Transcript Workshop on Orphan Drugs

Rare Diseases Task Force
Workshop on Orphan
Drugs
www.rdtf.org
30 May 2007
Working group on standards in health care
 WG1: WG on centres of reference
– Publication of the 2006 Report
– Dissemination through the Newsletter
– Through the High Level Group on Health Services
and Medical Care members
 WG2: WG on access to treatment
– ? How many drugs for how many patients
– As an contribution to the debate on affordability
Workshop on Orphan Drugs
 Topic: Forecast for Orphan Drugs and
eligible Rare Diseases Patients
 Place: Paris, 30 May 2007
Participants
 RDTF: S. Aymé, A. Kole, C. N. Ruperto, AR.
Stefanov, D. Taruscio
 DG Public Health: K. Freese
 EMEA: Jordi Llinares Garcia
 Experts:F. Bignami, C. Delval, F. Faurisson, R.
Salonen, C. Smit, S. van Weely
 Industry: Baxter, HRA Pharma, Orfagen, Orphan
Europe, Genzyme
Outcome of the workshop
 Necessity to publish overview of current
situation regarding treatment of rare diseases
– Type of diseases target for drug treatment
– Proportion treated so far
 Necessity to provide a forecast of orphan
drugs
– Based on the model established with the US
designations
– Applied to the European situation
• Based on EU designations
 Necessity to communicate differently
Orphan Drug Designations (449)
Designation
solid tumors
oncohaematology
neurology
transplantation
inflammation
infectious diseases
cystic fibrosis
pneumology
cardiovascular
endocrinology
metabolic
lysosomal
hepato-gastro
ophthalmology
dermatology
hematology
muscular dystrophies
toxicology
Number
106
79
31
26
24
23
22
19
18
17
16
14
12
10
9
9
6
6
Percent
24
18
7
6
5
5
5
4
4
4
4
3
3
2
2
2
1
1
Orphan Drug Designations (449)
Product Type
Number
Percent
chemical
261
58
biotechnology
136
30
cellular therapy
15
3
natural
13
3
oligonucleotides
12
3
gene therapy
12
2
Expert opinions
 75% of products examined by Companies
would be OD
 Success rate is 15%
 Booster of innovation/ celebration of
successful science/ Good investment
 Share funding between Industry and public
sector
 200 new Orphan Drugs in 2017
– Replacement/ not cumulative
 Smaller compounds are coming
 Cell therapy will be difficult to commercialize
 Emphasize on equity and solidarity
Plans for the future
 Preparation of a new report on Centres of
reference in Europe
– on the methods to assess the added-value of CR
– and of the added-value of Reference networks
– Second semester 07
 Establishment of an updated version of the
Inventory of national incentives in the field of
rare diseases
– Annual report
 Preparation of the RDTF work programme
2008-2010
– Emergency guidelines / personal cards
– List of essential tests