illustration of pills shaped into a dollar sign ($)

How effective is your medicine? This seemingly simple question is surprisingly complex. In many cases a pill’s benefits are barely detectable.

Before a drug can be approved by the Food and Drug Administration, its maker must demonstrate that it is “safe and effective.” That’s the law. The trouble is that the legal definition used by the FDA may be quite different from the way most people understand “safe and effective.”

The FDA’s Definition of Effective is Bizarre:

The agency requires that most medications be tested in two randomized controlled trials. The active compound has to be statistically better than an inactive sugar pill.

That doesn’t mean it has to be very effective, though. It may be necessary to use complicated statistics to detect a difference that is almost invisible to most patients.

This is clearest when we look at important outcomes such as survival or disappearance of disease. Many medicines are able to move markers such as cholesterol, blood pressure or blood sugar, but they don’t necessarily change how long or how well a person lives.

Statins: Incredibly Popular but How Effective?

As just one example, let’s consider statins. Many physicians consider them lifesavers and one of the greatest advances in modern medicine. According to the Agency for Healthcare Research and Quality (AHRQ), a division of the U.S. Department of Health & Human Services, statins are among the most prescribed drugs in the country.

In its Statistical Brief #458 (November 2014) AHRQ reports on Trends in Statin Therapy among Adults (Age ≥ 18), United States, 2000 to 2011:

  • The number of adults (age ≥ 18) who reported using prescribed statins increased from an average 17.6 million annually in 2000-2001 to 40.8 million in 2010-2011.
  • The number of statin prescriptions filled by adults (age ≥ 18) increased from an average 102.2 million annually in 2000-2001 to 237.9 million in 2010-2011.
  • Among adults (age ≥ 18), annual total expenditures (in constant 2011 dollars) for all statins rose, on average, from $11.5 billion in 2000-2001 to $21.6 billion in 2010-2011.”

Do statins deserve their reputation and popularity?

Billion dollar drugs like atorvastatin (Lipitor), fluvastatin (Lescol), lovastatin (Mevacor), pitavastatin (Livalo), pravastatin (Pravachol), rosuvastatin (Crestor) and simvastatin (Zocor) lower cholesterol levels extremely well, but do they actually improve and prolong life? After all, that is presumably the point of the exercise.

An analysis published in BMJ Open (online Sep. 24, 2015) found that in the most important randomized controlled trials of statins:

“The survival gains we found are surprisingly small. The highest value was 27 days, found in the 4S study, achieved by 5.8 years of simvastatin therapy in participants with a history of unstable angina or myocardial infarction [heart attack].”

Put another way, the survival advantage from taking a statin is modest, far less than most physicians imagine. By and large, patients have no idea that the “median postponement of death” attributed to statins is between three and four days.

Wait! Please stop and consider that last statement again. When a doctor puts a patient on a statin the expectation by the prescriber and the patient is that this medicine will ultimately prolong life substantially by preventing a heart attack, stroke or some other bad event.

“Death was postponed between -5 and 19 days in primary prevention trials and between -10 and 27 days in secondary prevention trials. The median postponement of death for primary and secondary prevention trials were 3.2 and 4.1 days, respectively.”

[Primary prevention usually means the study subjects were otherwise healthy people who had elevated cholesterol levels. Secondary prevention means that subjects had already experienced one heart attack or had been diagnosed with heart disease. Most experts support statins for secondary prevention. Primary prevention with statins has been controversial for years.]

In discussing the significance of these startlingly small benefits the authors note:

“Experience from studies of preferences, when presented with similar scenarios, shows that as many as 70% of lay persons would not accept such a treatment.”

What About Statin Side Effects?

The authors conclude:

“…if the patient has intolerance or unpleasant side effects from statins, for example, muscular problems, physicians should not be too insistent on the patient continuing them. Also, for patients whose life expectancy is short, the benefit of statin therapy in terms of survival gain may be quite limited.”

What About Billion Dollar Drugs for Depression?

Antidepressants are another example of billion dollar drugs that don’t always measure up to expectations. Meta-analyses of randomized controlled trials of antidepressants show that these drugs are statistically better than placebo (Contemporary Clinical Trials, July, 2015). According to the authors, however, their benefits are barely noticeable in everyday life.

Another group of researchers analyzed both published and unpublished results from clinical trials involving antidepressant medications. In this meta-analysis of drugs like fluoxetine, nefazodone, paroxetine, and venlafaxine they discovered that the newer generation antidepressants they reviewed were no more effective in relieving mild to moderate depression than placebos (PLoS Medicine, Feb. 2008). The conclusion they shared:

“Given these data, there seems little evidence to support the prescription of antidepressant medication to any but the most severely depressed patients, unless alternative treatments have failed.”

How Good Are Drugs for Nail Fungus?

Nail fungus is not life threatening, but many of the prescription medications used to treat it are not particularly effective. They are pricey, however.

Television commercials for efinaconazole (Jublia) make it seem as if this topical medicine can tackle tough toenail fungus because the FDA approved it. Dig a little deeper into the official prescribing information, though, and you will learn that it takes 48 weeks of daily applications to achieve a compete cure of the fungus in 15.2 to 17.8 percent of the people using it. Given the price of this medicine, many patients would anticipate a much higher cure rate.

Patients expect that when their doctors prescribe a drug, it will work for them. By work, they assume it will relieve the malady or prolong their life. Physicians need to become familiar with the data on true effectiveness of the medicines they prescribe so that they can be honest with their patients about what to expect.

Please share your own experience and thoughts on drug effectiveness in the comment section below and please vote on this article at the top of the page.

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  1. Jim

    When you look at the reliable evidence, the truth about most prescription drugs and the standard medical establishment is even more disappointing.

    Let’s take statins:

    It is important for the public to recognize that most of the “scientific” research in favor of cholesterol-lowering statins is flawed and fraudulent, in favor of the drugs..

    The most reliable evidence has long tied statin use with memory problems, muscle disorders, liver damage, cataracts, nerve damage, pancreatitis, erectile dysfunction, brain dysfunction, diabetes, and with an increased risk of cancer and higher mortality (statins only somewhat reduce the risk of non-fatal heart attacks).

    The physiological mechanisms of how statins do serious damage are also well understood, such as by their impairment of oxidative cell metabolism, the increase in inflammation and cell destruction, the lowering of cholesterol and steroid hormone production, the promotion of pancreatic injury, etc. – rather thoroughly explained in this scholarly article on how statins, and a cholesterol-lowering popular diet pill advertised by Dr. Oz, promote diabetes by googling “Do Garcinia Cambogia Side Effects Boost Diabetes?” (at supplements-and-health dot com site) – look at Figure 7 to see how irrational it is to block the production of cholesterol!

    Yet despite of the existence of that scientific knowledge, the medical business and the public health authorities keep ignoring it and continue to recommend statins to diabetics and make claims that they have a low risk profile despite that they are also significantly linked to cancer and higher mortality (just look at the propaganda put out by the Mayo clinic on statin drugs: “the risk of life-threatening side effects from statins is very low”).

    And because of such medical propaganda, few people are aware that the medical claims of benefits of statins are mostly based on studies conducted by people with vested interests. And, logically, it’s mostly the corporate medical business and other people with similar vested interests tied to it (eg, mouthpieces, hacks) who promote the alleged value of these highly lucrative products.

    Also, older people with HIGH cholesterol live longer than those with low cholesterol levels (see above mentioned article for numerous scientific study references confirming this).

    Because the cholesterol-heart disease theory, or rather medical dogma, is wrong, the use of statins is also wrong by logical extension.

    So the real truth is that statins have almost no real benefit in the very vast majority of users. They do more harm than good (read Uffe Ravnskov’s “The Cholesterol Myths” and Malcolm Kendrick’s “The Great Cholesterol Con”). It’s one of many “scientific” scams of the mainstream medical business.

    • Brian
      PA - Pennsylvania

      Thank you for your impassioned and truthful comment. Information such as this needs to be brought to the forefront. It is so disheartening to see people blindly believe whatever is told to them by their physicians. I sincerely hope that people will start educating themselves about what they’re putting in their bodies.

  2. joy

    I had a large area of toenail fungus on my big toe. rRather randomly, I applied, Vicks Vaporub, Listerine, and tea tree oil (each separately). Within a few months, the toe nail grew out clear and clean.
    With no expensive or dangerous drugs!
    I am 76 years old and always choose alternate treatments first.

  3. l. MARTINEZ

    doctors will darn near go ballistic on you if you refuse statin drugs… red yeast rice is excellent… not expensive. I gave husband cup of oats in the am… and one of his doctors called me and told me … stop the statin… his cholesterol was getting low enough… I did not tell him I never gave them to him.. I did the oats trip instead. Even tho we have been together 56 years this man of mine trusts the doctors and gets confused between us… after all it is the M.D. who has had all the schooling. Very frustrating… and the acceptable level of cholesterol is set very low… now they want to automatically put seven year olds on it so they will never have to worry about it in the first place.

    This is madness.. and drugs are given that kill the coQ10 and they never tell you to supplement it… wild…. it is the drug companies and their lust for money…. they really don’t care about you, period… I, myself am on no meds… drives my doctor nutz… am 85… and doing great… I do take hawthorne twice a day for the heart. astasanthin. and msm for pain. I have some knee pain and it works great…. to all ? investigate for your own life. doctors will tell you what the drug companies are telling them… and bottom line ? it is all about the money for them….

  4. Laurian
    Naperville, IL

    I guess most people don’t realize that the heads of government agencies (e.g., FDA, EPA, SEC) are subject to political pressures. That means when high powered lobbyists call congressmen on behalf of the drug companies, that pressure is transmitted to the relevant agency. The result is regulations that often don’t follow common sense and drugs that lack important information for the consumer.

  5. Gary
    Buffalo, NY

    In many cases, lifestyle interventions work better than medicines. Of course, there is much less profit to be made when people adopt a whole food plant-based diet and exercise more. But such a lifestyle would produce far better health improvement than taking statins. Consumers need to be better informed.

  6. David

    It amazes me that the FDA would approve an excessively expensive product for nail fungus when the anticipated success rate is so low.
    We can do our own research if we have the ability to search the web (but so many do not and you must get a reputable site), so doctors should most definitely give us the information of the percentage of success. Unfortunately, if an insurance program will cover the cost, many people will use it in hope…..but the cost to the health industry is exorbitant and will only increase insurance costs in he long run. If it is not a very successful drug, why does it cost so much?!?!

  7. David

    80 year old male with many health conditions (but happily soldiering on). Heart by-pass at age 59 and I was on Simvastatin for very many years (fortunately no muscle ache side effects), further heart attacks later and a blocked carotid artery I am now on Atoravastatin 40 mg and my cholesterol is very low.

    Now is this a good thing, as my cardiologist seems to think, or am I setting myself up for other problems? I am concerned about brain fog, but feel we all get a little forgetful at times. No serious mishaps to date, just normal forgetfulness. How can anyone possible state that these drugs can give so many days extra life, when there are so many different variations of the time a person has been on the drug. I can understand perhaps this might occur with an older person having a heart attack and no previous knowledge of the condition (which would be difficult to imagine in this day and age, but I guess it could).

    Then maybe the extra days have some correlation…..but if a person has been on a cholesterol lower drug for many years, surely one imagines it has been protective and helped in their longevity?

  8. Barbara

    I am one of those people who didn’t use Jublia but tried a different topical nail treatment for toenail fungus with very little improvement, after 18 months!! When another podiatrist wanted to prescribe it to me again he assured me that this time it would work. This time I was also prescribed an anti-fungal prescription in addition to the topical treatment and it did indeed work. But it is my belief that the anti-fungal prescription is what made the difference not the topical treatment. Interesting though is that my insurance would cover the topical treatment but not the anti-fungal prescription. Luckily it was not that expensive, so I paid for it out of pocket. But I think the topical nail fungus treatments are a total waste of time and money.

  9. Robert L Jones
    United States

    Timely and valuable information about statins.

  10. Moyra P.
    London, UK

    ARE there any ‘honest’ GPs, when their income top-up depends on their prescribing habits? The converse scenario (despite the oath they take) wd be their ‘ignorance’ of the harms caused by their psychological force-feeding of both ineffective AND toxic drugs. The medical model, with its multiplicity of hidden financial agendas, is now a global disaster. Untold millions of patients’ lives are destroyed… many stories so well-documented, but still ignored… by doctors’ refusals to listen to adverse feedback.
    I would advise anyone not yet harmed to read Dr. David Healy’s book, PHARMAGEDDDON.

  11. Rhonda

    I worked for a pharmaceutical company in research and development. We tested the topical so for fungus and found them very ineffective. I truly believe that drug companies should not be allowed to advertise outside of journals. These tv ads are extremely expensive and that cost, as always, is tacked onto the drug. It also has a way of making people think it is a wonder drug, when in fact it is not.

  12. Conie

    I believe the placebo effect is likely the only thing positive that comes from over half of doctor visits. Even then, the medicine they give, especially to people over 80 years old, likely hurts more than helps. I am 85. Possible exceptions are replacement medicines, for example, for a thyroid that is hypo or estrogen for older women. I do believe most doctors are moral– that they teach what they learned in medical school and believe it themselves. The only answer I can see is for consumers to do their homework (research) and for modern science to go forward, which is happening–fast! It would help, also, if doctors were good listeners.
    That can’t happen unless patients are good talkers–if they did their homework, wrote down their questions, and didn’t chat.

    • Randa

      Listerine and tee tree oil work great cheap and no toxic side effects like the RX!

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