Orphan Drug Designation
A drug becomes an “orphan” when it receives orphan designation from the Office of Orphan Products Development, FDA. An orphan drug is a drug intended to treat diseases or conditions affecting fewer than 200,000 people per year in the United States. The costs of research and development of an orphan drug may exceed sales of the drug when it is marketed. Therefore, the Orphan Drug Act was passed as an incentive to develop and manufacture drugs that treat rare conditions. Orphan designation qualifies the sponsor to receive certain benefits, such as 7 years marketing exclusivity, from the U.S. Government in exchange for developing the drug. However, the drug must successfully complete the new drug approval process and be approved by the U.S.-FDA before these benefits can be realized. More than one sponsor may receive orphan drug designation of the same drug for the same rare disease or condition. When another sponsor seeks orphan drug designation for a subsequent drug for the same rare disease or condition, an explanation of why the proposed variation may be clinically superior to the first must be made.

Are orphan products approved faster than other drugs?
Historically, the approval time for orphan products as a group has been considerably shorter than the approval time for all drugs. This is due to the fact that many orphan products receive expedited review because they are for serious or life-threatening disease.

How much do orphan drugs cost?
The cost of orphan products is determined by the sponsor of the drug and is not controlled by the FDA. The costs of orphan products vary greatly. Generally, health insurance will pay the cost of orphan products that have been approved for marketing.

The above information is reprinted from the FDA Office of Orphan Products Development: http://www.fda.gov/orphan/index.htm.

Clinical Trials
Testing Drugs in People
by Ken Flieger

“Most of us understand that drugs intended to treat people have to be tested in people. These tests, called clinical trials, determine if a drug is safe and effective, at what doses it works best, and what side effects it causes—information that guides health professionals and, for nonprescription drugs, consumers in the proper use of medicines.

Personal Participation in Clinical Trials
Clinical trials are carried out at major medical research centers such as teaching hospitals, at specialized clinics ….., and even in doctors’ offices. Although they often involve hospitalized patients, many clinical trials are conducted on an outpatient basis, with participants more or less going about their normal activities.

Although investigational drug studies vary widely, some things should be expected by participants in virtually any clinical trial…… Tests to assess disease status might be more frequent. Participants are often required to keep detailed records of their symptoms and follow strict schedules.

Trials in Humans
Phase 1
Number of Patients: 20-100
Length: Several months
Purpose: Mainly safety
Percent of Drugs Successfully Tested*: 70 percent

Phase 2
Number of Patients: Up to several hundred
Length: Several months to 2 years
Purpose: Some short-term safety but mainly effectiveness
Percent of Drugs Successfully Tested*: 33 percent

Phase 3
Number of Patients: Several hundred to several thousand
Length: 1-4 years
Purpose: Safety, dosage, effectiveness
Percent of Drugs Successfully Tested*: 25-30 percent

* For example, of 100 drugs for which investigational new drug applications are submitted to FDA, about 70 will successfully complete phase 1 trials and go on to phase 2; about 33 of the original 100 will complete phase 2 and go to phase 3; and 25 to 30 of the original 100 will clear phase 3 (and, on average, about 20 of the original 100 will ultimately be approved for marketing).

The above information and additional information can be found at the FDA website: http://www.fda.gov/

Additional information on clinical trials for patients with rare disease or conditions is provided by the Office of Rare Diseases of the NIH and can be accessed at the following sites:

“Testing Drugs in People” originally appeared in the July-August 1994 FDA Consumer . The companion article “Skeptic’s Guide to Medical Breakthroughs” was taken from the November 1987 FDA Consumer Special Report on New Drug Development in the United States (January 1995). Ken Flieger is a writer in Washington D.C.