/Case Studies
Case Studies 2015-10-08T14:35:01+00:00

Case Studies

Tysabri® (natalizumab) – US approval in 2004

Tysabri® treats multiple sclerosis (MS), but was taken off the market two months after approval after several patients developed a rare, serious brain infection called PML. Although laboratory tests can indicate which patients are at higher risk for PML, it is not possible to determine who will become infected and there is a no good way to prevent this infection from occurring. Despite these safety challenges, MS patients asked the FDA to make the drug available with strict controls on its use. After an FDA panel unanimously recommended bringing Tysabri® back to the market, FDA took this step by requiring a restricted distribution program – the Tysabri® Outreach Unified Commitment to Health (the TOUCH™ Prescribing Program), and FDA’s strictest warning (a “boxed warning”) about PML risk.  Under the TOUCH™ program, every Tysabri® patient is closely monitored for PML.  Because TOUCH™ was in place when the REMS law passed, it was converted to a REMS. Other drugs that carry a risk of PML have also had a REMS put in place to address this risk.

Xyrem® (sodium oxybate) – US approval in 2002

Xyrem® treats cataplexy (loss of muscle control) and excessive daytime sleepiness in narcolepsy patients.  Before approving Xyrem®, FDA had to consider the drug’s serious risks, including seizure, coma, and potential to stop breathing. FDA also needed to determine the potential for Xyrem®, a controlled substance, to be diverted and misused to facilitate sexual assault. Due to these serious safety risks, strict distribution controls have been in place for Xyrem® since its approval, including limiting dispensing to a single central pharmacy; a centralized database of all patients, prescribers and prescriptions; and patient and prescriber monitoring.

Thalomid®  (thalidomide) – US approval in 1998

For decades, the FDA’s refusal in the 1960s to approve thalidomide as a treatment for morning sickness during pregnancy exemplified FDA’s role in making sure that drugs are safe. In other countries where thalidomide was given to pregnant women, the drug caused thousands of severe birth defects – babies born with missing and deformed limbs.

However, after thalidomide was removed from the market worldwide, studies showed it was effective in treating a condition of leprosy and certain types of cancer. Therefore, FDA approved thalidomide in 1998 with restrictions to ensure pregnant women never receive it. Specifically, women of childbearing age must have monthly pregnancy tests before receiving thalidomide, and prescriptions are limited to 28 day supply. In addition, both patients and prescribers are enrolled in a program to ensure they understand the safety risks of thalidomide. In 2008, these restrictions were converted to an approved risk evaluation and mitigation strategy (REMS).

Isotretinoin – US approval in 1982

Isotretinoin, sometimes known by the brand name Accutane, is used for severe acne.  Like thalidomide, isotretinoin can cause serious birth defects if a woman takes it while pregnant. After years of trying various other risk management strategies without success, in 2006 the FDA and manufacturers required safety restrictions before patients could get the drug. After the REMS program went into effect in 2008, these requirements were converted to a shared REMS system, which requires patients and prescribers to enroll in a database, and for female patients to show they are using birth control and are not pregnant before receiving their prescription each month.  While the REMS requires doctors and patients to take actions to prescribe and use isotretinoin, these extra steps are designed to prevent birth defects, allowing isotretinoin to stay on the market and available to patients.