The FDA Must Review Safety AND Efficacy of New Drugs
A recent opinion piece in The Wall Street Journal (7/25/2011) by Michele Boldrin and S. Joshua Swamidass called “A New Bargain for Drug Approvals” lobbies for a fundamental change in FDA oversight for new drug approvals. Basically, they are advocating for a system that puts safety first and allows for the proof of effectiveness later. Specifically, the authors are proposing that the FDA “should return to its earlier mission of ensuring safety and leaving proof of efficacy for post-approval studies and surveillance. It is ensuring the efficacy – not the safety – of drugs that is most expensive, time-consuming and difficult.”
What is their reason for proposing such a change? Among other things, they are concerned that the necessity of proving a drug’s efficacy, which is the primary driver for the approximately $1 billion price tag in developing a drug, is limiting the number of compounds that the biopharmaceutical industry can advance through FDA approval. The authors believe that if a company only had to invest in showing that a compound was safe, the entire process would be cheaper and then companies would be able to have the funds to “unleash the next wave of medical innovations.”
They go on to propose that: “In exchange for this simplification, companies would sell medications at a regulated price equal to the total economic cost until proven effective, after which the FDA would allow the medications to be sold at market prices.”
There are a variety of obstacles that such a proposal would face. First of all, there is a reason that the FDA’s remit was expanded decades ago. In evaluating a medicine, you need to be able to put the safety profile of a new drug in perspective with its benefit profile. A breakthrough cancer drug with some serious side effects might be justifiably approved because of its life-saving effects, whereas a compound that lowered LDL but which has this same side-effect profile might be deemed unapprovable based on the benefit-risk profile. You need to have full safety and efficacy data to judge a new drug fully.
The second major issue is economic. Drug prices are already regulated throughout the rest of the world and will likely be regulated to a certain extent in the US at some point in the future. In addition, payers (HMOs, etc.) want to know the safety and efficacy benefits of a new drug before they will allow it to be prescribed in their health networks. Thus, having efficacy data is needed to help get a new drug prescribed as well as to get a reasonable price. Without having full efficacy data, the drug will not be made broadly available to patients.
Finally, and maybe most importantly, why would a physician prescribe a new drug without having a full understanding of the beneficial effects of such a compound as well as knowing how the new drug stacks up in comparison to the treatments that she/he is already using? Just because it may be deemed safe isn’t a good enough reason to have a physician give it to patients. Even when you have these data now, physicians are reluctant to try a new medicine over something that they have already had good patient success with.
Call me old-fashioned, but I like having the FDA being the independent evaluator of both safety and efficacy. Such approval gives doctors and patients confidence in the health care system. Abandoning this practice would be a big mistake.