Information Day Leeds, UK 27 October 2015 Treating and managing disease

Transcription

1 The societal challenge 'Health, demographic change and well-being' Work programme Information Day Leeds, UK 27 October 2015 Treating and managing disease Image credit: Adam Fagen Rallying for medical research / Creative commons usage license

2 Understanding the call topics: example 'PHC ' 'Specific challenge' 'Scope' 'Expected impact' 'Type of action'

3 SC1 PM New therapies for rare diseases Rare diseases Clinical trials on substances where orphan designation has been given by EC (by date of call closure) and design takes account of EMA assistance Small molecule to gene or cell therapy. May include novel interventions, repurposing Feasibility assessment based on clinical / preclinical. Patient organisation, MS heath authorities, efficacy / clinical benefit, health economics to be included Must follow IRDiRC 4-6 million (60)

4 200 therapies and means to diagnose most rare disease by Research and Innovation

5 Over 40 Committed members Europe Academy of Finland (FI) E-RARE 2 Consortium (EU) European Commission (EU) European Organisation for Treatment & Research on Cancer (BE) EURORDIS (EU) BioMarin Nederland B.V (NL) French Foundation for Rare Diseases (FR) French Muscular Dystrophy Association (FR) French National Research Agency (FR) Children's New Hospitals Management Group (GE) German Federal Ministry of Education and research (DE) Italian Higher Institute of Health Research (IT) Italian Telethon Foundation (IT) Lysogene (FR) Netherlands Organisation for Health Research and Development Prosensa (NL) Spanish Carlos III Health Institute (ES) UK National Institute for Health Research (UK) rtguest/fotolia.com Australia National Health and Medical Research Council Asia BGI (CN) Chinese Rare Disease Consortium (CN) Japan Agency for Medical Research and Development (JP) Korea National Institute of Health (KR) Saudi Human Genome Project (SA) WuXi AppTec (CN) Research and Innovation North America Canadian Institutes for Health Research (CA) Genome Canada (CA) FDA Orphan Products Grants Program (US) Genetic Alliance (US) Genzyme (US) Isis Pharmaceuticals (US) Mendelian Disorders Genome Centres (US) National Centre for Translational Sciences (US) National Cancer Institute (US) National Institute of Neurological Disorders and Stroke (US) National Institute of Arthritis and Musculoskeletal and Skin Diseases (US) National Institute of Child Health and Human Development (US) National Eye Institute (US) NKT Therapeutics (US) NORD (US) Office of Rare Diseases (US) PTC Therapeutics (US) Pzifer (US) Sanford Research (US) Shire (US)

6 SC1 PM New therapies for chronic diseases Chronic diseases (including chronic infectious) Not rare diseases or regenerative medicine Clinical trials on proof of concept of clinical safety and efficacy of novel therapies or repurposing. To include feasibility assessment. Feasibility assessment based on clinical / preclinical. Take into account gender and age Take into account patient views where relevant 4-6 million (60)

7 SC1 PM Comparing the effectiveness of existing healthcare interventions in the adult population Compare currently available preventive or therapeutic interventions in adults No restriction on diseases or interventions BUT preference for those with high public health and socioeconomic impact Cost effectiveness must be included. Randomised controlled trials, pragmatic trials, observational studies, large scale databases and meta analyses may be considered for this topic. Gender and socioeconomic elements affecting health equity should be considered. 4-6 million (40)

8 SC1 PM /17 Clinical research on regenerative medicine Any stage clinical work can be proposed, later stages preferred. Phase I proposals must present preclinical and toxicology data. Later stage proposals must present appropriate preliminary results. Must include authorisation or provide evidence of regulatory engagement and that authorisation is imminent. Preference to be given for those with approval in place. Any disease or condition, justification of choice to be provided. Demonstration of novelty vis a vis existing treatments required. Gender to be taken into account where appropriate, as well as previous projects (see PP). 4-6 million (30/30)