When I first started working in the rare disease and gene therapy space, I thought the science would be the hardest part to wrap my head around. And don’t get me wrong—it’s complex. But what I didn’t fully appreciate at the time was just how challenging When I first started working in the rare disease and gene therapy space, I thought the science would be the hardest part to wrap my head around. And don’t get me wrong—it’s complex. But what I didn’t fully appreciate at the time was just how challenging commercialization would be.

Launching a gene therapy for a rare disease isn’t like launching a traditional drug. The patient populations are small and geographically dispersed. The cost is staggering. The logistics are intense. And yet, the potential to change lives—permanently—is unlike anything else in healthcare.

That’s why I believe competitive intelligence has never been more important. In this space, every decision—from indication selection to pricing to access—requires strategic foresight, not just technical expertise. would be.

Launching a gene therapy for a rare disease isn’t like launching a traditional drug. The patient populations are small and geographically dispersed. The cost is staggering. The logistics are intense. And yet, the potential to change lives—permanently—is unlike anything else in healthcare.

That’s why I believe competitive intelligence has never been more important. In this space, every decision—from indication selection to pricing to access—requires strategic foresight, not just technical expertise.

A Different Kind of Commercial Strategy

In rare diseases, everything about the launch is personal and nuanced. There’s no broad DTC campaign. There’s no “standard” payer model. There might be only a few hundred eligible patients in a country—or even fewer.

What’s needed is a hyper-targeted, insight-driven approach, and that’s where CI becomes indispensable. For me, it’s the tool that answers the questions that don’t show up on the label or in the press release:

• Which competitors are building relationships with patient advocacy groups?
• How are early access programs being rolled out—quietly or aggressively?
• What kind of real-world evidence is being collected post-launch to justify reimbursement?

When you’re trying to commercialize a one-time treatment that costs $2 million, you can’t afford to be reactive. CI helps you anticipate where the roadblocks will be—and where the white space is.

Getting to Patients—Faster and Smarter

I’ve seen gene therapy teams stall because they underestimated the diagnostic bottleneck in rare diseases. Many patients go years without a diagnosis. And when they finally get one, they’re often ineligible due to disease progression.

CI helps us understand where other companies have succeeded—or failed—at finding patients faster. Are they investing in newborn screening partnerships? Deploying decentralized diagnostic tools? Working directly with academic centers?

The teams that win in this space are the ones who embed themselves into the diagnostic journey, not just the treatment decision. Competitive intelligence gives us the map for how to do that.

Reimbursement: The Next Frontier

Gene therapies are rewriting the rules of value and access. And while the science may be curative, payers still live in a world of budgets and actuarial math.

CI helps uncover:

• How are payers reacting to outcomes-based agreements?
• What’s the uptake of annuity-based payment models or risk-sharing structures?
• Where are HTA bodies pushing back—and where are they showing flexibility?

I’ve worked with teams that shaped their pricing and access strategies by understanding exactly how competitors stumbled—or succeeded—in front of payers. That kind of insight doesn’t just inform tactics. It changes boardroom decisions.

Building the Right Partnerships

No one launches a gene therapy alone. Whether it’s manufacturing scale-up, distribution, data collection, or global expansion, partnerships are everything. But again, not all partners are equal—and not all partnerships are public.

CI gives us the edge by revealing:

• Which CDMOs have real experience with viral vector delivery?
• What are advocacy groups actually saying about existing therapies?
• Which territories have local players with payer credibility?

In a space this capital-intensive, I’ve seen competitive intelligence make or break a deal. When you’re investing hundreds of millions into an ultra-rare asset, you need to know who’s truly aligned—and who’s just along for the ride.

Final Thoughts

Gene therapy and rare disease commercialization isn’t just hard—it’s redefining how we think about delivering healthcare. The promise is massive, but the margin for error is razor-thin.

That’s why I lean so heavily on competitive intelligence. In a field where every patient matters, every dollar counts, and every move is scrutinized, CI helps me navigate uncertainty with confidence.

It’s not about watching the competition for the sake of it. It’s about understanding the full landscape so we can bring life-changing therapies to the people who need them—faster, smarter, and more sustainably.