Raise your hand if you want to be in the placebo group…

We opted for a “doctor supervised off-label test” instead of a randomized control test. Here’s why.

American Society of Hand Therapists (ASHT)

Dr. Fauci has consistently dissed the use of hydroxychloroquine because there hasn’t been a large-scale, double-blind control test proving its efficacy.

There are plenty of field reports, patient testimonials and doctors prescribing the drug “off-label” (or even using it themselves as a prophylactic).

But, Dr. Fauci advises that we all just chill for a year or so until the “proper” scientific testing is done.

That point-of-view hits close to home.

Here’s a very personal story…


A couple of years ago, my wife was diagnosed with an early stage but very aggressive and invasive form of breast cancer.  Her’s was not a garden variety breast cancer since she was a “double negative”.  The practical implication was that, at the time,  there were only a few approved post-chemo biological drugs to use. 

Double negative” means testing negative for hormones estrogen and progesterone, but positive for the protein HER2.  Most cancer fighting drugs get “carried” around the body by attaching to the two hormones. Since most cancer patients have an adequate representation of one or both of the hormones, most cancer fighting drugs are developed for that profile.

One of the candidate drugs for my wife was a relatively new-to-the-market drug that had demonstrated high efficacy in controlled tests. When used in combination with the primary HER2 drug, it increased the long-term survival rate by multiples.

That was good enough for us.

The problem: This wonder drug had very tight label restrictions.  That is, patients were required to meet certain health-based  criteria.  My wife was outside one of the label criteria by just a little bit.  Doctors didn’t think that the disqualifying criteria was material for my wife and wanted to prescribe the drug,

Pursuing one angle, the docs found a spot in a randomized controlled clinical trial that was in process.

The rub: In the trial, my wife might get the drug., but might not. There was a 50% chance that she’d get a placebo instead of the drug.

We wanted to use the drug and a 50% chance of getting a placebo wasn’t acceptable.

So, we turned it down.

Then, the docs came up with a new angle: a doctor supervised off-label test.

The groundrules: My wife would get treated with the drug, she would get extra supervisory care from an “A Team” of Georgetown doctors and, to top it off, Genetech — the drug’s supplier — would foot the entire bill.

We jumped at that opportunity.

Fast forward:: My wife is a 5+year survivor and is still going strong.


My point: our story is roughly analogous to the hydroxychloroquine situation.

In our case, there wasn’t control group evidence for our specific situation, but  there was plenty of compelling evidence on the periphery that the drug was a safe, life-extending treatment.

For us, taking the drug was a no brainer: lots of upside, minimal downside.

A lot like hydroxychloroquine for coronavirus patients.

When time is of the essence and survival is at stake, most patients are very willing to take some modicum risk.

My view: In life or death situations, scrap the bureaucratic barriers, and let doctors and patients decide.



  • The FDA approved off-label use of hydroxychloroquine HCQ) on COVID-19 patients.
  • On Sunday, the Coronavirus Task Force (which includes Dr. Fauci) voted unanimously to “surge” millions of doses of HCQ to hospitals and pharmacies in the corona hot spots
  • The Michigan governor launched a 3,000 patient doctor-supervised off-label test in Henry Ford hospital … making the HCQ available and gathering data on its efficacy. That’s not the same as a randomized, controlled test, but it’s prety darn close since there are plenty of corona patients in other places who won’t be having the drug prescribed.
  • Yesterday, Gov. Cuomo relaxed his “hospital use only” restriction on HCQ

That’s all good news…


Follow on Twitter @KenHoma

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