Drug Truths

A site devoted to teaching about drug discovery and development.

The FDA Must Review Safety AND Efficacy of New Drugs

with 4 comments

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.


Written by johnlamattina

July 28, 2011 at 3:08 am

4 Responses

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  1. I completely agree with you on the importance of having the risk-benefit profile to not only convince physicians to prescribe a drug, but also determine inclusion in formularies for reimbursement. The latter will become even more important as we become more conscious of drugs costs (especially if we eventually start focusing on cost by QALY). And all of this might be harder given a growing number of problems with approved drugs (e.g., Avastin for breast cancer, Mylotarg, Avandia…)

    Let’s assume this proposed system is adopted–do you think it will actually save on absolute costs? Or will it simply shift the cost burden away from the pharma to the healthcare system which pays for the drugs at “economic cost” until efficacy is proven. And if uptake of drugs is slower using this regulatory model, will proof of efficacy take longer than under the current system, extending the time until “market prices” can be charged and effectively reducing the profitability of a drug to pharma?

    Perhaps a better system is smarter trial design under the current system (which may require smarter pre-clinical R&D), and a better-funded FDA to provide more guidance and reduce regulatory backlog.

    Chris Franco

    July 28, 2011 at 1:53 pm

    • I really hope that this WON’T be adopted. I think that it would be a very poor business model for the biopharmaceutical industry and cause their sales to drop. But more significantly, I think it would be a disater for patients. The FDA’s review of both safety and efficacy remains to be the gold standard. – John


      July 28, 2011 at 6:44 pm

  2. Suggestion made in WSJ article basically means put the cart before the horse. I believe marginally better drugs have no value and should not be on the market. How does one determine a new drug is safe when they do not know its value to cure a disease. FDA’s mission is good and of value. Let us not compromise.


    August 1, 2011 at 4:45 pm

    • I agree. My sense is that marginally better drugs will be commercial failures nd at lest the big pharmas will shy away from them. – John


      August 1, 2011 at 5:42 pm

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