It’s happened: gene therapy is available, at least for hemophilia B patients in the US. The idea that hemophilia can be treated as a “cure,” or at least using one shot to cover your bleeding episodes for at least a few years, is tantalizing. And if not that, we still have recombinant products that have never touched human blood.
We just updated our factor chart here, which compares all the products available on the market for hemophilia A and B. You have lots of treatments to consider! But why are we still making plasma-derived products?
A few reasons. But first, understand where plasma-derived products come from.
Plasma-derived products come from human blood plasma. Plasma donors undergo strict screening for disease risk factors, and their plasma is tested for several viral diseases. Recombinant products are not derived from blood; they originate from genetically engineered mammalian cells containing the human gene for factor. Recombinants are produced in large bioreactors, with human and animal proteins used in the culture medium in first- and second-generation recombinant factor. Third-generation products contain no human or animal proteins in the culture medium or the final product. Fourth-generation products are made from human cell lines.
Although plasma-derived products potentially risk transmitting blood-borne viruses, all US factor products, whether plasma derived or recombinant, are considered safe by the FDA.
Why would anyone intentionally choose a plasma-derived product instead of recombinant? After all, recombinant factor is the product recommended by NHF’s MASAC. And now we have gene therapy for hemophilia B. Why inject anything other than the missing factor into your child?
For some people, it’s all about cost. Plasma-derived factor is less expensive than recombinant factor. And gene therapy is expected to cost about $3.5 million per shot. People who have high out-of-pocket expenses need safe products, but may choose less expensive ones. Sometimes, the decision depends on the type of bleeding disorder being treated. For example, intermediate purity factor VIII products contain factor VIII combined with VWF (the way it’s naturally found in the blood) and are useful in treating von Willebrand disease.
When choosing your factor therapy, always discuss options with your HTC! Use our factor product guide here, which now includes bispecific antibody products and gene therapy, and bring it with you, and ask what’s best for your loved one or you.
The NHF ushered in new leadership at the 60th Annual Meeting in Denver this past week, just as our country has voted in new leadership. With many parallels to be made, there is a feeling that our community is ready for change. Val D. Bias, person with hemophilia and already a prominent leader in our community, was hired as the new CEO of NHF. The community faces a multi-front challenge to its current status: increasing pressure from payers to lower reimbursement rates; payers forming specialty pharmacies to switch patients away from traditional home care companies and 340B programs; tightening of funds to donate; speaking with one voice and one message; forming coalitions with other rare disorder groups; rising obesity in the bleeding disorders community. Many of these challenges were addressed in depth in the various workshops
About 2,000 attendees were said to be present at this historic meeting. For most it was a great reunion of long time friends and colleagues, and for many it was a first glimpse of the community with all it has to offer.
I have been attending NHF meeting since 1992, and saw many long time friends. In speaking with representatives of industry: home care companies, HTCs, pharmaceutical companies and the NHF chapters and hemophilia nonprofits, the most frequently mentioned word could easily have been “change.” Things are changing in our community, and without vigilance, a voice, a strategy and action, we could stand to lose many of the gains we have fought so hard to win over the last decade in choice and access of services and products.
Opening Ceremony: Theatrics or Thunder?
Perhaps this is why the opening ceremony was so different than any other I have seen. The room was a Democratic or Republican National Convention theme. Vertical banners with state names were placed in sections; a large screen projected images from the podium; and red and blue balloons poised overhead in a net, waiting to be spilled. Different, definitely different, people murmured. There was a cordoned off area with red, white and blue streamers: only certain people were to sit there, apparently.
Then the lights went out and the somber and foreboding 12-minute trailer of “Bad Blood” was shown. When it was finished, lights went out again, then a spotlight shown on Val, alone on the podium, seated on a chair. Val spoke for most of an hour, sharing his vision of where NHF should lead the community. No doubt aware of the audiences’ curiosity about the trailer, he stressed, “Let’s put our past in a place of honor, where it belongs, and move forward.” With that he introduced the Campaign for Our Future, which was already introduced to chapter leaders in June.
The slogan is: Access to Care Today, Achieving a Cure Tomorrow. Val stressed four areas of focus:
Government awareness and support Education for all life stages Access to care at HTCs Research and training
A check for $2 million was presented from Novo Nordisk to support the campaign. Most if not all the manufacturers have also contributed to the campaign, which hopes to raise $10 million to implement the campaign. Then the balloons overhead were released and everyone cheered and applauded. It seemed to be a new start for the NHF, and most people left feeling upbeat.
Later on, as people filtered out of the conference hall, there seemed to be second thoughts and questions by people. Many asked each other, “What did you think?” The trailer for “Bad Blood” was grim, dark, and pointed fingers some said. What was the point of the film? Have we not had 23 years of success, with no documented viral transmission? “What does the movie have to do with the new campaign?” one attendee asked. “If it’s for the future, why are we bringing up this past again?” Some people said they cried just watching the trailer as it evoked strong emotions of children contracting AIDS in the 70s. Some had nodded their heads approvingly as the film was being shown. Others feared the film was too strong, too biased, that it set the wrong tone for the NHF meeting: the movie would stir up negative feelings and fear in the absence of adequate general information about blood safety to new parents at a time when plasma products are just making a comeback. Everyone suddenly seemed to have a strong opinion. It was indeed a different kind of NHF opening: thunderous to make people take action, or theatrical to gain attention?
There was a lot to think about, and a lot to do. I was privileged to attend the Medical and Scientific Affairs Council (MASAC) to present my factor donation program, Project SHARE. As we donate millions of units of factor each year, we thought it a good idea to let MASAC know how we operate and answer any questions they might have.
The two and a half days NHF conference were filled with workshops, lectures, and symposia on all types of subjects related to hemophilia and von Willebrand Disease. Social events included lunches, raffles, and the final event, sponsored by Baxter: a wonderful and lively band, dancers, plenty of food, picture taking and dancing. Everyone got in on the dancing, which went till 11 pm. NHF planners and staff are to be commended for their ability to accommodate so many people with so many needs in so many sessions so perfectly and graciously.
If you attended NHF’s Annual Meeting this past week, please feel free to leave a comment! I’ll write next week about some of the meetings I attended while at the meeting.
It’s been a long time since I’ve seen a withdrawal or recall of factor. About six years ago we had a lot of them, and not everyone got word that their particular product was recalled. Then along came the Patient Notification System (PNS), a program that allows you to register to get alerted via email, telephone, fax or snail mail to any recalls or withdrawals. It’s a good time now to ask yourself if you have registered with PNS.
Here’s why: Just this week we had a voluntary recall of factor.
NHF’s Medical Advisory #407 of August 20 announced that CSL Behring initiated a voluntary recall of four lots of Monoclate-P
that were distributed beginning on August 4, 2008. It reads: “This action is being taken with the knowledge of the United States Food and Drug Administration (FDA). These lots are being voluntarily recalled as a precautionary measure because they do not meet the potency specification when stored for three months at 5 degrees C. No specific safety issues have been identified and neither have adverse incidents been reported. CSL Behring is requesting that the use of these lots be immediately discontinued and the product be returned to the company.”
When you register with PNS, you will be notified only about the product you use, so if you change brands, you may need to update your registration. If you are not registered, you may not hear at all about recalled brands. Please sign up with PNS today. Go to http://www.patientnotificationsystem.org to register. Don’t be the last to know!
Needless to say you should also register with NHF’s eNews, which will keep you updated with recalls and all news related to hemophilia.
Book I Just Read Fifty Things to Do When You Turn Fifty by Ronnie Sellars (ed). This is a compilation of 50 essays by 50 authors, doctors, celebrities about turning 50 and what you need to know. It was pretty good, a general summary on changes you will face in areas like fitness, attitude, career and friendship. Thing is, most of us at age 50 are already aware of these things! So I guess it’s a good book to read when you are 45. Best part is that all profits are donated. Two stars.
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