The US Food and Drug Administration's (FDA's) Priority Review, Accelerated Approval, Fast Track, and Breakthrough Therapy programs have been successful in facilitating the drug approval process and getting medications to patients quicker. To qualify for one or more of these FDA programs, a sponsor must meet specific criteria. Once a drug is given one or more designations, benefits can range from frequent meetings with FDA representatives to appointed FDA senior managers to facilitate the approval process. This paper will review the major guidelines set forth by the FDA, highlight advantages to the patient and health care community as a result of receiving specific designations, and provide several examples for illustration. As a result of these designations, many patients with rare diseases or life-threatening conditions have been afforded earlier access to effective therapies. As the pharmacist's role continues to expand, it is crucial to understand the nature of these accelerated programs and to advocate for increased access to new drug therapies.
Objective: To examine methylphenidate extended-release chewable tablets (MPH ERCT) dose patterns, attention-deficit/hyperactivity disorder (ADHD) symptom scores, and safety during the 6-week, open-label (OL) dose-optimization period of a phase 3, laboratory classroom study.Methods: Boys and girls (6–12 years) diagnosed with ADHD were enrolled. MPH ERCT was initiated at 20 mg/day; participants were titrated in 10–20 mg/day increments weekly based on efficacy and tolerability (maximum dose, 60 mg/day). Dose-optimization period efficacy assessments included the ADHD Rating Scale (ADHD-RS-IV), analyzed by week in a post hoc analysis using a mixed-effects model for repeated measures with final optimized dose (20, 30/40, or 50/60 mg), visit, final optimized dose and visit interaction, and baseline score as terms. Adverse events (AEs) and concomitant medications were collected throughout the study.Results: Mean MPH ERCT daily dose increased weekly from 29.4 mg/day after the first dose adjustment at week 1 (n = 90) to 42.8 mg/day after the final adjustment at week 5 (n = 86). Final optimized MPH ERCT dose ranged from 20 to 60 mg/day. Mean final optimized MPH ERCT dose ranged from 40.0 mg/day in 6–8 year-old participants to 44.8 mg/day for 11–12 year-old participants. There was a progressive decrease in mean (standard deviation) ADHD-RS-IV total score from 40.1 (8.72) at baseline to 12.4 (7.88) at OL week 5, with similar improvement patterns for hyperactivity/impulsivity and inattentiveness subscale scores. Participants optimized to MPH ERCT 50/60 mg/day had a significantly higher mean (standard error) ADHD-RS-IV score at baseline compared with participants optimized to MPH ERCT 20 mg/day (42.4 [1.34] vs. 35.1 [2.55]; p = 0.013). Treatment-emergent AEs were reported by 65/90 (72.2%) participants in the dose-optimization period.Conclusions: Dose-optimization period results describing relationships between change in ADHD symptom scores and final optimized MPH ERCT dose will be valuable for clinicians optimizing MPH ERCT dose.
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