Customizing Prescription Drugs

by Food and Drug Administration (FDA)

Two thousand years ago, according to Christian belief, three wise men traveled to Bethlehem to worship the Christ child, bringing with them the gifts of gold, frankincense and myrrh. The aromatic resin myrrh, historically treasured for its medical and cosmetic usefulness, is still used in medicine today, mostly to treat inflammation of the mouth and pharynx.

Myrrh is not approved by the Food and Drug Administration to treat these or any other conditions. But the medical use of this unapproved drug may become legally acceptable under a new pharmacy compounding law that allows pharmacists and doctors to tailor-make some drugs for specific patients.

Considering the Alternatives

Health professionals are compounding when they prepare a specialized drug product to fill an individual patient's prescription when an approved drug can't fill the bill.

Compounding sometimes involves nothing more than crushing a pill into a powder with a mortar and pestle and then mixing it into a liquid, says Wayne Mitchell, an FDA regulatory counsel and member of the agency's internal pharmacy compounding steering committee. On the other hand, some types of compounding involve sophisticated scientific operations. Preparing sterile drug products, for example, can require complex steps to ensure a germ-free work environment.

The pharmacy compounding law, which is part of the FDA Modernization Act of 1997, defines the limits of legitimate compounding. By limiting the scope of the practice, the law aims to protect patients from the unnecessary use of compounded drugs, which carry intrinsic risks. Patients are often better served by taking commercially manufactured drugs that have been scientifically tested, approved by FDA, and manufactured under controlled conditions.

Compounded drugs can be "a great alternative when nothing is commercially available," says Randy Juhl, Ph.D., dean of the University of Pittsburgh's pharmacy school and chair of FDA's pharmacy compounding advisory committee, a group that includes non-government doctors, pharmacists, and consumer advocates. "But," Juhl says, "as a rule of thumb, if there is a commercially available drug, that's always better because of the quality controls that we as pharmacists can't provide for something we make up as a single-patient batch."

No Seal of Approval

The pharmacy compounding law carves out a limited exception to the requirement that prescription drugs be approved by FDA based on studies demonstrating their safety and effectiveness. Because pre-approval drug studies are performed on a very specific drug formula and dosage, even relatively small compounding changes can convert an approved drug (a product tested as a tablet, for example) into an unapproved one (such as a liquid form made by the pharmacist).

As unapproved drugs manufactured without FDA oversight, compounded medications involve an extra risk factor compared to approved ones. "Any time you make a change to a drug, there is the potential for reactions between ingredients or other problems," says Fred Richman, an FDA compounding expert. "You could end up with something that is less effective and that, at worst, could harm the patient."

Patients have been injured and have even died after taking pharmacy-prepared drug products. Examples include three infants who died after receiving an intravenous solution incorrectly prepared in a pharmacy, and a patient who became blind in one eye from pharmacy-prepared eyedrops that weren't sterile.

Taking the Risk

So why would a doctor choose to prescribe a compounded drug for a patient despite the inherent risks? A doctor may determine, in his or her professional judgment, that the compounded drug's benefits over any approved alternative justify the risk for a particular patient.

Drugs for certain conditions just aren't made by manufacturers. But even if a drug is mass-produced for a medical condition, patients might need a custom-made version for a variety of reasons.

Sometimes a patient can't use the standard version of a drug because of an allergy to one of its ingredients. If that ingredient is "inactive," meaning it has no therapeutic role, a compounder might make a similar drug minus that ingredient — say, without the dye used for coloring; or without lactose, a filler in many classes of drugs, from decongestants to antibiotics to chemotherapy medicines.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.