Alternative endpoints

The FDA issued a new set of draft guidelines for pharmaceutical manufacturers the presents what has previously been referred to as “alternative endpoints.”

I posted about this in March. Here’s that previous post.

I caught a few minutes of the White House opioid summit yesterday and the phrase “alternative endpoints” caught my attention.

That’s Alex Azar speaking. He’s the Secretary of Health and Human Services.

Here’s what he said [emphasis mine]:

At the NGA (National Governors Association) we also highlighted two forthcoming Food and Drug Administration guidances that will expand and accelerate medication-assisted treatment. One of them will help the development of long-acting depot formulations like the monthly shot that was approved this past fall, and one that will open up new ways of assessing medically-assisted treatment effectiveness by looking at metrics besides just achieving abstinence, complete abstinence, so alternative endpoints.

I have two problems with that statement.

1. The idea that complete abstinence is the metric for medically-assisted treatment (MAT) effectiveness is just not true. I reviewed some of the most frequently cited evidence for MAT here. Most studies don’t even report information on abstinence.
2. I have no objection to measuring outcomes like reduced drug use, mortality, criminal activity, or disease transmission. Those are important public health outcomes. I also have no objection to providing treatments to patients who do not choose abstinence as a goal. However, these measures as endpoints for researchers and treatment providers? Creating systems focused on these as “endpoints”? If that’s what they are proposing, that’s neglect.

I do not know the contents of Secretary Azar’s mind and I do not claim to know his  motives. However, it’s important to be aware of his perspective. Here’s a portion of his bio:

From 2001 to 2007, Azar served at the U.S. Department of Health and Human Services – first as its General Counsel (2001–2005) and then as Deputy Secretary. During his time as Deputy Secretary, Azar was involved in improving the department’s operations; advancing its emergency preparedness and response capabilities as well as its global health affairs activities; and helping oversee the rollout of the Medicare Part D prescription drug program.

In 2007, Azar rejoined the private sector as senior vice president for corporate affairs and communications at Eli Lilly and Co. From 2012 to 2017, he served as president of Lilly USA LLC, the company’s largest affiliate.

That doesn’t mean he’s an evil Pharma shill, but it does say something about his worldview, his networks, and it may be considered a conflict of interests.