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U.S. Department of Health and Human Services

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Orphan Drugs

<< Return to FY 2005 Budget Proposal


Department of Health and Human Services
Food and Drug Administration
Fiscal Year 2005


Office of Orphan Products Development


  FY 2003
FY 2004
Enacted 1/ /2
FY 2005
Increase or

Program Level

$16,002,000 $16,006,000 $16,006,000 -


$13,270,000 $13,192,000 $13,192,000 -


$2,732,000 $2,814,000 $2,814,000 -
1/Contains a Budget Authority rescission of 0.59 percent for a total of $2,694,000.
  2/Cost of living increase included in Other Activities. 



The Orphan Drug Act (ODA) (P.L. 97-414) amended the Federal Food, Drug, and Cosmetic Act, as of January 4, 1983, and established that the Federal government would provide incentives to assist and encourage the identification, development, and availability of safe and effective treatments for rare diseases/disorders.  Orphan drugs are medications developed under a special program that assists the manufacturers of products needed to treat a potential patient population that is too small for profitable drug research, development, and marketing.  Orphan drugs provide important breakthroughs for patients who would otherwise be left lacing therapy. 

Since its creation in 1982, the Office of Orphan Products Development (OOPD) has been dedicated to promoting and encouraging the clinical development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions, affecting fewer than 200,000 persons or affecting more than 200,000 persons but not expecting to recover development costs, plus a reasonable profit, within seven years following FDA approval.




OOPD administers the major provisions of ODA that provide incentives for sponsors to develop products for rare diseases.  OOPD is organizationally located within the Office of Science and Health Coordination.  The orphan products development program includes identification of orphan products and the facilitation of their development.  In addition, the OOPD administers the Orphan Products Grant Program that provides funding for clinical research in rare diseases.  Although this grant has been expanded to include clinical studies for medical foods and devices that meet the "orphan" criteria established by Congress, ODA pertains primarily to drug and biological products.

The 1983 ODA guarantees the developer of an orphan product seven years of market exclusivity following the approval of the product by FDA.

Since the passage of the ODA, OOPD has designated 1,263 products to treat rare diseases.  245 of these orphan products have received FDA market approval, and are now available to treat a potential patient population of more than 12 million U.S. citizens. Thirty-three of these approved orphan products were developed with funding from the orphan product grant program.

In contrast to this pace of designating drugs to treat rare diseases, the decade prior to 1983 saw fewer than 10 such products come to market.  OPD received 152 applications for orphan designation during FY 2003.  Based on reviews conducted by OPD medical, and pharmaceutical review staff, 90 products received designation as orphan products.  Fourteen orphan products received FDA market approval for the treatment of rare diseases in FY2003.  See Table below.


Orphan Product Drugs Approved in FY 2003
Generic Name Trade Name Indication
Ceramide trihexosidase/alpha-galactosidase A agalsidase-beta (USAN) Treatment of Fabry's disease.
Buprenorphine hydrochloride   Treatment of opiate addiction in opiate users.
Buprenorphine in combination with naloxone Buprenorphine/naloxone Treatment of opiate addiction in opiate users.
Tositumomab and iodine I 131 tositumomab Anti-B1 Ab & I-131 Anti-B1 Ab Treatment of non-Hodgkin's B-cell lymphoma.
Nitazoxanide   Treatment of cryptosporidiosis.
Pegvisomant Treatment of acromegaly.
Icodextrin 7.5% with Electrolytes Peritoneal Dialysis Solution Treatment of those patients having end stage renal disease and requiring peritoneal dialysis treatment.
laronidase rHalpha-L-iduronidase Treatment of patients with mucopolysaccharidosis-I.
miglustat   Treatment of Gaucher disease.
Synthetic porcine secretin For use in the evaluation of exocrine pancreas function.
porfimer For the ablation of High-Grade Dysplasia in Barrett's Esophagus in patients who are not considered to be candidates for esophagectomy
nitazoxanide Treatment of intestinal giardiasis
ribavirin Treatment of chronic hepatitis C in pediatric patients
bortezomib To treat multiple myeloma


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