Risk-reducing mastectomy questioned for BRCA mutation carriers with prior ovarian cancer

July 12, 2017 – The study finds little survival benefit and high costs from the preventive procedure.

Mutations in the BRCA gene correspond to a higher lifetime risk of developing breast and ovarian cancers, and many women who carry these mutations consider undergoing mastectomy or removal of the ovaries and fallopian tubes as preventive measures.

But for the subset of women with BRCA mutations who have already had ovarian cancer, risk-reducing mastectomy might not be worth the price tag. New findings from Duke researchers, including the DCRI’s Evan Myers, MD, finds that for many women in this unique group, prophylactic mastectomy does not produce a substantial survival gain and is not cost-effective.

The finding is especially noteworthy because of updated National Comprehensive Cancer Network guidelines recommending that many women with ovarian cancer be considered for genetic testing regardless of family history. Now, more than ever before, some women with ovarian cancer are also learning that they carry a BRCA mutation.

“Risk-reducing mastectomy is costly and can require many months of follow-up and recovery,” said Charlotte Gamble, MD, the study’s lead author and a resident physician at Duke University School of Medicine. “Our results emphasize that prophylactic mastectomy should be used selectively in women with both a BRCA mutation and a history of ovarian cancer.”

In the study, published online July 11 in the journal Annals of Surgical Oncology, Gamble and co-researchers constructed a statistical model comparing risk-reducing mastectomy to breast cancer screening, including mammogram and MRI. The model incorporated clinical factors such as the age at ovarian cancer diagnosis, time between ovarian cancer diagnosis and risk-reducing mastectomy, BRCA mutation status, cancer survival rates and treatment costs. Risk-reducing mastectomy was compared to breast cancer screening performed every six months following ovarian cancer diagnosis.

The study’s authors also considered a cost-effectiveness measurement called the incremental cost effectiveness ratio. Healthcare interventions where this ratio is less than $100,000 per year of life saved are commonly considered cost-effective in medical literature. The authors used the same threshold in this study.

According to the authors’ analysis, the benefit of risk-reducing mastectomy over screening alone largely depended on the patient’s age at the time of her ovarian cancer diagnosis and time to mastectomy:

  • For women diagnosed at any age with BRCA 1 and 2 gene mutations and within the first four years after ovarian cancer diagnosis, prophylactic mastectomy was associated with a negligible gain in survival and was therefore not found to be cost-effective;
  • For women diagnosed at age 60 or older, regardless of time since ovarian cancer diagnosis, the gain in survival months was also negligible and the procedure was not cost-effective;
  • For women diagnosed at age 40 to 50 with BRCA 1 and 2 mutations and at least five years after an ovarian cancer diagnosis, the procedure was associated with a survival benefit of two to five months compared to screening and found to be cost-effective.

“Our study provides clarity on how a woman’s age and timing of a risk-reducing mastectomy after an ovarian cancer diagnosis impact the benefit of this procedure,” Gamble said. “Within the first five years, nobody benefitted from risk-reducing mastectomy and after that threshold, survival gains were seen mostly in the youngest, healthiest ovarian cancer patients.”

“There is no right or wrong answer on how to manage breast cancer risk in this unique population,” added senior author Rachel Greenup, MD, assistant professor of surgery at Duke. “However, we hope that our findings provide guidance to women and their doctors deciding if and when prophylactic mastectomy is beneficial following ovarian cancer treatment.”

In addition to Myers, Gamble, and Greenup, Duke co-authors include Laura Havrilesky, Junzo Chino, Scott Hollenbeck, Jennifer Plichta, P. Kelly Marcom, E. Shelley Hwang, and Noah D. Kauff.

The authors report no financial disclosures.

DCRI’s Alex Fanaroff receives AHA Funding Award

July 11, 2017 – The award supports cardiovascular researchers who are at the beginning of their careers.

Incoming Co-Chief DCRI Fellow Alex Fanaroff, MD, was recently named a recipient of the competitive American Heart Association (AHA) Fellow-to-Faculty Transition Award, which provides funding for beginner physician-scientists in cardiovascular or stroke research.

“The fact that an organization like the AHA thinks it worthy to commit its resources to me is extremely humbling,” said Fanaroff. “For an avid clinical researcher, there’s nothing quite like devoting the majority of their time doing research without worrying about how they are going to fund it.”

Currently pursuing research focused on describing and improving processes of care for patients with acute coronary syndromes and stable angina, Fanaroff has been a student, intern, resident, and fellow in the Duke School of Medicine for more than ten years. He says that this grant is the opportunity of a lifetime, which will not only enhance his chances of obtaining a high-quality faculty or staff appointment but also improve his success and retention in an investigative career in cardiovascular science.

With around 60 applicants each year, the applicant success rate for the program is about 20 percent.

“It is rare for a career development grant to be funded on the first application,” said Fanaroff, “but my grant mentors at the DCRI and in the Division of Cardiology encouraged me throughout the application process to really think about the direction of my career and my research, which provided focus and gave me confidence and hope.”

Tracy Wang, MD, DCRI’s Fellowship Associate Program Director and a proud mentor of Fanaroff for the past two years, says that it has been wonderful to watch his research skills maturing.

“Alex is the type of fellow who takes the ball and runs with it,” said Wang. “This AHA award is a demonstration of that. I have no doubt that he will be a leader within our field in the next ten years.”

Antibodies halt placental transmission of CMV-like virus in monkeys

July 6, 2017 – The finding advances a human vaccine for CMV, which afflicts 1 million babies a year.

Long before the Zika virus became a global fear, cytomegalovirus, or CMV, was commonly infecting developing fetuses and causing many of the same brain and developmental impairments.

The virus, one of only a handful known to be transmitted through the mother’s placenta to a fetus, infects nearly 1 million infants a year worldwide and is a leading cause of microcephaly, hearing and/or vision loss, and nervous system damage.

With effective interventions lacking, development of a vaccine remains an urgent public health mission. Now researchers from Duke University School of Medicine, and Tulane National Primate Research Center report findings in monkeys that demonstrates a vaccine approach that appears to be capable of protecting the animal’s fetus from infection.

“The presence of potent antibodies at the time of the mother’s primary infection seems to  prevent viral transmission and severe disease in the developing fetus, and therefore should be a primary target of vaccines to prevent neonatal infection,” said co-senior author Sallie Permar, MD, PhD, professor of pediatrics at Duke and member of the Duke Human Vaccine Institute. Permar and colleagues, including co-senior author Amitinder Kaur, MD, of Tulane National Primate Research Center and the DCRI’s Michael Cohen-Wolkowiez, MD, PhD (pictured), published their findings in the July 6 issue of the journal JCI Insight.

Using rhesus monkeys — a recent advancement in modeling congenital CMV transmission in humans — the researchers tested whether the offspring of mothers exposed for the first time to the rhesus form of CMV (RhCMV) could be protected from infection if the mothers first received RhCMV-specific antibodies.

The question is important, because the answer clarifies the type of immune response a successful vaccine approach should target.

“Ending congenital CMV infection is likely going to require an effective vaccine given before pregnancy, similar to how the rubella vaccine has eradicated congenital rubella syndrome in the Americas,” said first author Cody Nelson, a Duke Medical School MD/PhD student. “It’s important to know whether a successful CMV vaccine can rely on antibody responses alone, or whether we need to introduce other immune responses, such as T cells.”

In experiments with pregnant monkeys, the RhCMV-specific antibodies protected the mothers from losing the developing fetuses, which is what can happen when the mothers are infected with RhCMV for the first time during pregnancy.

A higher dose of the antibodies completely blocked transmission of the virus in each of the three monkeys that received it, and also reduced the virus’s ability to reproduce and mutate.

“Most vaccines on the market today work through an antibody mechanism, so our study demonstrates that a vaccine for CMV can likely go down that traditional path,” Permar said.

In addition to Permar, Kaur, Cohen-Wolkowiez, and Nelson, study authors include Diana Vera Cruz, Dollnovan Tran, Kristy M. Bialas, Lisa Stamper, Huali Wu, Margaret Gilbert, Robert Blair, Xavier Alvarez, Hannah Itell, Meng Chen, Ashlesha Deshpande, Flavia Chiuppesi, Felix Wussow, Don J. Diamond, Nathan Vandergrift, Mark R. Walter, Peter A. Barry, and Katia Koelle.

The study received support from the National Institutes of Health Nonhuman Primate Reagent Resource (R24 676 OD010976) The National Institute of Allergy and Infectious Diseases (HHSN 272201300031C); NIH (DP2HD075699, OD011104, R01AI103960, R01AI63356, F30HD089577).

Study finds children carry implicit bias toward peers who are overweight

June 23, 2017 – Participants were more likely to think positively of children who are healthy weight.

Even children as young as 9 years old can carry a prejudice against their peers who are overweight, according to a new study led by Duke Health researchers. They might not even realize they feel this way.

The study, published online today in the journal Pediatrics, sheds important insight into implicit weight bias in children and could serve as a starting point for further studies on the subject.

“When children are stigmatized for being overweight, it can cause further weight gain and other health consequences,” said Asheley Skinner, PhD, associate professor of medicine at Duke University School of Medicine, member in the DCRI, and the study’s lead author. “Given that, we felt that it was important to determine if we could identify unconscious attitudes towards weight in this 9-to-11 age group.”

The study included 114 children. The authors used a research method that primes subjects by using quick flashes of a series of carefully selected images that depict the study topic, juxtaposed with neutral images. Skinner said the study is the first to use this method, known as the Affect Misattribution Procedure, to consider attitudes that children have about weight.

The image used to measure bias showed a child engaging in an activity that was either related or unrelated to weight, like running or participating in class. Photographs alternately showed children involved in the activities who were either healthy weight or overweight, but were otherwise similar.

Study participants first viewed an image of a child engaging in an activity, followed by a neutral, abstract image. They then rated the abstract image “good” or “bad.”

After study participants saw these photographs, they were shown a neutral, abstract image, and asked to rate it as “good” or “bad.” On average, participants rated 64 percent of the abstract images preceded by images of children who were healthy weight as “good,” but did so for only 59 percent of second images preceded by children who were overweight, a difference that was statistically significant among the participants.

In the absence of bias, the study’s authors suggest that there would have been equal percentages of “good” ratings, since the preceding images of children performing the same activity were similar except for weight. The gap between “good” ratings therefore represented an overall implicit bias rate of 5 percent against the children who were overweight, according to the authors.

Previous studies by Keith Payne, PhD, of the University of North Carolina at Chapel Hill (UNC) and a co-author on the current study, have measured similar rates of implicit racial bias among adults. In this study, participants who were healthy weight had higher rates of implicit bias than those who were underweight or overweight.

“The main takeaway is that weight bias and a preference for thin people appears to start at a fairly young age,” Skinner said. “Knowing that this kind of implicit bias exists among children this age allows us to potentially be more aware of the unintended ways that children who are overweight might be stigmatized.”

“Implicit bias is important because it may underlie decisions among children about friendship, participation on sports teams and even bullying,” added senior author Eliana Perrin, MD, professor of pediatrics at Duke and the study’s co-principal investigator. “It’s essential to raise awareness about this kind of bias because it can have real consequences for children.”

Perrin said the work benefited from an interdisciplinary approach, with a research team that included physicians and social scientists, including co-principal investigator Andrew Perrin, PhD, a sociologist at UNC.

In addition to Skinner, E. Perrin, Payne and A. Perrin, co-authors include Janna Howard, Abigail Panter, Anna Bardone-Cone, Cynthia M. Bulik, and Michael J. Steiner.

The study was supported by the National Institutes of Health/National Cancer Institute (R24CA186212). The authors report no financial conflicts.

Drones carrying automated external defibrillators could prove lifesaving for cardiac arrest patients

June 20, 2017 – Investigators call for more research to see whether drone delivery of automated external defibrillators (AEDs) has the potential to be a “game changer” for patients who experience sudden cardiac death (SCD).

An editorial by Duke researchers published today in Circulation, the official journal of the American Heart Association, analyzes a potentially revolutionary solution to overcoming delays in getting an AED to the site of a patient who experiences cardiac arrest within a suitable window of time after a 911 call – AED-equipped drones.

SCD, or cardiac arrest, is a sudden unexpected loss of heart function, usually due to development of a very rapid disorganized heart rhythm that makes it impossible for the heart to continue to pump blood to the body. SCD is a leading cause of death in the United States and worldwide. The current treatment is primarily CPR and calling emergency medical services to shock the heart with an AED and administer medications.

However, each minute that passes lowers the survival rate by an additional 10 percent. Overall current survival to 30 days after out-of-hospital cardiac arrest is very low and remains one of medicine’s most vexing public health problems. When cardiac arrest occurs in a small area with an AED close at hand, such as an airplane or a casino, survival rates are much higher – 40 percent or more.

“An AED is basically a portable device that checks the heart rhythm and can send an electric shock to the heart to try to restore a normal rhythm,” said the DCRI’s Daniel Mark, director of outcomes research.

According to Mark, first author of the editorial, anyone can use an AED to help save a life. However, the only two current options of making these lifesaving devices accessible for use in a devastating emergency are placing retrievable static AEDs in public locations or having first responders physically bring one to the emergency site.

“The problem with static AEDs is that they are static,” said Mark. “And while community-wide emergency medical systems are in place to attempt delivering interventions to cardiac arrest patients within eight minutes of a 911 call, the absolute survival for out-of-hospital cardiac arrest continues to remain at or below 10 percent and we need to be able to get AEDs to the patients who need them much faster than we do now.”

Recent research published in multiple medical journals, including Circulation and JAMA, have begun to look at a novel answer: drone delivery of AEDs. However, there are many technical challenges in designing a drone for this sort of work. The drone must be not only very fast and capable of carrying the weight of an AED but also highly reliable, able to function in all weather conditions, and include advanced onboard collision avoidance technology and radar.

“In the United States, despite much interest and excitement, there are a considerable number of barriers to operating drones for medical purposes,” Mark said. “The Federal Aviation Administration that regulates our airspace, for starters, will require serious persuasion in the form of rigorous safety information before they will likely be willing to let drones fly beyond the line of sight of the operators in the U.S.”

“It is undoubtedly a revolutionary idea that needs to be explored,” Mark said. “But even as the engineers develop better drone technology and all the regulatory hoops are jumped through, we still have to prove the idea really works. We have to show that we can get a drone to a cardiac arrest site within three minutes of a 911 call and that once it arrives, the AED is used for its intended purpose.

“It’s not enough to show this can be done in a few cases. We need to show it can be done routinely and that doing this actually improves survival after cardiac arrest at the community level. And of course, we need to show that establishing and maintaining this service is economically viable.”

“I don’t have any doubts that eventually we would get the right combination of technology and regulation to be able to see the idea of AED-equipped drones through,” Mark said. “But are we close? No. Not yet.”

In addition to Mark, other contributing authors included Steen M. Hansen, Monique L. Starks, and Mary L. Cummings.

DCRI names Bray Patrick-Lake director of stakeholder engagement

June 15, 2017 – She will lead participant engagement work on several DCRI projects.

The DCRI has appointed Bray Patrick-Lake as its director of stakeholder engagement.

Bray Patrick-LakePatrick-Lake previously served as director of stakeholder engagement for the Clinical Trials Transformation Initiative (CTTI) and director of patient engagement for the Duke Clinical and Translational Science Institute (CTSI). She also served as co-chair of the advisory committee to the NIH Director for the Precision Medicine Initiative Cohort Program (now All of Us), which was launched by the Obama administration in 2015 to create new models of patient-powered research and provide clinicians with new tools and therapies. Recently, Patrick-Lake was named to the National Academies of Science, Engineering, and Medicine Health Science Policy board.

In her new role, Patrick-Lake will lead the DCRI’s participant engagement work on Project Baseline, a partnership with Verily, Duke, and Stanford to develop a well-defined reference, or “baseline” of health, and the NIH Environmental Influences on Child Health Outcomes (ECHO) program.

“The DCRI is committed to researching, understanding and supporting participant engagement in clinical trial design, conduct, and oversight,” Patrick-Lake said. “Multi-stakeholder teams can better advance high-quality, efficient research that’s also patient-centric. The culture at the DCRI supports a multi-stakeholder approach—we value all partners and all people. And when we do this, we succeed and innovate.”

Patrick-Lake has founded and led several non-profit organizations including the PFO Research Foundation, which she launched in response to the lack of definitive scientific information regarding the condition of patent foramen ovale (PFO). Patrick-Lake was motivated to work on issues of clinical trial design, informed consent, and data sharing after she participated in an aborted PFO clinical trial during which she had a device implanted in her heart. The data from that trial were never published or shared with patients and investigators.

“By understanding the challenges in research and bringing all parties’ unique skills, assets, and perspectives to the table, we can develop innovative solutions and do better research,” Patrick-Lake said.

Patrick-Lake holds a BS in zoology from the University of Georgia and a Masters in Forensic Sciences degree from National University in La Jolla, California.

American Heart Association funds trials to study, treat childhood obesity

June 14, 2017 -The projects include both clinical and basic science research on the causes and effects of obesity in children.

Duke Health and DCRI researchers will launch four projects this summer to better understand and treat the health impacts of childhood obesity.

The projects include clinical and population health research on the most effective treatments for childhood obesity and basic science research on differences in gut bacteria among children who are overweight compared to those in a healthy weight range, and how those differences might influence their risk of obesity and response to treatment.

Duke will conduct the studies as part of the American Heart Association’s Strategically Focused Research Network (SFRN) for children, which will provide $3.7 million over the next four years for the research. The DCRI’s Jennifer Li, MD (pictured), chief of the Division of Pediatric Cardiology at Duke University School of Medicine, will lead the work.

“Unfortunately, up to a third of children are obese or overweight,” said Li, who is also a professor of medicine and of pediatrics. “This is a generation of kids who might not do as well as their parents because they face a future risk of heart attacks, diabetes and stroke. This grant can help us figure out the best interventions, including those that might work on a larger scale in communities across the country.”

The clinical research portion will focus on the effectiveness of two existing Duke programs called Bull City Fit and Healthy Lifestyles, which the health system has offered since 2012. These programs combine regular exercise, nutrition classes, family involvement and monthly medical evaluations.

The AHA-funded clinical trial will enroll 350 youngsters and will be the most comprehensive study to date on the program’s impact on weight, physical fitness, quality of life, family engagement and more, said principal investigator Sarah C. Armstrong, MD, a pediatrician and associate professor of pediatrics.

“The proposed work aligns in mission with the American Heart Association’s goals of improving child health and well-being,” Armstrong said. “It’s exciting to get to work with an organization that so clearly supports children and their future health.

Cardiologist Svati Shah, MD, an associate professor of medicine and member of the Duke Molecular Physiology Institute and the DCRI, will lead the basic science research defining how molecular pathways associated with children’s gut bacteria could influence their obesity risks and other outcomes. Shah is also leading a project for a different AHA SFRN launched in 2016 to study heart failure in adults.

Asheley Skinner, PhD, associate professor of medicine, will lead the population-health project examining obesity treatment programs across the country to evaluate different models and their effectiveness.

For the fourth project, Skinner, also a member of the DCRI, will lead training for scientific, clinical and population health professionals on issues related to childhood obesity, cardiovascular disease and their prevention and treatment.

In addition to Duke, the AHA will fund projects at three other institutions to study pediatric heart health and diseases: Children’s National Health System in Washington, D.C., the University of Utah and Northwestern University.

Study creates benchmarks for evaluating community-based palliative care

June 13, 2017 – Understanding the components of a community-based palliative care model is the first step to designing incentives to encourage its spread, researchers say.

Use of palliative care, an interdisciplinary approach to caring for individuals with life-limiting illness focused on improving quality of life, is increasing as our population ages and evidence about its benefits grows. However, there is still little information about which care activities are necessary for delivering high-quality palliative care in the community.

A team led by Nrupen Bhavsar, PhD, MPH, of Duke’s division of general internal medicine and Donald H. Taylor, Jr, PhD (pictured), of the DCRI, the Margolis Center for Health Policy, and the Sanford School of Public Policy, worked with partners at Four Seasons Hospice organization to conduct a time and motion study at three care settings, resulting in a detailed process map. The team calculated the time spent on patient care, administrative duties, care coordination, and other activities.

“There is wide variation in how providers deliver palliative care,” said Taylor, “this study is the first step to designing incentives for high-value palliative care, including the development and sharing of best practices and generating evidence that can help us establish a value-based payment model.”

The study was published in the Journal of Palliative Medicine. Other members of the research team include Janet Bull, MD, MBA of Four Seasons, and Kate Bloom, MPH, Jonathan Nicolla, MBA, Callie Gable, BA, Abby Goodman, BS, Andrew Olson, MPP, and Matthew Harker, MPH, MBA.  Olson and Harker are members of the DCRI and Duke-Margolis.

Funding for this research was made possible, in part, by the Centers for Medicare & Medicaid Innovation through grant 1C1CMS331331.

DCRI’s Neurosciences Medicine concludes an active spring

June 7, 2017 – DCRI faculty and staff presented their most recent research at several conferences over the past few months.

As DCRI’s Neurosciences Medicine therapeutic area grows, faculty and business development have prioritized developing a presence at top neurological and psychiatric meetings around the world. This spring, the DCRI exhibited at two such conferences to demonstrate excellence in thought leadership and research: the American Academy of Neurology (AAN) conference in Boston, Massachusetts, and the World Intracranial Hemorrhage Conference (WICH) in Baltimore, Maryland.

neurosciences-med-sponsor.pngAAN Conference

Jeffrey Guptill, MD, assistant professor of Neurology and associate director of the Early Phase Unit (DEPRU) and Aatif Husain, MD, professor of Neurology and Global Sleep and Epilepsy Trials lead at DCRI observed and contributed to scientific presentations at AAN and met with potential sponsors focused on early phase trials and the importance of EEGs in research. Danny Laskowitz, MD, director of Neurosciences Medicine, also represented the group at AAN and co-hosted the “AAN continues to be a crucial platform for DCRI Neurosciences to have visibility in our industry,” said Laskowitz. “It is the largest arena for us to showcase our latest research and the excellence in thought leadership we’ve assembled in Neurosciences Medicine.”

​Other attendees from the DCRI included Chief Operating Officer Baljit (Boo) Samra, Jennifer Hart, assistant director Business Development for Neurosciences Medicine, and members of the Grants and Proposal Services team: Andrew Miles, Jen Fitzpatrick, and Betsy Younce. The reception served as a “thank you” to current stroke and ADHD sponsors for their dedication and collaboration with DCRI’s Neurosciences Medicine. In addition to current booth.pngsponsors, potential sponsors attended as well, creating a great networking experience for all.​

World Intracranial Hemorrhage Conference

Following AAN, DCRI’s Neurosciences Medicine traveled to Baltimore in early May. WICH is the first conference of its kind—dedicated exclusively to acute hemorrhagic brain conditions. Due to DCRI’s work on a phase II study in intracranial hemorrhage (CATCH trial), it was essential for the coordinating center to have a presence and voice at this groundbreaking event.

Michael Luke James, MD, anesthesiologist and critical care specialist-neurologist, and Jen Hart presented, “A proof of concept study to evaluate the administration of CN-105 in participants with acute supratentorial intracerebral hemorrhage.” CN-105, after 20 years of research, culminated in a promising new therapeutic for a patient population with no current options. Laskowitz and James designed the trial, and the study presentation was a collaboration between DCRI Neurosciences Medicine and Duke’s Neurology and Anesthesiology departments​.

Transforming registries into robust platforms for clinical trials

June 6, 2017 -Newly released recommendations from the Clinical Trials Transformation Initiative’s (CTTI) Registry Trials Project pave the way for efficiently transforming patient registries into reusable platforms for conducting clinical trials.​

CTTI recently unveiled a new set of recommendations to utilize existing and prospective patient registries to facilitate high quality, efficient registry-embedded clinical trials. The recommendations provide an essential framework for assessing and designing registries and can be applied to existing registries or used for developing new ones.

John Alexander

“The goal of these recommendations is to increase the practice of leveraging existing patient registries to facilitate high-quality clinical trials at lower costs,” said DCRI’s John Alexander, MD (pictured), co-chair, CTTI. According to him, existing registries collect data for the purpose of generating clinically usable information and evidence. “While registries have long been used to support non-randomized safety evaluations, their use for randomized or non-randomized efficacy evaluations is a newer practice,” he said.

“Depending on type-characteristics, some registries are more appropriate than others for conducting clinical trials. Regardless, CTTI’s Registries Trials Project has found a clear opportunity for registries to create a sustainable infrastructure to conduct clinical trials,” added Jules Mitchel, PhD, president, Target Health Inc. According to Mitchel, the new recommendations are the next step towards evolving how both researchers and sponsors think about clinical trial conduct, with faster, fit for purpose clinical trials as the end goal.

To determine if an existing registry is appropriate for embedding clinical trials, the new CTTI recommendations propose assessing whether the registry data demonstrates relevancy and robustness to support regulatory decision-making. The registry data must also have assurance of patient protections with the maintenance of patient and data privacy. To design a new registry suitable for embedding clinical trials, the CTTI recommendations advocate following software industry guidelines, as well as guidance documents provided by regulatory agencies, to assure that the registry complies with both industry and regulatory standards.

While the scale and scope of this project was limited to patient registries, CTTI’s multidisciplinary team believes that many of the principles and tools listed in these recommendations can potentially be applied to other health care systems and existing data sources, or those available within claims databases, to facilitate more cost-effective and competent clinical trials.

“We hope that with these recommendations, people will approach the design and conduct of clinical trials in a more systematic way that will ensure useful, high-quality data, including for FDA approval, on new devices and drugs,” Alexander said.

Established by the FDA and Duke University in 2007, the CTTI is a public-private partnership hosted by Duke University and based at the DCRI with a mission to develop and drive adoption of practices that increase the quality and efficiency of clinical trials.

Project team leaders included Duke’s James Tcheng, Celgene’s Dawn Flick, FDA’s John Laschinger, and Medtronic’s Ted Lystig. Other team members were Duke’s Sunil Rao and Emily Zeitler, Chunrong Cheng and Kristen Miller from the FDA, Christopher Dowd from the Cystic Fibrosis Foundation, Nicolle Gatto from Pfizer, Lauren Mclaughin from the Michael J Fox Foundation for Parkinson’s Research, Patient Representative Stephen Mikita, Daniel Mines from Merck, Magnus Petterson from AstraZeneca, Celgene’s Arlene Swern, and Mitchel. The current and previous project managers were Duke’s Sara Calvert and CTTI’s Steve Mikita, respectively.

More information about CTTI’s recommendations is available both on the Registry Trials Project page and in a webinar recording.