Access to the Best Care
by Joe Pugliese, President and CEO

The Hemophilia Alliance hosted one of the more extraordinary meetings held in the bleeding disorders community, June 20 and 21st in Chicago. The meeting was extraordinary for several reasons. First, we had sent out an invite to all members, the three national patient organizations, the Regional Directors and Administrators, ATHN, and HRSA, with little notice and asked people to travel on Father’s Day. We had over eighty attendees, which included NHF and the Coalition for Hemophilia B (HFA was planning to attend but had travel difficulties), four regions, representatives from ATHN and 44 HTCs were represented, including twenty-five doctors, nurses, pharmacists, and administrative staff.

The key take away from this multi-stakeholder meeting is that gene therapy in the bleeding disorders community should come through the federally-recognized network of HTCs. It is the best way of assuring the most clinically effective and cost-efficient introduction of the therapy to the community. The meeting focused on how to make sure all centers were prepared to bring gene therapy to the patient community. There are follow-up meetings being scheduled at the upcoming NHF BDC in Houston in August (click here for more information) well as the Alliance member meeting October 2-4th. The Alliance is also developing a moderated blog on our website for members to discuss issues related to gene therapy. You can access it here.

On another note, it has been close to a year since the first batches of DDAVP NS, manufactured by STAQ Pharma Inc, a 503B compound manufacturer, were shipped. It has been a community effort, with NHF, HFA and the Hemophilia Alliance, working together to get the product covered by insurance and into the hands of the patients. Our work together has been as recent as last week, when after multiple contacts, Express Scripts confirmed to NHF that the product was added to the national formulary effective July 1, 2022. They are joined by Optum (who fills scripts by exception), Ingenio and Accredo, who have added it to their national formularies. One would think getting coverage for a product that is far more convenient, is 10 to 20 times less expensive and offers appropriate clinical coverage would be easy. Nevertheless, we know there is still work to do, but I’m heartened by the progress we’ve made together. Finally, you can access a document laying out lots of the billing details that can be helpful to you in negotiating with any payer here.

I’m also pleased to highlight the amazing work of the World Federation of Hemophilia in helping to meet the needs of people with bleeding disorders in Ukraine. You can see an update that WFH shared here and they expect that additional funds will also be sent to the WFH national member organization in Lithuania and to the HTC in Germany, which continues to welcome new patients/families who are emigrating there. We will share more details as we have them. You can also learn more about other WFH and Alliance activities in this article on the Travel Grant recipients at the WFH congress and this article on the IHTC fellowship program.

Finally, as always, please remember that the Alliance works for you. Please contact me with any questions or concerns about these or any other issues that your HTC is facing.