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Harlan Krumholz
@hmkyale
, 7 tweets, 4 min read Read on Twitter
Today I'm @US_FDA w/superstar heart failure specialists, industry experts, patients, regulatory science experts, statistical experts; exploring new approaches to drawing inferences from clinical trial data. Can we do better than the traditional approach? Probably.
@US_FDA Conversations about TOPCAT, study of spironolactone for patients with heart failure and preserved ejection fraction. ~10 cents a day. Reasons to believe the drug is effective. Likely Russian & Georgian sites contaminated the trial. It is not strongly endorsed in the guidelines.
@US_FDA This is imp because no good treatments for these patients. And if you exclude Russia & Georgia, this trial is positive. And strong reasons to believe those sites enrolled people w/o the condition. Cheap drug. No profit motive. So no group pushing for re-examination of data.
@US_FDA This is about heterogeneity of effect, but in this case it derives from concerns about the integrity of some sites; the question is do we favor an analysis that assumes homogeneity of effects, even with reasons to think otherwise because of site behavior. @mfiuzat
@US_FDA @mfiuzat It is recurrent issue of subgroup analyses. Are we stuck with overall results even with knowledge that some regions may have contaminated the trial. How much do we discount the evidence? Meanwhile HF specialists in the room all believe, based on the trial, that the drug works.
@US_FDA @mfiuzat Another issue being raised… interpretability of the statistics for patients. How do we translate effects… and uncertainty? Not just about the math…but about the translation to practice. How do we inform decisions? @MinnowWalsh @coconnormd
@US_FDA Hearing about Spironolactone Initiation Registry Randomized Interventional Trial in Heart Failure W/Preserved EF (SPIRRIT). Based on registry in Sweden & large simple design in US. Enrollment in US is slow. We need to change our trial culture and capability in US. @mfiuzat
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Harlan Krumholz
@hmkyale
"Hospitals, Some Members Congress Concerned About Patients Making Uninformed Decision To Share Medical Data W/3rd Party Apps” Ironic since every day hospitals send patient data to 3rd parties w/no idea what is happening to it. @politico @ChrisMurphyCT @rosadelauro @SenBlumenthal
@politico @ChrisMurphyCT @rosadelauro @SenBlumenthal The bottom line is that patients should be informed where their data goes - & should have clear, easily understandable explanations. Most often now they do not even know who has their data. Important because it could contain info that they consider highly personal and sensitive.
@politico @ChrisMurphyCT @rosadelauro @SenBlumenthal And public health and certain research exceptions might exist for the use of deidentified data…but society needs to come together and be clear what is acceptable. The default should be the patient’s preference - with explicit permission.
Read 5 tweets
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Harlan Krumholz
@hmkyale
1/Lots of sturm and drang about national readmission program. @JAMA_current and @nytimes give platform to vocal critics of the policy. Worth exploring this a bit.
@JAMA_current @nytimes 2/~16% who are hospitalized get so sick during recovery that they need to be readmitted w/i 30 days. Many, including @leorahorwitzmd @ehbvassar us & others have identified quality gaps that contribute to readmission risk. E.g.: ahajournals.org/doi/full/10.11… jamanetwork.com/journals/jamai…
@JAMA_current @nytimes @leorahorwitzmd @ehbvassar 3/ The risk of readmission is a property of the hospital, independent of the patient. Same patient admitted with same diagnosis to a hospital with poorer readmission performance will have a higher risk of readmission. Published in @nejm… nejm.org/doi/full/10.10…
Read 16 tweets
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Harlan Krumholz
@hmkyale
1/ Is my dream that future of medical research can include category for people-powered data collaboratives. Barbara Evans and I lay out a path. Here is #tweetorial. academic.oup.com/jamia/advance-… @PCORI @EmCollective @gatesfoundation @NIH @EricTopol @wilbanks @YaleMed @sharonfterry
@PCORI @EmCollective @gatesfoundation @NIH @EricTopol @wilbanks @YaleMed @sharonfterry 2/First, we acknowledge most research done person at a time, w/individual consent. But what if people facing common conditions provided collective consent to their health data to researchers they choose. People powered. academic.oup.com/jamia/advance-…
@PCORI @EmCollective @gatesfoundation @NIH @EricTopol @wilbanks @YaleMed @sharonfterry 3/What could it be like to speed research if people could gain agency over their data & seek others to move quickly & responsibly to leverage data for discoveries. To treat the community of people as partners. Respectfully. Appropriately. Sharing insights. academic.oup.com/jamia/advance-…
Read 11 tweets

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