US regulators have finally released draft guidlines for companies seeking to combine two or more drugs into one therapy, opening the door to the speedier development of drug cocktails that target serious diseases including cancer.
The much anticipated guidelines, released by the US Food and Drug Administration (FDA) yesterday, come as drugmakers are increasingly focusing on combination therapies. In 2009, two pharmaceutical giants, Merck and AstraZeneca, announced that they would team up to test two cancer drugs in combination: AstraZeneca’s AZD6244, which targets a protein called MEK, and Merck’s MK-2206, a compound that blocks a protein called AKT. The hope is that blocking both MEK and AKT can simultaneously shut down an escape route cancer cells use to become resistant to the drugs when they are used alone. (See ‘Drug giants unite to develop cancer therapy’.)
Since then, the drive to overcome drug resistance has turned other fierce competitors into collaborators, but the practice is still uncommon. In addition to the obvious business concerns, companies weren’t sure how the FDA would evaluate combination therapies. Trials that test drugs in combination often provide less information about the safety of each individual drug than if the drugs were tested singly. In the past, it was often necessary to do a complete evaluation of each drug individually first, dramatically slowing the development timeline and raising the cost of testing drug cocktails.
The FDA’s new guidelines now create a path that sidesteps this requirement, allowing accelerated development of cocktails to treat severe illnesses, such as late-stage cancers, when a combination therapy is needed. Genentech’s cancer chief Ira Mellman told Bloomberg that the new pathway could trim five years off of combination-therapy development.
In exchange, the FDA may demand additional preclinical studies (for example, in animal models) to justify the need for a combination therapy and an accelerated approval pathway. “We’re looking for the home run here,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (WSJ).
The guidelines are open for public comment until 14 February. A final version is expected in six months.