Helping Children with Skin Diseases through Collaborative Research

PeDRA ON THE HORIZON: WHERE ACHIEVEMENT MEETS VISION An Update Report – March 2016

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Why PeDRA

Where We Are as 2016 Begins

In just three years and as of November 2015, there are now 63 total projects in process or in development, and several early studies completed. With the support of the Society for Pediatric Dermatology, the National Institutes of Health, industry, and advocacy groups, PeDRA is literally changing the present and future of pedi-atric dermatology research.

PeDRA investigators are working collabo-ratively, studies are progressing and being completed, and investigators are engaged at unprecedented levels in pediatric dermatology research.

Who We Are

THERE ARE NOW 32 PeDRA MULTICENTER STUDIES SUBMITTED FOR IRB APPROVAL, 63 TOTAL PROJECTS IN PROCESS, AND SEVERAL EARLY PROJECTS COMPLETED.

It has been so inspiring for me to stand behind an organization that is not only putting research collaborators together but also working hard to help fund resulting studies. Not only does PeDRA have the vision for what needs to happen in pediatric research, but the Alliance also acts on its word to make the concept a reality.”

–Elena Pope, MD, MSc, Chair, PeDRA Grants Committee

diseases. With this reality, pediatric dermatology leaders recog-nized that investigations could not make meaningful progress without collaborative efforts.

Thus, PeDRA was conceived as a research network in 2012 and we haven’t looked back since. Now with nearly 230 investigators part-nering to conduct studies spanning several disease areas, PeDRA is beginning to positively impact the lives of children who live with difficult and life-altering dermatologic diseases and conditions.

The MissionPeDRA’s mission is to promote and facilitate high quality collaborative clinical, translational, educational and basic science research in pediatric dermatology. Along with this mission is the vision to create sustainable collaborative research networks to better understand, prevent, treat and cure dermatological diseases in children.

PeDRA RESEARCH STUDY GROUPSPeDRA has focused research and collaboration in several disease areas:

• Birthmarks (and Procedural)—includes vascular and pigmented lesions

• Inflammatory Skin Disease—includes subgroups: atopic dermatitis; psoriasis; connective tissue disease; hair, nail & autoimmune; acne and hidradenitis

• Genetic Skin Disease—includes subgroups: epidermolysis bullosa; disorders of cornification; basal cell carcinoma nevus syndrome

• Skin Tumors and Reactions to Cancer Therapies (STARC)

• Neonatal SkinStandardized treatment protocols and clinical guidelines are sorely lacking for pediatric skin disorders,

including some conditions that are truly life threatening. Yet recruiting children for studies is challenging and projects are often underpowered, especially with rare

PeDRA is a network that multiplies the power of individual researchers by linking them together in collaborative studies. It is a rapidly growing organization, run by leading

dermatologists in the United States and Canada who volunteer their time. These professionals also lead the Society for Pediatric Dermatology (SPD), which launched PeDRA with generous seed funds and other important start-up resources.

PeDRA streamlines research by making available a coalition of pediatric specialists—scientists and clinicians—poised to conduct clinical trials and other studies to better understand pediatric skin disease and potential solutions.

With the pooling of resources through this collaboration and the resulting exponential expansion of the patient base, PeDRA will bring us closer to new treatment opportunities and solutions for young patients.

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PeDRA EARLY INVESTIGATORS

PeDRA ANNUAL CONFERENCE

Established in 2013, the conference is the only meeting venue that specifically aims to foster multicenter studies in all major areas of pediatric dermatology. The meeting provides

the platform from which investigators learn about and join quality collaborative studies. These partnerships help individual researchers “gain their footing” and enable patients to access studies that otherwise would be difficult.

The Annual Conference sows the seeds for collaboration, bringing people together to share common interests and identify research gaps. The energy and enthusiasm for this is palpable when investigators come together annually at this conference. These are dedicated professionals who try to help young patients in an era when relatively few answers or solutions are available. Now, clinician/investigators are enjoying a renewed sense of empower-ment with the concept of PeDRA as a research alliance taking hold and becoming a reality.

Early on, leaders conceptualized PeDRA as a “legacy project,” enabling younger clinician researchers to

engage in collaborative research and invest in the future of pediatric derma-tology. This purpose has resonated with trainees/early investigators, who in 2015 made up half of the Annual Conference attendees.

PeDRA has made sure to incorporate early career perspectives and priorities in the planning process by forming the Early Investigator Committee (“EIC”). That group, comprised of early and mid career clinician researchers, has launched the PeDRA Mentorship Program, which pairs early investigators with more experienced ones in one-on-one mentoring relation-ships. Also, the EIC is instrumental in ensuring appropriate early career content at the Annual Conference.

PeDRA FUNDS FOR RESEARCH

Reflecting our core commitments, PeDRA awarded its first seed grants for internal research projects in 2015. There was a competitive application process, overseen by a

grant review committee comprised of experienced clinician investigators in a variety of pediatric dermatology sub-fields. Four grants totaling $18,900 were awarded focusing on: skin cancer in children and young adults; morphea “mapping” toward a better understanding of the disease; pediatric psoriasis comorbidity screening; and the predictive value of hemangioma for risk of PHACE syndrome.

These funds are in addition to external grants raised for PeDRA projects. For example, the International Psoriasis Council provided $150,000 for a project that brought together PeDRA and a group of European pediatric investigators for a compar-ative study of systemic interventions in pediatric psoriasis.

The National Psoriasis Foundation also partnered with PeDRA with a $30,000 grant to support the Pediatric Psoriasis Comorbidity Screening Initiative to develop guidelines for pediatric psoriasis screening procedures.

FOR 2016, PeDRA IS DEDICATING $60,000 FOR INTERNAL RESEARCH FUNDING, which again will be awarded through a competitive grant application process. New this year, PeDRA and SPD are working together to harmonize their respective grant programs and combine research funding with the goal of supporting the most impactful applications for the field.

These are dedicated professionals [in PeDRA]

who try to help young patients in an era when

relatively few answers or solutions are available. Now,

clinician/investigators are enjoying a renewed sense

of empowerment with the concept of PeDRA as a research alliance taking

hold and becoming a reality.

When it comes to helping children, the National Psoriasis Foundation’s (NPF) dedication to improving the lives of those affected with psoriatic disease takes on special meaning. PeDRA, a unique organization that helps children with psoriasis and other devastating diseases through research and collaboration, has strengthened the NPF’s impact in making a difference for kids. We are proud to support PeDRA and excited by the progress it’s made in these few short years since its inception toward bringing more answers for children.”

– Michael Siegel, PhD, Director of Research Programs National Psoriasis Foundation

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How PeDRA Operates

ADVOCACY FOR OUR PATIENTS

PeDRA’s infrastructure is comprised of an Executive Committee and Board of

Directors that drive the policies and priorities of the Alliance. There are also several working committees and ad hoc task forces that convene on an as needed basis—for example, an Ethics Task Force produced Ethics Guidelines for PeDRA. All this leadership is volunteer.

PeDRA’s bedrock is made up of these volunteer medical profes-sionals who care for pediatric patients, run clinical trials, conduct bench research, lead translational research projects, consult on drug development, start registries, publish data critical to advance the field—and in their spare time,

PEDRA EXECUTIVE COMMITTEE

CO-CHAIRSLawrence Eichenfield, MD University of California, San Diego Rady Children’s Hospital, San Diego

Amy Paller, MS, MD Northwestern University Ann & Robert H. Lurie Children’s Hospital of Chicago

EXECUTIVE COMMITTEE MEMBERS

Anna Bruckner, MD University of Colorado School of Medicine Children’s Hospital Colorado

Ilona Frieden, MD University of California, San Francisco Benioff Children’s Hospital

Moise Levy, MD Dell Children’s Medical Center, Dell Medical School, University of Texas at Austin Baylor College of Medicine

Kimberly Morel, MD Columbia University, Morgan Stanley Children’s Hospital of New York—Presbyterian

Dawn Siegel, MD Medical College of Wisconsin Children’s Hospital of Wisconsin

Jeffrey Sugarman, MD, PhD University of California, San Francisco

EARLY INVESTIGATOR LIAISON TO THE EXECUTIVE COMMITTEE

Lisa Arkin, MD, Rush University

EXECUTIVE DIRECTOR

Sheila Rittenberg

2015 PeDRA FELLOWS

Jennifer Day, MD

Pamela Gangar, MD

PeDRA has structured a powerful relationship with patient advocacy organizations that repre-sent the spectrum of disease areas under the

PeDRA umbrella. Representatives of these patient organizations attend and sponsor the PeDRA Annual Conference. There, they lead a special Advocacy Forum and also participate in disease-specific breakout sessions. In these ways, along with extensive networking opportunities, patient advocacy organi-zations have a meaningful role in shaping the PeDRA research agenda.

Engagement with advocacy organizations has brought the patient voice into research partnerships. This synergy has been rewarding for everyone involved and has spawned some important collaborations and contributions—for example, the Foundation for Ichthyosis and Related Skin Types (FIRST) provided $50,000 for an ichthyosis collaborative study; and the Basal Cell Carcinoma Nevus Syndrome (BCCNS) Life Support Network directed a portion of a significant private contribution to PeDRA.

PeDRA has also engaged with the FDA Pediatric Division in an ongoing dialogue to discuss patient

needs and patient safety as related to drug devel-opment. Specifically, PeDRA mounted a powerful advocacy position, making the case for children to be included in atopic dermatitis (AD) clinical trials. PeDRA mobilized a broad set of dermatology organizations including the American Academy of Dermatology, the Society for Investigative Dermatology, and the Society for Pediatric Dermatology to support this position, which was heard at a 2015 FDA hearing. PeDRA’s participation at the hearing, along with that of the American Academy of Pediatrics and the National Eczema Association, successfully argued for including children in appropriate AD trials. The FDA hearing subsequently took a favorable position in allowing children to join the trials.

Following that success, Dr. Elaine Siegfried launched an initiative to produce a Guidance Document on the conduct of pediatric atopic dermatitis trials, which will inform both industry and the FDA on the optimum parameters and requirements for these studies. PeDRA is a major financial sponsor of this effort and PeDRA leadership is intricately involved in the process of producing this consensus-driven Guidance Document.

run PeDRA. The Alliance has a unique culture, shaped not by organizational preoccupations but by real needs in the field that these devoted professionals bring to PeDRA’s work.

PeDRA has a part-time Executive Director and two PeDRA Fellows who lend some assistance with the administration of PeDRA. A fiscal management firm handles PeDRA’s accounting and budgeting processes.

The National Institutes of Health, the Society for Pediatric Dermatology, patient advocacy organizations, industry, and dermatologists and family foundations make up PeDRA’s funding base.

Conclusion

Despite the unparalleled scientific advancements in cutaneous biology and new therapeutics that are emerging, there has been a relative paucity of discovery related to pediatric skin disorders

and certainly few translations into new interventions. Importantly, PeDRA has brought together many talented and dedicated individuals who will collectively study rare and common pediatric disorders in a way never before pursued at a single site or even through a few collaborating centers. PeDRA is transforming the science of pediatric dermatology through this research collaboration that addresses the important gaps in our under-standing of skin disorders of childhood.

The Alliance has a unique culture, shaped not by organizational preoccupations but by real needs in the field that devoted

professionals bring to PeDRA’s work.

www.pedraresearch.orgPediatric Dermatology Research Alliance—PeDRA on Facebook.

PeDRA’S MISSION is to promote and facilitate high quality collaborative clinical, translational, educational and basic science research in pediatric dermatology. Along with this mission is the vision to create sustainable collaborative research networks to better understand, prevent, treat and cure dermatological diseases in children.

Please visit www.pedraresearch.org for more information on PeDRA.

Questions? Please contact PeDRA Executive Director: Sheila Rittenbergsheila.rittenberg@gmail.com