economic
Commission in MoH
• Reimbursement decision made by MoH; only an appeal
to the court possible
10EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

11. Process of assessment of HTA analysis
of reimbursement dossier
11
Minimal
require-
ments
checking
MAH
supple-
ments
HTA
analysis
(optional)
Critical
assessment of
technology on
the base of
MAH analysis
Public
consul-
tations
Transp
Council
position/
/President
recommen-
dation
14 days 7 days
60 days (+14 days)
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

12. Problems with drug reimbursement in
Polish health care system
• In Poland there are no specific way of proceeding the
reimbursement process for orphan drugs
• According to reimbursement Act the threshold for
economic effectiveness is stiff: 3 x GDP per capita (in
2013 about 25 000 euro) with no exception for orphans
• Manufacturer may improve economic effectiveness
proposing risk sharing sheme (RSS) – eg. decreasing
the real price in confidential agreement with Ministry of
Health
• Transparency Council/AOTM President are not binded
by the threshold and may consider special patients
needs when shaping recommendation
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014 12

13. 13
Stakeholders involvement
in HTA assessment process
• Ministry of Health – originator of the process, recipient of
recommendations, by law independent decision maker
• National Health Fund – payer, involved in the process as consultee
and data provider; has the representative in TC
• Professionals – their opinion are asked by AOTM in the
assessment/appraisal process
• Patients – if organized in association, may be involved in
assessment/appraisal process as professionals; Patients Rights
Attorney representative in TC
• MAH (producer) – when applying for reimbursement is obliged to
provide HTA analysis and is entitled to comment on the assessment;
no appeal procedure to AOTM recommendations is forseen
• General public – may provide comments to analysis in the public
consultations – however rarely used
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

14. Conclusions
• In Polish system patients have the possibilities to provide
their opinions in HTA/reimbursement process
• We vastly need educated and well informed patients
being involved in HTA process
• We are looking forward for the effects of EUPATI project
promoting patients consciousness of innovative
therapies and their value in real therapeutic practice
14EUPATI 2014 Workshop, Warsaw, April 2nd, 2014

15. 15
Thank you for your attention
Anna Zawada
a.zawada@aotm.gov.pl
EUPATI 2014 Workshop, Warsaw, April 2nd, 2014