The pricing controversy surrounding Duchenne muscular dystrophy (DMD) treatment

The latest events surrounding the pricing of a Duchenne muscular dystrophy (DMD) treatment

In the United States, a commonly prescribed drug used in countries such as the UK and Germany to treat the disease Duchenne muscular dystrophy (DMD), was approved by the FDA for use last month. The drug commonly referred to by its generic name deflazacort, was approved with a cost price of $89,000 per year under the new name Emflaza, a 90-fold increase compared to prices outside the US of approximately $1000. The FDA approvalunder the Orphan Drug Act which encourages the development of drugs for rare diseases, enabled the pharmaceutical company a seven year monopoly on the DMD indication for deflazacort, even though the drug has long been available as a generic in other countries. After an intense backlash, the company then sold the drug to PTC Therapeutics.

US pharma giants Marathon Pharmaceuticals, who were responsible for this considerable price hike, triggered widespread concerns amongst those affiliated with DMD, a chronic degenerative muscle disorder which mainly affects young boys. As a result, the pharmaceutical company then temporarily suspended the rollout just four days after its announcement, 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.”

For patients of DMD, a chronic degenerative muscle disorder which affects approximately one in 3600 newborn boys worldwide¹, deflazacort which is also known as Calcort, has shown to prolong movement, and preserve cardiac and respiratory function. It is most commonly prescribed off label for the treatment of DMD, and the most commonly used DMD treatment in countries such as Canada².

“While these incentives such as the ‘Orphan Drug Act’ and ‘priority review vouchers’ rightly exist to enhance new treatments for rare paediatric diseases, one can’t help but think they are being exploited when they are associated with a drug which has been around for decades, and at a fraction of the price.”

Attention then spread globally leading to the involvement of a US congressional inquiry on not only Marathon’s pricing decisions, but the FDA’s approval of the drug. US Senator Bernie Sanders and Representative Elijah Cummings expressed concerns in a formal letter to the FDA over decisions that were made based on research carried out years ago. “We write to express our concern and request information regarding the unusual circumstances surrounding the recent approval of a brand name version of the more than a 20-year-old drug, deflazacort,” Sanders and Cummings stated in their joint letter.

The additional scoring of a ‘priority review voucher’ led to further unease regarding Marathon’s motive for the price hike. The voucher allows companies who gain approval for paediatric orphan drugs faster approval for another drug in the pipeline. Marathon can either use the voucher for itself or sell it to another company. These vouchers have been known to sell for up to $350 million.

Sjaak Vink, founder and CEO of everyone.org, has stated that “while these incentives such as the ‘Orphan Drug Act’ and ‘priority review vouchers’ rightly exist to enhance new treatments for rare paediatric diseases, one can’t help but think they are being exploited when they are associated with a drug which has been around for decades, and at a fraction of the price.”

To add further speculation, Marathon announced the selling of the drug to PTC Therapeutics, for $140 million in cash and stock upfront, along with a $50 million sale bonus. In addition, PTC will be required to pay Marathon an annual sales-based payment starting from 2018.

PTC who are well known within the DMD community due to their Duchenne treatment drug named Translarna, are yet to disclose how much they will be charging for Emflaza. PTC stated that their goal is to “ensure Emflaza is studied, understood and available to any Duchenne patient who needs it, and we determined that this transaction is the best path for ensuring that will happen.”

According to a 2015 population-based study presented by the Centers for Disease Control and Prevention, about 2 in 10 (22%) DMD patients took deflazacort³. In recent years, the FDA has allowed DMD patients to import the drug. It should be pointed out, as Senator Tom Cotton did when addressing US congress, that many people with DMD are happy about the FDA approval, as now insurance companies may cover the cost— indicating that families from lower income backgrounds will now have access to the drug.

Whether this is the case and access to deflazacort will improve remains to be seen. What is evident however, is an increase in negative public attitude towards an industry already desperately seeking acceptance and credibility. While the world awaits PTC Therapeutics’ pricing announcement, the FDA approval and subsequent pricing of deflazacort has inadvertently created confusion and criticism. everyone.org, a real patient’s buyers club, was created to help families and patients who may be affected by approval and pricing constraints, access effective treatments at the best possible prices. It is through social enterprises like everyone.org that help patients who are at the forefront of these complex approval issues, receive the latest information and access to both innovative and effective medicines at the best possible prices.

You can find more information about Duchenne muscular dystrophy treatment on our Duchenne muscular dystrophy information page.

References

1.Falzarano MS, Scotton C, Passarelli C, Ferlini A. Duchenne muscular dystrophy: from diagnosis to therapy. Molecules. 2015 Oct 7;20(10):18168-84.

2. McAdam LC, Mayo AL, Alman BA, Biggar WD. The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy. Acta Myol. 2012 May;31(1):16-20.

3. Kim S, Campbell KA, Fox DJ, Matthews DJ, Valdez R; MD STARnet. Corticosteroid treatments in males with Duchenne muscular dystrophy: Treatment duration and time to loss of ambulation. J Child Neurol. 2015 Sept;30(10):1275-80.