Commercial Considerations in the World of Rare Diseases

Emily, it’s a pleasure to see you today could you please introduce yourself and briefly describe what you’ll be speaking about at ATW23?

I’m Emily Chee, I’m the general manager for Novartis Gene Therapies responsible for the Americas. This includes the U.S., where we launched our gene therapy Zolgensma back in 2019, which has so far treated over 900 babies, Latin America, and Canada.

Zolgensma is a gene therapy for spinal muscular atrophy, without which those babies wouldn’t live past the age of two. It is remarkable from a clinical perspective, and has also been super fascinating from a commercialization perspective. Commercializing a $2m product has all kinds of interesting challenges, but it’s been a lot of fun pioneering. We are also now really close to reimbursement in Argentina and Brazil. It’s extra challenging really to bring these high-cost advanced therapies to middle and lower-income countries.

During my talk I will be sharing lessons learned from commercializing gene therapies. I wanted to share some of the lessons from our successes in the U.S., but also our journey as we are preparing to launch in Latin America. I thought that it could be meaningful for the audience to consider commercializing in both the more traditional first-wave countries that everyone launches in, but also the really important considerations for launching in lower and middle-income countries.

In terms of commercialization, I will consider the specific differences and difficulties in the rare disease space.

What are some of the challenges associated with commercializing these rare therapies?

There are a couple of key challenges in commercializing gene therapies. The first one is that the majority of diseases that we are targeting with gene therapies are rare diseases. There are often challenges with identifying these patients and getting them diagnosed with the right disease. Typically, these are also diseases that the earlier you treat them the better the outcome for patients. So we need to consider how to find patients and get treatment to them quickly.

I think the second thing I’d call out in terms of a challenge is that most advanced therapies, including Zolgensma, are high cost, and being able to demonstrate that value to payers to ensure access for patients, has been challenging in the U.S. It is even more challenging in lower and middle-income countries.

What do you think is needed in order for these therapies to improve the advanced therapies landscape?

There is a lot that’s needed to improve access to these advanced therapies. I think a key part is actually the strengthening of health care systems in general, particularly when we’re talking about lower and middle-income countries. Another key part does come down to patient identification – being able to identify those patients with rare disease.

Where do you see the field in five to ten years’ time?

I’m really excited for the future of gene therapies. It’s been great to see that other companies are starting to get approvals.

We are though still in the early stages, particularly when we’re thinking about the science. In five years’ time we’ll hopefully have multiple gene therapies on the market, and we’ll be providing therapies to rare disease patients who have been waiting a long time for a solution.

As an industry, we need to improve the science and manufacturing, and also tackle the commercialization challenges. 

What are you looking forward to about attending and speaking at Advanced Therapies Week 2023?

I’m looking forward to being able to network with other industry leaders, to be able to exchange learnings, lessons, and to figure out what needs to be done to continue to advance this space.

Don’t miss Emily’s session, ‘Commercializing Gene Therapies: Building Your Strategy From Translation Through to Commercial’ on Thursday, January, 19th 2023.
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