What Baby KJ Taught Us About Patient & Physician Communication
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
I learned early on in my writing career that important industry challenges are often reiterated three times from conference podiums.
In the biosimilar space, which is where I began, we often repeated the term “education, education, education.” Today in the mRNA space, we often hear that “delivery, delivery, delivery” is our prime objective. (And to be clear, this isn’t wrong!) But over the past few years, I think it’s also become clear that we need to take a page from the biosimilar industry and steal its “education, education, education” mantra.
This came through loud and clear during the recent AMM and Franklin Biolabs joint event called Building RNA Medicine Excellence. Over the past few weeks, I’ve been sharing my takeaways from the overall event, as well as from a specific panel featuring JHU’s Jeff Coller, CHOP’s Rebecca Ahrens-Nicklas, and Plowshare Therapies’ Kevin Strauss. Together, these three discussed the biggest learnings and takeaways from the miraculous Baby KJ experience that we very much hope to replicate in the future.
I’ve broken these three takeaways into a series of three separate articles — the first on what we learned about sustainable mRNA development, and the second about COGS. However, as you can also imagine, patient and physician communication played a large role in the discussion at hand.
To Strauss who works closely with the Amish and Mennonite communities, it’s important to keep in mind differences in education that can limit a community’s understanding of common drug development vernacular/abbreviations. For these communities, for example, many only get up to an eighth-grade education.
“I would encourage folks to think about how you can take these concepts about gene therapy and break them down into simple language and messages that people can understand,” Strauss explained. “Even when we’re trying to explain things simply, we’re not aware of how much jargon and how many abbreviations we use.”
But if we step back even further, both Strauss and Ahrens-Nicklas underscored the importance of realistic communication about the drug development paradigm — especially in the wake of an experience as miraculous as Baby KJ’s development which could go on to set an unrealistic expectation for other kids/families. In fact, as Ahrens-Nicklas clarified, she did not approach KJ’s family until there was a development roadmap and editing solution in hand so as not to provide false hope.
“We have to be honest about what real timelines look like in drug development right now,” Ahrens-Nicklas explained. “I challenge everyone on the development and manufacturing side to be honest with families and give realistic expectations. This means letting them know a lot can go wrong. Many things aligned to write the KJ story. Everyone dropped everything. We all know this is not the reality of drug development. So, we have to paint a realistic picture for families, and that’s a big part of what I do in my clinic.”
Painting a realistic picture of drug development also means being as transparent as we can about safety issues that arise both preclinically and in our clinical trials — particularly for the sake of physicians. As Ahrens-Nicklas emphasized, navigating the “whispers down the lane” from patients remains a big challenge. It goes without saying that participating in an early phase clinical trial is a big lift and sacrifice for families. Not having the full picture behind a safety event makes it that much harder for a physician to recommend a clinical trial.
“How can I judge whether or not I should be recommending or talking through a specific trial with a family when I don’t have clarity about what happened with the adverse event from that patient halfway across the country?” Ahrens-Nicklas concluded. “We need transparency around safety so we can analyze a trial’s risk-benefit for each individual patient.”
Missed part 1 & 2? Catch them both here: