Providing children and families a voice in health, medicine, research and innovation February 2015

Background (UK Experience)

What is KIDS? Kids and Families Impacting Disease Through Science (KIDS) is an advisory group of children, adolescents and families focused on understanding, communicating and improving health, medicine, research, and innovation for children. KIDS is a collaboration between the American Academy of Pediatrics (AAP) Section on Advances in Therapeutics and Technology (SOATT), local AAP Chapters, children’s hospitals, local schools and other partners.

KIDS Objectives for medicine, research and innovation that improves the health and well-being of children Learn, teach, advocate in the process through projects and consultation activities with hospitals, researchers, and other partners in the public and private sectors Engage input on research ideas, innovative solutions, unmet pediatric needs and priorities Provide to the design and implementation of clinical studies for children (e.g., assent, monitoring tools, schedules, etc.) Contribute as a critical voice for children and families in the medical, research, and innovation and processes Serve

KIDS Connecticut Pilot  Established first KIDS Team in Connecticut  Broad collaboration  AAP Section on Advances in Therapeutics and Technology  Connecticut Chapter of AAP  Connecticut Children’s Medical Center  Yale-New Haven Children’s Hospital  Pfizer Inc  Local schools  Serve as model for future KIDS Teams

KIDS Speak… I want to help…  “I want to help kids have more effective treatments and experiences”  “We would be doing stuff that really could help, not just talking about it”  “I have always wanted to play a role in impacting the medical system I just never knew how”  “I want to help heal kids in the best way possible”  “I just want to help children with medical conditions and give input on how to make their medical experience better” I want to learn…  “I would like to learn more about research in children”  “I am particularly excited to learn more about unmet pediatric needs”  “I don’t know understand very much about our medical system right now. I want to join to become an educated person and see if I can make a difference”

KIDS Speak… My opinion matters…  “I know what it’s like to be a kid”  “I’ve had pectus carinatum and know what it feels like”  “Adults will be in need of a child’s perspective for different medical circumstances that involve children”  “Since I’m a kid, I will be able to relate and connect to other kids better than an adult could”  “I’m particularly interested in contributing to the design and implementation of clinical studies for children”  “I would like to voice my opinion and engage in projects that will help children in need of innovation”

Empowering Children Worldwide iCAN is a worldwide consortium of KIDS Teams, Young Person’s Advisory Groups (YPAGs), and similar organizations working together to provide a voice for children and families in pediatric medicine through synergy, communication, and collaboration. International Children’s Advisory Network (iCAN) KIDS and Families Impacting Disease Through Science (KIDS) KIDS is an advisory group of children, adolescents and families focused on understanding, communicating and improving health, medicine, research, and innovation for children. KIDS is a collaboration between the American Academy of Pediatrics (AAP) Section on Advances in Therapeutics and Technology (SOATT), local AAP Chapters, children’s hospitals, local schools and other partners.

iCAN Objectives Connect With similar groups around the world Share Ideas, best practices, challenges, culture Learn From one another and capitalize on expertise Collaborate For research, innovation, children and families Advocate The health and well- being of children around the world

International Children’s Advisory Network  United States  KIDS Current (5) Connecticut Kansas City Utah Ohio Georgia  KIDS In Development (7) Illinois Indiana Miami Michigan New Jersey Texas Washington, DC  International  Current (7) KidsCan (Vancouver) Young Person’s Advisory Groups (UK) Birmingham Bristol Liverpool London Nottingham Scotland  In development (5) Australia (Sydney) Germany (Berlin) Italy (Rome) France (Lyon) Spain (Barcelona)

Sample Individual Team Projects  KIDS Connecticut  Pfizer R&D Summit  University of Connecticut Undergraduate Research Consult  ScotCRN Young Persons Group  British Science Festival, Focus Group  KidsCan  MonsterManor Mobile App  CRN Young Person’s Advisory Group  GenerationR Conference

iCAN Collaborations  Pediatric assent review for industry-sponsored study  iCAN reviewed and delivered feedback from children and families in the U.S., Canada, and the United Kingdom with very short turnaround time  Global Alliance for Pediatric Therapeutics Assent Project  Project evaluating current practices, challenges and unmet needs in attaining pediatric assent for clinical trials  iCAN collected ~50 survey responses from children and families in the U.S., Canada, and the United Kingdom  Plan is for additional iCAN involvement as project evolves  SPIRIT-C/CONSORT-C  Early product development feedback request  Plan to engage full network later in 2014 as requested by innovator  And we have just begun

KIDS Online & On The Road  AAP Healthy Children Expo  March 8-9, Chicago, IL  CHES Rounds/Toronto SickKids  April 21, Toronto, ON  Real World Health Care Invited Blog  April 23, /2014/04/kids/  Pediatric Academic Societies  May 3-6, Vancouver, BC  Children’s Brain Tumor Foundation Gala  May 28, New York, NY  Institute for Pediatric Innovation Annual Meeting  June 19, Cambridge, MA  European Network on Pediatric Research at the EMA (Enpr-EMA) Annual Workshop  June 26, London, England  SPIRIT-C/CONSORT-C Consensus Mtg  September 15-16, Toronto, Canada

KIDS Online & On The Road  KIDS Connecticut/UConn Undergraduate Research Program  September 19, Storrs, CT  NCDChild Panel  September 22, New York City  UNICEF ACTIVATE Talk  September 23, New York City  aking-young-voices-ncds/ aking-young-voices-ncds/  Clinical Trial Day  October 2, Rome, Italy  Sickle Cell Disease Association of America’s Annual Convention Oral Presentation  October 3, Baltimore, MD  American Academy of Pediatrics National Conference and Exhibition  October 10-14, San Diego, CA  “Assent in Pediatric Clinical Trials: An International Children’s Advisory Network Survey”  March 2015, Platform Presentation, ESPR, Philadelphia, PA  April 2015, Poster Presentation, PAS, San Diego, CA

KIDS On The Road (AAP Expo, Chicago)

KIDS On The Road (PAS, Vancouver)

KIDS On The Road (IPI, Cambridge)

Spirit-C/CONSORT-C (Toronto, Canada)

UConn Undergrad Research Program

NCDChild Event (New York City)

UNICEF ACTIVATE Talks (New York City)

Better Medicines for Children Conference (London, England)

Clinical Trial Day (Rome, Italy)

Sickle Cell Disease Association Oral Presentation (Baltimore, MD)

KIDS Georgia Kick-Off (December 2014)

Feedback On KIDS/iCAN  “I think this is a great way to help us to create more operationally feasible trials. Getting the perspectives of children will help us to pay special attention to details prior to startup. I think having a standard ‘KIDS advisory panel’ prior to the kickoff of any study would be good practice and would help with the implementation of our studies.” ~Development Operations Specialist Pharmaceutical Industry  “As a children’s organization, UNICEF always strives to bring children’s voices to the work we do, and an organization like iCAN Research is very meaningful to us because it provides children’s opinion to the work that matters most to them. We look forward to many more great projects by iCAN Research and hope to continue the conversation for the well-being of the children around the world.” ~Hayon Nam Programme Officer UNICEF

Feedback On KIDS/iCAN  “Many thanks on behalf of NCD Child and UNICEF, and also the American Academy of Pediatrics, the International Pediatric Association, and the Healthy Caribbean Coalition for your participation in our side event, ‘Next steps in NCDs for children and youth: UNICEF Facts for Life and life-course priorities for the Post-2015 Development Agenda,’ on Monday, September 22, We were very pleased with both the diversity of audience and lively discussion generated by our panelists. As you know, NCD Child actively seeks the input of youth voices in all of its activities to ensure children and adolescents are prominently recognized on the global NCD agenda. The work of iCAN and KIDS Connecticut is impressive, and we are very fortunate to work with your network.” ~Jonathon Klein, MD FAAP Executive Director, NCD Child Associate Executive Director, American Academy of Pediatrics

iCAN Launch & Research Summit  Date & Location  June 22-26, 2015  Washington, DC  Objectives  Provide children and families with world- class educational opportunity  Provide researchers and regulators with mechanism to hear important feedback/advice from children on pediatric issues/projects  Provide legislators/staffers with chance to hear from informed children about importance of pediatric research and innovation  Provide an interactive and collaborative forum for stakeholder groups and young advisors to network with one another and develop synergies with the growing iCAN network  Partners  American Academy of Pediatrics/NCD Child  Biopharmaceutical companies  Capitol Hill  Children’s National Medical Center  Contract Research Organizations  European Medicines Agency  Food and Drug Administration  National Institutes of Health  National Organization for Rare Disorders  Nuffield Center for Bioethics  PhRMA  Others  Attendees  leaders, youth advisors and parents from around the world  Colleagues from partner organizations

iCAN Launch Draft Agenda Monday (June 22) 8:00AM-6:00PMArrivals in Washington, DC 6:00PM-9:00PM Special Event #1 (Opening Ceremony) Dinner & Keynote Speakers (Lindsey Elsaesser) Teambuilding Event Tuesday (June 23) 8:30AMDepart Hotel for Meeting Venue 9:00AM-9:30AMWelcome, Introductions, Overview 9:30AM-10:00AMIcebreaker/Networking Game 10:00AM-10:15AMBreak 10:15AM-11:30AMA Journey Around iCAN (poster session) 11:30AM-12:00PMiCAN Working Together 12:00PM-1:00PMLunch 1:00PM-1:30PMiCAN Working Together, continued 1:30PM-3:00PM Long Road to Drug Discovery Game (Terri Swanson & team) 3:00PM – 3:15PMReturn to Hotel 3:15PM-5:30PMFree time 5:30PMDepart Hotel for Special Event #2

iCAN Launch Draft Agenda Wednesday (June 24) 7:30AMDepart Hotel for Children’s National Medical Center 8:00AM-10:00AMChildren’s National Medical Center 10:00AM-10:15AMBreak 10:15AM-11:45AM Regulatory Science (Food and Drug Administration, European Medicines Agency, Health Canada) 11:45AM-12:15PMTravel to Meeting Venue 12:15PM-1:00PMLunch 1:00PM-2:15PMNational Institutes of Health 2:15PM-2:30PMBreak 2:30PM-3:00PMNuffield Center for Biomedical Ethics 3:00PM-4:30PMHow A Bill Becomes Law (Lisa Kaeser) 4:30PMReturn to Hyatt Place 4:45PM-8:00PMFree time & Dinner On Own Thursday (June 25) 7:30AMDepart for Meeting Venue 8:00AM-9:00AMNational Organization for Rare Disorders 9:00AM-10:00AMAAP NCD Child 10:00AM-10:15AMBreak 10:30AM-11:30PMAAP Federal Affairs 11:30-12:00PMTravel to PhRMA Stakeholder Briefing 12:00PM-1:00PMPhRMA Stakeholder Briefing/Lunch (90 minutes) 1:00PM-1:30PMTravel to Capitol Hill Visitor Center 1:30PM-4:30PM Capitol Hill Overview, Tour, Activities (Visitor Center, House/Senate Floor, Committee Rooms) 4:30PM-5:00PMTravel to Hyatt Place 5:00PM-6:00PMFree time 6:00PM-9:00PMSpecial Event #4 (Closing Ceremony)

ICAN

Questions?

Please contact Hadleigh Thompson Development Intern, iCAN For More Information