Policy, Surveys

The compassionate use poll: A lopsided vote favors posting policies, with plenty of fretting

We ran a snap poll yesterday on compassionate use policies, and there’s clearly plenty of positive sentiment among our readers backing some sort of easily accessible, online statement explaining each biotech’s policies on expanded access to experimental drugs.

The vote landed 70% for, 30% against posting a compassionate use policy online among the 149 votes we collected in the last 24 hours. But maybe that has a lot to do with the fact that a very similar sized group also said that such policy statements don’t apply to their companies. Among the 49 who said it did apply, 33 said their companies didn’t post a policy, against 16 who said they did.

That two-to-one ratio against, though, is still a far cry from the negligible 4% of small biotechs tracked by Avalere Health which posted such policies.

No, said one poll respondent: “The decision to try an experimental medicine is very complex (scientifically, ethically and regulatorily) and making it ‘easy’ is going to open a Pandora’s box that we’re not ready for.”

The ‘no’ camp also expressed concerns that a single adverse event from compassionate use could derail a program, as well as worries about the cost of providing drugs for small companies years away from making any money.

A lot of the comments for the “yes” vote echoed this sentiment:

“Posting compassionate use policies on your website does not promise that your case will be accepted, but at least it provides transparency on their policy and offers the potential to be treated, ” said one.

Yes, but. “Compassionate Use should only be offered for drugs successfully shown to be both efficacious and safe in Phase IIb/III trials. Prior to that, it is not advisable. Remember “first do no harm,” says another.

Indeed, one reader also steered me to BioMarin’s policy, saying that it’s common for such statements to restrict any compassionate use to drugs which have finished Phase III, when you have solid efficacy and safety data, but before the FDA has acted.

Art Caplan

Art Caplan

That’s a narrow window. NYU’s noted bioethicist Art Caplan tells me he’s seen compassionate use requests for drugs in everything from animal-stage testing on up. Some small companies will start entertaining requests as early as Phase I. And no biotech should expect the pleas to be restricted for use in the designated diseases being studied. It could be for a different dose as well as a separate indication, which is what caught Chimerix in the middle of an online mob as the parents of one young boy sought access for its lead therapy through a public lobbying effort that suddenly went viral.

Ironically, I checked out Chimerix’s web site and couldn’t find any mention of a compassionate use policy. The biotech, which has experienced a couple of setbacks with the program, declined comment.

Any time you get press coverage of a new drug, Caplan tells me, you raise the chances of compassionate use requests. And Caplan doesn’t sign on with the post-Phase III only camp. The important thing is making sure the drug has a well-established safety profile and at least a hint of efficacy. For Caplan, Phase IIb is where you hit the sweet spot for considering these requests.

And he adds that he wasn’t in the least bit surprised to hear that few biotechs have clearly enunciated policies spelled out online.

“If you have a company with one CEO and 20 people chained to a bench, website development is not a top priority,” says Caplan. But that doesn’t mean they shouldn’t do it.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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