We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them. Our innovation begins with understanding people living with rare diseases, which fuels all of our efforts. For us, every patient story is important and inspiring, for Diplomacy&Commerce says Vladko Borić Business Unit Director Rare Diseases AstraZeneca Balkans. People suffering from rare diseases also have their dreams, wishes and aspirations, they have families, loved ones and friends. We have medications indicated for the treatment of atypical hemolytic uremic syndrome, paroxysmal nocturnal hemoglobinuria, enzyme replacement therapy in patients with hypophosphatasia, enzyme replacement therapy in patients with lysosomal acid lipase deficiency, etc, says Mr. Borić.
1. AstraZeneca has a developed portfolio in many therapeutic areas. What is the company’s motivation to enter the rare disease market?
Whether a population of 5 or 5,000 will be affected, behind each number is a concrete human destiny. And this is our mission – to help patients have a better quality of life. Rare diseases are actually a special therapeutic area because they affect fewer than 5 in 10,000 people. On the face of it, it seems like a small percentage, but if we look at the global picture, we arrive at the significant figure of nearly 400 million people who suffer from some form of rare disease worldwide. Add to that the fact that more than 7,000 rare diseases are known to date, but only 5% of them have a therapy or an approved treatment, and the picture becomes even more complex. Unfortunately, 50% of those suffering from rare diseases are children. Given all that, I think it’s more than clear why we’re committed to turning this statistic around. There is a great unmet need, and in order for progress to be made, a strong focus on research and development is required, in which we as a pharmaceutical leader are investing heavily. Over the past decade, innovative treatments have succeeded in making the transition from supportive care to treatment of the underlying disease, significantly changing the quality of life for people with rare diseases and their families. That is why, with joint efforts with our partners from the health system, institutions and patient organizations, we strive together to contribute to access to innovative therapies. Even before the acquisition of the company Alexion, which is extremely focused in this area, we at AstraZeneca had experience with rare diseases and provided solutions for some of them such as e.g. neurofibromatosis type 1. From October 2022, for the territory of the Balkan countries, we are working even harder in the field of rare diseases thanks to the formed specialized team. I am happy and proud of the dedication with which our colleagues work and the results are there – by the end of April this year, nearly 100 patients with rare diseases in the Balkans are being treated with one of our innovative therapies.
2. What are the therapeutic indications of your medicines in the field of rare diseases and what is the company’s focus on rare diseases?
We have medications indicated for the treatment of atypical hemolytic uremic syndrome, paroxysmal nocturnal hemoglobinuria, enzyme replacement therapy in patients with hypophosphatasia, enzyme replacement therapy in patients with lysosomal acid lipase deficiency, etc. We have many goals to focus on, but for starters, we are working hard to raise awareness of rare diseases because it plays a key role in diagnosing patients and prescribing the right therapy at the right time. I am extremely grateful to the colleagues of the team of Rare Diseases in the Balkans cluster, who take the mission to ensure access of innovative treatments to heart and work dedicatedly with local partners, institutions, doctors and patient organizations for a better health ecosystem and access to treatment.
Working in a multinational environment and a cross-functional model, they draw knowledge and experience from
each other, thereby applying best practices that ultimately lead to greater benefits for patients and society.
3. After the acquisition of Alexion and AstraZeneca’s entry into the rare disease market, what is the company focusing on in the Balkan countries and more specifically in Croatia?
As mentioned, the main challenges we face in the Balkan markets and on which our focus is directed are patient access to medicines and awareness of these diseases. Rare diseases are not a frequently discussed topic and the public, as well as a large part of specialists, are not aware of their specifics. For rare diseases, timely diagnosis is still an extremely complex process. In the most general case, until the correct diagnosis is reached and until it is established that a rare disease is present, patients have to visit doctors from different therapeutic areas repeatedly and waste valuable time. This is mostly due to the fact that many of the symptoms overlap with other diseases. Unfortunately, in many cases, late diagnosis ends fatally for the patient. Having said all this, Croatia is no exception. With our colleagues, we are working hard on the awareness and awareness of both partners and healthcare institutions, as well as the wider public. Our focus is also on early diagnosis, as well as ensuring access to medicines to reach the Croatian market, and from there to the Croatian patient.
4. You work as one team for the entire Balkan cluster, but each country has its own specifics when it comes to rare diseases. Is there any successful formula that applies to all markets?
That’s right, in each country there are many factors that determine whether a patient will receive therapy for the respective rare disease. We recognize the complexity of the situation facing healthcare systems, so we work with all stakeholders to find a way to help more patients have access to innovative therapies by organizing early access programs or by stimulating discussions and conferences on national registries and traceability of patient numbers. Something that we find very useful and that we try to implement is to connect health professionals from the Balkan countries to exchange valuable experiences and thus contribute to the faster diagnosis of patients. It is inspiring to be able to help even 1 patient, because in fact we will help at least a few more people – his relatives, to improve their quality of life. And they all have their own specific human story, and this is what motivates all of us to work even harder and more focused in the search for solutions. The truth is that a diagnosis, even if there is no available therapy for the disease in question, can at least help patients and their loved ones clarify the situation – what to expect and think about how to deal with what lies ahead. Our strength lies above all in common and united efforts. We interact with colleagues from other countries as well. We work in cross-functional teams to deliver end-2-end strategies and leverage the experience and knowledge of everyone involved in the process