A Memoir of a Mother’s Determination and Her Micro Preemie’s Struggle to Beat the Odds
I recently had the honor and pleasure of participating in the 3rd Annual FDA-INC Neonatal Scientific Workshop in Bethesda, Maryland.
My role, as co-chair of the Communications Workgroup, was to plan and execute a 90 minute Breakout Session entitled “Communication Strategies to Promote a Research Culture”.
Christina Bucci-Rechtweg (a pediatrician by trade who now works for Novartis Pharmaceuticals) was the other co-chair and I could not have asked for a better partner. Christina and I have known each other for a little over a year and I have had the pleasure of speaking with her on Capitol Hill on two different occasions (both addressing the Senate and the House of Representatives) on behalf of S.2041 and H.R. 5182. Christina is brilliant and hard-working and was instrumental in planning our Breakout Session activities.
But before I get into what our session was about and what we accomplished, I’m betting that you would like to know what INC and C-Path are and what the goal of the 3rd Annual FDA-INC Neonatal Scientific Workshop actually was.
C-Path (Critical Path Institute) was founded in 2005 in Tucson, Arizona, and is an independent, non-profit organization dedicated to bringing scientists from the FDA, industry and academia all together to collaborate and improve the drug development and regulatory process for medical products.
INC (International Neonatal Consortium) launched on May 19, 2015, and is C-Path’s ninth consortium – a global collaboration formed to forge a predictable regulatory path for evaluating the safety and effectiveness of therapies for neonates.
“By uniting stakeholders from research institutions, drug developers, regulatory agencies, patient advocacy and other organizations,” said Janet Woodcock, Director of the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (FDA/CDER), “INC can develop practical tools that can be incorporated into clinical trials for neonates, which will then lead to more successful, efficient trials and provide this population with better treatments.”
INC’s Statement of Purpose:
INC will accelerate the development of safe and effective therapies for neonates. The consortium will engage the global neonatal community – families, neonatal nurses, academic scientists, regulators, pharmaceutical investigators, advocacy organizations, and funders – to focus on the needs of the neonate. Through teams that share data, knowledge, and expertise, INC will advance medical innovation and regulatory science for this underserved population.
I’m honored to be a part of INC!
At this workshop, co-sponsored by the FDA and Critical Path Institute, participants reviewed the progress of INC, discussed the challenges in conducting registration trials to prevent preterm birth, and the breakout groups tackled specific regulatory science issues for developing safe and effective therapies for neonates. Regulators from around the world engaged in working sessions with academic experts, interested companies, neonatal nursing organizations, NIH institutes, family groups and nonprofit organizations.
Day 1 began with several meetings of the working groups, including Severity for Neonatal Adverse Events, Retinopathy of Prematurity, and the Seizures Workgroup, and it ended with a workgroup dinner reception at the Bethesda North Marriott Hotel.
Day 2 began with Dr. Jonathan Davis (Tufts University, INC Co-Director) making the opening remarks. I also know Dr. Davis for over a year as he was the spearhead behind the Senate and House bills I mentioned above, and I had the pleasure of speaking on Capitol Hill with him. Dr. Davis has dedicated his entire career to bettering the outcomes of premature babies. Next, Susan McCune (Director of Office of Pediatric Therapeutics, FDA) gave a presentation titled “A New Era for Developing Neonatal Therapeutics”. After she was done speaking, each workgroup had a few minutes to share updates about what they have done and looked to accomplish in their Breakout Sessions. Soon after the workgroup summaries were complete, a presentation titled “Developing Endpoints for Use in Regulated Neonatal Trials” was given by Gerry Baer (FDA) and Ralph Bax (European Medicines Agency).
After a short coffee break, there was a panel session titled “Plenary Session on Challenges in Conducting Registration Trials to Prevent Preterm Birth”. Several people participated in this session, including Mark Turner (University of Liverpool, INC Co-Director), Olof Rugarn (Ferring Pharmaceuticals), Yosuke Komatsu (GlaxoSmithKline), Louise Kenny (University College- Cork Ireland), Errol Norwitz (Tufts Medical Center), Mehali Patel (Bliss), Deb Discenza (Preemie World and member of Preemie Parent Alliance), Ralph Bax (European Medicines Agency), and Barbara Wesley (FDA). Nicole Thiele from EFCNI (European Foundation for the Care of Newborn Infants) also called in and gave a presentation via WebEx.
Following the Plenary Session, we all took a break to eat lunch and get ready for the Breakout Sessions.
There were several concurrent Breakout Sessions, including the one that I was a part of, so I can only summarize what went on in the other sessions, after giving details about mine.
Christina Bucci-Rechtweg and I led the Breakout Session called “Communication Strategies to Promote a Research Culture”. We had a few goals. We wanted to build strategies that embed neonatal research that is family-centered in the NICU culture. In order to do this, we looked to first identify communication challenges in neonatal units that impede successful implementation of research.
We planned a mapping activity where we asked the participants to answer four questions regarding how parents are approached to enroll their babies in clinical trials and what makes them more likely to consent. This activity gave us a great deal of information on the current practices surrounding clinical trials in the NICU. Both Christina and I shared an informal literature review of some of the articles previously published on clinical trials in the NICU, from both the medical and the parent perspective. We also shared results from a survey we had previously given to parents of premature babies who are members of the Preemie Parent Alliance. This was also very telling and we are in the process of analyzing the entire survey, along with the parent comments and we will use that information to harness multi-stakeholder collaboration to address communication challenges and improve the quality and efficiency in neonatal research. Our session ended with a phone presentation given by Nicole Theile of EFCNI on a white paper they put out on the topics we were discussing.
Although Christina and I led the Breakout Session, we had a lot of input from the other members of our workgroup as well. I only recently joined INC and this workgroup so I am especially thankful to the other workgroup members for all of their work. They include Wakako Eklund (National Association of Neonatal Nurses), Lynn Hudson (C-Path), Carole Kenner (Council of International Neonatal Nurses), Jeff Ming (Sanofi Pharmaceuticals), Lily Mulugeta (FDA), Min Soo Park (Yonsei University), Ronald Portman (Novartis Pharmaceuticals), Randy Prescilla (Boston’s Children Hospital), Thomas Salaets (University of Leuven), Catherine Sherwin (University of Utah), Ine Skottheim Rusten (Norwegian Medicines Agency & PDCO), Adina Tocoian (Shire), Mark Turner (University of Liverpool), John Van Den Anker (Children’s National Health System), Sander Vinks (Cincinnati Children’s Hospital Medical Center), Kelly Wade (Children’s Hospital of Philadelphia), Siri Wang (Norwegian Medicines Agency and PDCO), and Anne Zajicek (National Institute of Child Health and Human Development/National Institutes of Health).
Two other Breakout Sessions were running concurrently to ours and those were titled “Multiple Enrollment in Clinical Trials” (Co-Chaired by Dr. Jonathan Davis of Tufts Medical Center/INC Co-Director and Gerri Baer from the FDA) and “Long-term Outcomes” (Co-Chaired by Neil Marlow from the University College London and Mark Turner from the University of Liverpool/INC Co-Director). Although I could not attend either of those sessions, I heard their summaries at the end of the day and it seems as if both sessions were very successful.
The conference continued on with more concurrent Breakout Sessions after a short coffee break. I attended the session titled “Regulatory Challenges in Conducting Trials to Prevent Preterm Birth” (Chaired by Mark Turner). The other two Breakout Sessions were “Developing a Neonatal Common Protocol Template” (Co-Chaired by Ron Portman from Novartis Pharmaceuticals/INC Co-Director and Anne Cropp of Echelon Pharma Solutions) and “Applying Generic Severity Grading Criteria to Persistent Pulmonary Insufficiency of Prematurity” (Co-Chaired by Karel Allegaert and Thomas Salaets from the University of Leuven).
Towards the end of the day we all assembled again as leaders from each Breakout Session reported back to the group with how their Breakout Session went, what was accomplished and what their next steps would be.
There were two more presentations given before Mark Turner’s closing remarks and our dinner reception.
First was “The Role of INC in Pediatric Trial Networks” by Edward Connor (PTC, I-ACT for Children) and William Tremm (Janssen Research & Development) and second was “Nonclinical Models of Neonatal Therapies” by Susan McCune (FDA). Both gave us a lot to think about as we me move towards our next steps on our INC committees.
Although I have spoken at several NICU events and nonprofit fundraisers in the realm of premature births, this was my first time participating in a conference where I felt that real change was being enacted towards preventing premature births as well as improving the treatments and medications used on our world’s current and future premature babies. As a member of The Morgan Leary Vaughan Fund’s Board of Directors, I can speak for the whole organization in saying that we are excited at the thought of new clinical trials being done to produce new medications to treat and cure Necrotizing Enterocolitis.
I can only hope that my present inexperience as a member of the International Neonatal Consortium (INC) will lead to vast experience in this space, as I continue to serve on INC’s Communications Workgroup. I look forward to what we will accomplish.
One of our first goals following this workshop is to create and publish INC’s quarterly newsletter.
Be on the lookout for it.
I want to mention two members of the Preemie Parent Alliance (PPA for short) that attended the event. Deb Discenza runs Preemie World, which she started after her daughter’s premature birth 13 years ago. Deb also spoke at the workshop. Dr. Yamile Jackson attended the workshop as well. She runs Nurtured by Design, a company she started after designing The Zaky and Kangaroo Zak. They are developmental products used on premature babies in the NICU and I highly recommend them both. She started her company after her son’s premature birth 15 years ago.
I would like to personally thank Christina Bucci-Rechtweg for her leadership and patience as I acclimated to the INC Communication Workgroup.
Alicia West, Laura Butte and Lynn Hudson of C-Path deserve a lot of thanks (and rest!) for all of their hard work in putting the workshop together.
I would also like to thank Wakako Eklund (NANN) for taking such wonderful photos at the event and allowing us to share them. She is a very talented Neonatal Nurse Practitioner and photographer!
And last, I would like to thank Dr. Jonathan Davis (Tufts University/INC Co-Director) who suggested that I get involved in INC and allowed me to make such meaningful connections. The future premature babies of the world will be much better off because of the dedication of Dr. Davis and his passion for neonatology.
Please visit INC’s website to see how you can help us with our goals. After all, advancements in neonatology cannot happen without research.
Oh, and I even got to see a Cherry Blossom. It was not down by the Potomac, but at least I got to see one!
As always, thank you for reading and for your support 🙂
Please comment on anything you found interesting or would like to know more about.