CTSI Success Stories: Research Help, other CTSI resources, makes life easier for study coordinators

Elizabeth Werner, senior health project coordinator, and Ann Marie Scorsone, senior human subject research coordinator, have utilized CTSI resources for several of their research studies with the Division of Neonatology. They recently spoke to CTSI Stories about their experiences.

Elizabeth Werner, left, and Ann Marie Scorsone, right.

Thanks for taking the time to chat! Tell us a little bit about your work and how you came to use the resources within the CTSI.

Werner: We’re research coordinators in the Neonatal Intensive Care Unit (NICU), and I’m currently doing a test with infants where we put stretchy bands around their chest and abdomen and measure whether their breathing is synchronous or asynchronous, which can have health implications. However, the band itself is not FDA approved, so our study is a device trial because the FDA wants to ensure that the babies are safe and that they won’t be harmed by this.

I’ve personally used the Research Help Desk for their clinicaltrials.gov support, because that site can be really hard to navigate. Carrie Dykes — and before her, Cindy Doane — have both come over and helped me through the system and have identified the problems that I’d been having.

Scorsone: I’m part of the National Institute of Child Health and Human Development (NICHD), and within that is the neonatal research network. So we currently have about 10 studies right now that we’re doing in the NICU as part of that network.

One of those studies is something we’ve been engaged in for 15 or more years: Dr. Dale Phelps’s study on retinopathy of prematurity (ROP), which is one of the leading causes of early childhood blindness or long term eye dysfunction. We’re in a Phase III FDA trial with that right now and it includes a long term follow up, so we had Ann Dozier come speak with us about research subject retention. And Dr. Dozier really helped us in Rochester come up with ideas on how to help approach the families and talk about the long term follow up right in the beginning.  We get a great follow-up rate at their 2-year visits – it’s over 90 percent – and she’s been a big help with that.

What types of suggestions did she make?

Scorsone: She talked about really understanding what gets in the way in terms of participation, and why you tend to lose people as a trial goes on. We’ve really tried to maintain contact with our families so they feel like they’re not just part of a research project, but actually a part of a family here.

Werner: We talk to people about how this is a research institute, and how things that are helping your baby today started with studies that other families have participated in, in the past. That attitude, I think, has helped shape our whole division. We’ve really worked hard over the past five years to make both the patients and the staff feel like part of a research community. One thing we have done to help this is to set up a NICU research tent at the Stroll for Strong Kids to present study information there.

Scorsone: And we also have high visibility in the NICU itself. We make sure the research team is down there, and we’re talking to families all the time – not just showing up when we need a blood draw. The idea is these people aren’t just subjects. We’re creating ties, which is what Ann had talked to us about – shaping the culture. So we present research to the staff down there, and we’ve identified nurse champions that are interested in research and want to help us. And they’ve really embraced it, because I think they can see the difference.

What other programs have you used through the CTSI?

Scorsone: I’ve been to Adam Tatro’s eRecord training, and he very graciously asked me to be part of his eRecord leadership group, so I gave a couple presentations as well. He’s extraordinary. We’ve actually had him come here and give our coordinators a private workshop because we do so much data extraction for all of our studies.

Werner: We’re working with Adam on i2b2 as well, and we’ve leaned on the CTSI for REDCap support. I have Amanda Davin on my speed dial. I also like many of the seminars that are run over there – I’ve learned a lot from those.

Have you used the Research Request Dashboard?

Scorsone: Yes. Dr. Patricia Chess was thinking about doing a pilot study of a device. There was a company potentially interested in potentially buying the patent. So that involved some techie licensing stuff that goes way beyond my coordinator role. But through the dashboard, I was just able to write “Hey, I have an investigator who wants to do this,” and they connected me with Joan Adamo.

So that was really neat, they’re able to connect you with all these resources in-house with the click of a button, and I didn’t have to struggle to figure out those protocols myself.

UR CTSI Working to Advance Regulatory Science Training

Dr. Joan Adamo and Dr. Scott Steele recently published an article in the Clinical and Translational Science Journal entitled “Advancing a Vision for Regulatory Science Training.” Regulatory science is a new and burgeoning field and is defined by the FDA as “the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality and performance of FDA-regulated products.” Training programs in regulatory affairs, which is learning what the regulations are and how to apply them to projects is very common. However, training in regulatory science is less developed. In this article, Drs. Adamo and Steele advance regulatory science by identifying 11 thematic areas of training that contribute to regulatory science. These themes were identified by a Regulatory Science workgroup and then vetted by a group of interdisciplinary experts during a workshop in Washington, DC last fall. In support of this workshop, an extensive Regulatory Science survey was conducted of experts from industry, academia, government, not-for-profit organizations, associations and foundations to further prioritize and refine the proposed Regulatory Science competencies. Some examples of the Regulatory Science competencies include regulatory policies and process, research ethics, drug discovery and development, clinical trials, post-marketing and compliance, and communication.

This work was done in collaboration with other CTSAs. Future plans include developing a training network by identifying which academic institutions have expertise in the various thematic training areas, creating opportunities to share training amongst institutions and providing experiential learning opportunities in the training areas. In the future, “investigators will be able to pick and choose training topics related to regulatory science that will bolster the work they do in their field of expertise”, says Adamo.

Many investigators engage in regulatory science and don’t realize they are doing it. “The CTSI is helping faculty realize that their research often impacts this field, even if their work does not directly involve the regulation of a product” says Steele. What scientists work on now may help speed the development of other drugs or devices in the future. Put simply, “Regulatory science is any science that comes up with new ways so that products can be regulated and be brought out safely”, says Adamo. This includes reducing costs and the time it takes to get a safe and effective product approved. Steele notes, “A key element to advancing regulatory science and translational science is enabling research and educational collaborations across FDA, NIH, industry, academia and foundations.”

Stay tuned. In the fall, the UR CTSI will offer a series of seminars on regulatory science. Experts from academia, industry and the FDA will be here to present their latest work in the field of regulatory science.

Winner of URMC’s “America’s Got Regulatory Science Talent” competition presents idea to FDA

In some patients, acute myeloid leukemia strikes hard and fast. And in one subset of these individuals, researchers now know why: a mutation in their DNA which results in a more aggressive path for the disease.

With this knowledge, providers can potentially screen for this mutation, allowing them to treat the patients more intensively. The discovery was another success in the world of genome sequencing, which is being studied more and more frequently as scientists and physicians learn more about how variations in a person’s DNA can affect how they respond to disease and medication.  This approach is often referred to as personalized or precision medicine.

From left to right: Scott Steele, Ph.D.; Corey Hoffman; Stephen Ostroff, M.D.; Joan Adamo, Ph.D.

To further advance personalized medicine, Corey Hoffman wants all of the genomic data from trials utilizing genome sequencing to be accessible in the same database. This will allow researchers and scientists to easily expand on previous work, identifying and using these genetic markers to speed drug development and aid in drug safety by targeting specific populations likely to respond to a given treatment.  Hoffman’s idea won URMC’s “America’s Got Regulatory Science Talent” competition in February, and earned him a trip to Maryland, where he presented his database idea at the FDA’s 2015 Office of Regulatory Science and Innovation Science Symposium.

“There are two potential outcomes that I could see,” said Hoffman, a predoctoral student at URMC, told CTSI Stories in March. “The first is that you could screen a patient’s genome so that when patients do respond well in a trial, maybe there’s a genetic element they share.

“Secondly, certain individuals don’t respond well to certain drugs because of how their body metabolizes the drugs… so you could screen people for mutations in their CYP gene to give indications for drug response, and who is and isn’t a good candidate for a certain drug.”

Hoffman, who was joined at the conference by University of Rochester’s Scott Steele, Ph.D., and Joan Adamo, Ph.D., presented his idea to the FDA’s Acting Commissioner, Stephen Ostroff, M.D. and other leaders and staff at the FDA.

URBEST opening enrollment for grad students, postdocs

Medical research training — for students and postdocs alike — is heavily geared towards an academic career path. But only 40 percent of Ph.D. holders go on to become professors, according to a 2012 study from the National Institutes of Health.

“There isn’t as much funding for PIs as anyone would like, and people are getting nervous as to what students and postdocs are going to be doing if there isn’t enough funding for them in academia,” said Tracey Baas, Ph.D.

Steve Dewhurst, Ph.D. (above, right) and Sarah Peyre, Ed.D., are the PIs for URBEST.

That’s where URBEST comes in. The program, which launched in October, is one of 17 NIH-funded BEST (Broadening Experiences in Scientific Training) programs throughout the country, and provides career training to graduate students and postdocs who are considering careers outside of academia.

BEST programs differ from institution to institution, but Rochester’s program places emphasis in two main areas.

First, URBEST is designed around self-determination theory. This means the curriculum is very flexible, allowing enrollees to dip their toe into the water or dive much deeper to obtain the experience they desire. (In order to obtain a URBEST certificate, students need to accumulate 120 “points” within the program, but those points can be accumulated in a variety of ways.)

Second, URBEST places heavy emphasis on connecting students to internships, which isn’t done at every BEST program.

“But we thought the internship would make it most attractive for both the students and for future potential employers,” said Baas, executive director of URBEST.

The program allows students to pick one of three pathways, each with its own associated coursework and internship options:

• Industry, manufacturing, and entrepreneurship (director: Paul Dunman, Ph.D.)
• Regulatory affairs, compliance, and review (director: Joan Adamo, Ph.D.)
• Science and technology policy (directors: Katrina Korfmacher, Ph.D.; Scott Steele, Ph.D.)

The program will be accepting applications for its next cohort from April 6-17. To apply, or for more information, visit the URBEST website.

Meet the winner of “America’s Got Regulatory Science Talent”

Corey Hoffman, a predoctoral student in the Medical Center, was the winner of the second annual “America’s Got Regulatory Science Talent” student competition held on Feb. 10 at the Clinical and Translational Science Institute. Hoffman will soon travel to Maryland to speak with the Food and Drug Administration about his project. This competition is organized by the University of Rochester Regulatory Science program, led by Joan Adamo, Ph.D. and Scott Steele, Ph.D. and held in conjunction with the Center for Excellence in Regulatory Science and Innovation at the University of Maryland.

Hoffman recently spoke with CTSI Stories.

Tell us a little bit about your proposal.

Right now, there are certain areas in regulatory science where the FDA wants to advance science, and within each area there are a few more specific focuses. The focus that stuck out to me is a clinical one that centers on personalized medicine, and looking for new types of biomarkers.

So what we want to know is: Is there something within the human body, a protein or DNA sequence that might be different in a healthy individual than someone who might be at a predisposition for a disease or who might have a disease. And how can we identify methods for finding these and then validating them?

Can you give an example of that?

There was one study on people with leukemia, and there was a small subset of patients that had a more aggressive disease and didn’t respond to chemotherapy. So the study was asking if there was something genetic that was different in those individuals, and it showed that there was indeed a commonality. So that was the example I used in the presentation.

What I’m proposing is to take advantage of all the clinical trials being done that are using sequencing like that, and put them all into a database. This could allow others to prevent redundancies in future trials and could be a reference point for future clinical studies or basic science studies.

Very cool. In a perfect world, what do you see as the potential outcomes from this type of database?

There are two potential outcomes that I could see. The first is that you could screen a patient’s genome, so that when patients do respond well in a trial, maybe there’s a genetic element that they share. Or, if there’s a commonality in people that don’t respond, then you could screen future patients ahead of time before you even offer the treatment. That way you wouldn’t have to put them through something if you knew ahead of time that they weren’t going to respond.

Secondly, certain individuals don’t respond well to certain drugs because of how their body metabolizes the drugs. This is determined by things called CYP genes, and certain people have mutations in these genes which affect how they respond to the drug. So you could screen people for mutations in their CYP gene to give indications for drug response, and who is and isn’t a good candidate for a certain drug.

Are there any challenges you see to developing a database like this?

I think the biggest concern before a database is ever constructed is protecting the patient health information of these genetic sequences, and I think that’s something that remains unexplored. There needs to be efforts that go into de-identifying the genomes. I’m not a geneticist, but it seems possible that a certain sequence of DNA could be unique to an individual that it could tell you who that person is. So, how can we take advantage of genetic information without putting any risk to the patients? And if these genome sequences are in the database, who has access? Those are two things that need to be considered.