The US Food and Drug Administration has a long wish list of changes it is considering for approving new drugs and making more prescription medications available over-the-counter. In a meeting with several reporters that I attended on Wednesday, FDA Commissioner Dr. Margaret Hamburg said the agency “is at the beginning of a process to determine how we can be more innovative” to improve access to certain drugs while at the same time curtailing the overuse of others.
Here are three initiatives on their agenda:
1. A new over-the-counter-plus approval: Try to envision many of the most commonly prescribed medications—statins, high blood pressure pills, asthma inhalers, migraine drugs, and the EpiPen for allergic reactions—going over-the-counter. You wouldn’t need a prescription from your doctor if you ran out, just a brief discussion with your local pharmacist to make sure you’re taking the drug appropriately. The FDA is considering instituting an “OTC-plus” designation to address common conditions that are undertreated, often because patients fail to get prescription renewals.
When I asked whether birth control pills and other hormonal contraceptives would be added to this list, Hamburg avoided answering directly and said the plan was still in its early stages. (She had recently supported making the Plan B emergency contraceptive available over-the-counter without age restrictions but was overruled by Health and Human Services Secretary Kathleen Sebelius.) The agency plans to get more input on its OTC-plus initiative at a public meeting later this month.
2. Curtailing off-label use of certain drugs: The overuse of antibiotics “has reached crisis proportions,” said Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, who also attended the meeting. One solution the agency is exploring is a new category of approved drugs with limited uses that would attempt to curtail the use of new antibiotics—capable of fending off infections that don’t respond to multiple drugs—by approving them only for very narrow indications.
Doctors would understand from the special designation that a particular antibiotic shouldn’t be given for, say, garden-variety pneumonia or strep throat but only to those suspected of having a drug-resistant infection. Hamburg said the agency is working with Congress to support a new piece of legislation—called the Generating Antibiotic Incentives Now (GAIN) Act—to provide incentives to drug companies to develop antibiotics needed to treat new and emerging resistant bugs.
If implemented, the narrow approval designation could also be used for other classes of drugs likely to be over-prescribed, such as those approved for obesity. “It’s very clear that the morbidity from untreated obesity,” Hamburg said, would make side effects from drugs to treat it more tolerable, whereas those same side effects would be unacceptable in a larger population of individuals with, say, 20 or 30 pounds to lose who don’t face the same health risks.
3. Getting more drugs on accelerated approval track: While the FDA has a fast-track system in place for getting new potentially life-saving HIV or cancer drugs on the market, the agency would like to see more drugs for other conditions such as Alzheimer’s disease, chronic lung disease, and rare orphan diseases get accelerated approval. For this to occur, said Woodcock, researchers need to develop new “surrogate end points” that can validly measure the effectiveness of drugs—such as, perhaps, increased strength in an isolated muscle or faster walking pace—instead of relying on “gold standard” end points such as lower death risk or slower disease progression.