Highlights from the HAEA 2007 National Patient Conference

Over 260 members of the HAE community attended the 2007 HAE Association National Patient Conference that was held in St. Louis this past weekend.

Below are the highlights of the conference:

Representatives from each of the five drug companies provided updates on the status of their US clinical programs.
- Lev Pharma (C1 inhibitor--double nano filtered) has successfully completed their "acute attack" and "attack prevention" trials. Their application for a US license has been accepted by the FDA, and the company anticipates approval and product launch in early 2008.
- CSL Behring (C1 inhibitor) announced it has completed its phase 3 trial and plans to file for FDA approval as soon as possible.
- Dyax (Ecallantide) has reported favorable data for its phase 2 and phase 3 trials. The company is completing a confirmatory phase 3 trial and expects FDA approval by the end of 2008.
- Pharming (recombinant C1 inhibitor) expects to complete its clinical trial within the next month or so and then file for FDA approval shortly thereafter.
- Jerini AG (Icatibant) has completed its clinical trials and plans to file for FDA approval in the third quarter of '08.
The Conference Keynote Speaker, Dr. Hilary Longhurst from London, England, provided participants with an overview of the treatment method she uses for the 80 HAE patients under her care. C1 inhibitor concentrate is available in England, and Dr. Longhurst is a proponent of providing patients with the training, equipment, and follow-up services necessary to provide home access to the product. A recent paper authored by Dr. Longhurst succinctly summarizes her view regarding the optimal treatment method for C1 inhibitor--the only non steroid currently approved for treating HAE, "C1-inhibitor concentrate home therapy for Hereditary angioedema: a viable, effective treatment option."
Two HAE friends from Europe--Ursula Huffer from Germany and Henrik Boysen from Denmark--chronicled their HAE treatment experiences. Danazol's 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) featuring regular injections of C1 inhibitor. Ursula and Henrik described the switch from steroids to regular use of C1 inhibitor as "...life altering."
HAEA Medical Advisor Professor Bruce Zuraw discussed ongoing efforts in designing a HAE Patient Registry. Dr. Zuraw and the HAEA are working together to design a first rate 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 because it is data that will entice researchers to continue searching for better HAE treatments.
HAE specialists--Dr. Zuraw, Dr. Longhurst, Dr. Henry Li from Wheaton, MD, and Dr. David Hurewitz from Tulsa, Oklahoma answered HAE patients' questions for 90 minutes.
A registered nurse with years of experience working with insurance companies to obtain maximum benefits for rare disease treatments conducted sessions on how to play the drug reimbursement game and win. As we go forward, there is no more important issue than insurance reimbursement. The HAE Association is working to establish an infrastructure to assist individual patients' efforts get access to medicine and receive adequate reimbursement.
An experienced counselor conducted uplifting sessions on how to cope with a chronic disease.
The HAE Association officers presented ongoing programs and plans for the HAE Registry and Patient Health Care Network (HAE NET, see below). We also conducted a 90 minute Questions and Answers session.

Over the next several months, the HAE Association staff will be sending out newsletters with updates on the status of medicines that are awaiting FDA approval. We will also keep you apprised on progress in building the HAE Patient Registry and HAE NET--our patient health care network. HAE NET will feature a "patients helping patients" model with specially trained HAE friends who will assist individual patients who are making the transition to new therapies.

There is strength in numbers, and in order to provide HAE friends with first rate services, we need to continue showing the pharmaceutical industry and government that the HAE patient community is galvanized and ready to fight for access to new therapies.

The best way to help the cause and make sure we can keep you informed about forthcoming new treatments is to ensure that you and all of your affected family members have signed up as HAE Association members. Remember its free!

So, please, if you have not already done so, fill out the membership form. Keep in mind, we go to great lengths to ensure your personal information is secure and protected. Access to information is strictly limited and tightly controlled. We have never and will never disclose any personally identifiable information to an outside party.

Tony Castaldo, President US HAEA
Dennis DeMarinis, Director of Operations
Donna K. Davis, Director of Clinical Programs and Patient Health Care Services