Wednesday, September 29, 2010

A 21st Century FDA

We have been learning a lot about the new generation of cancer drugs, some of which have the potential to bring us tantalizingly close to the holy grail of "cure". Many are in the controlled trial stages of FDA approval, where the treatments are scrutinized for their long term effects before hitting the open market. I ran across this NYT article last week, which weighs the pros and cons of the  approval process against the very real need for dying patients to receive proven treatments.

The story goes that two cousins happened to be diagnosed with invasive melanoma around the same time that a novel therapy was entering its final Phase III trial. Both cousins qualified for the trial, but only one was given the new treatment (the other was placed in the 'control' group with a less effective treatment). The first cousin's condition improved; the second succumbed to the disease.

It is complicated scenarios like this one that exposes the limitations of our outdated FDA approval process. The cumbersome path puts doctors in the awkward position of telling a patient "Sorry, you can't have this life-saving treatment" and more importantly puts patients lives in jeopardy. Worse, the current 'fast-track' process would limit approval to only the sickest patients, which gives drug companies an incentive to wait for long-term results in order to gain approval for a wider market.

The debate is full of complexity, including factors like cost effectiveness, quality vs. length of life, and insurance coverage. Obviously, we want a rigorous safety/efficacy test for emerging treatments so we don't poison our patients (Vioxx, Avandia, etc), but 21st century research deserves an equally responsive FDA. The "priority review voucher" law passed in 2007 has the potential to speed up the process, which helped accelerate approval of Coartem, an anti-malaria drug. The idea may soon expand to some cancer research as well, where a bill was recently introduced to include rare pediatric cancers in a very capacity. The expansion of Health Information Technology (HIT) and health reform's Patient Centered Outcome Research Institute must play crucial roles in the evolution of obtaining such research data, as well, though I'll leave these concepts for a future post.

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