How do doctors know if a new drug is really better than the ones already available?
This is an important question because new drugs come out every year and if one works better at, say, reducing the chance of a heart attack, then it's probably worth prescribing.
On the other hand a new drug will have no established track record, so there will often be a lot we don't know about it. Also, it will almost certainly be far more expensive than the drugs it's trying to replace. So it might cost our patients thousands of dollars for no added benefit. Worse, we know from studies that patients will often stop an expensive medication mid-course if they can't afford it. Might it be better in those cases to prescribe a generic medication that is almost as good?
This came up when a patient of mine who had just had a heart attack told me his cardiologist had put him on a new drug called Effient. It cost him $100 a month even with insurance. Heart attacks are caused by blocked coronary arteries and a common therapy is to use a tiny metal cage called a "stent" to hold the blocked artery open. The problem is that blood clots can form in these cages, causing new heart attacks. Drugs like Effient are prescribed to prevent this.
Another, more common drug for this is Plavix. Plavix is generic now (clopidrogel), cost as little as $10/month without insurance and has worked well for over a decade. But my patient told me his Cardiologist had run a test on him that showed that Plavix would not be as effective for him as Effient.
So, my patient is spending more than $1,000 a year extra to minimize his chance of having another heart attack, or so he's been told. But is that really what my patient is buying? Let's look at the evidence.
Plavix has been used since 1997 to help prevent blood clots from forming in arteries, and many studies showed that Plavix is especially good at preventing clots from forming in stents. But a few years ago it was discovered that up to one fourth of the people given Plavix didn't metabolize it well, so perhaps Plavix was less effective in these people.
But there's a tricky point here: "less" isn't always the same as "not enough." After all, two aspirin work as well for your headache as five. So studies were done to check if deaths, strokes or heart attacks occurred more often in patients treated with Plavix if they didn't metabolize it properly. Some studies seemed to show a difference, and others didn't. Still, the FDA decided to issue a warning for Plavix in 2010, stating that a drug other than Plavix should be used on the poor metabolizers.
Conveniently, at about this time, Effient was approved by the FDA. Effient is metabolized differently than Plavix so perhaps it was better for the poor metabolizers--no one wants to take a chance with heart attacks. Still, Effient has very little track record and costs more than many people on a fixed income could afford.
So before we get out the checkbook, we should probably make sure that Effient is really better. In fact, do we even know if it's as good as Plavix? No one had done a study to prove that switching people to Effient, even if they metabolized Plavix poorly, really helped. Finally, in November 2012, a study was published in the New England Journal of Medicine to address this.
There were over 2,400 people in the study. All of them had recently received stents for severe heart disease. Half of them were treated the traditional way (no blood tests to warn against Plavix resistance) and the other half were screened for Plavix resistance and treated accordingly. Both groups were followed for a year after receiving their stents.
And it didn't matter. It made no significant difference which treatment these people received. In fact, the group that was screened and had their therapies modified did slightly worse.
Now, the makers of Effient will tell you that this is only one study and we shouldn't jump to conclusions before more studies are done. That's good advice. It might still be that Effient is a little better and the next study will show that. But it might also be that Plavix is a little better and the next study will show that instead. And we've been using Plavix since the '90's, so it's very unlikely we'll get a nasty surprise about it. That isn't always true for the newer drugs.
There are a number of lessons that can be learned from this example and it's important for all doctors to consider the following whenever we write prescriptions:
-- New drugs are sometimes hugely beneficial, but they are always expensive and experimental (by definition). Several medications have been taken off the market after their release because of serious (and previously unknown) side effects. Some of our best drugs are older than my oldest patients. So before you try the expensive "new kid on the block," try to make sure it's fixing something that really needs to be fixed.
-- Almost all initial research on a new medication is done by the pharmaceutical company that makes it. Even with the best intentions, they're more likely to see what they want to see, and there are many examples where their intentions weren't the best. Often, new medications that did better in industry-funded studies don't do so well in follow up studies. Be suspicious of any study that's funded by a pharmaceutical company.
-- Prescribing very expensive medications when inexpensive alternatives are available doesn't just waste people's money. If someone can't afford their medication, they often won't buy it. No medication is effective if it isn't taken.
Remember, new and expensive doesn't always mean better. Sometimes that extra cost is just money for nothing.
Technology has placed mountains of medical information at our fingertips. Knowledge is power, even if your doctor thinks a little knowledge (yours, not his) can be a dangerous thing. The reality is that being able to learn things on our own alters the balance of power in the doctor-patient relationship. We can do our own research and ask our doctors more questions. We are getting second, third and fourth opinions online from other patients who have walked down these same illness paths before us. Heck, we can even sign up for alerts on our medications and be the first to know when a generic for the all-mighty (and all-expensive) Lipitor is available.
Remember that joke that asks, "What do you call the guy who graduated at the bottom of his medical school class?" The answer is "doctor." Let's just say it: Not all doctors are created equal. And as the boomer bubble swells into the next stage of our lives, chances are, we are going to insist on the best. We have formed online communities to recommend hotels, electronics and pretty much everything else. You can expect to see an uptick on online communities that recommend doctors and hospitals.
There are sites like PatientsLikeMe that hook you up with others who share your diagnosis. This site, with about 1,000 diseases covered, is especially noteworthy, says Harvard's Herzlinger because it just organized the first patient-run clinical test. Clinical tests have remained the purview of drug companies who hope to market a profitable product. In this case, it was a bunch of patients who wanted to test lithium's effectiveness in treating Lou Gerhig's disease (ALS). They found it wasn't, but the world learned in the process that patients can take things into their own hands and not wait for Big Pharma to figure things out for them.
Baby boomers like convenience, which is why the house call movement is picking up steam. Also watch for increased evening and weekend office hours by doctors. Pharmacies already stay open late; why not your doctor?
Walgreens just opened a two-story, 27,000-sq.-ft. downtown Chicago store that represents the future of pharmacy. It offers a health clinic offering a wide range of services including vaccinations, health tests, physicals and treatments for common illnesses and minor injuries. The pharmacy also features an "Ask Your Pharmacist" desk, consultation rooms, a Health Corner space to host health and wellness community events and Express Rx kiosks for swift checkout. (There's a sushi bar and mini-spa to boot.)
Telemedicine enables patients to "see" their doctors using video conferencing or services like Skype. It eliminates distance barriers and could bring a higher level of care to those living in rural areas. It also could just make patients' lives a whole lot simpler. The doctor calls at a pre-arranged time. You can download your glucose readings straight from your hand-held meter into the computer for him to see. Herzlinger says that a phone call appointment with the doctor is in the not-too-distant future for minor health events, which would cost $30 to $50, she said.
Keeping medical records online may have made the life of your doctor's office manager easier, but up until now, they haven't done much for patient health. The reason is that there are more than 2,000 IT systems in place tracking patients and those systems, unbelievably, don't talk to each other. Watch for a common IT system that enables all your doctors to have the same information on you. No more faxing test results between offices and having things lost.
Employers are already implementing programs that reward workers with prizes and low health care premiums for maintaining a health lifestyle, such as Virgin's Healthmiles program. A website called HealthPrize collects daily compliance data from users, verifies their prescription refills, and rewards them for adherence with prizes. In the future, expect to see your insurance premiums go down if you agree to have your retina scanned when you go to the gym and wear a device that measures how much oxygen you have flowing through your blood to make sure you aren't just sitting on that exercise bike reading a book.