Queue No More

Canada's surprising proposal for fast tracking new drugs to patients

From Canada, the land of long health care queues, comes a genuinely promising idea for speeding new medicines into the hands of patients—a fast track approval process called progressive licensing. Which is exactly what the U.S. needs. In 2007, the Food and Drug Administration (FDA) approved only 19 new drugs, the lowest number since 1983. Last year saw a minor uptick to just 24 new medicines.

In 2007, Health Canada, the Canadian government's lead agency on health care issues, launched a national discussion on how to transform the country's drug approval system. Currently, the Canadian drug approval process operates much like the FDA's version does. Pharmaceutical companies submit new drug applications to regulators who then set out criteria for securing bureaucratic approval of the drug—including a series of clinical trials to prove that the medicine is safe and effective.

This venerable drug approval model focuses on prohibiting sales until new products have been carefully tested and then approved by regulators. The chief goal is to keep unsafe drugs from reaching patients. As we shall see, regulators are much less worried about mistakenly rejecting safe and effective drugs.

To keep unsafe drugs out of the market, new pharmaceuticals must undergo a series of clinical trials. Phase 1 trials, involving a few subjects, evaluate how a new drug acts in the body and looks for dangerous side effects. Phase 2 tests the new drug for effectiveness in a few patients. Phase 3 expands the trials to confirm effectiveness and to obtain further indications about risks versus benefits. Increasingly, regulators now ask for Phase 4 trials as well, which are post-marketing studies that evaluate the treatment's risks and benefits once the public has begun using it. Rare side effects frequently don't show up until the drug has been used by hundreds of thousands of patients.

Part of the domestic slow down in drug approvals comes from the fact that since the 1980s FDA regulators have more than doubled the number of clinical trials required to get a new drug approved from 30 to about 70. This increase in trials has raised the cost of getting a new drug through the regulatory maze to over $1 billion, thus limiting the number of new drugs that pharmaceutical companies can afford to pursue.

This is where progressive licensing could rescue our creaky pharmaceutical regulatory system. While the final regulations in Canada are still being hammered out, one exciting possibility is that drugmakers could submit some of their new medicines for approval after completing relatively fast and inexpensive Phase 1 and 2 trials. Such trials would provide preliminary information about a drug's safety and efficacy. In exchange for this fast track pre-marketing approval, drugmakers would agree to greater post-marketing surveillance of drug safety. Which means that patients using a new drug would essentially enroll in the equivalent of a Phase 4 trial. This post-marketing information would allow companies and regulators to continually adjust the balance of benefits and risks over the life cycle of new drugs. One important caveat is that such post-marketing scrutiny must not become as costly as the current system of pre-market regulatory review.

Following Canada's preliminary framework, progressive licensing would initially apply just to drugs that address previously unmet medical needs and in those instances where obtaining extensive clinical information is difficult (such as drugs that treat only a small numbers of patients with rare diseases). But why stop there? Canada's free-market Fraser Institute thinks progressive licensing has the potential to fix the current over-regulation of all drugs. Every beneficial drug also has accompanying risks, after all; the question is who gets to weigh the risks and the benefits.

Currently, regulators make the crucial decisions about the risks and benefits of treatment. But this leads to unbalanced benefit-risk evaluations. Remember that from the point of view of pharmaceutical regulators it's far more important to avoid a single highly publicized death from a new drug than it is to worry about the hundreds of unknown patients who die because of delays in approving new life-saving therapies.

In a 2007 report, the Fraser Institute looked at how progressive licensing could be transformed into a more radically open system that allows patients and physicians to evaluate the benefits and risks of new therapies rather than relying on the judgments of timid bureaucrats. In the report, Fraser's Brent Skinner looked at how the risks of new treatments compare to the risks of alternative treatments that the public already accepts.

For example, consider the case of the over-the-counter pain reliever ibuprofen versus the new drug Vioxx. A novel painkiller introduced in 1999, Vioxx was withdrawn from the market because it was found to increase the risk of heart attacks. But further research indicated that many non-steroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen, also increase the risk of heart attacks among users.

Both types of medicine effectively relieve pain, but Vioxx had the benefit of reducing the risk of gastrointestinal bleeding, which NSAIDs exacerbate. But who should weight the risk of dying from heart disease versus the risk of dying from bleeding ulcers versus effective pain relief for rheumatoid arthritis? One 1999 study estimated that there are 103,000 hospitalizations and 16,500 deaths in the United States due to complications from NSAID-associated gastric ulcers. As Skinner notes, a patient who is at high risk from gastrointestinal complications might well choose to take the cardiovascular risks associated with Vioxx. Why not let patients and their physicians have this risk information and choose for themselves?

Progressive licensing could modernize the current process from one where bureaucrats grant extensive permission before new drugs hit the market into a system based on initial indications of safety and effectiveness followed by ongoing risk evaluations. This would give patients greater say in their treatment, allowing those who willing to accept a certain amount of risk early access to the latest treatments, while risk-averse patients and physicians could wait until further information became available. It would also increase the scope for private groups—perhaps along the lines of the Underwriters' Laboratories certification process—to evaluate benefits and risks.

Progressive licensing might turn out to be just what the doctors (and patients) ordered for reducing the backlog of new drugs awaiting the nod from overly cautious regulators.

Ronald Bailey is Reason magazine's science correspondent. His book Liberation Biology: The Scientific and Moral Case for the Biotech Revolution is now available from Prometheus Books.

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  • That Guy||

    Link is 404'd.

  • ||

    It's got a space in the URL. Go here until they fix it.

  • ||

    Crap. I mean here

  • ||

    PL,

    You better get off my turf, man. We are totally heading for a dance fight!

  • ||

    I just insulted you in the Lohan skank thread. I must be psychic today.

  • Brett Stevens||

    Why not test drugs on prisoners?

  • ||

    Back to the topic at hand, it figures that Canada will start getting their healthcare system straightened out, just as we dive from 60% socialized medicine to 100%.

  • Warty||

    Speaking of Lindsay Lohan, desperation is hi-larious.

  • ||

    Warty,

    Man, that's why I called it the Lohan thread, because I referenced that video. I recommended that Episiarch should date her, too. They'd be so cute together.

  • ||

    They'd be so cute together.

    Until they died of Mega-AIDS.

  • ||

    Not to mention that much psychotic behavior in one couple. . . .

  • ||

    I have already beaten mega-AIDS, ultra-syphilis, and space herpes. Lohan doesn't frighten me.

  • Jeff P||

    But the real question is how many new fake diseases were invented over the same period?
    Things went downhill after Restless Leg Syndrome...

  • ||

    Jeff P,

    It's a phase. These interludes with no major breakthroughs in curing real diseases require that pharmaceutical companies stage breakthroughs in curing fake diseases. It's kind of like florists pushing fake holidays.

  • Jeff P||

    So your saying I'm stuck with my therapy for Twitchy Cock, which consists of beating it like it owes me money and sticking it in wet places.

  • phalkor||

    I bet you can get a medical marihuana recommendation for restless leg syndrome.

  • mi coño es su coño||

    "But the real question is how many new fake diseases were invented over the same period?
    Things went downhill after Restless Leg Syndrome..."


    OH MY GOD I GOT THE CRAZY LEGS!!!

  • ||

    No, the San Francisco 49ers got Crazy Legs.

  • Warty||

    Man, I knew my decision to stop reading every single thread on the blog would end up with me making a monkey out of myself. Fuck. *kicks stone*

    Speaking of monkeys...

  • Warty||

    I have the restless leg thing, and I can attest that it's AWESOME. I totally have a ready excuse any time I want to kick my girlfriend when she's asleep.

    KICK! "Ooops, sorry babe. You know how twitchy my legs get when you don't feel like blowing me." KICK! KICK! KICK!

  • Jeff P||

    Restless Leg would make a great excuse for goosestepping...

  • ||

    Which means we're taking too long to get life-saving new therapies to patients.

    It could mean that...

    It could also mean that there haven't been any significant drug related breakthroughs.

    last I checked, many drug companies were looking at ways to remix existing patented drugs to get a "new" patent because their pipelines were quite flimsy and not very promising.

    I know PFizer's (which I own stock in) pipeline is currently pretty weak, and they have some patents expiring soon and are looking for new ways to generate revenue.

    Maybe the pharma industry just isn't very successful as of late?

  • ||

    So your saying I'm stuck with my therapy for Twitchy Cock, which consists of beating it like it owes me money and sticking it in wet places.

    You've got it too? Jeez it's an epidemic!!

  • ||

    CT,

    I think that's mostly correct. We're probably on the verge of several important breakthroughs, but verge don't pay the bills this year.

  • ||

    last I checked, many drug companies were looking at ways to remix existing patented drugs to get a "new" patent because their pipelines were quite flimsy and not very promising.

    Yah, there's a lot to that, but you need to ask the next question:

    Why are drug companies focusing on extending existing patents with drugs that have already been through the brutally long and expensive approval process?

    Could it be that they are responding rationally to the incentives that we have created, namely, a brutally long and expensive process for new drugs that drastically skews the ROI on new research?

    Just askin', is all.

  • ||

    RC,

    As someone in the biotech industry, I would never defend the number of hurdles we have to go through, because it simply boggles the mind how much valueless work goes into getting a new drug approved, but...

    The concept of taking "new science" and turning it into a saleable product is pretty damn labor intensive in and of itself (i.e. without the silly approval process.) That's part of the reason that regulations that would be unthinkably onerous in most industries are met with a "well, that's just one more straw on the back" in the drug development world.

  • ||

    Legate Lamar,

    It's a wonder any science gets done in the field. And it's likely to get worse.

  • ||

    Oops, make that Legate Damar.

  • The Angry Optimist||

    Legate Lamar, leader of the Kardashian Union.

  • ||

    TAO,

    Heh, heh.

  • ||

    I have restless truculent libertarian syndrome. Some of you may have noticed.

  • Jeff P||

    Syndromes in the works include:
    Turgid Chode
    Quivering Bowel
    Asymetrical Taint
    Eighties Hair
    Swank Hipster
    Caustic Harpy

    Actually those all sound like H&R commenter aliases...

  • Libertarian Keyboard Warriors||

    Aw, that's cute. Ronnie even added "land of long health care queues," just in case the extremists were growing suspicious.

    Hey, at least it keeps him away from yet another muddled, Global Warming piece.

    "I have restless truculent libertarian syndrome. Some of you may have noticed."

    Oh, we've noticed. It's called "immaturity." Children demonstrate ego complexes similar to yours. It's the defining characteristic of your behavioral type. In other words, you're a cliche.

    Also, adding "liberty," or "freedom" to your handle on a Libertarian site is like Bill O'Reilly claiming that you've just entered a "No Spin Zone." Yes, I know, the deck must be stacked carefully.

    Ultimately, you're easy to pick apart because your outlook is so simplistic.

  • economist||

    Libertymike,
    I'm in the early stages. As someone who's lived with it longer, can you give me some advice on coping?

  • economist||

    So anyway, I'm pretty sure LKW, Run Paul, Markets are Magical, and a few other assorted random assholes are in fact all the same person.

  • Libertarian Keyboard Warriors||

    Watch me suck myself off!!!

  • ||

    Libertarian Keyboard Warriors,

    What a lucid and compelling argument!

  • economist||

    "Ultimately, you're easy to pick apart because your outlook is so simplistic."

    Which must be why, rather than schooling us with your amazing intellect, you'd rather throw random insults.

    Don't be scared to use your real handle, Edward.

  • economist||

    Dammit, ProL, you beat me to the punch!

  • Libertarian Keyboard Warriors||

    "Could it be that they are responding rationally to the incentives that we have created, namely, a brutally long and expensive process for new drugs that drastically skews the ROI on new research?"

    Oh, of course Dean. We get it. Every big business failure to advance comes back to the government. It could never be some other rather mundane factors like a lack of foresight, or a meaningful drug therapy break through, or most corporation's favorite malfunction: A general lack of creativity, and proper management.

    It's obviously the government's fault. I'm sure you won't accept any other explanation.

    I'm just saying.

  • economist||

    I will not feed the troll.
    I will not feed the troll.
    I will not feed the troll.
    I will not feed the troll.

  • Libertarian Keyboard Warriors||

    "Which must be why, rather than schooling us with your amazing intellect, you'd rather throw random insults."

    Oh, enough. It's common practice around here for you, and other Libertarians to shift into ad hominem mode when nuance enters the frame.

    You, and many others on here, have been indulging the very mindset that you are lashing out against now. I'm simply giving you a taste of your own medicine, and obviously you don't like it. That, alone, should tell you something.

    Anyway, do I have to dig your sorry ass up in the archive for maximum effect?

    Interestingly, my lack of substance certainly inspired you take time out of your busy Libertarian schedule that I'm certain is spent polishing your overman complex.

    Finally, I'm not Edward. Everyone who takes an interest in highlighting the extent of the Libertarian personality disorder is not simply the same person under a different screen name. Again, that's a fairly egotistical way to approach the matter. No surprises there.

    Finally, who says I'm disputing everything that is said on this thread? Sometimes holding a mirror up to a person in order to reveal to them the sheer absurdity of their thinking, is enough.

    Hey, it was worth a shot.

  • Libertarian Keyboard Warriors||

    "I will not feed the troll."

    I'm not sure that anything will stop you from feeding your ego.

    With each reply you reveal your soreness.

  • economist||

    "You, and many others on here, have been indulging the very mindset that you are lashing out against now."

    And what mindset is that?

  • Libertarian Keyboard Warriors||

    "So anyway, I'm pretty sure LKW, Run Paul, Markets are Magical, and a few other assorted random assholes are in fact all the same person."

    Wow, you must have a catalog of all of your online enemies, and their screen names. But, yeah, they're comments are meaningless, right?

    Like a business that has grown to big to be managed effectively, you lack priorities.

  • Libertarian Keyboard Warriors||

    "And what mindset is that?"

    I assume you're an "economist," but I also assume that they taught people like you how to read as well. I suggest that you go back and do that.

    Maybe words just aren't your game.

    Anyway, we're almost twittering.

  • economist||

    No, really, LKW, what mindset is it? I'd really love to know.
    And no, I don't have a catalog of my online "enemies". It's just amusing to note that there's either one person with a penchant for posting under several different handles or several people with nothing better to do than post "OMG teh libertards are STOOPID!"

  • Paul||

    Also, adding "liberty," or "freedom" to your handle on a Libertarian site is like Bill O'Reilly claiming that you've just entered a "No Spin Zone." Yes, I know, the deck must be stacked carefully.

    Right, kind of like "Center" for "Science" in the "Public Interest" or "Democracy" Now! or Employee "Free Choice" Act.

    This is fun!

  • economist||

    "Maybe words just aren't your game."

    I know I could never hope to be master wordsmith like you.

    "Like a business that has grown to big to be managed effectively, you lack priorities."

    You may have joe's memorial lawed yourself. It's "too".

  • economist||

    Paul,
    The troll is busy showing his massive intellect through grammatical errors.

  • Libertarian Keyboard Warriors-||

    I'm not Edward, even though I sound exactly like him!

  • economist||

    Perhaps LKW is an embittered joe who's come back to wreak his vengeance on the libertarian posters for not appreciating his wit.

  • economist||

    Oh, please, LKW, don't destroy me with you mad debate skills. I think it would kill me *sarcasm*.

  • economist||

    "You, and many others on here, have been indulging the very mindset that you are lashing out against now."
    I must admit I'm confused. Does LKW mean that there was a certain mindset that we had here, and now others are copying it, and that makes us angry? That doesn't really make sense. Perhaps...no that doesn't work either. It seems far more likely that clarity isn't LKW's game.

  • ||

    libertarians are childish and immature and...and selfish! and also immature and childish and...well they just repeat the same old tired cliches and are selfish !

  • jtuf||

    I like the Canadian proposal. Plenty of people who can afford it make sure to go to cutting edge research hosptials if they have a difficult illness. Part of the attraction of these hospitals is the possibility of joining a drug trial if that drug is the best one for their disease. Why not let all patients have the benefits of trying out a drug early?

  • Ben||

    > Why not let patients and their physicians
    > have this risk information and choose for
    > themselves?

    From a liberty standpoint, no reason.

    From a practical standpoint, because lawyers, legislators, and judges would turn it into a huge clusterfrack. Just as they have done with everything else even remotely related to informed, consensual personal choice.

  • Libertarian Keyboard Warriors||

    "No, really, LKW, what mindset is it? I'd really love to know."

    Okay, but I expect some kind of reimbursement.

    The mindset that you, and others on here seem to loath, yet perpetrate yourselves, is expressed through strawmen arguments, ad hominem attacks, either/or fallacies, and a host of other abuses of reasoning that you readily chide your opponents about. In other words you're a classic hypocrite. There's no one here to keep you honest.

    You know as well as I do that this is the case. However, existing in an echo chamber has left many of you ill prepared for the most basic arguments.

    Again, I've been around this site for years, and the archives would be an easy place to start. You're behaving better than you normally do. Don't push it.

    "libertarians are childish and immature and...and selfish! and also immature and childish and...well they just repeat the same old tired cliches and are selfish !"

    Those traits cover a lot of behavioral ground. Besides, no one ever said that all cliches are inherently untrue.

    If you are being labeled with cliches, then it is more than likely due to the fact that you are a cliche. But educating a fully entrenched Libertarian on matters of the self is like trying to teach a retarded kitten how to do a back flip.

  • nike shox||

    is good

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