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Advancing Pediatric Clinical Trial Innovation

2020 Annual Report

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In Service of Children

Our Mission

We work to accelerate and enhance the quality of pediatric clinical trials so that children have access to medicines that have been proven safe and effective for them.

We believe that children of all ages deserve innovative medical therapies that are developed with the same level of urgency and commitment afforded adults.

Table of Contents

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From the Leadership

I-ACT for Children: A Timeline of our Growth

2020 Fiscal Year Financial Activities

Key 2020 Achievements

Our I-ACT for Children Members

Looking Forward

Leadership, Board & Collaborators

Letters From Our

Leadership

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This is the first formal Annual Report for the Institute for Advanced Clinical Trials for Children and represents a summary of key elements of the Institute’s history, with a focus on 2020. We are grateful for all the support and engagement from diverse stakeholders who have helped launch the Institute as a trusted forum to help advance drug and device development for children and a collaborative network of centers, therapeutic area experts, product development specialists and advocates. In many ways, 2020 was a milestone in moving the conceptual framework developed from 2014 to 2016 into a strong foundation to enable durability and impact.

On behalf of the Board of Directors, I want to recognize the commitment and hard work of the Institute’s leadership and staff during this time of growth and development. Starting a new entity takes expertise and fortitude, and we appreciate all your efforts.

In 2020, we began a strategic planning initiative to shape and focus the Institute’s work over the next several years. This included feedback from representatives from our major stakeholders and others not yet formally part of I-ACT for Children. The process affirmed the Institute’s mission, vision, values and core strategies, and identified ways to ensure that we are providing real value in the pediatric product development enterprise. We anticipate that this will be completed in 2021 and serve as the foundation for I-ACT for Children for 2021 and beyond.

Edward Connor, MD, MBE, FAAP
Chairman of the Board

Five years ago, a group of creative-minded, determined stakeholders in pediatric research gathered to find a solution to the intractable problems related to medicines development for children. The Pediatric Trials Consortium saw an opportunity to create an independent, neutral organization that could work with public and private stakeholders to help expedite innovative drug development to advance the health of children. The concept included a hub of experts to advise regarding the feasibility and efficiency of pediatric protocols, combined with a standing network of qualified pediatric clinical trial sites.

Today, we are proud to say that we have built the foundation for what our founders envisioned. Over the last three years since its launch, The Institute for Advanced Clinical Trials for Children has built a strong foundation from the ground up. Its network now stands strong at 80 sites in the United States, South America, Australia and Saudi Arabia, and we provide global interoperability through agreements with the MICYRN pediatric network in Canada and a Belgian hub in Europe. Our staff provides guidance on clinical trial strategy, design and execution, complemented by a panel of 50 expert consultants in 18 priority therapeutic areas. We have taken on 26 projects since we launched operations, ranging from site identification to feasibility assessments, advisory boards and protocol design consultation. And we have completed several critical pre-competitive projects that address unmet needs and align with key regulatory priorities. The Institute now counts five biopharmaceutical sponsors among its members; was awarded a grant from the US Food and Drug Administration to support its work; and has established collaborations with many like-minded organizations representing a cross section of stakeholders.

In 2020, we endured the COVID-19 pandemic with the rest of the world, working closely with our sites and our members as the pandemic transformed clinical trial operations. With patient and staff access to research sites restricted, new methods for trial conduct were necessary. The positive result was a new set of practices that in many cases, may be here to stay. For example, virtual study screenings, eConsent, use of home health care and home drug delivery are now commonly used at I-ACT for Children sites. At the same time, we have adapted our own processes, including conducting virtual site visits to qualify new sites for our network. It was during 2020 that we implemented the first clinical trial in the network. This was a critical milestone – one that was hindered in part by pandemic-driven “pauses” on many studies. But as start-up capabilities for non-COVID-19 pediatric research are being restored both at our sites and within our members’ organizations, all signs point to 2021 being a busy year within the I- ACT for Children Site Network.

The initial three “start-up” years of the Institute have been formative, as the roadmap created by the Pediatric Trials Consortium met the proverbial road. With these learnings in mind, in 2020 we began a strategic planning process to define the next phase of the Institute’s growth and development. This work will shape and focus the plans for 2021 and the next three to five years as we move forward toward achieving the vision and mission of the organization.

Of course, none of our achievements this year occurred in a vacuum. Our growth – and our success – has been possible because of our partners and collaborators, our advocates and of course, our members. We would not be at this point without the unwavering advocacy and partnership of these researchers, study sponsors, regulators and most importantly, patients and their families.

We are still a very young organization, and we have only just begun to demonstrate the value we can provide. But the stage has been set. Proof of principle has been established. And our mission remains clear.

Thank you for your support and encouragement as we have grown from the spark of an idea into the organization we are today. Heading into 2021, we are more committed than ever to advancing innovative and efficient research to benefit all children with medical needs.

Laura Gordon
CEO

I-ACT For Children:

A Timeline of our Growth

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I-ACT For Children’s
2020 Fiscal Year Financial Activities IACT_Web_2020_AnnalReport_Assets_Graphs_Revenue

Membership Support

$4,100,000 (58%)

Government Grant

$1,208,019 (16%)

Program Services

$1,126,578 (16%)

Contributions

$695,054 (10%)

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Management & General

$3,252,543 (62%)

Grant Expenses

$1,211,199 (23%)

Program Expenses

$619,783 (12%)

Consortium

$151,240 (3%)

Net Assets:

Net Change

$2,336,239

Net at December 31, 2018

$3,783,942

Net at December 31, 2019

$6,120,181

Making an Impact

Key 2020 Achievements

Creating an Efficient, Sustainable Infrastructure

Advancing Innovation in Clinical Trial Design

Thought Leadership

A New Kind of Network

Creating an Efficient,
Sustainable Infrastructure

The I-ACT for Children Site Network was created with the belief that doing things the traditional way – creating a small group of sites from scratch every time a pediatric clinical trial was needed – wasted precious time, as children with medical needs waited for treatments that had been proven safe and effective for them. Months and even years were often lost in this process, as sites had to be identified, contract terms and budgets were negotiated for each study and IRB reviews took weeks or months to complete.

None of the stakeholders – sponsors, sites, regulators and certainly not patients and their families – believed that this “traditional” way was acceptable. When I-ACT was launched, our most urgent goal was to build a standing pediatric trial network that could be activated quickly and was designed for maximum speed and efficiency.

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In 2020, several critical factors came together to make the Institute’s Site Network a global, interoperable pediatric clinical trial network:

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Beyond our diverse geography, we are building efficiencies into the site network to reduce study start-up delays – which are a significant driver of overall study delays for many drug developers.

  • Working with both our sites and sponsors, we developed a Master Start-Up Cost Agreement template and tools to help sites improve their budget negotiation cycle time. We also are working with our members to put MCDAs and MCTAs in place with sites, establishing the core elements of confidentiality and contracting so that individual studies can be processed more smoothly and efficiently.
  •  We trained 40 of our sites for the January 2021 launch of our Pediatric Improvement Collaborative for Clinical Trials & Research (PICTR) initiative, a quality improvement program through which we will measure and continue to enhance the efficiency of our site network. We are proud to be partnering on this important initiative with Peter Margolis, MD, and his team at the James M. Anderson Center for Health Systems Excellence. In 2021, we will collect baseline measures from all participating sites and incorporate QI change packages within a select subset of “trailblazer” sites, so that we can make and measure improvements as rapidly as possible.
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These achievements – combined with the speed of our site network’s central IRB – reflect not just the urgency of our mission, but also the culture of our site network. 

“While many GCP training programs exist for PIs and research staff, they typically have little information specific to pediatric research. We worked with the Duke Clinical Research Institute to develop a pediatric-focused GCP training supplement for our site network that helps fill this gap – and is harmonized with training requirements from our biopharmaceutical member companies.”

I-ACT for Children As Convener

Advancing Innovation in Clinical Trial Design

Advancing methodologies for more efficient pediatric product development is a high priority for both regulators and sponsors. I-ACT for Children leveraged our strength in convening stakeholders in a pre-competitive environment to complete several important projects in 2020 that focus on innovative approaches to improving pediatric trial speed and efficiency.

Duchenne Platform Trial

Inclusion of Adolescents in Adult Trials

Prioritizing Pediatric Planning

Other Accomplishments of Note

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Duchenne Adaptive Platform Trial

More than 40 companies are actively involved in drug development for Duchenne Muscular Dystrophy and there are more than a dozen active clinical trials. But with a rare disease such as Duchenne, it is difficult to recruit and run large robust clinical trials. The Institute led an initiative to design an adaptive platform trial protocol for Duchenne in collaboration with Parent Project Muscular Dystrophy and the Critical Path Institute. Using this design, multiple potential drugs can be tested in parallel, using common placebo groups as appropriate. As individual drugs enter and exit the study pending success or futility, the trial can be modified. This avoids multiple trials competing for the same limited group of patients and creates multiple efficiencies, with the goal of accelerating much-needed Duchenne therapies.

The protocol was informed by a stakeholder meeting we convened in 2019 to seek input from patients and their families, regulators, researchers and drug developers. The protocol’s scientific leadership incorporated outcomes from the meeting into the development plan, and the next step is to meet with regulatory agencies to address key aspects of the proposed design before implementation.

Inclusion of Adolescents in Adult Trials

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The inclusion of adolescents in adult clinical trials as a way to accelerate access to safe and effective medicines for this older pediatric population is a focus of active discussion among researchers, drug developers and regulators in many countries. In the US, regulatory guidance has been issued in oncology and the approach has been used in areas such as HIV drug development. Stakeholders in Europe have published similar positions. The Institute convened a workshop in 2019 to address the scientific, ethical and operational challenges of such inclusion and developed consensus recommendations from that meeting, which included high-level participants from across the United States as well as Spain, England, Canada and Germany and included leading scientific researchers, FDA representatives, patient advocates and industry experts. The recommendations from this meeting were recently accepted for publication in the journal Therapeutics Innovation and Regulatory Science.

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“Although the volume and severity of COVID-19 illness in adults has been greater, we cannot afford scientific unknowns for children. Studying the infection in children now could lead to valuable information that helps us successfully treat COVID-19 and prevent its further spread.”

Gary Noel, MD

I-ACT for Children Chief Medical Officer

Prioritizing Pediatric Planning

Planning for pediatric studies as early as possible in the drug-development process is critical to achieving rapid, efficient start-up of those trials once there is suitable evidence in adults to proceed. This early planning is an important priority for regulators, developers and of course, for patients and their advocates. As the race began to develop treatments and vaccines for COVID-19, we publicly advocated for prioritizing the needs of children by publishing considerations in pediatric COVID-19 research in Nature Pediatric Research that called for urgency in understanding how SARS-CoV-2 behaves in children and outlined the key areas of pediatric research that required immediate action.

In addition, we hosted a Virtual Workshop with regulators, biopharma developers and experts on the front lines of treating children with COVID-19. The workshop participants shared the state of pediatric COVID-19 illness as well as innovative approaches to collecting the data required to advance safe and effective pediatric COVID-19 therapies. The workshop was co-moderated by I-ACT for Children Founder and Board Chair Edward Connor, MD, MBE, and Susan McCune, MD, Director of FDA’s Office of Pediatric Therapeutics and recommendations from the workshop have been published in Nature Pediatric Research.

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Promoting the Urgent Need

Thought Leadership

An important part of our mission is to serve as a voice for children by raising awareness of the unmet needs in pediatric research and advocating for the medical needs of children to be addressed as urgently as those of adults. In 2020, this advocacy included publishing manuscripts and presenting at prominent conferences.

icon-bullhorn-blue “Key Clinical Research Priorities for the Pediatric Community During the COVID-19 Pandemic”

We published “Key Clinical Research Priorities in the Pediatric Community During the COVID-19 Pandemic” in Nature Pediatric Research, calling for urgency in understanding how SARS-CoV-2 behaves in children and outlining the key areas of pediatric research that required immediate action.

“Pediatric Infectious Disease Journal”

We published a Commentary in the Pediatric Infectious Disease Journal about the important milestone represented by FDA’s inclusion of pediatric dosing in the Emergency Use Authorization for remdesivir in treating serious COVID-19 disease.

“Ensuring continued progress for development of COVID-19 therapeutics in children”

We published proceedings from our Workshop on COVID-19 Therapeutics Development in Pediatric Research.

Inclusion of Adolescents in Adult Trials

Proceedings from our Pediatric Research Innovation Forum on Inclusion of Adolescents in Adult Trials have been accepted for publication in Therapeutic Innovation and Regulatory Science.

International Neonatal Consortium

Our medical and scientific leadership including CMO, Dr. Gary Noel, and SVP for Clinical & Scientific Development, Collin Hovinga, presented models for Adaptive Platform Trials and advocated for innovative trial design/execution for studies of neonates and the use of RWE/RWD to inform these trials at the national meetings (International Neonatal Consortium, DIA, CTTI, FDA, International Congress of the European Society for Developmental, Perinatal and Paediatric Pharmacology).

Remote Patient Monitoring

Our site network team co-hosted with Elin Haf Davies, PhD, MSc and colleagues from Aparito, a Virtual Meeting on Remote Patient Monitoring along with collaborators in Canada and Connect for Children (C4C) in Europe dealing with innovations needed in the era of COVID-19.

Advancing Innovation Together

Our I-ACT for Children Members

We work closely with our biopharmaceutical members to help them plan and execute their pediatric trials of innovative medicines and to advance pre-competitive initiatives that address unmet pediatric research needs. Our Sustaining Members hold five-year memberships, while our Annual Members hold one-year memberships. Each of our members shares our commitment to accelerating pediatric research and ensuring that children have access to medicines that have been proven safe and effective for them.

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Sustaining Members

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Annual Members

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Our I-ACT for Children Leadership Team

I-ACT Board of Directors

Key External Collaborators (2020)

  • Scientific Review Committee: John Bradley, MD, Rady Children’s Hospital (Chair) and Adelaide Robb, MD, Children’s National Hospital
  • External Advisory Committee: Jonathan Davis, MD (Chair), Tufts Children’s Hospital and William Smoyer, MD, Nationwide Children’s Hospital and Ron Portman MD, Novartis (Coordinating Committee)
  • Best Practices, Education and Tools Committee: Grace Wentzel, CCRP, CHRC, Nationwide Children’s Hospital and Amanda Galster, MPH, CCRP, University of Minnesota (Co-Chairs)
  • FDA Grant Partners: Kolaleh Eskandanian, PhD, MBA, PMP (Device Development) and John van den Anker, MD, PhD (Pharmacology) Children’s National Hospital, Peter Margolis, MD, PhD, Cincinnati Children’s Hospital (QI and Learning Research Systems), Christopher B. Forrest, MD, PhD, Children’s Hospital of Philadelphia, (Real World Data), Klaus Romero, MD, MS, Critical Path Institute (Regulatory Science)
  • DMD Platform Trial Consortium: Jane Larkindale, DPhil, Critical Path Institute, Pat Furlong, Parent Project Muscular Dystrophy, Abby Bronson, MBA, Edgewise Therapeutics, Deborah Ascheim, MD, D2A Ltd , Scott Berry, PhD, Berry Consultants Richard Finkel, MD, St. Jude Children’s Research Hospital and Craig McDonald, MD, UC Davis Health (Scientific Co-Chairs)
  • International Leads: Mark Turner, BSc, PhD, MBChB, DRCOG MRCP(UK), FRCPCH, FFPM(Hon), University of Liverpool, c4c, Thierry Lacaze-Masmonteil, MD, PhD, Alberta Children’s Hospital Research Institute, MICYRN, Hidefumi Nakamura, MD, PhD, National Center for Child Health and Development (Tokyo, Japan)
  • Other Acknowledgments: We would like to recognize Pfizer for providing financial support during the launch of the Institute and Lucy Vereshchagina, PhD, PhRMA and Danielle Friend, PhD, Biotechnology Industry Organization for their support of I-ACT for Children programs.

Funding for some of the programs described in this annual report was made possible, in part, by the US Food and Drug Administration through grant (1 U18 FD 006297). Views expressed in written materials or publications or by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices or organization imply endorsement by the United States Government.