Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis.

A meeting was organized to bring together multiple stakeholders involved in the testing and authorization of new medications for juvenile idiopathic arthritis (JIA) to discuss current issues surrounding clinical trials and access to new medications for children and adolescents with JIA. The Childhood Arthritis and Rheumatology Research Alliance invited representatives of regulatory agencies (Food and Drug Administration and European Medicines Agency), and major pharmaceutical companies with JIA-approved products or products in development, patient and parent representatives, representatives of an advocacy organization (Arthritis Foundation), and pediatric rheumatology clinicians/investigators to a 1-day meeting in April 2018. The participants engaged in discussion regarding issues in clinical trials. As the pharmacologic options to treat inflammatory arthritis rapidly expand, registration trial designs to test medications in JIA patients must adapt. Many methodologies successfully used in the recent past are no longer feasible. The pool of patients meeting entry criteria who are willing to participate is shrinking while the number of medications to be tested is growing. Suggested solutions included proposing innovative clinical trial methods to regulatory agencies, as well as open discussions among stakeholders. Ensuring that new medications are authorized in a timely manner to meet the needs of JIA patients worldwide is critical. Approaches should include open dialog between regulatory agencies, pharmaceutical companies, and other stakeholders to develop and implement novel study designs, including patient and clinician perspectives to define meaningful trial outcomes, and changing existing study plans.

Experience of care from the perspective of individuals with cystic fibrosis and families: Results from 70 CF Foundation accredited programs in the USA.

INTRODUCTION: In 2012 and 2013, 30 adult and 40 pediatric CF Foundation-accredited programs across the United States recruited patients and families to complete an experience of care survey. This paper reports the positive attributes and the opportunities for improvement in CF care from the perspective of individuals with CF and families. METHODS: Patients and families completed the survey by web, interactive voice response, or with the help of a telecommunication professional. Funnel plot was used to determine positive attributes and improvement opportunities. Chi-square tests and 95% confidence intervals were used to determine differences between group and logistic regression models were used to determine factors associated with the experience of "best" care. RESULTS: 2090 adults with CF or parents of children with CF, 29% of the 7113 potential respondents, completed a survey. Both the adult and pediatric survey respondents reported the same 5 positive attributes of experience of care: courtesy and respect shown, easy to understand explanations, involved in decision-making, their questions were answered, and enough time with providers. Potential areas for improvement included assessing mental health and improving inpatient hospital staff's knowledge of CF. In general, results from the pediatric survey were significantly better than the adult survey. Variables predictive of "best" care experience from both adult and pediatric respondents were treatments always working and two self-care factors of finding information and working out solutions. CONCLUSION: The CF Foundation developed an experience of care survey to systematically collect and learn directly from individuals with CF and families about their impressions and observations of CF health care delivery. Respondents reported positive and respectful experiences and improvement opportunities were identified, which can help programs target specific areas to enhance the care experience.

Improving chronic care delivery and outcomes: the impact of the cystic fibrosis Care Center Network.

Cystic fibrosis (CF) is a multisystem, life-shortening genetic disease that requires complex care. To facilitate this expert, multidisciplinary care, the CF Foundation established a Care Center Network and accredited the first care centres in 1961. This model of care brings together physicians and specialists from other disciplines to provide care, facilitate basic and clinical research, and educate the next generation of providers. Although the Care Center Network has been invaluable in achieving substantial gains in survival and quality of life, additional opportunities for improvements in CF care exist. In 1999, analysis of data from the CF Foundation's Patient Registry detected variation in care practices and outcomes across centres, identifying opportunities for improvement. In 2002, the CF Foundation launched a comprehensive quality improvement (QI) initiative to enhance care by assembling national experts to develop a strategic plan to disseminate QI training and processes throughout the Care Center Network. The QI strategies included developing leadership (nationally and within each care centre), identifying best CF care practices, and incorporating people with CF and their families into improvement efforts. The goal was to improve the care for every person with CF in the USA. Multiple tactics were undertaken to implement the strategic plan and disseminate QI training and tools throughout the Care Center Network. In addition, strategies to foster collaboration between care centre staff and individuals with CF and their families became a cornerstone of QI efforts. Today it is clear that the application of QI principles within the CF Care Center Network has improved adherence to clinical guidelines and achievement of important health outcomes.