Safe and Effective?

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Over the years, I’ve had the opportunity to deal with FDA (Food and Administration) approvals for a variety of drugs and devices.  I admit, sometimes, it did not quite seem to be an ‘opportunity’, but, generally, I was most appreciative of the process.

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Of course, we had to be very careful to never say the products we developed had FDA approval, since the government agency refuses to be on the hook for its actions. Instead, the FDA avers that, given the data an entity submits, the product is considered to be”safe and effective” or “substantially equivalent” to another item that has been reviewed by the agency.

Our track record is and was pretty good in this area.  While many a firm awaits the process end for seven years or so, we managed to cut that time significantly over the years.  (It actually became a competitive advantage for our firm.)

So, it was with initial dismay that I perused a report by Nicholas Downing (lead author, med student at Yale; his co-authors were Drs. Aminawung, Shah [Mayo Clinic], Krumholz, and Ross- all the rest at Yale).  They examined the approvals of some 188 drugs that underwent FDA review over a 7 year period (2005-2012) and reported their results in the Journal of the American Medical Association.  I was drawn to the report due to various “news” reports about their findings.

It turns out that some drugs were required to be evaluated via multi-site trials, while others (about 1/3) only needed evaluation at a single site.  And, while the authors seem to rail about the latter, most of those approvals involved only cancer drug approvals, which are severely life-threatening and for which there are no viable alternatives- yet.  Given the complexity of those situations, I am not sure this practice is as heinous as it sounded to me at first reading.   Except that many of these trials did not involve the use of placebos [there often was not another drug with which it could be compared] and also employed surrogate end points.

[A surrogate end point means we are employing a measurement of the effect of the drug that may correlate with a clinical milestone- but it may not.   It may indicate how a subject feels, functions, or survives- but may not be associated with the cure of the patient’s condition(s).  One such surrogate measurement is cholesterol in lieu of monitoring heart disease.  I have already explained how poorly this sort of surrogate measurement is employed in medicine.]

The law does require that the FDA demand “adequate and well controlled investigations” when determining the efficacy of various drugs.   This is why the FDA guidance documents state that at least 2 trials be submitted to obtain approval, but that same document also states that the agency retains “flexibility” in applying this guideline.

So, you understand that a drug to treat athlete’s foot would require multiple trials and comparisons with existing drugs.  But, a drug to treat “rara-carcinoma” (a new cancer I just invented) with 1000 known new cases a year could only involve a small trial with no placebo and maybe not have the best test markers.  But, if that is a disease you or your loved one is facing, do you really blame the FDA for loosening its guidelines?

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20 thoughts on “Safe and Effective?”

  1. I have never understood how this all works. What I do know is that I have friends here in the US that need drugs. The drugs exist and are used in other countries but the US won’t approve them…for years to come. I know each country has their own rules and guides but surely they can use what was found in other countries to help speed up the process. But no, that does not seem to be the case.
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    1. Many of the drugs that exist elsewhere are made by companies (Ranbaxy, for example) that fail to protect the public, make adulterated products, etc. Or, they make drugs that have alternatives that are more effective or less expensive. (One, unfortunately, cannot compare prices charged in India with those in the US- since firms have a policy of socking it to the US resident and selling the drug for less abroad. One can only compare prices of drugs within the same market.) Or, the drugs you mention may not have had complete testing- and are doing so as we speak. (For major, wide-spread diseases, multi-site trials are the norm; they are not the norm for other jurisdictions.)
      I’m not saying that is always the case- just usually, Shawn.

  2. Your point is well taken, If I or someone I loved had cancer, I would not want my next of kin to find out later that we had been given a placebo. When we’re talking death as the answer to the test, I’d opt to go with the new drug because its a 50/50 or better chance that it would help me, whereas there is no chance with the placebo. That seems like a no brainer to me. I also agree with Shawn, The German governments “FDA” has a very good track record and has approved drugs our country has not even looked at. I think that is partly because some of our companies would not make any money on them if approved for sale in this country. Business is Business in a country where money talks louder than people.
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    1. Oh, I won’t buy that logic for a New York minute, Chef William. I would put the FDA up against the German counterpart any day of the week. And, the 2011 practice the government (not their drug approval system) imposed to put price controls has cut the availability of many drugs.
      It’s like our vaccine system in the states- it does not provide enough to keep everyone in the system. That’s NOT an improvement, at all.

    1. Do you really think patients can make such trade-offs, Muriel? I, for one, know that to be NOT the case. Just look at how many folks are running around half-cocked about GMO’s- like we haven’t been manipulating the genes of our foodstuffs for millenia…
      And, without the studies, how would one know the risk and the efficiency???

    1. That would require there being a drug to accomplish that task, Ann.
      And, let’s assume they speed up the process (I am not quite sure what that means)- and because they found the shortcut (which usually means no long term testing), the patient no longer has memory loss, but will die within a year, because there was no long term safety study. I don’t think that’s a wonderful tradeoff, at all.

    1. I don’t know how speeding up would be done- unless one decides that long-term safety is not important, Alessa. Which is kind of what the article was talking about is done for cancer patients- since no treatment would be no life after a while- so long-term safety is less critical.

  3. A good friend of mine is facing a serious situation. She had cancer years ago, and while they cured it, she reached the lifetime limit on her chemo drug. Fast forward some 30 years and she has a different cancer. First line treatment – you guessed it, the drug she can’t take anymore of. And the next choice? Well, in many cases, it stops working after about 18 months. She went on it and….. Now,it’s two years later and the cancer, which was in remission, is growing again. So now what? Fortunately they had other drugs to formulate a treatment plan, but how many drugs are left? I have a feeling she would take that limited trial drug in a New York minute, if it gave the hope of control and remission. I’ve never been in that situation, and I hope I never am.

    1. Alana:
      This is exactly the reason why the FDA has been approving cancer drugs under much less stringent protocols than other drugs. Because the nature of the illness is such that there is no alternative drug, a placebo would mean abandoning the patient to die, etc.
      Thanks for making the situation clearer!

  4. I can certainly understand why the FDA would loosen it’s guidelines for drugs to treat certain cancers, as I’m sure those who are afflicted with those types of cancers and their loved ones, would greatly appreciate. That said, I certainly would want them to be as careful as possible when putting new medications on the market, which, of course, requires time.

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