1. www.eurordis.org
EU INITIATIVES
TO IMPROVE ACCESS TO CARE
FOR RARE DISEASE PATIENTS
Christel Nourissier
General Secretary, EURORDIS
Balkan Congress Rare Diseases_26-27 June 2009
3. EU legislations stimulating R&D
and improving access to treatments for rare diseases
⢠The EU Regulation on Orphan Medicinal Products in 1999 (> 2000)
⢠The EU Regulation on Pharmaceutical Products in 2003 (> 2004)
⢠The EU Regulation on Paediatric Drugs in 2006 (> 2008)
⢠The EU Regulation on Avanced Therapy Medicinal Products in 2007
(> January 2009)
3 Cluj-Napoca,26-27 June 2009
4. Member States have EU common procedures for:
⢠ODD= Orphan Drug Designation: Committee for Orphan Medicinal
Products, COMP/EMEA
⢠PA= Protocol Assistance: Scientific Advice Working Party,
SAWP/CHMP/COMP/EMEA
⢠PIP = Paediatric Investigation Plan: Paediatrics Committee, PDCO/
EMEA
⢠CAT = Committee for Advanced Therapies
⢠MAA = Marketing Authorisation Application: Committee for
Medicinal Products for Human Use, CHMP/EMEA
- Revision of orphan designation criteria at MAA
(including Significant benefit): COMP/EMEA
⢠Post-MA obligations, additional studies, follow-up registries
assessed by CHMP/EMEA
4 Cluj-Napoca,26-27 June 2009
5. Availability from the patientsâ point of view
Designation, Marketing Authorisation : European process
EMEA
EU
National availability for patients ?
5 Cluj-Napoca,26-27 June 2009
6. A new policy base to improve availability
⢠The EU Pharmaceutical Forum Conclusions & Recommendations
(adopted November 2008) :
ď§ Guiding Principles on ÂŤ Improving Access to orphan medicines for all
affected EU citizens Âť
ď§ Guiding Principles on Relative Effectiveness
⢠The Commission Communication on Rare Diseases, Chapter 5.3
ÂŤ Access to Orphan Drugs Âť (adopted december 2008)
⢠The Council Recommendation on Rare Diseases, Chapter 5
ÂŤ Gathering the expertise on rare diseases at the European
level Âť 5.5 ÂŤ therapeutic or clinical added value of orpahn drugs Âť
(adopted June 2009)
6 Cluj-Napoca,26-27 June 2009
7. Key Facts & Values relevant to availability
⢠Rarity of patients
⢠Scarcity of expertise & knowledge
⢠Overeaching values of universality,
access to good quality care, equity,
solidarity, social justice
7 Cluj-Napoca,26-27 June 2009
9. Inequalities of access: a Europeâs Challenge
⢠EURORDIS Surveys on Availability of all Orphan Medicines
across all EU Member States, performed in 2001, 2003, 2005, 2007
⢠Major inequalities of patient access between Member States,
between regions inside a Member State, between hospitals
⢠Slow placing on the market, can take several years for life saving
drugs; no placing on the market for some medicines:
ď§ No relation between the delay of placing on the market & patient
availability, and the value of the drug for patients treatment
ď§ Responsibilities of delays are on both sides: (a) Member States which
either donât have the expertise on these rare diseases or just wish to save
money (b) Pharmaceutical & Biotech companies which donât have the
ressources to apply to over 27 different procedures in a short period of
time or ar not interested by some too small national markets
9 Cluj-Napoca,26-27 June 2009
10. Available OMPs by country (source 2007)
Number of available OMPs
20-21
15-19
10-14
5-9
0-5
10 Cluj-Napoca,26-27 June 2009
11. 2001
2002
2003
2004
2005
r
ar
ob
ot
Ph lt
ia
Pr m
re
Xy tio
a
ev r
R va
a
ex n
N dre
Availability by product
so
Ly id
gr
Xa din
Number of countries
a
rf
O
k
ta x
Li lve
i
us s
B avi
nt l
Ve aga
l t
ep r
R ave
m x
So no
e
is a
T r sc e
v e ym
Za raz
u
ld r
A lee e
ac m
11 Cluj-Napoca,26-27 June 2009
Tr azy
br
Fa c
e
liv
G in
ilz
W a
de
Pe
30
25
20
15
10
5
0
12. Averaged OMP price compared to European mean price
Ex factory price Users price
>110 %
105-109 %
100-104 %
95-99 % d
90-94 %
< 94 %
12 Cluj-Napoca,26-27 June 2009
14. The European Solution:
From Agreed PrinciplesâŚ
⢠New agreed principles (EU Pharma Forum):
ď§ ÂŤ Member States, stakeholders and the Commission should
strenghten their efforts to ensure access to orphan medicines
in all EU Member States Âť
ď§ EU Exchange of Knowledge on the Scientific Assessment of
the Clinical Added Value of Orphan Medicines
ď§ Specific Conditional Pricing & Reimbursement mechanisms
ď§ Early dialogue on research & development
ď§ Increased awareness on rare/orphan diseases
14 Cluj-Napoca,26-27 June 2009
15. The European Solution:
⌠to Action and new Good Practices
⢠New practices at EU Level (Commission Communication &
Council Recommendation):
ď§ European Collaboration for the Scientific Assessment of the
Clinical Added Value of Orphan Medicines
ď§ Creation of a Working Party to produce European Scientific
Common Assessment Report on this Clinical Added Value
ď§ Regular revision & update of clinical added value reports
based on post-MA data from real life studies
15 Cluj-Napoca,26-27 June 2009
16. National Strategies or Plans on Rare Diseases:
from EU agreed principles to concrete measures
⢠New practices at national level, including new EU Member states,
in the next national strategies & plans on rare diseases 2009-2013
(and recommended to all European countries):
ď§ Commitment to take active part in this new EU collaboration
ď§ Commitment to use the European Common Assessment
Report to speed up its national decision making process and
to do its appraisal on Pricing & Reimbursement
ď§ Promote Conditional Pricing & Reimbursement with regular
revisions, based on revised & updated Reports
ď§ National payment, even when prescribed and distributed in
hospitals
16 Cluj-Napoca,26-27 June 2009
17. Why Romania and Balkan countries should be
willing to implement these agreed principles and
new practices?
⢠Because Romania and Balkan countries have taken part to the
development of these new principles and have agreed to them
(EU Pharma Forum and Council of Health Ministers of the
European Union)
⢠Because Romania and Balkan countries would:
ď§ Benefit from quicker, better quality and more consistent assessment
thanks to their collaboration with other Member States: scientific and
medical data on which clinical added value is assessed are the same and
are valid across Europe
ď§ Coordinate their post marketing requirements with other Member States,
asking for real life studies or registries for instance, with higher chances of
obtaining more rapidly more reliable data
ď§ Be able to do have better value for its money when paying for orphan
medicines thanks to (a) better medical practices & prescriptions (b) taking
advantage of conditional pricing & reimbursement (c ) better targeted
patients responsive to the treatment within the therapeutic indication
17 Cluj-Napoca,26-27 June 2009
18. www.eurordis.org
Yann Le Cam Fabrizia Bignami François Houÿez
Chief Executive Therapeutic Development Health Policy
ylecam@eurordis.org fbignami@eurordis.org fhouyez@eurordis.org
Flaminia Macchia Anja Helm Paloma Tejada
European Public Affairs Relations with Patient Groups Communications
fmacchia@eurordis.org ahelm@eurordis.org ptejada@eurordis.org
18 Cluj-Napoca,26-27 June 2009