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Interview with Daniel Leonard, Director, Global Patient Advocacy, UniQure

Posted on: 11 April 2017, source: Gene Therapy for Rare Disorders 2017
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.




How do you think the gene therapy field will evolve over the next 5 years? It is incredible to see how many gene therapies are in development right now, and the prevailing wisdom says that we’ll see the first gene therapy approved in the US soon. Issues of access, pricing and reimbursement will have to be solved. If that can be accomplished, we could see a revolution in medicine.

What are the most significant challenges the field will need to overcome in order to fulfill its potential? There will be unique challenges for each specific development program, and there will be challenges that are common across all programs. Those include trial design, manufacturing and eventually, patient access.

What is your role at UniQure? I am the Director of Global Patient Advocacy. My primary focus is on hemophilia, and specifically supporting our gene therapy development program for hemophilia B.

What were the main challenges you encountered in building the patient advocacy function? uniQure didn’t yet have any connections to the hemophilia community. I needed to start from scratch in terms of mapping the advocacy landscape, and making the initial outreach. Having never worked in gene therapy, I needed to understand the technology, as it impacts how you interact with the community. Having never worked in hemophilia, I needed to understand the disease, and very importantly, the history of the community. The intersection of the technology and the history is certainly one challenging aspect.

Do you have any advice for companies looking to engage patient communities more effectively? Do not wait to engage. Even if there is risk in engaging early, be up front about the risk – you can even get advice on how to mitigate risk from patient community leaders.

How have you communicated with the rare disease community effectively? Understand where the mutual interest lies, and let that be your guide. This will show you the best path to partnership. It is not the same for every company or even every development program.

What challenges have you encountered that are unique to gene therapies? Grappling with the difficult past of gene therapy; dealing with the fact that expectations and hopes for gene therapy have been unfulfilled for many years; properly setting expectations for what can be expected from a gene therapy and dealing with the “cure” word; envisioning the access to gene therapies when there are little to no precedents.

What are you most looking forward to at the upcoming Gene Therapy for Rare Disorders conference? I am seeing all of the challenges and opportunities of gene therapy through my limited lens – I am looking forward to hearing from others who can share with me their experiences, as I am sure it will broaden my understanding of this fascinating world we are working in.

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Posted on: 11 April 2017, source: Gene Therapy for Rare Disorders 2017
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.
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