Navigating the complex realm of drug access in paediatric care.

A Drug Access Navigator (DAN) is a role that has been established in patient care settings with the goal of expediting the process by which patients obtain drugs through the Health Canada Special Access Program (SAP), individual drug companies' patient access programs (PAP), and public and private drug plans. In the paediatric setting, the process of accessing many drugs has grown increasingly complex. The majority of paediatric prescriptions involve 'off-label' drug use. This leads to an increased need for drug access through the SAP and/or PAPs and demonstrates that the need for a DAN may be particularly prominent in the paediatric population. To our knowledge, there is no evidence in the literature of a DAN functioning in a paediatric setting, though there is a demonstrable need. We plan to address this need by introducing a DAN for shared use at BC Children's Hospital, a paediatric tertiary care centre.

Improving value and access to specialty medical care for families: a pediatric surgery telehealth program

Background: In Canada, access to subspecialty surgical services for children imposes inconvenience and financial hardship on geographically remote families. The purpose of this study was to evaluate a recently implemented pediatric surgical telehealth pilot program from the family and provider perspectives. Methods: Enabled by an existing telehealth infrastructure for pediatric subspecialty medicine and mental health, a pilot telehealth program for surgical consultation was established by a single surgeon in British Columbia. Following establishment of eligibility criteria, patients from remote communities requiring new consultation or clinical follow-up were offered a telehealth alternative. At the end of the encounter, both the parent and patient (if appropriate) provided feedback via a questionnaire. Provider satisfaction was also assessed via a questionnaire. We estimated costs avoided and analyzed data on pediatric surgery consultation wait time. Results: Between September 2014 and November 2017, 80 patients were seen in 19 remote telehealth centres, 23 as new referrals and 57 in follow-up consultation. Among new referrals, the commonest diagnosis was chest wall deformity. The average travel distance avoided was 705 km, with an estimated direct cost avoidance of $585. Sixty-four families (80%) completed the questionnaire. Almost all (63 [98%]) indicated high overall satisfaction with the telehealth experience. Provider satisfaction was similarly high, in terms of both the technology user interface and clinical effectiveness. Overall pediatric surgical consultation wait times were unaffected. Conclusion: Implementation of telehealth technology in a pediatric surgical practice offered high value to patients/families and, from the provider's perspective, yielded an acceptable alternative to in-person assessment.

Contexte: Au Canada, l'accès aux services chirurgicaux surspécialisés pour enfants est source d'inconvénients et de difficultés financières pour les familles vivant en région éloignée. L'objectif de cette étude était d'évaluer le point de vue des familles et des fournisseurs de services sur un programme pilote de télésanté pédiatrique récemment mis en oeuvre. Méthodes: Ce programme pilote de consultation chirurgicale à distance a été mis sur pied par un chirurgien de Colombie-Britannique, qui s'est servi d'une infrastructure de télésanté préexistante permettant la prestation de services surspécialisés de médecine et de santé mentale pour enfants. Une fois leur admissibilité établie, les patients vivant en région éloignée et ayant besoin d'une consultation ou d'un suivi clinique se sont vu offrir des services de télésanté. Après la consultation, les parents et le patient (lorsque c'était approprié) ont rempli un formulaire de rétroaction. La satisfaction des fournisseurs de soins a été également évaluée au moyen d'un questionnaire. Nous avons estimé les coûts évités et analysé les données sur le temps d'attente pour une consultation en chirurgie pédiatrique. Résultats: Entre septembre 2014 et novembre 2017, 80 patients ont consulté 19 centres de télésanté : 23 pour une nouvelle consultation et 57 pour un suivi. Parmi les nouveaux cas, le diagnostic le plus fréquent était une malformation de la paroi thoracique. En moyenne, 705 km de déplacements ont été évités, ce qui représente un coût estimé de 585 $. Soixante-quatre familles (80 %) ont rempli le questionnaire, et presque toutes (63 [98 %]) se sont dites globalement satisfaites de leur expérience de télésanté. La satisfaction des fournisseurs de soins était également élevée, tant en ce qui a trait à l'interface utilisateur des outils technologiques qu'à l'efficacité clinique. Le programme n'a eu aucun effet sur le temps d'attente général pour une consultation en chirurgie pédiatrique. Conclusion: L'utilisation de services de télésanté en chirurgie pédiatrique a été d'une grande valeur pour les patients et leur famille. Du point de vue des fournisseurs de soins, elle a constitué une solution de rechange acceptable aux consultations en personne.

Pediatric Melanoma: A 35-year Population-based Review.

BACKGROUND: Melanoma is a rare neoplasm in the pediatric population. Recent publications suggest a possible increase in incidence over the past few decades. The purpose of this study was to analyze trends in pediatric patients diagnosed with malignant melanoma in British Columbia (BC) in the past 35 years. METHODS: A retrospective review was performed. All patients in BC diagnosed with melanoma before 18 years of age from 1979 to 2014 were included. Patient demographics, melanoma description, treatment details, and survival data were collected. RESULTS: Seventy-eight subjects were identified for the study. Patients were equally distributed by sex. Sixty-one (78%) of the subjects were diagnosed in the postpubertal age (≥12 years old). The most common sites of occurrence were the extremities (n = 33) and the trunk (n = 27), with the location on the trunk showing the highest mortality rate (22%). All patients were surgically treated and some had additional chemotherapy (12) and/or radiotherapy (12). Fatal outcome was recorded in 12 of the 78 subjects, 10 of whom had postpubertal diagnosis. The average time from date of diagnosis to date of death was 9.3 years. CONCLUSIONS: The incidence of melanoma in the pediatric population remains exceedingly rare: less than 2.5 per million children younger than 18 years. The diagnosis is rarely made before puberty; the incidence is equal in males and females and has not changed over a 35-year time period in BC. Our study shows 85% survival with the majority of patients having had surgical excision only.

Validity of Commercial Activity Trackers in Children With Congenital Heart Disease.

BACKGROUND: Increasing physical activity levels is a high priority to optimize long-term health in children with congenital heart disease (CHD). Commercial activity trackers have been validated in adults and are increasingly used to measure and promote physical activity in pediatric populations, but they have not been validated in children. METHODS: In 30 children with CHD aged 10-18 years, we assessed the validity of physical activity form the wrist-based Fitbit Charge HR (Fitbit, San Francisco, CA) against hip-based ActiGraph (ActiGraph LLC, Pensacola, FL) accelerometers under free living conditions for 7 days. We assessed the association between devices using intraclass correlation coefficients (ICCs) and Bland-Altman plots. Receiver operating curves were used to identify Fitbit step cut points. RESULTS: There was a strong association between the 2 devices for daily steps across 138 analyzed person-days (ICC = 0.855; P < 0.001), but poorer agreement for time spent in physical activity intensities (ICCs < 0.7). Daily Fitbit steps of ≥ 12,500 identified meeting physical activity guidelines defined as ≥ 60 minutes of moderate-to-vigorous physical activity per day. Fitbit devices recorded more steps than accelerometers (-2242 steps per day, 95% limits of agreement of -7738 to 3253). Between-device differences were greater in boys vs girls. Fitbit devices were worn for longer than accelerometers (-36 minutes per day, 95% limits of agreement, -334 to 261), but overall differences in wear time explained little of the variance in step differences (7%, P = 0.048). CONCLUSIONS: Commercial activity trackers provide opportunities to remotely monitor physical activity in children with CHD, but absolute values might differ from accelerometers. These findings are important because of the increasing emphasis on physical activity promotion and monitoring in children with cardiovascular risk factors.

Physical Activity and Sedentary Behavior in Children With Congenital Heart Disease.

BACKGROUND: Children with congenital heart disease (CHD) are thought to have low levels of physical activity (PA), but few studies have used objective measures of PA in this population. METHODS AND RESULTS: We recruited patients with mild, moderate, and severe CHD and cardiac transplant recipients, aged 8 to 19 years, from pediatric cardiology clinics throughout British Columbia and Yukon, Canada. Participants were fitted with an ActiGraph accelerometer to be worn over the right hip for 7 days. Daily means were estimated for a variety of accelerometry-derived metrics, including moderate-to-vigorous PA and percentage of sedentary time if they had at least 3 valid days of accelerometry data. Participants also completed a PA questionnaire. We included 90 participants (aged 13.6±2.7 years; 54% male), of which 26 had mild CHD, 26 had moderate CHD, 29 had severe CHD, and 9 were cardiac transplant recipients. Median daily moderate-to-vigorous PA was 43 min/day (interquartile range: 28.9-56.9 min/day), and 8% met PA guidelines of 60 minutes of moderate-to-vigorous PA at least 6 days a week. There were no significant differences in any accelerometry-derived metric according to CHD severity. Boys were significantly more active and less sedentary than girls. Activity declined and sedentary behaviors increased with age in both sexes. Sports participation was common, including competitive out-of-school clubs (57%). PA restrictions from cardiologists were rare (15%). CONCLUSIONS: We found normal age-sex patterns of PA in children with CHD. There were no differences in PA by CHD severity, suggesting that sociocultural factors are likely important determinants of PA in these children.

Validity and reliability of the Physical Activity Questionnaire for Children (PAQ-C) and Adolescents (PAQ-A) in individuals with congenital heart disease.

OBJECTIVE: To assess the criterion validity, internal consistency, reliability and cut-point for the Physical Activity Questionnaire for Children (PAQ-C) and Adolescents (PAQ-A) in children and adolescents with congenital heart disease-a special population at high cardiovascular risk in whom physical activity has not been extensively evaluated. METHODS: We included 84 participants (13.6±2.9 yrs, 50% female) with simple (37%), moderate (31%), or severe congenital heart disease (27%), as well as cardiac transplant recipients (6%), from BC Children's Hospital, Canada. They completed the PAQ-C (≤11yrs, n = 28) or-A (≥12yrs, n = 56), and also wore a triaxial accelerometer (GT3X+ or GT9X) over the right hip for 7 days (n = 59 met valid wear time criteria). RESULTS: Median daily moderate-to-vigorous physical activity was 46.9 minutes per day (IQR 31.6-61.8) and 25% met physical activity guidelines defined as ≥60 minutes of moderate-to-vigorous physical activity per day. Median PAQ-score was 2.6 (IQR 1.9-3.0). PAQ-Scores were significantly related to accelerometry-derived metrics of physical activity (rho = 0.44-0.55, all p<0.01) and sedentary behaviour (rho = -0.53, p<0.001). Internal consistency was high (α = 0.837), as was reliability (stability) of PAQ-Scores over a 4-months period (ICC = 0.73, 95%CI 0.55-0.84; p<0.001). We identified that a PAQ-Score cut-point of 2.87 discriminates between those meeting physical guidelines and those that do not in the combined PAQ-C and-A samples (area under the curve = 0.80 (95%CI 0.67-0.92). CONCLUSION: Validity and reliability of the PAQ in children and adolescents with CHD was comparable to or stronger than previous studies in healthy children. Therefore, the PAQ may be used to estimate general levels of physical activity in children and adolescents with CHD.

Genetic Insurance Discrimination in Sudden Arrhythmia Death Syndromes: Empirical Evidence From a Cross-Sectional Survey in North America.

BACKGROUND: There is virtually no information assessing the insurability of families affected with Sudden Arrhythmia Death Syndromes (SADS) for the determination of the nonclinical implications of genetic screening. It is important to identify the barriers and challenges faced by families as a result of genetic screening for SADS to enable equitable access to insurance coverage. METHODS AND RESULTS: To explore the insurance coverage experiences of SADS-affected families, we administered a cross-sectional online survey across North America from April 28, 2012 to November 13, 2013. Participants included individuals with a SADS diagnosis and their relatives who have applied for insurance (health, life, travel, and disability) or have existing insurance coverage. Of 202 participants, 92% had a SADS diagnosis (92%) as either a proband (50%) or an affected relative (42%); 8% of participants were unaffected family members of a proband; and genetic confirmation was reported by 73%. Of the 54% of SADS respondents who applied for insurance, 60% were rejected by insurers. The preexisting SADS diagnosis was the major reason reported for rejection (57%). Most respondents (80%) had insurance coverage through a spouse/parent plan at the time of diagnosis; 14% experienced a subsequent negative effect on coverage. Thirty-nine percent of affected SADS respondents reported an increase in insurance premium rates. CONCLUSIONS: Increased genetic testing has negatively impacted insurability for SADS patients and affected family members. The challenges in obtaining life and health insurance are mainly because of the preexisting condition, even in the presence of protective laws in the United States.