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Out of order: Beyond the bubble
November 2018
by Randall C Willis  |  Email the author
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Science and medicine are about measurement. They are about the quantitation of parameters that determine whether something is normal or unusual, healthy or diseased.
 
Throughout the scientific and medical journals, as well as DDNews itself, we read about p-values and non-inferiority, minimal residual disease and PFS, IC50 and t½, sensitivity and specificity.
 
Want to convince a roomful of scientists about your new finding or approach? Show them the numbers.
 
Want the FDA and its compatriots to approve your therapy? Show them the numbers.
 
Want insurance companies or regional health programs to cover a therapy? Show them the numbers.
 
In science and medicine, we quest for understanding. We quest for knowledge. We quest for truth.
 
This is how it is, and for the past 18 years, this is how I have read, interviewed and written about it.
 
Something is changing, however. The nature of the quest is shifting or expanding.
 
Yes, we want understanding, knowledge and truth; but more and more it seems that these are no longer enough, are no longer sufficient without also including a quest for meaning.
 
What science and medicine are not is healthcare. And healthcare cannot exist without patients and caregivers, the very people often ignored or digitized in the parameterization of health and disease.
 
Over the last year or so, I have noticed the shift in my conversations with researchers and executives. Where once we only spoke of platforms and performance, we now also talk about people and impacts on daily lives.
 
For my August Special Report on Drug Delivery, I spoke with Mathias Schmidt, CEO of ArmaGen, a company developing novel enzyme replacement therapies to treat the neurological impacts of lysosomal storage diseases. And as eager as he was to talk about his company’s approach to the blood-brain barrier, Schmidt had more to offer.

He recounted with some emotion how game-changing the outcome of a clinical study was for some of the parents and patients.
 
“The parents aren’t looking for another Einstein," he explained. "They know about the [otherwise] very serious prognosis of the disease.”
 
“If they can keep their kids at the stage where the disease currently is—because they know the child is going to decline—they would be the happiest people on Earth,” he continued.
 
“This is what we should always be reminded of. Anything that we’re doing here is not for us. It’s not for the vanity of science. It’s about how do we create a better tomorrow for the patients whom we serve.”
 
Similarly, for my September Special Report on Stem Cells, I talked to Amander Clark of UCLA’s Broad Center and Kyle Orwig of the University of Pittsburgh, both of whom apply stem cell technologies to improve understanding of and perhaps one day treat infertility.
 
Both had heartfelt recollections of the people they met in pursuing their research.
 
“I get emails on a weekly basis from couples who are asking me when will stem cell—iPSC particularly—technology be available so that my partner and I can have a baby,” Clark recalled. “Clearly this is a topic and a technology that is desired by members of our community.”
 
“If you aren’t suffering with infertility yourself, it may be hard to know what’s going on inside other people,” offered Orwig, who is also director of fertility preservation in Pittsburgh. “I can guarantee you that there is a major psychological impact on the individual and also on the couple.”
 
Likewise, in this month’s Special Report on Molecular Diagnostics, I spoke with Kimberly Martin, vice president of reproductive health at Natera, about noninvasive prenatal testing using cell-free fetal DNA found in maternal blood.
 
Yes, Martin had plenty to say about the assay technologies, detection sensitivities and fetal fractions. Just as important to her, if not more so, however, was the human responsibility of not just providing the results to patients and partners, but also providing a sense of context, implications and limitations of the results.
 
“Every day, I saw the impact,” she recounts. “I tried very hard from when I was a resident until I became an attending physician and on into industry to meet the family where they are. To use language that they can understand. To try to make a bad situation as supportive as possible.”
 
In each interview, the weight of responsibility and the hope that they could make a change in even a single human life were palpable. I have heard that same tone in many other conversations, as well.
 
There will always be an analytical dispassion to science and medicine, as much of discovery and development takes place at a distance—both physical and temporal—from its intended benefactors, who deal with their conditions largely invisibly. In most cases, that is as it needs to be.
 
But I am heartened to see a greater human connection between one end of the process and the other, because with that connection comes compassion and with compassion comes meaning. And without meaning, what is the point?

Randall C Willis can be reached by email at willis@ddn-news.com

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