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With These Randomized Data: The Patient’s Perspective

With These Randomized Data: The Patient’s Perspective. Bray Patrick-Lake, President and CEO. Employer. Duke University. Disclosure Statement of Financial Interest.

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With These Randomized Data: The Patient’s Perspective

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  1. With These Randomized Data: The Patient’s Perspective Bray Patrick-Lake, President and CEO

  2. Employer Duke University Disclosure Statement of Financial Interest Within the past 12 months, I or my spouse/partner have had a financial interest/arrangement or affiliation with the organization listed below. Affiliation/Financial Relationship Company

  3. Let’s Thank the Real Heroes:The Patients STEP ONE: The study participants should be notified of the results in lay terms and profusely thanked. If you don’t provide them with information about your study Google and social media will. Never assume a study participant has been adequately informed of the results just because you published it in a peer-reviewed journal. Patients don’t have access to peer-reviewed journals. The information is too technical to be meaningful.

  4. What Did Patients Want to Know from RESPECT? “Three things: Safety- what are the long term risks of having the device. Efficacy- Does the device solve the problem that it was intended to solve- preventing recurring events. Unintended consequences- Are there any issues that have arisen that are unexpected and unwelcome- for example, the 10% of cases that develop aortic regurgitation as has been reported in other studies”

  5. More from Patients “I am hoping that it finds closing a PFO is more beneficial to the patient than drug therapy, therefore getting my insurance company & cardiologist on the same team.” “I am hoping that the Amplatzer stays in tact while I live out the rest of my life … and that I won't need open heart surgery to get it repaired or removed.” “RESPECT for every patient in this group so that we can finally get some attention and funding for all the questions being mentioned here.”

  6. What’s To Like from the Patient Perspective? • The clean population of RESPECT eliminated a lot of the noise. • Some patients were followed for 8 years! • The Afib rates were the same in both groups. • There were no erosions. • The SAE rates appear to be low. • The effective closure rate minimizes doubt around device performance affecting the ability to answer the scientific question. • PFO SCIENCE HAS TAKEN A STEP FORWARD

  7. What’s Not To Like From the Patient Perspective? • The immediate negative kneejerk reaction and devaluing of information that is better than what was previously available to help patients make treatment decisions • Expected from the media • Highly disappointing from our treating physicians • PFO Patients are tired of venture capitalists driving the conversation about our disease’s importance. • Stroke has a significant cost in the U.S., financially, in disability, and in lives lost. Patients will not tolerate being offered a less effective therapy over cost-related issues. [Incidentally, cost is not an efficacy end point in regulatory decisions.]

  8. Anger Management Placeholder

  9. Recruitment and Retention • Are we really surprised that 100+ patients were lost to follow-up or withdrew consent? • PFO patients did not have a collective voice or a seat at the table when the trial was designed a decade ago. • The study lasted much longer than anticipated. • There is a truth to this being a “hostage population” due to reimbursement issues despite patients willingly signing the informed consent paperwork. • Patients are allowed to withdraw consent at any time. A large number of “lost” patients speaks volumes about the lack of patient support for randomization and belief that equipoise exists. • In the future, we must build trust with the patient community and understanding of the value of randomized data to the patient population before expecting them to enroll in a study and see it through.

  10. Quality of Life Data: Some things are so obvious that they often get overlooked • Significant therapeutic burden exists with warfarin. • This burden affects both patients and their family members. • Patients care as much about feeling better as they do about safety and efficacy. • QOL end points are efficacy end points.

  11. What Now? • It has become increasingly clear that work on standardization of PFO needs to be done ASAP. ASE, we need your help! • Professional society collaboration between independent disciplines modeled after the ACC/STS TAVR collaboration would be of great benefit in improving PFO patient care. • All stakeholders need to be at the table for a discussion about the continued existence of clinical equipoise.

  12. Talking to Patients • Closure 1 and the ASD device safety issues reminded us that the closure procedure was low-risk, but not no-risk, and of the importance of treating appropriate patients. It would be a disservice to patients to abandon that thinking at this time. • It takes time to do quality patient education and the new data adds important layers to the conversation. If you and/or skilled nursing staff don’t have time to perform a thorough, unbiased discussion of treatment options then do PFO patients a favor and send them to a center that does. • The temptation to help desperate patients is hard to resist. Beware of overstating benefitbefore the FDA advisory committee has had a chance to analyze the data.

  13. Many Questions Remain • Are patients comparing apples to apples when making treatment decisions? • Should patients be on aspirin alone? • When comparing trials are we ready to say the benefits of PFO closure for recurrent cryptogenic stroke are definitively device specific? • Will the Kaplan-Meier curve continue to diverge or could this change over time as events are reported? • Are we headed for a recommendation of combo therapy?

  14. The Path Forward Nothing is predestined: The obstacles of your past can become the gateways that lead to new beginnings. - Ralph Blum

  15. Key Steps in Advancing PFO MedicineModel Compliments of John Spertus, MD Aggregate the Data! Improved Health Data from RoPE, Closure 1, RESPECT and PC Trial Prediction Models Outcomes Delivery Tool Patients using the data Delivering the Evidence Physicians using the data Shared-decision making tools

  16. Take Away Messages: • Do not give up on this complex situation and abandon PFO patients. • Patients need you to further investigate the relative risk reductions and long-term outcomes, and develop models that allow for meaningful and accurate comparison between medical and device therapies. • The worst thing that could happen is giving PFO/RCS patients a false sense of security that they are “cured” and no longer need to manage their stroke risk factors or attend follow-up visits. • Patients are more than a P-value. We should leave room in the conversation for patient preference and tolerance of risk as we move forward.

  17. I Have a Dream The PFO Research Foundation envisions that someday PFO patients will be able to walk into their physicians’ offices and receive: • Evidence–based care supported by multidisciplinary professional society guidelines that limits variability, • Accounts for personal characteristics, preferences, and tolerance for risk and • Measures longitudinal outcomes in an effort to continuously improve care for all PFO patients.

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