Implications of the Privacy Rule for Trialists and Academic Research

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Godfrey Stevenson
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1 Implications of the Privacy Rule for Trialists and Academic Research Robert M Califf MD Vice Chancellor for Clinical Research Duke University Medical Center

2 HIPAA Privacy Rule Because of the paralyzing effect of the interpretation of HIPAA (and other associated regulatory issues) it is likely that thousands to tens of thousands of Americans are dying or becoming disabled needlessly But, that s just my opinion and I can t prove it!

3 Academic Of theoretical interest but of no practical importance Pertaining to an effort to understand

4 Implications of HIPAA Privacy Rule Current issues Finding research subjects Getting consent appropriately Following research subjects over time Finding lost research subjects The future We are creating an aura around clinical research which is demoralizing, expensive and out of touch with modern views of the integration of clinical research and clinical practice This aura is contributing to the exodus of clinical research from the US and likely to the continued decline of US health outcomes relative to the rest of the world

5 I skate to where the puck is going to be, not to where it has been. Wayne Gretsky (the Puck Stops Here!)

6 Current Situation Clinical Investigators Demoralized 5.5% annual decrease in US investigators filing 1572 form with FDA At same time growth occurring elsewhere Central and Eastern Europe 16% annual increase Asia 45% annual increase Ken Getz Applied Clinical Trials 9/2007

7 Demoralization of Clinical Researchers in the US "The regulatory climate has become so oppressive in the recent past that two out of three principal investigators who have worked with us for 20-some years said they don't want the responsibility anymore," warned Maloy. "They don't have time for it. They're busy seeing patients in their practice." Ken Getz Applied Clinical Trials

8 Current Situation US Losing its Leadership Role Turnover rates doubled between 1990 and 2000 Half of first time researchers will not participate again Women only 12% of researchers filing 1572s GCP violations increased multiple fold GCP violations increased multiple fold Ken Getz ACT

9 Current Situation More Difficult to Screen for Patients Global Stroke Trial All European sites provided detailed screening logs so that generalizability of study could be determined Only 5 of 15 US sites provided data and these data were incomplete Kompanje and Maas, Critical Care Med 2006

10 Current Situation Skewed Skewed Access to Populations Unstable angina registry that spanned privacy rule implementation Consenting subjects: older, whiter, more married Dramtically lower mortality in consenting subjects Stroke registry Armstrong, Arch Int Med, % of subjects participated with passive effort and just over 50% with $500k active effort Biased sample with lower mortality and disability

11 Clinical Trials Example Industry sponsored international megatrials Up to 4% of people lost to followup at the end of study This can swing the result of a trial (esp( in common chronic diseases) What can be done to find patients? What if they withdrew consent? What if they have died?

12 Bureaucracy The only thing that saves us from the bureaucracy is inefficiency. An efficient bureaucracy is the greatest threat to liberty Eugene McCarthy ( ), Time magazine, Feb. 12, 1979 Bureaucracy defends the status quo long past the time when the quo has lost its status Laurence J. Peter ( ) Any sufficiently advanced bureaucracy is indistinguishable from molasses. Unknown

13 Goal Generalizability X Validity

14 Life Expectancy Around the World Country Life expectancy Men Women Angola Cambodia China US Cuba UK Canada Japan

15 The Cycle of Quality: Generating Evidence to Inform Policy 12 Transparency to Consumers 11 Pay for Performance 1 FDA Critical Path Discovery Science Outcomes Performance Measures 10 Evaluation of Speed and Fluency 2 3 NIH Roadmap Early Translational Steps Measurement and Education Clinical Trials Clinical Practice Guidelines Data Standards 9 4 Network Information Conflict-of-interestof-interest Management 5 Empirical Ethics 6 Priorities and Processes 7 Inclusiveness 8 Use for Feedback on Priorities Califf RM et al, Health Affairs, 2007

16 Goals for CRUSADE: Improve Adherence to ACC/AHA Guidelines for Patients with Unstable Angina/Non-STEMI Acute Therapies Aspirin Clopidogrel Discharge Therapies Aspirin Clopidogrel Evaluating the Process of Care Beta Blocker Beta Blocker An adherence score is applied to each patient. incorporating the components of Heparin (UFH( or LMWH) process ACE Inhibitor of care. The score from each patient then combined for all patients GP IIb-IIIa at IIIa each Inhibitor hospital. Typical Statin/Lipid scores ranged Lowering from 50 to 95%. All receiving cath/pci Smoking Cessation All 400 hospital adherence scores then ranked in quartiles - best to worst. Cardiac Rehabilitation Circulation, JACC ACC/AHA Guidelines update

17 Overall Adherence Score Trends Over Time 80% 77.9% 78.0% 79.3% 75.2% 70% 68.1% 69.6% 71.0% 72.3% 73.0% 73.6% 60% Q1 '02 Q4 '02 Q3 '03 Q2 '04

18 Link Between Overall ACC/AHA Guidelines Adherence and Mortality % In-Hosp Mortality Every 10% in guidelines adherence 11% in mortality <=25% 25-50% 50-75% >=75% Hospital Composite Quality Quartiles Adjusted Unadjusted Peterson et al, ACC 2004

19 STS: National Randomized Trial in Quality Improvement in Bypass Surgery The Society of Thoracic Surgeons T. Bruce Ferguson Jr. PI; Mary Eiken,, RN, MS; Meghan Carey, MS; Fred L. Grover, MD; The Duke Clinical Research Institute Eric D. Peterson, MD, MPH, Co-PI; Elizabeth R. DeLong, PhD; Laura P. Coombs, PhD; The Quality Assurance Foundations: : Iowa, Colorado Minnesota, Alabama Supported by AHRQ RO-1 1 HS 10403

20 Randomization All Sites N=399 Intervention #1: Preop ß-blockade N=124 Intervention #2: IMA Use in Elderly N=120 Control: No Intervention N=115 Regional QI: Both Interventions N=40

23 Society of Thoracic Surgeons Model Science and Technology Common database Improved Outcomes Education and Feedback Outcomes Gaps Trials and Outcomes Projects

24 National Consortium For Clinical Databases (NC2D)

25 Applicability of HIPAA and the Common Rule to the STS Databases Data to link to for QI purposes NOT RESEARCH Data to link to for research purposes STS Data w/ direct patient identifiers used for QI STS Data w/ direct patient identifiers forms a research repository Common Rule: NA HIPPA: BA agreement in place No need to approach IRB Common Rule: Waiver of consent HIPPA: Waiver of authorization Approach IRB with research repository protocol Most STS analyses including those performed for Access & Publications will be done with this dataset Common Rule: Waiver of consent HIPPA: Waiver of authorization Approach IRB with new study protocol HUMAN SUBJECTS RESEARCH Data with identifiers used for specific research study Limited data set used for research Common Rule: Not human subjects research HIPPA: Limited dataset with a data use agreement Approach IRB for declaration of Not human subjects NOT HUMAN SUBJECTS RESEARCH

26 -omics Technologies Can Help Predict Treatment Response. Cancer Diabetes Genetic & Genomic Profiling Responder Exercise + Diet A Adverse Exercise event + Diet B Non-responder Exercise + Diet + Medication

27 In 20 Years (or a bit longer) All people will have An electronic health record Biological samples stored to enable personal decisions Digitized images 3 Healthcare will be personalized or mass customized using an individual s images, samples and clinical data The health of a community will be monitored using aggregate records Clinical research will use these records to create learning health care systems

28 In 20 years (or a bit longer) Clinical research will be seen as a learning system built into health care delivery Electronic health records will provide inexpensive characterization and followup Disease specific registries will proved the deep and more precise data needed for detailed mechanistic and specialty based studies Randomization will be seen as an important tool inserted where appropriate into these ongoing data streams

29 Competing Views of Globalization of Clinical Research Networks of clinician investigators linked by common interest in health, disease, prevention, diagnostics and therapeutics Research embedded into clinical practice to gain both validity and generalizability Open to studies of high quality regardless of source of funding Multinational corporations hiring investigators at the lowest cost to work for them doing clinical trials Research separated from clinical practice as much as possible Separate government funded organizations to do evaluative studies and epidemiology using tax money

30 Clinical and Translational Science Awards

32 Integration of Clinical Research Networks Link existing networks so clinical studies and trials can be conducted more effectively Ensure that patients, physicians, and scientists form true Communities of Research

33 FDA PPP on Safe and Effective Clinical Trials First meeting on October 11 th Hope to raise funding to develop empirical base for FDA guidance on regulations for clinical trials Key players FDA, industry, academia, patient advocates, non- academic clinical research professionals

Liberating Clinical Trial Data: Pooling Data from Multiple Clinical Trials to answer Big Questions

Liberating Clinical Trial Data: Pooling Data from Multiple Clinical Trials to answer Big Questions Robert M Califf MD Vice Chancellor for Clinical and Translational Medicine The Premise Trial participants