Efficacy Requirement of the FDCA–a safeguard or a headache?

By: Arzoo Zaheer

Date:May-16-08

THE PRICE WE PAY:
The Efficacy Requirement for New Drugs Under the Food, Drug and Cosmetic Act
Corey B. Rubenstein

I.INTRODUCTION
The newly sworn Republican-controlled Congress has, as one of its primary objectives, the downsizing of government. Speaker Newt Gingrich has specically targeted the federal Food and Drug Administration (FDA) for the conservative wrath.1 The political climate that led to the change in power in Congress is partly based on the nation’s anti-regulatory demeanor { the belief, whether correct or mistaken, that the regulators have run riot, that there is too much power in the hands of a few appointed bureaucrats. The FDA draws specific attention because of the palpable eects of its rulings in the lives of all citizens. Specically, the requirements for the pre-market approval of new drugs have been criticized for being too cumbersome.2 This paper will attempt to draw a compromise between the observed problem of overregulation in that area and the still important policies underlying the federal Food, Drug, and Cosmetic Act (FDCA). It will call for the elimination of the efficacy requirements for new drug applications (NDA) as a way to reduce the costs of developing new drugs, while maintaining the safely requirement in order to ensure the public health.

The rest of the article is available here.

This article proved extremely fun to read since the author made his/her case really strongly. It’s almost like reading a philosophy paper where the authors provide their arguments, then that of the opposite side and then refute the arguments of the opposite side. J Here, I will provide a brief summary and my opinion about this article.

FDCA was enacted in 1938 due to the Elixir Sulfanilarnide disaster. The purpose of this Act was to ensure that the drugs that are marketed for public use are safe and won`t harm the consumers. Later on, the efficacy requirement was added to the drug amendments even though efficacy was never a real concern. The author argues that the allowing the efficacy requirements hamper the availability of the safe drugs to the general public.

Corey provides three reasons why he/she thinks that the efficacy requirement for the NDA must be waived. First of all, the entire NDA process is too costly. Pharma companies have limited resources and money to invest in the experimental drugs. Corey argues that decreasing the cost of the process would assist in making beneficial drugs. Secondly, FDA is too cautious and it enters into NDA review process with a bias against approval. Thirdly, the efficacy requirement interferes with the rights of the patients since it delays drugs availability and also prevents patients to voluntary take (only) safe drugs after consulting with their doctor.

Proponents of the opposite argument debate that the efficacy requirement is required for an insurance funded medical system since a patient will not internalize the cost of an inefficient drug. However, according to Corey, this argument ignores the fact that a doctor will not prescribe an ineffective drug to the patients unless the circumstances are exceptional.

In my opinion, both sides have a point. FDA must review and amend its own policies about NDA approval. Secondly, using due process and scientific evidence, regulatory authorities should figure out exactly which drugs (for particular illnesses) must have an efficacy requirement and for which drugs it’s ok to waive that requirement. For example, its best to have efficacy requirement for drugs for AIDS but it’s alright to waive it for cosmetics. Thirdly, the insurance companies should amend their policies to include a clause that would allow internalization of the cost of the drugs that have been approved by the regulatory agencies to be marketed without efficacy studies. But it must be kept in mind that the consumers, doctors – the entire pharmaceutical market must be properly educated as to exactly why its alright to take certain drugs even though efficacy studies haven’t been conducted on them. Lastly, an effort should be made to advance the research process so it’s possible to carry out safety and efficacy testing simultaneously. This would reduce the NDA approval time dramatically while settling this debate over whether the efficacy requirement should be waived or not.