The Pink Sheet reports that the FDA wants to make a better case for increasing the use of accelerated approval, potentially as an alternative to implementing a new expedited approval mechanism.
In addition to saving the agency the inevitable headaches associated with implementing a new pathway, it also could quiet discussions about placing a new “Progressive” and/or “Exceptional” approval route into the agency toolbox. Draft legislation outlining both has been circulating among industry and congressional circles for several weeks.
The progressive approval concept would allow a drug to be marketed if evidence is submitted for a candidate that is likely to predict clinical benefit for a designated population and use. Exceptional approval would be allowed using an alternative showing if the data needed couldn’t be generated ethically or feasibly, according to the draft legislation.
On the January 15th edition of “BioCentury This Week” Center for Drug Evaluation and Research Director Janet Woodcock said that, while there are differing views on what progressive approval entails, the consensus is that no new approval pathway should lower the standards for safety and efficacy.
Woodcock also said accelerated approval is not realizing its full potential as a vehicle for qualifying drug candidates. She said it could be used more often and more consistently.
It would seem to be making the case that increased use of accelerated approval could be a viable alternative to instituting the two new approval schemes.
“[Accelerated approval has] been limited by its use, it’s not limited technically,” Woodcock said. “I think FDA could go a long way to [expanding the pathway’s use] with new guidance and new policies. I believe currently what’s codified in fast-track regulation is a little bit confusing about accelerated approval. So that could be clarified as well.”
Woodcock said more internal and external guidance on accelerated approval, such as provisions for the use of intermediate clinical endpoints, would help increase interest.
Woodcock said the concept of “staged approval” is feasible, but also potentially problematic.
“If you’re really going to have less evidence you've got to have some quid pro quo on the other side,” she said. “You can’t just toss it over the wall to the market and say go at it. And REMS doesn’t work in this situation because REMS is about a known safety problem.”
FDA also could speed up its approval of potentially breakthrough drugs by considering the best course of action early in the process, Woodcock said.
At the point a dramatic treatment effect is discovered, whether it is in a small population or any development phase, she suggested everyone “ought to all stop, take a deep breath and figure out how can we evaluate that drug as rapidly as possible.”
She said in some cases years could be cut off a drug’s development period if the larger treatment effect is verified.
“Even at that point, if it’s real, that’s a drug that offers something that no other drug’s offered before in that disease,” Woodcock said. “So you have to think how do we verify if that is real or not and then how do we evaluate the safety profile in the most efficient way possible?”
Did somebody say “molecular diagnostics?” Did somebody say “personalized medicine?”