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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