Mutations in the gene encoding adenosine deaminase (ADA; a key enzyme of the purine salvage pathways) cause the autosomal recessive disease severe combined immunodeficiency (SCID). When ADA function is absent or impaired, the toxic metabolites adenosine, 2’-deoxyadenosine, and deoxyadenosine triphosphate accumulate in cells, causing severe lymphocytopenia affecting T‑ and B‑lymphocytes and NK cells.
Hang on, you’ve lost me. What does that ACTUALLY mean? And how useful was that little piece of text? It all depends on your level of scientific and medical knowledge of your audience.
For the general public
This paragraph would be simply dismissed— it’s impenetrable. For the more scientifically and medically literate, it may translate into “a mutation causes SCID, which sounds like bad news, a bit like AIDS.”
For a non-expert healthcare professional (HCP)
The key piece of information about lymphocytopenia may provide a bit of extra context about how the disease could impact patients, with the gravity of the condition extrapolated from the name (severe! [and] combined! immunodeficiency disease).
For the expert
The paragraph is probably redundant. You aren’t telling them anything they do not already know, and you’re possibly taking up valuable time.
Clearly, communicating your message in the right way for your target audience is key, and although we all have access to vast amounts of information at our fingertips, the job is not straightforward. This is especially true for complex and targeted cell and gene therapies with novel modes of action that leverage novel biomarkers and complex delivery methods.
The science behind these therapies is at the cutting edge of healthcare innovation, and there is a wide range of understanding (from none to highly literate) across both patient and healthcare professional populations that must be considered when planning a communication.
For patients, regulators, payers, and healthcare professionals alike, questions are also raised about the robust nature of the data—especially for accelerated approvals. As a result, conversations around the risk and benefit of these therapies become more challenging: Digestible communications are needed to help those who are not experts in the science make (the right) decisions.
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