Incentivizing Orphan Product Development: United States Food and Drug Administration Orphan Incentive Programs.

Over 30 years ago, the United States (US) Congress passed the Orphan Drug Act (ODA) to encourage the development of products for rare diseases or conditions ("orphan products"). The Act provided incentives to sponsors for developing products with orphan designation and established a grant program to fund studies of orphan products. Since its enactment in 1983, the ODA has been credited for bringing more than 590 orphan drugs to the market, inspiring the implementation of orphan legislation globally, and enabling the creation of other programs that extend existing knowledge of the natural history of rare diseases and stimulate the development of medical devices for children and patients with rare diseases. This chapter provides a brief overview of the main features and successes of 5 of the orphan incentive programs administered by the US Food and Drug Administration (FDA): the Orphan Drug Designation Program, the Humanitarian Use Device (HUD) Designation Program, the Orphan Products Clinical Trials Grants Program, the Pediatric Device Consortia (PDC) Grant Program, and the Orphan Products Natural History Grants Program.

Reasons for Encounters and Comorbidities in Adolescents with Intellectual Disability in General Practice: A Retrospective Analysis of Data from the Ask Study.

Adolescents with intellectual disability have substantial health needs. This retrospective analysis of data from the Ask Study describes reasons for primary care encounters and the prevalence and incidence of chronic physical and mental conditions among a cohort of community-dwelling adolescents with intellectual disability. Participants attended secondary schools in southern Queensland, Australia. Primary care data were extracted from primary care records. Demographic and health information was collected using carer-completed questionnaires. Reasons for primary care encounters, disease prevalence at age 16 years, and disease incidence through adolescence were reported. Data were obtained for 432 adolescents with intellectual disability (median follow-up: 4.1 years). Skin problems (29.4 per 100 encounters) were the most common reason patients presented for primary care, followed by psychological and behavioural problems (14.4 per 100 encounters) and musculoskeletal problems (13.8 per 100 encounters). Conditions with the highest prevalence were autism spectrum disorder (18.6%) and asthma (18.1%). The prevalence of epilepsy, visual impairment, and cerebral palsy were 14.7, 11.1, and 8.0%, respectively. Gastroesophageal reflux had the highest incidence (9.4 cases per 1000 person-years). Adolescents with intellectual disability have significant healthcare needs, which general practitioners need to be aware of and address. Study findings should inform the development of training programs for general practitioners.

Immune marker reductions in black and white Americans following sleeve gastrectomy in the short-term phase of surgical weight loss.

BACKGROUND: Surgical weight loss procedures like vertical sleeve gastrectomy (SG) are sufficient in resolving obesity comorbidities and are touted to reduce the burden of pro-inflammatory cytokines and augment the release of anti-inflammatory cytokines. Recent reports suggest a reduced improvement in weight resolution after SG in Black Americans (BA) versus White Americans (WA). The goal of this study was to determine if differences in immunoglobulin levels and general markers of inflammation after SG in Black Americans (BA) and White Americans (WA) may contribute to this differential resolution. METHODS: Personal information, anthropometric data, and plasma samples were collected from 58 participants (24 BA and 34 WA) before and 6 weeks after SG for the measurement of immunoglobulin A (IgA), IgG, IgM, C-reactive protein (CRP), and transforming growth factor (TGFß). Logistic regression analysis was used to determine the relationship of measures of body size and weight and inflammatory markers. RESULTS: Both IgG and CRP were significantly elevated in BA in comparison to WA prior to weight loss. Collectively, IgG, TGFß, and CRP were all significantly reduced at six weeks following SG. CRP levels in BA were reduced to a similar extent as WA, but IgG levels were more dramatically reduced in BA than WA despite the overall higher starting concentration. No change was observed in IgA and IgM. CONCLUSIONS: These data suggest that SG improves markers of immune function in both BA and WA. More diverse markers of immune health should be studied in future work.