The Unspoken Truth Of Bluebird Bio's EU Misadventures
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
It’s somehow that time of year again when my colleague Dan Schell at Life Science Leader magazine taps me on the shoulder and says it’s time to start thinking about your year-end outlook article. This will be the first year I’m covering the C&G manufacturing/outsourcing space and determining which trends and challenges will take shape and evolve in 2022.
This assignment inevitably prompted me to figure out which overarching stories have emerged about the C&G manufacturing space from the seemingly trillions of news pieces and technical articles I’ve read over the past year. Though there have been many themes to note, there is one story the industry is becoming infinitely more familiar with: The COGS for these therapies is proving to be a real damper for market access — especially when we look to the overseas markets. This was exemplified most recently by Bluebird Bio’s announcement that it will be exiting the European market post-haste, barely two years after expressing hope that it could woo over EU health agencies.
As pharmaphorum outlines in this article, safety issues (both past and present) accompanied by EU health agencies’ disinterest in paying $1.8 million for Zynteglo (starting with Germany) has encouraged the company to either “hot potato” Zynteglo to a partner or end EU commercialization altogether. At this point, I think it’s safe to say that Bluebird Bio has had a more rough-and-tumble year than John Wick.
First and foremost, this is heartbreaking news for patients. It goes without saying that this story will repeatedly be cited as a prime example of reimbursement systems’ shortcomings in paying for seven-figure-plus therapies — particularly in government run and/or thriftier healthcare systems.
But this also saddens me because of what this market limitation ultimately means for manufacturing innovation, know-how, and overall COGS for ex vivo C&G therapies. The logistically challenging nature of ex vivo therapies (including Bluebird’s Zynteglo) inherently limits the scope and breadth of what is possible to treat today. You know even better than I do the manpower and capacity needed to [often manually] develop and scale-out therapies for several hundred, maybe thousands of patients in clinical trials, let alone the commercial market.
However, this expansion to a broader, global patient population is also, oddly enough, exactly what the industry needs to finesse the overall manufacturing process and bring costs down.
The larger the patient pool, the more insights we’re granted into the inherent variabilities each patient’s cells bring to the ex vivo process and how to nurture those cells into becoming the best treatment they can be. (I know, it sounds like your child’s first day in kindergarten.) Secondly, to become its most efficient self, the manufacturing process craves industrialization — no matter how nascent or catered toward ultra-rare populations it may be. Working with a larger patient population adds even greater impetus to engineer and automate such biology to make the development process even more efficient. And finally, we can’t overlook the importance of attaining greater amounts of data, which will ultimately lend itself to more informed and reproducible manufacturing processes. (Of course, as I’ve written before, to achieve this final point, we also need the right people to help us make sense of that data.)
Bluebird’s latest news will most likely not be the last example we have of the commercial shortcomings of the C&G market. There’s also, unfortunately, no easy answer or quick solution to overcoming such a challenge beyond undertaking longer-term efforts to improve manufacturing, some of which I’ve touched upon recently in my other blog posts. But if one thing is clear, the industry is going to have to become comfortable living with and navigating the tension between increasing access and improving COGS — neither of which can truly be accomplished without the other.