Not Quite at the Finish Line Yet
By Joe Pugliese

Wow, after waiting 25 plus years for a gene therapy product for the hemophilia community, it never occurred to me that Biomarin’s product would be rejected by the FDA last week with a call for additional data on the durability of their product. If you’ve been traveling (unlikely) or if you just really, really took the week off, here are some links to more info, some of which include quotes from some familiar faces: NYT article, Stat News article, Biopharma Dive article.

The good news is we are blessed with a wide array of highly effective and safe products today, so the wait, which looks like it could be 24 months or so, will be very manageable. It is easy for me to say: I do not have hemophilia. But, I have spoken to many physicians and healthcare professionals around the country, who have dedicated their lives to constantly improving care for people with hemophilia and most agree that it is too soon. Gene therapy is unlike the broad array of fine products available today, since even the latest products have half life. Gene therapy is both irreversible and as has been discussed widely, will be “expensive” (while open to interpretation, the rumored price tags certainly raise the stakes). We have long believed that patients should have access to most appropriate product for them, that will never change.

Gene therapy just makes that decision-making process much more important and “final,” which is why it is a good reason to get it right the first time. I am sure the people at Biomarin are reeling today. In my past lives, I have been on the receiving end of some of these same messages. I also wear rose colored hearing aids; when you tell me no, I hear “not yet.” To all of the researchers and gene therapy companies that have done so much to get us to where we are today, I’d just say that the finish line is still so close, so please keep pushing!

The Alliance has been part of a gene therapy working group since the beginning of the year. It started pretty small, but now includes a group of about 40 clinicians from across the country, as well as NHF leaders. It is one of several working groups focused on making sure that as a community we are prepared to bring gene therapy to market the right way that ensures that patients get the greatest benefit from this exciting new therapy once approved.

We are also focused on making sure that all federally-supported HTCs are prepared to make gene therapy available to their patients. We believe HTCs are the most appropriate sites for the delivery of gene therapy and its follow-up care. There is no place or need for any middlemen. The Alliance has and will continue to build out in house capabilities so we be a full partner with any company bringing a gene therapy product to market.

It sounds like a broken record but gene therapy and all the promise it holds for the bleeding disorders community will be here soon. We will be ready to facilitate patient access to it through a well-funded, comprehensive treatment center network, which was created at the request of the patient community and will always be dedicated to supporting the clinical needs of the community including helping everyone get through these crazy times we are living in.

NHF has recently announced a COVID-19 bridge grant program. I’m proud that the Alliance was delighted to match the donation. See a letter here from our current and past Board members that went to all chapters receiving funds.