Magazine article Drug Topics

R.Ph.S and Orphan Drugs

Magazine article Drug Topics

R.Ph.S and Orphan Drugs

Article excerpt

Riutek, Eldepryl, DDAVP more and more orphan drugs are coming on the American market. Today, there are 122 orphan drugs approved by the Food & Drug Administration. This means, as the most readily accessible health professional, pharmacists are probably encountering more questions from patients on these products.

Many pharmacists are stymied when faced with a prescription for a drug that is unfamiliar, especially when they do not know the name of its manufacturer, it is not listed in the Physicians' Desk Reference, and it is not distributed through normal distribution channels. It can be difficult to trace the source of unusual drugs, and patients are often frustrated at how difficult it is to locate a pharmacy that might help them.

Many orphan drugs are not widely distributed through local pharmacies because of the scarcity of rare disease populations in any geographic area, although some of these products may have more widespread off-label uses. In addition, most community pharmacists do not like to lay out the large sums of money necessary to stock such medications. Betaseron, for example, can cost $10,000 to $12,000 per year retail. Even breakage of an orphan drug vial or expiration of shelf life becomes a major financial hazard to retail pharmacists.

It may be easier for hospital R.Ph.s to locate orphan drugs, since many such agents are biotech products, injectables, or IV formulations that often go through hospital distributors. However, a few orphan drugs, such as Eldepryl for Parkinson's disease or Betaseron for multiple sclerosis, are carried by local pharmacies.

For the rarest diseases (e.g., porphyria or severe combined immune deficiency-ADA), manufacturers may distribute the drug directly through a toll-free phone number. Other products may enter the normal distribution system, but the R.Ph.s may need advance notice to locate the drug stock.

As a nonprofit charitable organization, the National Organization for Rare Disorders (NORD) was developed to help patients with rare diseases. There are more than 5,000 of these orphan diseases, many of which are life-threatening, affecting an estimated 20 million Americans.

New role for NORD

In 1987, NORD began a new role-helping indigent transplant patients obtain the immunosuppressant drug cyclosporine (Sandimmune). Its maker, Sandoz Pharmaceuticals, asked NORD to develop an equitable method to distribute a limited supply of this drug at no cost to post-transplant patients who were uninsured, had exhausted their temporary Medicare Rx drug coverage, and could not afford to purchase this medication.

The NORD/Sandoz Medication Assistance Program grew to cover additional Sandoz products, including Clozaril, Sandoglobulin, Sandostatin, and Parlodel. …

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