Date: 12/01/2022

Holly Neale MD,Suz Schrandt JD,Becky M. Abbott MPH,Jennifer Austin,Callista Brand,Karen Camp MPA,Katherine Devenport,Michelle Hall,Lauren Isbell,Charlene Miciano,Sarah Pry,Paula Quinn,James Rittle,Francesca Tenconi MBA,Julia Terrell,Meredith Weiss,Sam Zavitz,Michael Siegel PhD

PMID: 36443263 DOI: 10.1111/pde.15199

Abstract

Background/Objectives

Patient and caregiver perspectives are critical in understanding dermatologic disease impact, presentation, and management in children. The Pediatric Dermatology Research Alliance (PeDRA) Patient Advisory Committee (PtAC), a group of patient representatives and parents of children with cutaneous disease, pursued a multistep, iterative, consensus-building process to identify comprehensive, high-priority research needs.

Methods

Building on discussions at the 2020 PeDRA Annual Conference, a research prioritization survey was developed and completed by PtAC members. Survey themes were aggregated and workshopped by the PtAC through a series of facilitated calls. Emerging priorities were refined in collaboration with additional PeDRA patient community members at the 2021 PeDRA Annual Conference. Subsequently, a final actionable list was agreed upon.

Results

Fourteen PtAC members (86.7% female) representing patients with alopecia areata, atopic dermatitis, vascular birthmarks, congenital melanocytic nevi, ectodermal dysplasias, epidermolysis bullosa, Gorlin syndrome, hidradenitis suppurativa, ichthyosis, pemphigus, psoriasis, Sturge–Weber syndrome, and pachyonychia congenita completed the survey. Following serial PtAC meetings, 60 research needs were identified from five domains: psychosocial challenges, health care navigation/disease management, causes/triggers, treatments to preserve or save life, and treatments to preserve or save quality of life.

Conclusions

Many pediatric dermatology research priorities align across affected communities and may drive meaningful, patient-centric initiatives and investigations.

1 INTRODUCTION

Patient and caregiver perspectives are important drivers for research and management of childhood skin disease. Eliciting insights from affected populations helps providers understand and honor patient preferences when co-creating treatment plans for children with cutaneous diseases such as atopic dermatitis, acne, psoriasis, and skin cancer.^1-3 Unique patient perceptions have led to the development of validated quality of life measurement tools, core outcome sets, and patient-centered approaches to counseling discussions in clinics.^4-6 Patient and caregiver voices also help fill knowledge gaps regarding the cutaneous clinical presentations and natural histories of rare syndromes.^2, 7 Efforts to highlight patient viewpoints in the academic pediatric dermatology community are ongoing and may drive further investigational focuses.

As patients are key players in achieving meaningful research efforts, it is important to involve representatives from impacted disease communities when prioritizing research questions. Identification of universal research needs is challenging because each disease brings distinct burdens, diversity of treatments, variable presentations, and differing impacts on quality of life. Consensus priorities for translational dermatologic research in the United Kingdom were proposed in 2015 through clinicians and patient support group representatives, identifying 97 priority research questions.⁸ Disease-specific research priorities, such as those for atopic dermatitis, psoriasis, and alopecia areata, have since been separately proposed by involving key disease stakeholders.^9-11 Importantly, children are a unique subset of dermatology patients, and there currently is no harmonized set of domains defining which pediatric dermatology research may be the most impactful for those affected.

In 2019, the Pediatric Dermatology Research Alliance (PeDRA) developed a Patient Advisory Committee (PtAC) as a forum for engagement between parents and patient representatives with cutaneous disease.¹² As of 2022, the committee consists of 15 representatives across 14 diverse dermatologic diseases including epidermolysis bullosa, facial vascular birthmarks, psoriasis, hidradenitis suppurativa, pemphigus, Gorlin syndrome, pachyonychia congenita, atopic dermatitis, Sturge–Weber syndrome, ichthyosis, alopecia areata, PHACE syndrome, congenital melanocytic nevi, and ectodermal dysplasias. Importantly, all representatives bring lived experience, with some participants also having population-level experience by virtue of leading (or involvement in) disease-focused patient groups. By closely involving patient representatives and caregivers in a pediatric dermatology research organization, affected communities have exposure to the latest relevant findings and close influence on emerging studies.

Here, input from patient representatives on the PeDRA-PtAC was gathered to organize a consensus-driven set of topics relevant to improving the lives of children with dermatologic diseases. Potential research questions and actionable items were proposed within each topic for future research and advocacy endeavors. Understanding such priorities from the patient and caregiver perspective is a pivotal step to advance the field of pediatric dermatology.

2 METHODS

This project received an exemption determination from the Genetic Alliance institutional review board. A consensus-style approach used combined survey and serial meetings under the direction of an experienced facilitator (Figure 1). Following an introduction of the concept at the 2019 PeDRA Annual Conference, PeDRA formed the PtAC by working with leaders from patient advocacy organizations and individual patient stakeholders with connections to PeDRA to individually recruit patient representatives with a strong personal connection and reliable channels of communication to a disease community. PtAC recruitment prioritized individual perspectives, inter- and intra-community communication capacity, and familiarity with PeDRA and pediatric dermatology research. The group included 15 members at the time of this project. In October 2020, PtAC members broadly discussed research needs during a breakout session at the PeDRA Annual Conference. In June of 2021, the PtAC reconvened to identify the most relevant topics to investigate further in a survey. Based on this meeting, a survey was drafted and revised by members of PtAC. Ultimately, 22 items were included in 5 domains: (1) demographic and disease or condition characteristic factors, (2) understanding physical impact of disease or condition, (3) understanding psychosocial impact of disease or condition, (4) disease or condition management and quality of life, and (5) research and project types. The survey was fielded to all 15 representatives in the PeDRA-PtAC from August to September 2021.

FIGURE 1

Key meetings from October 2020 to January 2022 for the Pediatric Dermatology Research Alliance (PeDRA) Patient Advisory Committee (PtAC) to develop a prioritized agenda for pediatric dermatology research needs.

Survey results (93.3% response rate) were aggregated and assembled before sharing data with the PtAC in a preliminary review discussion; results from that discussion and subsequent email dialogue resulted in a second iteration of prioritized research foci. This second iteration was further workshopped at the virtual 2021 PeDRA Annual Conference using World Café style idea generation breakout sessions,¹³ in which another subset of PeDRA patient community members (patients and parents registered for the 2021 conference who voluntarily chose to attend the patient-specific breakout session, two of whom were also members of the PtAC) were present. This included a mixture of organizational leaders and patients from the vascular birthmarks, eczema, ichthyosis, nevus, PHACE syndrome, Sturge–Weber syndrome, and vitiligo communities. No clinician or professional scientist was present. Here, addressing emerging themes and challenges identified, members were divided into small groups and participated in an exercise to organize broad research and intervention ideas by complexity and estimated time to accomplish. Actionable items were narrowed down through this exercise and presented back to the PtAC. In a final round of facilitated brainstorming, the PtAC refined needs relevant across diverse disease states, versus needs within a more limited disease-specific limited stratum, adjusting for relative weight. No research needs were completely excluded. A finalized agenda was agreed upon by the PtAC in early 2022.

3 RESULTS

Fourteen survey respondents (86.7% female) represented patients with the following conditions: alopecia areata, atopic dermatitis (2), vascular birthmarks, congenital melanocytic nevi, ectodermal dysplasias, epidermolysis bullosa, Gorlin syndrome, hidradenitis suppurativa, ichthyosis, pachyonychia congenita, pemphigus, psoriasis, and Sturge–Weber syndrome. These respondents identified as the parent (without dermatologic disease) of a child with a pediatric dermatologic condition (33.3%), affected patient with history of pediatric dermatologic disease without affected children (33.3%), or a patient with history of pediatric dermatologic disease with affected offspring (26.7%). The composition of patient and family advocates who participated in the results synthesis sessions shared a similar diversity of disease experiences.

The two domains under which the greatest number of research needs were classified were “psychosocial challenges” (n = 16) and “health care navigation/disease management” (n = 19). The next three domains were “causes/triggers” (n = 4), “treatments to preserve or save life” (n = 11), and “treatments to preserve or save quality of life” (n = 10). For each of the five primary domains, common themes were identified (Table 1). A total of 60 research questions and needs were identified and grouped by research type (Table 2).

TABLE 1. Major domains and themes identified for patient-centered research priorities in pediatric dermatology

• ^a See Table 2 for research needs identified.

TABLE 2. Patient-centered research priorities

• Abbreviations: B, bench; C, comparative effectiveness research; CT, causes and triggers; D, descriptive; H, health care systems; I, innovation; PC, psychosocial challenges; TL, treatments to prolong life; TS, treatment of symptoms.

4 DISCUSSION

Presented are the first comprehensive sets of pediatric dermatology research priorities derived from a patient and caregiver perspective, established by a unique group of stakeholders from different disease areas who share a connection to collaborative pediatric dermatology research and each other through the PeDRA-PtAC. The domains (based on cross-disease importance and commonality) this group arrived at were psychosocial challenges, navigation of the health care system/disease management, causes and triggers of disease, treatments to prolong life, and treatments to address symptoms. The 60 proposed items highlight patient-perceived gaps in patient-perceived existing research, clinical, and support efforts, and reveal common concerns of cancer, pain, itch, and visibility among other things. This list should serve as a useful reference for stakeholders while identifying meaningful pediatric dermatology research initiatives, and also be considered by funders, patient advocates, clinicians, and policy leaders to have high impact on affected pediatric communities.

Over half of the identified research needs (35 of 60) fell into domains of “psychosocial challenges” and “health care navigation and disease management,” making these domains highly prioritized. Psychosocial challenges are a critical cross-disease focus. Negative effects on quality of life, self-esteem, and interpersonal relationships for both patients and caregivers are a consequence of many childhood skin conditions across all ages.¹⁴ Atopic dermatitis, psoriasis, acne, and hidradenitis suppurativa are examples of the many pediatric dermatologic conditions associated with stigma, bullying, impaired relationships with family members, psychiatric impact, and suicidal behavior.^14, 15 The patient priorities here highlight an emerging theme within the psychosocial challenges domain: a need to empower patients in a supportive environment. Support groups and programs have positive effects on children with skin disease and family members,^16, 17 an area identified by the PeDRA-PtAC for needed continual focuses in order to identify best support strategies to improve existing programs and develop new strategies.

The second predominant domain, “health care navigation and disease management,” represents a common challenge for patients and caregivers. Existing barriers in pediatric dermatologic care include financial obstacles (treatment cost, selective insurance acceptance), geographic location, and prolonged wait times.^18, 19 Socioeconomic disparity additionally limits access to pediatric dermatologic appointments for minority patients.²⁰ Thus, the PeDRA-PtAC agenda includes items to compare health plans, create patient insurance education, improve access to providers, and coordinate care. In addition, conditions seen by pediatric dermatologists may affect multiple organ systems (i.e., epidermolysis bullosa, Gorlin syndrome, neurocutaneous syndromes) requiring multidisciplinary care.^21-23 The presented priorities encompass identification of effective interdisciplinary and multidisciplinary models, as well as innovations to help patients navigate this complex process.

The remaining three domains surrounding causes/triggers and treatments provide a patient-centered direction for further disease-specific research efforts. The priorities for “causes and triggers of cutaneous disease” highlight bench and innovation style research to increase understanding of the role of genetics, the microbiome, and the environment. For treatment domains, life-saving/prolonging treatments are largely applicable to higher mortality conditions (i.e., skin cancer), whereas the quality of life-preserving/improving treatment domain priorities apply across many conditions. Patient-centered methods in developing dermatologic management approaches have been previously successful, such as those for biologic decision making in psoriasis.²⁴ Future treatment investigations with a similar patient-centric approach may be broadly useful in pediatric dermatology.

While the community-level knowledge of disease experience among individual participants contributed to a broad corpus of findings, limitations for the implemented approach exist. Representatives were selected based on a collaborative approach between PeDRA leadership and stakeholders with existing connections to PeDRA (based on factors such as organizational history and disease-specific opportunity to participate in collaborative pediatric dermatology research). Openly acknowledging the impracticality of efficiently and effectively representing multiple patient populations, each composed of thousands to millions of individuals, the unique approach led to a small consensus group size, lack of comprehensive representation of all childhood skin diseases, and subjective assessments. As a result, not all items are applicable to every pediatric dermatologic disease, and the priorities are intended to serve as a flexible guiding tool that may inform future large-scale consensus initiatives, patient surveys, and focused research. In addition, as part of a semi-informal qualitative consensus process, the survey technique relied on the expertise of an experienced facilitator and no validated thematic analyses were performed. Individuals who contributed to the testing and development of the survey were the same participants completing the survey. While this approach provides the advantage of iterative steps that led to the inclusion of relevant items in a diverse group of stakeholders, bias in the form of an “echo chamber” may occur in which pre-existing beliefs are reinforced.²⁵ The anonymity of the survey, allowance to free-type additional issues, and incorporation of outside community members who did not develop or complete the survey at a concluding discussion session balance this limitation.

Generalizability challenges arose during the consensus process for unique needs between and across specific diseases, such as the variability of treatment approaches for diseases that require special care and wide diversity of symptomology severity for affected children. The group acknowledged that while some research needs are paramount within a given disease population, they are necessarily unique and narrowly applicable. Conversely, other research needs span disease populations and would equally benefit all patient communities. Both cross-disease and disease-specific priorities were included here to allow for patients, clinicians, and researchers to proceed with helpful initiatives.

Research priorities developed here should be interpreted with both methodologic strengths and flaws considered. The PeDRA-PtAC items identified provide a unique and actionable foundation for impactful patient-centric research in the context of a collaborative pediatric dermatology research network. Expanded efforts with attention to a systemic method of selecting stakeholders, engaging a larger group in a longer list of patient populations, in addition to using more formal methodologic thematic selection, may improve validity of research needs and provide valuable supplements to this work.

FUNDING INFORMATION

This project was partially funded through a Patient-Centered Outcomes Research Institute® (PCORI®) Eugene Washington PCORI Engagement Award (16347-PeDRA). The statements presented in this publication are solely the responsibility of the author(s) and do not necessarily represent the views of the Patient-Centered Outcomes Research Institute® (PCORI®), its Board of Governors or Methodology Committee.

CONFLICT OF INTEREST

Michael Siegel, PhD, and Katherine Devenport are employed by the Pediatric Dermatology Research Alliance. Holly Neale, MD served for 1 year as the Pediatric Dermatology Research Alliance Fellow. Jennifer Austin is employed by the International Alliance of Dermatology Patient Organizations.

© 2022 The Authors. Pediatric Dermatology published by Wiley Periodicals LLC.

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Grant support

PCORI/Patient-Centered Outcomes Research Institute/United States

Full Text Sources

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