Notes from Joe
Update on the Alliance’s Work on an Intranasal DDAVP
by Joe Pugliese
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.
Hill Day Recap
by Johanna Gray
The Hemophilia Alliance held another successful virtual Hill Day in early May. We had 17 teams of advocates – including HTC staff members, patient advocates, representatives from NHF and HFA, and Alliance contractors and staff – participate in nearly 130 meetings with Congressional offices. We discussed a handful of issues:
- Overview of bleeding disorders and HTCs: With so many new Members and staff, we spent time on bleeding disorders and HTCs 101.
- Overview of 340B program and its importance to HTCs: We walked through the importance of 340B program income to allowing HTCs to serve all of their patients and the ways that HTC participation in the program might differ from other covered entities.
- Discussion of new access to care issue – alternative funding model: This is the newest access to care challenge, where ERISA plans are dropping coverage of specialty drugs and then referring patients to manufacturer patient assistance programs to get access to their drugs.
We did not have a concrete “ask” for these issues this year; we decided that going back to educating Members of Congress and their staffs was worthwhile since there are so many new faces this Congress, plus it allows for more Alliance members to participate. For the alternative funding model issue, we did ask for offices to let us know if they want to work with us on the issue.
We received great feedback from the offices with whom we met. There was broad interest in learning more about our community, support for HTC participation in 340B, and concern about the alternative funding model. We believe that the advocates also all had a great time – with meeting on Zoom this year (a platform very familiar to staff and Members of Congress, as well as all of us!), many more Hill folks participated on video, which made for better meetings.
In follow up to the Hill Day, we are now planning to reach out to the handful of offices that indicated an interest in working on the alternative funding model, as well as the staff for the House and Senate Committees with jurisdiction over the issue (House Education and Labor, and Senate Health, Education, Labor and Pensions). We are hoping to work with these staffers to develop the policy solution to the issue that could be advanced this Congress. This Hill strategy complements other efforts that the Alliance is undertaking with our partners, including outreach to payers, state insurance commissioners, manufacturers, etc.
Thank you so much to all of the Alliance members who made time to participate in the Hill Day! We look forward to hopefully seeing everyone in person for the Alliance Hill Day in 2022!
340B Program Updates
by Elizabeth “Issie” Karan
On May 17, 2021, Health Resources and Services Administration (HRSA) Acting Administrator Diana Espinosa sent letters to six pharmaceutical manufacturers (AstraZeneca, Lilly USA, Novartis, Novo Nordisk, Sanofi, and United Therapeutics). The letters may be found here (look for “HRSA Correspondence to Stakeholders 2021).
The letters state unequivocally that these manufacturers are in direct violation of the 340B statute because of policies restricting 340B Program pricing to covered entities that dispense medications through pharmacies under contract. As you may remember, beginning in July 2020, these manufacturers implemented policies which limited covered entities’ utilization of 340B drugs at contract pharmacies. The manufacturers took different approaches, with some entirely refusing to provide the 340B price on all products sold to covered entities and dispensed through contract pharmacies, while others limited sales by requiring specific data submissions or selling drug products only after a covered entity has demonstrated 340B compliance. The Hemophilia Alliance was able to get an exception to these policies for products used by HTCs.
Now, for the first time, HRSA has publicly stated that these policies have resulted in overcharges to covered entities. HRSA expects changes, stating that “[The drug manufacturer] must immediately begin offering its covered outpatient drugs at the 340B ceiling price to covered entities through their contract pharmacy arrangements…” “[The drug manufacturer] must comply with its 340B statutory obligations and the 340B Program’s CMP final rule and credit or refund all covered entities for overcharges that have resulted from [this] policy. . . . Continued failure to provide the 340B price to covered entities utilizing contract pharmacies, and the resultant charges to covered entities of more than the 340B ceiling price, may result in CMPs as described in the CMP final rule.”
This last sentence is important as HRSA is putting manufacturers on notice that, if they were to ignore their requests, they could be subject to significant fines. As a reminder, the 340B Program Ceiling Price and Civil Monetary Penalties final rule states that any manufacturer participating in the 340B Program that knowingly and intentionally charges a covered entity more than the ceiling price for a covered outpatient drug may be subject to a Civil Monetary Penalty (CMP) not to exceed $5,000 for each instance of overcharging. These assessed CMPs would be in addition to repayment for an instance of overcharging.
These manufacturers likely will make changes to their existing approaches to contract pharmacies in 340B to limit their financial exposure. However, this is not likely to be the end to challenges to the use of contract pharmacies in 340B. We will keep monitoring the situation and update accordingly.
Revenue Cycle & A/R Management Assistance
by Kimberly Wolverton Lackman
It is not what you bill – it is the reimbursement that you receive! Did you know Hemophilia Alliance (HA) offers Revenue Cycle & A/R Management Solutions that are included in your annual membership dues?
We lead and facilitate mutually beneficial dialogs between the HTC, Brokers, and Payers, including action items related to Amendments, Appeals, contract language clarification, high dollar escalations, individual Letters of Agreement resolution, and other complicated reimbursement matters. We assist with A/R analysis along with developing and enhancing HTC Revenue Cycle best practices.
We want to continue to expand on the revenue cycle management services offered; we need your input to determine additional ways we can help. At the end of May, we will be emailing members our Billing & Revenue Cycle Management Survey to gain a stronger understanding of your revenue cycle management processes, needs and issues. Please look for the Survey in your Inbox; we encourage you to respond so we may assess your needs and offer additional services. We will be holding technical assistance webinars on May 26th and June 8th. Survey responses will be due at the end of June.
Over the past 10 months, HA has helped member HTCs collect over $1.5 million in outstanding A/R. Please reach out to our Manager of Reimbursement & Payer Marketing, Kimberly Wolverton Lackman at email@example.com, to discuss how we may be of assistance. We work for you!
Notes from the Community
A. ANNOUNCING: HEMOPHILIA ALLIANCE FOUNDATION 2nd-ANNUAL “LARGE” GRANT
The Hemophilia Alliance Foundation (HAF) is excited to offer its 2nd-annual funding opportunity for a “Large” Grant. This funding will allow an organization to fund a larger-scope project than what they are able to do with current HAF smaller grants. This will be a one-year grant with projects completed within the year.
The Grant Guidance and online application form are available here: https://hemophiliaalliancefoundation.org/grant-information
The goal of this grant is to encourage larger scale, creative, and unique projects, as well as to serve the community with resources that will improve the lives of patients with inheritable blood disorders. With this larger funding amount, the “Large” Grant aims to encourage projects that are larger in scope, unique, and generate resources that can be shared with the whole community.
- A non-profit 501 (c) (3) organization
- An organization that has a history and documented track record of serving the inheritable blood disorders community
- Completion of the application with all required documents attached
- The grantee will be responsible for a conference call meeting with HAF board members during and/or after project completion to discuss the process (e.g., what worked, what could be improved)
- These funds may not be used in conjunction with grants from the Hemophilia Alliance.
Collaborative projects between more than one organization are encouraged and given high priority.
Grant funds will NOT be awarded for the following costs:
- Administrative or overhead costs may not exceed 8% of budget
- Basic, laboratory research, or related equipment
- Underwriting or sponsorship of fundraising events
- Construction costs
- Recurring costs, such as:
- Office rent and utilities
- Monthly phone bills for the applicant organization
- Salaries of staff not related to the grant application
Applications are currently being accepted through June 30, 2021. The project period will begin on September 1, 2021 and conclude by August 30, 2022.
If you have any questions about this “Large” Grant process, please contact the Hemophilia Alliance Foundation Grant Committee Co-Chairs, Amy Marquez, at firstname.lastname@example.org, or Stephanie Raymond, at email@example.com.
B. HFA To Host Mental Health First Aid Training Opportunity
Hemophilia Federation of America (HFA) is pleased to offer free Mental Health First Aid training through an internationally renowned program by the National Council for Behavioral Health as part of HFA’s 2021 Mental Health and Wellness Initiative.
HFA will be offering four trainings throughout 2021 for bleeding disorders community members ages 18 and over. The training is also open to the staff of HFA’s member organizations and hemophilia treatment center staff throughout the country.
The training includes a two-hour pre-work commitment, plus a four- and one-half-hour live session online. Participants who complete both sections of the course will receive a Mental Health First Aid certificate indicating they have been trained in recognizing the signs and symptoms of common mental health and substance abuse challenges, how to have an effective conversation about those challenges, and where to direct people for mental health care and support. Click here for more information about HFA MHFA trainings.
The next training cohorts will meet on the following dates:
- Saturday, June 26th
- Saturday, September 25th
- Saturday, November 13th
Mark your calendar. More details will be shared as the dates approach.
Team Alliance Contact Information
We work for you! Please don’t hesitate to contact any of us with any questions or concerns: