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...

Robert Miller, the patient advocating for Hemispherx Biopharma Inc.'s Ampligen (rintatolimod) for chronic fatigue syndrome by going on a hunger strike in protest of the FDA's complete response letter, ended the hunger strike over the weekend. Progress was made, he said, as follows from a Facebook post. "1. Dr. Wanda Jones, who I trust and respect, in the Assistant Secretary of Health’s office has engaged with the Secretary of Health over the past days on the need for a serious commitment to address our unmet needs. 2. Dr. Jones committed to me that the Secretary’s office will be engaged in the...

About a week ago, my obituary arrived by email. But something related, which you might think is weirder, happened Tuesday afternoon, and all of it involves chronic fatigue syndrome (CFS), and Hemispherx Biopharma Inc.’s drug candidate for it, Ampligen. More specifically, it involves Bob Miller, 54, who is on a hunger strike. Miller stopped eating about the same time my obituary showed up, in protest of the FDA’s refusal to approve Ampligen (rintatolimod), the Toll like receptor 3 modulator for CFS that was given an unfavorable review by the FDA’s advisory panel in December, which I covered. I talked with...

When the FDA’s Rachel Sherman said the biggest challenge the agency faces with biosimilars is educating the public, she wasn’t exaggerating. An article in the New York Times shows just how big of a challenge it’s going to be. The article “Biotech Firms, Billions at Risk, Lobby States to Limit Generics” slams biotechs for encouraging states to adopt legislation limiting the automatic substitution of biosimilars, which the Times repeatedly called “generics.” As BioWorld Today reported, most of the bills being considered by states would require physician notification of what was substituted, enhanced recordkeeping and an opportunity for doctors (and, in...

A hunter walks into a bar and says, “Did you hear the one about the FDA?” “You mean the time it shot itself in the foot?” the bartender responds. “The left foot or the right foot?” another hunter asks, wiping the beer froth from his mouth. “What difference does that make?” the first hunter asks. “I want to know if it’s the one I’ve heard before. Or if this is a new one.” The first rule of thumb for hunters is to know what they’re doing. Otherwise, they might shoot themselves in the foot – or worse. The second is...