The Breakthrough Therapy Designation

Implications and Strategic Business Considerations

The breakthrough therapy designation has generated substantial interest since its creation one year ago, as this new pathway represents the most comprehensive set of clinical development benefits available for a pipeline agent. However, the breakthrough therapy pathway may also lead to potential commercial downsides and regulatory risk. Most notably, the limited dataset from the breakthrough clinical program may be insufficient to garner favorable pricing and reimbursement, particularly in Europe. In addition, the abridged clinical trials may not be considered satisfactory for regulatory approval in ex-U.S. markets. Taken together with several other commercial considerations, it is critical for manufacturers to develop a comprehensive understanding of these advantages and disadvantages prior to pursuing the breakthrough therapy pathway.