The payer backlash against PTC’s deflazacort for Duchenne MD begins with some damning comparison shopping

PTC Therapeutics CEO Stuart Peltz

PTC Therapeutics has a few weeks to go before it wraps its $190 million deal to buy the controversial drug deflazacort from Marathon Pharmaceuticals and starts marketing the old steroid with a new FDA approval for Duchenne muscular dystrophy. But even before the biotech decides its price, the payer kickback against this drug has already begun.

Unless it’s virtually free, it’s clear that for now some don’t want to pay for it.

A new analysis from the Washington State Healthcare Authority also raises fresh questions about PTC’s repeated assertions — echoing Marathon — that they were investing in a development program in order to make the steroid more widely available, beyond the small group that they said had been importing it from overseas suppliers.

Officials at the Washington State Healthcare Authority started their policy review by doing some comparison shopping between deflazacort and prednisone, a generic steroid that sells in the US for about 5 cents per pill, or $55 a year. And that’s the price that the authority is considering in deciding the affordability and coverage on deflazacort for state Medicaid patients.

When Marathon Pharmaceuticals priced deflazacort at $294 a pill, or $89,000 a year — after Duchenne parents had been paying about $1,000 a year to import a supply of the drug — the news triggered the latest in a series of embarrassing drug price scandals.

Marathon CEO Jeff Aronin decided to duck and cover, “pausing” the launch, and then PTC came along with $140 million in cash, plus milestone money, and a solution to the crisis. Now PTC CEO Stuart Peltz is in the hot seat, lining up a new wholesale price, adopting the same argument that deflazacort has been available to only a small percentage of the patients in the US.

Once he prices and launches, US patients will no longer have legal access to the drug at discount prices from a UK supplier.

The WSHA, though, decided to do some fact checking ahead of PTC’s deflazacort launch as they consider their policy decision.

In their report, dated for an April 19 review, the authority found three small studies comparing deflazacort and prednisone. These studies provided “low quality” evidence that there is no evidence of any difference between prednisone and deflazacort in improving muscle strength and some low quality evidence from one tiny and possibly biased study that deflazacort would trigger less weight gain than prednisone — though there has never been a study on that endpoint to provide statistical significance.

What about Marathon’s claim, now echoed by PTC, that fewer than 10% of the patient population have access to deflazacort?

The authority’s policy report cites CDC figures indicating that 22% of the boys take deflazacort and 14% alternated between prednisone and deflazacort. The rest take prednisone. Those figures also fall more closely to an estimate by patient advocate Christine McSherry that at least 25% of the boys with Duchenne MD take deflazacort, possibly reaching as high as 50%.

These patients have been paying more for deflazacort largely because they believe their children do better, with less weight gain, than they would experience with prednisone — a belief which has also driven use outside the US. But many are incensed that Marathon took a cheap alternative and put it through a development program that left them with the approval and a priority review voucher as a bonus — probably for far less cost than the company had implied in claiming that it would take years to cover the cost of research.

“Prednisone is the lower cost, equally effective alternative,” concluded Ryan Pistoresi, PharmD, Assistant Chief Pharmacy Officer at the authority. And that’s why prednisone should be the preferred corticosteroid, with a case-by-case review for any special request for deflazacort.

PTC may have an approved drug to market in the US, after the FDA repeatedly spurned their drug ataluren. But their executive team faces a steep market climb.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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