What Is "Orphan Drug Status?"

Why is it so difficult to develop treatments for rare diseases?

For one thing, when few patients have a specific disease, even fewer are available to participate in the clinical trials that help researchers evaluate new treatments—and that means researchers have less data to guide their work. At the same time, pharmaceutical companies look down the road and estimate how many patients could be expected to buy the drug in the future. When that number is small, there’s less financial incentive for the companies to invest in the drug’s development.

That’s why the Food and Drug Administration (FDA) established the Orphan Drug Designation program, which grants “orphan status” to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders. “Rare” means a disease that affects fewer than 200,000 people in the U.S., including diseases like glioblastoma that has an incidence rate of two to three per 100,000 adults per year. In some cases, the designation is given to drugs intended to treat more than 200,000 people a year when future sales of the new drug would not be enough to recoup the costs of researching and developing it.

Recently the FDA awarded orphan drug status to a brain cancer vaccine in development at Roswell Park. “We’ve been working for some time on a drug called SurVaxM, which is a cancer vaccine that is designed to target the deadly brain cancer glioblastoma,” says Robert Fenstermaker, MD, Chair of the Department of Neurosurgery at Roswell Park Comprehensive Cancer Center and Chief Medical Officer for MimiVax, a Roswell Park spinoff company created to develop new immunotherapies. Although it is uncommon, glioblastoma accounts for 52% of primary brain tumors and represents a highly aggressive cancer that is difficult to treat.

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“We’ve been conducting clinical trials with patients using SurVaxM for five years now and are very pleased with the recognition by the FDA in awarding orphan status,” says Dr. Fenstermaker. SurVaxM is currently being evaluated in a phase II clinical trial at Roswell Park, the Cleveland Clinic, Dana-Farber Cancer Institute, Massachusetts General Hospital and Beth Israel Deaconess Medical Center. The vaccine stimulates a patient’s immune system to produce white blood cells that specifically attack and kill tumor cells that contain a protein known as survivin. MimiVax is also conducting another study treating patients with a form of cancer called multiple myeloma using the SurVaxM vaccine.

“One of the things orphan drug status might enable us to do is to get SurVaxM into more advanced randomized clinical trials using smaller numbers of patients, and—provided the drug continues to show promise—get it to market a lot faster,” says Dr. Fenstermaker.

The orphan drug status could possibly enable the researchers to get the vaccine to market following a smaller randomized clinical trial of roughly 200 to 300 patients, instead of the standard 600 or more.

“If the drug looks effective at that time, then the FDA could elect to approve its use more widely, on condition that the company complete a full phase III study,” says Dr. Fenstermaker.

According to Fenstermaker, orphan status can also help attract bigger companies to assist in a drug’s development. “Orphan status gives a period of exclusivity to the manufacturer, and this can make it more enticing for bigger pharmaceutical companies to come in and assist academic institutions like Roswell Park and smaller companies like MimiVax in the development of the drug, getting it to patients quicker.”