You have to hand it to the cancer lobby. It really has the U.S. federal government wrapped around its little finger. Some will say I'm overstating the case, but is that really a solid argument? Let's examine the evidence. Mortality, economic impact boxes? Not checked First, let's look at overall mortality in the U.S. According to the National Center for Health Statistics, heart disease was responsible for more than 614,000 deaths in 2013, while the various cancers claimed fewer than 592,000 lives. If you add cerebrovascular deaths (more than 133,000) into the...

As the saga of Martin Shkreli continues to unfold in all its obnoxious glory, with every tweet and smirk signifying the former biopharma CEO’s apparent aspirations to a level of sheer jerkiness heretofore unseen on planet Earth, there’s one question that I – and probably a lot of other folks – can’t help but ask: Is this guy for real? I mean, seriously? From his gleefully unapologetic, 5,500-percent price hike for an in-licensed, generic drug, to his now-infamous gray-hoodied perp walk following his arrest on charges of securities fraud, to his juvenile Twitter taunts – not to mention his outlandish...

Duchesnay Inc.’s 15-minutes of fame for its morning sickness drug arguably turned into a few hours’ worth by time the drugmaker complied with an FDA warning letter demanding a corrective ad to offset the omission of risk information in paid social media endorsements by She-Who-Must-Not-Be-Named in the serious biopharma world. The first 15 minutes for Diclegis (doxylamine succinate and pyridoxine hydrochloride)came with You-Know-Who’s tweets and posts to her millions of followers on Facebook and Instagram. That fame increased exponentially when the FDA handed down the warning letter last month, daring to name the infamous reality TV maven and setting mainstream...

Patients have come a long way to get a say in the drug development and approval process. But there’s still some ground to cover if they’re going to move from the passive subject role to a 50-50 partnership that reminds researchers, drugmakers and regulators that, “Hey, this is all about us!” While major drugmakers have been hiring senior-level staff to connect and interact with patients and to incorporate the patient perspective into their R&D for a while now, the regulatory field is making slim – albeit positive – advances in that direction. In the past, patients often testified during the...

It’s not every day that drugmakers get a chance to take on an entirely new market. Biosimilars are offering that opportunity. And hundreds of drugmakers, research institutions and governments are hoping to make the most of it. The result is more than 700 follow-on biologics (FOBs) already approved or in the global pipeline, according to a new report by BioWorld. Those entering the field are as diverse as the FOBs they’re developing. Biologics pioneers, big pharma and generic drugmakers are being joined by “pure-play” start-ups, conglomerates, public-private consortia, contract manufacturers, contract research organizations, government agencies, nonprofits and research institutions. Some...

For more than five years, the FDA was criticized for failing to provide clear guidance on how drug developers could use social media without running afoul of its marketing restrictions. Last month, the agency finally came through with two crucial draft guidances. A webinar Thursday afternoon aimed at providing more color around the social media regulatory framework could only be described as an #EPICFAIL. The webinar, for which registration reportedly closed early due to overwhelming demand, suffered problems from the get-go, with many registrants unable to log in at all. Almost immediately, slides started crashing, prompting the agency to pause...

Including the patient voice in the drug approval process could produce a fundamental shift in the risk-benefit equation of a new drug. But first, the FDA has to figure out how to do it, and that may take a broader societal discussion, FDA Commissioner Margaret Hamburg said. Speaking at the annual meeting of the Pharmaceutical Research and Manufacturers of America, Hamburg ticked off a few issues that we as a society must address. For instance, in the absence of a cure, patients with chronic or terminal diseases want a better quality of the life they have left. But the FDA...

Before its unfortunate drop in quality in its last few seasons, the medical drama House was one of my favorite shows, with its title character, a cranky, possibly drug-addicted doctor solving diagnostic riddles and saving lives with Sherlockian brilliance, and quite often breaking every rule in the book to do it. One episode in particular had House facing off with billionaire investor Vogel, who had thwarted his attempt to enroll a very sick patient in a clinical trial without mentioning to the trial investigator that she wouldn’t fit the criteria, namely having just undergone a C-section. They have an impassioned exchange:...

U.S. and British government agencies are moving toward enabling what would be the first clinical trials of what is, in effect, human germline engineering – genetic modifications that would be passed down through the generations. The modifications would not be made to nuclear DNA. Instead, the procedure being considered is oocyte modification – creating an egg cell with the nuclear DNA of one woman and the mitochondrial DNA of another. The goal would be to allow women with mitochondrial diseases to have babies that share their nuclear DNA, but are not at risk of inheriting their mitochondrial disease. The UK...

As I write this, I’m sitting in the University of British Columbia/Vancouver General Hospital (UBC/VGH) Eye Care Centre, where my husband, Chuck, is completing post-tests at the conclusion of a six-month study on prosopagnosia, otherwise known as face blindness. The condition isn’t treatable with drugs – not yet, at least – but it’s nonetheless disabling, prompting researchers at several centers in North America and Europe to work collaboratively and seek to help patients carry on with their lives. Clinical studies of prosopagnosia have, so far, informed researchers more about the causes and structural manifestations of the disease than about potential...