Position Paper
Position Statement

Potential Impact of Growth Hormone Re-Classification
To a Schedule III drug on the US Population

The MAGIC Foundation
March 10, 2008

There are more than 6,000 rare, or “orphan,” diseases that affect about 25 million Americans. (A rare or “orphan” disease is one that affects fewer than 200,000 people in the United States.) Most endocrine disorders requiring growth hormone classify as rare disorders.

How many people does the re-classification of Growth Hormone to a Schedule III affect?
United States / Frequency  from: http://www.emedicine.com/PED/topic2087.htm#IntroductionFrequency
 “By definition, 2.5% of the population is short. However, the number of children with poor linear growth is higher given the frequency of chronic diseases of childhood. The Utah Growth Study is the largest population-based survey of growth in children published to date. These investigators assessed height and growth velocity in nearly 115,000 American children. Among the 555 children with short stature”, “only 5% had an endocrine disorder. In addition, 48% of the children with growth hormone deficiency (GHD) or Turner syndrome (TS) in this large cohort had been undiagnosed or untreated.”
According to the National Institutes of Health, severe “short stature may be a symptom” caused by a treatable “medical condition”. The Pituitary Network reports that 65 million Americans suffer from pituitary and hormonal disorders.

Causes of Growth Failure  (This list is NOT all-inclusive.)

• Pediatric AIDS
• Chronic diseases such as congenital heart disease, kidney diseases, asthma, sickle cell anemia, thalassemia, juvenile rheumatoid arthritis, and diabetes
• Craniopharyngioma (brain tumors)
• Cushing's disease
• Delayed puberty 
• Hypopituitarism
• Hypothyroidism that develops before birth
• Inflammatory bowel disease
• Malabsorption disorders such as celiac disease
• Pituitary dwarfism
• Precocious puberty 
• Small for Gestational Age
• Idiopathic Short Stature
• Russell-Silver syndrome 
• Turner syndrome
http://www.nlm.nih.gov/medlineplus/ency/article/003271.htm
Following is a table outlining a small sample of conditions and the number of children impacted by Growth Hormone based on the U.S. Dept. of Health and Human Svcs., Cntrs. for Disease Control and Prevention, National Center for Health Statistics. However, in reviewing these statistics it is important to note that:

1. According to the Utah study quoted above, 48% of the children with conditions impacted by growth hormone are not diagnosed. Therefore, one could deduct that this table may only reflect half of the affected population for Turner Syndrome and Growth Hormone Deficiency.

2. Other conditions such as Kidney Disease, Brain Tumors, Leukemia, AIDS (and many others), are NOT included in these totals. Incorporating statistics from those additional illnesses would dramatically increase the number of children (and consequentially affected adults) impacted. This table reflects the more rare and little known conditions for your basic and initial understanding of how wide spread the devastation will be with a Re-classification of growth hormone to a Schedule III drug.

Medical Condition	Ratio	Source	Newborn children affected in 2005 based on the births recorded	Minimum sample number of children affected based on the last Census 2000 reporting 80M+ children under the age of 19
Noonan Syndrome	1:1,000 to 1:2,500	http://rarediseases.info.nih.gov/Wrapper.aspx?src=asp/html/resources/info_cntr.html&PageID=4	1655 (or as much as 4,138)	48,338 (but could total as many as 74,484)
Turner Syndrome	1:2,500 female births	http://rarediseases.info.nih.gov/Wrapper.aspx?src=asp/html/resources/info_cntr.html&PageID=4	1655	48,338
Hypopituitarism	1:333,333 (3:1,000,000)	http://www.emedicine.com/ped/topic1130.htm	12	24,000
Panhypopituitarism		Office of Rare Diseases (ORD) of the National Institutes of Health (NIH)	21	200,000
Congenital GHD	1:50,000 births	http://www.emedicine.com/PED/topic1810.htm	82	1,476
SOD/ONH	1:10,000	Cathy Clark, FOCUS	143	2,574
CAH	1:10,000 to 18,000	NIH	230 (but could be as high as 413)	4,140
Small for Gestational age. IUGR	14:1,000	http://www.cdc.gov/nchs/fastats/birthwt.htm	338,565 (8.2% of all babies)	6,094,170
Idiopathic Short Stature	3:100	Pediatric and Adolescent Medicine, March 2006, Vol. 160, No. 3. Joyce Lee, M.D.		400,000 (ages 4-15 only)
Fetal Alcohol syndrome	1:100	http://www.nofas.org/faqs.aspx?id=12	40,000	720,000
Russell-Silver Syn.	1:100,000 (some estimates are 1:50.000)	The MAGIC Foundation	41	738
Prader Willie Syn.	1:12,000 to 1:15,000	http://www.pwsausa.org/	275 (to 345)	4,950
Growth Hormone Deficiency	1:3,480 derived from Utah Study	Journal of Pediatrics 1994:125:29-35, Lindsay R, et al	1,189	22,988
TOTALS			383,867 new children each year	7,551,712 children under the age of 19

(This chart does not reflect any adults also impacted by these conditions who would also be affected by the Classification change.)
 
In the article, Growth Hormone Deficiency, Updated: Apr 19, 2006, Stephen Kemp, MD, PhD, Professor, Department of Pediatrics, Section of Pediatric Endocrinology, University of Arkansas and Arkansas Children's Hospital reported that:  “Of more than 20,000 children receiving GH in the National Cooperative Growth Study (a database of patients receiving GH therapy), approximately 25% of the GH-deficient patients had an organic etiology.

These etiologies included the following:
• CNS tumor, including craniopharyngioma - 47%  
• CNS malformation - 15%
• Septo Optic Dysplasia - 14%
• Leukemia - 9%
• CNS radiation - 9%
• CNS trauma / infection- 4%
• Histiocytosis - 2%”

The MAGIC Foundation has more than 170 different medical conditions impacting children’s healthy growth referenced by parents in the database. However, worldwide the database P.O.S.S.U.M. reports in excess of 600 medical conditions correlate to growth failure. Families who have already braved their own private medical battle should not be forced to battle greater logistical issues due to constraints brought about by celebrity abuse. Who is being protected, the children, affected adults – their families or the athletes who broke the law to obtain the drug?

How will this change impact families?
The Federal Laws applied will enforce additional laws and restrictions applied on the state level and Insurance policies will all be “revisited” with regard to each child’s Growth Hormone Deficiency as it will become part of an entirely “new category” of medication. This re-evaluation could facilitate horrible delays pertaining to Insurance considerations, delay in obtaining refills and a world of paperwork nightmares which the majority of families endured when their child was initially diagnosed. Children and or adults who need this drug (for example) to avoid hypoglycemic comas could potentially face life threatening dangers without regular treatments.

One family submitted personal financial data to help us convey how Schedule III labeling and the subsequent State Schedule III laws will impact their child and family in New Jersey.  Ironically, New Jersey is not impacted as severly as families in New York and other states.

Miles (82 round trip)	$39.77
Tolls	$8.00
parking	$17.00
Co-Pay	$35.00
Lost income/trip	$344.44
Cost Per Trip	$444.21
Current Annual Cost	$1,776.86
If Schedule III is passed/ Future Annual Cost	$5,330.57
Additional Annual Cost to Family	$3,553.72