This quarterly publication will keep you informed on who we are and what we are doing, as well as on key news in the world of pediatric clinical development. In this edition, we want to help you better understand and prepare for the upcoming legislation change in the USA: The RACE for Children Act.

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Edition Highlights:

RACE for Children Act

Legislation such as the Better Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have helped to increase development of safe and effective pharmaceuticals for pediatrics in the us. While these have been effective for many diseases, they have had virtually no impact on the development of more treatments for childhood cancer.

Staggering statistics for pediatric oncology in tandem with research in personalized medicine have given rise to an amendment to PREA which we anticipate will result in more drugs in the pediatric oncology pipeline. Incorporated as Title V of the FDA Reauthorization Act (FDARA), enacted August 18, 2017, the Research to Accelerate Cure and Equity (RACE) Act will dramatically impact the landscape of pediatric oncology research.

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Join Us For a Free Webinar!

The RACE for Children Act: Collaborating for Success
June 22 | 1pm EDT

We will be hosting a free webinar with The Leukemia & Lymphoma Society (LLS) regarding the collaborative efforts critical for the success of the RACE for Children Act. During this open discussion, you will learn about the intended impact of the RACE Act as well as the ground breaking LLS PedAL Trial and the monumental collaboration already underway among non-profits, industry pharma partners, pediatric consortia, genomics providers and Clinical Research Organizations (CROs).

Register

Did You Know?

Pediatric cancer is the #1 cause of death by disease in children in the USA with an average 68.3 years of life lost to pediatric cancer. And there are over 900 drugs in the adult cancer pipeline, with just a handful of these in development for children.

The Center for Pediatric Clinical Development is powering innovation in pediatric clinical trial design and implementation - because half isn't whole.

See how our team of pediatric clinical development experts are prepared to help you navigate the changes coming with the implementation of the RACE Act. We have compiled a library of resources to help you prepare for this historic transition in our industry.

Access RACE Act Resources

Children's Oncology Group

Children’s Oncology Group (COG), a National Cancer Institute (NCI) supported clinical trials group, is the largest organization in the world devoted entirely to childhood and adolescent cancer research across all spectrums including diagnosis and supportive care. COG and its members’ goal is to “…cure all children and adolescents with cancer, reduce the short and long-term complications of cancer treatments, and determine the causes and find ways to prevent childhood cancer”. COG believes this is done quickest and most efficiently by engaging scientists around the world.

  • Over 200 sites with 9000 experts across North America, Australia, New Zealand and Europe
  • 90% of the 14,000 pediatric patients diagnosed each year in the US are cared for at a COG site
  • COG has approximately 100 active clinical trials open at any given time

Key to COG is understanding the special needs of developing humans to the cutting edge of molecular basis for childhood and adolescent cancers. COG trials include frontline treatment, new therapies, supportive care and survivorship issues. COG has forums and recommended guidelines on long term follow up care to address the complex needs of survivors. Having standards set in place helps providers with anticipatory guidance and ongoing promotion of health and quality of life.

Pediatric consortia such as COG are critical for the successful conduct of pediatric oncology trials, and thus collaborations with consortia will be important for implementation of the RACE for Children Act.

How Do We Safely Send Kids to School During a Pandemic?

The COVID-19 pandemic has led to the nationwide closure of schools and universities in more than 190 countries, impacting 1.57 billion students.

Is it possible to safely send kids—and the adults who work there—back to school in the midst of a pandemic?

We focus on the needs of the child first and foremost in pediatric clinical research. By incorporating strategies employed in pediatric trials, such as counseling, assent, and age-appropriate resources, into school reopening plans, we can better support our students’ transition back to school.

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Recent Publications

Dr. Mark Sorrentino the Vice President, Center for Pediatric Clinical Development published an article called "We Can Do Better: Key Considerations for Paediatric Trial Success" in the February issue of the International Clinical Trials journal. In the piece, Mark discusses the challenges around enrollment and retention and the improvements needed around design and selection.

Missy Hansen, Pediatric Strategy Liaison, Center for Pediatric Clinical Development authored an article titled "Challenges Arise as the Need for Pediatric Research Increases" for Outsourcing-Pharma in May. The piece discusses the challenges unique to pediatric clinical trials and how oncology drug developers can approach these challenges as they prepare to run more pediatric studies as required by the RACE Act.

Read full article

Master Protocols

A lack of suitable pediatric patients to participate in clinical trials is already an issue. With the RACE for Children Act we expect to see an increase in the number pediatric oncology studies to be conducted globally. Given the rarity of pediatric cancers and the limited number of potential patients to participate in these trials, it is likely that more complex protocol designs will be utilized.

Master protocols are one option and are encouraged by the FDA who have issued draft guidance on master protocols for oncology products. The guidance defines a master protocol as "a protocol designed with multiple sub-studies, which may have different objectives and involves coordinated efforts to evaluate one or more investigational drugs in one or more disease subtypes with the overall trial structure". The aim is to expedite drug development through more efficient clinical trial design.

Master protocols are designed to study either multiple therapies, with direct comparison to competing therapies, or to their respective controls in parallel, or multiple diseases. They can be split into three distinct types; basket trial, umbrella trial and platform trial.

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Meet the Newest Additions to the CPCD!

Martine baby pic

Dr. Martine Dehlinger-Kremer, PhD
Vice President CPCD

Dr. Dehlinger-Kremer has expertise that spans more than 30 years in the research industry, including experience in regulatory, medical affairs, and pediatric leadership. She has global drug development experience in more than 40 countries. Her vision and leadership extend to service with a number of professional organizations, including Enpr-EMA, the European Network of Pediatric Research at the European Medicines Agency, European Forum for Good Clinical Practice (EFGCP) Children Medicines Working Party, the International Children’s Advisory Network, and the European CRO Federation.

Missy baby pic

Missy Hansen MSN, APRN, CNP
Pediatric Strategy Liaison, CPCD

Missy comes to PRA with over 25 years of experience as a pediatric nurse practitioner, 15 of which in pediatric hematology/oncology. Missy will be using her expertise to continue to help develop and promote pediatric trials with her initial priority on those within the pediatric oncology area given the upcoming RACE Act.

Jami baby pic

Jami Thompson
Senior Pediatric Site Network Specialist

Jamie brings extensive feasibility and site intelligence expertise from her almost 14 years in these groups at PRA. She joins us to support the impressive growth the PSN has undergone in the past 2 years. Jami will focus on building and maintaining critical strategic relationships with world class organizations.

Q&A with LeAnn Jackson

LeAnn joined PRA in October 2018 as a Senior Project Manager, working primarily on oncology clinical trials. We sat down with her to hear her personal story about why she entered into oncology clinical research (spoiler alert - it has to do with that smiling little boy she is running with in the picture on the right) and to get her thoughts on the RACE for Children Act.

Read full interview
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World Children's Day 2020

During PRA Health Sciences’ celebration of UNICEF’s World’s Children Day on November 20, experts from LSU-Shreveport, UNICEF, the Center for Information and Study on Clinical Research Participation (CISCRP), Jumo Health, the International Children’s Advisory Network (iCAN), and Sproutel were among the participants that converged on Blue Bell, PA. These dynamic speakers shared their expertise and insight into how they are making a difference and making the clinical trial experience more positive for pediatric patients.

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The major theme of all these presentations was the absolute need to engage our children more directly. That way, we can fully understand what they are going through. When we design clinical trials, we become so concerned with the science, agency requirements, and regulatory or ethics considerations that we forget to consider how our design might impact our patients and their families.

Melanie Mendez-Villarrubia, the young patient and advocate, said it best: “We are people first and we have lives outside of your study.” If our industry can approach trial design with this sentiment front of mind and prioritize ways to engage with the patients from the beginning, we can expect increased participation, better compliance, and a more positive overall experience for the participants.

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Correction

In some prior references to Sproutel and their innovative Jerry the Bear, it may have appeared that PRA and Sproutel have a formal relationship. Though we do look forward to ongoing collaborations, there is currently no formal agreement in place. We were thrilled to have Aaron Horowitz, CEO of Sproutel, join us at our World Children’s Day event in the Blue Bell office last November.

Interested in learning more about any of the information mentioned here or want to talk with someone about a specific clinical study?

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