A new paper in the latest issue of Health Affairs is worth a look. “Drugs Cleared Through The FDA’s Expedited Review Offer Greater Gains Than Drugs Approved By Conventional Process“:
We investigated whether drugs approved by the Food and Drug Administration (FDA) through expedited review have offered larger health gains, compared to drugs approved through conventional review processes. We identified published estimates of additional health gains (measured in quality-adjusted life-years, or QALYs) associated with drugs approved in the period 1999–2012 through expedited (seventy-six drugs) versus conventional (fifty-nine) review processes.
The FDA has a strict process for drug approval, requiring a number of phases of testing to establish safety and efficacy. It can, however, expedite this process when it thinks a drug may address an unmet clinical need. Of course, you want such a process to be fair and work. You want it to be focused on “better” and “more important” drugs. The 21st Century Cures Act moved even further. Some worry that loosening standards will lead to increased exposure to harm, especially if there isn’t proof that these drugs provide more benefits.
Enter this study:
We developed a data set of incremental QALY gains associated with drugs the FDA included in the expedited review programs and those the FDA did not include. We used drug-indication pairs as the unit of analysis. In other words, when incremental QALY data were available for a drug for multiple indications, each indication was included separately in the data set. We previously used this data set and a similar methodological approach to compare the incremental health gains associated with specialty and traditional drugs
They then identified all new molecular entities approved by the FDA from 1999-2012, separating them into those that did and did not use the expedited process.
They found that drugs that used at least one expedited review program (there are four) offered greater gains than those that did not (0.182 vs 0.003 QALYs). When they looked at the individual programs, there were differences in three of the four: priority review (0.175 vs 0.007 QALYs), accelerated approval (0.370 vs 0.031 QALYs), and fast track (0.254 vs 0.014 QALYs).
There are limitations to this, of course. Not all drugs have QALY estimates, and those without can’t be studies. To be honest, I was surprised so many did. Clearly there are some other biases here as well – positive QALY outcomes are more likely to be published and pushed, especially by industry. QALY’s also aren’t perfect, but I’m not going to fault them for using an accepted standard.
Bottom line: This is evidence that drugs in expedited programs seem to have larger QALY gains than those in the conventional process. Drugs in more programs have bigger gains. The FDA program appears to be working. That doesn’t mean we should stop looking and evaluating, but we should acknowledge the apparent success of the process.