You’ve got a gift for writing, to be able to craft a narrative that was that long in the wind-up, but does in fact come full circle to culminate in a payoff that is both illustrative of the fundamental point that you wanted to make, but also hopeful and uplifting besides. Thanks for sharing your and your daughter’s story.
From a methodology standpoint, I think you’ve highlighted an important concept in evidence appraisal. A “negative” study (very generally) means the active arm failed to show a benefit over control that was greater than or equal to the pre-determined effect size that the study itself was powered to test for. It does not (and cannot) exclude some smaller effect size. So any such study can’t show a treatment “doesn’t work”, since the possibility of a smaller yet nonetheless real effect remains possible (of course, the burden of proof remains with the proponents to positively demonstrate any such benefit).
Separately, the approach to rare diseases continues to demand a precarious balance. It is impractical to demand proof of causation as we would for treatment of more common conditions (such as those typically seen in cardiology). And yet there must still be some gatekeeping as a society to protect against runaway costs in pursuit of what sometimes turns out to be false hope (spurred along the whole way by industry interests).
Excellent piece. This is a tall order, but it would be great to read a follow up in which you give some suggestions on how the system can strike the right balance between preventing massive expense and commitment to marginal, unhelpful, or harmful new drugs and treatments and also, as you noted, getting out of the way of innovators.
Think context matters. My gawd do anything in the freaking realm of reality when you have a child dying of a dead liver. But when it comes to marginal benefits and clear risks in older patients let’s have caution.
Best wishes to your daughter! I had no idea about the trials and tribulations with liver transplants. It is amazing how far medicine has advanced and what still lies ahead. We need to empower independent and innovative physicians to advance the practice of medicine and not to be constrained by regulators.
Stunningly beautiful piece. Well written, carefully argued, and important. Thank you.
Beautiful essay. Wishing your daughter a lifetime of health. ❤️
Every turn in this narrative reveals a new optic…which accumulates to twist around before plunging into the heart of this writing.
You’ve got a gift for writing, to be able to craft a narrative that was that long in the wind-up, but does in fact come full circle to culminate in a payoff that is both illustrative of the fundamental point that you wanted to make, but also hopeful and uplifting besides. Thanks for sharing your and your daughter’s story.
From a methodology standpoint, I think you’ve highlighted an important concept in evidence appraisal. A “negative” study (very generally) means the active arm failed to show a benefit over control that was greater than or equal to the pre-determined effect size that the study itself was powered to test for. It does not (and cannot) exclude some smaller effect size. So any such study can’t show a treatment “doesn’t work”, since the possibility of a smaller yet nonetheless real effect remains possible (of course, the burden of proof remains with the proponents to positively demonstrate any such benefit).
Separately, the approach to rare diseases continues to demand a precarious balance. It is impractical to demand proof of causation as we would for treatment of more common conditions (such as those typically seen in cardiology). And yet there must still be some gatekeeping as a society to protect against runaway costs in pursuit of what sometimes turns out to be false hope (spurred along the whole way by industry interests).
Excellent piece. This is a tall order, but it would be great to read a follow up in which you give some suggestions on how the system can strike the right balance between preventing massive expense and commitment to marginal, unhelpful, or harmful new drugs and treatments and also, as you noted, getting out of the way of innovators.
Think context matters. My gawd do anything in the freaking realm of reality when you have a child dying of a dead liver. But when it comes to marginal benefits and clear risks in older patients let’s have caution.
Beautiful peace. I cried the whole way through it. I have learned so much today. Thank you.
Did not expect this to become so personal at the end but I’m glad it did. Amazing job on this one.
This medical history is likely unknown to many in healthcare and learned so much! Thank you. The highest blessings and best wishes for your daughter.
Great piece of research and inspiring piece of writing.
Best wishes to your daughter! I had no idea about the trials and tribulations with liver transplants. It is amazing how far medicine has advanced and what still lies ahead. We need to empower independent and innovative physicians to advance the practice of medicine and not to be constrained by regulators.