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...

What’s in a biosimilar name? That depends on whether the vial is half empty or half full. Seeing it as half full, many generic makers focus on the similarities between a follow-on and its reference biologic. Because of those similarities, they say biosimilars and their reference product should share the same international nonproprietary name (INN) – as is the practice with traditional generic drugs. That’s the argument the Generic Pharmaceutical Association (GPhA) made in the citizen petition it submitted to the FDA earlier this month. Used to cashing in on the success of a small molecule brand drug by sharing...

A few years back my brother-in-law Sam was starting a new job and was required to get a physical as part of the hiring process. A standard X-ray revealed a spot on his lungs and further testing confirmed it was cancer. The diagnosis came about two years after he’d quit smoking, but he had been a smoker for many years and was somewhere in his early 40s. Thankfully, because it was caught and treated so early, he is still with us today and doing great. But what if he hadn't had that physical? Earlier this week the U.S. Preventive Services...

So urgently did I think I wanted to read Ben Goldacre’s Bad Pharma, that I ordered it from Amazon’s UK site last November, unwilling to wait for U.S. publication – which happened just as I reached the end, polishing off the book’s afterword, called “Better Data.” Goldacre, it turns out, is as much bothered by the secrecy of data as by its quality, and he has also set up an activist website, where the public can petition for “all [clinical] trials to be registered, for all summary results to be reported, and for full clinical study reports [CSRs] to be...

Patients getting their four minutes to say how an experimental drug changed their life or gave them hope have become a routine part of nearly every FDA advisory committee meeting – as routine as the call to order or the conflict of interest statement. And as routine as the adcom moving on with little regard for what they’ve said. The public hearing speakers, along with the usually silent patient member of the panel, seem to have one function – to let the FDA check off the box for patient involvement. But patients with orphan diseases and their families deserve more...

Despite the regulatory approval of new antiepileptic drugs (AED) over the past decade and the expansion of indications deemed suitable for epilepsy surgery, individuals with intractable seizures – roughly one in three epilepsy patients – still face a dearth of treatment options. That void is all the more frustrating because epilepsy remains a debilitating condition for reasons beyond the clinical manifestations, often resulting in chronic unemployment or underemployment, social isolation and poor socioeconomic status. It's no surprise, then, that the prospect of a new treatment for people with medically refractory epilepsy would be greeted with great enthusiasm. As an adjunct...