This health blog is devoted to your child’s health, not our wild politics, but now and then politics threatens our children’s health, so we need to take note.

The threat comes from a plan to dismantle the processes that insist any drug or device that is prescribed or used has been proven to be safe and effective.

I like these processes because they reflect what I consider the two questions I hold most important in weighing any advice we give:

1. Is the proposed drug or intervention effective, will its use actually make you or your child’s life better?
2. Is the proposed drug or intervention safe, will its use cause any harm to you or your child?

To me these two questions are so very important, if they are not asked, then the door swings open for people who want to make money to sell goods that don’t work or cause real harm.

The only protection any of us have in the marketplace is an organization called the FDA.  In the 1960’s, a drug used to control morning sickness in pregnant woman, thalidomide, was not allowed to be sold until it was able to prove it was safe and effective.  That delay kept its makers from selling it in the United States for an extra year or so, during which time many women in Europe who used it found it caused their babies to be born without arms or legs.  This catastrophe was only prevented here due to the rules of the FDA, which boil down to one:  no drug can be prescribed until proven to be safe and effective.

Now we learn that the White House is seriously going after these protections:  https://nyti.ms/2kavxyh

Here is an important point.  Sometimes we get wound up with political slogans like regulation, or big government, or interference of the government in our lives.  But in this instance the issue is not regulation, or the size of government.

It is really about whether in our country, if your doctor prescribes a drug or use of a device, whether you can possibly know if it works and if it is safe.

Right now we know.  I can follow the medical literature, I can rely on the FDA, I can find out if a drug works, it if really improves life, and I can find out if it is safe.

If the FDA is crippled from doing its work, no one will know, I won’t be able to find out.  The very foundation of my profession, of being a doctor, will be compromised, as my ability to find out if a drug or device helps or hurts is erased.

It would be very good news for the money makers.  After all, it takes a lot of money and time to find out if a proposed new drug or device really makes a difference or possibly will cause harm.  Imagine the riches to be realized if all those bothersome questions can be skipped, and all you have to do is make a flashy package, and a good ad campaign, and billions can be yours.

Some people in Europe made a lot of money selling thalidomide in the 60’s, and if the FDA is prohibited from ensuring our drugs and devices are safe and effective, perhaps some Americans can look forward to many bonanzas, too.

But I stand against the choice of money over health.  It would be heartbreaking to see one child or person hurt by an untested drug, and unproven device.  Especially if it was just to help someone get richer.

A key item to keep an eye on is the sneaky concept called surrogate endpoints.  Even companies that have no compunction about selling drugs and devices that do not work and, in fact could seriously hurt people, don’t like to come out up front and say so.

Here is there current pitch:

“We support requiring all drugs and devices be shown to work, but we don’t think they should be proven to not cause harm.  And, when it comes to working, we would like to say it’s enough to show a drug or device does its intended purpose, even if, ultimately, no health is actually improved.”  

That is a surrogate endpoint.  A great example would be a drug designed to lower cholesterol.   Research may show it does indeed lower cholesterol, but it might not really have an impact on what everyone really cares about when it comes to cholesterol, namely, having a heart attack.  Here, lowering cholesterol is a surrogate endpoint, since the real endpoint is living longer, having fewer heart attacks, but the new wave at the FDA threatens to chuck the burden of showing a drug actually helps, and only requiring it to show it does something.

BOTTOM LINES

1. It turns out the be wondrously difficult to create a drug or a device that actually makes one of us healthier.  Most such inventions fail to do so, in fact, about 90% of drugs reviewed by the FDA fail to get approval.
2. Every person I know wants the same thing for their child(ren) when it comes to drugs and devices.  They don’t want their child(ren) to be treated with something unless it clearly is beneficial.
3. That means we want our drugs to really help and to really not hurt.  BOTH.
4. The FDA, since 1962, has maintained a standard we all can support, that is, that prescribed drugs and devices cannot be sold without good proof they really work, really help, and really don’t hurt.
5. Forces are at work, RIGHT NOW, to gut the ability of the FDA to ensure the medications and devices prescribed in America really work, really help, and really don’t hurt.
6. If you think that’s a problem, you are right.  Keep in mind the medical landscape is littered with drugs and devices that did pass the current FDA approval tests, but over time were found to be worthless or harmful.  The market gets fooled all the time.  There is NO doubt that plenty of companies could give two hoots about your safety, they are in it just to get rich.

I care very deeply about this issue, anyone familiar with Advanced Pediatrics knows we go to great lengths to make sure we never recommend an intervention unless we have good proof it works, it helps and it doesn’t hurt.  We don’t even allow any samples or gifts or even visits from drug companies.

If the companies that seek to gut the FDA succeed, even our ability to make sure the advice we offer you on drugs and devices will be dangerously weakened.

I plan to let Senators Brown and Portman, and our Congresspeople know how I feel about this threat.  I don’t think it has anything to do with being conservative or liberal, Democratic or Republican, it’s just a simple foundation of medicine.

Our drugs and devices should be tested to find out if they work, if they help, and if they hurt, before any of us are exposed to them.  We know any other approach invites needless catastrophe.

To your health,
Dr. Lavin