How to Speed Up the Drug Approval Process

Unless you are an asthma sufferer, you probably didn’t see the Food and Drug Administration’s (FDA) recent announcement that beginning next year it will ban the sale of  Primatene Mist, the only over-the-counter asthma inhaler sold in the United States.

Do new tests show that the drug is harmful or that there are unanticipated side effects? No. In fact, Primatene Mist has proven to be one of the most effective treatments for temporary asthma symptoms. Instead, the drug is being pulled for environmental reasons -- yes, you read that right. The only over-the-counter asthma treatment will no longer be available because of an international agreement created to stop the substances that damage the environment. Because Primatene Mist uses chlorofluorocarbons (CFCs) as a propellant (spray), the medicine is considered an atmospheric pollutant.

Not only is this a heavy-handed response by the feds, but it could actually be dangerous. Rather than put the needs of asthma sufferers first, this FDA action will require patients to pay more for alternative medical treatment and limit the likelihood that temporary asthma symptoms can be treated quickly and effectively.

For patients without medical insurance, this decision could likely lead to black-market purchases or an increased willingness to use stale-dated products. This is far more dangerous than the effects of the inhalers on the environment.

However, such decisions demonstrate that the FDA has gone far afield from its responsibility to ensure that efficacious drugs are efficiently approved for sale in the U.S. In fact, it really is time to consider reforms to the FDA like the Heartland Institute’s Free To Choose Medicine (FTCM) initiative.

How does Free To Choose Medicine work? FTCM makes an end run around the costly and time-consuming FDA reform approval process -- a process that in some instances is approaching 10 years. While providing modest safety protection, the longer approval times give little comfort to a patient with a terminal condition. In fact, there are literally hundreds of cases where a drug that would ultimately prove safe and effective was denied to patients -- and tragically, the patients died before getting access to the drug.

Free To Choose Medicine relies on a two-tiered system. One tier keeps the status quo for any who still believe it is useful or effective. A second tier relies on an innovative waiver approval process whereby patients with either a terminal diagnosis or a chronic illness are allowed to access drugs that have already been submitted to the FDA -- well before their formal approval would occur in the first tier.

To participate in the second tier, pharmaceutical companies would have to make available all existing relevant information on the drug. Patients, in consultation with their doctors, would agree to accept that there are some risks associated with using tier 2 drugs.

Doctors would keep track of the patient’s medical progress with the results submitted to a nationwide Tradeoff Evaluation Database (TED) -- a database that would be open to medical practitioners nationwide.

As the treatment results from the tier 2 drugs develop, the FDA would be legally authorized to use the results to issue a preliminary approval or denial. This process could reduce by 4-6 years the amount of time that drug approval takes. Once a sufficient number of patients have successfully used the tier 2 drug after it has received preliminary approval, the FDA would be required to issue final approval for the drug.

This process would benefit both terminally and chronically ill patients in the short run and all patients in the end.

Companies would benefit as well. Today investors and drug developers have to assume that their ROI -- return on investment -- will be delayed by as much as a decade before going forward with a particular field. Since there is no guarantee that research will ultimately result in a new drug, delay only adds to the risk factor of investors and leads to higher pricing once drugs are finally approved. FTCM rewards drug makers whose drugs are effective by giving them the earliest possible approval times.

Instead of the FDA taking on responsibilities like protecting the environment -- which are far afield from its original purpose -- FTCM puts patients and doctors back in the driver’s seat when it comes to their medical needs and re-orients the FDA to its real mission. Patients will have access to more treatment options sooner and doctors will be able to better understand the results of various treatments. Moreover, since companies’ research costs are lower, everyone can benefit from the reduction in drug costs.

Notably, these results are not just theoretical. FTCM gives patients more access and hope than the current program that pushes a lucky few into the clinical trials where they still have a 50 percent chance of receiving a placebo instead of the real drug.

Ultimately, America can’t afford to have a health care system where it is acceptable to let people suffer or die needlessly due to delay in receipt of drugs.

FTCM would empower patients by giving them the ability to decide their future. Instead of expanding FDA duties away from drug approval, Congress should explore ways to restore drug approval as the FDA’s primary mission.