For patients with rare diseases to benefit, collaboration is key

On average, it takes 7 to 8 years for a patient with a rare disease to be diagnosed. Unfortunately, many times rare disease patients face wrong diagnosis, lack of cooperation and limited access to therapies. To make progress, strong collaboration between all three parties – patients, specialists and the pharma industry – is vitally important in order to benefit the patients. We asked HRA PHARMA RARE DISEASES , our valuable partner, several questions about the essence of this collaboration.

Why it is important to embrace a collaborative approach to effectively deliver therapies to patients?

In the case of rare diseases, the collaboration between the three main parties – patients, HCPs and the pharma industry – is crucial. With this collaboration, we mean working together on projects that help advancing science and research for the benefits of the patients and developing tailored projects that help patients cope with their disease. This is only possible when engagement, commitment, and trust exist inside this community.

Why do you think it is so important to have a dedicated and committed partner like Medis for orphan drug commercialization?

In order to meet the needs of patients, HRA PHARMA RARE DISEASES collaborate with dedicated and committed partners all over the world who share the same mission and vision, like Medis . These are partners who want to succeed and bring the medicines to patients while collaborating with their physicians.

What does it take, in your opinion, to have a long-lasting and?successful business partner relationship?

Common objectives including engagement towards patients and HCPs, respect, ethics and trust.

Commercialization of orphan drugs presents a unique challenge, especially in regions like the CEE with diverse and complex markets. What are, in your opinion, the main challenges and how can they be overcome?

Bringing orphan drugs to the markets, and in regions such as the CEE, could be indeed challenging. This happens due to the nature of rare diseases, mainly the delayed diagnosis and the misdiagnosis due to lack of awareness. Another challenge is the lack of knowledge around the rare disease and how to manage it inside the HCPs community. In addition, the access is a challenge, because of the different pricing that orphan medicines could have compared to common/non-orphan drugs, since not all payers have the experience and the knowledge of the rare diseases area.

Many rare diseases unfortunately still remain undiagnosed, so finding suitable patients is key. Therefore, advanced collaboration with specialists is crucial. What is Medis’ expertise that you find the most valuable when addressing this matter?

Medis is a valuable partner because they understand the business and are committed to organising activities to increase the awareness on different diseases through valuable collaborations with key opinion leaders and other HCPs, making sure to bring all necessary information to those who need it.