Legislative Advocacy: July/August 2007 Update

July/August 2007: Status as of this writing

The House and Senate Labor, Health and Human Services and Education Appropriations subcommittees completed a critical early step in the appropriation process in June with the “marking up” of their spending bills.

The full Senate Appropriations Committee and the full House Appropriations Committee met their plan to approve their respective bill in mid July prior to leaving for the summer congressional recess that started August 6th.

In working with Senator Sherrod Brown’s office and Representative Rosa DeLauro’s office we found out that the HHT language made it into both “marked up” bills however the funding request was not added. As reported by Representative DeLauro’s office, only 50% of her priorities were funded this year because of the tight budget. The specific reported language is listed below.

House FY 2008 LHHS Appropriations Bill

Hereditary Hemorrhagic Telangiectasia (HHT).-The Committee encourages CDC to establish a Hereditary Hemorrhagic Telangiectasia resource center to increase identification of people affected with HHT, and increase knowledge, education and outreach of this largely preventable life-threatening condition. The Committee encourages CDC to provide information on effective evidence-based interventions and treatments to prevent premature death in the HHT population, improve outcomes and the quality of life for people living with HHT by creating a database to collect and analyze data, support epidemiology studies, provide surveillance, and train health care professionals.

Hereditary Hemorrhagic Telangiectasia (HHT).-HHT is a multisystem vascular genetic disorder that affects 75,000 Americans, producing arterioveneous malformations in the brain and lung. The Committee recognizes that while HHT is largely preventable with proper intervention, twenty percent of children and adults with HHT die or become disabled due to lack of recognition by the medical community. The Committee encourages NINDS to support research that would improve the quality of life for people living with HHT.

Senate FY 2008 LHHS Appropriations Bill

Hereditary Hemorrhagic Telangiectasia.—The Committee is aware of interest in the establishment of a Hereditary Hemorrhagic Telangiectasia [HHT] National Resource Center. The Committee encourages the CDC to examine carefully proposals to establish such a center.


Positive Outcomes from HHT Legislative Advocacy

Understandably, we are disappointed that federal funding was not added into the bill at this point. Many people have worked very hard including Members of the Foundation, HHT Treatment Center of Excellence Directors, Board of Directors, and the Foundation’s staff. On a positive note, there are a couple things I would ask you to keep in mind as we continue to proceed with the HHT Legislative Initiative.


  • HHT’s pursuit of federal funding is a necessary activity but gaining the funding is a long and difficult process


  • HHT has benefited in several ways as a direct result of our HHT Legislative Initiative. As a result of our HHT Language being added to the House and Senate bill, HHT has benefited in the following ways:


    • The United States Government now recognizes HHT as a national health problem.


o The Center for Disease Control and Prevention (CDC) now has HHT on their radar. In our initial meeting with the CDC, HHT and the CDC agreed to participate in a two day conference to be held in November of 2007. This conference is entitled “HHT Health Initiatives for the 21st Century”. The goal is to develop a health initiative of surveillance, detection and treatment of this deadly disease. The HHT Foundation has the disease expertise, which married with the CDC’s experience, will certainly lead to improved care for patients with this disease. The CDC has awarded the HHT Foundation a grant of $32,000 to be utilized for the conference.


The specific objectives of this conference:


  • to develop a roadmap for HHT treatment and research
  • to implement Clinical Guidelines and effectively disseminate information to primary care providers to aid in identifying undiagnosed individuals
  • Data Base Development to establish methods for data collection related to screening, treatment, and outcomes of medical interventions

o The National Institute of Health (NIH) has HHT on their radar as well. The HHT Foundation’s researchers and clinicians write proposals to gain funding for their specific research projects. Since the HHT Language has been in the bill, these researchers have been awarded more funding for research. To follow is just a partial listing of Clinical Trails that have been funded:


  • Thalidomide Reduces Arteriovenous Malformation Related Gastrointestinal Bleeding. The purpose of this study is to determine whether Thalidomide is effective in the treatment of arteriovenous malformations in the gastrointestinal tract. (Study started October 2006 – Expected completion date - July 2008)
  • ATERO: A Randomised Study With Tranexamic Acid in Epistaxis of Rendu Osler Syndrome. The purpose is to demonstrate that oral intake of tranexamic acid significantly reduces the risk of epistaxis occurrence, estimated by the average monthly duration of episodes of epistaxis. (April 2007)
  • Molecular Studies on Hereditary Hemorrhagic Telangiectasia Families. Purpose: HHT is a disease inherited as an autosomal dominant disease. Analyzing DNA from affected and unaffected family members allows us to identify the location of disease genes by linkage analysis. Sequencing genes in the interval identified by linkage analysis allows us to identify which precise gene is mutated. Further functional studies can then determine why the mutations in that gene cause the disease.
  • Investigation of Plasma Proteins in Patients With Hereditary Hemorrhagic Telangiectasia and PAVMs. Purpose: HHT is a disease that leads to the development of dilated and fragile blood vessels, including arteriovenous malformations in the lungs (PAVMs). We hypothesize that the genetically-determined abnormality in the blood vessels of HHT patients leads to alteration in the concentrations or activity of several proteins in the blood stream. We propose to take blood samples from patients at defined times in order to study changes in blood protein levels and activity. (Study started – March 2005)

    There is still an opportunity to have funding added and the HHT Foundation is working side by side with a few of our HHT Treatment Center of Excellence Directors to influence this process with key individuals in the political arena. I want to be candid however; it will be difficult to have funding added at this point for FY2008. Regardless, we still have to try and will keep all informed if action is needed.


    The leadership will soon convene in the conference process. This is the process of reaching a compromise in the differences between the House and Senate bills. It is important to ensure our HHT Language stays in the final bill. Strong and persistent outreach by advocates now and through this conferencing process will be critical to assure HHT language stays in the bill for FY2008.

    Congressional Appropriations for Uncommon Disorders

    One of the common objections that a Member of Congress or their staffer will state is that the budget for FY2008 is very tight and that is why they may not be able to help us with the HHT federal funding this year. The budget has been tight for several years and will continue to be so in the future. Similar disorders have received millions of dollars in federal funding over the years and continue to do so. Most of these disorders are less prevalent than HHT. These disorders are worthy of federal funding and so too is HHT. This disorder has been ignored too long. It is our time.

    Key Congressional Committee

    Our HHT Legislative Initiative is seeking an appropriation; therefore, we are still focusing our efforts on the Appropriations Committee. Since HHT is a health issue, HHT falls under the domain of the Appropriation Subcommittee for Labor, Health and Human Services, and Education (Labor HHS). This is where we will focus a great deal of our efforts. Utilizing http://www.congressmerge.com website, you can find a list of the members of the House and Senate Appropriation Committees. Please see if your Senators or Representative are on this list. You can click on their names to view a wealth of valuable information about each member.

    These Representatives have the power to set aside funding for the HHT initiative. While these individuals are key to our cause, it is important to contact all your Senators and Representatives, regardless of whether they are on the committee or not, to gain their support. If they are not on one of these committees they can help us by letting their colleagues on the committee know that they support the HHT Legislative Initiative.

    The objective is to get appointments or make contact with your Member of Congress (through phone, email or fax) in an effort to obtain recognition and funding for HHT. Please see the Advocacy 101 section that follows for guidelines to help you accomplish this task.