Deleted Tweets From Scott Gottlieb, R-D.C.

The National Evaluation System for Health Technology coordinating center https://t.co/R5KhR1zRMz is our public-private enterprise for collecting medical device RWE; with agreements with 12 organizations from 195 hospitals, 4,000 outpatient clinics and 495 million patient records https://t.co/JNBInLUWr4

Sentinel data have eliminated the need for postmarketing studies on 9 potential safety issues involving 5 products. Such studies typically require yrs. to design and complete and cost millions. We’ve also used Sentinel to understand patterns of opioid use https://t.co/KwqwLw4kXP.

We’ve used RWE for premarket decisions on medical devices, particularly new indications for use, as we did in 2013 by extending a transcatheter heart valve to patients with inoperable aortic valve stenosis based in part on data from patient registries. https://t.co/EsPJ3bcP4q

It will soon launch pilot programs testing the use of patient registries and claims data to evaluate total joint and knee replacement surgery, different wound closure techniques and the safety of intervertebral body fusion devices for spinal conditions https://t.co/SeJQhhMjob.

Additional RWE use requires assessing RWD quality and our analytical methods. Claims data is structured with medical care info from many health systems. EHRs provide a wider range of info - lab results, imaging and clinical assessments but are often unstructured and inconsistent. https://t.co/wtc9juxgVB

FDA is funding a study by Brigham & Women's Hospital & Harvard Medical School, seeking to use claims databases to replicate 30 randomized clinical trials (RCTs) to determine whether RWE can reliably reach the same effectiveness conclusion about a product as an RCT.

Most RWE efforts to date have been focused on postmarket safety questions. But RWD data from patient registries, natural history studies of the course of a disease and chart studies have been used for comparison arms in some single arm cancer and rare disease trials. https://t.co/lUTS0baFwF

RWE will still need to meet the same high standards as all evidence submitted to FDA to support a regulatory decision. In most cases it won’t be the only source of evidence. Instead, it will help provide a broader picture of a product’s safety and effectiveness over its lifecycle

And #FDA is supporting the first RCT in Sentinel to test an educational intervention to address the underuse of effective medications to reduce stroke in patients with atrial fibrillation. It could serve as proof of concept for future RWE trials. https://t.co/rqWZvYvgpu https://t.co/47DsSsJsvQ

Moreover, extensive use of RWD in the conduct and design of traditional clinical trials has the potential to make our medical product development processes more efficient and help lower the cost of development.

Consider our MyStudiesapp https://t.co/mJGZiOTgqS which makes it easy for patients to input RWD and transfer it to their EHR for use in CTs and registries. We’ve released its source code and technical docs for others to use and there’s a Sentinel pilot of it underway. https://t.co/1kqkMkSmTa

INFORMED, a data group in our Oncology Center of Excellence, https://t.co/Vr0ZzuOGDE is collaborating with Flatiron Health and CancerLinQ in use of RWD to understand real-world use of new immunotherapies and assess completeness, accuracy and consistency of electronic health data

More RWD/RWE work is coming. This week we’ll be releasing a new 2019 Strategic Framework for evaluating the potential use of RWD and RWE in regulatory decisions. We discussed this framework at a recent workshop we participated in. https://t.co/Z2ZiwIiCxZ

#FDA approved a new test to assist in detecting cytomegalovirus in newborns less than 21 days of age. When used with results of other diagnostics, it may help providers more quickly identify the virus in newborns and determine the best approach for a child https://t.co/nojvanUThq

Our goal is to close the evidence gap between the information we use to make #FDA’s decisions and the evidence used and collected by the medical community to provide patients with better care and more informed treatment decisions. https://t.co/AK252aNVir

RT @SGottliebFDA: As a science-based agency we rely on evidence generated from data to inform regulatory decisions. #FDA's… https://t.co/pL44bYVagS

RT @FDAOncology: #FDAOncology’s Dr Ann T Farrell received the #ASH18 Outstanding Service Award for her leadership and dedication to… https://t.co/mU6coDHMi0

RT @FDAOncology: Discussion on #cancerresearch and NCI-FDA collaboration between @NCIDirector Ned Sharpless, @SGottliebFDA &… https://t.co/dDlrMsPcgu

Chag Sameach https://t.co/Oxd46uEwop

RT @FDAOncology: Patient advocates attending #ASH18, join us for an FDA Special Session: A Dialogue with Patient Representatives & A… https://t.co/YGDPGG2JMK

The National Evaluation System for Health Technology coordinating center https://t.co/R5KhR1zRMz is our public-private enterprise for collecting medical device RWE; with agreements with 12 organizations from 195 hospitals, 4,000 outpatient clinics and 495 million patient records https://t.co/JNBInLUWr4

Sentinel data have eliminated the need for postmarketing studies on 9 potential safety issues involving 5 products. Such studies typically require yrs. to design and complete and cost millions. We’ve also used Sentinel to understand patterns of opioid use https://t.co/KwqwLw4kXP.

We’ve used RWE for premarket decisions on medical devices, particularly new indications for use, as we did in 2013 by extending a transcatheter heart valve to patients with inoperable aortic valve stenosis based in part on data from patient registries. https://t.co/EsPJ3bcP4q

It will soon launch pilot programs testing the use of patient registries and claims data to evaluate total joint and knee replacement surgery, different wound closure techniques and the safety of intervertebral body fusion devices for spinal conditions https://t.co/SeJQhhMjob.

Additional RWE use requires assessing RWD quality and our analytical methods. Claims data is structured with medical care info from many health systems. EHRs provide a wider range of info - lab results, imaging and clinical assessments but are often unstructured and inconsistent. https://t.co/wtc9juxgVB

FDA is funding a study by Brigham & Women's Hospital & Harvard Medical School, seeking to use claims databases to replicate 30 randomized clinical trials (RCTs) to determine whether RWE can reliably reach the same effectiveness conclusion about a product as an RCT.

Most RWE efforts to date have been focused on postmarket safety questions. But RWD data from patient registries, natural history studies of the course of a disease and chart studies have been used for comparison arms in some single arm cancer and rare disease trials. https://t.co/lUTS0baFwF

RWE will still need to meet the same high standards as all evidence submitted to FDA to support a regulatory decision. In most cases it won’t be the only source of evidence. Instead, it will help provide a broader picture of a product’s safety and effectiveness over its lifecycle

And #FDA is supporting the first RCT in Sentinel to test an educational intervention to address the underuse of effective medications to reduce stroke in patients with atrial fibrillation. It could serve as proof of concept for future RWE trials. https://t.co/rqWZvYvgpu https://t.co/47DsSsJsvQ

Moreover, extensive use of RWD in the conduct and design of traditional clinical trials has the potential to make our medical product development processes more efficient and help lower the cost of development.

Consider our MyStudiesapp https://t.co/mJGZiOTgqS which makes it easy for patients to input RWD and transfer it to their EHR for use in CTs and registries. We’ve released its source code and technical docs for others to use and there’s a Sentinel pilot of it underway. https://t.co/1kqkMkSmTa

INFORMED, a data group in our Oncology Center of Excellence, https://t.co/Vr0ZzuOGDE is collaborating with Flatiron Health and CancerLinQ in use of RWD to understand real-world use of new immunotherapies and assess completeness, accuracy and consistency of electronic health data

More RWD/RWE work is coming. This week we’ll be releasing a new 2019 Strategic Framework for evaluating the potential use of RWD and RWE in regulatory decisions. We discussed this framework at a recent workshop we participated in. https://t.co/Z2ZiwIiCxZ

#FDA approved a new test to assist in detecting cytomegalovirus in newborns less than 21 days of age. When used with results of other diagnostics, it may help providers more quickly identify the virus in newborns and determine the best approach for a child https://t.co/nojvanUThq

Our goal is to close the evidence gap between the information we use to make #FDA’s decisions and the evidence used and collected by the medical community to provide patients with better care and more informed treatment decisions. https://t.co/AK252aNVir

RT @SGottliebFDA: As a science-based agency we rely on evidence generated from data to inform regulatory decisions. #FDA's… https://t.co/pL44bYVagS

RT @FDAOncology: #FDAOncology’s Dr Ann T Farrell received the #ASH18 Outstanding Service Award for her leadership and dedication to… https://t.co/mU6coDHMi0

RT @FDAOncology: Discussion on #cancerresearch and NCI-FDA collaboration between @NCIDirector Ned Sharpless, @SGottliebFDA &… https://t.co/dDlrMsPcgu

Chag Sameach https://t.co/Oxd46uEwop

RT @FDAOncology: Patient advocates attending #ASH18, join us for an FDA Special Session: A Dialogue with Patient Representatives & A… https://t.co/YGDPGG2JMK