How endpoints can expedite orphan drug approval

In Advocacy, Clinical Development, Regulation & Government by André Singer

In these past months, I have been developing the program for the World Orphan Drug Congress USA and found out that at least a couple initiatives has been helping to expedite the development and review of new orphan drugs. One is the well-known and recently implemented Breakthrough Therapy Designation, which is one of the provisions of the popular FDASIA (Food and Drug Administration Safety and Innovation Act).

According to biotechs like Alexion Pharmaceuticals the breakthrough designation has improved the assessment of value-based proposition through an optimized mensuration of the clinical benefits of a therapy. But does it mean that all biotechs are now able to easily determine an indication’s endpoint and through that get a quicker review process from the FDA? Probably not.

The biggest challenge here is to effectively determine the appropriate endpoints for a particular rare disease, and more than that, calculatedly speaking, how much money is it worth. Especially because a payer will look into a therapy and evaluate it from a clinical benefit standpoint.

The other potentially successful approach is related to the benefit-risk reassessment. In Japan, according to the Pharmaceutical and Medical Device Regulatory Science Society of Japan, a related reform is underway. The new system gives a conditional approval with a fixed term even when the efficacy of the product is only estimated and the balance of estimated benefit and risk is acceptable.  This should promote the development of innovative products.

At the 2014 edition of the abovementioned orphan drug conference, these initiatives will be presented and their implementation and development discussed for continuing optimization.