Approval of a drug for clinical use requires production of data on efficacy and safety through submission of results from randomized controlled trials (RCTs), in which the new molecule is usually compared with placebo (or an active comparator) for a set of outcomes that will serve as the basis for the drug's indications. These indications are crucial, because drugs are approved on the basis of ...

INTRODUCTION Methylphenidate is a piperidine derivative structurally and pharmacologically similar to amphetamine. Methylphenidate is indicated for Attention Deficit Hyperactivity Disorder (ADHD) in children aged 6 years of age and over when remedial measures alone prove insufficient. In adults, its indication, except in narcolepsy, is not defined. Methylphenidate received regulatory approval a...

O December 3, 2012, a three­ judge panel of a U.S. appeals court took a controversial leap toward what some fear will be license by the courts to invalidate a host of state and federal regu­ lations, including some applica­ ble to health care. In recent years, the Supreme Court has broadened the reach of the First Amend­ ment, defining “protected speech” in such a way as to curtail or eliminate...

BACKGROUND Off-label use of (orphan) medicinal products for (rare) diseases is quite common but not underpinned by clinical studies to confirm efficacy and safety. No risk-analyses by regulatory agencies are carried out. The objective of this study was to map off-label use of orphan medicinal products in Belgium in terms of attitude towards off-label prescribing, factors influencing off-label p...

Fluoride varnishes are fast becoming the standard of care as topical fluoride treatments. Fluoride varnishes still await approval from the FDA for use as caries preventive agents. In the meantime, their use for such purposes is considered "off-label." This article highlights the efficacy of fluoride varnishes as caries preventive agents and introduces some of the commercially available fluoride...

Behavioural and psychological symptoms of dementia (BPSD) occur in most patients with dementia. They cause great suffering in patients and caregivers, sometimes more so than the cognitive and functional decline inherent to dementia. The clinical features of BPSD include a wide variety of affective, psychotic and behavioural symptoms and signs. The causes and risk factors for BPSD are multiple a...

, number 5 , May 2016 :e 20160205 In a highly controversial decision, the Food and Drug Administration (FDA) approved, in August 2015, extended-release oxycodone for use by children between 11 and 16 years old with “pain severe enough to require daily, around-the-clock, long-term opioid treatment for which alternative treatment options are inadequate.”1 For the first time, doctors are provided ...

Not just drug manufacturers, but patient advocacy groups too, are pushing the FDA to ease its prohibitions on the dissemination of off-label medication (and medical device) information. The situation with regard to Genentech’s Avastin and Lucentis (bevacizumab) is just one reason why. Lucentis is indicated for the treatment of the wet form of age-related macular degeneration (AMD), and Avastin ...

The Second Circuit's December 2012 decision in United States v. Caronia striking down the prohibition on off-label marketing of pharmaceutical drugs has profound implications for economic regulation in general, calling into question the constitutionality of restrictions on the offer and sale of securities under the Securities Act of 1933, the solicitation of shareholder proxies and periodic rep...

on YouTube about using one of its products and the content prompts questions about off-label use, this would be a solicited request. The FDA may consider solicited requests evidence of a firm’s intent that a drug be used for off-label purposes. 2. The guidance also distinguishes between non-public and public unsolicited requests and recommends appropriate responses. For example, a telephone cal...