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In September 2005, Autoimmunity Research, Inc. (doing business as Autoimmunity Research Foundation or ARF) applied to the Food and Drug Administration (FDA) requesting that two antibiotics and olmesartan medoxomil be designated as Orphan Drugs for the treatment of sarcoidosis.

An orphan drug is a pharmaceutical agent to be used to treat a rare medical condition (a disorder affecting fewer than 200,000 people in the United States). Since these drugs might have a limited application (for a rare disease) and thus be largely unprofitable, the Orphan Drug Act was passed in 1983 to encourage pharmaceutical companies to develop drugs for diseases that have a potentially small market.

Orphan drugs follow the same FDA regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmaco-dynamics, dosing, stability, safety and efficacy. However, some financial burdens are lessened in an effort to maintain development momentum.

Designation of a drug as an orphan provides these advantages, in preparation for a phase III clinical trial:

• federal tax credits (up to 50% of the drug development costs) and marketing incentives.
• the usual number of patients (1,000) for a phase III clinical trial is not required, as that number may not be available in orphan diseases.
• waiver of normal FDA fees, such as marketing application fee and annual product fees.
• 7-year monopoly (exclusive marketing rights) for the company obtaining the first marketing approval of a drug for a specific orphan disease.
• Potential research grants [1]

Designating a drug an orphan drug does not mean that the FDA has granted approval for marketing or promoting its use in treating a particular disease. It simply means some regulations regarding the clinical testing of the drug are relaxed in order to facilitate testing to prove safety and efficacy and ultimately, the chance to meet the requirements for such FDA approval.

On March 26, 2006, the FDA designated Minocycline an orphan drug for the treatment of sarcoidosis.

On August 9, 2006, the FDA designated Clindamycin an orphan drug for the treatment of sarcoidosis.

The FDA application to designate olmesartan medoxomil (Benicar®) an orphan drug has not been approved.

FDA designation of Minocycline and Clindamycin as orphan drugs for the treatment of sarcoidosis may reassure some doctors and patients who understand the safety of these drugs, have weighed their risks versus benefits and compared them with other treatment options. The efficacy and safety of these drugs for the treatment of sarcoidosis, however, like many drug interventions currently in use, has not been demonstrated in a valid clinical trial.

Autoimmunity Research met with the FDA in 2006 in an effort to persuade them that the equivalent of a phase III trial was being conducted online, but they decided that the anecdotal patient reports posted on www.marshallprotocol.com did not meet their requirements for a clinical study.

Conducting a clinical study usually requires hundreds of thousands of dollars. A phase III study in the USA also requires affiliation with an accredited research facility, an investigative review board, a principal investigator, inclusion and exclusion criteria, etc.

Following a successful, properly conducted study, a drug company sponsor would have to apply for and receive FDA marketing approval in order to advertise and promote a drug for treating any orphan disease, even if an orphan drug designation had been made.

Patients who are looking for valid clinical studies should visit http://www.clinicaltrials.gov/.

1. US Food and Drug Administration, Information for Industry, Developing Products for Rare Diseases and Conditions http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm