Notes from Joe, February 2020

Notes from Joe

How Did We Get Here and Where Are We Going?
By Joe Pugliese

As noted here and everywhere else over the last several years, we have been blessed with an explosion on new, innovative therapies over the last 5 years. There are many more coming, including gene therapy, alternate rebalancing therapies and improved extended half-life products delivered subcutaneously. All of these advancements have been made possible through the existence of the comprehensive care network created by an act of Congress, at the request of the patient community. The pharmaceutical industry’s substantial investment in this area would not have been possible without the network of HTCs to aggregate patients.

After decades in the making, gene therapy is almost here. Unlike the almost here of 25 years ago or 5 years ago, almost here means before the year is over. Dr. Steve Pipe recently gave a webinar on gene therapy and the presentation is posted online; if you missed it I would encourage you to listen to it in its entirety. What struck me the most during the presentation was how far we have come and how much more is possible, with the focus on continuous improvement of both current therapies and therapies that suddenly are right around the corner.

Ironically, in this issue is an update on a program developed by the University of Michigan, with a grant from the Hemophilia Alliance Foundation, focused on teaching children with hemophilia how to self-infuse. Predictably it is the dedicated staff at a federally funded HTC giving their own time to not just teach these guys how to infuse but taking the extra time to make it fun. You will note the esteemed Dr Pipe in the photo – lower left on his hands and knees at the bottom of the pyramid.

It is clear to me that the only appropriate place for gene therapy is in the hemophilia treatment centers. The promise of this treatment and all of those to follow will only be realized if we stay focused on how we got here and where we would like to go.

Also in this Issue…

Alliance Update
· The More the Merrier: HTCs and Patients Wanted For Hill Day
· May Meeting for NEW HTC Staff
· Join us for an Informative Meeting at HTRS/THSNA in April
· 2020 Meeting Schedule

Washington Update
· Governmental Food Fight: GAO and CMS Sling 340B Related Publications Back-and-Forth?

Payer Update

Notes from the Community
· University of Michigan Launches Infusion U Program