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As patients clamor for greater access to experimental medicines, a survey released Tuesday finds that just 19 percent of 100 drug makers publicly post policies about their programs for obtaining these drugs, which are known as compassionate use. Moreover, only one of those companies posted information about specific procedures for making requests, and this company did not list any contact information.

More specifically 52 percent of the 25 companies with a market capitalization of greater than $10 billion analyzed by consulting firm Avalere Health disclosed policies on their web sites. By contrast, 14 percent of medium-sized companies — those with market caps between $1.5 billion and $10 billion — posted details and only 4 percent of smaller companies, with market caps of less than $1.5 billion, did the same.


The findings underscore arguments by a growing number of patient advocacy groups and lawmakers that the process for gaining access to experimental medicines is difficult, a complaint that has generated criticism of the US Food and Drug Administration and sparked social media shaming campaigns of some companies. The issue has, in turn, prompted federal and state legislation to alter the process.

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  • It is impossible for a drug company to craft a one size fits all policy for compassionate use. The protocol depends on many things, including size of patient pool and availability of drug, since many times supplies are limited and by definition therapy must be rationed based on demand. I ran a compassionate use program for NYHA Class 3 and 4 CHF for a novel vasodilator in the 1990’s. The drug was being discontinued so supplies were not abundant. We had a specific protocol for that drug alone. Each new compassionate use drug requires its own set of inclusion criteria based on the above criteria. Even if there were to be a general policy companies would be reluctant to publish it lest they be besieged with phone calls, which happen soon enough anyway once doctors know of its availability.

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