ICH E10 provides specific guidance on the choice of control groups for clinical trials, but there are additional considerations for choosing a control group for a pediatric trial. In this article, the author looks at different types of control groups and the specific challenges that present when considering the design for a pediatric trial, such as the medical condition being treated, the potential patient population, suitability of comparators, and availability of real-wo...

The US Food and Drug Administration (FDA) on Wednesday finalized guidance on developing new types of buprenorphine depot products for medication assisted treatment (MAT) for opioid use disorder amid the ongoing opioid epidemic.   Buprenorphine, along with methodone and naltrexone, are the only drugs approved by FDA to treat opioid use disorder.   Buprenorphine MAT works by reducing patients’ withdrawal symptoms and blocking the euphoric effects of other opioid drug...

The Government Accountability Office on Tuesday issued a report calling on the US Department of Health and Human Services (HHS) to assess the effectiveness of efforts to expand the use of medication-assisted treatment (MAT) for opioid addiction. The report follows a House hearing last week in which FDA Commissioner Scott Gottlieb announced several steps the agency will take to promote the development and use of MAT. Background The report and Gottlieb's testim...

In testimony before the House Energy & Commerce Committee on Wednesday, FDA Commissioner Scott Gottlieb said that the next steps in efforts to combat the ongoing opioid crisis will be difficult, but necessary given the spread of the epidemic. "We'll need to touch clinical practice in ways that may make some parties uncomfortable," Gottlieb said, pointing to restrictions on prescribing and mandatory education for physicians as possible steps. "Long ago we ran out of stra...

The US Food and Drug Administration (FDA) on Thursday sent a letter to health care providers noting the receipt of dozens of reports for two different types of adverse events linked to fluid-filled intragastric balloons used to treat obesity.  “The first type of adverse event involves the fluid-filled intragastric balloon over-inflating with air or with more fluid (spontaneous hyperinflation) in patients’ stomachs, resulting in the need for premature device removal...

The European Medicines Agency (EMA) on Monday released new draft guidance on the development of Alzheimer’s disease treatments that would allow treatments to be evaluated in earlier disease stages before dementia sets in. The new guidance comes as EMA recognizes that the field of Alzheimer’s disease (AD) research and development “witnessed a recent paradigm shift in the diagnostic framework of AD which is now considered a continuum with a long-lasting presymptomatic phas...

The European Medicines Agency (EMA) has launched an initiative focused on existing patient registries, with the aim of seeing how they can be better used as a source of post-authorization data, and how they can help to establish new registries. Background EMA defines a registry as a system using observational methods over time to collect uniform data on a population defined by a particular disease, condition, or exposure. A disease registry includes patients bas...

The word leprosy brings to mind a gruesome disease that separates its sufferers from society and continues to strike fear into communities, much like Ebola. While the genome for leprosy has been sequenced and there is a drug cocktail that effectively cures the disease, its mode of transmission still is unknown. A disease that dates back through millennia, leprosy so far has eluded efforts to find the final piece of information that might eradicate it from the world per...

Patients and physicians alike often have lofty expectations and aspirations about the effectiveness of a prospective treatment. But, as a new article in the Annals of Family Medicine argues, those expectations contribute significantly to both the increasing costs of healthcare and avoidable harms suffered by patients. At the core of the healthcare cost crisis right now is an unavoidable fact, writes Steven Woolf, MD: The cost of obtaining healthcare, and in particular ...