In spite of some recent bumps, gene therapy remains one of the most exciting and dynamic areas of healthcare. Since 2017, but especially over the past year, a number of companies have produced promising clinical results for their respective gene therapies and received regulatory approval from the Food and Drug Administration to bring their products to market.
Many of these therapies target rare diseases and give patients, who live with previously incurable or untreatable conditions, a chance to reclaim some aspects of their health. MM+M’s digital editor Jack O’Brien recently spoke with Tom McDonnell, president of the BW Health Group, Ben Tempel, principal at Frameshift Advisors, and Courtney Rice, principal at Acadia Strategy Partners, about best practices and strategies for launching gene therapy products.
What are some hurdles gene therapy companies encounter when preparing for a product launch?
McDonnell: Launching gene therapies requires a different playbook — and not even a playbook that can be leveraged from one product to another. There is a risk of using the same strategies and tactics that may have worked in years past and applying them to gene therapy. We are seeing that for the most part; it doesn’t translate. This is where there needs to be some change, some education, some learning.
Tempel: Every launch is completely different: you’ve got different therapeutic areas and specialists, different patient groups and even varying levels of clinical, financial and operational savvy at treatment centers, that need to be approached and communicated with in vastly different ways. It’s definitely a paradigm shift in the way we need to provide care to the patients. The operational role of the treatment center is really critical.
Rice: Not only are these gene therapy launches unlike specialty or large molecule launches we’ve seen previously, but with each subsequent gene therapy launch we’re learning from the previous one. We’re learning in real-time. You have to be comfortable with uncertainty and you have to be energized to navigate it without clear analogues or benchmarks. I’d also add that the role of evidence planning, registries and market access planning are crucial in light of the larger price tags, that are offsetting costs on both the medical and pharmacy benefit.
What are key factors that contribute to a successful gene therapy launch?
McDonnell: Historically in life science, there were traditional silos by functional area and each one of those silos had a job to do. In gene therapy it seems necessary to break down those age-old silos, embrace coordination and collaboration because these areas that are so critically dependent on each other, not only for treatment, but also to find and qualify patients. There may be different roles, different titles, emerging areas that we haven’t even thought about yet that will help in the future to break down those silos and what will emerge in their stead is more targeted and precise capabilities that meet the unique challenges these therapies come with. I think the most pronounced element we’ve seen in our clients’ gene therapy launches is how we are leveraging data to increase our precision in finding and communicating with stakeholders.
Tempel: Best laid plans — strategy doesn’t always go the way you want them to, and having flexibility in your strategy and how you set up your program is so important when you launch and when you start dosing your first patients. You can see what hasn’t gone as well as you wanted, and you need to be able to quickly pivot to optimize your program. You don’t want to get boxed into one way of doing things, whether it’s in your distribution channels or the way you’re working with your treatment centers and your physicians.
Rice: Each drug launch truly is unique to its therapeutic area, to its audience, to the age of the patient. All those fit into this multi-combination complex lock and you can’t just apply a templated, one-size-fits-all strategy to it. And no therapy can succeed without strong relationships with administration centers, which in turn demands a new set of skills from marketing partners.
What measures do you recommend for evaluating the impact of a gene therapy?
McDonnell: There’s certainly the number of patients that have received treatment and the number of centers of excellence that are administering these gene therapies. This brings up another key point which is that you can’t get to the number of patients unless you have taken the time to get your centers of excellence online and educated. Sometimes this is the first time these centers are administering a gene therapy, because they were not necessarily part of the clinical trial program. This makes communications even more critical.
Tempel: There are many metrics to follow but one important idea to keep in mind when setting KPIs is that in gene therapy the time to revenue is different. It’s not a traditional launch where on day one of launch, the channel buys a product from you and you have instant revenue, so you definitely need to have a more holistic approach to measuring success. The patient journey to treatment is quite complex and evaluating success at each and every step is important. Everything from how many patient referrals are coming in, how many patients are getting insurance clearance and eventually how many patients are getting treatment. If you track and evaluate success at each point in the journey, you will not only have a better handle on what is transpiring with your launch, but you will also be able to take action if there are any bottlenecks that arise.
Rice: We’re watching what everybody’s doing, across AAV, Lentivirus, Synthetic, LNP. We make prudent decisions based on the data at hand.
What are some industry trends that are influencing product launches?
McDonnell: We need to focus on creativity, health literacy and finding patients faster. We also need to be able to educate healthcare professionals that have been treating their patients one way for their entire career and then ask them to weigh a transplant versus a cell or gene therapy for example.
Tempel: Ensuring we always come back to the patient and that we’re making sure that we take as much burden from them and their families as possible. That’s something as an industry we’re very good at — let’s keep it going in gene therapy.
Rice: Being aware that we have to make some discerning choices. Our clients are often small biotechs who need a critical path to launch. We help them make deliberate inclusion and exclusion choices when it comes to steps in the launch. There’s an inclination to check every box if you come from Big Pharma with big resources and codified, regimented launch playbooks based on years of best practices. But in biotech and in this funding environment, we often have to launch on limited dollars and skeletal budgets. We often hear “I have to do more, with less.” Sometimes that means that we’re reliant on a lot of outsourcing, which is great … right up until it’s not. Getting great at launching on a slimmed-down budget demands a hunger to fight through complexity and unknowns and, identify a critical path to launch. It really demands discipline and data. Lots of data.
