Friday March 10th, 2017

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ДЕН НА РЕТКИ БОЛЕСТИ 2017

The Life With Challenges association, together with the National Alliance for Rare Diseases of the Republic of Macedonia, under the umbrella of the European Association for Rare Diseases – EURORDIS, celebrated the international Rare Diseases Day, February 28, 2017 this year as well under the motto “With Research, Possibilities are Limitless”. In addition, this year we are also focusing on parents of children with rare diseases and the difficulties and challenges they face. Their stories were on display in a poster presentation in the hall of the EU Info Center today and they were shared on social media.
After the new Law on Financing the Rare Diseases Program was passed in 2015, 21 new medicines were procured for approximately 12 rare disease diagnoses, which covered more than 150 patients. According to information available from the Ministry of Health’s public bids, the number of medicines in 2016 has increased by 29, which means that more diagnoses have been covered, so now the number of patients receiving medication has reached approximately 220. There are 46 primary diagnosis in the registry.
Statement by professor Aspazija Sofijanova, PhD, president of the National Commission for Rare Diseases:
As medical doctors we must be less emotional, however that’s not so in our case because we are in direct contact with sick children. When we save a child, it feels like we have saved the world, but when we lose a child, it feels like we have lost the world. This year we will continue to struggle for the patients with rare diseases, and the parents, the patients and the associations offer the greatest help in that struggle – if we join forces with the institutions, there is a lot that can be done. We know the issues. I just came from the clinic and I would like to say that Iskra says hi, that’s a child who has spinal muscular atrophy and who is still in intensive care at the clinic. I also know Daniel, who was helped a great deal by the US Embassy in Macedonia, that is Mrs. Mary Jo Wohlers. It is a new medicine, and we will try to find the financial means to bring it to Macedonia, like we bring medicines for other patients.

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Изјава, Проф. Др. Аспазија Софијанова, Претседателот на Националната комисија за ретки болести

Statement by academician Momir Polenakovic, Macedonian Academy of Sciences and Arts (MASA):
Within MASA we have a small genetics and diagnostics center, but we also collaborate with other countries and we are trying to forge the way ahead in terms of education and to push forward in diagnostics, which is as important as prevention. Early diagnostics is essential for early therapy and for providing timely information to the patients, so that their life quality is improved. The collaboration with the Wilson Disease Association – Macedonia has already resulted in improvement in the education for diagnosing Wilson Disease as well, and they provided us with reagents through a donation. We hope that in the future we will have more funds for providing other reagents in order to boost the diagnosis of a larger number of rare diseases.

Statement by Rashela Mizrahi PhD, Ministry of Health of the Republic of Macedonia:
The rare diseases program was launched two years ago with the creation of the rare diseases registry and the increased financial support for medicine procurement. There are many issues in terms of diagnosis, classification and systematization. There’s a whole apparatus working in the background at the Ministry and the Rare Diseases Commission, where clinicians work pro bono for the wellbeing of the patients. We have also established criteria and requirements for entering the registry, some 400 patients have been registered with more than 46 diagnoses, and we already have medication for 220 patients.
Statement by Rebeka Jankovska Risteski, president of NARBM (National Alliance for Rare Diseases of the Republic of Macedonia):
This year we are focusing on patients who are yet to receive medicine. Such are patients who have spinal muscular atrophy, children whose only hope is a medicine approved by the American Food and Drugs Administration last year. Then we have the medicines for patients who have rare forms of cancer, such as the MDS patients – two patients who were donated the medicines have already shown signs of improvement in their wellbeing and life. Therefore we believe that innovative medication should be made available in order to allow for a good quality life for the patients and their families. Then we have the medicine for juvenile arthritis – only one of the three that exist is being procured. Finally we would ask the Health and Pension Insurance Fund of Macedonia (FZOM) not to forget that even in 2015-2016 they promised the patients suffering from Chrohn’s disease and ulcerative colitis they would provide them REMICADE. Of course, we would also like them to provide patients with medical aids and meet other needs when there are no medicines available. Our vision as an alliance is for nobody to be left behind.

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Statement by Anja Bosilkova Antovska, vice-president of NARBM:
There is a great variety of rare diseases and anyone may contract them. We carried out research in collaboration with the Wilson Disease Association – Macedonia and we drafted some recommendations for changes in Macedonian legislation so that rare disease patients are not apprehensive of whether there will have a long-term therapy plan available or not. A national rare disease program and systematic, legal solutions must exist for us to ensure a future for the rare disease families.
Statement by Vesna Aleksovska, director of Life With Challenges:
No matter how much I speak I am bound to leave something out. On the one hand we are thankful for the understanding that the institutions have shown for us, rare disease patients, in the last two years, but on the other hand I must also say that there is a lot to be done, many people to be helped, and that we will always have new requests and new needs. The rare diseases are very unique, each one has a number of variations and each patient is a different story, with separate challenges, difficulties, issues, happiness and sorrow. Working together as part of the alliance I think that we can achieve much more, and I hope that we will be increasingly vocal and that our voice will be heard more and more. Each family deserves the best chance possible for a happy life. We will continue to meet up with the institutions, we will speak up in front of the media, we will get the public involved by organizing events… Each patient must be taken care of.

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Other events around Macedonia

Bitola
This year the rare diseases awareness raising campaign began with an event in Bitola on February 24, at 10 a.m. in downtown Bitola, on Sirok Sokak, where with the support of the Hepar Centre for Citizens who have Rare Diseases the Life With Challenges Bitola organized a press-conference and distributed flyers downtown in order to raise public awareness for the rare diseases.

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Ohrid

On February 28, 2017, at noon, a march began from the front yard of the Hristo Uzunov primary school. Some 200 students in a line marched along Pitu Guli street, and then along Jane Sandanski street, and then they joined forces with the students of the St. Clement of Ohrid secondary school and they continued together all the way to the Macedonia quay (in front of the Royal hotel). There, at 1 p.m., the students ran a cross race to the Ss. Cyril and Methodius monument. From there the students went to the city square, where they distributed information leaflets for rare diseases. On February 26 and 27, there were hiking activities organized as well, with the support of ZSH Avantura – Ohrid. The activities in Ohrid were also bolstered by the Animalija association, which supported the idea for rare disease awareness raising with the motto: “Adopt a pet, for some people that’s the only free therapy”. The event was organized by Gordana Loleska, a mother of a child suffering from Alport syndrome, with the support of the Ohrid Red Cross, the St. Clement of Ohrid secondary school, ZSH Avantura, the Animalija association and the citizens of Ohrid.

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Prilep

On February 24, 2017, a group that distributed flyers for rare disease awareness raising joined the Prilep masquerade. There was also a public lecture for rare diseases. The event was organized by Jasmina Hristoska, an acromegaly patient, with the support of SLAP – Prilep.

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About the Association Life With Challenges

Association of citizens for rare diseases LIFE WITH CHALLENGES Bitola

Our organization is founded with the help and support by the National Alliance of people with rare diseases of Bulgaria. The first meeting of families and doctors connected to rare diseases in Macedonia was organized by Bulgaria in Ohrid, Macedonia, in June, 2009.

Since than the patients in Macedonia decided to form their own organization and fight for their rights. This is how Life With Challenges was born.

In the next 3 years we were gathering members and learning how to manage and grow into a strong patient organization.

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Our Vision and Mission

Vision

Our vision is achieving the best possible quality of life of patients and families with rare diseases, through best possible social and health services.

Mission

Our mission is developing solutions and politics through implementation of activities for improving quality of life of patients and families with rare diseases. The goal of Life With Challenges is to help and support patients and families that face life with rare diseases. Our mission is realized through the following activities:

  • Helping and informing people with rare diseases, creating and sustaining communication between patients
  • Raising awareness in society through education about the problems of patients with rare diseases
  • Organization of public events, congresses and meetings with a goal to educate and inform
  • Support of research and education about rare diseases
  • Organization of info centres for people with rare diseases
  • Organization of cultural events for raising public awareness
  • Distribution and printing of info-materials about rare diseases
  • Social rehabilitation for people with rare diseases
  • Finding funds for treatment of patients
  • Documentation of patients with rare diseases

Important Issues

  • Advocating for patients’ rights.
  • Raising awareness in society about rare diseases, about physical and psychological consequences.
  • Helping and informing people with rare diseases.
  • Helping doctors to get information about diagnostics, treatment and rehabilitation of rare diseases.
  • Communication with other organizations about rare diseases in R. Macedonia and in the world.
  • Communication with health institutions in R. Macedonia for better treatment of people with rare diseases.

What are we doing?

  • Negotiating with the Ministry of Health
  • Informing the President of the Government and the Minister of Finance about the importance of treatment for patients with rare diseases
  • Raising awareness to get more support from the people in Macedonia
  • Cooperating with the media to raise the level of awareness about our problems
  • Cooperating with other patient organizations to act together about important patient issues

Rare diseases in the organization

The association of citizens for rare diseases Life With Challenges, is founded by patients and parents that face with life with Gaucher. Later in the association joined patients with Hereditary Angioedema, Allagile Syndrome, PKU and others.

The association is representing patients and parents with rare diseases that are members in the association. Rare diseases represented by the association are: Gaucher, Phenylkeotnuria, Hereditary Angioedema, Allagile Syndrome,  Tyrosinemia, Lenox Gastaut Syndrome, Congenital Muscular Dis trophy,  Congenital Cataract, Epydermolysis Bulosa, Carnitine plamitolitransferase deficiency, Pulmonary Hypertension, Adrenomyeloneuropathy, Wilson Diseases, Williams Syndrome, RET Syndrome, Amyotropihi lateral sclerosis – Lou Gehrig’s diseases, Myasthenia Gravis , Huntington disease, Porphyria, malignant paraganglioma feohromocitom tumor, arthogryposis multiplex congenital, Fibrodysplasia ossificans progressiva (FOP), Multiple myeloma, Myelodysplastic syndrome, MPS 4 – Morqio Syndrome,   Strümpell disease – Hereditary spastic paraplegia, Takayasu arteritis, Primary ciliary dyskinesia, Kartagener type, Mastocytosis, Juvenile dermatomyositis, Friedreich ataxia , Acute intermittent porphyria, Cystinuria, Stargart, Lowe Syndrom, Dextocardia Situs inversus, Heurofibramatozis, Phemgus Vulgaris, Wegener’s Granulomatosis, Ehlers – Danhlos Syndrom, Alport Syndrom, Polimiozitis, Mastocitozis, Burgada Syndrom, Fanconi, Niemann Pick C, Chronic inflammatory demyelinating polyneuropathy, Carney Complex,  Erythrodermia Ichthyosiformis congenita, Guillain Barreov Sindrom, Malignant Melanoma, Rendu-Osler-Weber disease -hereditary hemorrhagic telangiectasia(HHT), Systemic vasculitis-Sy.Churg-Strauss, …  and others that will join us and ask for help and support.

The association does not represent rare diseases that are not registered members in the association.

Some groups of patients have their own associations and we encourage and support other groups to form associations since we think it is important for each disease to have association or at least a support group.

The association also is a member of the National Alliance for Rare Diseases of R. Macedonia (14 organizations representing different rare diseases and patients rights).

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What have we accomplished?

 

Program for rare diseases at the Ministry of Health of Macedonia

The first program for rare diseases at the Ministry of health is from the year 2009 and it included 3 patients with MPS 2 – Hunter syndrome.

There were no further changes until 2015. From 2015 there is a register for RD at the Ministry of Health – more about that can be found in the program for RD from 2016.

The New Programme of the Ministry of Health for 2016 is as it follows:

  1. Diseases which can be treated with medicines;
  2. Diseases which are rare, but for which there is no treatment but which are important for the diagnostic and planning.

In the registry of patients with rare diseases are included solely the diseases listed in the ORPHAN list of rare disease – reviewed in July 2014

http://www.orpha.net/orphacom/cahiers/docs/GB/List_of_rare_diseases_in_alphabetical_order.pdf
In the Registry of rare disease patients are only included diseases which affect one in 2000 persons, but which do not exceed 20 affected at the level of Republic of Macedonia
Medicines are provided or the patients who are registered in the Registry of rare diseases through this programme. The provided medicines are necessary for the patients and represent an only choice of treatment of the rare disease, and they are not on the positive list of medicines

For the realization of this programme, treatment of rare diseases with therapy and medicines which have not been previously applied in rare disease treatment, for the provision of the necessary equipment for diagnosis of rare diseases, providing education for the staff who diagnoses and treats the rare disease patients, it is expected that in 2016 total funds in the amount of 203.000.000,00 denars will be needed.

  • Treatment of patients with a rare disease regardless of insurance status 197.000.000.00
  • Providing the necessary equipment for diagnosis and treatment of rare diseases 4.000.000.00
  • Providing education of the staff who diagnoses/treats the rare disease patients 2.000.000.00

We hope that changes will be made in the program for 2017 since as patients organizations we all reacted to the limitation of the number of patients (20 per diagnosis). And we reacted to the lack of further criteria and step by step explanation of how patients can be registered and get the needed treatment.

Rare disease registry in Macedonia

The register for rare diseases is consisted of collected data for all diagnosed cases of people with rare diseases in a population.

The registration is a process of systematic collection and data analysis so that it can provide information on the number of rare diseases and the number of people affected by them.

In Macedonia the register for rare disease is at the Ministry of Health. Patients need to send a request with appropriate diagnostic information so that they can be included in the registry. Also doctors can send information about patients so that they can be included at the registry.

The commission for rare disease is consisted from doctors from the clinic centre in Skopje. They gather to discuss about registering patients in the rare disease registry and further they discuss about the possible treatment and tis provision to the patients who need it.

For now the Program for rare diseases has limitation – it says that treatment will be provided to patients with rare diseases (where rare diseases are those 1 in 2000 people as from the list of www.orpha.net , but only if they are not more than 20 in R. Macedonia and have no treatment at all from others like from the Health Insurance Fund or from the Clinics). We hope that this limitation will go away and that all patients will get treatment. We also hope that drugs will be provided in a timely manner so that patients won’t be left without therapy for long periods of time (3 months) because of administration procedures.

In 2017 the Health Insurance Fund announced that they will cover diagnosis for patients when they can not get a diagnosis in the country in time of 6 months which is the same as the law in Serbia and we proposed it to the institutions right after we saw it was working in Serbia. We are very happy about this and we hope it will help families facing rare diseases.

All these accomplishments are made with cooperation with other rare disease organizations in the National Alliance for rare diseases of R. Macedonia.

Rare disease awareness

Rare disease day

We started marking rare disease day since 2012. In the past 2 years we are marking the day together with the organizations from the National Alliance for Rare Diseases of R. Macedonia.

Every year we manage to organize more events for raising public awareness with the help of our members and with cooperation with other organizations.

In 2014 we got the support from the wife of the USA ambassador in Macedonia Ms. Mary Jo Wholers and in 2015 and 2016 we had the support of the First Lady of Macedonia – Ms. Maja Ivanova.

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The part of “Life With Challenges” in Rare Disease Day, 2016 which we are very proud of was a promotion of the study for rare diseases that was realized in 2015 and can be downloaded at the following link – .

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Poster from the study was presented at ECRD 2016.

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We are proud to say that Rare diseases get more attention in the media and at the institutions in R. Macedonia. We hope that more events will contribute towards acceptance from the public and acknowledgment from the institutions.

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In 2016 we managed to organize events in Bitola with the help of Hepar Centre Bitola, in Prilep with the support of Jasminka Hristoska and in Ohrid with the support of Gordana Loleska and the Red Cross from Ohrid.

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Other important activities

Postal stamp for rare diseases

Last year a member of the association for rare diseases Life with challenges, a mother with a child with Alport syndrome, Gordana Loleska, employed in the post office in Ohrid, started an initiative for creating a postal stamp with the logo of rare disease day to help in raising awareness for rare diseases in Macedonia. This initiative was supported by many medical professionals in Macedonia who send letter of support to the Direction for philately to the Director Mr. Elez Elezi.

We did an internet search and we realized that this kind of stamp will be first of its kind – dedicated to Rare Disease Day to all the Children with Rare Diseases and it is a great initiative for the Macedonian post office, an accomplishment on world level.

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This year the post accepted this initiative and we hope that the stamp will be ready for distribution for rare disease day 2017. The name of the stamp will be Children with rare disease.

The stamp will be promoted by the national alliance fir rare diseases of Macedonia and also by the post office. It is a great idea that came true … a nice way to promote awareness … we hope that other countries will do this also.

We are very proud of Gordana Loleska who enthusiastically took this idea forward. We also want to say thank you to the general director of the post office Mr. Fadis Redzepi, to the director of the sector for philately Mr. Elez Elezi, to the director of the post office in Ohrid Mr. Sejfula Hani and to the council for philately at the post office of R. Macedonia, with its president Ms. Vasilevska Sonja.

Rare Disease Day Flag under water for New Year, 25 December, 2016, Ohrid, Macedonia

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Climbing the highest mountain in Western Europe – Mont Blank in France in support for rare diseases in Macedonia

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Climbing Korab, 4th of September, 2016

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Skopje Marathon – Running for rare diseases (from 2013 to 2016 and we will continue in 2017)

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Round table on rare cancers, 28th of October, 2016, Skopje, Macedonia with support from Serbia and Bosnia and Herzegovina

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Cooperation with doctors and researchersFIFTH MEETING OF SEE FOR RARE DISEASES, ON 12 NOVEMBER, 2016

The Macedonian Academy for science and art had the opportunity to organize the fifth meeting of SEE for rare diseases, on 12th November, 2016.

In the organizational committee of this meeting are: Momir Polenakovic, President, Zoran Gucev, Secretary, Nada Pop-Jordanova, Ilija Filipche, Zivko Popov, Velibor Tasic,Aspazija Sofijanova, Vesna Aleksovska, Dijana Plaseska Karanfilska

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Cooperation with others and membership in alliances

Internationally are actively participating in the board of directors of the European Gaucher Alliance – EGA, in the Drug Information, Transparency and Access Task Force in EURORDIS, and we are members of several organizations internationally and nationally: European Gaucher Alliance, EURORDIS, International Pompe Alliance, MDS Alliance, International Alliance of Patients Organizations, National Alliance for Rare Diseases of R. Macedonia,  Alliance for patient organizations – APO in Macedonia, an organization that is working on helping patients and families that face cancer. We also cooperate with organizations from the Balkan countries from Serbia, Croatia, Bulgaria, Bosnia and Herzegovina, Albania, Monte Negro, Kosovo, Slovenia.

Why do we think that cooperation is important?

  • Sharing knowledge in patient advocacy.
  • Sharing tools and best practices with patient organizations.
  • Learning how other organizations in the world accomplish their goals.
  • Getting support in policy development.
  • Getting support and new ideas for activities about raising public and institutional awareness about the problems and challenges of the patients and families.
  • Access to workshops, meetings, seminars and training on subjects important for development of strong patient advocates.
  • Access to new information of research and development of new medicines, new policies and new ways to help and support patients

Some pictures from conferences:

Second regional meeting of Gaucher patients and first regional meeting for lysosomal diseases  Sarajevo, Bosnia and Herzegovina,  5-7 April, 2013

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Second Balkan Conference for rare diseases – “Better communication – better treatment“, 20 – 22 April, 2013, Sofia, Bulgaria

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EURORDIS Membership Meeting, May, 2013, Dubrovnik, Croatia

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Patient Meeting Bulgaria – Macedonia, Skopje, 7 September, 2013

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NORBS – National Plan Conference, Belgrade, Serbia, 5-7 December, 2013

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DITA Task Force Meeting (Drug Information, Transparency and Access), London, UK, 8-10 December, 2013

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EUPATI – European patients academy on therapeutic innovation – 2014 – 2015

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Bulgaria – Macedonia Meeting, Kustendil, Bulgaria, 1-3 May, 2015

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