Rare Diseases & MPS Comments by Con. Ron Kind

Mr. DAVIS of Illinois. Mr. Speaker, I want to, first of all, thank the gentleman from California (Mr. Waxman) for yielding time to me, and commend him and the gentleman from Illinois (Mr. Shimkus) for introducing this legislation.

I also want to thank the former commissioner of the Chicago Metropolitan Water Reclamation District, the distinguished JoAnn Alter, for bringing this matter to my attention. Therefore, I rise in support of H.R. 4013, the Rare Diseases Act of 2002.

Mr. Speaker, with a large low-income population, 24 hospitals, 5 medical schools, and several research institutions in my district, I know firsthand the heartbreak faced by people who struggle to find the appropriate medical treatment for themselves and their families.

We have made tremendous strides in education, research, and medical protocols for individuals with diseases that affect large populations. Much funding has been dedicated and continues to be directed to treatment of diseases such as cancer, heart disease, and diabetes, and this is all good.

There are, however, a number of rare diseases which affect 200,000 or fewer Americans which continue to go underresourced. While statistically 200,000 people may be a small number, it is a large number when we consider it represents people needing medical treatment. However, if we aggregate the number of people suffering from at least 1 of the 6,000 known rare diseases and disorders, we are talking about 25 million Americans, 1 in 9, suffering from a rare disease.

Several months ago a mother and her young son, who suffers from Crohn’s disease, traveled hundreds of miles from Virginia to Northwestern Memorial Hospital in my district to see if he could be accepted into a special treatment program that was offered nowhere near his home. He wrote a letter to me thanking me for the fact that he was indeed able to get into Northwestern and to be considered for treatment for his very rare disease.

This bill, H.R. 4013, which establishing an Office of Rare Diseases at the National Institutes of Health, by increasing the national investment in the development of diagnostics and treatment for patients with rare diseases and disorders, and by allowing for rare disease regional centers of excellence, is a quantum leap in the right direction. I again commend my colleagues for its introduction and urge swift passage of this resolution.

Mr. WAXMAN. Mr. Speaker, I have no further requests for time, and I yield back the balance of my time. Mr. KIRK. Mr. Speaker, I rise today in strong support of H.R. 4013, the Rare Disease Act of 2002. Currently, more than 6,000 rare diseases affect 25 million Americans each year. While some progress has been made to fight rare diseases over the last twenty years, we must commit greater resources to this effort, particularly through the National Institutes of Health.

My own constituents have been very active in this fight. Families of Spinal Muscular Atrophy, based in Libertyville, Illinois, have been working to enhance research efforts of this and other rare diseases since 1984. I commend Audrey Lewis and all of the Families of SMA’s staff and volunteers for their continued dedication and hard work in this field.

SMA is just one of the thousands of rare diseases that continues to impact American families, and is the number one genetic killer of children under the age of two. A child of parents who both carry the SMA gene has a one in four chance of developing this devastating disease. Inflicted children are forced to live with neuromuscular deterioration that can affect crawling, walking, head control and swallowing.

We must make every effort to expand research efforts so that those with SMA and other rare diseases have a change to live long and healthy lives. I hope that Congress can send a united message of the importance of enhanced research efforts in this field by supporting this bill. An Office of Rare Diseases at the National Institutes of Health is an essential element in our efforts to raise awareness and research for SMA and the thousands of other rare diseases affecting Americans. I urge my colleagues to support H.R. 4013.

Mr. PITTS. Mr. Speaker, the legislation before us today represents the latest steps in Congress’ twenty year commitment to Americans with rare “orphan” diseases. Since its passage in 1983, the Orphan Drug Act has stimulated the development of 231 new marketed orphan products, with several hundred more in the pipeline for which we all have great hopes. Yet, there are still more than 5,000 rare diseases with no specialized treatment at all. For this reason, H.R. 4013 and H.R. 4014 represent major advances in stimulating new therapies for those with rare diseases, as well as improved care.

It is important to note, however, that even those 231 marketed orphan drugs, biologics, foods and devices are not always readily available to patients because of geographical and insurance barriers. One of the unfinished pieces of business facing this Congress is to make sure that Medicare is not contributing to this problem.

Three years ago, when Congress created the Medicare Hospital Outpatient Prospective Payment System, known as HOPPS, Congress placed all orphan products into a pass-through category where they would be paid at a higher rate. Even still, many hospitals have lost money when they stocked orphan drugs to treat patients with rare diseases.

Now we are faced with a situation where CMS has proposed a regulation for the 2003 HOPPS program that leaves most orphan drugs, biologicals and blood plasma therapies and their recombinant analogs such as clotting factors for individuals with Hemophilia without adequate reimbursement. Many hospitals will refuse to stock these drugs because of the large loss they will incur for treating a small number of patients. Without appropriate reimbursement, patients may be turned away from hospital emergency rooms or directed to alternative facilities if the location does not stock their product. The consequences of inadequate or non-existent access would be devastating, particularly in rural areas where the nearest hospital to stock a particular orphan drug may be a hundred or more miles away.

Mr. Speaker, it is critical that the House of Representatives act this year to ensure that orphan drugs are properly reimbursed.

Mr. KIND. Mr. Speaker, I rise in support of both the Rate Diseases Act (H.R. 4013) and Rare Diseases Orphan Product Development Act (H.R. 4014). A rare disease is defined as one that afficts fewer than 200,000 individuals. Our country has over 6,000 rare diseases that affect more than 25 millions Americans.

Both of these measures will provide necessary incentives to find a cure for these ailments. H.R. 4013 establishes an Office of Rare Diseases within the National Institutes of Health director’s office. In addition, the bill creates Rare Disease Regional Centers of Excellence to conduct research and training in the diagnosis, prevention, control and treatment of rare diseases. H.R. 4014 would double the funding to $25 million for the successful FDA grant program for research on orphan drugs. Since 1983, the FDAa has approved more than 200 treatments for rare diseases, this additional funding will be critical to increasing the number of treatments available.

A wonderful family in my hometown, the Kirches, brought the issue of rare diseases to my attention. I have had the opportunity to meet with Larry, Susan and their daughter Allyson to hear about their struggles and triumphs with Allyson’s battle with Mucopolysachharidosis (MPS) III. MPS III is a genetic disorder that results in the body’s inability to produce certain enzymes. This lack of enzyme production interrupts the usual breakdown of complex carbohydrates that are stored in almost every cell in the body. Without the breakdown, storage progressively builds in each cell causing damage in multiple systems within the body including respiratory, bones, internal organs, and nervous system. The results of this damage include mental retardation, short stature, cornea damage, loss of mobility, and most importantly a drastically shortened life span. At present there is no cure for MPS III.

Allyson’s future depends on investment in scientific and biomedical research by the public and private sector and, we owe it to all children with these disorders to make every effort to improve their quality of life and ultimately contribute to efforts in developing effective treatments. I urge my colleagues to support these measures and assist families like the Kirches all across our country.

Mr. DINGELL. Mr. Speaker, I rise in strong support of H.R. 4013, the “Rare Diseases Act of 2002,” of which I am proud to be an original cosponsor. I would like to thank my colleagues on both sides of the aisle for working together to introduce this important legislation and for working on behalf of the 25 million American people who suffer from rare diseases.

Rare, or orphan diseases affect fewer than 200,000 individuals in America. Nearly one million people in my home state of Michigan are afflicted with a rare disease. There are more than 6,000 rare diseases. Enactment of the Orphan Drug Act of 1983 provided incentives for drug and biological manufacturers to invest in treatment for rare diseases.

While the Orphan Drug Act has achieved dramatic increases in research into, and treatments for rare diseases, more still needs to be done. One positive step includes authorizing, in statute, the Office of Rare Diseases.

In addition, H.R. 4013 authorizes regional centers of excellence for rare disease research. This will enable the National Institutes of Health (NIH) to select sites to concentrate on finding cures and treatment methods for rare diseases.

I urge all of my colleagues to join me in support of H.R. 4013.

Mr. BILIRAKIS. Mr. Speaker, I rise today in support of H.R. 4013, the Rare Diseases Act of 2002. This bill, which was introduced by the gentleman from Illinois, Mr. SHIMKUS, would help improve research on rare diseases at the National Institutes of Health. I urge my colleagues to join me today in supporting this important piece of legislation.

A rare or “orphan” disease affects fewer than 200,000 people in the United States. There are more than 6,000 rare disorders that, taken together, affect approximately 25 million Americans. One in every 10 individuals in this country has received a diagnosis of a rare disease.

H.R. 4013 will help focus research on rare diseases at NIH. The bill also specifically gives NIH the authority to support regional centers of excellence in rare disease research. This bill will help strengthen our national research infrastructure in this area and improve our ability to treat and hopefully cure numerous rare diseases.

The Energy and Commerce Committee approved H.R. 4013 in late June, and I again urge my colleagues to support this important bill.

Mr. SHIMKUS. Mr. Speaker, I have no further requests for time, and I yield back the balance of my time.

The SPEAKER pro tempore (Mr. Boozman). The question is on the motion offered by the gentleman from Illinois (Mr. Shimkus) that the House suspend the rules and pass the bill, H.R. 4013.

The question was taken.

The SPEAKER pro tempore. In the opinion of the Chair, two-thirds of those present have voted in the affirmative.

Mr. WAXMAN. Mr. Speaker, I object to the vote on the ground that a quorum is not present and make the point of order that a quorum is not present.

The SPEAKER pro tempore. Pursuant to clause 8, rule XX and the Chair’s prior announcement, further proceedings on this motion will be postponed.

The point of no quorum is considered withdrawn.