The U.S. Food and Drug Administration's Experience with Ivacaftor in Cystic Fibrosis. Establishing Efficacy Using In Vitro Data in Lieu of a Clinical Trial.

On May 17, 2017, the U.S. Food and Drug Administration expanded the patient population for use of ivacaftor to include patients with cystic fibrosis with relatively rare mutations in the cystic fibrosis transmembrane conductance regulator gene.

Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: the ivacaftor experience.

Cystic fibrosis (CF) is a life-shortening inherited disease caused by mutations in the CF transmembrane conductance regulator gene (CFTR), which encodes for the CF transmembrane conductance regulator (CFTR) ion channel that regulates chloride and water transport across the surface of epithelial cells. Ivacaftor, a drug recently approved by the US Food and Drug Administration, represents the first mutation-specific therapy for CF. It is a CFTR channel modulator and improves CFTR function in patients with CF who have a G551D mutation.

Inhaled fluticasone propionate by diskus in the treatment of asthma: a comparison of the efficacy of the same nominal dose given either once or twice a day.

Study Objective: In September 2000, the US Food and Drug Administration (FDA) approved the use of Flovent Diskus (FD) [fluticasone propionate; GlaxoSmithKline; Research Triangle Park, NC], which is an orally inhaled, dry-powder corticosteroid, for the maintenance treatment of asthma at dosages of 50 to 1,000 microg administered twice-daily. Once-daily dosage regimens did not receive approval. This article will detail six clinical trials, five of which incorporated comparative once-daily and twice-daily treatment arms of the same nominal dose of FD.

Improving the quality of adverse drug reaction reporting by 4th-year medical students.

Purpose: Evaluate whether a 15-minute lecture intervention will improve adverse drug reaction reporting quality on standard MedWatch forms.

Methods: Seventy-eight 4th-year medical students were randomized to intervention 'Group-A' or non-intervention 'Group-B' on the first day of a required five-day clinical pharmacology rotation. Group-A participants attended a 15-minute lecture on completing a MedWatch form with quality information considered by the Food and Drug Administration as critical to adequate adverse drug reaction reporting.

Global Ratings of Student Performance in a Standardized Patient Examination: Is the Whole More than the Sum of the Parts?

Standardized patient examinations (SPE) are widely used in medical education to assess skills that cannot be measured with written examinations. Trained actors termed standardized patients (SPs) are used to simulate patients with specific medical problems. SPs typically use behaviorally specific checklists and rating scales to evaluate examinees.

Olanzapine-induced rhabdomyolysis.

Objective: To report a possible case of olanzapine-induced rhabdomyolysis with concomitant lithium-induced pseudo-infarction electrocardiogram changes.

Case Summary: A 13-year-old white boy was admitted to the hospital with profound weakness and electrocardiogram (EGG) changes suggestive of myocardial damage after starting olanzapine and lithium. An adverse medication effect was not considered at the time of the patient's admission.

Visual hallucination and tremor induced by sertraline and oxycodone in a bone marrow transplant patient.

The authors report a case of probable serotonin syndrome caused by the coadministration of sertraline and oxycodone. A 34 year-old male patient experienced visual hallucinations and severe tremor after dramatically increasing his dosage of oxycodone while on stable amounts of sertraline and cyclosporin. Discontinuation of cyclosporin did not result in resolution of his symptoms.

Learning disabilities and medical schools.

Learning disabilities are a group of disorders that affect 10-15% of the general population and 3% of United States medical students. Medical students with learning disabilities are typically unaware of their disorder and may only learn of it when the pace of medical school curriculum overtakes what were previously successful study techniques. Despite the significant prevalence of this disorder, medical educators' awareness, understanding and provision of services for people with these disabilities are inadequate.

Quantification of human H1 histamine receptor mRNA from peripheral blood.

Study Objective: To develop a reverse transcription (RT)-polymerase chain reaction (PCR) technique to detect and quantify human histamine1 (H1) receptor mRNA in peripheral blood.

Methods: Primer pairs were based on the human H1 receptor nucleotide sequence. A competitive reference standard (CRS) was developed that used the same primers as wild-type mRNA but contained a 92-bp deletion.

The impact of the location and structure of an ambulatory rotation on cognitive knowledge and performance.

With the increasing shift to community-based ambulatory education, it is essential to gain a better understanding of the impact of these changes. To assess the impact of the location and structure of an ambulatory internal medicine clerkship rotation on cognitive knowledge and clinical performance, students were assigned to one of the following: (a) a multidisciplinary ambulatory clerkship (MAC), (b) the office of a community-based general internist, or (c) a university-based internal medicine ambulatory clinic. The groups were compared on the internal medicine clerkship examination and preceptor ratings controlling for introduction to clinical medicine course performance via analysis of covariance.

Creating and evaluating an independent ambulatory internal medicine clerkship.

Patient care is shifting from an inpatient setting to an ambulatory setting. Despite this shift, most internal medicine clerkships provide the majority of medical student training in inpatient settings or in university tertiary care clinics, which are not representative of patient care in a community setting. We created a separate ambulatory clerkship that used volunteer community faculty at local and distant sites.

Setting standards and defining quality of performance in the validation of a standardized-patient examination format.

Purpose: To evaluate whether written standards increase the reproducibility of a physician-facilitated station in an objective structured clinical examination (OSCE) designed to assess history, physical-examination, and communication skills.

Method: The OSCE examination at the University of Texas Medical Branch-Galveston consists of ten eight-minute stations. Six of these stations consist of three History, Physical-examination, Problem-solving, and Plan (HPPP) station pairs.

The reliability of medical student ratings of clinical teaching.

This study estimated the interrater reliability of medical student evaluations of clinical teaching. Data consisted of 1,570 ratings evaluating 147 faculty over a 4-year period in a 3rd-year internal medicine clerkship. The number of ratings a typical faculty member receives in a year was also calculated and used to extrapolate the standard error of measurement for data typically available to evaluate faculty at different time intervals.