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Rare Diseases Act Elaine and I, as the USA portion of your list co-owners, have been working on something that needs the urgent attention of our members in the United States. Actually, it's something that may ultimately be very helpful to all of us even though it is happening in the United States, but only USA members need to act on this. The following information is from Diane Dorman, Senior Director for Public Policy for the National Organization for Rare Disorders (NORD). It talks about legislation recently introduced by Senator Edward Kennedy that could significantly increase funding and facilities for research and treatment of rare disorders, as well as increase support for funding the development of "orphan drugs". Diane suggests that anyone who would like to support this legislation can (actually we've added a couple of suggestions to hers):
Thanks, Elaine Jacobson and Joan Reed From Diane Dorman of NORD On August 3 Senator Edward Kennedy and Senator Orrin Hatch introduced the Rare Diseases Act of 2001 (S. 1379). Not since the passage of the Orphan Drug Act has such an important piece of legislation been introduced that positively impacts the entire rare disease community of 25 million Americans. In the text of S. 1379, Congress made the following findings: For many years, the 25 million Americans suffering from over 6,000 rare disorders were denied access to effective medicines because prescription drug manufacturers could rarely make a profit from marketing drugs for such small groups of patients. The Orphan Drug Act (ODA) created financial incentives for research and development of such "orphan drugs." Despite the tremendous success of the ODA, rare diseases and disorders deserve greater emphasis in the national biomedical research enterprise. The National Institutes of Health (NIH) has received substantial increases in research funding from Congress for the purpose of expanding the national investment in behavioral and biomedical research. Notwithstanding such increases, funding for rare diseases and disorders at the NIH has not increased appreciably. The Food and Drug Administration (FDA) supports small clinical trials on new treatments for rare disorders through Orphan Products Research Grants. Yet the appropriations in Fiscal Year 2001 for such research grants were less than in Fiscal Year 1995. What does the Rare Diseases Act of 2001 do? 1 Provides a statutory authorization for the existing Office of Rare Diseases (ORD) at the NIH. 2. Increases the national investment in the development of diagnostics and treatments for patients with rare disorders. 3. Authorizes regional centers of excellence for rare disease research and training. 4. Increases funding for the NIH Office for Rare Diseases to $24 million for fiscal year 2002 and "such sums as may be necessary for each subsequent fiscal year." 5. Increases the funding for the FDA's Orphan Product Research Grants program, which has provided vital support for clinical research on new treatments for rare disorders to $25 million for fiscal year 2002, and "such sums as may be necessary for each subsequent fiscal year." Suggested letter for your local Senators as per suggestion (1) in our Post #1 Write to your U. S. Senators asking them to cosponsor the Rare Diseases Act of 2001. This same letter could be used for recommendation (3), writing each member of the Health, Education, Labor and Pensions Committee. You can get their contact information by clicking on their individual names under their pictures. Sending to them at U.S. Senate, Washington, DC 20510 may well work, though they do have more complete addresses that might reach them quicker. Sen. Dodd, for instance, is located at 448 Russell Senate Office Building. If you don't have time to look them up but would send off letters, the Chairman of that Committee is Sen. Edward M. Kennedy. The Majority (Democratic) members of the committee are Sens. Christopher Dodd, Tom Harkin, Barbara Mikulski, James Jeffords, Jeff Bingaman, Paul Wellstone, Patty Murray, Jack Reed, John Edwards, and Hillary Clinton. The Minority (Republican) members of the Committee are Sens. Judd Gregg (Ranking Member), Bill Frist, Mike Enzi, Tim Hutchinson, John Warner, Christopher Bond, Pat Roberts, Susan Collins, Jeff Sessions, and Mike DeWine. Note that Sen. Kennedy is sponsoring this legislation, and that Sens. Hollings and Bingaman have signed on as co-sponsors. Sen. Bingaman is a member of this Committee, but Hollings is not. We need as many Senators to sign on as co-sponsors as possible. Those that co-sponsor we know will vote for it. At least in NY, only the majority members are "allowed" to sign on as co-sponsors. I don't know if that's the case in the U. S. Senate. I would hope not, but you never know. Politics.... We can ask all to co-sponsor anyway. Diane Dorman of NORD provided the following, to which we've made a few changes. Because of the number of lines our listserver allows, and the fact it doubles the number if we type in anything but basic plain text format as it has to convert everything we type otherwise, I've not included any of the highlighting she had in her original. Therefore, I've put asterisks around what should be bold if you can do that. You might also highlight the bill number every time you see it, also your state's name and the number of estimated folks with rare diseases in your state. Please note there are a few places within the letter for you to include your own state-specific information. Read it carefully. We've put asterisks, as well as the brackets she used, to help you notice where these things must be inserted. Then, of course, delete all information that should not be in the final version. Also, make sure you put your name and address at the top of the letter as I've indicated. Anonymous letters don't go far. Note the last paragraph suggested by Diane Dorman. It puts them on notice that we'll be looking for their support of this bill. "Working closely with them" doesn't mean a lot on a personal basis. It may include sending another letter or making a phone call. The more they hear from us, the better the chances, so don't be afraid to do that. If you prefer not to use that wording, though, feel free to change it. The fewer "form" letters they receive, the better...but not the fewer letters in all. Please DO send something, even if you feel you're going to miss the deadline of the presumed committee meeting next week. If this bill passes their committee, it will most likely go on to another and still need your support. Note that you need the Census information in Post #3 to complete this letter. --Elaine Jacobson and Joan Reed
SAMPLE LETTER ASKING YOUR U.S. SENATOR TO COSPONSOR THE RARE DISEASES ACT OF 2001 (S. 1379) (start at center-->) (Your street address) (Your city, state, zip code) (Date of letter) The Honorable _____________________ United States Senate Washington, DC 20510 Dear Senator __________________: I am writing to you today to ask that you sign on as a co-sponsor of an important Senate bill. On August 3, 2001, Senators Orrin Hatch and Edward Kennedy introduced the **[make this bold] Rare Diseases Act of 2001 (S.1379).** The legislation provides a statutory authorization for the existing **[make this bold] Office of Rare Diseases (ORD)** at the National Institutes of Health (NIH) and increases the funding for the Food and Drug Administration's Orphan Product Research Grants Program, which has provided vital support for clinical research on new treatments for rare disorders. S.1379 also increases the national investment in the development of diagnostics and treatments for patients ith rare disorders, authorizes regional centers of excellence for rare disease research and training, and boosts funding for the ORD to $24 million for fiscal year 2002. [The second paragraph should go into detail about how S. 1379 will personally benefit you, your family, other rare disease patients and their families.] [We're including the following 3 paragraphs as a suggestion for this information. Use it if you like; or better yet, write your own. Try not to make it too long (mine's a bit long) and adjust your margins and font size so that you can get it on one side of the page if possible....if you're sending it by regular mail. Remember, it's the next **3** paragraphs that are XLH-specific and which you may want to word yourself.] The particular condition which affects my family is called X-Linked Hypophosphatemia, or XLH for short, caused by a genetic mutation of a gene on the X chromosome. Nearly all patients with XLH develop rickets in childhood, causing bowing of the lower extremities and slowing of growth, regardless of the amount of vitamin D in the diet or the amount of time spent outdoors in the sunshine. Because it affects only approximately one in 20,000 people around the world, it is very difficult to find a physician who has ever seen a case, let alone one who has experience in treating it. Although it was published 43 years ago to be a known hereditary disorder, we are often at the mercy of clinicians unfamiliar with this rarely-seen condition who either fail to diagnose the problem early enough to prevent life-long deformities, even with a known family history, or who treat it in a way that seems right but actually is not. Some insurance companies don't recognize our need for specialty care. Astonishingly many medical labs do not post pediatric normals for phosphorus on their report forms, nor do they warn that they don't, which has led physicians to believe their patient is not affected by this disorder until the child falls below the much lower adult normal range. Every human being carries several genetic mutations; some are evident and some are not. XLH happens to be one that causes a medical problem, and new spontaneous cases are being diagnosed all the time. Children should not have to needlessly suffer the effects of a very treatable condition. We need to make it possible for more physicians to learn how to diagnose and treat this and other rare conditions. Another problem we periodically encounter is temporary production stops of medications we need to replace what our body does not appropriately utilize. We're currently experiencing that with one product for which there is no direct substitute, and supplies in warehouses are running out. Other medications that had been found in research trials to be effective in treating our condition have been discontinued because they were found not profitable enough to the drug companies because they weren't also helpful for disorders with a larger market. The Rare Diseases Act of 2001 would encourage the development of new treatments and the continuation of those that help fewer people. It would also enable more medical students to learn about our condition, so that proper treatment is more accessible. Diagnosis should come easier and earlier, and S.1379 will help that to happen, and it may well encourage a cure to be found. I urge you to cosponsor the Rare Diseases Act of 2001 to ensure that the nearly **[see census chart in Post #3 for number of constituents in your state suffering with rare diseases]** constituents in **[your state]** with rare disorders receive the care and treatment they so desperately need and deserve. I look forward to hearing from you very soon, as we wish to work closely with you to ensure passage of the Rare Diseases Act of 2001 that will provide increased clinical research and treatments for the 25 million Americans in the United States suffering with rare "orphan" diseases. Sincerely, (type your name and sign it above) ------------------------------------------------------------------
This is the Census information you'll need to complete the letter in Post #2: --Elaine Jacobson and Joan Reed Census Info Chart Don't send this chart with the letter, but refer to it for the information you need in the letter. The state populations are from April 2000 Census numbers. U.S. State Est. # of Residents State Population with Rare Disorders (9.5%)* Alabama 4,447,100 422,475 Alaska 626,932 59,559 Arizona 5,130,632 487,410 Arkansas 2,673,400 253,973 California 33,871,648 3,217,807 Colorado 4,301,261 408,620 Connecticut 3,405,565 323,529 Delaware 783,600 74,442 District of Columbia 572,059 54,346 Florida 15,982,378 1,518,326 Georgia 8,186,453 777,713 Hawaii 1,211,537 115,096 Idaho 1,293,953 122,926 Illinois 12,419,293 1,179,833 Indiana 6,080,485 577,646 Iowa 2,926,324 278,001 Kansas 2,688,418 255,400 Kentucky 4,041,769 383,968 Louisiana 4,468,976 424,553 Maine 1,274,923 121,118 Maryland 5,296,486 503,166 Massachusetts 6,349,097 603,164 Michigan 9,938,444 944,152 Minnesota 4,919,479 467,351 Mississippi 2,844,658 270,243 Missouri 5,595,211 531,545 Montana 902,195 85,709 Nebraska 1,711,263 162,570 Nevada 1,998,257 189,834 New Hampshire 1,235,786 117,400 New Jersey 8,414,350 799,363 New Mexico 1,819,046 172,809 New York 18,976,457 1,802,763 North Carolina 8,049,313 764,685 North Dakota 642,200 61,009 Ohio 11,353,140 1,078,548 Oklahoma 3,450,654 327,812 Oregon 3,421,399 325,033 Pennsylvania 12,281,054 1,166,700 Rhode Island 1,048,319 99,590 South Carolina 4,012,012 381,141 South Dakota 754,844 71,710 Tennessee 5,689,283 540,482 Texas 20,851,820 1,980,923 Utah 2,233,169 212,151 Vermont 608,827 57,839 Virginia 7,078,515 672,459 Washington 5,894,121 559,941 West Virginia 1,808,344 171,793 Wisconsin 5,363,675 509,549 Wyoming 493,782 46,909 Total U.S. Population Estimate of those with Rare Disorders 281,421,906 26,735,081 Source: U.S. Department of Commerce, U.S. Census Bureau. *Based on FDA and NIH estimates that approximately nine to ten percent of the U.S. population suffers with rare disorders
Sample Letter to Senator Kennedy & Hatch --Elaine Jacobson and Joan Reed
SAMPLE THANK YOU LETTER TO HATCH AND KENNEDY The Honorable Edward M. Kennedy, Dear Chairman Kennedy: **OR, for letter to Sen. Hatch:** The Honorable Orrin Hatch Dear Senator Hatch: As a member of The XLH Network whose family numbers among the 25 million Americans suffering with rare disorders, I want to thank you for introducing the Rare Diseases Act of 2001 (S. 1379). This landmark legislation addresses the needs of the rare disease community, which have been neglected and overlooked for too long. The XLH Network represents patients suffering with **[detailed information about your organization. Also go into detail about the disease group you represent and how passage of S. 1379 will impact your organization - this is most important. The ORD and the Research Grant Program are the only two federal resources available to the rare disease community.] Note from Joan and Elaine: You could include the same 3 XLH-specific paragraphs from the letter in Post #2, which I'll paste in below. If you wish to use your own wording (the preferred choice if you can), delete these *3* paragraphs or the parts you're not using. Again, try to make this fit on one side of a letter by adjusting margins and type sizes.** The particular condition which affects my family is called X-Linked Hypophosphatemia, or XLH for short, caused by a genetic mutation of a gene on the X chromosome. Nearly all patients with XLH develop rickets in childhood, causing bowing of the lower extremities and slowing of growth, regardless of the amount of vitamin D in the diet or the amount of time spent outdoors in the sunshine. Because it affects only approximately one in 20,000 people around the world, it is very difficult to find a physician who has ever seen a case, let alone one who has experience in treating it. Although it was published 43 years ago to be a known hereditary disorder, we are often at the mercy of clinicians unfamiliar with this rarely-seen condition who either fail to diagnose the problem early enough to prevent life-long deformities, even with a known family history, or who treat it in a way that seems right but actually is not. Some insurance companies don't recognize our need for specialty care. Astonishingly many medical labs do not post pediatric normals for phosphorus on their report forms, nor do they warn that they don't, which has led physicians to believe their patient is not affected by this disorder until the child falls below the much lower adult normal range. Every human being has several genetic mutations; some are evident and some are not. XLH happens to be one that causes a medical problem, and new spontaneous cases are being diagnosed all the time. Children should not have to needlessly suffer the effects of a very treatable condition. We need to make it possible for more physicians to learn how to diagnose and treat this and other rare conditions. Another problem we periodically encounter is temporary production stops of medications we need to replace what our body does not appropriately utilize. We're currently experiencing that with one product for which there is no direct substitute, and supplies in warehouses are running out. Other medications that had been found in research trials to be effective in treating our condition have been discontinued because they were found not profitable enough to the drug companies because they weren't also helpful for disorders with a larger market. The Rare Diseases Act of 2001 would encourage the development of new treatments and the continuation of those that help fewer people. It would also enable more medical students to learn about our condition, so that proper treatment is more accessible. Diagnosis should come easier and earlier, and S.1379 will help that to happen, and it may well encourage a cure to be found. Your long-standing support of the rare disease community is deeply appreciated, and we applaud your efforts to increase funding for both the Office of Rare Diseases at the National Institutes of Health and the Orphan Product Research Grants Program at the Food and Drug Administration. You can be assured that the XLH Network and its members will work tirelessly on your behalf to ensure passage of the Rare Diseases Act of 2001. Sincerely, (type your name and sign it above) Last modified Aug 8, 2007 XLH is also known as X-Linked Hypophosphatemia (sometimes also spelled as hypophosphataemia), X-Linked Hypophosphatemic Rickets, Familial Hypophosphatemia, Vitamin D-Resistant Rickets (VDRR) Rickets and even Genetic Rickets. Its notable characteristics are bowed legs, short stature, poor teeth formation causing spontaneous dental abscesses, and low blood phosphorus levels.
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