Transparency in drug regulatory decision making Why transparency? Birthe Byskov Holm, Rare Diseases Denmark Copenhagen October

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1 Transparency in drug regulatory decision making Why transparency? Birthe Byskov Holm, Rare Diseases Denmark Copenhagen October

2 Transparency - A tool to drive change and innovasion?

3 Breaking the Acces Deadlock to Leave One Behind A work-in-progress contribution on possibilities for patients' full and equ access to rare disease therapies February 2017

4 Who are we? EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of over 700 rare disease patient organisations from more than 60 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services. 4

5 EURORDIS in brief Founded in member patient organisations 68 countries (28 EU countries) 41 National Alliances of RD Patients Organisations 58 European Federations of specific rare diseases Outreach to over 1800 patient groups 40+ staff, offices in Paris, Brussels, Barcelona Over 320 Volunteers: +80 patient advocates and +250 moderators 5 Million Budget 5

6 Who are we? Our vision Better lives and cures for people living with a rare disease Our mission EURORDIS-Rare Diseases Europe works across borders and diseases to improve the lives of people living with a rare disease 6

7 What is a rare disease? A rare disease is defined in Europe as affecting fewer than 1 in 2,000 citizens Over 6,000 distinct rare diseases 6% of the population affected in the course of their lives Many are of genetic origin % rare diseases are non genetic, over 30% of patients Many children are affected by rare diseases. Onset occurs in childhood for 50% of rare diseases. 30 million people living with a rare disease in Europe More than 300 million people living with a rare disease across the world Some main groups: metabolic, neuro-muscular, autoimmune, developmental anomalies, bleeding disorders, cardiovascular, respiratory, skin diseases, rare cancers 7

8 Breaking the Acces Deadlock to Leave One Behind A work-in-progress contribution on possibilities for patients' full and equ access to rare disease therapies February 2017

9 EURORDIS Press Release Multi-Stakeholder Symposium Brussels February Even though ever-advancing science and technology now create rapidly growing opportunities to develop more innovative medicines, the rare disease community is still facing problems accessing these medicines in Europe because of their affordability. This reflection paper, still a work in progress, is intended to challange a number of existing misconceptions around rare disease medicines. It sets out how to design a new and improved model to ensure patients full access to these medicines, a model that addresses the current imbalance and restores in parallel transparency and also trust between payers and pharmaceutical companies, paving the way for common value principles and fair pricing of medicines.

10 EURORDIS Press Release Multi-Stakeholder Symposium Brussels February Eurordis is calling for a change to the system to ensure that medicines are immidiately accessible to patients, affordable and sustainable for national healthcare systems. The system should create a predictable and sustainable environment for companies and investors We will not take no for an answer. We cannot passively curb under the weaknesses of the current model. We, rare disease patients, are not the problem: we are part of the solution. The ambition of Eurordis by 2015 is to have 3 to 5 time more new rare disease therapies approved per year, 3 to 5 time cheaper than today.

11 What has this to do with transparency in drug regulatory decision making?

12 Involved in EMA Committees COMP: Committee for Orphan Medicinal Products 1 EURORDIS representatives (Vice-Chair) + 1 Observer PDCO: Paediatric Committee 2 EURORDIS representatives (full member & alternate) CAT: Committee for Advanced Therapies 1 EURORDIS representative (full member) PCWP: Patients' and Consumers' Working Party 2 EURORDIS representatives (full members) Scientific Advice & Protocol Assistance CHMP: Committee for Human Medicinal Products 12 12

13 EURORDIS REPRESENTATION IN EXTERNAL NETWORKS, ORGANISATIONS AND INSTITUTIONS IN 2017 EMA European Medicines Agency European Commission European Reference Networks (ERNs) COMP Committee for Orphan Medicinal Products PDCO Paediatric Committee CAT Committee for Advanced Therapies PCWP Patients & Consumers Working Party SAWP Scientific Advice Working Party CHMP Committee for Medicinal Products for Human Use EU clinical trials portal and Union database stakeholders group EMA Task Force on Registries Commission Expert Group on Rare Diseases Commission Expert Group on Cancer Control EU Health Policy Forum Joint Research Center EU Platform Rare Diseases Registration (JRC) Health Technology Assessment (HTA) HTA Network EUnetHTA Joint Action 3 MEDEV / MOCA ERN BOND - European Reference Network on bone disorders ERN CRANIO - European Reference Network on craniofacial anomalies and ear, nose and throat (ENT) disorders Endo-ERN - European Reference Network on endocrine conditions ERN EpiCARE - European Reference Network on epilepsies ERKNet - European Reference Network on kidney diseases ERN-RND - European Reference Network on neurological diseases ERNICA - European Reference Network on inherited and congenital anomalies ERN LUNG - European Reference Network on respiratory diseases ERN Skin - European Reference Network on rare and undiagnosed skin disorders ERN EURACAN - European Reference Network on adult cancers (solid tumours) ERN EuroBloodNet - European Reference Network on haematological diseases ERN eurogen - European Reference Network on urogenital diseases and conditions ERN EURO-NMD - European Reference Network on neuromuscular diseases ERN EYE - European Reference Network on eye diseases ERN GENTURIS - European Reference Network on genetic tumour risk syndromes ERN GUARD-HEART - European Reference Network on diseases of the heart ERN ITHACA - European Reference Network on congenital malformations and rare intellectual disability MetabERN - European Reference Network on hereditary metabolic disorders ERN PaedCan - European Reference Network on paediatric cancer (haematooncology) ERN RARE-LIVER - European Reference Network on hepatological diseases ERN ReCONNET - European Reference Network on connective tissue and musculoskeletal diseases ERN RITA - European Reference Network on immunodeficiency, autoinflammatory and autoimmune diseases ERN TRANSPLANT-CHILD - European Reference Network on Transplantation in Children VASCERN - European Reference Network on Rare Multisystemic Vascular Diseases 13

14 European Not-for-Profit Organisations: DIA: Drug Information Association EFPIA Think Tank: European Federation of Pharmaceutical Industries and Associations EUROPABIO Patients Advisory Group EUCOPE EURORDIS REPRESENTATION IN EXTERNAL NETWORKS, ORGANISATIONS AND INSTITUTIONS IN 2017 EPF: European Patients Forum EFGCP: European Forum for Good Clinical Practice FIPRA International Policy Advisors Friends of Europe Rare Cancer Europe Social Platform Maladies Rares Info Service (French Helpline for RDs) Rare Disease Platform in Paris PFMD - Patient Focused Medicines Development Initiative European network of parliamentarian advocate for rare diseases: European parliament interest group on Rare Diseases Advocates in national parliaments International Institution s and Not-for-Profit Organisations: NGO Committee for Rare Diseases (United Nations, New York) NEWDIGS: New Drug Development ParadIGmS IAPO: International Alliance of Patients Organizations IRDiRC: International Rare Disease Research Consortium ICORD: International Conference on Rare Diseases and Orphan Drugs Member of European Networks: E-Rare EuroBioBank ECRIN BBMRI Stakeholders Forum Treat NMD RD-Connect SCOPE Joint Action (Advisory Board) OpenMedicine IMI EUPATI IMI ADAPT-SMART Partnership Learned Societies: European Federation of Internal Medicine (EFIM) European Hospital & Healthcare Federation (HOPE) - International Federation of Social Workers Europe (IFSW-Europe) European Society of Human Genetics (ESHG) International Society for Pharmaco-economics and Outcomes Research (ISPOR) 14

15 EURORDIS Summer School Started 2008, new format was launched in 2015: Expert Patient and Researcher Eurordis Summer School (ExPRESS) 2015 and 2016 saw the inclusion academic researchers as participants for the first time. So far, over 400 patient representatives and researchers have been trained, coming from 40+ different countries and representing 75+ diseases 5-day annual training on: Clinical Trials & Medicines Development EU Regulatory Processes & EMA HTA, Reimbursement, Patient Access Translational & International Research 15 Alumni involved in regulatory processes at the EMA and/or in collaboration with sponsors and/or as EURORDIS Volunteers 15

16 EURORDIS GOVERNANCE CHART 2017 MEMBERS Financial Audit Deloitte European Public Affairs Committee EURORDIS Panel of Experts GENERAL ASSEMBLY BOARD OF DIRECTORS BOARD OF OFFICERS CHIEF EXECUTIVE OFFICER STAFF President Vice President General Secretary Treasurer Officer EURORDIS International Circle of Ambassadors EURORDIS Action Groups & Task Forces Therapeutic Action Group (EMA) Policy Action Group (Commission Expert Group on RDs) Policy Action Group Rare Cancers (Commission Expert Group on Cancer) DITA TF (Drug, Information, Transparency & Access) ECRD 2018 Vienna 24 European Patient Advocacy Groups (epags) EURORDIS STANDING COMMITTEES & COUNCILS Council of Rare Diseases International (specifc by-laws for autonomous governance) Council of National Alliances on Rare Diseases Council of European Federations on Rare Diseases European Network of RD Help Lines (ENRDHLs) EURORDIS PROGRAMS & PROJECTS COMMITTEES & WORKING GROUPS Health Policy: - Social Policy Advisory Group - EUROPLAN Advisors - RareConnect Steering Committee - RareConnect Advisory Committee - Rare Barometer Advisory Committee - Rare Barometer Topic Experts Committee Communication: - Editorial Committee - Rare Disease Day Steering Committee - Black Pearl Evening Committee Research & Therapies: - RD-Connect Joint Patient Advisory Council - Summer School Faculty 16 Cross-cutting - Operating Grant Steering Committee - EURORDIS Membership Meeting 2017 Budapest

17 Eurordis is calling for radical change. This change will not be easy but is urgently needed to translate innovasion into health benefits for patients. Improving access can only be achieved by establishing a collaborative multistakeholder structured approach. Our goal is to leave no one behind. Thank you. 17

18 Eurordis is calling for radical change. This change will not be easy but is urgently needed to translate innovasion into health benefits for patients. Improving access can only be achieved by establishing a collaborative multistakeholder structured approach. Our goal is to leave no one behind. 18