Real-life stories

Meet Jana


Jana and the uncertainties of access to SMA medicines in Bulgaria


Her smile bright, her enthusiasm contagious, Jana is a 33-year-old patient advocate from Bulgaria who lives with SMA.

The daughter of medical doctors, Jana was raised aware of her condition and of the possibility of gradually losing her abilities. This motivated her.

At 15 years old, she became involved in patient advocacy, first locally, then at the European level. Today, with her extensive experience and vast knowledge, Jana sits on many prominent advocacy positions in SMA, neuromuscular disease, and beyond.

When she first started her advocacy journey, Jana could never have imagined reaching the point of achieving medicines for SMA.

At one point, all these clinical trials started to really provide results! The SMA community became more visible, stronger, and more united. And at the same time, I wanted to pinch myself several times, just to make sure that this is really happening, and I'm not dreaming!

In Bulgaria, patient advocates began fighting for SMA medicines early on. Local families living with SMA formed lobby groups, met with politicians and national regulators, and pushed hard on the local government with the support of leading medical experts. A first medicine was made available for children in 2019. Then, in 2022, two of the three available SMA medicines became available to adults as well. The medicines are reimbursed through the National Health Insurance Fund, coming at no cost to individual patients. 

Access to medicines, however, is more complicated in practice than in theory. A national commission approves a total number of patients who are to receive each medicine, making access difficult for any new person seeking medicines, or anyone wanting to switch between medicines. New patients with more severe disease presentation are prioritized over milder cases. Treatment decision-making is shared between patients and clinicians, but a national commission has the final say on medicine approval. This approval, however, is only valid for six months, meaning that those who receive the medicine must resubmit updated lab and clinical assessments proving continued eligibility twice a year. 

In practice, this proves complex. Bulgaria only has two qualified medical centres who can run the assessments and administer the medication, both located near the capital Sofia. People living with SMA who do not live in the capital have to travel there often for their care, with financial and practical difficulties. Applications for reapproval cannot be submitted before the last dose of medicine has been consumed. Collecting all the documentation takes time, and the labs’ validity may expire before the request is even evaluated. Once the reapplication is received, it can take around two months to receive approval from the national commission. Then, the National Health Insurance Fund must officially notify local pharmacies before they can hand over the medicine. This means that people living with SMA who are already receiving medicines may have to wait weeks to continue their therapy, while all the bureaucracy is settled. 

People don't really realise what this costs to the mental health. All these tests every six months, and the fear that you might not be approved. We are still in a very delicate situation where we don't feel fully supported, as patients, that we are going to receive this treatment, no matter the circumstances.

All in all, Jana and her fellow patient advocates still have much to fight for, despite their success in achieving access and reimbursement for SMA medicines for all in Bulgaria. Their next goals are to raise awareness about SMA and available medicines. In adults leaving with SMA, many of whom are still wary of initiating treatment through medicines because they lack solid information in their native language. In local clinicians, to improve time-to-diagnosis and the delivery of state-of-art information about treatments. In the local government, to address pending needs including those for medical equipment that can improve quality of life. And, finally, in the society, to make sure that people living with SMA have equal opportunities for work, schooling, and full participation in social life.

Interview conducted on 4 March 2024

Maintaining access to SMA medicine is a challenge, not only achieving it. 

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