HAE Association Update January 22nd 2006

January 22, 2006

Dear HAE Friends,

    I am writing today to provide a brief overview of the HAE Association’s 2006 plans, and to discuss the critical need for all HAE friends to participate in clinical trials.

Dallas Regional Meeting

    We are planning a series of regional patient meetings in various parts of the country during 2006, and the first one will be held in Dallas on March 18, 2006. The meeting will feature plenty of time for patients to interact and discuss their lives with HAE. There will be an informative presentation from one of the HAE Association’s medical advisors, and almost all of the drug companies sponsoring clinical trials will attend to discuss their medicine and clinical development program. For further information on the meeting, please visit our web page HAE Home Page or contact our Board member/events coordinator Dennis DeMarinis at the following link Dennis DeMarinis. The Association has a limited amount of financial aid available for patients who would like to attend, but could otherwise not afford to do so. Our goal is to have at least 70 attendees, so please mark you calendars and plan to join us in Dallas.

Clinical Trial Participation

    As many HAE friends already know, there are 5 HAE clinical trials going on simultaneously in the United States. We have a unique opportunity to seize upon this good fortune and maximize our chance to have multiple HAE therapies available in the United States.

    While in many ways a blessing, this situation also poses significant challenges for such a small patient community. It is important that we provide each of the five companies with a sufficient number of patients to complete their trials. This would acknowledge the significant investment each company has made in studying our disease, and should also lead to getting several medicines licensed. That is why the HAE Association, under the leadership of Clinical Programs Director, Donna Davis, is vigorously supporting each company’s clinical development effort by identifying and referring HAE friends to the trial center that is geographically closest to their residence.

    HAE friends, we desperately need your help and participation to ensure these trials get done. Our worst fear is that one or more of these companies will discontinue their US trial because not enough patients have signed up. We have such a unique opportunity, and now is the time to seize upon it. To be sure, participating in a trial involves some sacrifice, but it is well worth it because getting multiple therapies approved will ensure our children and their children will not have to suffer, and will have more choices than just anabolic steroids.

    If you have not already done so, please go to our web page HAE Home Page and sign up on our clinical trial database, or email the HAE Association’s Clinical Programs Director at the following link Donna Davis.

    If you are already signed up for a trial, please go into the trial center whenever you have an attack that fits the eligibility criteria. If you are unsure of what is considered “eligible,” please call the coordinator at the trial site for clarification. Several pharmaceutical companies have voiced concerns that enrolled patients may not be contacting trial centers when they are experiencing an attack. We recognize it's very difficult to get up and travel when you are so sick, and we wish there were another way to get drugs approved. Unfortunately, we are stuck with the current system, and getting to the center to test the medicines when we are horribly sick is the only way we are going to get FDA approval.

Other 2006 Initiatives

    2006 will be another busy year for the HAE Association! Board member and web master Scott McCoy, along with HAE friend Joyce Wilmot, will continue to keep the web page informative, up to date, and aesthetically pleasing. We will continue to devote significant time and effort in assisting companies conducting US clinical trials.

    A sister rare disease organization that has been in existence for over a decade is working with us to implement some unique technology they developed to enhance the Association’s efforts to communicate with HAE friends. The primary goal of this affiliation and the technical assistance being provided is to establish an electronic infrastructure for expeditious and systematic data collection that, ultimately, will help researchers and pharmaceutical companies better understand HAE. As part of this project, we are putting together a HAE patient brochure, and, in the very near future will have the means to readily help researchers and pharmaceutical companies collect data on HAE in a timely and systematic manner.

    Finally, each of the therapies being tested are going to be very expensive because, among other reasons, they have obtained “Orphan Drug” status.. The HAE Association wants to ensure that insurance carriers reimburse patients who use any newly approved medicine. As a result, we are working with consultants to design an HAE Association sponsored “pharmaco-economics” study that will support insurance reimbursement for all products. At some point, the HAE Board will ask our 1300 HAE family “network” to provide data that can be used to complete this study. In addition, we are already exploring ways to ensure that patients without insurance or other means to pay for therapy can get access to newly approved medicines.

Warmest regards to all HAE friends,
Tony Castaldo
President, US HAE Association