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Rare Diseases Task Force Workshop on Orphan Drugs 30 May 2007.

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Presentation on theme: "Rare Diseases Task Force Workshop on Orphan Drugs 30 May 2007."— Presentation transcript:

1 Rare Diseases Task Force Workshop on Orphan Drugs 30 May 2007

2 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

3 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

4 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

5 Orphan Drug Designations (449) DesignationNumberPercent solid tumors10624 oncohaematology7918 neurology317 transplantation266 inflammation245 infectious diseases235 cystic fibrosis225 pneumology194 cardiovascular184 endocrinology174 metabolic164 lysosomal143 hepato-gastro123 ophthalmology102 dermatology92 hematology92 muscular dystrophies61 toxicology61

6 Orphan Drug Designations (449) Product TypeNumberPercent chemical26158 biotechnology13630 cellular therapy153 natural133 oligonucleotides123 gene therapy122

7 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

8 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 –Emergency guidelines / personal cards –List of essential tests

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