Breakthrough drugs in the fast lane to approval

In Clinical Development, Regulation & Government by Tim Peplow

breakthrough drug therapy fda orphan (A_of_DooM

US drug developers have cheered on the introduction of a new regulatory pathway that could slash the time taken for a new drug to be approved, according to Reuters.

The FDA already has programs in place to expedite drug development for unmet medical needs – Fast Track, Accelerated Approval, and Priority Review – but there’s still room for another. The pathway in question is the FDA’s new “Breakthrough Therapy” designation, which the FDA says is “intended to expedite the development and review of drugs for serious or life-threatening conditions”.

A drug must demonstrate a substantial improvement over available therapy on at least one clinically significant endpoint, and as of July 26th 2013, 25 drugs have been granted the designation.

One drug to have received Breakthrough Therapy designation is Vertex Pharmaceutical’s orphan drug Kalydeco. Dr Jeffrey Leiden, Chief Executive of Vertex, said that the experience of working with the FDA for the cystic fibrosis drug was dramatically different from the the normal approval process. A key difference was the speed and ease of communication between the company and the FDA. “We pick up the phone and talk in real time,” Leiden said. “It makes the process immeasurably smoother.”

The process is moved on as quickly as possible, with communications taking minutes instead of weeks or months.

Dr Richard Pazdur, the director of the FDA’s Office of Oncology and Haematology Products, also highlighted the ease of communication, telling Forbes magazine that a breakthrough designation makes it more likely for a company to get an answer on the phone, and that the senior FDA management gets involved sooner.

Another Breakthrough Therapy-designated drug is J&J and Pharmacyclics’ orphan drug ibrutinib for chronic lymphocytic leukaemia, Waldernstrom’s macroglobulinaemia and mantle cell lymphoma. Dr Jay Siegel, head of global regulatory affairs at Johnson & Johnson, said that he expects that the designation will shave two years off the time it will take the FDA to review the drug.

What do you think? Should other regulators such as the EMA implement a similar breakthrough designation pathway?

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The World Orphan Drug Congress Europe brings together pioneers from the orphan drug community, including key opinion leaders from the payer, HTA, public health and patient advocacy bodies. Read more about the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.

Read more at Reuters >