by Beth Mole
Ars Technica / 2017-02-13 19:33
In an unexpected move, Marathon Pharmaceuticals announced Monday that it is pausing the roll-out of its now FDA-approved drug for Duchenne muscular dystrophy—which has an $89,000-per-year list price.
The announcement comes in the wake of intense outcry from patients, the public, and lawmakers over the drug’s eye-popping price, which Marathon announced last week.
The drug, deflazacort, is a steroid treatment that slows the progression of Duchenne, a rare, devastating neuromuscular disorder that leaves mostly boys unable to walk by their teens (it’s X-linked recessive). The disorder also shortens their lifespan to 25-30 years.
Deflazacort is an old drug that’s available as a generic overseas. Previously, US patients could import a year’s worth for around $1,200. But on Thursday, Marathon announced it had received FDA-approval to sell its version in the US under the brand name Emflaza. Because the company had it approved through the FDA’s ‘orphan drug’ program—that is, a priority review for drugs that treat rare disorders—the company now has the seven years of market exclusivity that comes with that program. And, also part of that deal, the company has received an FDA voucher to expedite a future review, which is intended to act as an incentive for pharmaceutical companies to develop orphan drugs. The vouchers are transferrable, though, and often sell for hundreds of millions of dollars.
Anticipating blowback from the drug’s steep list price, Marathon quickly explained that rebates, discounts, financial programs, and insurance coverage will make sure patients can get the drug at an affordable price. The company said it expects patients will pay $20 out of pocket, or less.
Still, none of Marathon’s actions or assurances have set well with patients, advocates, and lawmakers.
As Ars reported last week, health experts worry that some patients will slip through the cracks and be stuck with a crushing bill.
Dana Edwards, a New Jersey mother whose 12-year-old has taken deflazacort since he was five years old, told Kaiser Health News Monday that the company was: “robbing my insurance company.”
Also Monday, Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) wrote a strongly-worded letter to Marathon. In it, the two legislators chastised and questioned the company over its pricing and use of the “orphan drug” program.
“Marathon’s apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89,000 a year is unconscionable,” Sanders and Cummings wrote in their letter. “Exorbitantly pricing potentially life-saving medications that should be widely available for a fraction of the price hinders patient access and drives up costs for the entire health care sector.”
In their letter, the two lawmakers requested information about how Marathon priced the drug and how much money they stood to earn.
In response to the general outcry, the company said it would put a hold on its plans to roll out the brand-name drug until it could hear and review concerns.
In a statement, Marathon Chairman and CEO, Jeff Aronin said:
“Since last week’s approval, we have heard both support from the community and concerns about how the pricing and reimbursement details will affect individual patients and caregivers, such as how it affects coverage of other components of Duchenne treatment. Based on these questions, today we are announcing:
1. We are pausing our commercialization efforts in order to meet with Duchenne community leaders and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on the resulting plan of action
2. We will continue to maintain our Expanded Access Program for patients receiving EMFLAZA
3. Patients currently receiving deflazacort from other sources may continue to have that option
The statement went on to explain the drug’s pricing:
When we set the price of EMFLAZA, we considered the following: the resources we invested to get to this point, the resources needed to complete the phase 4 clinical studies and FDA post-marketing study commitments, the ability to fund future research and development, and the ability to ensure broad patient access through insurer reimbursement and our own patient assistance programs. There is confusion that this is a generic drug. In the United States, FDA considers deflazacort a new drug, and we had to get it approved. Our tablets are manufactured in the United States.
The resources we invested were substantial and we don’t expect to recoup our investment for several years, and we have only seven years of market exclusivity.
… I want to assure caregivers and others that our preliminary meetings with the payer/insurer community have gone well, and many have acknowledged the price was appropriate given the very small patient population…
We will continue the discussion on pricing and access with you until there is a full understanding of how pricing decisions directly impact the revenues needed to fund past, current, and future research as well as opportunities to purchase new therapeutic treatments.