Expedite-Review-Programs-chart

Breakthrough Therapy designation, is it now the only pathway?

In Regulation & Government by André SingerLeave a Comment

Definitely not! Although this was the hype of last week’s FDA/CMS Summit in Washington D.C. when even FDA’s Commissioner Margaret Hamburg spent most of her time on stage talking about the recent and first 3 drugs to be approved under the breakthrough therapy designation program. Two of these, Gazyva from Genentech and Imbruvica from Pharmacyclics, are therapies for rare diseases – chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), respectively.

However, when at that conference I spoke with Richard Moscicki – CDER’s Deputy Director for Science Operations – he could not but emphasize that there’s a variety of different expedited review programs and that industry needs to have a better understanding of which is the most appropriated review program for each orphan drug candidate.

Breakthrough Therapy designation was implemented in July 2012 as one of FDA Safety and Innovation Act’s provisions. A therapy is granted breakthrough status when:

  • it intends alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and;
  • preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

The other expedited review programs are:

  • Accelerated Approval
  • Fast-track Designation
  • Priority Review

Here’s an interesting chart I found at the Friends of Cancer Research website that compares these programs above and the breakthrough designation:

Definitely not! Although this was the hype of last week’s FDA/CMS Summit in Washington D.C. when even FDA’s Commissioner Margaret Hamburg spent most of her time on stage talking about the recent and first 3 drugs to be approved under the breakthrough therapy designation program. Two of these, Gazyva from Genentech and Imbruvica from Pharmacyclics, are therapies for rare diseases – chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), respectively. However, when at that conference I spoke with Richard Moscicki – CDER's Deputy Director for Science Operations – he could not but emphasize that there’s a variety of different expedited review programs and that industry needs to have a better understanding of which is the most appropriated review program for each orphan drug candidate. Breakthrough Therapy designation was implemented in July 2012 as one of FDA Safety and Innovation Act’s provisions. A therapy is granted breakthrough status when: it intends alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and; preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. The other expedited review programs are: Accelerated Approval, Fast-track Designation and Priority Review.

If you would like to find out more about where industry is stumbling or benefiting from these expedited review programs, be sure to join us at the World Orphan Drug Congress USA 2014 where regulators and industry, among other rare diseases stakeholders, will be gathered to discuss this topic.

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