FDA novel drugs:
Source: FDA Voice
Source: FDA Voice
There’s some debate as to whether cancer patients should have drugs that are proven to work, or whether instead they should have access to as many drugs as possible that might work. At least that’s what we’ve gathered from a recent study identifying that more than half of cancer drugs approved by the EMA between 2009 and 2013 showed no benefits for either survival or quality of life. While that’s not extremely terrible considering, 1) the EMA focuses on timely patient access to new drugs and, 2) trials focusing on survival outcomes are expensive and (more importantly) time-consuming, one has to wonder if we can’t do better than half. Otherwise we’re just provoking cancer patients to the point of rampage.
What do Michael J. Fox, Billy Graham and Billy Connolly (funny dude, swears a lot) have in common? Well, two things. One, Parkinson’s Disease and two, they finally have a new treatment option. That’s right, the US FDA has approved the first new Parkinson’s treatment in over a decade. Europe gave the thumbs up in 2015, but the FDA has been dragging its feet since 2010. The reluctance to green light it had something to do with abuse liability and withdrawal effects. Apparently, these aren’t issues in 2017. Patients receiving Newron’s drug, Xadago, experienced more “on” time (less uncontrollable, involuntary movement) when taken with the current treatment. With expected annual sales of $450M and heavy US contribution, expect to see a US celebrity face put to it soon.
Fresh off the clock, ex-FDA commish Robert Califf recently vented a few concerns he has with speeding up drug approvals. #1: Faster approval does nothing to address popular concern for the cost of drugs. Pharma will still say they must recoup hefty development costs and people will still not trust that explanation. #2: Are speed and safety at odds? As Califf puts it, “Declaring a drug safe after very little information is treacherous.” All drugs have risk and this risk is only uncovered through evidence. Check out 22 case studies where the bottom fell out between Phase II and III. And #3: Drugs can’t be approved faster if the FDA needs more FDA-ites – employees. We’re not sure what they call themselves.
Beginning in April of this year, NHS England will have the ability to withhold patient access to select approved medicines, according to The Times. Even if NICE considers a new drug of appropriate value, it may be withheld or rationed if it costs the NHS more than £20 million per year. While this might not be too alarming for those seeking help to lower cholesterol, the prospect of a drug being withheld for a rare disease – even if it is considered good value – is no bueno. Or what if one of the many potential Alzheimer’s drugs in development works? The size of the addressable patient population could make the £20 million threshold look puny. Stay tuned for more unpopular decisions to come.
According to QuintilesIMS, total consumer drug spend is estimated to top $1.5 trillion by 2021. That’s a lot of green. It’s a brave new healthy world out there and while the cash may well roll in for pharma companies, it’s not all fun and games. In the words of the not-so-immortal Biggie Smalls, “mo money mo problems.” While much of it will surely go toward development of the projected 45 new launches per year, these kinds of figures will not go unnoticed in popular media and political circles. Prepare for more pricing pressure and calls to demonstrate product value through health outcomes studies, comparative effectiveness research, and numerous other versions of payers telling pharma to “prove it.”
Maybe you should talk to your doctor to see if a HealthyDose™ of biosimilars is right for you. Side effects include: euphoria caused by an acute awareness of feeling yourself getting smarter, accusations of being a know-it-all by your colleagues, and more euphoria due to objectively knowing you are in fact smarter than your colleagues.
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That’s what Médecins Sans Frontières / Doctors Without Borders, WHO, and other experts are discussing. The West African Ebola crisis led to a push for accelerated access to treatments, arguing that using a potential therapy with promising results in animal studies is OK during a major public health crisis. Meaning, even if the FDA hasn’t approved it for use in humans, patients should still have access (in some circumstances). MSF’s article in The Journal of Medical Ethics seeks to define these “exceptional circumstances” and the goals of using unproven therapies. This could be a tipping point for research ethics. Good news, since there’s still no approved treatment for Ebola.