News/Press Releases

The European healthcare landscape is a dynamic, innovative ecosystem. Industry is increasingly identifying new approaches, solutions and business models that adapt to both mature and emerging growth markets. The pace of innovation is relentless, the potential benefits for society many.

At the same time, this is a highly regulated industry, working to the highest possible standards of safety and trust. Regulatory pathways are also evolving to adapt to the challenges posed by new therapies, new delivery methods and new health challenges.

To maintain user-driven innovation as an attractive investment for all stakeholders, the value chain needs to be ever more cohesive. Healthcare innovators, decision makers, regulators and payers must to work together to shape their environment. Future barriers should be minimised, potential benefits to society maximised and treatments delivered as quickly as possible to the patients that need them.

EuroMeeting 2015
The DIA EuroMeeting 2015 offers the ideal platform for healthcare innovators, patient advocates, decision makers and regulatory professionals to connect. They can network, scope potential new business partnerships and explore options for concrete solutions. This is why the EuroMeeting 2015 offers a significant return on investment for companies seeking to proactively manage a successful shift to a service oriented model.

How will you benefit?
Your core staff will not only learn about current and future market conditions issues. They can also spend time actively networking and building relationships with the regulatory and policy counterparts who are shaping trends. The resulting knowledge transfer and lasting partnerships will reduce the burden of regulatory barriers, benefiting your business now and further down the line.

Who will you meet?
The EuroMeeting attracts:
•    Corporate Executives
•    Pharmacovigilance professionals
•    Clinical development professionals
•    Regulators
•    Decision Makers
•    CRO’s/Consultants

What makes the EuroMeeting 2015 different? 

The DIA EuroMeeting 2015 is unique in presenting a microcosm of the entire drug development process ranging from the policy environment to market uptake and regulation.  It attracts attendees & exhibitors from all stages of the value chain, ranging from those conducting original R&D to the patients benefiting from innovation

– See more at: http://www.diahome.org/en/Flagship-Meetings/EuroMeeting2015/About-the-Conference.aspx#sthash.TxfQPRV1.dpuf

In an intense and stimulating three days, you will not only learn, you will be exposed to fresh thinking and the latest ideas. You will meet not only people you know, but also those that you want to know and those that you should know. – See more at: http://www.diahome.org/en/Flagship-Meetings/EuroMeeting2015/About-the-Conference.aspx#sthash.TxfQPRV1.dpuf

13 patients were attending this conference through the fellowship grant program of DIA. Vesna Aleksovska as a representative of Life With Challenges attended the conference. Patients interested in attending the conference next year are welcome to contact Life With Challenges and ask for information about applying for the fellowship grant.

The Gaucher Expert Summit is an international meeting which brings together, from around the world, healthcare professionals involved in the treatment and management of patients with Gaucher disease with the focus of “Driving Excellence in Patient Care”.

This educational event provides an opportunity to meet with other Gaucher physicians and to exchange views on best practice approaches to the management of patients with Gaucher disease.

Gaucher disease is a rare inherited disease caused by the deficiency, absence or incomplete functioning of an enzyme called glucocerebrosidase. Over time, this can result in the accumulation of a waste substance called glucocerebroside in cells, causing progressive damage to tissues and major organs including the spleen, liver and bones.

The Gaucher Expert Summit is an educational meeting dedicated to Gaucher disease. This international meeting brings together healthcare professionals involved in the treatment and management of patients with this rare condition.

With world-class faculty and through plenary lectures, lively panel debates and interactive workshops, delegates have the opportunity to engage, discuss and learn.

At this Gaucher Expert Summit, ‘Gaucher disease – Driving excellence in patient care’, an exciting and informative programme has been created to examine the key steps involved in delivering effective care for patients with Gaucher disease.

The international multidisciplinary faculty  discussed a range of topics that include:

• Strategies for earlier diagnosis
• Quality of life assessment
• Optimising therapeutic management over the long term

Chairs of this meeting were Professor Ari Zimran and Professor Hans Aerts.

This year the event was held from 17 to 18 of April in Amsterdam. Vesna Aleksovska was attending the event invited as a Board Member of the European Gaucher Alliance.

The GLF is a scientific meeting sponsored by Genzyme that brings together approximately 200 participants from around the world, including physicians, academics, researchers and patient organization representatives to discuss current care for patients with Gaucher disease and the direction of future disease management. This year’s GLF will be chaired by Timothy M. Cox, Professor of Medicine at the University of Cambridge. The theme for 2015 is: “New Clinical Science of Gaucher Disease” and will focus on the following objectives:

• Advancing therapeutics through greater understanding of pathogenesis (molecular & otherwise)
• Fostering collaborative and interdisciplinary research
• Inspiring (recruiting) the next generation of clinician researchers and scientists

“In designing this year’s GLF program, we have sought to encompass the hugely diverse aspects of Gaucher disease as they impinge on the lives and well-being of patients all over the world. It is chastening to realize that despite radical improvements over the last 25 years, many aspects of this condition remain very hard to treat; and even when the diagnosis eventually has come to light, not every patient can gain access to the best standards of care,” said Prof. Cox. “This meeting will give time for open sharing of experience and seeks to explore under-recognized aspects of the disease that are often neglected in the heat of routine practice.”

The opening plenary session highlighted the most recent scientific discoveries in the field of Gaucher disease research. Additionally, several interactive working groups have been established to focus on: (1) Spectrum of Clinical Manifestations in Gaucher Disease; (2) Gaucher Disease Type 3: Addressing Patients’ and Families’ Concerns; (3) Unmet Needs in the Assessment of Bone Disease in Gaucher; and (4) Gaucher Disease and Cancer: Mechanisms and Management.

“The GLF is an excellent example of global collaboration and partnership, with the Gaucher community working together to propel research forward and further our understanding of Gaucher disease,” said Genzyme’s Acting Head of Rare Diseases, Richard Peters, MD, Ph.D. “While much progress has been made, great need still exists, particularly around the neuronopathic forms of Gaucher disease. We look forward to lively discussion while we work to achieve our collective goal to support the needs of the global Gaucher community.”

From Macedonia, Vesna Aleksovska attended the forum, invited as a member of the Board of Directors of the European Gaucher Alliance.

EUPATI is a patient-centered team of 30 organizations, led by the European Patients’ Forum, with partners from patient organizations (The European Genetic Alliance, the European AIDS Treatment Group, and EURORDIS), university and not-for-profit organisations expert in patient and public engagement, along with many European pharmaceutical companies. The common goal is to help patient advocates be more educated and involved in health care.

The European Patients’ Academy (EUPATI), will provide scientifically reliable, objective, comprehensive information to you on the research and development process of medicines. It will increase your capacity to be effective advocates and advisors, eg, in clinical trials, with regulatory authorities and in ethics committees. It will also offer patients and the public basic information, training and education around health issues.

EUPATI aims to make a visible difference, showing you ways that you can have a more meaningful involvement in areas like drug discovery, the safety of medicines and access to treatments. We want to raise public awareness, your awareness, and make you better at navigating your health system. Find out more about the European Patients’ Academy on the link below.

http://www.patientsacademy.eu/index.php/en/

The first training course is already happening with 50 participants from all around EU. From Macedonia we have a participant from Life With Challenges. We hope to realize a basic training in Macedonia next year after the course ends and we translate materials in Macedonian language.

Next training course is starting at the end of this year.

At the congress you will also have a possibility to see the exhibition Hug Rarity developed by Lice v Lice, NARDM and Life With Challenges.

Rare Disease Day- Life with a Rare Disease: Day by Day, Hand in Hand

Respected,

On the occasion of the World Day of Rare Diseases the Association of Citizens with Rare Disease “Life with Challenges”, this time with joint forces of 12 organisations from the National Alliance for Rare Disease in Macedonia (NARDM) organized and event to mark the day with support from the activism platform “Face to Face” which issues a magazine with the same title.

Mrs. Maja Ivanova, First Lady of R. Macedonia and honorary patron of Life with Challenges stated:

“Rarity in the world represents uniqueness and prestige. Rare diseases are also unique, and patients with a rare disease are equally unique and their lives precious. Because every life is equally valuable it is important to have early diagnosis and suitable treatment for rare diseases.

I was priviledged to be  honorary patron of the event orgaised by EURORDIS in Brussels where  together we discussed the support in the area of research, treatment, early diagnosis as well as support for the patients. Rare diseases should be a priority both on European and national level.”

Mrs. Maja Ivanova greeted the patients and their families whose everyday struggle helps to raise the awareness on rare diseases. She urged not to emphasise the differences, but to build a better life together.

Mr. Ognen Janeski, public persona and activist of the platform “Face to Face”stated:

“The 29 stitches on my chest remind me of my struggle and my strength to persevere.” He reminded us how important it is to have and to give strength and support to rare disease patients through Azemina’s example, a girl suffering from the rare disease Gaucher,  who fights the rare disease with a smile. Ognen recounted his personal touching story when as a child he underwent a surgical procedure which saved his life and he emphasized that patients always come first and above all.

Mrs. Vesna Aleksovska, President of Life With Challenges  and NARDM stated:

“Rare diseases are a complex challenge which can only be surpassed with joint collaboration between institutions, patients, families, doctors and the pharmaceutical industry. In R. Macedonia for a very long time only a small part of the rare diseases were addressed (fully or partially) through the Health Insurance Fund. After the adoption of the Rare Disease Programme within the Ministry of Health in 2009 patients suffering from MPS 2 – Hunter Syndrome were taken care of . This year with the new law on excise tax one denar from each packet of cigarettes goes directly into financing the Rare Disease Programme. We are grateful for this measure both as associations and patients and I know that the doctors are grateful as well because they will be able to save more lives. With this change in the financing of the Programme we expect patients who have not been addressed by now by any institution and who struggled individually to receive proper treatment  to finally get the appropriate help. There are around 30 different diseases and the list of these patients is being compiled at the Ministry of Health in cooperation with the associations, patients and doctors. We hope that all patients will receive suitable help with which the families suffering a rare disease will finally get better quality of life. We will continue to work on improving the health and social services for patients and families with rare diseases , knowing that medicines are not the only thing we need, but a multidisciplinary approach which will also provide social help, necessary orthopedic aids and psychological help.”

At the event Certificates of Gratitude were awarded to the following individuals, state institutions and private companies:

1. Mrs. Maja Ivanova, First Lady of R. Macedonia
2. Mr. Nikola Todorov, Minister of Health of R.Macedonia
3. Mr. Momir H. Polenakovik, Macedonian Academy of Sciences and Arts
4. Prof. MD Aspazija Sofijanova
5. Agency for Medicines, R.Macedonia
6. Foundation Open Society Macedonia
7. US Embassy, Macedonia
8. Genzyme –Sanofi Aventis
9. Central European Initiative- CEI
10. Hoffmann-LaRoche
11. Vinary Brzanov
12. Brewery Star Grad
13. Foundation Makedonika
14. ONE
15. VIP
16. Kontura
17. CONCEPT Marketing and Communications
18. ALKALOID KONS Ltd
19. ALKALOID AD Skopje
20. Prof. MD Aco Kostovski
21. Prof.MD Zoran Guchev
22. Prof.MD Vesna Grivchev Panovska
23. Ass.MD Ivan Milev
24. Prof. MD Velibor Tasikj
25. Ass. MD Biljana Chonevska Jovanova
26. Nurses from the hematology ward, University Clinic for Children’s Diseases
27. NaTochak
28. To4ak
29. Red Cross of Municipality Gazi Baba
30. ZPK Korab
31. Natasha Dimitrievska Krivoshev
32. Zoran Mihajlovski
33. Dragan B.Kostikj
34. Valentina Bachvarovska
35. 

International Gaucher Day

25th of July (Friday) 2014, Skopje R. Macedonia

10:30 – 11:30 h.

Dear members and supporters,

The Association of citizens for rare diseases LIFE WITH CHALLENGES – Bitola, will organize an event for 26th of July, the International Gaucher Day, for the first time this year, as it is launched by the European Gaucher Alliance. The 26th of July 2014 has been selected as it is the 160th anniversary of the birth of French dermatologist Philippe Charles Ernest Gaucher who was born in Nievre on the 26th of July 1854.

 On 25th of July we are organizing a bicycle ride with the support of the City of Skopje, To4ak, NaTocak and Genzyme – Sanofi Aventis Company. The  event has a goal to raise public awareness about Gaucher disease. 

The bicycle ride will begin at 10:30 h., on 25th of July (Friday), from the park Jane Sandanski, with end point at Zena Park at 11:00 h., (map – http://0.mk/VozenjeGOUCHER) where we will have a short press conferences with statements from members and supporters of LIFE WITH CHALLENGES.  

Gaucher disease was named after Philippe Charles Ernest Gaucher as in 1882 whilst still a student he discovered the disease in a 32-year old woman who had an enlarged spleen. At the time Gaucher thought it to be a form of splenetic cancer and published his findings in his doctorate. It was not until 1965 that the true biochemical nature of Gaucher disease was properly understood.
The development of Enzyme Replacement Therapy in 1991 offered life-saving treatment to many Gaucher patients however there remains much unmet medical need and in many countries patients cannot get treatment as their governments do not have reimbursement programmes in place or are unable to support all patients who need treatment. It is for this reason the EGA has launched International Gaucher Day with the aim of:

• highlighting the existence of Gaucher disease and its signs and symptoms to create greater awareness to ensure the patients are at an early stage to prevent misdiagnosis and incorrect treatment
• highlighting the existence of available treatments
• highlighting unmet medical need particularly the lack of treatment for patients with rare neuronopathic manifestations
• highlighting the tragedy of countless patients in many parts of the world who are unable to access life-saving treatments
• highlighting the importance for further research into Gaucher disease with particular reference to the links that are emerging between Gaucher disease and more common conditions such as Parkinson’s disease

Throughout the 40 member countries of the EGA patients, families, doctors, scientists and healthcare professionals will be marking IGD by holding events and activities using promotional materials made available through the EGA office provided by the generous support of our corporate sponsors.

Sincerely,

Vesna Aleksovska,

President of the Association of citizens for rare diseases

LIFE WITH CHALLENGES Bitola

Contact: +389 70 705 446, vesna.stojmirova@gmail.com, zivotsopredizvici@gmail.com

Dear friends,

Association of citizens for rare diseases “Life With Challenges“, will join the Skopje Marathon, with a group running under the motto – Running for a better life.

Link to Skopje Marathon page – http://skopskimaraton.com.mk/ 

Link for participation in the marathon –  http://skopskimaraton.com.mk/prijavuvanje/5km/

Note: If you are applying to participate in our group for rare diseases, in the part where it says GROUP, please write TRCAME ZA PODOBAR ZIVOT, that is the name of the group running to raise public awareness about rare diseases.

Also please write to us so we know that you are running in our group and you’ll get a t-shirt with our motto.  (zivotsopredizvici@gmail.com)

In Macedonia there are over 18 different rare diseases and over 200 patients who live with rare disease. For some of these diseases there is a treatment and for some research is in progress.

Everyone who wants to support us as a participant or sponsor is welcome.

For now we have the support of HAE Macedonia and the Association of Physiotherapists of Macedonia and HEMOLOG.

Let’s raise public awareness about rare diseases in Macedonia together!

PRESS RELEASE

First CEI Conference on building Regional Network of Rare Disease Organisations

23-24 March, Sunday-Monday, 2014, Hotel Porta,

Skopje, R. Macedonia

The Rare Disease Citizen Association “Life with Challenges” has held the First CEI Conference on building regional network of Rare Diseases Organisations, on 23-24 March, 2014 in Skopje R. Macedonia.

This conference is a project that has brought to strengthening the capacities of organisations and increase of networking and communication among rare diseases organisations in the region of Central Europe. With this conference we contribute towards raising awareness for rare diseases (in reference to general public, institutions and medical professionals).

The wife of the President of RM, Mrs Maja Ivanova, the managing director of the Bureau for Medicines of RM, Mrs. Katerina Aleksovska, prof PhD Aspazija Sofijanova,MD, managing director of the Children’s Clinic in Skopje, as well as representative from the Health Insurance Fund (HIFM) and Prof. PhD Dijana Plasheska Karanfilska from MASA (Macedonian Academy of Science and Arts) addressed the conference.

As a honorary sponsor of the European Organisation for Rare Diseases, Mrs. Maja Ivanova emphasized the importance of rare diseases in the health care policies at a national and European level “Rarity does not mean rare involvement, on the contrary, it means great and dedicated work for changes in this area.” She also pointed out that in Macedonia through the relevant ministries programs for treatment of rare disease patients are regularly adopted through approval of funds for appropriate treatments. The involvement of the authorities in this direction is recognized in the fact that two years ago the Committee on rare diseases was established. She also called for the provision of better treatment, early diagnosis and to help in the accessibility in efficient treatments. Merely, working for better quality of life for each patient.

From the Bureau for Medicines of RM  it was emphasised that they appreciate the cooperation with patients’ organisations. It was pointed out that they also work on faster registration of medicines for rare diseases. Through a government project equipment has been provided for early detection of metabolic disorders. At the end Mrs. Katerina Aleksovska stated that through joint organised endeavors they will continue to provide therapy and treatment for rare disease patients.

Saving a child’s life is priceless. It is one thing to say, and it is something different when you meet the patient and when you need to provide treatment.  Thanks to Vesna from “Life with Challenges”, who made us think about these patients and take action to provide treatment, I stand in front of everyone to say what is necessary. With the Committee we make small steps, but what we do is relevant. The challenges wait for no man, as a parent, as human it is my duty to do everything I can for these children.” – stated the managing director of the Children’s Clinic in Skopje prof. PhD Aspazija Sofijanova, MD.

Mr. Branko Adjigogov, counsellor of the managing director of HIFM, Mrs. Maja Parnadjieva-Zmejkova, stated that so far they have provided gluten free products for several rare diseases, and in the following 30 days treatment for Alagille Syndrome and Huntington disease will be provided.

Prof. PhD Dijana Plasheska- Karanfilska from MASA greeted the initiative regarding rare diseases and emphasised that what is important for rare diseases aside from research and study is the early detection and prevention.  The centre in MASA does not stop researching them. We live in time of fast technological advancement when sequenced research enables us to recognize certain genetic mutations and recognize more easily rare disorders, with screening detection and prevention will be conducted more easily, and with that provide better quality of life for those suffering from rare disease.

The president of the rare disease patients’ organisation “Life with Challenges” emphasized the need of adopting National Plan for rare diseases.  The aim of the National Plan for rare diseases is improvement of the quality of life of those suffering from rare disease and their families in Macedonia. Health is basic human right, regardless whether it is about rare or common disease.

The lack of existence of this strategy would mean a threat to human rights of patients with rare diseases and their families Also a systematic access to the treatment of rare diseases would be disabled which as a consequence would have the lowering of quality of life of patients and their families, inability to work, increase of physical and mental disabilities and high incidence of births of children with rare diseases. This would therefore mean lack of relevant plans for health protection in the field of rare diseases.

Within the conference a Manifesto was signed with which the rare disease patients ask for sooner adoption of the National Plan for rare diseases. The Manifesto was publicly signed and supported by patients’ organisations from the region. The patients cannot wait any longer.

The second part of the conference was devoted to presentations by doctors and medical professionals who work in the area of rare diseases. Experiences were exchanged with medical professionals from the country and the region.

The conference is supported by CEI (Central European Initiative), Genzyme- Sanofi Aventis and Celegene Corporation.

PRESS RELEASE

Conference on creating National alliance for rare diseases of R. Macedonia

22 March (Saturday) 2014 hotel “Porta”, Skopje, R. Macedonia

The rare disease citizen association “Life with Challenges”-Bitola together with The Association for Hemophilia, rare coagulopathies  and Von Willebrand- HEMOLOG and the Association for Emancipation, Solidarity and Equality of Women of Republic of Macedonia- ESE oranised a Conference on Creating National Alliance for Rare Diseases of R. Macedonia  on  22 March, Saturday in hotel “Porta”, Skopje. The conference is part of a project that is realized within the framework of the USAID Civil Society Project implemented by the Foundation Open Society- Macedonia. Also the project is supported by Genzyme- Sanofi Aventis and Celgene Cooperation.

“I am very proud that such a project is supported by the USAID Project on Civil Society implemented by the Foundation Open Society. Working with Vesna from Life with Challenges I have come to know the problems and challenges people with rare diseases in Macedonia face. Regardless whether it is about rare, very rare or extremely rare diseases there is need of joint support and cooperation to provide necessary care. This alliance will strengthen the cooperation and will give patients a stronger voice in front of  the institutions. I have met few patients and every time I am amazed by the courage and perseverance of these people who do not allow the rare disease to define them. They are the heroes of our times. I encourage institutions in Macedonia to make all that is within their abilities in order to help the families facing rare diseases.“-stated  Mrs. Mary-Jo Wholers,  Embassy of the USA in Macedonia, honorary member of The Rare Disease Citizen Association “Life with Challenges”- Bitola.

Prof. PhD Elena Kjosevska, specialist in social medicine, Public Health Institute of Republic of Macedonia, managing director of the Department for promotion of health, analysis and prevention of contagious diseases stated: “The Public Health Institute will be involved in the activities that lead to improvement of data gathering, monitoring and situation analysis and with the support of the Ministry of Health  to establishing registries which will offer basis for careful planning of measures for improvement of conditions in the area of rare diseases. With joint efforts we can move towards improvement of the situation with rare diseases and I hope that we will make successful steps forward for the benefit of all families facing rare diseases.“

Borjan Pavlovski,  coordinator of the Programme on Public Health and Women’s Health  in the Association for Emancipation, Solidarity and Equality of Women of R. Macedonia-ESE, stated: When the Associations Hemolog and Life with Challenges approached us for the first time with the idea for and Alliance we were taken by the enthusiasm that they fight the fight for people with rare diseases. Human rights in health care guarantee that every citizen has the right to health. We are aware that these are complex diseases and the treatment is often expensive, but this does not mean that these people should not be provided for in order to contribute society as healthy citizens. We know that the problems cannot be resolved at once, but step by step the aim can be achieved.“

Mr. Branko Adjigogov, counsellor of the managing director of the Health Insurance Fund of R. Macedonia, stated: “Unfortunately in Macedonia still there is not an official registry for rare diseases and that is a big problem, especially because of the fact that we cannot know the financial implications in order to bring right decision regarding treatment and care for patients. We have managed to contribute towards provision of appropriate diet which is medicine for certain diseases such as PET syndrome and Phenylketonuria and we continue with the endeavours to provide suitable supplements for Alagile Syndrome and Tyrozynemia. We hope that with such small steps we will contribute towards normalisation of the situation.“

Vesna Aleksovska, president of the Rare Disease Citizen Association “Life with Challenges”- Bitola, stated: “The idea for an alliance emerged from our cooperation and we are aware that joined we can have a stronger voice as rare patients. We are aware of the lack of sufficient epidemiological data, the need of screening, the need for improved diagnosis, the obvious need for an increase of the budget for rare diseases and amendments of the legislation on rare diseases and many other steps that need to be made in order to reach better quality of life for patients with rare diseases. The Proposal National Plan that we have created according to European Regulative is what we consider as a foundation for improvement of the conditions.“

Cveta Nakjeska, founder and member of the Managerial Board of HEMOLOG – Association for Hemophilia, Von Willebrand and rare coagulopaties  stated: Entering this alliance is a benefit for all of us. We are creating it in order to work towards better quality life, and not survival. The motto of the World Hemophilia Alliance this year is – Speak out: create change.  From small steps and locked doors we walk towards greater steps and unlocked doors. I believe and I am certain that this alliance will achieve its aim and form paper it will move into action.“

The main aim that was achieved with this project is uniting about ten rare disease associations with the aim for joint endeavours in fostering and support of reform in the health-care system in the area of rare disease. The main focus with the establishing of the Alliance was defining the National Plan on Rare Diseases  which will provide long-term solutions for the problems and challenges rare disease patients and their families face.

Among patient representatives, patient organisations and representatives from relevant institutions from Macedonia, representatives of EURORDIS (European Organisation for Rare diseases), EGA (European Gaucher Alliance) and representatives of patient organisations from Bulgaria, Serbia, Slovenia, Bosnia and Herzegovina and Croatia were also present. They represented their national alliances and federations and National Plans and Strategies on Rare Diseases in their countries accordingly.

Contact:

Borjan Pavlovski ESE 02/3298 713, 02/3298 295, bpavlovski@esem.org.mk

Vesna Aleksovska, Life with Challenges 070 705 446, info@challenges.mk, zivotsopredizivici@gmail.com, vesna.stojmirova@gmail.com

Dragi Lulevikj, Hemolog, 071 796 247, info@hemolog.org, dragi.lulevik@yahoo.com