Today our spotlight is on how well a gene therapy approved in early 2024 has performed with a lookback at several years preapproval and over a year post launch.
The specific therapy is not the primary focus of this report. Rather, it serves as an example to assess whether a therapy truly performs as advertised.
This ‘one-and-done’ therapy was introduced at a price of $3.5 million. The pre-approval trial results were exceptionally favorable, meeting the aspirations of any manufacturer. The outcomes were so impressive that the manufacturer confidently offered insurers a warranty providing “financial protections” in case the therapy failed to deliver as promised or even if its efficacy did not persist. It is important to note that the trials were relatively small for this orphan therapy, further challenging the confidence level of good outcomes in a larger population.
It is uncommon to encounter such positive feedback as the following:
· After the first year, no treatment-related adverse events were reported.
· Of the nine serious adverse events documented in four participants, none were related to the treatment.
· There was no discontinuation or dropout from the study resulting from adverse events or death.
· No participant resumed legacy therapy during the trial.”
A comprehensive review of the article could serve as a benchmark for any manufacturer aiming to achieve excellence in the launch of new drugs.
—————————————————————————————–
