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A rock and a hard place

When insurance companies exclude drugs from their coverage
A rock and a hard place

Last month the FDA approved Sarepta's Duchenne muscular dystrophy (DMD) drug known as Exondys 51. The noteworthy aspect of this approval was that the FDA committee was decidedly split on whether the data presented as part of Sarepta's submission actually demonstrated a benefit in patients. One reviewer went as far as to refer to the therapy as an 'elegant placebo'. Others were far more blunt in their assessment of the data. And yet others still thought the data actually warranted the therapy's approval.

And then mere days after this decision, Anthem (one of Americas's largest health insurers) declared that they would not cover Exondys 51 until further data had established its efficacy - effectively relegating the drug to investigational status in its own eyes and ignoring the FDA approval. They went on to add that the therapy is not 'medically necessary'.

It's bad enough that the FDA can't unanimously agree on a drug approval (which has always been the public's litmus test that everything is good with the submission), but these things happen. In 2013 the FDA approved a powerful painkiller called Zohydro - a pure hydrocodone drug. And immediately there was a swell of backlash because the drug's manufacturer had not developed a tamper-resistant packaging solution for Zohydro. Without the tamper-resistant packaging, critics argued that the FDA was ignoring the high potential for opioid abuse and the ramifications that came along with that. The FDA's own Anesthetic & Analgesic Drug Advisory Committee (ie expert panel) voted against making Zohydro available. And it wasn't close. It was 11-2 against approving Zohydro. The expert panel cited 'achievement of narrow targets for safety and efficacy' and the potential for abuse as the main reason(s) for overwhelmingly voting against approval. A dissenting vote here or there is nothing new.

What's troubling are the whispers and the questions over the data and over basic issues around efficacy and safety for Exondys 51. Achievement of narrow targets and the potential for abuse are very different issues from 'I'm not even sure this thing works'. This leaves the door open for finger-pointing, hand-wringing and precedent-setting. The FDA has gone to great pains to assure us that this Sarepta approval process is an outlier. In fact, a senior CDER official delivered a strongly worded message to that effect in front of a group at the National Organization of Rare Diseases meeting in Washington last month. OK. If you say so. But one has to wonder how a drug with this type of study design was allowed to be submitted in the first place and why it was even reviewed: it had a small sample size (12 patients), used a questionable biomarker as a surrogate marker for clinical benefit and failed to conduct a larger trial with a placebo arm as requested by the FDA.

Do we have the right to tell people with an illness that they can't try a therapy if they can afford it?

And then amidst this backdrop of mass confusion and hysteria, lie the insurance companies and pharmacy benefit managers who now refuse to cover this drug. Anthem's insurance pool covers 38 million American lives. And others will soon follow with their refusal to cover this therapy. Now we have entered the realm of the 'slipperiest of slopes.' Now we are at the point where private companies have motivations for excluding drugs on their coverage lists that are in direct conflict with their for-profit mandate. Now we begin to wonder when the next insurance company will disagree with the data on a drug's approval and limit - or completely exclude - coverage for its membership. And we begin to wonder whether this exclusion will be for profit-driven reasons or true scientific reasons.

Of course I'm sensitive to the orphan drug aspect of this story and the complete lack of alternative therapeutic options for patients with DMD. But shouldn't that only matter once the efficacy and safety are not in question? Maybe. Maybe not. There's a larger policy issue at play here: to what extent, if any, do we have the right to tell people with an illness that is essentially a death sentence that they don't have the right to try a therapy if they can afford it. Remember that some members of the FDA committee reviewing the data on Exondys 51 thought the drug should be approved. And remember that patients with this form of the disease have absolutely no other therapeutic option.

Ladies and gentlemen, meet the 'rock' and meet 'the hard place'.

Article by
Rohit Khanna

is managing director of Catalytic Health, a healthcare communications, advertising and strategy agency. He can be reached at:

14th November 2016

From: Regulatory



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