Looking Back, Planning Ahead
By Joe Pugliese

As is typical in our December Newsletter, most of this issue will be focused on looking back over the past year and planning ahead for 2021. But before we dive deeper into all we’ve been through and accomplished this year, I want to give a very exciting breaking news highlight for the community. What makes this particularly nice is it was a community effort. Led by Johanna Gray and Ellen Riker, HTCs, local and national patient organizations and our manufacturing partners coalesced around this single critical issue for Medicare patients in the bleeding disorders, who needed rehabilitation after surgery or injury.

Congress Enacts the Hemophilia SNF Access Act: Congress included the provisions of the Hemophilia SNF Access Act in HR 133, the Consolidated Appropriations Act of 2021, which means that starting on October 1, 2021, skilled nursing facilities will be able to bill separately for bleeding disorders treatments provided to Medicare beneficiaries. Congress passed the bill late on December 21st and it now goes to President Trump for his signature over the next week. This has been a long-time advocacy goal for the community, and we are thrilled that the bill was enacted this year!

Now, to our regular year in review….

2020 is finally coming to an end, although I am afraid that the hangover is going to be nasty and prolonged. The first vaccines are now shipped and being administered, and I hope that more vaccines and more doses get to the market in short order. It was a year we would like to forget and never will. We give thanks to for all the essential workers, healthcare providers, policemen, firemen, garbagemen, trucker, retail employees, our military, postal workers, UPS, FedEx, and anyone else I missed. They have all performed heroically during the craziest year ever. Our manufacturing partners performed basically flawlessly, our early concerns in March about potential product shortages never materialized.

Closer to home, 2020 was also the year that gene therapy for hemophilia was going to be a reality, then wasn’t or at least not yet. Just this week, UniQure announced that the FDA has placed a clinical hold on its HOPE-B hemophilia gene therapy trial due to a patient enrolled in the trial being suspected of developing liver cancer. They are beginning an investigation into whether the patient does in fact have cancer, and if he does, whether it is related to receiving the treatment. You can learn more in their press release, in the recording of a call they held on the issue on December 21, 2020, and on a new website they have created to answer questions. The good news is that gene therapy will eventually get here, and, in the meantime, we have many excellent therapies available that are increasingly effective and more convenient to use.

On a brighter note, HTCs reimagined what outstanding clinical care looks like in a pandemic and beyond as we zoomed into the world of telemedicine (thank you, Roshni, for sharing your insights on one of our first weekly webinars!). While nothing will soon take the place of hands-on clinical care, virtual everything is not going away ever.

I am also proud that the Alliance Pharmacy never missed a beat in supply life-saving products to centers across the country. The Alliance suspended all business travel on March 12th. We have the benefit of being a virtual organization by design. We quickly set up weekly webinars and weekly team meetings aimed at keeping us and our members connected and informed. Like so many others, we pivoted to virtual and held member meetings and even hosted a very successful virtual Hill Day.

The promise we made to the community years ago to reinvest back into the community was also very apparent. The Alliance Board approved $500,000 in emergency patient assistance funding in March when we saw many of our community members were going to need a hand up to cope with the economic crises. The Board then approved an additional $500,000 in funding for a matching grant program with NHF focused on helping the local chapters weather the crisis.

After decades of traveling across the country and around the world, I suddenly found myself home. I find I do not miss the schlep to the airport, nor do I miss boarding the plane, countless Ubers and too many hotel rooms. I do miss the personal contact with colleagues and friends. There are people I have seen once or twice a year forever. It is those connections that make this community so special and unique; the silver lining of chronic disease.

We are looking forward to being back on the road as soon as it is safe – hopefully, next year, though it may be too early to tell. A good rule of thumb is to hope for the best and plan for the worst. Regardless, our focus for 2021 is to make sure that Alliance members have the resources and the community support you need to care for patients in the bleeding disorders community. We wish you good health and a prosperous and safe 2021.