2014 REPORT ON THE STATE OF THE ART OF RARE DISEASE ACTIVITIES IN EUROPE

Transcription

1 2014 REPORT ON THE STATE OF THE ART OF RARE DISEASE ACTIVITIES IN EUROPE STATE OF THE ART OF RARE DISEASE ACTIVITIES IN LATVIA This work was financed by the EUCERD Joint Action: Working for Rare Diseases N

2 This document has been produced by the Scientific Secretariat of the European Union Committee of Experts on Rare Diseases (EUCERD) Joint Action through the EUCERD Joint Action: Working for Rare Diseases (N , Coordinator: Kate Bushby, University of Newcastle, United Kingdom), within the European Union s Second Programme of Community Action in the Field of Health. The European Union Committee of Experts on Rare Diseases (EUCERD) was established in 2009 and its mandate ended in It is replaced from 2014 by the Commission Expert Group on Rare Diseases. The EUCERD Joint Action continues to support the activities of the new Expert Group until More information on the activities of the former European Union Committee of Experts on Rare Diseases and the EUCERD Joint Action can be found at Disclaimer: The findings and conclusions in this report are those of the contributors and validating authorities, who are responsible for the contents; the findings and conclusions do not necessarily represent the views of the European Commission or national health authorities in Europe. Therefore, no statement in this report should be construed as an official position of the European Commission or a national health authority. Copyright information: The 2014 Report on the State of the Art of Rare Disease Activities is copyrighted by the Scientific Secretariat of the EUCERD Joint Action : Working for Rare Diseases (N ). This product and its contents may be used and incorporated into other* materials on the condition that the contents are not changed in any way (including covers and front matter) and that no fee is charged by the reproducer of the product or its contents for their use. The product may not be sold for profit or incorporated into any profit-making venture without the expressed written permission of the EUCERD Joint Action Scientific Secretariat. Specifically: 1) When the document is reprinted, it must be reprinted in its entirety without any changes. 2) When parts of the documents are used or quoted, the following citation should be used. *Note: The 2014 Report on the State of the Art of Rare Disease Activities in Europe contains material copyrighted by others. For material noted as copyrighted by others, the user must obtain permission from the copyright holders identified in the document. To quote this document: Rodwell C., Aymé S., eds., 2014 Report on the State of the Art of Rare Disease Activities in Europe, July European Union,

3 ACRONYMS CAT - Committee for Advanced Therapies at EMA CHMP - Committee for Medicinal Products for Human Use at EMA COMP - Committee on Orphan Medicinal Products at EMA DG - Directorate General DG Enterprise - European Commission Directorate General Enterprise and Industry DG Research - European Commission Directorate General Research DG Sanco - European Commission Directorate General Health and Consumers EC - European Commission ECRD - European Conference on Rare Diseases EEA - European Economic Area EMA - European Medicines Agency ERN - European reference network EU - European Union EUCERD - European Union Committee of Experts on Rare Diseases EUROCAT - European surveillance of congenital anomalies EUROPLAN - European Project for Rare Diseases National Plans Development EURORDIS - European Organisation for Rare Diseases FDA - US Food and Drug Administration HLG - High Level Group for Health Services and Medical Care HTA - Health Technology Assessment IRDiRC International Rare Diseases Research Consortium JA - Joint Action MA - Market Authorisation MoH - Ministry of Health MS - Member State NBS - New born screening NCA - National Competent Authorities NHS - National Health System PDCO - Paediatric Committee at EMA RDTF - EC Rare Disease Task Force WG - Working Group WHO - World Health Organization 3

4 GENERAL INTRODUCTION This document was produced by the Scientific Secretariat of the European Union Committee of Experts on Rare Diseases (EUCERD), through the EUCERD Joint Action: Working for Rare Diseases (N ), which covers a three year period (March 2012 February 2015). The European Union Committee of Experts on Rare Diseases (EUCERD) was established in 2009 and its mandate ended in It is replaced from 2014 by the Commission Expert Group on Rare Diseases. The EUCERD Joint Action continues to support the activities of the new Expert Group until The present report aims to provide an informative and descriptive overview of rare disease activities at European Union (EU) and Member State (MS) level in the field of rare diseases and orphan medicinal products up to the end of A range of stakeholders in each Member State/country have been consulted during the elaboration of the report, which has been validated as an accurate representation of activities at national level, to the best of their knowledge, by the Member State/country representatives of the Commission Expert Group on Rare Diseases. The reader, however, should bear in mind that the information provided is not exhaustive and is not an official position of the European Commission, its Agencies or national health authorities. The report is split into six parts: Part I: Overview of rare disease activities in Europe Part II: Key developments in the field of rare diseases in 2013 Part III: European Commission activities in the field of rare diseases Part IV: European Medicines Agency activities and other European activities in the field of rare diseases Part V: Activities in EU Member States and other European countries in the field of rare diseases Part VI: Activities at National level in each EU Member State and other European countries in the field of rare diseases Parts I V also include a description of the methodology, sources and validation process of the entire report, and a selected bibliography and list of persons having contributed to the report. The present document contains the information from Parts II and V of the report concerning Latvia. A list of contributors to the report and selected sources are in annex of this document. For more information about the elaboration and validation procedure for the report, please refer to the general introduction of the main report. Each year, there are around downloads of the different sections of the report combined. 4

5 RARE DISEASE ACTIVITIES IN LATVIA Definition of a rare disease Stakeholders in Latvia accept the European Regulation on Orphan Medicinal Products definition of a prevalence of no more than 5 in individuals and that rare diseases are life-threatening or chronically debilitating diseases. National plan/strategy for rare diseases and related actions Work has recently been finished on a national plan by the working group, which included health care specialists and representatives from the Ministry of Health. In December 2011, the plan was written and submitted to the Ministry of Health for evaluation. The costs related to rare diseases are currently included in the national health care budget. A public consultation of the plan was launched in 2012 and the results were analysed by the Ministry of Health. A number of meetings with different stakeholders were held, and as a result, the plan was further elaborated. The plan was adopted in 2013, no additional funding has been secured, therefore major activities are related to update of regulations concerning rare diseases. The activities are now being implemented including the approval of an act concerning registries and plans to include Orphacodes and ICD codes in the congenital anomalies and cancer registries. A National Cancer Control Programme ( ) was stipulated by regulations No.48 of the Cabinet of Ministers of the Republic of Latvia (adopted on the 29 January, 2009), and included rare cancers. In August 2009, a regulation was introduced which allowed for the compensation of medicines for children with rare diseases. The Cardiovascular Health Improvement Action Plan ( ) was adopted in 2013 and includes activities in field of health promotion, improving cardiovascular diseases treatment and early diagnostic on congenital malformation of the heart. Centres of expertise There are currently no official designated centres of expertise for rare diseases in Latvia but a meeting was held in 2013 to discuss possible criteria for national centres of expertise. A legal framework for centres of expertise, including those for rare diseases, is expected in the future. Currently the Latvian State University Children s Hospital provides genetics services, hospital specialists deal with children with haematological, oncological and endocronological diseases. The Riga East University Hospital has a specialised clinic (Chemotherapy and haematology clinic) in which haemophilia A, haemophilia B, Factor XII deficiency and von Willebrand disease receive diagnostics and treatment (in this hospital, rare oncological diseases also can be treated, e.g. Burkitt s lymphoma, Langerhans cell histiocystosis, Mantle-cell NHL, multiple endocrinology neoplasia, Erwing s sarcoma, Wilm s tumour, Waldenström macroglobulinemia and others). Pauls Stradins University hospital has services in different rare diseases area: cardiology, nephrology, vascular diseases (Arteriovenous vascular malformations, lymphatic disorders, aortic pathology, endarteritis, carotid tumors, etc.), oftalmology, oncology, gastroenterology, endocrinology, pulmonology. A rare cardiovascular diseases network (Poland, Lithuania and Latvia through the P. Stradins Clinical University Hospital, Centre of Cardiology), started in May 2011 and finished in January The Ministry of Health, Orphanet team and experts from 3 University hospitals have been started work on developing national criteria for centres of expertise to be registered in Orphanet database and met in February 2013 to discuss this issue. Registries There is no separate registry for rare diseases but the National Plan for rare diseases foresees activities for evaluation and improvement of existing patient registries to start centralised data collection about rare disease patients. The Centre for Disease Prevention and Control is the supervising authority and keeper of Register of patients with particular diseases, including cancers, congenital anomalies (some of these are rare diseases). There is a plan to pilot use the Orpha code as well as OMIM codes for rare diseases in the register of patients with congenital anomalies and cancers. Latvia contributes to the EUROCARE (Eurocare-5 study) European registry, RARECARENet, and the Joint Action EUROCAT. Specialists from university hospital centres collect rare disease patient data, for example, at the Latvian Cardiology Centre is Pulmonary Arterial Hypertension patient's data base. The Centre of Endocrinology of Pauls Stradins University hospital has created several data bases of patients with rare endocrine diseases: 5

6 acromegaly, Cushings disease, MEN syndrome. These data bases are created for follow-up purposes, as well as serve to as a source of scientific information. Congenital malformation data base is held by the Genetics clinic at the Latvian State University Children s Hospital. Neonatal screening policy In Latvia, newborns are screened for phenylketonuria and congenital hypothyroidism Data about screened newborns in maternity units are collected in the Newborns Registry supervised by The Center for Disease Prevention and Control. Genetic testing The Regulations of the Cabinet of Ministers prescribe genetic analysis paid via the state budget. Any laboratory, that has a contract for the provision of services and appropriate technical resources, can perform these tests. In Latvia are not genetic analysis reference laboratories. A number of cytogenetic laboratory technologies have been approved for number of chromosomes and structure determination as well as molecular biological laboratory technologies for the determination of gene mutation. Regulations of the Cabinet of Ministers describe the procedures for the organisation and financing of health care and the types and amounts of medical treatment services that are be paid from the State budget, including genetic analysis. If genetic analysis for some reason cannot be performed in Latvia and that this analysis is necessary to prevent irreversible deterioration of health status or loss of vital functions, these tests can be performed abroad and they can be funded using the State budget. Genetic testing is available in Medical Genetics Clinic of Latvian State University Children s Hospital, Molecular Laboratory, Riga Stradins University, Scientific Laboratory and in Latvian BioMedical Research and Study Center. No national guidelines and specific conditions for reimbursement of expenses related to the tests have yet been determined. Genetic testing in other EU and EFTA states is possible with a E112/S2 form if genetic testing is a health care service usually financed from the state budget and this service cannot be provided in the Republic of Latvia or cannot be provided within a reasonable period of time. Mostly it is provided for children with life-threatening or treatable conditions. In some cases university clinics collaborate and send samples to Latvian Biomedical Centre of Research and Studies for genetic testing. For example, genetic testing of all family members of patients with MEN syndrome is being financed in term of scientific project. Diagnostic tests are registered as available in Latvia for 10 genes and an estimated 11 diseases in the Orphanet database 1. National alliances of patient organisations and patient representation There is no alliance in Latvia, but there is a patient organisation dedicated to rare diseases, the Latvian Rare Disease Organisation Caladrius 2 launched in The mission of the organisation is to provide patients with the relevant information and support and to represent patients. In 2010 Caladrius established a fund to help rare disease patients who could not otherwise fund their treatments: the organisation had obtained public benefit organisation status to legally collect funds for this action. There are plans to create an alliance of rare diseases patient organisations and chronic patient organisations at national level. Until now there were 8-9 organisations who share information and collaborate together in this area. Rare diseases patient organisations lack the capacity to establish an alliance. In Latvia are a number of other rare diseases and rare diseases-related patient organisations, including Latvia Haemophilia Society, the Society for People with Disabilities Motus Vitae, the Phenylketonuria Society, Pulmonary Hypertension Society and Society of Cystic fibrosis. These organisations often collaborate with each other and in 2013 had many activities, for example, Motus Vitae joined the International ALS/MND Alliance and arranged the international conference VII Nordic ALS Alliance meeting in Latvia "Baltic Bridge": Services for people living with ALS/MND (there participated patients with their assistants, medical professionals, social workers and Health Care Institutions from Denmark, Finland, Estonia, Russia, Iceland and Latvia). On 21 June 2013 I AM BREATHING was screened in Latvia by Motus Vita. 1 Information extracted from the Orphanet database (January 2014)

7 Latvian patient associations participated actively in 2013 in a Cystic Fibrosis Europe Association organised campaign which included the presentation of the film about cystic fibrosis patients of Latvia in European Parliament in Brussels on 14 November The aim of the campaign was to work out a strategy to obtain equal health care rights and to promote the accessibility of Cystic Fibrosis diagnostics and therapy for Cystic Fibrosis patients in all Europe. The Latvia Hemophilia Society has strong cooperation with European Hemophilia Consortium and World Federation of Hemophilia, as well as other people with bleeding disorders communities around the world. Ziedosim.lv is a non-governmental organisation which financially supports children and families to confirm a diagnosis of rare diseases by sending patients or medical samples abroad 3. Sources of information on rare diseases and national help lines Orphanet activities in Latvia The Ministry of Health of the Republic of Latvia has designated The Centre for Disease Prevention and Control as the representative of the Republic of Latvia to participate in the Joint Action Orphanet Europe since April The Orphanet team is currently hosted by the Centre for Disease Prevention and Control and is in charge of collecting data on rare disease related services (specialised clinics, medical laboratories, ongoing research, registries, clinical trials and patient organisations) in Latvia for entry into the Orphanet database. The Orphanet Latvia country site was launched in April 2012 and regularly updated by the Orphanet team. Official information centre on rare diseases There is no information centre for rare diseases in Latvia other than Orphanet. Web based information is available for a limited number of diseases (rare and non-rare) and certain information is maintained by using the state budget. Information was added about some common conditions in Latvian for the patients and general practitioners. According to Directive (2011/24) of patients rights in cross-border healthcare, Latvia has a focal point for providing information concerning the Latvian health care services and the services offered in different countries, including rare diseases. The Latvian National Health Service functions as this contact point. Help line There are non-rare disease specific help lines run by the state, some by the state budget, to help patients to access health care and psychological support, but no help line dedicated to rare diseases. A helpline for Cystic fibrosis patients is offered during office hours at the Children s University hospital. Other sources of information on rare diseases Information on rare diseases is available regarding paediatric rheumatic diseases 4, lysosomal diseases (Gaucher disease, Fabry disease and Hunter syndrome) 5, pulmonary hypertension 6, bleeding disorders 7, and via PHL Latvia 8. Guidelines In Latvia clinical guidelines for RD at national level have not been approved. The Centre of Endocrinology of Pauls Stradins University hospital in collaboration with Riga East University hospital endocrinologists and Latvian State University Children s Hospital endocrinologists issued in 2013 Diagnostic algorithms of rare endocrine diseases. These recommendations contain information of patients with a suspected rare endocrine disease in an organised and short form. The recommendations are aimed to help general practitioners and endocrinologists to think about rare diseases when presented with certain types of patients. Training and education initiatives Every year the Baltic metabolic specialist meeting is held; the 2013 was held in Riga. The meetings bring togetehr most geneticists and laboratory specialists from Baltic countries. Pauls Stradins University hospital organises post-diploma educational courses for different specialists in most areas of medicine, endocrinology

8 among them. The programme usually covers not only the most common clinical conditions but rare diseases also. In 2013, the Latvian Haemophilia Society intensified cooperation with Lithuanian Haemophilia Society in order to provide disease specific training for physiotherapists who work with people with bleeding disorders in Latvian State University Children s Hospital and the Riga East University Hospital. The adult haemophilia treatment centre in The Riga East University Hospital also runs various training sessions for nurses and other medical staff to raise awareness and knowledge of bleeding disorders and related health issues. National rare disease events in 2013 The Rare Disease Association Caladrius and the Centre for Disease Prevention and Control of Latvia marked the day by an event entitled Rare and Friendly. In support of rare disease patients in Latvia. Children, musicians Jenny May and Intars Busulis and hockey club Dinamo Riga participated in a photo shoot with patients. These sessions were not only to provide a pleasant surprise for patients with rare diseases, but also to show that people with rare illnesses are as important as the other members of the community, but they need public support to obtain the necessary health care. The event was followed by a press conference for public and media representatives about genetic and rare diseases In July 2013, Latvia Hemophilia Society, one of the oldest patient organisations in Latvia, celebrated its 20th anniversary. Hosted rare disease events in 2013 No hosted events were reported. Research activities and E-Rare partnership National research activities Funding is available for rare disease projects (through state budget, charities and pharmaceutical companies) although funds are not specifically earmarked for rare disease research. However rare diseases have not been included in the priority directions of science and research. Local activities are organised by the University hospitals, for example, Pauls Stradins University Hospital collaborated with Latvian Biomedical Centre of Research and Studies in term of research in the area of rare endocrine diseases. This collaboration includes genotyping of family members for specific mutations, genetic counselling, search for genetic markers etc. Participation in European research projects A research team in Latvia participates in one of the FP7 projects related to rare diseases. E-Rare Latvia is an observer of the E-Rare project. IRDiRC Latvian funding agencies do not currently contribute funds to the IRDiRC. Orphan medicinal products The State Agency of Medicines of Latvia is responsible for regular collecting and distributing of the information on medicines, including orphan medicinal products, as well as for collecting and compiling the information on the safety, evaluating drug risks and coordinate measures of medicine use risk mitigation, according to Regulations No of the Cabinet of Ministers (adopted on December 7, 2004) State Agency of Medicines Statutes. Orphan medicinal product committee A representative of Latvia is a member of the Committee for Orphan Medicinal Products (COMP) of European Medicines Agency. Orphan medicinal product incentives The Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products reported that in Latvia the State Agency of Medicines is entitled, due to considerations of health protection, to make a decision (after discussion with the Minister for Health) regarding the fee exemption or reduction for activities associated with the evaluation, 8

9 registration or re-registration of a medicinal product if the medicinal product (with or without orphan designation pursuant to Regulation 141/2000) is intended to the treatment of a rare disease. 9 Under the centralised procedure, companies submit a single marketing-authorisation application to the European Medicines Agency. Once granted by the European Commission, a centralised (or Community ) marketing authorisation for Orphan medicinal products is valid in all European Union (including Latvia) and EEA-EFTA states. Orphan medicinal product market availability situation The State Agency of Medicines of Latvia s includes the medicinal products registered in the Republic of Latvia and the centrally registered medicinal products (including orphan medicinal products) in a register 10 of medical products of the Republic of Latvia (according to Regulations No. 376 of the Cabinet of Ministers (adopted on May 9, 2006) Procedures for the Registration of Medicinal Products ). The following orphan medicines were marketed in Latvia in 2013: Cystadane, Diacomit, Evoltra, Exjade, Gliolan, INCRELEX, Jakavi, Kuvan, Myozyme, Myrin 100, Nexavar, Pedea, Revatio, Sprycel, Tasigna, Volibris, Wilzin. In 2013, compared to 2012, 3 new orphan drugs were launched on the market: Evoltra, Exjade, Myozyme and 3 orphan drugs were no longer placed on the market: Litak, Mozobil, Yondelis. Medicinal products: Aldurazyme and Ventavis which previously were designated as an orphan medicines and were placed on the market in 2012 are no longer designated as orphan medicines in Europe. Orphan medicinal product pricing policy There are no specific provisions for the pricing of orphan drugs. There have not been any developments in this area Orphan medicinal product reimbursement policy Since 2009, some orphan medicinal products for children are available as a part of the special programme Medical treatment of rare diseases for children for Children s University Hospital, Riga. Within this programme, there are provided some orphan medicinal products like Elaprase, Cystadane, Increlex, Kuvan. Orphan medicinal products are partially available via the reimbursement system. Dasatinibum, Nilotinibum are included in the positive reimbursement list. 2% of reimbursement budget is intended to individual reimbursement with limitation up to LVL/year for a single patient. Within this individual reimbursement, the following orphan medicinal products are provided: Revatio, Volibris, Nexavar, Cystadane, Diacomit, Thalidomide. Other initiatives to improve the availability of orphan medicinal products The Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products reported that in Latvia The State Agency of Medicines may issue [...] distribution authorisation for medicinal products not registered in Latvia if the medicinal product is intended for treatment of a rare disease (for an individual patient on the basis of prescription or for use in a health care institution on the basis of a written request). 11 Orphan devices There were no orphan devices placed on the market in 2012 Other therapies for rare diseases No specific information reported. Specialised social services Respite care services are available and the categories of patients eligible for reimbursement are described in the Procedures for the Organisation and Financing of Health Care (Regulations of the Cabinet of Ministers No. 1046, adopted on 19 December 2006). Therapeutic recreational programmes are also available and costs are included in the national health care budget. There are existing government-run services promoting social integration of those with handicaps, including the school environment and work place. 9 Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products (2005 revision) (pp16-17) Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products (2005 revision) (pp16-17) 9

10 As of 1 January 2013 a new service for persons with disabilities (including persons with disabilities of rare diseases) has been launched, a municipality based service of an assistant for performing activities outside home (to get to a place where person work, learn, get to the rehabilitation institution etc.). An assistant service is eligible to: persons with Group I or Group II disability 12, on the basis of conclusion by the State Medical Commission for the Assessment of Health Condition and Working Ability on the necessity for a service of an assistant; persons with disability aged 5 to 18 years 13, on the basis of conclusion by the State Medical Commission for the Assessment of Health Condition and Working Ability on the necessity for special care due to severe functional impairments. The service of an assistant amounts to 40 hours a week within the territory of Latvia (except for persons with Group I visual disability who receive a benefit for using a service of an assistant 10 hours a week and who receive a service up to 30 hours a week if a service of assistant exceeds 10 hours a week that is specified by the municipality social service office). DEVELOPMENT OF RARE DISEASE ACTIVITIES IN 2013 IN LATVIA National plan/strategy for rare diseases and related actions Work has recently been finished on a national plan by the working group, which included health care specialists and representatives from the Ministry of Health. In December 2011, the plan was written and submitted to the Ministry of Health for evaluation. The costs related to rare diseases are currently included in the national health care budget. A public consultation of the plan was launched in 2012 and the results were analysed by the Ministry of Health. A number of meetings with different stakeholders were held, and as a result, the plan was further elaborated. The plan was adopted in 2013, no additional funding has been secured, therefore major activities are related to update of regulations concerning rare diseases. The activities are now being implemented including the approval of an act concerning registries and plans to include Orphacodes and ICD codes in the congenital anomalies and cancer registries. The Cardiovascular Health Improvement Action Plan ( ) was adopted in 2013 and includes activities in field of health promotion, improving cardiovascular diseases treatment and early diagnostic on congenital malformation of the heart. Centres of expertise There are currently no official designated centres of expertise for rare diseases in Latvia but a meeting was held in 2013 to discuss possible criteria for national centres of expertise. A legal framework for centres of expertise, including those for rare diseases, is expected in the future. The Ministry of Health, Orphanet team and experts from 3 University hospitals have been started work on developing national criteria for centres of expertise to be registered in Orphanet database and met in February 2013 to discuss this issue. Guidelines In Latvia clinical guidelines for RD at national level have not been approved. The Centre of Endocrinology of Pauls Stradins University hospital in collaboration with Riga East University hospital endocrinologists and Latvian State University Children s Hospital endocrinologists issued in 2013 Diagnostic algorithms of rare endocrine diseases. These recommendations contain information of patients with a suspected rare endocrine disease in an organised and short form. The recommendations are aimed to help general practitioners and endocrinologists to think about rare diseases when presented with certain types of patients. 12 For the persons from 18 years of age depending upon the level of limitation of physical or mental abilities shall be determined to have the following: a) Group I disability very severe disability, b) Group II disability severe disability, c) Group III disability moderately expressed disability. 13 For a person up to the age of 18 disability is determined without being divided into groups. 10

11 Training and education initiatives Every year the Baltic metabolic specialist meeting is held; the 2013 was held in Riga. The meetings bring togetehr most geneticists and laboratory specialists from Baltic countries. Pauls Stradins University hospital organises post-diploma educational courses for different specialists in most areas of medicine, endocrinology among them. The programme usually covers not only the most common clinical conditions but rare diseases also. In 2013, the Latvian Haemophilia Society intensified cooperation with Lithuanian Haemophilia Society in order to provide disease specific training for physiotherapists who work with people with bleeding disorders in Latvian State University Children s Hospital and the Riga East University Hospital. The adult haemophilia treatment centre in The Riga East University Hospital also runs various training sessions for nurses and other medical staff to raise awareness and knowledge of bleeding disorders and related health issues. National rare disease events in 2013 The Rare Disease Association Caladrius and the Centre for Disease Prevention and Control of Latvia marked the day by an event entitled Rare and Friendly. In support of rare disease patients in Latvia. Children, musicians Jenny May and Intars Busulis and hockey club Dinamo Riga participated in a photo shoot with patients. These sessions were not only to provide a pleasant surprise for patients with rare diseases, but also to show that people with rare illnesses are as important as the other members of the community, but they need public support to obtain the necessary health care. The event was followed by a press conference for public and media representatives about genetic and rare diseases In July 2013, Latvia Hemophilia Society, one of the oldest patient organisations in Latvia, celebrated its 20th anniversary. Hosted rare disease events in 2013 No hosted events were reported. Specialised social services As of 1 January 2013 a new service for persons with disabilities (including persons with disabilities of rare diseases) has been launched, a municipality based service of an assistant for performing activities outside home (to get to a place where person work, learn, get to the rehabilitation institution etc.). An assistant service is eligible to: persons with Group I or Group II disability 14, on the basis of conclusion by the State Medical Commission for the Assessment of Health Condition and Working Ability on the necessity for a service of an assistant; persons with disability aged 5 to 18 years 15, on the basis of conclusion by the State Medical Commission for the Assessment of Health Condition and Working Ability on the necessity for special care due to severe functional impairments. The service of an assistant amounts to 40 hours a week within the territory of Latvia (except for persons with Group I visual disability who receive a benefit for using a service of an assistant 10 hours a week and who receive a service up to 30 hours a week if a service of assistant exceeds 10 hours a week that is specified by the municipality social service office). In 2013 PHA Latvia financially supported the first pulmonary endarterectomy for CTEPH patient, organised the Summer Health camp for 40 rare disease patients and their relatives (caregivers), including children with rare diseases and their parents, managed the Charity Sport games to seek fundraising for PAH patients, and proceeded the oxygen home care therapy supporting for PAH patients. 14 For the persons from 18 years of age depending upon the level of limitation of physical or mental abilities shall be determined to have the following: a) Group I disability very severe disability, b) Group II disability severe disability, c) Group III disability moderately expressed disability. 15 For a person up to the age of 18 disability is determined without being divided into groups. 11

12 LIST OF CONTRIBUTIONS 16 Contributions in 2010 Rita Lugovska and Zita Krumina (Orphanet Latvia, Medical Genetics Clinic of the Latvian State, Children s University Hospital, Riga) Monta Forstmane (Department of Health Care, Ministry of Health) Ieva Grinfelde (Medical Genetics Clinic of the Latvian State, Children's University Hospital, Riga) Baiba Lace (Caladrius and Medical Genetics Clinic of the Latvian State, Children s University Hospital, Riga) Contributions in 2011 This report has been compiled in collaboration with members of the Ministry of Health Centre of Health Economics, Health Payment Centre, State Agency of Medicines and Medical Genetics Clinic of the University Children's Hospital, Riga, including: Dainis Krievins (COMP Member for Latvia, University of Latvia) Rita Lugovska (Orphanet Latvia, Medical Genetics Clinic of the Latvian State, Children s University Hospital, Riga) Antra Valdmane (EUCERD Representative Latvia, Ministry of Health of the Republic of Latvia) Contributions in 2012 Collaboration with members of the Ministry of Health, the National Health Service, the State Agency of Medicines, the Riga East University Hospital, the Paula Stradins Clinical University Hospital, Caladrius (The Latvian Rare Disease Organisation), the Society for People with Disabilities Motus Vitae, the Haemophilia Society and the Pulmonary Hypertension Association. Contributions in 2013 Jana Lepiksone (Orphanet Latvia team coordinator, The Centre for Disease prevention and Control of Latvia) Antra Fogele (The National Health Service) Elma Gailīte (The State Agency of Medicines) Zane Straume (Children s University Hospital, Riga) Anita Kalēja (P. Stradins Clinical University Hospital) Ieva Plūme (Pulmonary Hypertension Society) Valerijs Rakovs (The Association Motus Vita) Contributions in 2014 Kristina Pure (Ministry of Welfare) Jana Lepiksone (Orphanet Latvia team coordinator, The Centre for Disease Prevention and Control of Latvia) Solvita Štelmahere (The National Health Service) Elma Gailīte (The State Agency of Medicines) Zane Straume (Children s University Hospital, Riga) Aldis Rozenbergs (Pauls Stradins Clinical University Hospital) Ieva Plūme (Pulmonary Hypertension Society) Valerijs Rakovs (The Association Motus Vita) Baiba Ziemele (Latvia Hemophilia Society) Alla Beļinska (Society of Cystic fibrosis) Validated by: Antra Valdmane (ECEGRD Representative Latvia, Ministry of Health of the Republic of Latvia) 16 The contributors and validators of the report have contributed information which is accurate to the best of their knowledge. However, readers should take note that the contents of this report are illustrative and not exhaustive. 12

13 SELECTED BIBLIOGRAPHY AND SOURCES 17 The State Agency of Medicines National Cancer Control Programme ( ) polsis.mk.gov.lv/view.do?id=2932 Orphanet Latvia national website Latvian Rare Disease Organisation - Caladrius Palidzesim.lv 17 All websites and documents were last accessed in May