Page 4 - HFA_Dateline_2017_Q2_Fall_Special_Edition

EXECUTIVE CORNER
Dear Community Members,
When my son Nicholas was born 14 years ago, gene therapy
was the subject of conversation in the community, and longer-
lasting factor was barely mentioned. Over the years, the
emphasis seemed to flip and, for a variety of reasons, gene
therapy took a bit of a back seat while new, longer-lasting
factor products were introduced to the market. Recently gene
therapy, along with other novel therapies such as RNAi and
bi-specific antibody, have reestablished themselves as the
subject most on community members’ minds.
In the fourteen years that I’ve been fortunate to have Nicholas
in my life, 18 new products have been released onto the
market. As a mom trying my best to navigate my child’s health-
care needs, I’ve felt that the amazing growth within the indus-
try has added to the everyday challenges that hemo-moms and
-dads face. And as the product options have changed, so too
have my thoughts on those options. When Nicholas was a baby
starting prophylaxis, my husband and I just wanted to find a
product that worked well for him while we prayed for no inhibi-
tor development.
Once we were several years into three-days-a-week prophylax-
is, a long-lasting product came to market and I thought without
hesitation that I’d want Nicholas to try it as soon as possible. deep dive into how clinical trials work (p. 22), to the infograph-
Even if it cut down just one infusion per month, I thought it
would be a big step forward for us: just one less needle stick
would be worth it. Complicating the conversation a bit is the
fact that Nicholas is older and his medical care is no longer
completely my decision. While he’s not quite ready to make all
of his own medical decisions, he is old enough to have a say.
4 Dateline Federation | Special Issue Fall 2017
After our most recent HTC visit, he asked about the longer-
lasting factor: “why am I not doing that?” We talked about both
the factor products that are available and some of the consid-
erations that would go into making the decision to switch. His
answer? He’d think about it.
I admit, it can be overwhelming to think about these topics and
engage in conversations with my son. I feel fortunate to have
not only a wonderful HTC, but also HFA as a great source of
information and support.
As overwhelming as it may be with so much change in our com-
munity, it is an exciting time. Just look at the comprehensive
list of products on the market (p. 9) and the growing list of new
and emerging therapies being produced and studied (p. 17)!
What a time to be involved in this special community! To our
knowledge, this issue offers the first time such a detailed list
of products and therapies has been published and I’m thrilled
to be able to share it with you all. Our team at HFA worked
diligently with manufacturers to ensure that the information in
this issue is as timely and accurate as possible.
Also in this issue are a number of informational resources and
educational tools to help us all better understand some of the
more complex issues we encounter in the community. From a
ic of how a drug is brought to market by the FDA (p. 30), this
issue is packed with great information. I couldn’t put the issue
down until I read it from cover to cover, and hope you enjoy it
as much as I did!
Warm regards,
Tracy Cleghorn
Board Chair
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