What Baby KJ Taught Us About mRNA COGS

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

Over the past few weeks, I’ve been publishing a series of articles from the AMM/Franklin Biolabs event, “Building RNA Medicine Excellence.” In my first takeaway from this event, I discussed three core pillars of expansion for the mRNA industry. But as I prefaced in my most recent article, “What Baby KJ Taught Us About Sustainable mRNA Development,” there was one panel discussion that deserved a second look, particularly because it laid bare many of the under-discussed realities behind the Baby KJ story.

As we all know, such a heroic tale is worthy of celebrating; after all, this is just the kind of experience we needed in the mRNA industry to help showcase mRNA’s therapeutic capacity. However, as I outlined in part one, there were also reasons why the Baby KJ story was a unique (and at times, quite lucky) situation. This is why I loved the discussion that took place between JHU’s Jeff Coller, CHOP’s Rebecca Ahrens-Nicklas, and Plowshare Therapies’ Kevin Strauss. Together, these experts delved into where the extraordinary and the realistic converged within the Baby KJ story and shared how we could move forward in a more sustainable fashion.  

Here, in part 2 of this three-part series, I share a few details that came to light about the COGS behind Baby KJ’s therapy production. Obviously, we know why it’s important to keep these figures in mind as we move forward; but I particularly liked Strauss’ reminder that it’s our job to “think systematically about how we’re going to create a safety net of sustainable, accessible product that can be widely distributed at an affordable cost.” And to do that, transparency around the cost of this experience can go a long way to helping us understand where we are starting and where we can and must go from here.

The Real Math on N-of-1 COGS vs. Standard of Care

Arguably, one of the most important “curtains” we needed to pull back on the story of Baby KJ was that of the costs associated with the development and treatment. As we already know, access was provided via an expanded access IND, meaning we weren’t dealing with traditional drug payment/reimbursement practices. As Ahrens-Nicklas admitted, some of the COGS were paid for and some of the effort was “in kind.” But we should all be heartened to hear that rumors of such a therapy coming with an $8 million-dollar price tag are inaccurate. 

In most conversations about drug pricing, we often make comparisons between a novel therapy’s cost and the cost of the current standard(s) of care. This conversation was no exception. For urea-cycle disorder, pediatric patients typically receive a liver transplant — a procedure that costs roughly a million dollars. Prior to this surgery, most patients will receive nitrogen scavenger medications, which come with a $750,000/year price tag.

If we consider this, Ahrens-Nicklas acknowledged that a single batch of an LNP-based editing drug product comes in under current standards of care costs. “In terms of manufacturing of batches, it is more on the order of magnitude of what you would expect for a liver transplant,” she said.

However, it’s also important we remember that making a therapy accessible will require a BLA, which is accompanied by the high costs of assay validation, PPQ batches, and more.

“If I’m giving my patient an approved therapy, I want to make sure it’s safe and that it’s manufactured in a way that is reproducible and robust,” she added. “But that will drive up costs.”

That said, of course, we’re not up a creek without a paddle; we’ve had numerous discussions around platforming over the past few years, and these conversations have not been lost on the physician community, either. In fact, Ahrens-Nicklas has been encouraging the FDA to consider situations in which we can, for instance, repurpose tox studies, biodistribution studies, and PPQ batches. 

“You cannot justify a $2 million price tag for an ultra-rare disease, just because there are 20 patients in the U.S. that might benefit from it when you’re leveraging all the same data that you would be leveraging for a drug targeting a more common indication,” she clarified. “So, we have to figure out ways of working across diagnoses and leveraging all of the fundamental foundational data to drive costs down.”

Miss part 1? You can catch it here

Stay tuned for part 3!