STAT readers on patient advocacy, the utility of diagnostic tests, and more

STAT now publishes selected Letters to the Editor received in response to First Opinion essays to encourage robust, good-faith discussion about difficult issues. Submit a Letter to the Editor here, or find the submission form at the end of any First Opinion essay.

“Alzheimer’s drug approvals show we need a re-evaluation of patient advocacy,” by Jon Hu

As a former FDA associate commissioner, I strongly disagree with the unfair and uninformed comments of the author. The author casts unconfirmed aspersions on the agency. I can say, because of personal experience as an “insider,” that the idea that patient groups inappropriately sway FDA decisions is absurd. It’s not surprising there’s no mention of the agency’s successful (and congressionally-mandated) Patient-Focused Drug Development Program. The author owes the FDA an apology.

— Peter Pitts, Center for Medicine in the Public Interest

***

As a patient advocate myself, I find it demoralizing to read articles like this. Being in Canada, the process is quite different. Governments pay for whatever drugs we have access to, so we don’t get access to everything, only those with the least uncertainty. Is that a good thing, though? Having provided input into the health technology assessment process here in Canada, we advocate for treatments that we have heard from patients provide benefit to them. So without knowing why these groups are advocating for access, why write such an article? If patients are telling me — and it may not be all patients — then is it not my duty to fight for them and then let the FDA deliberate on the scientific evidence? There is so much bad press directed to hard-working advocates, who are always representing the voice of the patient communities they serve.

— Sabrina Hanna, the Cancer Collaborative

“When do tests hurt more than help?” from the “First Opinion Podcast”

A very astute analysis by the two discussants, but they miss a critical input: utility. Utility is not statistical, it is subjective. It exists in the mind of the patient. In the example where a genetic abnormality occurs in 1/20,000 of the population, the question they fail to address is, “What is the (negative) utility of giving birth to a severely disabled child?” For me, the utility would be -Inf (negative infinity), so no matter the probabilities they lay out, if a test read positive, I’d abort. (I’m a man, so this is a thought experiment.) I’d guess that for most prospective mothers desiring a child, the (negative) utility of having an abortion would be far outweighed by the (negative) utility of bearing a severely disabled child. It follows that to complete the analysis, a doctor or social worker must interview the patient, have her assign a utility to the various possible outcomes, and compute the expected utility of each possible course of action for that patient. Then, pick the course of action that optimizes utility and explain to the patient why this course of action is her best option.

— John Van Praag

“It’s time to rethink the Medicare annual wellness visit,” by Jeffrey Millstein

He nails it, and I would add these appointments are an insult and degrading. Focus is questions like remember three words and repeat them later, basically questioning that seems to imply everyone likely to have dementia. I am an active professional working and traveling and want an exam that checks for cancer or other physical ailments, not one making me feel like I’m a suspect for incarceration due to Alzheimer’s.

— Cliff Berg