Reducing drug risk is achievable

The problem of serious physical risks associated with medications is not going away. Consider the following recent drug recalls: Vioxx (rofecoxib, Merck), Bextra (valdecoxib, Pfizer), and Tysabri (natalizumab, Biogen Idec).

Some people fault the Food & Drug Administration, claiming that it does not sufficiently monitor drugs for safety during clinical trials. Based on my own years of experience in the biopharmaceutical industry, I consider the FDA to be extremely rigorous in its standards for drug safety during the approval process. But no matter how carefully the FDA and drugmakers monitor for safety, the potential for a low incidence of serious side effects will always exist. There is simply no way to guarantee that pharmaceuticals will ever be entirely risk-free.

That said, there are actions legislators and scientists can take to minimize serious health risks associated with pharmaceutical agents. What's more, this can be done without unduly delaying the development of new medical treatments. These measures fall into two categories:

The newest generation of biomarkers, still in its incubation phase, will enable a new treatment paradigm known as "theranostics," which involves a tight coupling of diagnostics and therapies, such that a biological indicator determines in advance which patients will respond to a particular treatment. By targeting these patients, the biomarkers favorably alter drugs' risk-benefit profiles, allowing nonresponders to avoid side effects unbalanced by therapeutic gain. These biomarkers promise to eliminate much of the uncertainty involved in prescribing drugs-and result in significant healthcare savings.

One theranostic currently on the market is the biomarker for Herceptin (trastuzumab, Genentech), a drug for suppressing tumor growth in patients with metastatic breast cancer, which is used to test biopsy samples of tumors. A sample that tests positive indicates that the patient can be helped by the drug. Given that Herceptin is effective in only about one-quarter of patients, the biomarker for this drug is particularly useful.

The next phase of biomarker research, already under way, is to develop biological indicators identifying patients likely to experience adverse effects from a drug. Not only will such biomarkers reduce the incidence of side effects from medications, they will also be a means to help keep widely effective, potentially lifesaving drugs available to the public.

Theranostics is taking us closer to what, arguably, is our ultimate goal: true personalized medicine. It's difficult to estimate the true costs involved with theranostics. Third-party payers may resist coverage of theranostics to begin with, although in the long term, I think savings will be realized because the use of companion diagnostics will narrow the treatment population and produce higher rates of efficacy and fewer side effects.

Pharmaceuticals will never be 100% free of risk. But with greater FDA control of drugs already on the market and continued advances in biomarker research, we can greatly reduce the risk of serious side effects for the millions of people who take these medications.