HAE Association’s 2008 National Patient Conference and International Leadership Forum Notes

31 Oct

Some of you may remember (and some of you won’t care) that I decided at the last minute to attend the Hereditary Angio Edema (HAE) Association’s 2008 National Patient Conference and International Leadership Forum in Chicago a few weeks ago. You may also remember that I sort of promised to recap whatever I learned, especially regarding the FDA’s approval of the C1 Inhibitor treatment, Cinryze.

What some of you know is that I collected some nasty bugs while I was in Chicago and have been deadly ill since I returned, so I’m only now making good on that promise. Using my own notes and cribbing off the minutes distributed by Janet Long, the new VP of the HAEA, here’s the skinny:

My flight was uneventful, but getting from the Midway Airport to the O’Hare Airport and then on to the Marriott was rather interesting.

The Midway shuttle was running late and about 30 people were huddled in the underground busing terminal where the shuttles and buses for a conglomerate of different purposes were coming and going. Despite being told the shuttle they needed was on the next concrete pad over, 26 people continued to huddle on the Midway platform until, like a startled flock of birds, they suddenly migrated to the right pad when that’s where their shuttle stopped, abandoning four women on the Midway shuttle, which we thought was just hilarious. When our shuttle finally arrived, the driver made up for lost time by getting in the left-hand turning lane at every stoplight and then barreling straight ahead and cutting off all the driver’s to his right. As I said, rather interesting.

When I arrived at O’Hare and trekked over to the Marriott shuttle stop, I didn’t have long to wait. However, the shuttle went to more than one Marriott (something the website forgot to mention). Everything I’d read called MY Marriott the Airport Marriott, but the driver didn’t call any of the Marriott’s that, so I had to ask for clarification and found that the O’Hare Marriott and the Airport Marriott were one and the same. The lady in the seat across from me had a more difficult problem: Her paperwork simply said “Marriott.” Then she said she was going to a conference and I asked her which one. When she said the HAE conference, her problem was solved! We were soon chatting away like we were old friends.

The cocktail party that evening was quite interesting also. Waiters came to each table bearing hot hors d’oeuvres and there was an open bar, as well as several cold buffets. I was very good and only had a single glass of merlot with a few chunks of cheese (I was feeling very hungry). The interesting highlight of the night is when two women who were researching the effects of HAE on the lives of patients came to the table where I had met up with my friend, Sand. I asked them to join us and proceeded to give them an earful, as did the rest of the patients at the table. They left us feeling very illuminated and quite amazed at the horrors we face and how bravely we face them.

Below are the highlights of the conference itself:

  • Almost 300 people attended the 2008 conference on October 17-19, 2008.
  • HAEA President Anthony Castaldo kicked off the meeting with a discussion of “Exciting Times for the HAE Patient Community.” His key points were:
    • The approval of C1 inhibitor in the United States is the first step in preventing future HAE-related deaths (there was a solemn moment of silence during the conference opening in honor of three HAE friends that died of laryngeal swelling in 2008) and significantly improving the lives of those in our community who are disabled by HAE.
    • HAE friends interested in getting access to C1 inhibitor or finding out more about the medicine should contact the HAEA’s Patient Services group.
    • The HAEA advocates that every HAE patient has a fundamental right to choose a treatment program that best fits their individual needs. The approval of C1 inhibitor opens up a new world for treating HAE as evidenced by the presentations from European HAE friends (see below), and the HAEA will help each and every HAE friend by providing the information necessary to support whatever therapy option is chosen.
    • The HAEA will work closely with ViroPhama (the company that received the approval for C1 inhibitor) to support and represent each HAE friend in the insurance reimbursement process. ViroPharma also has a financial assistance program available for patients who might not be able to afford the medicine. The HAEA Patient Services staff will be able to provide information on that program as soon as more is known.
    • The HAEA’s Burden of Illness study conclusions were presented. Almost 500 patients (including myself) participated in this landmark study designed to provide insurance companies with scientific data that proves new medicines are needed to adequately treat HAE. A press release that provides an overview of the study will appear on the HAEA web page shortly, and the HAEA will ensure that the data gets into the hands of those who are responsible for insurance reimbursement. The bottom line conclusion of the study is that HAE poses significant financial, social, and emotional burdens that the current medicines do not address.
    • The HAEA is going to be conducting the largest scale study of the effect of androgens on HAE patients ever attempted. The study will be accessible over the internet and totally anonymous. The goal is to collect scientifically valid data that will once and for all provide accurate information regarding the safety, effectiveness, and side effects of HAE medicines. Tony emphasized that patient participation in this important effort will help to improve the lives of all HAE patients, as well as provide a brighter future for our children. The androgen study will be rolled out soon – watch for more information on how you can participate.
  • The Conference Keynote Presentation, “Decades of safe and effective use of C1 inhibitor for treating and preventing HAE attacks in European patients; and the effectiveness and safety of home infusion,” was delivered by Dr. Marcel Levi, Professor of Medicine at the University of Amsterdam
    • Dr. Levi discussed his long-term observations (and published studies) of HAE patients in the Netherlands who have been using C1 inhibitor to control their HAE attacks. His research has unequivocally shown that
      • C1 inhibitor has a decades-long track record as a remarkably safe and effective therapy; and
      • HAE patients who have home access to C1 Inhibitor generally have no problems self-infusing the medicine or relying on a family member to give the injections.
      • Dr. Levi and his staff require that patients complete a training course before they are allowed to begin home infusion.
  • Representatives from each of the five drug companies provided updates on the status of their US clinical programs
    • ViroPharma (formerly known as Lev Pharma)
      • ViroPharma’s C1 inhibitor (the brand name is Cinryze) was approved by the FDA as an attack prevention therapy. The company anticipates that product kick-off will be in early to mid-December according to Janet, but my discussions with the case managers leads me to believe that it will not happen until early next year. ViroPharma is putting together an infrastructure to make Cinryze available to every HAE friend that could benefit from the medicine.
      • The program is called Cinryze Solutions, and the HAEA is ready to (1) provide you with a direct referral into the Cinryze Solutions program, and (2) act as your advocate and confidant, and help you work with your doctor, the specialty pharmacy (there are only two speciality pharmacies authorized by ViroPharma in the US—one is CareMark; Cinryze will be manufactured in Amsterdam) who will ship your medicine, drug company representatives, and insurance providers. Please contact the HAEA’s Patient Services group for more information.
      • Here’s what else I found out: ViroPharma is planning on coding Cinryze not as a pharmaceutical drug (despite the fact that it is being dispensed by a specialty pharmacy), but as a medical procedure! This critically impacts how your insurance will determine your co-pay. In my case, this means that I will not be able to get a 90 day supply of Cinryze for $60; instead I will have to pay 25% of the actual costs because (1) it is coded as a medical procedure and (2) the treatment is being provided out of network (i.e., by CareMark and my speciality pharmacy is MedCo). This automatically means that I will not be able to afford to pay for my treatments, even though my physician is willing to let me do my own infusions at home (i.e., I am the one who is going to be performing the medical procedure!). I quickly discussed this with Tony in the Saturday night buffet line (he has the same insurance that I have) and his response was “That doesn’t make any sense. So we’ll fight it!” I’m thinking Blue Cross/Blue Shield might have something to say about it, too! But the ViroPharma case manager who told me (and who claims to sit right next to my own case manager) looked at me like I had grown six heads when I questioned the validity of doing it this way—and I was so mad, I ended up in the Ladies, crying—and that’s very unusual for me.
      • Nobody would even give me a hint as to how much each vial was going to cost—but in Europe, one vial of Cetor is going for about $800, so…no matter how I look at it, I’m gong to have to get some help from the non-profit group that helps people pay for their medication—something I would really rather avoid, but which it looks like I’m about to be forced into.
    • CSL Behring
      • Their Berinert brand of C1 inhibitor has completed its Phase 3 trial and they expect Berinert to be approved for treating acute HAE attacks (attacks that have begun) sometime in December 2008 (Berinert only has a half-life of 30 hours and so cannot be used to prevent attacks). CSL Behring officials noted that the company is a global leader in the plasma protein biotherapeutics industry, and are experts in producing products to treat rare diseases, and that Berinert has been safely and effectively used throughout the entire world for decades. I asked them why it took them so long to bring Berinert to the US since it has been in use in Europe for 30 years; the cost of passing FDA drug trials was most often cited.  Why doesn’t the FDA accept data from European drug trials and 30 years of accumulated safety data?
    • Dyax
      • Ecallantide (formerly DX-88) completed its second Phase 3 clinical trial for its subcutaneous HAE attack treatment and reported that the data generated during the trial indicates the medicine is an effective and safe therapy. Ecallantide is a recombinant product that inhibits kallikrein–an enzyme that plays a key role in the biochemical processes that lead to HAE-related swelling. The company filed for FDA licensure in September 2008, and the product could be approved in early 2009.  The half-life of this treatment is also too short to be used preventatively.
    • Pharming NV
      • They announced successful clinical trial results for their product, Rhucin, a recombinant human C1 inhibitor protein that is derived from the milk of genetically altered rabbits (interesting aside: I had lunch Saturday with a doctor who was interested in this treatment and he had actually seen them milk a bunny—people are really paid to milk rabbits!). Clinical trials have shown that Rhucin is a safe and effective treatment for acute HAE attacks (again, half-life is too short to be used preventatively). The company plans to apply for a US license in the near future.
    • Jerini AG
      • The US clinical trial for Firazyr did not meet the statistical significance requirements for effectiveness required by the FDA (because it did not include a double-blind placebo). Firazyr is delivered through a subcutaneous injection in the stomach (he said it was a short needle, but still–yuk!) and works by blocking the peptide that causes HAE-related swelling. The product has been licensed in Europe and Jerini is now working on plans to conduct another clinical trial in the US.  Short half-life again.
  • HAE friends from Europe Discussed How Access to C1 Inhibitor Changed Their Lives
    • Ursula Huffer from Germany and Henrik Boysen from Denmark–chronicled their HAE treatment experiences. Danazol’s adverse side effects and general lack of effectiveness led both Ursula and Henrik to stop steroid therapy and take advantage of a treatment regimen (offered by compassionate physicians in Germany and Denmark, but not necessarily elsewhere in Europe) that features regular injections of C1 inhibitor. Ursula and Henrik described how switching from steroids to using C1 inhibitor to prevent attacks was life changing. They are now both leading normal lives and are especially happy that they can travel without having to worry about life threatening throat swelling or the awful side effects they experienced while on androgens. They both described how they have stopped on the autoban on the way to the airport to give themselves infusions, stopping HAE attacks that would have otherwise derailed their travels plans—way kool!
  • HAE Association Study Results: Quality Of Life Aspects Of C1 Inhibitor Home Access Compared Receiving Therapy At A Medical Facility
    • HAEA Medical Advisor Professor Bruce Zuraw summarized the results of a HAE Association sponsored study (I participated) that compared the safety, effectiveness and quality of life of patients who had home access to C1 inhibitor concentrate to those that traveled (moi) to a medical facility for their treatments. Professor Zuraw provided the study’s top line results: “Physician Supervised self-managed C1 Inhibitor infusions are a safe and cost effective alternative to treatment in a medical facility and also enhance the patients’ quality of life.”
  • HAE Association’s Scientific Registry
    • Dr. Zuraw later presented information on the HAE Association’s Scientific Registry. Dr. Zuraw and the HAEA, working together, have designed a state-of-the-art registry that will produce high quality “scientific grade” data. Dr. Zuraw stressed the importance of obtaining maximum patient participation once the registry is up and running–it is the collected data that will entice researchers to continue searching for better HAE treatments and, ultimately, a cure!
  • Physician Questions and Answer Period
    • HAE specialists HAEA Medical Advisor Professor Bruce Zuraw, Dr. Henry Li from Wheaton, MD, and Dr. Marc Riedl from UCLA,and Dr. Andrew Grant from Galveston, TX fielded a wide variety of questions from the patient community. There is not enough space to post the detailed questions and answers in this summary. However, the HAEA is happy to field any and all questions from each and every member of our community, and get definitive answers from world class HAE scientists. HAE friends can contact their patient services group or submit a question through the HAEA website at this link: http://www.haea.org/contactus.php.  I will add this:  It was continually stressed that FDA approval only limits what a company can print/claim in its literature regarding a licensed product; what a doctor determines to be in his patient’s best interests and how a doctor decides to use a product “off-label” is not governed by the FDA.  In other words, although Cinryze is currently only approved by the FDA for prophylactic use (i.e., preventative use), this does not preclude doctors from using it for acute attacks.  Note:  ViroPharma is currently seeking FDA approval for acute attacks so its use for acute attacks can be documented in the product’s literature.  Additionally, the FDA has only approved Cinryze to be administered in a clinical environment; this does not preclude doctors from allowing their patients to self-administer if the doctor determines it is in the patient’s best interest to do so.

I was unable to attend any of the activities scheduled for Sunday morning because they moved everything to be an hour later and I had to catch my flight, so these are strictly Janet’s notes:

  • The HAE Association 2008-2009 Program
    • Officers presented ongoing programs and plans that will benefit all HAE patients: The programs discussed included:
      • Expanding the HAEA’s Patient Services–We are growing to ensure we are well positioned to serve as the primary problem solver for HAE friends who are interested in exploring new therapies, or have any HAE-related issues or needs.
      • Implementing a robust research infrastructure that includes offering medical research grants aimed at bettering the lives of HAE patients.
      • Rolling out and recruiting patients for our HAE Scientific Registry.
      • Conducting regional meetings, workshops, and lectures for patients and physicians interested in learning more about HAE.
      • Establishing a 24/7 toll free number to help patients deal with HAE issues that arise.
      • Continuously upgrading our web page to ensure it remains the most authoritative source on the web for HAE-related information.

There you have it.  I met some very incredible people from all over the world and learned even more.  I got to hug people I’ve known for years over the phone and the web, but never seen.  I laughed and I cried and I ate Chicago deep dish pizza.  I hope I can go to the next conference, too.  It was even worth having my throat swell up when I got back, just thank the Goddess I’m still on the Cinryze trial “medical procedure” and had Dr. Hornberger to get me through THAT crisis!


8 Responses to “HAE Association’s 2008 National Patient Conference and International Leadership Forum Notes”

  1. Red October 31, 2008 at 8:37 PM #

    I am so jealous that you went! I wanted to go so bad! And you got to see Sand! How cool! Thanks for all the notes, I’ll have to come back and re-read them again. **hugs** Lots of wishes and blessings for no swell your way!

  2. Faerie♥Kat October 31, 2008 at 11:09 PM #

    Hi Red!

    The one thing I feel so bad about is that I think I may have talked Jerry into not going because I wasn’t–and then I went at the last minute (with HAEA financial assistance). But I never would have done it if I hadn’t found a fantastically low airfare into Midway; all the airfares in O’Hare were astronomical and required 3-4 hour layovers at either JFK, Charleston or someplace equally idiotic!

    I also met Sand’s two girls–Taylor (the newly teen)was so adorable! She hooked up (but not in the sense the kids use today!) with two other kids her age (one girl and one boy) and the three were inseparable (and very giggly–just like you’d remember being at that age–so cute!). Jackie (the older daughter) was very quiet.

    Getting to finally meet Donna (from Hawaii) and Tony after talking to them via phone and email for so many years was another highlight for me. Donna showed me pictures of her son (only person in her family with HAE); he’s now playing football, thanks to Cinryze! Donna’s mom was there, too; they were both so terrific. And Tony…even more friendly in person, if that can be imagined. So huggable! All the HAEA workers (Janet, Michele, etc.) were so friendly and caring. Sand had a attack and Donna arranged an infusion for her! How perfect was that?

    I really didn’t know what to expect, so wasn’t really expecting much, but the whole conference was really professional and I was very impressed. I’m totally onboard to attend everything I can from now on!

    Hugz to you my swell friend!


  3. gt281 November 1, 2008 at 3:57 PM #

    I read the whole thing…Interesting how the cogs of science move
    thru space….very slowly….Good question about the U.S. FDA and
    the Euro FDA….Why don’t they accept each others studies?….hmmm…
    I’ll bet it has something to do with the French…
    ….And who’s this Tony person!!!!!!!!

    • Faerie♥Kat November 1, 2008 at 6:33 PM #

      I’m positive you’re right about the French; after all, they were the ones
      that came up with the idea to eat frog legs and snails, which must have set
      civilization back at last one thousand years.

      Although Tony may be the President of the United States and International
      HAE Associations, and therefore the savior of my pitiful life, you, my dear
      Bard of the Cornstalks, have both made me laugh insanely and saved my
      sanity more than once, which IMO is much harder to do, and living would be
      pointless without either insane laughter or occasional sanity. So never
      shall Tony rise higher in my heart or esteem than thou, and when Puff flies
      thee hither some day, I shall hug thee four times more and six times harder
      than ever I did poor Tony. You have been fairly (and faerie) warned!


      Your Faerie Queene

      > [Original Message]

  4. Cheryl November 2, 2008 at 2:39 PM #

    Hi Kat….this was very interesting. Thanks for sharing your experiences. xoxo

    • Faerie♥Kat November 2, 2008 at 2:47 PM #

      Thanks for popping in, Cheryl!

      Faerie hugz, Kat

      > [Original Message]

  5. Seneca November 3, 2008 at 10:09 AM #

    I heard in a recent news article that pharmacists are being poorly paid and they have a heavy workload and that this is leading to a high error rate… I wonder what effect this will have on how many more pharmacy students we will have graduate in the near future…

    • Faerie♥Kat November 3, 2008 at 2:59 PM #

      Interesting (and anonymous) comment from (purportedly) a pharmacy company.
      Since I can’t read the supposed article without a reference, I’m left to
      wonder where the supposed article got its statistics. Could it have been
      from the pharmacy industry? This comment looks very spammy to me, but I’m
      going to let it ride as a perfect example of Internet terrorist and scare
      tactics being used against our own citizens right here in our own country.
      Remember, dear readers, not everything we read on the Internet has been
      blessed by the Goddess with the kiss of truth.

      > [Original Message]

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