One of pharma's key strategies for growing a drug's sales is expanding into bigger groups of patients. Sometimes new meds hit the market for small-scale--even rare--diseases and then win approval for more common ones. Cancer drugs can begin as treatments for patients who've repeatedly failed on other drugs and then move into frontline use.
Thing is, the BMJ contends, those follow-up studies are often less stringent than they should be.
In a new study, the British Medical Journal looked at 295 supplemental applications approved by the FDA from 2005 to 2014, covering 164 different drugs. About half of the new approvals came in diseases different from the initial nod, and the rest covered new populations--kids versus adults, for instance--or different ways of using a drug, such as monotherapy rather than as an add-on to an older med.
The studies behind those approvals ranged widely in quality, the study found. Applications to expand a drug into pediatrics from adult use tended to be the lowest-quality overall, the BMJ study said. About half of them either included no additional clinical trials at all or relied on trials without a control arm.
Meanwhile, studies looking at orphan drugs in more common diseases were more likely to match up to the original trials--for better or worse. Getting orphan drugs to market justifies placebo-based trials and clinical endpoints rather than outcomes-oriented goals, the journal said, but for diseases affecting more people, with alternative treatments available, there should be a higher standard.
But often, those follow-up trials were as likely to compare a new drug to placebo as the original studies did, and to set clinical measures--aka surrogate markers--as endpoints, rather than actual outcomes.
All of this isn't to say that all studies for new indications fall short of the BMJ's ideal, but that there's a wide range of study quality, and at a time when the FDA is focusing on getting new treatments to patients as quickly as possible.
And that, in turn, means that tracking drugs after approval is more important than ever, the BMJ study concludes. Collecting info on side effects and treatment effectiveness after drugs hit the market, and following up with studies to confirm earlier trials, are necessary checks, the journal stated. Plus, the FDA should make it easier for doctors and patients to find out more about supplemental drug applications, so they can judge the quality of the evidence for new uses.
- read the BMJ article
Special Reports: Top 20 orphan drugs by 2018