II 109th CONGRESS 1st Session S. 1774 IN THE SENATE OF THE UNITED STATES September 27, 2005 Mr. Cornyn (for himself and Ms. Mikulski) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions A BILL To amend the Public Health Service Act to provide for the expansion, intensification, and coordination of the activities of the National Heart, Lung, and Blood Institute with respect to research on pulmonary hypertension.

1.

Short title

This Act may be cited as the Pulmonary Hypertension Research Act of 2005.

2.

Findings

Congress finds the following: (1)In order to take full advantage of the tremendous potential for finding a cure or effective treatment, the Federal investment in pulmonary hypertension must be expanded, and coordination among the national research institutes of the National Institutes of Health must be strengthened. (2)Pulmonary hypertension (PH) is a serious and often fatal condition where the blood pressure in the lungs rises to dangerously high levels. In PH patients, the walls of the arteries that take blood from the right side of the heart to the lungs thicken and constrict. As a result, the right side of the heart has to pump harder to move blood into the lungs, causing it to enlarge and ultimately fail. (3)In the United States it has been estimated that 300 new cases of PPH are diagnosed each year, or about 2 persons per million population per year; the greatest number are reported in women between the ages of 21 and 40. While at one time the disease was thought to occur among young women almost exclusively, we now know, however, that men and women in all age ranges, from very young children to elderly people, can develop PPH. It also affects people of all racial and ethnic origins, with African Americans suffering from a mortality rate twice as high as that affecting Caucasians. (4)The low prevalence of PPH makes learning more about the disease extremely difficult. Studies of PPH also have been difficult because a good animal model of the disease has not been available. (5)In about 6 to 10 percent of cases, PPH is familial. The familial PPH gene is located on chromosome 2 and was discovered in July 2000. This discovery provided new insights for determining the molecular basis of PPH and opened new avenues of study for understanding the fundamental nature of the disease. (6)In the more advanced stages of PPH, the patient is able to perform only minimal activity and has symptoms even when resting. The disease may worsen to the point where the patient is completely bedridden. (7)PPH remains a diagnosis of exclusion and is rarely picked up in a routine medical examination. Even in its later stages, the signs of the disease can be confused with other conditions affecting the heart and lungs. The use of new diagnostic standards has been positively related to the rates of diagnosis. (8)In 1981, the National Heart, Lung, and Blood Institute established the first PPH-patient registry in the world. The registry followed 194 people with PPH over a period of at least 1 year and, in some cases, for as long as 7.5 years. Much of what we know about the illness today stems from this study. (9)As research progresses, so do treatments for PH. Currently, there are 4 FDA-approved medications for PH and 3 more in trials. However, all medications are not effective on all patients. Lung transplantation is often considered a treatment of last resort for PH. (10)Because we still do not understand the cause or have a cure for PPH, basic research studies are focusing on the possible involvement of immunologic and genetic factors in the cause and progression of PPH, looking at agents that cause narrowing of the pulmonary blood vessels, and identifying factors that cause growth of smooth muscle and formation of scar tissue in the vessel walls. (11)Secondary pulmonary hypertension (SPH) means the cause is known. Common causes of SPH are the breathing disorders emphysema and bronchitis. Other less frequent causes are the inflammatory or collagen vascular diseases such as scleroderma, CREST syndrome, or systemic lupus erythematosus (SLE). Other causes include congenital heart diseases that cause shunting of extra blood through the lungs like ventricular and atrial septal defects, chronic pulmonary thromboembolism, HIV infection, and liver disease. Sickle cell anemia is also linked to SPH, with preliminary studies suggesting that approximately one third of sickle cell patients develop SPH.

3.

Expansion, intensification, and coordination of activities of National Heart, Lung, and Blood Institute with respect to research on pulmonary hypertension

Subpart 2 of part C of title IV of the Public Health Service Act (42 U.S.C. 285b et seq.) is amended by inserting after section 424B the following section:

424C.

Pulmonary hypertension

(a)

In general

(1)

Expansion of activities

The Director of the Institute shall expand, intensify, and coordinate the activities of the Institute with respect to research on pulmonary hypertension. (2)

Coordination with other Institutes

The Director of the Institute shall coordinate the activities of the Director under paragraph (1) with similar activities conducted by other national research institutes and agencies of the National Institutes of Health to the extent that such Institutes and agencies have responsibilities that are related to pulmonary hypertension. (b)

Centers of excellence

(1)

In general

In carrying out subsection (a), the Director of the Institute shall make grants to, or enter into contracts with, public or nonprofit private entities for the development and operation of centers to conduct research on pulmonary hypertension. (2)

Research, training, and information and education

(A)

In general

With respect to pulmonary hypertension, each center assisted under paragraph (1) shall— (i)conduct basic and clinical research into the cause, diagnosis, early detection, prevention, control, and treatment of such disease; (ii)conduct training programs for scientists and health professionals; (iii)conduct programs to provide information and continuing education to health professionals; and (iv)conduct programs for the dissemination of information to the public. (B)

Stipends for training of health professionals

A center under paragraph (1) may use funds provided under such paragraph to provide stipends for scientists and health professionals enrolled in the programs described in subparagraph (A)(ii). (3)

Coordination of centers; reports

The Director shall, as appropriate, provide for the coordination of information among centers under paragraph (1) and ensure regular communication between such centers, and may require the periodic preparation of reports on the activities of the centers and the submission of the reports to the Director. (4)

Organization of centers

Each center under paragraph (1) shall use the facilities of a single institution, or be formed from a consortium of cooperating institutions, meeting such requirements as may be prescribed by the Director. (5)

Number of centers; duration of support

The Director shall, subject to the extent of amounts made available in appropriations Acts, provide for the establishment of not less than 3 centers under paragraph (1). Support of such a center may be for a period not exceeding 5 years. Such period may be extended for 1 or more additional periods not exceeding 5 years if— (A)the operations of such center have been reviewed by an appropriate technical and scientific peer review group established by the Director; and (B)such group has recommended to the Director that such period should be extended. (c)

Data system; clearinghouse

(1)

Data system

The Director of the Institute shall establish a data system for the collection, storage, analysis, retrieval, and dissemination of data derived from patient populations with pulmonary hypertension, including, where possible, data involving general populations for the purpose of identifying individuals at risk of developing such condition. (2)

Clearinghouse

The Director of the Institute shall establish an information clearinghouse to facilitate and enhance, through the effective dissemination of information, knowledge and understanding of pulmonary hypertension by health professionals, patients, industry, and the public. (d)

Public input

In carrying out subsection (a), the Director of the Institute shall provide for means through which the public can obtain information on the existing and planned programs and activities of the National Institutes of Health with respect to primary hypertension and through which the Director can receive comments from the public regarding such programs and activities. (e)

Reports

The Director of the Institute shall prepare biennial reports on the activities conducted and supported under this section, and shall include such reports in the biennial reports prepared by the Director under section 407. (f)

Authorization of appropriations

For the purpose of carrying out this section, there is authorized to be appropriated $50,000,000 for each of the fiscal years 2006 through 2010.

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