Notes from Joe, March 2023

Notes from Joe
  • Dateline: Washington DC
  • by Joe Pugliese, President and CEO

    NHF Washington Days was live for the first time since 2020. Turnout was strong with over 400 people from the bleeding disorders community turning out to lobby our representatives to support the community, including some folks from team Alliance and several people from HTCs.

    The first issue we tackled is HR 830, the HELP Copays Act, which was introduced Reps Carter (R-GA) and Barragan (D-CA). We were asking other representatives to co-sponsor this bill and for Senators to introduce a companion bill. The measure is designed to prohibit the use of copay accumulator adjustor programs. Why is this important? The annual out-of-pocket (OOP) maximum can be as high as $9,100 for an individual and $18,200 for a family. This means patients with chronic expensive conditions can look forward to paying ruinous copays every year, if insurers are allowed to pocket the copay assistance paid on behalf of a patient, and then turn around and demand the same amount from the patient.

    I spent the day with the Florida contingent. We were well-received at all the offices we visited, but one visit really stood out. Two affected women in our group who are constituents of Representative Posey (R-FL) were describing the challenges they face in part because of copay accumulator adjustors. In the middle of the discussion, Rep. Posey looked to his staffer and announced that he was going to cosponsor HR 830. Heather and Samantha immediately broke out in tears. I would say it was contagious. The difference between being well received and getting a firm commitment to sponsor the bill is huge, and shows how effective these advocates were!

    We also asked to maintain the current funding levels for the federal hemophilia programs at the CDC and HRSA, and to ensure that HTC participation in 340B is maintained (more on this shortly). Finally we asked for ongoing NHLBI funding. While we were in town President Biden proposed a $6.8 trillion budget, which makes the millions we’re seeking for our programs less than a drop in the bucket.

    Other breaking news while we were in town was the joint announcement made by PhRMA and NACHC (the National Association of Community Health Centers) saying they have formed a new organization, the Alliance to Save America’s 340B Program—ASAP 340B. The new group released a set of principles about how they intend to save the program. We have been asked by any number of organizations what we think of this development. First, let me say we are of course very much in favor of saving 340B. The at-risk population we serve absolutely depends on HTCs having access to 340B as noted above. However, we are extremely concerned about what we’ve heard so far and believe that there could be several harms to the bleeding disorders community.

    I do have a couple of questions. Why was all of this done in secret, without the involvement of other grantees like HTCs? Why are organizations with a long history of opposing 340B part ASAP 340B? Why did PhRMA not reach out to HTCs for input? It seems counterintuitive at best.

    As we learn more about this group it is clear they either don’t know or do not care about the negative impact their proposals would have on our community. You will be hearing much more about this over the coming weeks and how you can help educate policymakers about how they can really protect 340B, HTCs, and they patients you serve. But the first “to-do:” please plan on coming to Washington, DC for the Alliance Hill Day this June to make sure our voices are heard. See below for more info.

  • Product Update
  • by Joe Pugliese, President and CEO

    Aminocaproic acid / Brand Name Amicar
    On March 3, 2023, it was announced that Illinois-based generics firm Akorn had filed for Chapter 7 bankruptcy in Delaware, which affects us since they are the main maker of Amicar. We immediately reached out to the trustee assigned to manage the Akorn chapter 7 filing, the Office of Pharmacy Affairs, Mikart the contract manufacturers, and our now good friends at STAQ Pharma who brought DDAVP NS to market. (More on that below)

    We did not hear from the trustee nor Mikart. The Office of Pharmacy Affairs was very responsive, but this issue is outside of their preview. STAQ Pharma started looking into the feasibility of producing first the aminocaproic acid liquid then the tablets. Since STAQ is a 503B outsourcing facility, the product needs to be listed on the FDA drug shortage list to manufacturer the product. Len Valentino reached out to the FDA asking to have aminocaproic acid added to the drug shortage list. He made a similar request of FDA for Stimate.

    The FDA has been in close communication with the other manufacturers of aminocaproic acid and has received assurances that they will be able to supply the marketplace. If you experience difficulties sourcing the product, please let me know immediately. I can tell you that The Alliance Pharmacy has shipped all the inventory they had available.

    I shared this information with Miriam Goldstein, the Vice President of Public Affairs at the Hemophilia Federation of America, and she was very helpful in providing some additional contacts. I wrote to the accounting firm overseeing the bankruptcy filing with an offer to purchase all the remaining inventory of Amicar. We will of course let you know if anything comes of this request.

    As a reminder, DDAVP nasal spray is readily available from STAQ Pharma and you must purchase direct from STAQ if you plan on dispensing yourself. Where physician dispense is allowed you do not need to have a pharmacy license, they ship directly to the clinic using an NPI number.

    Reimbursement remains challenging although we have some successes. Optum has been very helpful in making sure patients have access to product; I wish I could say the same for CVS Caremark and Express Scripts. We have spoken to the FDA on several occasions about getting regular approval for the product, which would definitely help address the reimbursement challenges. Our efforts to date have not yielded any results. We have had individual Medicaid plans reimburse, but without FDA approval Medicare is yet to provide reimbursement.

    As a reminder, Dr. Amy Shapiro has provided us with a letter of medical necessity. It highlights the safety and efficacy of the product.

    It you have questions about ordering or our DDAVP challenge program, please email me at

    Many thanks to Len, Miriam, Amy and a host of others for your efforts to helping address these supply issues.

Also in this Issue…

Washington Update
· Register for the Hemophilia Alliance Hill Day this June!

Alliance Board Update
· Alliance Seeking Board Nominations

Alliance Update
· The Alliance Upcoming Events Webpage – A New Look & Feel

Payer Update
· HANS PPO Agreement 42 signed, 65 To Go

Notes from the Community
· Update on Partners Physician Academy
· Collaboration Updates with our National Organization Partners

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