Notes from Joe, May 2021

Notes from Joe

Update on the Alliance’s Work on an Intranasal DDAVP
by Joe Pugliese

Dear Community:

In July 2020, Ferring Pharmaceuticals, announced a voluntary recall of all batches of STIMATE nasal spray (a solution of desmopressin acetate used to control bleeding in patients with mild hemophilia A and/or type 1 von Willebrand disease). The US recall was part of a global recall of the product, initiated when Ferring detected out-of-specification assay results in some vials of STIMATE marketed outside the US. By letter dated February 3, 2021, Ferring noted that it had completed its investigation, was working to address the issues, and advised our organizations that it expects to restart manufacturing, making first deliveries of STIMATE to the market no earlier than in the second half of 2023.

The timing of the letter was ironic, earlier the same day I had sent an email to all the Alliance member HTCs asking how the shortage was affecting their ability to care for patients and how many patients were in need of product. I received 40 responses within the first three hours. The respondents identified upwards of 800 patients and a variety of work arounds that clinicians across the country were using to fill the gap. The work arounds showed just how imaginative and dedicated the HTC staff members around the country are when it comes to caring for their patients. They were however work arounds and none were ideal for meeting patient needs. The end of February I presented to the MASAC board on the work we had done to try to find a suitable alternative for the intranasal spray. Jim Jorgenson, who serves on MASAC, put me in touch with STAQ Pharma.

After a series of exploratory discussions and a site inspection by Paul Limberis RPh and Steve Powell from the Rocky Mountain Hemophilia Treatment Center in Denver, the Hemophilia Alliance Board agreed to pay for the upfront costs of having STAQ develop a Stimate “biosimilar.” I’m pleased to report that they have successfully done exactly that and have product up on stability. We expect to have the first vials of DDAVP intranasal spray available in mid – July. The product will only have about 60 days dating at launch. Steve Pipe has volunteered to have MASAC organize a system to triage product availability in the early stages, to ensure patients going to surgery and those with the most consistent need get access to the product first. This will also allow us to minimize waste.

There are a number of additional issues we need to address over the next 60 days. STAQ is presently licensed in 26 states (see map). The Hemophilia Alliance, Hemophilia Federation of America, National Hemophilia Foundation, and World Federation of Hemophilia will send a letter asking for expedited license review so that patients across the country will have access to the drug. We will also contact HTCs and state and local chapters for their assistance in relevant states. We will be providing everyone in the targeted states with the appropriate contact information. We will be having a similar effort in reaching out to payers. It is safe to assume reimbursement in the near term will be challenging but I think with a concerted effort, we can benefit the patients who have been missing this product. Please see these video highlights from our May member webinar.

Also in this Issue…

Washington Update
· Hill Day Recap

Legal Update
· 340B Program Updates

Payer Update
· Revenue Cycle & A/R Management Assistance

Notes from the Community
· Announcing: Hemophilia Alliance Foundation 2nd Annual “Large” Grant!
· HFA To Host Mental Health First Aid Training Opportunity