Notes from Joe
Harmony in Hemophilia
by Joe Pugliese, President and CEO
When we started the group purchasing organization in 2005, we promised the community that we would invest in supporting them. This support has taken many forms. We provide funding to the ATHN data managers, NHF Advocacy, the USHTC network, Hemophilia Federation of America, Hemophilia Alliance Foundation, World Federation of Hemophilia, disaster relief and lobbying efforts on behalf of the bleeding disorders community.
Last year we broke new ground and funded the development of a DDAVP NS to meet a critical need for patients. We also co-funded the publication of Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders. Our lobbying efforts paid off with the introduction, passage, and implementation of the Hemophilia SNF Access Act, which will facilitate access to skilled nursing facilities for Medicare beneficiaries with bleeding disorders. In 2021, we were also national sponsors for NHF’s Unite Walk campaign. Karen Bowe, our Director of Community Relations, led a record setting effort by HTCs across the country in raising money for Chapters nationwide.
Also last year we signed a memorandum of understanding with the Hemophilia Federation of America. The memorandum outlines the importance of the HTCs’ gold standard of care. HFA committed to educate the community about how critical these centers are to the well-being of the patients in the bleeding disorders community. The effort is already paying dividends with this article having just been published in HFA’s Dateline Federation magazine. An excellent article, it highlights the history, challenges, and successes of the HTC network. Look for more information during the year.
While this is all well and good, there is much that remains to be done. The health care environment is quickly changing with new therapies that promise great advances yet represent challenges for how to fund the treatment center network to ensure access to care in the future. We created the HTC Growth Initiative for a subset of our members in the fall of 2021. The response has been tremendous, and we are looking at sharing the teachings from this series of workshops. You can expect to hear much more about what we have learned and programs to assist HTCs in meeting the challenges they face.
One program we had already developed was the New Member meeting. We have long heard that staff turnover is a significant challenge for HTCs across the country. We had scheduled a meeting for new HTC staff last year but had to cancel because of the pandemic. Earlier in February, we had an in-person meeting with 55 participants from across the country. I can safely say everyone had a wonderful time. It is hard to estimate what we have missed by not being in Real Life. The presentations from the Alliance staff and our manufacturing partners were informative and collegial. We want to thank Biomarin, CSL Behring and Sanofi for their support and participation.
Grants Management that Makes Sense
by Elizabeth “Issie” Karan, Legal Counsel
Hemophilia Treatment Centers (HTCs) often must grapple with grey areas when it comes to compliance with the rules for their federal funding. The Hemophilia Alliance team works to demystify these guardrails and steer HTCs towards effective operations. We want to highlight an area of grants management that often creates confusion.
Many HTCs are lucky enough to accrue a balance of program income during their grant period. This is not common for federally funded grants. As a result, many accountants familiar with other grant programs expect HTCs to zero out their accounts at the end of the fiscal year. Because revenue cycles involving high-cost therapies can be volatile, HTCs should consider prudent accounting practices. The Uniform Grants Guidance allows federal grantees to carry forward balances to subsequent periods of performance with permission from their awarding agency. The Maternal and Child Health Bureau (MCHB), which oversees the regional HTCs and the grant program more generally, has the authority to allow this practice.
At 45 CFR § 75.308 (d)(3), Revision of budget and program plans, the UGG states:
“… HHS awarding agencies are authorized, at their option, to waive prior written approvals… [s]uch waivers may include authorizing recipients to … [c]arry forward unobligated balances to subsequent periods of performance…”
If HTCs do not currently carry forward unobligated balances or receive pressure not to, we recommend that you speak with your regional grantee about your policy. HTCs should embrace grants management policies that sustain their comprehensive care model for generations of bleeding and clotting disorder patients to come.
Join us for the Alliance Hill Day!
by Johanna Gray, Advocacy Consultant
We are excited to be planning for the 2022 Hemophilia Alliance Virtual Hill Day in April, 2022! All Alliance members and their patients are invited to join us to advocate for the bleeding disorders community from home. Here’s what you need to know:
What: We will schedule phone/video calls on your behalf with Congressional offices. The benefit is that it will be easy for everyone to participate from home or work, without having to come to DC!
When: Tuesday, April 5, 2022. We will notify you as meetings are confirmed but ask that you do your best to block the whole day from 9-5 eastern (though don’t worry West Coasters, we’ll make sure that no meetings start that early!). We will notify you when your meetings are confirmed so you can free up any other times besides your scheduled calls.
Why: We will be educating Senators and Representatives and their staff members about bleeding disorders, HTCs, and the importance of the 340B Program to our community. We will also be discussing two affordability and access issues affecting the community – copay accumulator adjustors (we’ll be lobbying for the HELP Copays Act) and alternative funding (we’ll be educating offices about this emerging issue).
Note: This means that we will be lobbying in our meetings. That means that HTC staff members cannot do these meetings on regular work time – you would be required to take vacation or charge the time to a different cost center. Again, your time spent on calls with offices will be only a few hours that day. If you have any questions or concerns about your ability to lobby, please contact me and we can discuss!
Who: We hope that many Alliance members will participate! We encourage you to also recruit a patient from your center to join you.
Next Steps: Please click here to register to participate. The deadline for registration is March 7, 2022. The Alliance will start scheduling meetings on your behalf. We will be holding a training webinar for all participants a few weeks before the event to share logistical and substantive details that you will need to know to make your meetings successful.
Schedule issues? If you can’t participate in meetings on April 5th and are willing to send emails to your Congressional offices, please register via that option on the form. We can provide you with contact info and a sample email to use.
Any questions? Please contact me at email@example.com or 703-304-8111 with any questions.
Request for Coverage of Desmopressin Acetate Nasal Spray (DDAVP)
In August of 2021, in a combined effort with the National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA), we submitted a Desmopressin Acetate Nasal Spray (DDAVP) letter to all payers, PBMs, and brokers for which the Hemophilia Alliance has a relationship. The letter outlines the request for coverage of DDAVP, including the necessity of the drug to replace the voluntary recall of all batches of Stimate Nasal Spray. In particular, Exhibit A references dosage, corresponding NDC number, J code, WAC and AWP. As previously discussed STAQ is continuously updating their stability testing and extending the dating on DDAVP NS, current inventory has an expiration date of 6/28/2022. We expect to have 12 month dating by the middle of 2022.
For Medicaid claims:
As a rule, Medicaid agencies are not allowed to cover drugs that do not have a contract with CMS to pay rebates for each unit used and paid for by Medicaid. If the rebate contract is not in place, CMS will not provide matching funds (called federal financial participation or FFP) to the states for these products. As with many things, there are exceptions. Drugs that are used in compounding a prescription for Medicaid patient are exempt from this requirement. (Also, a state may choose to pay for the product with all state funds which is also allowed.)
In the case of the STAQ product, while it is part of the exception, it does not have the “look and feel” of a traditionally extemporaneously compounded product. Normally these compounded products are required to use a specific claim form that requires the pharmacist to list the active ingredients and the main “carrier” products. They also don’t have a unique NDC. And finally, the 503B outsourcing process approved and monitored by the FDA is relatively new and not familiar to many state pharmacy programs.
If you ask you Medicaid agency to cover the STAQ DDAVP product and they resist, please remind them of the letter. We sent to them last summer before the first batch was released explaining all of this in detail before the first batch was released. If there are still issues, feel free to provide George Oestreich with the detail of the state encounter. He will be happy to assist if you.
Additionally, there seems to be some confusion about STAQ Pharma’s status below is brief checklist describing their operation.
- STAQ Pharma is a FDA registered 503B Outsourcing Facility. While STAQ prepares compounded products, our process is nowhere near the same as what you might think of relative to a “local compounding pharmacy.”
- STAQ Pharma adheres to and are inspected to the Current Good Manufacturing Practices standards (cGMP). cGMP is the same manufacturing standards that generic and branded drug companies adhere to and are inspected by the FDA. STAQ is also inspected by the FDA (and by State Board of Pharmacy like California and by Hospital Systems).
- Not only are STAQ’s products produced according to the FDA cGMP standards, their product formulation and stability work is performed and governed by those same standards. The cGMP standard is a very difficult standard to meet and maintain. STAQ has had 2 FDA inspections, their first one is already closed, and was closed in what they believe is record time for our industry. Their 2nd inspection happened this past October and they have completed their responses to FDA and await communication from the FDA on whether there are any next steps, or we proceed to closure.
- Their Stability protocol and testing has recently passed the 180-day mark, so that is how they are dating the DDAVP product now. Their final timepoint for the product is 1 year.
- Their Stability work is not simply potency over time. STAQ does a complete and full stability test of their drug products including container closure tests, microbiology tests, endotoxin tests, spray geometry, spray droplet size (as is required for Intranasal delivered products).
- STAQ’s ownership structure includes some of the US most recognized names in Health Systems – University of North Carolina, Froedtert Health System and College of Medicine of Wisconsin, Children’s of Colorado, Nationwide Children’s, Cincinnati Children’s, University Hospitals, and Louisiana Children’s and Medical Center.
- The 503B Outsourcing Facility designation and regulatory category is relatively new in the United States, having only been developed through Congressional Act in 2014 and FDA Guidance becoming more clearly defined in 2016 and 2018. STAQ is most definitely not a local compounding pharmacy. They produce drug products in ready to administer forms that are very much needed by our nation’s healthcare system, including drug products on the FDA 506E shortage list. DDAVP is on that 506E list.
- The way the FDA categorizes their products, internal to Health and Human services, is also changing. Internally, the systems FDA uses to track their products are using the internal category designation of their products as “Outsourcing Facility Compounded Human Drug Product (Exempt From Approval Requirements)”. This was only recently announced by the FDA so it may take a year or two for that to work its way into CMS and other payor related systems and formularies.
If you have any questions, please contact a Team Member of the Hemophilia Alliance.
1st Annual Hemophilia Alliance Pharmacist CE Conference
by Stevan Mizamakoski, RPh, Indiana Hemophilia and Thrombosis Center, Pharmacist Conference Committee Member
As many of you know the 1st Annual Hemophilia Alliance Pharmacist CE Conference was held in San Diego at the end of January. The program was offered to pharmacists of HTCs across the country and included an in person and virtual option for attendees. The conference was a great experience for everyone and far exceeded expectations. For those attending in person, a networking reception was held on January 26th that enabled us to meet new people and see some familiar faces. The conference consisted of ten live presentations, providing fourteen hours of live continuing education, from eleven amazing providers.
The experience was fantastic, and we are already looking forward to hosting the 2nd annual conference next year. In addition to the informative presentations the event allowed us to make connections with pharmacists from a variety of different centers, discuss concerns we are facing, and share best practices. I would strongly encourage all Alliance member pharmacists to plan on attending a future conference.
I would like to thank all of our presenters for taking the time to create and present such great and impactful information. I would also like to thank The Hemophilia Alliance for deciding to host such an ambitious event. For those of you like me that do not plan events like this, it is a pretty big undertaking to host a live and virtual event, consisting of in-person and virtual speakers, with approved ACPE credits. I would also like to thank Angela Kellum and Resham Sahijram for volunteering their time and expertise to assist in planning the event.
by Mark Plencner, Committee Chair
Our thanks again to our outstanding speaker panel:
Steven Pipe, MD; Robert Sidonio, MD; Kim Mauer, MD; Sonia Nasr, PHD; Carol Johnstone, MA; Duc “Bobby” Tran, MD; Maissaa Janbain, MD; Angela Kellum, PharmD; Nicole Reynaud, RN; Jennifer Maahs, PNP; Regina Butler, RN BC
Our thanks also go to:
George Oestreich, PharmD and Angie Johnson of GLO Associates and Sean Singh and Kiet Huynh, Hemophilia Alliance technology experts
And our sponsors, Takeda and TAP
Team Alliance Contact Information
We work for you! Please don’t hesitate to contact any of us with any questions or concerns: