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  Departments of Labor, Health and Human Services, and Education, and ..., Part 3
  By United States. Congress. Senate. Committee on Appropriations. Subcommittee on Departments of Labor, Health and Human Services, Education, and Related Agencies
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  has enabled D.E.B.R.A. to direct patients and families to several sources of excellent medical care and information. Additionally, the registry has stimulated cooperative clinical and laboratory studies on such subjects as wound healing, cellular grafting, and gene mapping
  Yet despite all the encouraging news, I am worried about future progress. Since its establishment as a separate institute in 1987, NIAMS has not been fully funded. The substantial amount of additional funds that were allocated in fiscal year 1991 were certainly appreciated. However, 73 percent of approved research grants still go unfunded.
  D.E.B.R.A. strongly supports the recommendations of the coalition of voluntary and professional associations concerned with the programs of NIAMS. And they urge you to add $47 million to fiscal year 1992 budget. Without this additional support there will be no new centers, no new clinical trials, and fewer and fewer approved research grants.
  PREPARED STATEMENT D.E.B.R.A. continues to work at providing support, information, and assistance to our families, but there are some things we cannot do, and it is for these that we come to you, to ask you to continue to build and strengthen your investment in EB research through adequate funding to NIAM.
  [The statement follows:)
  STATEMENT OF SHARON Wanat, BOARD OF TRUSTEES, DYSTROPHIC EPIDERMOLYSIS
  BULLOSA RESEARCH ASSOCIATION (D.E.B.R.A.) Mr. Chairman, members of the subcommittee, I am Sharon Wanat, a member of the Board of Trustees of the Dystrophic Epidermolysis Bullosa Research Association (D.E.B.R.A.) of America. I am also a newly appointed member of the National Advi. sory Board for the Arthritis and Musculoskeletal and Skin Diseases Institute of the National Institutes of Health.
  This is the eleventh year that a representative from D.E.B.R.A. of America has come before this committee and I thank you for once again giving us this opportunity.
  I am here to speak to you on behalf of the more than 50,000 infants, children and adults who suffer from Epidermolysis Bullosa. Epidermolysis Bullosa is a group of inherited diseases in which an unknown defect in the skin causes painful blistering and wounds. The cycle of repeated blistering and inadequate healing to fragile skin can lead to scars which form over the entire body surface as well as in the mouth, throat, eyes and other internal organs. Although Epidermolysis Bullosa primarily affects the skin, this disease has dire essects on many other systems of the body. Common complications include malnutrition, chronic anemia, hand and joint deformities and skin canær, which can lead to amputation and even death.
  This is a disease that severely assects the quality and duration of life. It is disfiguring, severely disabling and very often fatal. People find it unbelievable that a skin disease could be fatal! However, most infants with the junctional form of EB do indeed die before their first birthday. My own son, Edward, died at eleven months as a result of respiratory and urinary tract complications. Others with the severe forms of recessi dystrophic disease die in childhood. Many patients do reach adulthood but are locked in a malnourished, child-like body with little hope of reaching an adult's normal physical stature or maturation. The progressive nature of the disfigurement and disabilities makes independent living a rare event. The social and emotional consequences and the financial burden to both the patient and the family are enormous.
  DEBRA of America was founded in 1980 to offer support and information to patients and families with EB. Soon after its inception, it became clear that the only way to conquer this dread disease was through medical research. Since 1981, when DEBRA first came to the Congress, the support of this committee has been outstanding. Your support has dramatically increased the amount of money being spent on EB research. We are very grateful. I am please to report that the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) is presently supporting several research projects on EB. Although to date there has been no clear breakthrough, there has been significant progress in understanding the basic molecular structure of the basement membrane zone, that area between the outer and inner layers of skin. There is also an exciting possibility that gene mapping research already in progress will lead to the discovery of a genetic marker for Epidermolysis Bullosa.
  In 1986, the National EB registry was created by the mandate of this committee. Since then, over 1,500 patients have been enrolled in the registry. The registry has enabled DEBRA to direct patients and families to several sources of excellent medi. cal care and information. Additionally, the registry has stimulated cooperative clinical and laboratory studies on such subjects as wound healing, cellular grafting, skin cancer, and gene mapping.
  Yet, despite all the encouraging news, I am worried about future progress. Since its establishment as a separate Institute in 1987, NIAMS has not been fully funded. The substantial amount of additional funds that were allocated in fiscal year 1991 were certainly appreciated. However 73 percent of approved research grants go unfunded. Furthermore, the lack of resources for career development awards and training fellowships for NIH as a whole, and specifically for NIAMS, is discouraging young people from pursuing careers in research. And what about the opportunities and hope that the National EB Registry holds? We need to be assured that NIAMS will have sufficient funds to renew the contract for the registry.
  My concerns also include support for research centers. Unfortunately, because of inadequate funding, NIAMS has only been able to establish two core skin centers rather than the six that were mandated. We urge you to remain committed to the support of centers.
  It is for all the reasons that I have just given that we strongly support the recommendations of the Coalition of Voluntary and Professional Associations Concerned with the Programs of the National Institute of Arthritis and Musculoskeletal and Skin Diseases and urge you to add $47 million to the fiscal year 1992 budget for NIAMS. Without this additional support, there will be no new centers, no new clinical trials and fewer and fewer approved research grants will be funded.
  D.E.B.R.A. has entered its second decade of service and accomplishments. We salute the thousands of patients and families who have helped to build our organization. We continue to work at providing support, information and assistance to our families. But there are things I cannot do. And it is for these things that we must come to you-to ask you to continue to build and strengthen your investment in EB research through adequate funding for the National Institute for Arthritis and Musculoskeletal and Skin Diseases. Your support has made a difference. I urge you to continue to make a difference. Senator BURDICK. Mr. Mumber.
  Mr. MUMBER. Good morning, Senator. Our daughter, Rebecca, was born in 1989 and diagnosed with a junctional variety of EB. We were told most infants do not survive more than a year. In fact, Rebecca survived 13 months; she died last December.
  In her 13 short months of life, Rebecca was hospitalized 150 days. This imposed a terrible strain on our entire family, since we also had a very healthy and active 2-year-old son, Nicholas. We often would not see our son for weeks at a time when Rebecca was hospitalized, and would be sent to live with relatives out of State. We could not really leave Rebecca alone in the hospital, because doctors and nurses really would not know how to care for her. They would try to treat her like any normal child, handle her normally, and doing that would greatly compromise her skin.
  When Rebecca was not hospitalized she required pediatric home nursing care for 40 hours a week at a cost of roughly $1,600 a week. Rebecca received the best medical care at Children's Hospital in Philadelphia, but while my health insurance coverage is one of the better ones, we still incurred significant out-of-pocket expenses. We had to do daily dressing changes on the open areas of her skin. It took 2 hours a day to do it.
  Dressing supplies cost over $50 a day in the beginning, and it escalated to over $100 a day toward the end. Obviously, the majority of families cannot afford this, and most insurance companies do not reimburse for routine dressing supplies.
  Our Rebecca was a beautiful child, and she was a fighter with an indomitable spirit, despite her many afflictions. What she had to endure in her life was more than any infant should bear, should have to suffer.
  These kids really have a strong will to survive, and despite all her problems, Rebecca never complained, except when we were doing her dressing changes. Even when we were done, she always gave us a warm smile.
  It is for this spirit that I really ask you to support the research for these children.
  Senator BURDICK. Thank you. How much in additional funding would be necessary to renew the contract for the registry?
  Ms. WANAT. I am not sure of that number, Senator, right off. We will submit that number to you, but I am afraid I do not have that number right offhand.
  Senator BURDICK. Can you do it within 5 days? Ms. Wanat. Yes; of course. Senator BURDICK. Very fine. That is all, thank you. Ms. WANAT. Thank you very much. [The information follows:] Funding needed to extend the contract for the National Epidermolysis Bullosa Registry. The proposal has been under review by the coordinating Center and the four participating clinical centers since April because the scope of the Registry has been expanded. The ænters now estimate that the direct first year costs for the EB Registry will be slightly over $1 million.
  The Registry seeks to foster and support research which is aimed at the following purposes: to search for the basic defect(s) in EB; to define improved means of diagnosis of EB; to develop esfective means of treatment and prevention of EB; and to study the genetic and epidemiologic factors which are operative in EB. STATEMENT OF ROBERT HUMPHREYS, EXECUTIVE DIRECTOR, NA
  TIONAL COALITION ON IMMUNE SYSTEM DISORDERS ACCOMPANIED BY MARCIA BOYLE, PRESIDENT, IMMUNE DEFICIENCY
  FOUNDATION Senator BURDICK. Robert Humphreys, executive director, and Marsha Boyle, president, Immune Deficiency Foundation. Welcome to the committee.
  Mr. HUMPHREYS. Good morning, Senator. I am Robert Humphreys, I am here before you this morning in my capacity as executive director of the National Coalition on Immune System Disorders. And with me is Marcia Boyle, who is the president of the Immune Deficiency Foundation, and is also a parent of a child with immune deficiency disease.
  The coalition is an organization which supports basic biomedical and clinical immunological studies through not only the National Institute on Allergy and Infectious Diseases, but also through all the other NIH Institutes that engage in such research.
  Our coalition membership includes such widely diverse groups as the American Association of Immunologists, the American Academy of Otolaryngology, Head and Neck Surgery, Crohn's and Ileitis Foundation of America, the Immune Deficiency Foundation, as I mentioned, the Myasthenia Gravis Foundation, and the Multiple Sclerosis Society. In short, our membership is as diverse as are the diseases that affect the immune system, Senator.
  We believe that less and less attention, relatively, has been spent on immune system diseases that are non-AIDS related in the wake of the very strong and increasing interest of the Congress and in the Nation in AIDS research. We feel as a result that non-AIDS immunology research has, if not been given short shrift, at least has not been given the kind of attention that it should have been over the past several years.
  PREPARED STATEMENT
  At this juncture, Senator, I would like to ask Mrs. Boyle to reflect for you some of the activities that her foundation undertakes, but also from her own personal experience what the needs in immune system research are.
  [The statement follows:]
  STATEMENT OF THE NATIONAL COALITION ON IMMUNE SYSTEM DISORDERS The National Coalition on Immune System Disorders (NCISD) is an organization open to any society, group, or association whose members support research in basic and clinical immunology. NCISD supports such research whether it takes place under the auspices of the National Institute of Allergy and Infectious Diseases, or through other NIH institutes. Coalition members include such widely diverse groups as the American Association of Immunologists; American Association Academy of Otolaryngology-Head and Neck Surgery; Crohn's and Ileitis Foundation of America; Immune Deficiency Foundation; Myasthenia Gravis Foundation; and National Multiple Sclerosis Society.
  In addition, the Clinical Immunology Society, the Asthma and Allergy Foundation of America, the American Academy for Otolaryngic Allergy, the American Academy of Dermatology, and the American Diabetes Association support the coalition's efforts. Essential support is provided through the Coalition's sustaining members, currently Sandoz Pharmaceutical Corporation and Abbott Laboratories, and from Immunetech.
  Although the general public associates the National Institute of Allergy and Infectious Diseases (NIAID) chiefly with research on HIV and AIDS, the Institute is much more than that. This Coalition supports expanded NIAID research activities in non-AIDS areas. We believe non-AIDS research has suffered in recent years because of the pressure to devote increasing resources to HIV research. NCISD does not oppose AIDS research, but we do strongly urge that renewed attention be devoted to other badly needed immune system research relating to diseases, disorders and conditions which deserve a much higher research priority.
  Just as NCISD's membership is diverse, so are the diseases and disorders with respect to which we urge increased research effort: cancers, rheumatoid arthritis, lupus, multiple sclerosis, myasthenia gravis, Sjogren's Syndrome, psoriasis, diabetes, severe combined immune deficiency and other primary immune deficiency diseases, otitis media, and many others have proven or suspected immune system involvement. At least 50 million Americans have one or more of these diseases and disorders.
  NCISD endorses a level of funding for new and competing non-AIDS research project grants (RO-1s) which will support an award rate of 50 percent of approved grants, and which will fully fund continuation grants at council-approved levels. This contrasts with the fiscal year 1991 situation, under which NIAID could support only 26.5 percent of approved new and competing grants, and the President's fiscal year 1992 budget, under which NIAID support would be reduced to 24.4 percent of approved grants.
  If funding were approved at these recommended levels, NIAID research attention would be directed at research areas such as the genetic basis and molecular pathophysiology of the primary immune deficiency diseases; autoimmunity in diabe, tes mellitus, celiac disease, and inflammatory bowel disease; environmental immunotoxicology; and new vaccines for immunologic diseases, just to name a few exciting new and expanded research avenues.
  At the same time, full funding for investigator-initiated research grants and research centers would be possible, rather than the anticipated “programmatic reductions", or "downward negotiations” that will result in grants which are 10 to 16 percent smaller than those approved by the peer reviewers and council.
  For a number of years, the members of NCISD have been deeply concerned that the Nation's research priorities have been skewed to the point that important scientisic discoveries have been delayed, probably for many years. Research that holds the promise of revealing the causes ol, cures, and therapies for countless immune system disorders simply has not been done. Congress can begin to reverse this damaging process by adopting the funding levels for NIAID non-AIDS research which NCIŠD endorses.
  Attached as an appendix to this statement is a table showing comparative dollar levels for the various activity categories of NIAID for fiscal year 1991, the President's budget for fiscal year 1992, and NCISD's recommendations for fiscal year 1992.
  Thank you for your consideration of this request.
  Ms. BOYLE. Thank you very much. The Immune Deficiency Foundation represents over 80 primary or genetic immune deficiency disorders ranging from severe combined immune deficiency, which I will refer to as SCID—that is the disease of the boy in the bubble—to less severe disorders found in 1 out of 500 Americans.
  To illustrate where we are today, my neighbor came to me the other day concerned because one of her friend's baby had just been diagnosed with SCID. When my son was diagnosed 13 years ago with an immune deficiency, we had to wait several days to discover whether it was an antibody disorder or whether it actually was SCID. Luckily, it was an antibody disorder. For then, had it been SCID, it would have been a death sentence.
  The good news is that today the SCID baby has a decent chance at survival because of research advances that have brought about