by Dr. Kathryn Wagner
about the author:
Dr. Kathryn Wagner is a clinician-scientist with over 20 years of caring for children and adults with neurological disease and directing clinical and translational research to develop novel therapies for these disabling and frequently fatal disorders.
“Transformative” Medicine?
Today I write not as an employee of a pharmaceutical company but as an individual who has recently lost a child she helped raise. A remarkable young person full of love and joy who has succumbed to Duchene Muscular Dystrophy (DMD).
My experiences and opinions are mine and not necessarily representative of the DMD community nor of the entire rare disease community. I am an anecdote.
Still, having stepped in to mother a child with DMD, having been a clinician treating boys and young men, deeply connected to many of these families and the larger DMD community, having run a lab researching novel therapies and conducted clinical trials in academia, and then more recently participated in drug development for DMD in the pharmaceutical industry, I do have an unusual perspective.
Words Matter
I would like to write today about the use of the word “transformative.” This word is misunderstood and exploited.
A “transformative” medicine is something that patient-families long for. A “transformative” medicine is a medicine that would dramatically, and not incrementally, change the course of disease. There have been transformative medicines for sure–consider the effects of insulin for diabetes.
However, I find the word used for anything and everything that a scientist or a company feels a need to promote—for a paper, for a grant, for investors, for regulatory approval. The word is used without an understanding or a care of the damage it causes.
Could we please give up the word “transformative”?
– Kathryn Wagner
Not everyone in the community can or will dissect the data. They will see the word “transformative” and to them it may mean that their child doesn’t lose ambulation, doesn’t lose the ability to feed themselves, doesn’t die as a young adult. They will make decisions to donate, to participate in a clinical trial, to try and keep using a medication based on this word.
I even encourage reconsideration of the phrase “potentially transformative.” Given the fact that approximately 90% of drugs fail in development and those that are approved are frequently underwhelming, what is the justification to use purely aspirational language when it produces such elevated expectations in patients and families?
“Meaningful”
Not everyone agrees with me. Another mother and researcher of DMD, is helping me with my grief, and counters that the community is far better off than the nihilistic state it was a couple decades ago. Very true. There is much value in hope. But not in hype. We should never give up hope, we should never give up developing novel and better drugs.
Perhaps we could use the word “meaningful” when we talk about the effects we hope our drugs will have on patients. A drug can surely be meaningful without being transformational.
Perhaps clinicians, researchers and drug developers could ask themselves, what would “transform” their own lives, before they spoke.
I’ve asked myself this very question, but what would have transformed my life is now, since my loss this past week, no longer possible.
– Kathryn Wagner
More To Consider…