Measuring What Matters to Patients in Dermatology Drug Development: A Regulatory Perspective.

Incorporating the patient voice into drug development and regulatory review process allows for the science of drug development to be more patient-centered. Dermatology is one therapeutic area where patients have the potential to provide valuable perspectives on symptoms, functional impacts, and aesthetic outcomes. Patient-reported and observer-reported outcomes play an important role in capturing concerns related to the disease or condition and its treatment. Patient experience data from well-designed trials are critical for regulatory decision-making and ultimately enable prescribers and patients to make better informed treatment decisions at the point of care.

FDA Approval Summary: Selumetinib for Plexiform Neurofibroma.

On April 10, 2020, the FDA approved selumetinib (KOSELUGO, AstraZeneca) for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas. Approval was based on demonstration of a durable overall response rate per Response Evaluation in Neurofibromatosis and Schwannomatosis criteria and supported by observed clinical improvements in plexiform neurofibroma-related symptoms and functional impairments in 50 pediatric patients with inoperable plexiform neurofibromas in a single-arm, multicenter trial. The overall reponse rate per NCI investigator assessment was 66% (95% confidence interval, 51-79) with at least 12 months of follow-up. The median duration of response was not reached, and 82% of responding patients experienced duration of response ≥12 months. Clinical outcome assessment endpoints provided supportive efficacy data. Risks of selumetinib are consistent with MAPK (MEK) inhibitor class effects, including ocular, cardiac, musculoskeletal, gastrointestinal, and dermatologic toxicities. Safety was assessed across a pooled database of 74 pediatric patients with plexiform neurofibromas and supported by adult and pediatric selumetinib clinical trial data in cancer indications. The benefit-risk assessment for selumetinib in patients with inoperable plexiform neurofibromas was considered favorable.

Exploration of baseline patient-reported side effect bother from cancer therapy.

BACKGROUND: Patient reports of expected treatment side effects are increasingly collected as part of the assessment of patient experience in clinical trials. A global side effect item that is patient-reported has the potential to inform overall tolerability. Therefore, the aim of this study was to examine the completion and distribution of such a global single-item measure of side effect burden in five cancer clinical trials. METHODS: Data from five trials from internal Food and Drug Administration databases that included the Functional Assessment of Cancer Therapy-General single-item measure of overall side effect burden (i.e. impact on degree of bother) were analyzed. Completion rates for the side effect bother item, items adjacent to this item, and two non-adjacent items on the Functional Assessment of Cancer Therapy-General that are related to health-related quality of life were calculated at the baseline assessment and at the 3-month assessment. To evaluate the distribution, the percentage of patients reporting high levels (quite a bit or very much bother) of side effect bother at baseline and 3 months was assessed. RESULTS: Completion rates for all items were at least 80% regardless of time point or trial population. However, in three of the five trials, completion rates for the side effect bother item were lower at baseline compared to adjacent and non-adjacent items. This difference was not observed at 3 months. Up to 9.4% of patients reported high levels of side effect bother at baseline. CONCLUSION: Patients may enter trials already reporting some bother from side effects. This can make interpretation of results with respect to the investigational agent under study challenging. Patients may skip an item evaluating side effect bother at baseline, suggesting some difficulty with interpretation of what is being asked. Further study of the wording and utility of a baseline side effect bother assessment is warranted.

Public-private partnerships in transplant drug development.

The Transplant Therapeutics Consortium (TTC), a public-private partnership (PPP) led by the Critical Path Institute (C-Path), recently published a whitepaper titled "The Importance of Drug Safety and Tolerability in the Development of New Immunosuppressive Therapy for Transplant Recipients" by Stegall et al in the American Journal of Transplantation. As staff members of the Food and Drug Administration's (FDA), Center for Drug Evaluation and Research (CDER), Office of New Drugs and Office of Translational Science, and the Oncology Center of Excellence, we would like to provide our perspective on the TTCs efforts and the whitepaper.

Informing the Tolerability of Cancer Treatments Using Patient-Reported Outcome Measures: Summary of an FDA and Critical Path Institute Workshop.

The US Food and Drug Administration and the Critical Path Institute's Patient-Reported Outcome (PRO) Consortium convened a cosponsored workshop on the use of PRO measures to inform the assessment of safety and tolerability in cancer clinical trials. A broad array of international stakeholders involved in oncology drug development and PRO measurement science provided perspectives on the role of PRO measures to provide complementary clinical data on the symptomatic side effects of anticancer agents. Speakers and panelists explored the utility of information derived from existing and emerging PRO measures, focusing on the PRO version of the National Cancer Institute's Common Terminology Criteria for Adverse Events. Panelists and speakers discussed potential ways to improve the collection, analysis, and presentation of PRO data describing symptomatic adverse events to support drug development and better inform regulatory and treatment decisions. Workshop participants concluded the day with a discussion of possible approaches to the patient-reported assessment of an investigational drug's overall side effect burden as a potential clinical trial end point. The Food and Drug Administration reiterated its commitment to collaborate with international drug development stakeholders to identify rigorous methods to incorporate the patient perspective into the development of cancer therapeutics.

U.S. Food and Drug Administration Approval: Neratinib for the Extended Adjuvant Treatment of Early-Stage HER2-Positive Breast Cancer.

On July 17, 2017, the FDA approved neratinib (NERLYNX; Puma Biotechnology, Inc.) for the extended adjuvant treatment of adult patients with early-stage HER2-overexpressed/amplified breast cancer, to follow adjuvant trastuzumab-based therapy. Approval was based on data from ExteNET, a randomized, double-blind, placebo-controlled multicenter trial. Women with early-stage HER2-positive breast cancer and within 2 years of completing adjuvant trastuzumab were randomized to neratinib (n = 1,420) or placebo (n = 1,420) for 1 year. The primary endpoint was invasive disease-free survival (iDFS), defined as the time between randomization date to first occurrence of invasive recurrence (local/regional, ipsilateral, or contralateral breast cancer), distant recurrence, or death from any cause, with 2 years and 28 days of follow-up. The trial showed a statistically significant treatment effect favoring neratinib with a stratified HR of 0.66 [95% confidence interval (CI), 0.49-0.90, P = 0.008]. The estimated iDFS rate at 2 years was 94.2% (95% CI, 92.6%-95.4%) in patients treated with neratinib versus 91.9% (95% CI, 90.2%-93.2%) in those receiving placebo. Diarrhea was the most common adverse event (AE), with a 40% incidence of grade 3 or 4 diarrhea, and represents the most common AE leading to treatment discontinuation. Other frequent AEs (>10% incidence) were nausea, abdominal pain, fatigue, vomiting, rash, stomatitis, decreased appetite, and muscle spasms. Other than diarrhea, neratinib is associated with a low incidence of severe AEs; toxicities are generally reversible and manageable with dose interruptions, dose reductions, and/or standard medical care. This article summarizes FDA decision-making and data supporting the neratinib approval. Clin Cancer Res; 24(15); 3486-91. ©2018 AACRSee related commentary by Unni et al., p. 3483.

U.S. Food and Drug Administration Approval: Cabozantinib for the Treatment of Advanced Renal Cell Carcinoma.

On April 25, 2016, the FDA approved cabozantinib (Cabometyx; Exelixis, Inc.) for the treatment of advanced renal cell carcinoma (RCC) in patients who have received prior antiangiogenic therapy. The approval was based on data from one randomized, open-label, multicenter study in which patients with RCC who had received prior antiangiogenic therapy were treated with either cabozantinib 60 mg orally once daily (n = 330) or everolimus 10 mg orally once daily (n = 328). The major efficacy outcome measure was progression-free survival (PFS) as assessed by a blinded independent radiology review committee in the first 375 randomized patients. A statistically significant improvement in PFS was seen, with a median PFS of 7.4 and 3.8 months in the cabozantinib and everolimus arms, respectively [hazard ratio (HR), 0.58; 95% confidence interval (CI), 0.45-0.74; P < 0.0001]. At a second interim analysis, a statistically significant improvement in overall survival (OS) in the intent-to-treat population was also demonstrated, with a median OS of 21.4 and 16.5 months in the cabozantinib and everolimus arms, respectively (HR, 0.66; 95% CI, 0.53-0.83; P = 0.0003). The most common (greater than or equal to 25%) adverse reactions included diarrhea, fatigue, nausea, decreased appetite, palmar-plantar erythrodysesthesia syndrome, hypertension, vomiting, weight loss, and constipation. Clin Cancer Res; 23(2); 330-5. ©2016 AACR.

Development and Validation of the Eyelash Satisfaction Questionnaire.

BACKGROUND: Patient-reported outcome (PRO) measures have been used to assess treatment benefit in a variety of therapeutic areas and are now becoming increasingly important in aesthetic research. OBJECTIVES: The objective of the current study was to develop and validate a new PRO measure (Eyelash Satisfaction Questionnaire [ESQ]) to assess satisfaction with eyelash prominence. METHODS: The content of the questionnaire (including conceptual framework and questionnaire items) was generated by review of literature, participant interviews, and expert opinion. Cognitive interviews were conducted to pilot test the questionnaire. Psychometric properties of the questionnaire were examined in a combined sample of participants (n = 970) completing Internet- (n = 909) and paper-based (n = 61) versions. Item- and domain-level properties were examined using modern and classical psychometrics. RESULTS: Content-based analysis of qualitative data demonstrated the presence of 3 distinct domains (Length, Fullness, Overall Satisfaction; Confidence, Attractiveness, and Professionalism; and Daily Routine). Initial confirmatory factor analysis (CFA) results of 23 items revealed insufficient model-data fit (comparative fit index [CFI] of 0.86 and a non-normed fit index [NNFI] of 0.82). A revised model using 9 items (3 per domain) achieved appropriate fit (CFI of 0.99 and NNFI of 0.97). Analyses revealed measurement equivalence across the Internet- and paper-based versions. The 3 ESQ domains had strong internal consistency reliability (Cronbach's α [range] = 0.919-0.976) and adequate convergent and discriminant validity. CONCLUSIONS: The ESQ was found to be a reliable and valid PRO measure for assessing satisfaction with eyelash prominence. LEVEL OF EVIDENCE 3: Therapeutic.

Development and validation of a novel patient-reported treatment satisfaction measure for hyperfunctional facial lines: facial line satisfaction questionnaire.

BACKGROUND: Facial lines or wrinkles are among the most visible signs of aging, and minimally invasive cosmetic procedures are becoming increasingly popular. AIMS: The aim of this study was to develop and validate the Facial Line Satisfaction Questionnaire (FLSQ) for use in adults with upper facial lines (UFL). METHODS: A literature review, concept elicitation interviews (n = 33), and cognitive debriefing interviews (n = 23) of adults with UFL were conducted to develop the FLSQ. The FLSQ comprises Baseline and Follow-up versions and was field-tested with 150 subjects in a US observational study designed to assess its psychometric performance. Analyses included acceptability (item and scale distribution [i.e. missingness, floor, and ceiling effects]), reliability, and validity (including concurrent validity). RESULTS: In total, 69 concepts were elicited during patient interviews. Following cognitive debriefing interviews, the FLSQ-Baseline version included 11 items and the Follow-up version included 13 items. Response rates for the FLSQ were 100% and 73% at baseline and follow-up, respectively; no items had excessive missing data. Questionnaire scale scores were normally distributed. Most domain scores demonstrated good internal consistency reliability (Cronbach's α ≥ 0.70). Most items within their respective domains exhibited good convergent (item-scale correlations > 0.40) and discriminant (items had higher correlation with their hypothesized scales than other scales) validity. Concurrent validity correlation coefficients of the FLSQ domain scores with the associated concurrent measures were acceptable (range: r = 0.40-0.70). Six FLSQ items demonstrated reliability and validity as stand-alone items outside their domains. CONCLUSIONS: The FLSQ is a valid questionnaire for assessing treatment expectations, satisfaction, impact, and preference in adults with UFL.

Subject satisfaction with onabotulinumtoxinA treatment of glabellar and lateral canthal lines using a new patient-reported outcome measure.

BACKGROUND: Patient satisfaction with treatment is an important outcome in facial aesthetic medicine. OBJECTIVE: To evaluate subject satisfaction with onabotulinumtoxinA treatment of glabellar lines (GL) and crow's feet lines (CFL) using the validated Facial Line Satisfaction Questionnaire (FLSQ). METHODS: In this randomized double-blind study, subjects with moderate/severe GL and CFL received onabotulinumtoxinA (20 U, GL; 24 U, CFL) or placebo. Over 120 days, the following were assessed: satisfaction, achievement of treatment expectations, satisfaction with duration of treatment (FLSQ), severity of GL and CFL (Facial Wrinkle Scale [FWS]), and aesthetic improvement (Global Aesthetic Improvement Scale). RESULTS: Satisfaction in the per-protocol population was significantly greater at Day 60 in the onabotulinumtoxinA group (n = 60) compared with placebo (n = 57) for GL (81.7% vs 0%; p < .001). Most subjects treated with onabotulinumtoxinA remained satisfied up to 120 days. Achievement of treatment expectations (86.7%; Day 60), satisfaction (81.7%; Day 60), and satisfaction with the duration of treatment (61.6%; Day 90) were significantly better with onabotulinumtoxinA than placebo (p < .001) for GL and CFL combined. Efficacy (FWS) and aesthetic improvement were observed in most subjects at Days 30 and 60, respectively. CONCLUSION: High satisfaction rates are achieved and sustained in subjects treated with onabotulinumtoxinA for GL and CFL combined.

Acne-Related Quality of Life Among Female Adults of Different Races/Ethnicities.

BACKGROUND: Acne impairs quality of life, but its effect on different races/ethnicities is unclear. This study evaluated racial/ethnic differences in acne-related quality of life and psychological symptoms among female adults. METHODS: A Web-based survey was conducted with U.S. female adults (25-45 years old) with facial acne (≥25 visible lesions). Outcomes included sociodemographics, clinical characteristics, acne-related quality of life (Acne-Specific Quality of Life Questionnaire), psychological symptoms (Patient Health Questionnaire), and work/school productivity. Racial/ethnic differences were evaluated using descriptive statistics and analysis of variance/chi-square analyses. RESULTS: Three-hundred twelve subjects (Black = 30.8%, Hispanic = 17.6%, Asian/other = 17.3%, White = 34.3%) completed the survey (mean age = 35.3 ± 5.9 years). Acne negatively impacted quality of life for all subjects. Black subjects reported significantly less negative impact on self-perception versus Asian/other (Black = 12.6 ± 9.9, Asian/other = 8.4 ± 8.6; p = .05). Social functioning was less negatively impacted in White and Black subjects versus Asian/other (White = 12.7 ± 7.5, Asian/other = 8.4 ± 7.8, p < .05; Black = 12.1 ± 9.2, Asian/other = 8.4 ± 7.8, p = .06). Over one third (total sample = 40.7%, Black = 31.3%, Hispanic = 36.4%, Asian/other = 50.0%, White = 46.7%) reported moderate/severe anxiety/depression symptoms. Acne also impacted ability to concentrate on work/school. CONCLUSION: Racial/ethnic differences were observed in acne-related quality of life and psychological symptoms in female adults; acne negatively impacted self-perceptions and social/emotional functioning.

Validation of a New Patient-Reported Outcome Measure for Facial Acne: The Acne Symptom and Impact Scale (ASIS).

OBJECTIVE: The objective of this study was to evaluate the psychometric performance of the Acne Symptom and Impact Scale (ASIS) for use in adolescents and adults with acne vulgaris. METHODS: Psychometric evaluation was performed using both traditional psychometrics in line with proposed US Food and Drug Administration (FDA) criteria and new psychometric methods, Rasch Measurement Theory (RMT). Assessment of equivalence was also evaluated between Caucasians and Non-Caucasians on individual items. RESULTS: One-hundred fifty subjects completed baseline and follow-up assessments (89 [59.33%] in the Caucasian group and 61 [40.67%] in the Non-Caucasian group). Psychometric analyses demonstrated that the ASIS Sign and Impact domains both performed well. Each domain fulfilled traditional psychometric criteria (Cronbach's alpha=0.79-0.92; test-retest reliability=0.75-0.78) and mostly satisfied Rasch psychometric criteria (person-reliability=0.72-0.93; person-separation=1.61-3.69). Select individual ASIS Items also performed well across all measures and were shown to be reliable and valid as stand-alone items. A similar pattern of results were found for both Caucasian and Non-Caucasian racial subgroups. CONCLUSIONS: These findings provide empirical evidence that the ASIS is a reliable and valid PRO measure that can accurately assess the severity of symptoms and impacts associated with acne vulgaris.

Safety and Efficacy of Bimatoprost for Eyelash Growth in Postchemotherapy Subjects.

OBJECTIVE: To evaluate long-term efficacy and safety of bimatoprost for treatment of chemotherapy-induced eyelash hypotrichosis. DESIGN: One-year, multicenter, double-masked, parallel-group study. SETTING: Twenty-one centers in the United States and one center in the United Kingdom. PARTICIPANTS: This study randomized (3:1) 130 subjects to bimatoprost 0.03% or vehicle applied topically to upper eyelid margins for six months. All subjects used bimatoprost for a second six months. MEASUREMENTS: Responders for the primary composite end point achieved ≥1-grade improvement in Global Eyelash Assessment score and ≥3-point improvement in Confidence, Attractiveness, and Professionalism domain score of the Eyelash Satisfaction Questionnaire at Month 4. Secondary assessments included eyelash length, thickness, and darkness, using digital image analysis. RESULTS: The responder rate was significantly higher with bimatoprost versus vehicle at Month 4 (37.5% vs. 18.2%; p=0.041) and Month 6 (46.9% vs. 18.2%; p=0.004). Significant improvements favoring bimatoprost occurred in eyelash length (p=0.008), thickness (p<0.001), or darkness (p=0.029) at Month 4, with similar results at Month 6 (p<0.001, length; p<0.001, thickness; p=0.002, darkness). Responder rates reached 61.5 percent at Month 12 for subjects continuing bimatoprost and 67.6 percent for those switched from vehicle to bimatoprost. Conjunctival hyperemia (16.7%) and punctate keratitis (9.4%) were the most common adverse events. CONCLUSION: Bimatoprost provides rapid eyelash recovery, whether started shortly after chemotherapy (4 to 12 weeks) or delayed for six months, with minimal adverse events. CLINICAL TRIAL REGISTRY: NCT00907426.

Acne treatment patterns, expectations, and satisfaction among adult females of different races/ethnicities.

BACKGROUND: Limited data are available on acne treatment patterns, expectations, and satisfaction in the adult female subpopulation, particularly among different racial and ethnic groups. OBJECTIVE: Describe acne treatment patterns and expectations in adult females of different racial/ethnic groups and analyze and explore their potential effects on medication compliance and treatment satisfaction. METHODS: A cross-sectional, Web-based survey was administered to US females (25-45 years) with facial acne (≥25 visible lesions). Data collected included sociodemographics, self-reported clinical characteristics, acne treatment use, and treatment expectations and satisfaction. RESULTS: Three hundred twelve subjects completed the survey (mean age, 35.3±5.9 years), comprising black (30.8%), Hispanic (17.6%), Asian/other (17.3%), and white (34.3%). More than half of the subjects in each racial group recently used an acne treatment or procedure (black, 63.5%; Hispanic, 54.5%; Asian/other, 66.7%; white, 66.4%). Treatment use was predominantly over-the-counter (OTC) (47.4%) versus prescription medications (16.6%). OTC use was highest in white subjects (black, 42.7%; Hispanic, 34.5%; Asian/other, 44.4%; white, 59.8%; P<0.05). The most frequently used OTC treatments in all racial/ethnic groups were salicylic acid (SA) (34.3%) and benzoyl peroxide (BP) (32.1%). Overall, compliance with acne medications was highest in white versus black (57.0±32.4 vs 42.7±33.5 days, P>0.05), Hispanic (57.0±32.4 vs 43.2±32.9 days, P>0.05), and Asian/other (57.0±32.4 vs 46.9±37.2 days, P>0.05) subjects. Most subjects expected OTC (73.7%) and prescription (74.7%) treatments to work quickly. Fewer than half of the subjects were satisfied with OTC treatment (BP, 47.0%; SA, 43.0%), often due to skin dryness (BP, 26.3%; SA, 44.3%) and flakiness (BP, 12.3%; SA, 31.1%). No statistically significant differences were observed among racial/ethnic groups in their level of satisfaction with OTC or prescription acne treatments. CONCLUSION: Racial/ethnic differences were observed in acne treatment patterns in adult females, while treatment expectations were similar. Results indicate that treatment patterns and expectations may impact treatment satisfaction and medication compliance.

Impact of female acne on patterns of health care resource utilization.

BACKGROUND: Limited data are available on acne treatment patterns in females through their adult years. OBJECTIVE: The purpose of this analysis was to evaluate health care resource utilization (HRU) and treatment patterns in cohorts with and without the use of acne medication and predictors of use. METHODS: A cross-sectional, web-based survey was administered to US females (25-45 years) with facial acne (≥ 25 visible lesions). Data collected included: sociodemographics and self-reported clinical characteristics, acne treatments, and health care professional (HCP) visits. Subject characteristics associated with medication use were examined by logistic regression. RESULTS: Approximately half of the total sample (N=208, mean age: 35 ± 6) ever visited an HCP for acne and reported more over-the counter (OTC) medication use (51.0%) than prescription (Rx) medication use (15.4%). Subjects did not use medications daily, averaging from 12-18 days over the previous 4 weeks. Logistic regression showed that race and prior HCP visits for acne were significant predictors of medication use (P<.05). CONCLUSIONS: Adult females generally self-treated their acne using primarily OTC medications; however, poor compliance was observed for Rx and OTC. Race and prior HCP visits for acne were significant predictors of current medication use.

Effects of OnabotulinumtoxinA treatment for crow's feet lines on patient-reported outcomes.

BACKGROUND: Although millions of aesthetic procedures are performed annually, few patient-reported outcome (PRO) measures have been used in this setting. OBJECTIVE: To evaluate the impact of onabotulinumtoxinA treatment for crow's feet lines (CFL) on relevant psychological variables and self-perception of age/appearance in subgroup populations. MATERIALS AND METHODS: Facial Lines Outcomes (FLO-11) Questionnaire, Self-Perception of Age (SPA), and Subject Global Assessment of Change in CFL (SGA-CFL) were PRO measures administered in 2 Phase 3, double-blind placebo-controlled trials for the treatment of CFL alone or CFL/glabellar lines (GL). Patient-reported outcome measures were analyzed by subgroups (age, gender, and baseline CFL severity). Subject satisfaction with appearance was also analyzed. RESULTS: Most subgroups receiving onabotulinumtoxinA demonstrated significant improvements in psychological impact (FLO-11 Items 2, 5, and 8) versus placebo at Day 30 (p ≤ .05). OnabotulinumtoxinA-treated subjects consistently rated themselves as looking younger on SPA versus placebo in all subgroups at Day 30 (p ≤ .05) and showed significant improvements in CFL appearance versus placebo at all time points on SGA-CFL. Overall, subjects were satisfied with their appearance. CONCLUSION: OnabotulinumtoxinA-treated subjects experienced significant improvements in perceived appearance, attractiveness, tiredness, age, and satisfaction versus placebo. Subjects treated for CFL and GL experienced even greater effects.

The impact of upper facial lines and psychological impact of crow's feet lines: content validation of the Facial Line Outcomes (FLO-11) Questionnaire.

BACKGROUND: Treatments for upper facial lines (UFL), the most visible sign of aging, are of interest to patients and clinicians alike. Patient-reported outcomes (PROs) are valuable in evaluating the impact of such treatments; however, regulatory recommendations have stipulated that the patient perspective be central in developing these assessments. OBJECTIVES: (1) To evaluate the content validity of the Facial Lines Outcomes Questionnaire, a PRO instrument developed to assess upper facial line impacts, according to the regulatory guidance of the United States Food and Drug Administration and (2) assess whether it adequately measures the psychological impacts associated with crow's feet lines (CFL) (lateral canthal lines) from the patient perspective. METHODS: Two patient groups participated in face-to-face qualitative interviews. One group included patients with UFL (Group 1, n = 25 interviews), and the other included patients specifically with CFL (Group 2, n = 41 interviews). Each interview consisted of a concept elicitation and cognitive debriefing phase. RESULTS: Interviews with both groups elicited all key concepts of the instrument, including "bothered by facial lines"; "looking older"; "looking less attractive"; and looking "tired," "stressed," or "angry." Most Group 2 patients (n = 35, 85%) agreed that the instrument adequately assessed the psychological impacts associated with CFL. During cognitive debriefing, the majority of patients in both groups agreed the instrument was understandable, comprehensive, and easy to complete. CONCLUSIONS: The Facial Line Outcomes Questionnaire is an appropriate and valid tool to assess the impact of UFL and the psychological impacts associated with CFL.

Racial differences in clinical characteristics, perceptions and behaviors, and psychosocial impact of adult female acne.

OBJECTIVE: Limited data are available on racial differences in clinical characteristics and burden in adult female acne. The objective was to describe racial differences in clinical characteristics, psychosocial impact, perceptions, behaviors, and treatment satisfaction in facial adult female acne. DESIGN: Cross-sectional, web-based survey. SETTING: Diverse sample of United States women. PARTICIPANTS: Women between the ages of 25 and 45 years with facial acne (≥25 visible lesions). MEASUREMENTS: Outcomes included sociodemographic characteristics, psychosocial impacts, perceptions, behaviors, and treatment satisfaction. Racial differences were evaluated using descriptive statistics and t-test/chi-square analyses. RESULTS: 208 females participated (mean age 35±6 years); 51.4 percent were White/Caucasian and 48.6 percent were non-White/Caucasian women [Black/African American (n=51); Hispanic/Latina (n=23); Asian (n=16); Other (n=ll)]. Age of acne onset (mean 14.8±5 vs. 17.0±8 years, p<0.05) and acne concern occurred earlier (16.6±7 vs. 19.3±9 years, p<0.05) in White/Caucasian than non-White/Caucasian subjects. Facial acne primarily presented on chin (28.0%) and cheeks (30.8%) for White/Caucasian women versus cheeks (58.4%) for non-White/Caucasian women. Non-White/Caucasian women experienced more postinflammatory hyperpigmentation than White/Caucasian women (p<0.0001). Facial acne negatively affected quality of life (QoL) in both groups, and most participants (>70%) reported some depression/anxiety symptoms. More White/Caucasian than non-White/Caucasian women were troubled by facial acne (88.8% vs. 76.2%, p<0.05). Lesion clearance was most important to White/Caucasian women (57.9 vs. non-White/Caucasian 31.7%, p<0.001); non-White/Caucasian females focused on postinflammatory hyperpigmentation clearance (41.6% vs. Caucasian 8.4%, p<0.0001). CONCLUSION: RESULTS highlight racial differences in participant-reported clinical characteristics, attitudes, behaviors, and treatment satisfaction. These findings may inform clinicians about racial differences in facial adult female acne and guide treatment recommendations toward improving care.

Development of a new patient-reported outcome measure for facial acne: the Acne Symptom and Impact Scale (ASIS).

OBJECTIVE: Facial acne has been associated with impaired health-related quality of life, which is an essential patient outcome for evaluating the success of acne treatment. In consideration of the US Food and Drug Administration's (FDA) new recommendations on patient reported outcome (PRO) measures, the objectives of this study were to (1) establish the need for a new PRO measure that assesses facial acne outcomes and satisfies the criteria set forth by the FDA and (2) develop the content of a new facial acne PRO measure appropriate for use in both adolescents and adults as well as adherent to the FDA PRO Guidance. METHODS: A literature and PRO review, patient interviews (concept elicitation), and input from clinical experts were used to develop a conceptual framework for the outcomes deemed important to facial acne patients, and to construct items for a preliminary PRO measure: the Acne Symptom and Impact Scale (ASIS). Cognitive interviews were conducted to pilot test the ASIS. RESULTS: A review of the literature and PROs revealed that, of the 34 measures identified, no suitable PRO measure for the population of interest was available. The conceptual framework comprised two main themes: symptoms and psychosocial impacts. Concept elicitation interviews included a diverse set of patients (n=48) with facial acne, of various ages: 12-17 years (n=15), 18-25 years (n=20), and 26-50 years (n=13). The most frequently reported symptoms were: pimples, oily skin, scabs/scars/marks, blackheads, acne, and whiteheads. The most frequently reported impacts were impacts on appearance, self-consciousness, annoyance, bothersomeness, mood, social criticism, embarrassment, confidence, and social withdrawal. These reported symptoms and impacts constituted the 15-item draft ASIS. The draft ASIS was modified following the analysis of 20 cognitive interviews, resulting in the current 17-item ASIS. CONCLUSIONS: Results from both the concept elicitation and cognitive interviews demonstrated that the ASIS is content valid in both adolescents and adults with facial acne. The ASIS will undergo psychometric evaluation to further support its validity in both adolescents and adults with facial acne.