Abstract
At the 2018 annual meeting of the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA), the International Dermatology Outcome Measures (IDEOM) Psoriasis Working Group presented an overview of its efforts to enhance clinical care and research in both the clinical setting as well as in clinical trials for psoriasis. First, the group discussed the results of a Delphi survey conducted in collaboration with the American Academy of Dermatology to agree on a unique physician-reported global assessment to measure the quality of care delivered to patients with psoriasis and other chronic inflammatory dermatoses. Second, the group summarized its efforts to select outcome measures for “PsA symptoms” and “treatment satisfaction,” 2 of the domains of the psoriasis core domain set that were established by IDEOM. Finally, the Psoriasis and Psoriatic Arthritis Clinics Multicenter Advancement Network (PPACMAN) presented an update on its clinical, educational, and research missions to foster the development of combined clinics for psoriatic disease, increase disease awareness, and accelerate management.
The International Dermatology Outcomes Measures (IDEOM) group is a nonprofit organization that was founded in 2013 with this mission statement: “[To] establish patient-centered measurements to enhance research and treatment for those with dermatologic disease”1. Following the Outcome Measures in Rheumatology (OMERACT) model2, IDEOM has involved patients, health economists, payers, nonprofit organizations, physicians, and regulatory agencies in multiple projects to advance research on outcome measures for psoriasis, psoriatic arthritis (PsA), hidradenitis suppurativa, alopecia areata, and acne in both clinical practice and clinical trials.
At the 2018 Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) annual meeting in Toronto, Ontario, Canada, IDEOM presented an update on its work. Initially, Dr. Alice Gottlieb presented IDEOM’s efforts for clinical practice. Together with the American Academy of Dermatology (AAD), IDEOM conducted a Delphi survey to agree on a unique physician-assessed global tool to measure the value of care across severe chronic inflammatory dermatoses3. Results from this study were then discussed among a larger group at the IDEOM 2018 Annual Meeting.
Next, IDEOM presented an update on the development of the core outcome set (COS) for psoriasis clinical trials. A COS is the minimum set of outcomes that should be measured and reported in all clinical trials for a given disease state4. To that end, IDEOM first defined a core domain set for psoriasis clinical trials (i.e., the minimum set of relevant domains and subdomains that should be measured), which includes the following domains: skin manifestations [primary: body surface area (BSA)/erythema/induration/scale], location of skin lesions (palmar-plantar and scalp psoriasis), investigator global, psoriasis and PsA symptoms, patient’s global, treatment satisfaction, and health-related quality of life5. To select or develop the most appropriate outcome measures to measure these domains, IDEOM has established domain-specific workgroups. At the meeting, Dr. Lourdes Perez-Chada and Dr. April W. Armstrong presented the results of the PsA Symptoms and Treatment Satisfaction Workgroups, respectively.
IDEOM-AAD Collaboration for Clinical Practice
To date, dermatologists need to prove the value of care they provide to their patients, policy makers, payers, and patients6. Indeed, the quality of care delivered by dermatologists to their patients plays a critical role in payment. For example, quality represents 50% of the score of the Merit-based Incentive Payment System7. Unlike other specialties, feasible, standardized, and valid outcome measures that reflect quality of healthcare (e.g., hemoglobin A1c for diabetes) are not readily available in dermatology. Thus, there is a need to achieve consensus on feasible disease severity outcome measures that can be incorporated into a quality measure to improve patient outcomes6.
Quality measures are measures that assess how a given practice is doing in the management of different areas of patient treatment and care8. Quality measures can provide critical data for continuous quality improvement and payment, foster patient empowerment, and support shared decision making. Most quality measures are constructed as proportions or percentages and can use a provider-reported score or a patient-reported outcome measure to calculate the quality of care provided9.
The denominator of the proportion consists of the number of patients treated by a provider during a defined period of time who were at risk of, or eligible for, the numerator event (e.g., patients with psoriasis receiving systemic therapy). The numerator consists of the number of patients in the denominator who received the appropriate treatment or diagnostic test, or the number who present an adverse outcome (e.g., patients with psoriasis achieving a BSA < 3%)9.
At the 2018 annual GRAPPA meeting, Dr. Gottlieb presented IDEOM and AAD’s collaborative efforts to reach consensus on a valid and feasible provider-reported global disease severity measure that can be used across many inflammatory skin diseases. Toward this end, the IDEOM/AAD collaboration initially conducted a literature review of current provider-reported global disease severity metrics used in inflammatory dermatoses3. Then, the data collected from identified instruments informed a 2-round Delphi survey that was conducted at a meeting held in New York in February 2018. Through an anonymous automated response system, participants were invited to vote on (1) which inflammatory dermatoses should be assessed by a global measure; and (2) which scale type would be more appropriate to quantify global disease severity [i.e., a 5-point Likert scale with or without descriptors, a numeric rating scale (NRS), or a dichotomous clear/almost clear vs not outcome]. Consensus was defined by prespecified endpoints for each question3.
Thirty-six IDEOM and AAD members attended the meeting, including dermatologists, rheumatologist-dermatologists, pediatric dermatologists, quality measures experts, AAD members, 1 patient, and a research fellow. Among 23 inflammatory skin diseases discussed, psoriasis, atopic dermatitis, and acne were selected as the dermatoses to be assessed by a global measure. Regarding the quantification methods, consensus was reached on a 5-point ordinal scale (clear = 0, almost clear = 1, mild = 2, moderate = 3, severe = 4) with disease-specific descriptors provided through referenced electronic platforms (e.g., AAD Website, phone applications)3.
The results of the Delphi survey were then presented and discussed at the May 2018 IDEOM Annual Meeting in Washington, DC, where a larger group (n = 86) was involved. Attendees included healthcare providers (HCP), industry partners, patients, and patient-association representatives. At the IDEOM Annual Meeting, participants agreed that treatment response should be evaluated by both the provider (94%) and the patient (88%) and recorded in every patient chart. In addition, participants agreed that these assessments should not be combined into a composite score (82%). Similarly, participants agreed that, in treat-to-target strategies, the goal should be to maximize provider-reported and patient-reported outcome measures. Finally, attendees responded that payers should not make decisions to cover treatment costs (96%) and reimbursements (82%) based only upon a patient-reported instrument.
To reach consensus on a patient-reported instrument to be incorporated into a quality measure, IDEOM and AAD held a meeting in October 2018 that included HCP and patients with acne, psoriasis, and atopic dermatitis. The results of this meeting are expected to be presented during the 2019 annual GRAPPA meeting in Paris, France.
PsA Symptoms in Psoriasis Trials Delphi Update
PsA is a chronic, progressive, and potentially disabling disease that occurs in about 30% of patients with psoriasis10,11. Despite this, PsA is rarely assessed in psoriasis clinical trials. During the process of establishing the core domain set for psoriasis clinical trials, “PsA signs” and “PsA symptoms” were considered as candidate domains. Because “PsA signs” entails the assessment of enthesitis, dactylitis, and arthritis by a trained physician, and because dermatologists tend to feel uncomfortable conducting these examinations, “PsA signs” was not included in the core domain set. Conversely, “PsA symptoms,” a patient-reported domain, was selected as a core domain5.
PsA symptoms is a complex domain that encompasses the subdomains of pain, physical function, and patient global12. To select the most appropriate instrument to measure this domain, IDEOM established a PsA Symptoms Workgroup, which included Dr. Gottlieb, Dr. Joseph F. Merola, Dr. Alexis Ogdie, Dr. Jeffrey Cohen, and Dr. Perez-Chada. At the 2018 GRAPPA annual meeting, Dr. Perez-Chada presented an update on the PsA Symptoms Workgroup’s work.
The goals of the workgroup were to (1) define whether patients should be screened for PsA prior to the measurement of PsA symptoms in psoriasis clinical trials; and (2) reach consensus on what is the most appropriate instrument to measure PsA symptoms in this context. The overall process consisted of 4 steps following the guidelines published by the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN), Core Outcome Measures in Effectiveness Trials13, and the OMERACT filter 2.114: (1) a 2-stage literature review; (2) a pre-Delphi exercise that modeled the content of the Delphi survey upon the input of various participants; (3) an international Delphi survey; and (4) a consensus meeting.
The literature review was organized into 2 stages. First, the group sought to identify available PsA screening tools and evidence on their performance as well as measures for PsA symptoms (i.e., pain, physical function, and patient’s global). Then, the group systematically searched for studies on the measurement properties of candidate PsA symptoms measures. Data collected from this search were used to create a preliminary draft of the Delphi survey, which was tested during the 2017 IDEOM Annual Meeting12.
During the 2017 IDEOM Annual Meeting, attendees voted on the role of screening for PsA in psoriasis clinical trials and on the validity, responsiveness, and feasibility of 3 candidate PsA symptoms measures: the Psoriatic Arthritis Impact of Disease 9 (PsAID9), the Routine Assessment Patient Index Data 3 (RAPID3), and the Patient Global Assessment of Arthritis (PG-arthritis). Upon review of voting results and discussions with experts in PsA, the final Delphi survey was designed and included the PsAID9, RAPID3, PG-arthritis [combined with pain visual analog scale (VAS)/NRS], and PG-PsA (combined with pain VAS/NRS).
Participants were asked to vote on the role of PsA screening in psoriasis clinical trials and on the validity, responsiveness, feasibility, and reliability of these instruments. To define consensus, prespecified endpoints were established. In addition, participants were asked to rank the instruments in order of preference. The survey was distributed through Partner’s REDCap to all members of the IDEOM, GRAPPA, and Psoriasis and Psoriatic Arthritis Clinics Multicenter Advancement Network (PPACMAN). Finally, the group presented and discussed the results of the Delphi survey at a consensus meeting held at the 2018 IDEOM Annual Meeting.
The Delphi survey involved 297 international participants including rheumatologists (45.1%), dermatologists (25.9%), patients (7.4%), industry partners (8.8%), dermatologist-rheumatologists (5.1%), patient association representatives (3.4%), and others (4.3%). Results showed that 91.2% of them agreed that all patients enrolling in a psoriasis trial should be asked about a rheumatologist diagnosis of PsA, and 87.5% agreed that all patients enrolling in a psoriasis trial should be screened for PsA, regardless of having a known diagnosis of PsA. Among the candidate PsA symptoms measures presented, PsAID9 was voted as the instrument of highest quality. When ranked in order of importance, PsAID9 was chosen as the first choice, and RAPID3 was voted as an acceptable second choice.
The results of the Delphi survey were then endorsed (n = 40) at the consensus meeting held at the 2018 IDEOM Annual Meeting. In all, 77% of attendees agreed PsA screening should be included in all psoriasis trials and that no further Delphi rounds were needed given that prespecified endpoints defined for the Delphi survey had been met. In addition, 76% of participants agreed that the PsAID9 was the best PsA symptoms measure to be recommended for psoriasis clinical trials. Finally, 86% of participants favored the use of the PsAID9 over the PsAID12 in the context of psoriasis trials. The audience argued that fewer questions decreases questionnaire burden, and that the questions about depression and social isolation that are not included in the PsAID9 are identified within the other measures of quality of life that are already included in the psoriasis core set.
In all, the IDEOM PsA Workgroup recommends that the screening and measurement of PsA symptoms should occur in psoriasis clinical trials and registries. Screening for PsA is not only consistent with good clinical practice because it provides a unique opportunity to detect cases of PsA, but it will further allow investigators to match patients to studies for which they may be more appropriate (e.g., inform whether patients with PsA should be enrolled in a trial for topical therapy). Similarly, measuring PsA symptoms in the broader at-risk psoriasis population could allow HCP to identify the early transition to PsA, offer the opportunity for early intervention, and facilitate the understanding of therapeutic interventions for psoriasis on the natural history of PsA, which is especially useful in registries and longterm extension studies.
Next steps involve the dissemination and implementation of results in the psoriasis clinical trials and registries.
Treatment Satisfaction in Psoriasis Trials Update
Dr. Armstrong presented the progress to date on IDEOM and GRAPPA’s effort to measure treatment satisfaction in psoriasis. First, based on an international consensus effort, treatment satisfaction was defined as 1 core domain to be measured in psoriasis clinical trials5.
Treatment satisfaction refers to the extent to which patients perceive that a given therapy fulfills their health needs15. Indeed, it reflects the patients’ experience with attributes of both the treatment process and the treatment outcomes. Treatment process attributes include treatment location, frequency, duration, route of administration, formulation, and cost. Treatment outcome attributes include treatment benefit (probability of treatment benefit, magnitude of treatment benefit, duration of treatment benefit, and time until treatment benefit) and side effects (probability, severity, and reversibility)15.
It has been proposed that a patient’s decision to continue or change treatment is based on a decision balance. In this decision balance, treatment effectiveness is weighed against side effects and the inconvenience of using the medication16.
To select the best instrument to assess treatment satisfaction in psoriasis clinical trials, the Treatment Satisfaction Workgroup conducted a systematic literature review (SLR) to identify all treatment satisfaction instruments that are used in dermatology and critically appraise their quality (measurement properties). Following the COSMIN guideline for systematic reviews, the methodological quality of studies on measurement properties, as well as the quality (i.e., measurement properties) of the treatment satisfaction instruments themselves were assessed. This methodology allowed for an evidence-based recommendation on the overall quality of treatment satisfaction instruments to support whether a treatment satisfaction tool is selected17.
The SLR identified 11 treatment satisfaction instruments that were classified as generic, dermatologic-specific, and psoriasis-specific. Generic treatment satisfaction instruments include the Beliefs about Medicines Questionnaire and the Treatment Satisfaction Questionnaire for Medication. Dermatology-specific treatment satisfaction questionnaires include Patient Benefit Index (PBI), Vehicle-preference score, Topical Therapy Adherence Questionnaire, and Patient Preference Questionnaire. Psoriasis-specific treatment satisfaction instruments include REFlective evaluation of psoriasis Efficacy of Treatment and Severity, Spanish Satisfaction with Treatment of Psoriasis Questionnaire, Nail Assessment in Psoriasis and PsA-PBI, Psoriasis Satisfaction Questionnaire, and Desired Improvement Tool.
Results showed there is variability in validity, reliability, and responsiveness among the different measurements. Many of these instruments lacked information on the measurement properties. As a result, IDEOM agreed that a new treatment satisfaction questionnaire was needed for patients with psoriatic diseases.
Finally, Dr. Armstrong presented a draft of a treatment satisfaction questionnaire currently in preparation. To inform the design and content of the instrument, the workgroup reviewed the instrument forms of existing treatment satisfaction tools and conducted nominal group discussions with patients who have psoriasis. This questionnaire centered on 4 domains: effectiveness, safety, convenience, and global treatment satisfaction. This was well received among the attendees.
Psoriasis and PsA Clinics Multicenter Advancement Network (PPACMAN)
Psoriatic disease is a multisystemic disease state that requires an interdisciplinary approach to ensure accurate and timely diagnosis and management18,19. To address this need, the PPACMAN, a nonprofit 501(c)(3) organization, was established “to nucleate psoriasis/PsA combined clinics to advance a multi-level approach to psoriatic patients, increase disease awareness and accelerate management”20.
At the 2018 annual GRAPPA meeting, Dr. Merola, Dr. Jose Scher, Dr. Soumya Reddy, and Dr. Ogdie presented PPACMAN’s goals and updates on their work. Having established a network of combined clinics and local/regional partnerships, PPACMAN’s goals are to (1) improve education for trainees and practicing HCP about the importance of early identification of PsA and the value of collaborative care for patients with psoriatic disease; (2) support the formation of similar clinical models and regional dermatology-rheumatology partnerships; and (3) conduct research that demonstrates the effectiveness of these care models as well as to promote and conduct clinical translational research in psoriatic disease by harnessing the unique environment of its combined clinics network.
Dr. Ogdie and Dr. Scher presented the research mission. Dr. Ogdie then presented the study, “Identifying barriers and facilitators of co-management of PsA among dermatologists and rheumatologists in the US,” which will be funded by Pfizer. For this study, input will be collected from patients from combined clinics through telephone interviews and from HCP through focus groups and meetings. Dr. Ogdie reviewed ongoing efforts to create a network of shared data from multiple combined clinics for longitudinal patient observation. Dr. Scher introduced the study “Preventing Arthritis in a Multicenter At-risk Population (PAMPA) Study Group,” which was established to facilitate clinical, translational, and basic research into the preclinical and earliest clinically apparent phases of PsA.
Dr. Merola and Dr. Reddy presented the clinical and educational missions projects. Under the clinical mission, PPACMAN is promoting the use of virtual clinics, Web-based conferences, tele-mentoring, and shared electronic medical records (EMR) data to foster relationships and communication between community-based centers. Also, PPACMAN is developing a “Clinic Building Toolkit” to guide the establishment of new combined clinics and “EMR Templates Toolkit” to promote best practices and ensure clinicians are collecting necessary data for shared decision making. Regarding the educational mission, PPACMAN is offering observerships in combined clinics for providers and administrative staff as well as provider meetings, the first of which was held in the New York City area on September 29, 2018.
Finally, Dr. Reddy presented results of a recent PPACMAN survey on the needs and challenges of combined clinics, and Dr. Merola and Dr. Scher highlighted PPACMAN’s achievements in promoting the next generation of researchers, including Dr. Perez-Chada and Dr. Julia Manasson.
DISCUSSION
Herein, we present the updates of the skin research workgroups from the GRAPPA 2018 Annual Meeting. IDEOM will continue its work to identify instruments to measure quality of care in dermatology as well as outcome measures for the core domains set for psoriasis clinical trials. Similarly, PPACMAN remains committed to working on its research, clinical, and educational missions to further the understanding of psoriatic disease, raise awareness, and advance care. Updates on IDEOM’s and PPACMAN’s work will be presented at the 2019 GRAPPA annual meeting in Paris, France.
Footnotes
As part of the supplement series GRAPPA 2018, this report was reviewed internally and approved by the Guest Editors for integrity, accuracy, and consistency with scientific and ethical standards.
