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Annual activity report – 2012

ANNUAL REPORT– 2012
Association of citizens for rare diseases LIFE WITH CHALLENGES – Bitola

The Association of citizens for rare diseases LIFE WITH CHALLENGES – Bitola is founded by patients and parents that face life with Gaucher disease in the year 2009. Gaucher is a disease that comes from lack of enzyme in the organism and there are problems because of storage of Gaucher cells in the liver, the spleen and the bone marrow. Because of that these patients have low hemoglobin levels, enlarged spleen and liver,weak bones, often fractures, lost of energy, fatigue etc.

Today in Macedonia there are 13 patients diagnosed with Gaucher disease, 4 of them are on Cerezyme donation bu the ICAP program and 1 of them is on clinical study on Eliglustat. They are receiving therapy for 3-4 years and all the symptoms are almost totally gone.  This therapy is a life therapy and patients should receive it every two weeks through infusion.

Later in 2012, the association started growing, more patients and parents joined with other rare diseases. First parents with children with Phenylketonura joined. This diseases causes brain damage and mental retardation, associated with physical difficulties. In Macedonia there are 7 patients with this disease and they provide food and medicine on their own wo they can provide normal life.

With Alagille Syndrome there is one child diagnosed in Macedonia. This genetic disorder is characterized with progressive loss of the liver function and in 30 to 50% of the cases there is cirrhosis, yellow skin, itching, problems with vision. To improve their condition and maintain normal development and growth this child needs treatment for the liver, the skin and the eyes and special diet.

In the association there are also patients with HAE, rare disease manifested by swelling (edemas) on different body parts. There is no cure, but there is treatment with C1 Inhibitor. In Macedonia there are around 16 patients with HAE.

There are other rare diseases in Macedonia as: multiple myeloma, phenylketonuria, myelodysplastic syndrome, gastrointestinal stromal tumors, recidivans, acromegaly, gastroenteropancreatic neuroendoc rine tumors, mucopolysaccaharidosis, hereditary tyrosinemia, galacorosemia, and hereditary fructose intolerance.

The association is open for members and other patients and parents that face life with rare diseases. In Macedonia there is a low level of diagnosis of rare diseases and we need to improve the area of diagnosis and treatment further. Patients often go in other countries for treatment and diagnosis.

Until now the Ministry of Health is treating only 2 patients with mucopolysaccaharidosis.

Activities and events in 2012:

• Continuous activity form 2009 until today is regular communication with the Ministry of Health for improvement of conditions for treatment of patients with rare diseases.

•  Continuous cooperation with Children Clinic Skopje and realization of meetings with the director of the Chidlren Clinic – Aspazija Sofijanova, who become a supporter of the association towards realization of our goals.

• In 2012, on 29th of February, Rare Disase Day, with a goal of raising public awareness and informing of citizens and patients, we organized a press conference with media and we talked publicly about our probelms and challenges. more information about the event you can read on the links bellow from media.
1. http://www.dnevnik.com.mk/default.asp?ItemID=6A2E3A7D78373743942077317D4FDEDB
2. http://www.denesen.mk/broj66/
3. http://kanal5.com.mk/default.aspx?mId=37&egId=13&eventId=87829
4. http://24vesti.com.mk/index.php/makedonija/293-makedonija/4774-2012-02-29-15-19-04
5. http://www.plusinfo.mk/vest/35152/Drzhavata-gi-ostavi-na-cedilo-pacientite-so-retki-bolesti
6. http://vesti.alfa.mk/default.aspx?mId=36&egId=6&eventId=46777
7. http://kurir.mk/makedonija/vesti/62626-Alarmantno-Za-deca-so-retki-bolesti-nema-pari-za-terapija
8. http://press24.mk/story/zdravje/%E2%80%9Ezhivot-so-predizvici%E2%80%9C-bara-terapija-za-site-pacienti-so-retki-bolesti
9. Telma television – news- http://telma.com.mk/index.php?task=content&cat=6
10. http://www.sky.mk/makedonija/16355.html?task=view
11. novinska agencija – http://mia.mk/default.aspx?mId=35&lId=1
12. http://vecer.com.mk/?ItemID=CF9BD888BBEDEE4AAE6F86A85D53EFA4
13. Focus daily newspaper
14. Sitel television – morning show and news
15. Alfa television – news, Portik and morning show

• Cooperation with associations and organizations: HAE Macedonia, HEMOLOG, Associations form Bulgaria, Serbia, Croatia, Slovenia, Bosnia and Herzegovina etc.
On EU level we are members of European Gaucher Alliance and now in 2013th we are also full member of EURORDIS.

• Projects: In 2012 we won the PAL award and we will use the resources in 2013th according to planned activities for campaign for raising public awareness for rare diseases (press conferences, events, publishing info material).

• Web page: http://challenges.think.mk/ , We have a new web page where we publish activities and information. We continue with communication through facebook group and fan page: https://www.facebook.com/groups/312483895490987/ , https://www.facebook.com/LifeWithChallengesi .

• Honorary member: Ms Mary Jo Wohlers, wife of ambassador of USA in Macedonia.

• Companies: cooperation with Genzyme (Sanofi Company) continues for raising public awareness about rare diseases. CONCEPT Marketing Communications is also a supporter of the association in the field of public relations, design, promotion materials etc.

• Media: media cooperation is on a very high level. All of them publish stories, articles and interviews about rare diseases and some of them filmed TV shows on the subject.

Future activities:

During our development we focused on contacting patients, uniting families and offering support and help in terms of information and treatment. As the membership is growing the association becomes stronger and more influential.

We are also making efforts in creating a register for patients with rare diseases so we can see the real situation in Macedonia regarding rare diseases.

Other activities are planned in cooperation and communication with the members and supporters of the association. We expect to come to long-term solution for provision of treatment for rare diseases in Macedonia in communication and cooperation with the Commission for rare diseases, the Ministry of Health, the Health Insurance Fund of Macedonia, the Bureau of Drugs and the Children Clinic. .

Sincerely,
Vesna Aleksovska
President od the Association of citizens for rare diseases
LIFE WITH CHALLENGES – Bitola


Rare disease day – 28th of February

On 28th February we will organize a media club/round table with journalists, representatives from the rare disease association “Life With Challenges”, doctor and representatives from the Health Institutions in Macedonia. The association will present its goals and explain the problems/issues of the patients with rare diseases. Further in an interactive discussion the doctors will answer questions from the present audience and they will advice about improving quality of life of the patients.

This event has a goal to raise public awareness and open a dialog between the interested parties. Patients and journalists will have a possibility to talk about the challenges they face every day and about the further activities of the association.

More details about the event … soon …


Balkan regional meeting of Gaucher associations

Dear members and supporters,

From 5th to 7th of April, in Sarajevo, Bosnia and Herzegovina, there will be a Gaucher meeting organized by the Balkan Associations from Slovenia, Macedonia, Bosnia and Herzegovina and Serbia.

The goal of the meeting is for patients, parents, doctors and nurses to talk together about life with Gaucher and how to improve quality of life in the future.

At the same time, this is a first meeting of patients with lysosomal diseases at Balkan region level.

The interactive program is on the following subjects:
– Mutual understanding and support

– Exchange of opinions and experience

– Questions and discussion about life with Gaucher

The meeting will be in hotel Europa in the center of Sarajevo and as official language we chose Serbian-Croatian with a goal to understand each other easier and better and to establish and maintain contact with each other.

This meeting is organized from the following patient organizations:
Association of patients for rare diseases from Bosnia and Herzegovina  – http://www.rijetkebolesti.ba
Association of citizens for rare diseases “Life With Challenges” – R. Macedonia– http://challenges.think.mk/
Association of citizens for help of patients with Gaucher  Serbia  – http://www.gose-srbija.rs
Association of patients with Gaucher – Slovenia  – www.gaucher-drustvo.si

Logistic and financial support for organization of the patient meeting is provided by:
Genzyme (Sanofi Company)
Association XY – www.xy.com.ba/bih

Sincerely,
Vesna Aleksovska
President of Association of citizens for rare diseases “Life With Challenges”


PAL Award – Patient Advocacy Leadership Award

The Association of Citizens for Rare diseases “Life With Challenges” from Bitola is a winner of the  PAL (Patient Advocacy Leadership).  As winner of this award are 8 more organization from the world.

The PAL Award recognizes patient advocacy organizations for extraordinary work and innovation in helping to raise awareness of lysosomal storage disorders (LSDs), a family of rare diseases that often affect only a small number of people around the world. LSDs typically cause progressive and debilitating health problems (http://www.genzymeadvocacyawards.com/2012-awards.aspx)

Fifty patient organizations from around the world applied for 2012 PAL Awards by submitting proposals for innovative campaigns and projects to support the LSD patient community.

Proposals were submitted from organizations that represent people affected by Gaucher disease, MPS disorders, Fabry disease, Pompe disease and Niemann-Pick disease, among other rare diseases. Applicants came from six continents, and 2012 winners were recognized in Chile, France, Hong Kong, Macedonia, Poland, Thailand, United Kingdom and the United States.

“For 3 years, “Life With Challenges” has been working to help people learn about and cope with Gaucher and other rare diseases. The PAL Award will help us to continue to build on these important efforts in the years ahead. We are very excited about the opportunity to expand our work and our impact through this important honor,” said Vesna Aleksovska, President of “Life With Challenges”.

According to the Global Genes Project, an estimated 350 million people worldwide are affected by rare disease. Only five percent of rare diseases currently have approved treatments and more than 50 percent of rare diseases do not have foundations, advocacy groups or organized community support to help expand awareness and improve access to care.
“The organizations that were selected to receive PAL Awards for 2012 showed a remarkable level of innovation and dedication in helping others to learn about and cope with lysosomal storage disorders. Genzyme is honored and very pleased to be able to support the work of “Life With Challenges” through the PAL Award program this year,” said Vulnet Ferrati, Genzyme representative from Macedonia.

 


Program for rare diseases in Macedonia

 

PROGRAM FOR TREATMENT OF RARE DISEASES FOR 2009 IN R. MACEDONIA

Rare or orphan diseases are diseases who appear in 5 cases in 100 000 population. The small number of cases with rare diseases is a limited circumstance for the pharmaceutical industry to undertake further research for these diseases. Although from every rare disease for now there are only few patients, treatment is socially and ethically relevant. The small number of potential patients is limiting for the economical attraction for undertaking, researching and development of treatment for rare diseases. The production process should be on a high level of safety, quality and efficiency as for the other developed treatments for other diseases.

According to the European Regulative for rare medical products (Regulative (E3) no. 141/2000) rare medicine (orphan drug) is defined as treatment a) for the condition that is life threatening and exhausting, b) condition where not more that 5 / 1000 000 persons are affected, or as treatment for which small return is expected economically and in c) condition for which there is no other alternative way of treatment or for which the new treatment will contribute a lot to the patient’s condition (benefits for the patient compared to the situation before treatment).

Unlike for other diseases, health experts have limited knowledge and skills for diagnosing and treatment for rare diseases. The small incidence of these diseases allows only limited number of health experts, usually in specialized centers, to build expertise for diagnose, care and treatment for patients with rare diseases.

Building awareness and expertise for rare diseases on EU level. Controled use of treatment can be connected with creating standardized registers for patients on international level and network of centers for expertise. Registers will allow direct estimate for number and profile of patients for research and budget purposes. Other key benefit of these kind of registers is direct knowledge about where the patients live and how they can be included in research for new treatment, what will be the benefit for them and for the company. At the same time, forming registers for the rare diseases will make easier to generate more data for benefits of further treatment.

This data will also contribute in creating basis for future decisions for prices and reimbursements. All registries and registrations should be in compliance with regulative for data protection and other relevant national regulative. For higher level of benefit, efforts should be coordinated on national and international level.

Early diagnosis for these kind of diseases, which are mostly genetic, is the best guarantee for efficient treatment from the aspect of therapy ant expenses. Therapy is mostly not curative, but usually provides limited or extensive symptomatic support.

National program for treatment of rare diseases has a purpose to provide availability of new treatment for the citizens of R. Macedonia. Until now in Macedonia there are 20 patients with rare diseases. The treatment in most cases is impossible to be provided from the family without any help from the state.

Diagnosis of rare diseases is conducted by experts in the clinics, depending of the nature of the disease, but mostly it is done in the Clinic for Children Diseases. In most cases, tests are done in highly equipped health institutions out of R. Macedonia, so the diagnosis can be confirmed.

Patients with rare diseases are treated in the Clinic for Children Diseases, and adult patients in other clinics. Most rare diseases are result of lack of certain enzymes.

In R. Macedonia the following diseases appear: Morbus Gaucher, Hereditary angioedema, multiple myeloma, phenylketonuria, myelodysplastic syndrome, gastrointestinal stromal tumors, recidivans, acromegaly, gastroenteropancreatic neuroendoc rine tumors, mucopolysaccaharidosis, hereditary tyrosinemia, galacorosemia, and hereditary fructose intolerance.

In the future there may be new diagnosis of other rare diseases and more patients with already diagnosed rare diseases, and they all should be included in treatment according to the medical criteria.

Measures and activities for treatment of patients with diagnosed rare diseases will be undertaken by public health institutions, depending form the nature and character of the diseases and the treatment. Public health institutions are obligated to report regularly to the Ministry of Health about diagnosed patients with rare diseases who are already mentioned in this program and for new diagnosed rare diseases that should be included in the program and therapy and treatment should be provided for them accordingly.

FINANCE NEEDED FOR REALIZATION OF THE PROGRAM

For realization of the program for treatment for rare diseases in R. Macedonia for 2009, with therapy and drugs that are used in treatment for rare diseases in 2009, 27 800 000 Denars are needed in the budget.

Number Activities Unit Total cost
1. Treatment for patients with rare diseases regardless of their insurance state 20 persons 27 800 000

Press – media announcements

Press from 29.02.2012 – Rare disease day – press conference

Daily newspapers

  1. http://www.dnevnik.com.mk/default.asp?ItemID=6A2E3A7D78373743942077317D4FDEDB
  2. http://www.denesen.mk/broj66/
  3. http://vecer.com.mk/?ItemID=CF9BD888BBEDEE4AAE6F86A85D53EFA4
  4. http://www.dnevnik.com.mk/default.asp?ItemID=099CEDCB5472FA47A4ABE965ED408DA2
  5. Focus – on line link not available

National televisions

  1. http://kanal5.com.mk/default.aspx?mId=37&egId=13&eventId=87829
  2. http://vesti.alfa.mk/default.aspx?mId=36&egId=6&eventId=46777
  3. News at 18.30h on Telma- http://telma.com.mk/index.php?task=content&cat=1&rub=6&item=14908
  4. Sitel – News at 15.00h- on line link not available
  5. Sitel – morning show – 8.30h – on line link not available
  6. Alfa – portik – tv show – 10.00-12.00 h. – on line link not available
  7. Local television from Tetovo – on line link not available

Новинска агенција / News agency

  1. http://mia.mk/default.aspx?mId=35&lId=1

Internet portals

  1. http://24vesti.com.mk/index.php/makedonija/293-makedonija/4774-2012-02-29-15-19-04
  2. http://www.plusinfo.mk/vest/35152/Drzhavata-gi-ostavi-na-cedilo-pacientite-so-retki-bolesti
  3. http://kurir.mk/makedonija/vesti/62626-Alarmantno-Za-deca-so-retki-bolesti-nema-pari-za-terapija
  4. http://press24.mk/story/zdravje/%E2%80%9Ezhivot-so-predizvici%E2%80%9C-bara-terapija-za-site-pacienti-so-retki-bolesti
  5. http://www.sky.mk/makedonija/16355.html?task=view

Press information

The government is ignoring 20 patients with rare diseases
With the budget cuts, the hope of the patients is gone!

A 12 year old girl with Gaucher will have to be operated if the Ministry of health does not make an intervene import of the needed medicine. The budget for the program with rare diseases has been cut during the last budget rebalance and there is no budget for treatment of the patients with rare diseases in Macedonia.

Mence Atansasova – Toci

The medicine exists but is not available for the 12 year old A.K. who has Gaucher. She will have to be operated because her condition is getting worse. Her spleen is enormously enlarged and her liver also. That is why the spleen has to be removed. But if she starts enzyme replacement therapy as soon as possible, she won’t need the operation. The girl was diagnosed 6 month ago and the parents noticed the signs when her abdomen was getting bigger because of the enlargement of the spleen and liver. The necessary therapy should be provided by the Ministry of Health. Her family is desperate, trying to provide the needed medicine. They contacted the Ministry but there is still no answer.

– My daughter has constant pain in her abdomen. The liver and the spleen are enlarged and she has to start treatment immediately. Otherwise her spleen has to be removed and that is not good. – says her father Shani K.

The medicine is expensive and it is not available in Macedonia. The company that produces the medicine is ready to provide the first 50 units of the medicine and they will wait for payment for a year if needed (if the government is not able to pay sooner). The association “Life with Challenges” is negotiating with the Ministry of Health to provide the needed medicine and save the life of the girl.

– We hope that the Ministry of Health will bring the right decision – providing the needed medicine. It is about saving human lives that we are talking and we need long-term solution for the patients with rare diseases in R. Macedonia. – says Vesna Stojmirova, the chairman of the association.

The newspaper “Den” asked the Ministry of Health for an answer about the pending issues regarding the condition of the patients with rare diseases, but there was no answer from the Ministry. It is still not known if the girl will have a normal life or a life with lower quality and psychical and physical obstacles.

In Macedonia there are around 20 patients with rare diseases and most of them are children. In the program for rare diseases it is stated that 1 million Euros will be provided for the treatment of these patients. With the last rebalance of the budget this program was ignored and with the budget cuts, the hope of the patients is lost. From the association “Life with Challenges” they say that through the program only 2 children with the rare disease – mucopolysaharidosis are treated and all the other patients are left without therapy.

The doctors say that if these patients are treated they can have normal lives. Without treatment they are facing serious health problems and most of them die at young age. The treatment for these patients will cost approximately 500 000 Euros. The company that produces the medicine for Gaucher is already donating treatment for 5 patients with Gaucher in Macedonia. In the past 4 years they donated around 5 million Euros for treatment and diagnosis. They are committed to treating the patients and that is a huge help for the government in Macedonia.

For raising public awareness about rare diseases in Macedonia, the association “Life with Challenges” will organize a campaign with the resources from the PAL reward (Patient Advocacy Leadership). For the PAL award, 50 countries in the world submitted applications and only 9 of them received the reward. The project “Do not cry fight for your rights!” will be a huge support in the fight for patients’ rights in Macedonia.


Documents for download

Click on the document that you want to download, there are documents in English and Macedonian:

Magazine “Face to Face”, 2015, Rare Disease Day

– licevlice br. 14  In Macedonian language

– Face to Face in English language

Annual report for 2015:

Izvestaj od aktivnosti za 2015

Activity report for 2015

Annual report for 2014:

Activites in 2014 – annual report

Finansiski izvestaj od Centralen registar 2014 – Zivot so predizvici – Financial report from Central register for 2014 – in Macedonian

Brochures from the conferences in March, 2014

Brochure – European regulative and recommendations in the field of rare diseases

Nacionalen plan za retki bolesti – broshura – National plan for rare diseases – Macedonian and English

Brochure from Rare Disease Day – 2014

Брошура – Ден на ретки болести Web – Rare Disease Day 2014 in Macedonian, translated from the rare disease day message and EURORDIS brochure princeps_document-EN

Brochures with patient stories

– Brosura – Prikazni na pacienti – Macedonian Version

– Brochure – Patient Stories – English version / Англиски

Membership Application

–  Пријава за членство – Macedonian, English and Albanian

Informative brochures for rare diseases in Macedonain 

– – Болест на Гоше – Gaucher Disease

– Епидермолисис Булоса – Epydermolysis Bulossa

– Наследна Тирозинемија – Hereditary Tyrosinemia

– Фенилкетонурија – Phenilketonyria

– Хередитарен Ангиоедем – Hereditary Angioedema

– Болеста Помпе – Pompe disease

Laws in R. Macedonia – regarding rare diseases and health

– Програма за лекување на ретки болести во РМ за 2012 година – Македонски

– PROGRAMA ZA LEKUVAWE NA RETKI BOLESTI VO REPUBLIKA MAKEDONIJA ZA 2013 GODINA – Македонски

– ZAKON_ZA_ZDRAVSTVENATA_ZASTITA_PRECISTEN_TEKST, Македонски

Reports and documents of the association

– Activities of Life with Challenges in 2013 – Annual Report 2013

– Official financial report 2013 from central register

– Life with Challenges – report 2012 – Report for 2012, Англиски

– Godisen izvestaj – Zivot so Predizvici – Rerport 2012, Macedonian version

– Статут на Здружението –  Macedonian version

– Do not cry fight for your rights – presentation at Gaucher  Patient Advisory Meeting at Waterford, Ireland, English

– zavrsna smetka 2011 – 2012 – Financial statement in Macedonian – revenues less than 2500 EUR in 2011 and 2012


Membership

Membership is free of charge and voluntary.
Members of the association are the founders, and all members of  the official bodies in the association.
Members of the Association can be Macedonian or foreign citizens, patients with rare diseases, members of families with patients with rare diseases, supporters of the mission, goals and activities of the association.

You can become member in the Association with filling out an application form.
Each members is being informed timely for the activities of the association.

If you want to become our member, please download the application bellow – just click and it will start with download. After you fill it out, send it to  zivotsopredizivici@gmail.com, or info@challenges.mk.

– Prijava-za-clenstvo1 / Membership Application


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