Interview with Nick Sireau, CEO of AKU Society

In Orphan Drug Congress by wingyun

The 5th World Orphan Drug Congress on 12- 14 November in Brussels is just a couple of months away and I recently sat down with one of our key speakers, Nick Sireau (the Chairman and CEO of the AKU Society) for a short interview.

Dr Sireau will be addressing the importance of having an all-stakeholder perspective of clinical trials for the rare disease AKU (Black Bone Disease). Dr Sireau is also the Co-founder and Chairman of Findacure, a new social enterprise that raises awareness into, and helps patients with, extreme and exceptional diseases that advance understanding of medicine and help discover potential new treatments. As a committee member of Rare Disease UK, the national alliance of rare disease groups in the UK and of the International Rare Disease Research Consortium (IRDiRC), Dr Sireau has extensive experience in putting together rare and ultra-rare clinical trials.

Nick Sireau, CEO AKU Society, image edited from

Nick Sireau, CEO AKU Society, image edited from

What is the importance of having an all-stakeholder perspective of clinical trials, addressed in your talk?

Clinical trials increasingly need an all-stakeholder approach in order to be successful. Our experience with DevelopAKUre, a consortium developing the drug nitisinone for the rare disease AKU (Black Bone Disease), shows that bringing together clinicians, academics, industry and patients on equal terms is an excellent way of overcoming the challenges involved in rare disease trials. Each party brings its own perspective, resources and skills to the consortium – allowing us to develop innovative ways of devising and implementing the trials.

What are some of the most pressing issues you are facing in implementing this?

As is often the case, a key challenge is funding. Despite the €6 million from the European Commission (EC) and the in-kind contributions of consortium members, we are pushing our resources to the limit. On the one hand, this has forced us to be even more creative in our approach. On the other hand, it shows how difficult it is to carry out public-private partnerships in areas of market failure such as in many rare diseases. Without funding from the EC, it would be difficult for many rare disease groups to work on new treatments for their disease. That’s why we need a variety of funding avenues, not just from international sources such as the EC, but increasingly from national and philanthropic sources.

What do you think will be the single biggest trend or change to the orphan and rare disease sector between now and 2025?

I hope the biggest change in the orphan and rare disease sector over the next 10 years will be a surge in patient involvement in clinical trials. Patient groups have a passion and clarity of vision that can bring together all the other actors in the process by focusing on the need to develop cures fast and effectively. They are also increasingly skilled in their approach, thanks to the training and support provided by umbrella groups such as EURORDIS. If this continues, I hope we will see more projects where patients are leading the clinical development of treatments.

What would you like to gain from our congress?

The World Orphan Drug Congress is a fantastic opportunity for finding out about the latest developments in the field of orphan drugs and rare diseases. It’s also an excellent networking opportunity. I’ve made a number of very good contacts there in the past that have led to some of the key collaborations that are the centre of our DevelopAKUre consortium. I hope to gain similar knowledge and contacts at this year’s congress.

Click here to book your place so you can meet key speakers like Nick Sireau as they share solutions for collaborative orphan drug clinical trials.