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Personalized medicine may drive down popularity of randomized controlled trials

November 18, 2014

Medical Team Works on Clinical Trial

For years, researchers conducting experiments to evaluate potential new drugs did so through randomized controlled trials, which have long been considered the gold standard in biopharmaceutical development. However, with the rise of personalized medicine, this experimental design is becoming less commonly used. Furthermore, drug companies interested in personalized medicine are noting that randomized controlled trials are not cost-effective because of patient enrollment protocols and the time needed for experiments.

Fortunately for personalized medicine development, regulatory authorities, such as the U.S. Food and Drug Administration, created various research tracks that allow for expedited evaluations of new medications, as reported by Reuters. Still, there are many questions that continue to surround the future of clinical trial design.

FDA has 4 alternative tracks
In the interest of providing access to effective treatment for diseases, the FDA encourages drug developers to take advantage of four alternative development tracks, which are intended for candidates that would act as first-line treatment or may be more effective than existing therapies:

  1. Fast track. This is for medications that can treat serious conditions and fulfill unmet needs.
  2. Breakthrough therapy. This expedites evaluations for medications that may be more effective than currently available drugs.
  3. Accelerated approval. Experiments on this track evaluate drugs for unmet needs by using surrogate endpoints.
  4. Priority review. The FDA takes action on priority review candidates within six months.

When it comes to personalized medicine, these alternative tracks to clinical trial design may be more appropriate than randomized controlled trials because this treatment strategy approaches patients based on their individual genetic profiles rather than through a population health strategy. This may be particularly pivotal within the context of cancer treatment, as more researchers discover that the genetic profiles of malignancies can be very heterogeneous, even among individuals who have the same type of cancer. Furthermore, some genetic variants make various forms of cancer more aggressive than others.

According to Reuters, different drug companies have faced ethical questions because of these dynamics. For example, if a medication candidate is designed to work only for patients who carry a certain genetic profile, it is unclear whether a randomized controlled trial is appropriate. Some companies may be quicker to jump on the opportunities afforded by more targeted clinical trials, and this may give them a competitive edge.

"We won't have to do those dinosaur trials," Alexander Eggermont, chief executive of Institut Gustave-Roussy, France's largest cancer center, told Reuters. "It will change the whole attitude in drug development."

International standards, pricing may pose obstacles
One dynamic that is complicating matters is the fact that alternative research tracks are generally quicker and cheaper than randomized controlled trials. In theory, these lower research and development costs should provide drug companies with an added incentive to adopt alternative tracks; but ironically, some are reluctant to jump on the bandwagon because higher research costs actually create the perception of an altruistic financial investment.

Furthermore, international collaborations may become more difficult in partnering nations that do not accept the alternative track standards of the FDA.

"In Europe, it's a different world because you can get a drug approved by the European regulatory agencies – but if the governments won't approve funding for it, people can't access it," George Demetri of the Dana Farber Cancer Institute told Reuters.