How do I understand what tests, procedures, medications can be predicted to be helpful for my circumstances, and avoid those that may be redundant, un-actionable, and potentially harmful?

We bring our problems to our doctors and we expect them to figure out what’s wrong and fix it. The figuring out process will probably involve diagnostic tests, and we expect that any doctor worth their salt will write a prescription or recommend a procedure as part of the fix.   And our doctors always know what’s right, don’t they?  And they always give us the best fix, don’t they?

Well … no. Not always.  At least not without our help.

 

To increase the probability of receiving the most effective and highest value care available, you need to collaborate with your doctor ask them the right questions.

The approach represented here combines several concepts that have achieved various levels of adoption; providers playing The New Game have generally embraced these concepts, while those playing The Old Game sometimes consider them controversial or have resisted them.

They include:

Collaborative vs Paternalistic relationships with your doctors, including Shared Decision Making and Precision Medicine.

Our doctors will continue to have unique knowledge and experience that we need to stay healthy or to understand our conditions and evaluate treatment options. Our responsibility as patients is to seek and foster a relationship where they appreciate that these are our decisions to make in the context of our own self-interest; our goals, values, beliefs, and circumstances.   Then, if we’re diagnosed with a condition, we want to understand what all of our treatment options are, which may be most effective relative to our goals, and what our physician’s advice is. We want our physicians to understand us and treat as complete beings, including our values and beliefs. Not simply as conditions. Then it’s up to us to decide which treatment option to pursue and to comply with that treatment until we either achieve our goal, or, if we don’t, try other options until we do.

Informed Consent.

Done well, a complete informed consent process consists of seven elements:

Discussing the patient’s role in the decision-making process.

Describing the clinical issue and suggested treatment

Discussing alternatives to the suggested treatment (including no treatment)

Discussing risks and benefits of the suggested treatment (and comparing them to the risks and benefits of alternatives)

Discussing related uncertainties

Assessing the patient’s understanding of the information provided

Eliciting the patient’s preference (and thereby consent)

When done poorly, the process and documents for informed consent can intimidate us, hide treatment options from us, and cause us to withdraw from our role in our own treatment decisions.  Poor informed consent can also obligate us financially to unplanned, unnecessary or unwanted procedures – often excluded benefits or work from out of network providers – which can result in financial devastation.

Download one or more of these to move the conversation with your doctor along …

One Page Review of Treatment Options

Questions to ask regarding diagnostic tests or procedures

Questions to ask regarding prescription or OTC medications

Questions to ask when evaluating therapeutic options or procedures

Want a second opinion?  If your treatment plan includes a surgical procedure, is anticipated to be expensive, or you’re just not sure, a second opinion might give you more confidence in your decision.  Try The Cleveland Clinic’s My Consult.  If your not sure, read about another patient’s experience in a recent Washington Post article or check out other options at various prices here.

A lot of really smart people have been doing a lot of really good work on FAQ’s, treatment options, comparative effectiveness research, patient satisfaction, and other information are already available for the specific test, medication, therapy or procedure you’re considering. Take a peek:

Option Grid is a series of decision aides – brief, easy to read tools to help patients and providers compare alternative treatment options.  Brought to you through a collaborative under the Dartmouth Institute for Health Policy and Clinical Practice.

PatientsLikeMe is ‘…committed to putting patients first, by providing a better, more effective way to share real world health experiences in order to help yourself, other patients like you, and organizations that focus on your conditions.’   Users can learn from others by comparing treatments, symptoms and experiences with others similar to themselves. They can also share experiences and give and get support to improve their lives.

Smart Patients is ‘an online community where patients and caregivers learn from each other about treatments, clinical trials, the latest science, and how it all fits into the context of their experience’.

Iodine helps ‘compare treatments, find to-rated medications, and read people’s experiences for hundreds of medications’.

Worst Pills, Best Pills is an independent second opinion on more than 1,800 prescription drugs, over the counter medications, and supplements.

Choosing Wisely is an initiative of the American Board of Internal Medicine Foundation in partnership with Consumer Reports. Resources include patient friendly materials to educate patients about what care is best for them and the right questions to ask their physicians.

Your Health: Fact, Not Fiction. An information sheet on how to be a smart patient and live longer.   Included are general guidelines for High Value Care, which includes ‘To Do’ lists for preventative care, as well as ‘To Question’ list of tests and procedures that may not add value to your diagnosis or treatment. From the Choosing Wisely project in conjunction with Consumer Reports and the American College of Physicians.

Advice for Caregivers: Treatments and Tests for Seniors. Recommendations regarding cancer; heart disease and ICD’s; diabetes and high blood sugar; dementia and antipsychotic medicines, long term medicines, and feeding tubes; sleep issues; appetite stimulants and high calorie supplements; social support and hand feeding; urine tests, urinary tract infections, and avoiding antibiotics; chronic pain; screening tests; and palliative and hospice care. From the Choosing Wisely project in conjunction with Consumer Reports and the ABIM, in English and Spanish.

 

Examples of good questions to ask:

“Why are we doing this diagnostic test, and what will you do with the results?  Does the additional diagnostic information merit delaying treatment?  And what are my options for achieving the same diagnostic information?  What are the chances of negative side effects or harms for each of my options?”

“What are my alternatives, and how can I decide what’s best for me?

What if I do nothing?

What if I change my lifestyle habits?

What are my medical alternatives?  For prescription meds, is there a generic or OTC alternative?  For surgery, is there a medical management alternative?  If not, is there a minimally invasive alternative?”

“For each of my treatment options;

What are the anticipated benefits, and for every 100 patients treated, how many more experience the anticipated benefits compared to patients who do nothing? Is there Numbers Needed to Treat (NNT) data available?

What are the possible harms or side effects to me, and for every 100 patients treated, how many more experience the harms side effects compared to patients who do nothing? Is there Numbers Needed to Harm (NNH) data available?

Are there any predictive indicators to determine whether I’m more likely or less likely to experience the anticipated benefits or side effects? Family history? Past medical history? Genetic screening? Age, race, sex, or ethnicity?

Are there any known uncertainties?

Are there comparative effectiveness studies available? If not, what do other studies say will be most effective?

With all of the above, and understanding my own personal beliefs, what would you do if you were me?”

To illustrate the difference these questions can make, bear with me through two (admittedly hokey) stories to illustrate the value of these questions.

 

Here is the way most of us think the game is works …

Shane K. walks into his primary care physician’s office. When his turn comes and he’s face to face with Dr. Sarah, he explains that he has what he thinks is a chronic condition; “Doc, I’m always thirsty”. Dr. Sarah asks a few questions about Shane’s family and past medical histories, does a quick physical exam, then turns to Shane with confidence and says, “Good news and bad news, Shane. The bad news is you were right – you do have a chronic condition, and it’ll be with you for the rest of your life. But the good news is that we have just the thing for you, and if you take it, your symptoms will go away. Here’s your prescription”. She rips it off the pad in a flourish, asks if he has any questions, flashes a quick smile, then purposefully moves off to the next exam room. Shane looks down at the prescription pad, and it simply says “Drink 1 pint + of H2O 3x daily”.

Shane shrugs, figures Sarah knows what she’s talking about – after all, our doctors are our best and brightest, right? – and leaves the office. Two weeks later Shane runs into Dr. Sarah at the youth soccer fields where both of them have young ones playing. “Dr. Sarah”, Shane gushes, “I can’t thank you enough! It worked! I feel so much better. You’re the greatest!”. Shane’s belief in his doctor, and overall faith in our medical system, have been reinforced. Sarah feels good, because she has once again made a difference in someone’s life and has fixed what ailed them.

Several months later, Shane has another problem and heads back to see Dr. Sarah. He’s not quite sure Dr. Sarah can help personally with this one, but he doesn’t know where else to turn. Once in the office, he explains; “Dr. Sarah, I’m hoping you can help me out. It’s … it’s …” he has difficulty speaking candidly. “Go ahead”, says Sarah. “You can speak freely. Everything we say is confidential here”. “Ok”, stammers Shane. “It’s my car. It’s just so … dirty!”.   Sarah grins. “You’d be surprised, Shane, how many people have the same problem. The good news is that we can get to the bottom of this pretty quickly with a simple diagnostic test using the latest technology. It’s called a ‘digital photograph’. I’ll place the order now, and a technician should be in touch in the next couple of days.” Shane’s confidence in Sarah is high, so even though his problem isn’t fixed yet, he feels better.

Several days later, he gets the call. “Shane, this is Jim. Dr. Sarah referred me to you for your digital photograph. I’ve got slots open this afternoon, on Wednesday, and again next Monday. When would it be convenient for you to bring your car over?”. Shane is anxious to get to the bottom of this, so he rearranges some work appointments and goes to see Jim that afternoon. On arrival, Jim checks Shane’s insurance, sees that he is ‘in network’ and that digital photographs are an included benefit, but also notes that Shane hasn’t yet reached his deductible so he collects the fees for the digital photographs (Jim believes 4 will be necessary) from Shane before proceeding. Shane then follows Jim out to the parking lot, where Jim whips out his very expensive looking professional digital camera and takes his pictures.

The next day, Shane gets the call. “Shane, it’s Dr. Sarah. I’ve gotten the photos back from Jim and they confirm my diagnosis. You have a dirty car. I can’t help you here. We just don’t have the equipment. But I know just what to do. You need a common procedure performed by a specialist. It’s called a car wash. Once you have one done, your car will be nice and clean again.” Shane is relieved. We know what the problem is! He knew he could count on Dr. Sarah.

Off Shane goes to the car wash. Once again, when he arrives, the car wash attendant checks to see if he’s ‘in network’ and is an included benefit, which he is. But once again, Shane hasn’t met his deductible so he pays out of pocket. Afterwards, Shane feels better. His car is clean. But he also has some nagging questions. Is my car as clean as it could possibly be? Did I pay too much for my car wash?   Should I have had it washed somewhere else?   But he trusts Dr. Sarah. So why should he feel uncertain?

 

Here is the way it should work …

The next month, Sarah spots Shane at the weekend soccer tournament and seeks him out where they can speak in private. “How’s the car, Shane?” she asks in a hushed voice. “Oh, it’s pretty good. I appreciate your help”, replies Shane. He’s genuinely grateful, but his nagging questions remain. “Listen, Shane,” continues Sarah. “We’re going to have a lot of down time during the tournament this weekend, so if you’re interested, I’d like to recap our two recent encounters and talk about how they might have gone better.” This perks Shane up. He finally has an opportunity to ‘peak inside the magic world’ of medicine and really understand how it works.

“The key,” begins Sarah, “is in the questions. You have to ask yourself the right questions before you see me, then, when you see me, you have to ask me the right questions. The first question you should have asked yourself was …

 

Do I really need to go to my PCP’s office and see her/him for this problem?

“No. Not always,” explains Sarah. “There are a number of problems, aches, and pains that are simple and straightforward, and that you can fix without the help of a medical professional. Websites provided by trustworthy sources such as the Mayo Clinic and the Cleveland Clinic can help you distinguish and can often give you suggestions for self-treatment. In the case of your thirst problem, you didn’t need to see a medical professional at all. You could have fixed it yourself. Other examples of conditions you can feel comfortable self-treating include many muscle aches, headaches, and stomachaches as long as they don’t continue over a prolonged period.   In the case of your dirty car, the web site may not have had specific information or you may have been left uncertain. In those circumstances, you might have stopped at a Walk In Clinic. They could have diagnosed your problem just as well as I could, and chances are they would have been more convenient to visit and cost you less money. And if they couldn’t handle your problem, they’d admit that and bump you up to me.

“Let’s say you considered all of that, but decided to come see me anyway. And after collecting your reason for visit/chief complaint, your current conditions/meds, your family medical history, your past medical history, and a physical exam, I had drawn a tentative diagnosis but wanted to confirm with some further diagnostic tests, as was the case with your dirty car. Even the diagnostic testing recommendations are an opportunity for patient/doctor collaboration. You should have asked …

 

Why are we doing this diagnostic test, and what will you do based on the results?   Does the additional diagnostic information merit delaying treatment? And what are my options for achieving the same diagnostic information? What are the chances of negative side effects or harms for each of my options?

“In the case of my request for the digital photographs, I was 99.99% sure you had a dirty car and needed a car wash. But I wanted to be certain. If you had asked the questions above, we could have discussed whether the value of having that additional .01% of confidence was worth delaying your treatment and the additional cost. If we’d discussed it at the time we may have agreed that it was not. In fact, I’ve seen studies indicating that approximately half of all diagnostic tests ordered by physicians are either unnecessary or counterproductive.

But if we had talked, and you agreed that a photo of your dirty car was worth the time and money, we also could have discussed whether you could have had the digital photographs done someplace else. I love Jim to death, and he always has the latest and greatest equipment, but his fees reflect that. Truth be told I could just as well have sent you to Barbara who just uses her iPhone and charges far less. For your situation the additional high resolution of Jim’s equipment didn’t make any difference at all in aiding my diagnosis. And for that matter, if the images had come back indeterminate, I would have still recommended the car wash. Which means it probably was altogether unnecessary and unhelpful.

With all of that to consider you may have decided to just skip the digital image and gone straight to the car wash.   I remember an almost tragic case from early in my career when an overworked resident ordered a CT scan for a patient with abdominal pains he was almost certain were a hot appendix. Unfortunately, the imaging department was backed up and the patient probably wouldn’t have gotten the scan until a day later. Thankfully, a senior resident caught what was happening and sent the patient straight into emergency surgery. Had they waited for the imaging result, the appendix could have burst and things would have gotten really ugly.

When we’re talking dirty cars and car washes this whole thing isn’t a big deal. But I have to admit, my colleagues and I tend to over-use diagnostic testing. In medical school we’re taught a bias to use everything at our disposal, and we carry that bias into practice. The easiest examples are labs and images. We’ll often order labs or bundles of labs out of habit, even if they don’t play a clear role in differentiating our diagnosis. And images are worse since some of them – CT scans especially – expose patients to significant amounts of radiation. Over a patient’s lifetime that accumulates and the latest figures I’ve seen indicate we have as many as 30,000 cases of radiation induced cancer a year. Maybe half of those patients die from it.

And here’s something else that most people don’t realize; technology has become so advanced that we’re continuously redefining ‘normal’. Think back to your dirty car example. In the old days, we would have judged your car as dirty or not based on clear visual symptoms – just like we do physical conditions with your body. If there were empty juice boxes, two week old stray french fries, and dirty soccer clothes strewn all over, then bingo, it’s dirty. If not, it’s clean, right? But if you send me your supposedly ‘clean’ car, and I’m a typical physician, I may order a battery of electron microscope images on it and, as you can probably guess, find all sorts of microbes and gross creepy crawly things in that minivan. Suddenly it’s dirty again, and subject to more invasive procedures. The problem with that is the advanced technology is re-defining ‘normal’ far beyond visual symptoms, which isn’t always helpful. Sometimes it’s downright harmful. If you have no symptoms or known problems, but I do a full body MRI on you just to be sure, I will almost certainly find something ‘abnormal’ somewhere in your body. Only recently have we appreciated that if we use cutting edge, high resolution imaging devices we can ALWAYS find something abnormal.   Absent symptoms, that’s often lead to unnecessary anxiety and further testing or even exploratory invasive surgery – all of which have the risk of or guarantee of negative side effects. Much the same can be said of much of the cancer screening we’ve started doing in recent years. Look hard enough, and you can always find something. It’s only been recently that the medical community has begun to discuss the idea that there is no ‘normal’, perfect, flawless human body. The presence of small fatty growths, for example, is starting to be considered normal.

“So if an image or lab test is not determinant in diagnosis, question why you should do it. Especially absent symptoms. And if you need an image, explore low/no radiation options like ultrasounds, which are relatively safe and low cost compared to other options.

“And there is a really dark secret here as well, Shane. Diagnostic tests have quality related attributes called ‘sensitivity’ and ‘specificity’. Sensitivity is the ability of the test to detect what it’s looking for if it’s present. For example, if you have 100 people who have a certain cancer, and you give the test to all 100, and the test indicates that 96 of them are positive for that cancer, then the test has a sensitivity of 96%. And 4% of the people tested have what’s called a ‘false negative’, since they really should have tested positive for that test. But believe it or not a sensitivity of 96% is exceptionally high for most diagnostic tests.

“Specificity is the ability to detect only what the test is looking for. So if you have 100 people who you know do not have this same cancer, and you give the test to all 100, and the test indicates that 8 of them are positive (we call them ‘false positives’), then the test has a specificity of 92%.   Those 8 patients then go through the misery and anxiety of further testing over time before learning they were a false positive. The shortcomings in sensitivity and specificity explain why physicians often won’t change their diagnosis or treatment based on a diagnostic test result that doesn’t support it.

“So far, we’ve been talking about testing to support the diagnostic process. Problems with sensitivity and specificity sometimes get really bad when diagnostic tests are used for screening – just to ‘be sure’ when there are no other symptoms indicating a person might have the condition in question. Here’s why; many conditions are only found in very few people. Let’s say our cancer in question above is only found in 1 out of every 10,000 people. If physicians order our cancer test above ‘just to be sure’ on 10,000 people, only 1 should come back positive. But for our test above with a sensitivity of 96% and specificity of 92% (which actually makes it a very good test relative to all labs), we’ll likely find the one positive, but also get false positive results on an additional 800 (10,000 x 8%) people!   That’s 800 people who have to deal with the anxiety and expense of disproving a finding for a condition that they don’t have!   And keep in mind – very few diagnostic tests have sensitivity and specificity as high as the example above.

“Finally, and unfortunately, there is also a significant financial element to diagnostic testing that illustrates how your best interests are not always aligned with your providers. Hospitals are especially notorious for doing every diagnostic test they can get paid for. Physicians who do their own diagnostic testing ‘in house’ aren’t immune, either. Their public rational is that they want to be as certain as possible in their diagnosis and screen for asymptomatic conditions, but many of us would question the degree to which they’re really motivated by financial incentives. Diagnostic tests are extremely profitable. And as technology becomes more sophisticated, they become more expensive and more even profitable – even when they’re not more helpful. And the nature of the equipment for much of diagnostic testing is such that the hospital/physician must pay for the equipment up front (such as a CT scanner), then hope they can do enough scans quickly to pay off the equipment, then use the equipment as often and as long as they can since subsequent scans are almost pure profit. And to make things even more perverse, guess who gets paid to treat all the false positives?

When insurance paid for everything, the financial aspect of this didn’t seem to be a big deal to patients, but now that there are so many people on high deductibles, things are different. My sister-in-law showed me a bill for $324 in lab tests and $2,268 for an MRI (both of which she had to pay out of pocket) and I couldn’t see how any of them directly aided in diagnosing her condition or making treatment recommendations. But she asked me too late.

Often times you do need diagnostic tests and they do play a key role in diagnosing or helping determine your best treatment options. But be sure you’re making an informed decision before you have the tests done.”

 

What are my alternatives, and how can I decide what’s best for me?

“Now that we’ve collaborated on a diagnosis, the next major question is what treatment is most appropriate. Do not assume that you have only one option. The right questions to ask include …

What if I do nothing?

Sarah sighs. “Our basic expectation is that our doctors will do something and fix us. This expectation is further compounded by our social inclination to immediate gratification. But sometimes, a better approach is to do nothing. This obviously wasn’t the case in your two examples; you would have died of thirst, and your car wasn’t going to wash itself. But believe it or not, for many conditions the body has amazing abilities to heal itself or needs only minor help from modern medicine. This obviously isn’t always the case, but in some common conditions, including low grade infections, knee/joint pain, and angina (chest pains), we have a propensity to over medicate or to do surgery when there is no available evidence indicating that the surgery will make the patient any better off.”

What if I change my lifestyle habits?

“Unfortunately, this is probably the most underutilized option of all,” Sarah continues. “Roughly 80% of people afflicted with chronic conditions, including most of those with diabetes, heart related problems, and hypertension/high blood pressure; as well as many of those with allergy, respiratory, anxiety, depression, and other mental health related conditions; and even some of those with cancer, can be cured (not mitigated – cured!) with lifestyle changes.

A balanced regime of nutrition, exercise, sleep management, stress management, and connectedness with others is often the most effective, least expensive, and least dangerous (in terms of possibility of adverse side effects) option compared to prescription medications or invasive surgical procedures.

Too often, we physicians mention this in passing as we’re scribbling on our prescription pads. It simply takes too long to council patients on good lifestyle habits, and their compliance with any lifestyle changes happens outside of our office and outside of our control. Patients expect a prescription – an immediate perceived fix, even though very, very few medications do anything more than treat or suppress symptoms. And the prescription pad is easy, and it’s within our control. Some patient satisfaction surveys even indicate that some patients perceive their care as being poor quality because they didn’t receive a prescription. Most patients value the perception of immediate gratification that comes with a prescription or even an invasive surgical procedure.”

What are my medical alternatives?  For prescription meds, is there a generic or OTC alternative?  For surgery, is there a medical management alternative?  If not, is there a minimally invasive alternative?

“There are many cases where doing nothing or lifestyle changes would be completely ineffective,” Sarah is quick to point out. “Otherwise I’d be out of work. A bad infection requires medication.   An aortic aneurism is a ticking time bomb until stinted.   But even under these examples, there are still alternatives.

“Is a prescription called for? If so, does it need to be a brand name, or is a generic just as good? This is a tricky one, since pharma companies have enormous profits and correspondingly, enormous budgets to throw at public relations and marketing activities aimed at both prescribing patients and prospective patients. The games they play as brand name drugs are coming off of their patients are disturbing. For example, they might seek a patient for a new drug that is the same as an old drug coming off patient, except the new drug is at a 50mg dosage where the old was 25mg. Yet because the old now has generic competitors, the cost for two tablets of the old might be 10% of the cost of one tablet of the new; both with equivalent effectiveness.   Or, even worse, they might make a molecular change to the new patient, but because they have no scientifically valid studies to back up increased effectiveness, they suppress what studies they are and resort to pure marketing.   Generic companies, in contrast, have very narrow profit margins and do almost no marketing. That’s why it’s worth asking. Last time I heard, approximately half of all prescriptions written for brand name drugs could have been substituted for far less expensive, but medically equivalent, alternative or generic drugs.

“Is a surgical procedure the only alternative, or is medical management (therapy, medications, or other less violent interventions) a possibility? If surgery, what are the available techniques and is there a minimally invasive option available? Minimally invasive techniques, such as laparoscopy, use relatively tiny incisions and variations of cameras and micro tools to accomplish the surgical task, whereas ‘big cut’ procedures let the surgeon get in up to their elbows and go to work. In general, minimally invasive techniques have a fraction of the post-surgical complications (including infections), and much shorter recovery times.

“And if you decide on a surgical or other procedure, the questions of who and where can have an enormous impact on both quality and price. In your car wash example, if you went to a commercial car wash, the cost/quality tradeoff has pretty much been defined by a competitive market. Not so in health care. In healthcare, it’s more like going to two different nephews and asking them to wash your car. One might charge you $10, the other $60. But price wouldn’t necessarily reflect quality.”

“OK, Shane,” Sarah intones seriously. “If you’re inclined to consume adult beverages, we need to get you one before we continue. Most physicians aren’t even clear on what I’m about to tell you.

“If you’re a patient, and I, as your doctor, prescribe a med or recommend a surgery, you’re natural presumption is that if you take the med or do the surgery, you’re going to be better off because of it, correct? Obvious, even? But in reality, that’s not always the case. As an institution, we are guilty of over stating the expected benefits and understating the predictable risks of many, many interventions.

“Quiz time. Lets say I write you a script and explain the expected benefits, and you agree to take the meds as directed. I then tell you I’ve written the exact same script for 199 other patients that were experiencing the same condition. Like you, 99 others agreed to take the meds. But the other 100 refused and opted to do nothing.

Question #1: Of your group that took the meds, how many of the total 100 would you expect to experience the anticipated positive benefit of that prescription drug?

Question #2: Of the other group of 100 who refused to take the drug, how many would you expect to experience the anticipated positive benefit regardless?

“If you answered 100 to question #1 and zero to question #2, you’re not only wrong. You’re way, way off. Those would have been the answers for your thirst problem and my water solution, but they’re in no way reflective of a typical prescription medication. Refill your drink and sit down, because you are not going to like what you’re about to hear.

“For a prescription drug to get approved by the FDA, it needs to pass two standards; safety and effectiveness. Simple enough. But the problem is how those standards are measured. A clinical trial is basically a group of people with a specific health/illness profile specific to the drug being tested. Half the group is given the drug, and the other half a placebo. But these can be fairly small groups – as few as a hundred each. And the trial may go for a fairly short time – usually less than a year, and sometimes as short as six weeks.

“Safety is a subjective assessment of all of the negative effects experienced by those involved in the clinical trial group, with no effort to establish that the negative effect was caused by the drug being tested. So an adverse side effect may have been coincidental and not causative, resulting in over stating possible side effects. But since the study is fairly short, it’s also possible that slow developing side effects are undiscovered and unreported.

“Effectiveness is the real deception here. If there are 100 people each in the group receiving the drug and the control group (receiving the placebo), and 2 people in the placebo control group experience the expected positive effect of the drug (for reasons obviously unrelated to the drug), but 3 people in the test group that received the drug experience the expected positive effect, then the drug is deemed effective even though only 1 person out of 100 could be seen to actually be positively impacted by the drug!

And if you think that’s bad, it gets worse:

The drug companies themselves are either running the clinical trials, or are paying a third party (called a Contract Research Organization, or CRO), whose business is totally dependent on maintaining the drug companies as clients.

Many epidemiologists would argue that the differences in results between the test and control groups are often statistically irrelevant, and that there is not enough isolation from confounding factors to consider the test drug to be validated as causative for the positive impact.

The drug companies can run several simultaneous clinical trial groups, and can throw away the results that don’t favor their application. For example, they might run 3 simultaneous groups; one produces less positive effect than the placebo, the second equal effect, and the third a minor beneficial effect such as the one illustrated above. The drug company can throw away the results of the first two clinical trials and use the third to successfully achieve FDA approval. Incidentally, this drives everyone but the drug companies nuts, since it essentially proves that the trials are not reproducible, which calls into question their overall validity.

There is little to no effort made by the drug companies at this stage to identify sub-populations within the test groups that may be either advantageously or adversely impacted. For example, if all of those experiencing the expected benefit have a common race, or all of those experiencing adverse side effects are within a certain age bracket, these findings are not published.

The test groups may, to a degree, be ‘cherry picked’ or selected for financial reasons. For example, a clinical trial for a drug that is anticipated to be marketed toward the elderly may include a substantial number of participants who are children. This benefits the drug company since they get an automatic additional 30 months on their patent, and it may skew the results in their favor since children have such a substantially different (arguably less vulnerable) metabolism than the elderly do.

There is absolutely no requirement on the part of the FDA to compare the new drug to one that actually exists and is already effective. So the newly approved drug might actually be less effective that one that’s already on the market – and this potentially includes generics.

The FDA receives a substantial % of total funding from the fees associated with drug applications. FDA employees and advisory board members are not precluded from receiving ‘consulting’, speaking, advisory, or other funds from drug companies. This is a clear conflict of interest that most of science (and even some of academia and government) attempts to steer clear of, but we tolerate it at the FDA. Who exactly does the FDA ultimately work for, anyway?

“So let’s go back to our quiz, and use some specifics. You have elevated LDL (‘bad’ cholesterol), which is assumed to indicate an increased risk of heart attack. The class of drug known as statins (such as Lipitor, Mevacor, Zocor, and Crestor – just to name a few) has been heavily marketed to reduce LDL. But LDL is what’s known as a ‘surrogate’ measure, with the assumption that lowering LDL leads to a reduced chance of heart attack.  But that assumption isn’t supported in long term studies.  Since clinical trials are over relatively short time frames, statins were tested against surrogate outcomes instead of actual outcomes.

“Let’s look at what actual independent comparative effectiveness studies based on double blind randomized controlled tests show:

Question #1: Of the group that took statins, how many of the total 100 would you expect to experience the anticipated positive benefit of that prescription drug?

Answer to #1: Three experienced heart attacks; two of the three were not fatal.

Question #2: Of the other group of 100, who took a placebo, how many would you expect to experience the anticipated positive benefit regardless?

Answer to #2: Four experienced heart attacks; two of the four were not fatal.

“When independent effectiveness studies are conducted, the goal is to establish effectiveness in terms of how many in the test group benefitted (called the ‘numbers needed to treat’, or NNT) and how many in the test group were harmed (called the ‘numbers needed to harm’, or NNH). These are MUCH clearer predictive indicators than the self-promoting literature produced by the drug manufacturers.   Additionally, independent Comparative Effectiveness studies pit interventions with similar goals against each other; for example a new statin vs both a placebo AND an existing, relatively effective statin. Bias is now eliminated since the study is independent and is a double blind randomized control test, and the bar to define a ‘win’ is an improvement on the status quo. Is one option measurably better than the best currently available in terms of NNT and NNH.

“All of these same concepts can be applied to evaluating the options for either using a surgical procedure (vs an alternative, less violent option), or considering one procedure over another. But care must continue to be used when differentiating surrogate vs actual outcomes measures. For example, if the actual desired outcome is better ambulation and range of motion for a knee surgery, the NNT for medical management and physical therapy is as good as or better than the NNT for knee surgery. For a lower risk of heart attack and stroke, the NNT for medical management is as good as or better than the NNT for bypass surgery. And the NNT for lifestyle changes could be argued to be far superior than that of either medical management or surgery. Better yet, medical management combined with simultaneous lifestyle changes may offer the best NNT available. And don’t forget that all surgical and pharmaceutical interventions have an NNH, and that NNH is almost certainly higher – usually MUCH higher – than alternative lifestyle changes or even doing nothing.”

By this time, Shane was simply shaking his head. “I had no idea,” he muttered. “This makes it sound like a lot of what I thought was modern medicine is really a sham.”

“Not all of it, by any means,” replied Sarah. “But enough of it is questionable, and even doctors aren’t always self-conscious of their own bias. Nor do they always realize just how much money has been spent by the pharma, diagnostic, and medical device manufacturing industries to get them to use their products freely. And the fact that Medicare, private insurance, and even Medicaid pays for so much of this has made it very easy to do a lot of procedures, feel like you’re doing a lot of good, and, incidentally, become very wealthy.

“What can you do about all of this, you’re probably wondering? Well, if you’re leaning toward using a prescription med or surgical procedure, one other piece of this conversation that you can try and have with your doctor is this …

Is there any way to predict which of the three basic populations (those benefited, those not effected, and those harmed) that you personally fall into?

Sometimes there is; sex, age, race, and family history can sometimes help predict. One possible tool for some prescription drugs is a pharmcogentics screen. This is a subset of genetics screening that just looks at a tiny subset of your DNA for information on your likelihood to respond well (or poorly) to a small subset of drugs. No silver bullet, by any means, but if you and your physician are considering a drug that can be tested for, this may be an excellent option. Another possibility is a review of the literature for what are called ‘secondary studies’. These look at past clinical trials and other effectiveness studies, and try to isolate sub groups by some definition (age band, race, blood type, existing conditions, allergies, etc) to better predict those helped and harmed.

“So … with that understanding, prepare yourself to ask the following questions when collaborating with your physician on a prescription drug or therapeutic or surgical procedure. This gets us to our second set of ‘Rules of 100’s’:”

For each of my options:

What are the anticipated benefits, and for every 100 patients treated, how many more experience the anticipated benefits compared to patients who do nothing? Is there Numbers Needed to Treat (NNT) data available?

What are the possible side effects to me, and for every 100 patients treated, how many more experience the side effects compared to patients who do nothing? Is there Numbers Needed to Harm (NNH) data available?

Are there any predictive indicators to determine whether I’m more likely or less likely to experience the anticipated benefits or side effects? Family history? Past medical history? Genetic screening? Age, race, sex, or ethnicity?

Are there comparative effectiveness studies available? If not, what do other studies say will be most effective?

“And finally, the big question. This is where your PCP can really do you a great service …”

 

“There is no one good answer for any of these questions, Shane. The best answer comes from a patient who understands all the alternatives available to them, and a recommendation from a physician who understands not just the scientific elements of the question at hand, but all of the other intangibles unique to that patient; things like the patient’s values and belief system, their religious feelings, and their emotional state. An engaged, collaborative physician will take these factors into consideration.

As the patient, it’s important to appreciate how this last question is nuanced. It’s better to ask your physician questions phrased to get a personal response as contrasted with a professional opinion. ‘What would you do if you were me’ specifically elicits your physician’s advocacy for you as a unique individual. You’re looking for advice that might be different for the next patient in similar circumstances. You can also ask ‘What would you advise one of your family members to do in my circumstances?’   That question also solicits a personal response that might be more candid than a professional opinion.   Your doc might be much more likely, for example, to thoroughly discuss or even advise a ‘do nothing’ or ‘lose some weight’ option with a relative than with a patient. Why? Because that might be what’s in your physician’s heart, but they’re delicate and different conversations to have with patients.

Both of these are contrasted with the more usual ‘What should I do, Doc?’. That question elicits a professional referral or recommendation. Professionally, your physician may be influenced by pressures from their peer network or from the hospital(s) where they have privileges to channel patients down paths of over-utilization that value the goals of specialists or hospitals over those of patients.”

Shane was still reeling from what he’d just heard. “Well,” he finally replied. “I’ve had some interesting conversations during the down time at these soccer tournaments, but this one is really giving me something to think about. But one final question; why didn’t we have this conversation when I first came in with my thirst problem?”

Sarah paused before answering. “Simple. I’m part of the problem, even when I try to be part of the solution. I’m a primary care doc. I earn a fraction of what my medical school classmates who chose specialist roles make. And we both carry about the same med school debt, which is pretty significant. My husband and I have two kids, and all the same financial pressures that come with raising a family on a middle class income. For me to make ends meet, I need to average 32 patients a day. When I walked into your exam room – both times – I was dealing with the best outcome both you and I could get from that moment forward. I wasn’t going to chase you out of my office and not bill for the visit; that wouldn’t have helped either one of us. And I simply didn’t have the time to have this discussion with you then. I had 4 minutes before I needed to see the next patient in order to stay on schedule. Now that we’ve had the time this weekend, I’m totally OK if this cost me some future office visit revenue because you do something different next time.

“Why? Because I’m still clinging onto the reasons I decided to be a doctor in the first place. To help people.”