Gaucher Disease Patient and Activist Successfully Improves the Care for Other Rare Disease Patients in Her Country

This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia.

North Macedonia is a developing country located above Greece. It has 2 million inhabitants. Unfortunately, very few doctors in the country are well-informed (let alone experts) on rare diseases. When you have a rare disease, you want your doctor to be an expert. For the longest time there were no patient registries in the country and therefore, the Health Ministry had no knowledge of how many of their citizens had a rare condition or even which rare diseases existed in the nation. Additionally, the national health insurance fund only covered a few select therapies for a few select rare conditions. Funding for treatments for Gaucher disease for instance, were not covered due to their expense. This was just 10 years ago.

Vesna Aleksovska

When Vesna Aleksovska was born in North Macedonia there was no newborn screening. She wasn’t diagnosed with Gaucher disease until the age of 7. She was only able to receive the treatment she needed (Cerezyme) through help of a special program created by the manufacturer of the drug. The now 34-year-old is determined to make a change in the way her country handles rare diseases. She believes that no rare disease patient should have to struggle to get the treatment they need.

Vesna is founder and president of Life With Challenges, an organization formed in 2009 dedicated to improving the care of rare diseases in the country. She is also the chair of the International Gaucher Alliance. Vesna’s very first goal when she began her advocacy journey was to stop the complacency she clearly saw among healthcare professionals regarding rare conditions.

First Steps

First, Vesna knew she had to plan her approach on how to get politicians more aware of rare diseases and the challenges rare patient’s face. Thankfully, she already had the resources and experience she needed. She had 10 years of work experience in the journalism field including radio, television, print, and online news. This experience told her that turning to the media with compelling stories is a fast and effective way to spread awareness and spark change.

Although some patients were reluctant to share their stories, Vesna was able to recruit three other storytellers for her mission. Together, they held their very first press conference in 2012.

The outcome was better than they could have ever imagined. The conference attracted not only the government’s attention, but the attention of rare patients around the world. There was an influx of messages from individuals across the globe wanting to get in contact with Life with Challenges. It was just what the rare community in North Macedonia needed.

The team didn’t stop there, and they didn’t stop with one disease. They discussed pulmonary hypertension, Alport syndrome, spinal muscular atrophy (SMA), and Huntington’s disease among others. Vesna was not afraid to ask the health minister, finance minister, and the prime minister, why they weren’t taking the action that was needed. She and her team participated in interviews and made appearances on talk shows. There were newspaper articles published about their mission.

Dragan Dimitrijoski was one of Vesna’s brave storytellers. His son who is just 6 years old has phenylketonuria (PKU). There are treatments for PKU, but unfortunately none of the therapies were available in Macedonia and Dimitrijoski actually moved his family all the way to Sweden so that his son would have access to PKU resources and specialists.

For too many families, these were the hard decisions they were forced to make.

The Outcome

Vesna’s efforts produced an exquisite result. Healthcare experts across the world took note of her publicity. After three years of dedicated work, Macedonia’s Health Minister, Prime Minister, and the head of the health insurance fund vowed to make a change.

The Health Ministry created a patient registry, the insurance fund expanded coverage to numerous rare diseases and their treatments (including Cerezyme), and the budget for rare disease coverage has been increased.

The change of Health Ministers in 2018 did not sway progress, with the new representative continuing the momentum that had begun. In fact, he has engaged with more rare disease medical professionals and patient advocacy organizations than his predecessors.

 “The Health Ministry has gone from ignoring the community to partnering to help rare disease patients and caregivers.”

Vesna’s success is proof of the impact a singular voice can have on the state of rare healthcare. Of course, Vesna could not have done this without the help of her other storytellers and the journalists who covered her events. But it was her spark, determination, and drive, that ensured that this project began and was completed.

That said, the work is never truly done. There is always more progress that can be made- in North Macedonia and across the globe and Vesna has continued to use her voice to encourage rare patients to speak out for their rights.

You can read more about Vesna’s story and her impact here.

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