脑部疾病的治疗价值（英文版）.pdf
<p>POLICY WHITE PAPER TOWARDS OPTIMIZING RESEARCH AND CARE FOR BRAIN DISORDERSV TEBC RESEARCH PROjECT - THE VALuE Of TREATmEnT FOR BRAIN DISORDERSEarly InTErVEnTIon: BrIdgIng ThE Early dIagnosIs and TrEaTmEnT gap2the Value Of treatment FOR BRAIN DISORDERSThe European Brain Council (EBC) is a non-profit organisation gathering patient associations, major brain-related societies as well as industries. Established in March 2002, its mission is to improve the lives of those living with brain disorders by advancing the understanding of the healthy and diseased brain through bringing together science and society.3the Value Of treatment FOR BRAIN DISORDERSEBC Final Conference, 22 June 2017, held under the auspices of the maltese EU presidencymUlTIplE goals Target unmet needs to achieve high value for patients Conduct cost-effectiveness analysis in health care (comparing costs and values of defined interventions) and assess the benefits of seamless, coordinated care in the prevention and treatment of brain disorders Propose solutions with a societal impact and reflect on new research developments Release evidence-based policy recommendationsa BoTTom-Up mEThod WITh CasE sTUdIEs daTa analysIs and ConVErgEnCE oF EVIdEnCE To polICy4the Value Of treatment FOR BRAIN DISORDERSTABLE OF CONTENTSAuTHORS PART1 7ACkNOWLEDGEMENTS 8FOREWORD 14 EXECuTIVE SuMMARY 16PART 1: VALuE OF TREATMENT RESEARCH PROjECT OVERALL OBjECTIVES 22 AND POLICY RECOMMENDATIONS BACkGROuND - WHAT ARE BRAIN DISORDERS? 22ABOuT THE VALuE OF TREATMENT PROjECT 231. CONTEXT 241.1. Epidemiology and impact of diseases 241.2. unmet needs and treatment gaps 261.3. Why early intervention is key for brain disorders 281.3.1. Early detection and intervention Potential risk reduction capacity in mental health care 281.3.2. Early detection and intervention Essential in neurological disorders 291.3.3. Addressing stigma 311.4. State of play of research initiatives and public health policies at European level 311.4.1. Medical research and frameworks of action 311.4.2. Public health policies and frameworks of action 352. RATIONALE: VOT RESEARCH METHODOLOGY 365the Value Of treatment FOR BRAIN DISORDERS3. kEY FINDINGS ACROSS CASE STuDIES 423.1. Case studies findings 423.2. Challenges to bridge the treatment gap 543.2.1. Health services challenges 543.2.2. Other challenges: non-adherence to treatment and unaffordable access to care 564. CONCLuSIONS AND POLICY RECOMMENDATIONS 59REFERENCES 68PART2: SuMMARIES OF THE CASE STuDIES 73AuTHORS PART2 74Schizophrenia 75Restless Legs Syndrome 80Parkinsons disease (PD) 84Multiple Sclerosis (MS) 89Stroke 94Normal Pressure Hydrocephalus (NPH) 97Headache 100Epilepsy 105Alzheimers disease (AD) 110REFERENCES 113SPONSORSHIP 1166the Value Of treatment FOR BRAIN DISORDERS7the Value Of treatment FOR BRAIN DISORDERSAuTHORS PART 1:EBC team: Quoidbach V1, Esposito G1, Destrebecq F1, kramer S1EBC academic partners: Tinelli M2, knapp M2, Guldemond N3Working group leaders: Dodel R4, Gaebel W5, kälviäinen R6, kehler u7, Mitsikostas D8, Sørensen PS6, Trenkwalder C9, Vanhooren G10, Visser Pj11EBC executive board: Nutt D1, Boyer P1,5, Di luca M1,12, Oertel W1,41. European Brain Council (EBC)2. London School of Economics (LSE)3. The Institute of Health Policy & Management (iBMG), Erasmus university4. Philipps-universität Marburg5. European Psychiatric Association (EPA)6. European Academy of Neurology7. Dept. of Neurosurgery, Asklepios klinik Altona, Hamburg, Germany8. European Headache Federation (EHF) 9. Paracelsus Elena klinik and university of Goettingen10. European Stroke Organization (ESO)11. Vu university Medical Center Amsterdam12. Federation of European Neuroscience Societies (FENS)8the Value Of treatment FOR BRAIN DISORDERSACkNOWLEDGEMENTSSchizophrenia Working GroupWolfgang Gaebel European Psychiatric Association (EPA) Silvana Galderisi European Psychiatric Association (EPA)Danuta Wasserman European Psychiatric Association (EPA)Cyril Höschl National Institute of Mental Health, klecany, Czech Republic; 3rd Faculty of Medicine, Charles university, Prague, Czech RepublicPavel Mohr European Psychiatric Association (EPA); National Institute of Mental Health, klecany, Czech Republic; 3rd Faculty of Medicine, Charles university, Prague, Czech RepublicPetr Winkler National Institute of Mental Health, klecany, Czech Republic; Health Service and Population Research Department, IoPPN, kings College LondonPatrice Boyer European Brain Council (EBC) and European Psychiatric Association (EPA)Aagje Ieven The European Federation of Families of People with Mental Illness (EuFAMI) Hilkka kärkkäinen Global Alliance of Mental Illness Advocacy Networks (GAMIAN) - EuropePaul Arteel Global Alliance of Mental Illness Advocacy Networks (GAMIAN) - EuropeAmir Inamdar Takeda Development Centre, Europe, Ltd.Meni Styliadou Takeda Development Centre, Europe, Ltd.Christoph Van der Goltz H. Lundbeck A/SVeronica Zilli janssen EMEACristiana Maria janssen EMEABart Malfait janssen-Cilag, Beerse, Belgium kristel van Impe janssen EMEAkarin Becker Boehringer Ingelheim GmbH9the Value Of treatment FOR BRAIN DISORDERSDementia Working GroupPieter jelle Visser Vu university Medical Center; Amsterdam Neuroscience;Sietske Sikkes Alzheimer center, Department of Neurology, Vu university Medical Center; Amsterdam Neuroscience; Department of Epidemiology & Biostatistics, Vu university Medical Center, Amsterdam, the NetherlandsRon Handels Department of Psychiatry and Neuropsychology and Alzheimer Centre Limburg, School for Mental Health and Neurosciences, Maastricht university, Maastricht, The Netherlands; Department of Neurobiology, Care Sciences and Society, Center for Alzheimer Research, Division of Neurogeriatrics, karolinska Institutet, SwedenLisa Vermunt Alzheimer center, Department of Neurology, Vu university Medical center; Amsterdam Neuroscience, Amsterdam, the NetherlandsPhilip Scheltens Vu university Medical Center; Amsterdam Neuroscience;joel Bobula Pfizer IncCharles Faid Pfizer IncAmir Inamdar Takeda Development Centre, Europe, Ltd.Meni Styliadou Takeda Development Centre, Europe, Ltd.Michele Potashman BiogenTresja Bolt BiogenFrancois Chazelle Merck Scharp & Dohme Europe Inc. (MSD) Boris Azais Merck Scharp & Dohme Europe Inc. (MSD) Veronica Zilli janssen EMEAjan Sermon janssen EMEACristiana Maria janssen EMEAHeadache Working GroupDimos Mitsikostas European Headache FederationMatilde Leonardi European Brain Council (EBC) WHO Liaison; Neurology, Public Health, Disability unit- Foundation IRCCS Neurological Institute Carlo Besta, Milan, Italykoen Paemeleire European Headache FederationTimothy Steiner Lifting the BurdenElena Ruiz de la Torre European Federation of Neurological Associations (EFNA)Annik k Laflamme Novartis Pharma AG, Basel, SwitzerlandTeva Pharmaceuticals10the Value Of treatment FOR BRAIN DISORDERSEpilepsy Working GroupReetta kalviainen Epilepsy Center/Neurocenter, kuopio university Hospital and university of Eastern Finland, kuopio, Finland Chantal Depondt ILAE; Department of Neurology, Erasme Hospital, université Libre de Bruxelles, Brussels, BelgiumTony Marson ILAE; Department of Molecular and Clinical Pharmacology, university of LiverpoolAngela Boland Liverpool Reviews and Implementation Group (LRiG); university of Liverpool Rumona Dickson Liverpool Reviews and Implementation Group (LRiG); university of Liverpooljames Mahon Liverpool Reviews and Implementation Group (LRiG); university of LiverpoolPaul Boon European Academy of Neurology (EAN)Philippe Ryvlin European Academy of Neurology (EAN); Epilepsy Alliance Europe; Department of Clinical Neurosciences, CHuV, Lausanne, Switzerlandjanne Martikainen School of Pharmacy, university of Eastern Finland, kuopio, Finland Ann Little European Federation of Neurological Associations (EFNA); International Bureau for EpilepsyPeter Dedeken uCB Biopharma, Brussels, Belgiumnormal Pressure Hydrocephalus (nPH) Working Groupuwe kehler Dept. of Neurosurgery, Asklepios klinik Altona, Hamburg, Germany; International Hydrocephalus Society ISHCSF11the Value Of treatment FOR BRAIN DISORDERSmultiple Sclerosis Working GroupPer Soelberg Sorensen European Academy of Neurology (EAN); Danish Multiple Sclerosis Center, Dpt. Neurology, university of Copenhagen Rigshospitalet, Copenhagen, Denmarkkerstin Hellwig Department of Neurology, St.josef Hospital, Bochum GermanyMaura Pugliatti European Academy of Neurology (EAN); Dept. of Biomedical and Surgical Sciences, university of Ferrara, ItalyAndreea Antonovici European Multiple Sclerosis Platform (EMSP)Monica Moroni School of Medicine, university of Ferrara, ItalyBettina Hausmann European Multiple Sclerosis Platform (EMSP)Rob Hyde BiogenEmanuele Degortes BiogenAnnik Laflamme Novartis AG Teva PharmaceuticalsRocheParkinsons Disease Working GroupRichard Dodel European Academy of Neurology (EAN)Günther Deuschl European Academy of Neurology (EAN); Prof. Dr. med, Department of Neurology, ukSH kiel, CAu kiel, GermanyWolfgang Oertel European Academy of Neurology (EAN); Hertie Senior Professorship; Dept. Neurology, Philipps university Marburg, GermanyLizzie Graham European Parkinsons Disease Association (EPDA)Eleanor Flanagan Grayling Belgium; European Parkinsons Disease Association (EPDA)Frank Michler jung & Parkinson, Germanyjutta Ahmerkamp-Böhme jung & Parkinson, GermanyGudula Petersen Grünenthal GmbHDaniela Myers Pfizer IncCharles Faid Pfizer IncMarja kolis Grünenthal GmbHSilke Walleser Autiero Medtronic12the Value Of treatment FOR BRAIN DISORDERSRestless Legs Syndrome (RLS) Working GroupClaudia Trenkwalder Paracelsus-Elena-klinik, Center of movement disorders, kassel, Germany/ university of Goettingen, Goettingen, Germanyjoke jaarsma European Alliance for Restless Legs Syndrome (EARLS)Roselyne Rijsman Center for Sleep and Wake Disorders, Haaglanden Medical Center, The Hague, The NetherlandsGiorgios Sakkas Department of Health, Plymouth Marjon university, Plymouth, uk & university of Thessaly, Trikala, GreeceRaffaele Ferri Department of Neurology IC, Oasi Research Institute (IRCCS), Troina, ItalyYves Dauvilliers Sleep Disorders Center, Department of Neurology, Gui-de-Chauliac Hospital, Inserm, u1061, Montpellier, FranceWolfgang Oertel European Academy of Neurology (EAN); Hertie Senior Professorship; Dept. Neurology, Philipps university Marburg, GermanyAnna Mezzacasa Vifor PharmauCB Biopharma, Brussels, Belgium13the Value Of treatment FOR BRAIN DISORDERSStroke Working GroupGeert Vanhooren European Stroke Organization (ESO) Department of Neurology, AZ Sint-jan Brugge-Oostende, Brugge, BelgiumVincent Thijs Belgian Stroke Council; Florey Institute of Neuroscience and Mental Health, Melbourne university, Melbourne, AustraliaEivind Berge Norwegian Stroke Organization, Dept. of Internal Medicine, Oslo university Hospital, and Institute of Clinical Medicine, university of Tromsø, NorwayAlastair Webb European Stroke Organisation (ESO); Department of Clinical Neuroscience, university of Oxford, ukAna Catarina Fonseca universidade de Lisboa, Faculdade de Medicina, Institute of Molecular Medicine, Portugal Bo Norrving European Stroke Organization (ESO); Dept. of Clinical Sciences Lund, Neurology, Lund university, SwedenValeria Caso European Stroke Organization (ESO)Franz Fazekas European Academy of Neurology (EAN)jon Barrick Stroke Alliance for Europe (SAFE)Gary Randall Stroke Alliance for Europe (SAFE)Stephanie Roso Pfizer Inc.Charles Faid Pfizer Inc.We would like to thank jean Georges, Dianne Goveand Ana Diaz (Alzheimer Europe) for their contribution to the case study on Alzheimer disease. We would also like to thank Alberto Raggi, Licia Grazzi, Domenico DAmico and Giuseppe Lauria (Foundation IRCCS Neurological Institute Carlo Besta, Milan, Italy) for their contribution to the case study on Headache. Finally, we would like to thank Eveline Sipido (European Academy of Neurology, EAN) for her support to the entire project.We are also taking the opportunity to thank all EBC staff members for their support to the entire project.Current EBC Team Members :Frédéric DESTREBECQ, Giovanni ESPOSITO, Stephanie kRAMER, Tadeusz HAWROT, Vinciane QuOIDBACH, Cécile GREBET, Vania PuTATTI and Marijn SCHOLTEPast Members :Ester BONADONNA, Alison TuRNER, Lara INGRAM and Monika kALkuSOVA14the Value Of treatment FOR BRAIN DISORDERSFOREWORDUnprecedented innovation in technology and medical processes is rapidly revolutionizing human life. Current health systems, however, have not been able to adapt quickly enough to maximize the value to patients. This is particularly true for brain disorders, and is particularly challenging for policy makers. Value-based healthcare is currently gaining traction in Europe as the desired solution or path forward in improving health systems. This holistic approach towards seamless care models critically intertwines wider patient and societal outcomes with efficient spending of resources. Doing this should lead to both a more sustainable framework for payers and improved care for patients.on 22 June 2017, the European Brain Council (EBC) will release the Value of Treatment (VoT) 2015-2017 research project policy White paper with case studies findings and policy recommendations, at a final conference organized under the auspices of the maltese EU presidency. The research project is building on the EBC Report “The Economic Costs of Brain Disorders in Europe” published in 2005 (Balak and Elmaci 2007) and updated in 2010 (Gustavsson et al. 2011) that provided robust estimates of the costs of brain disorders in Europe and enlightened necessary public health policy implications for more patient- oriented and sustainable care models as well as the need for more research. This policy paper focuses on “prevention and Early Intervention, Bridging the Early diagnosis and Treatment gap”. Starting from case studies data analysis covering a wide range of brain disorders and ending in evidence-based policy recommendations, the project assesses the treatment gap and the cost of non- or inadequate treatment. our findings recommend early intervention and the promotion of a holistic healthcare approach (as opposed to fragmentation in separate medical “silos”), address combined research and public health policy gaps and opportunities at the EU level, and translate the findings into policy recommendations.With this new study, EBC is not only looking at the socio-economic impact and value of healthcare interventions, but is also emphasizing how timely care pathways are likely to need greater integration and how better collaboration can be achieved in the future for the benefit of those living with or at risk of a brain disorder.We addre</p>
