Summary
The phase 2 trial data presents results with a better outcome for superficial bladder cancer patients as those treated with standard procedures. A complete remission result after 2 years of 67% is dramatically better then adjuvant Chemoinstillation of immune therapy by BCG. The most important question for the near future will be. Is the study design innovative and can it prevent mistakes to exclude different risk distributions between the two or more groups compared in study. As a second important question the design in phase 3 with a comparison between placebo and EOquin is to week for FDA decisions because the comparison to established standard therapies in adjuvant treatment strategies are missing. The consequence will be that one or two more comparing arms in study should be integrated. This will be cost and time consuming but will prevent Spectrum Pharmaceuticals for the disaster of rejection of the collected data at the FDA after the period of data collection
Analysis
The described study in a phase 3 trial with a comparison between EOquin and placebo will produce data which will be discussed critically because the new substance has no direct comparison to standard procedures. As an ideal design a comparison to mitomycin C and BCG will bring clear results for an FDA approval, because the new substance can be compared with established medical treatments as standards actually. Of course such a design will be extreme expansive because of four comparing arms and the recruiting time will be long. But this design will produce the only acceptable data pool for FDA decisions. In most cases the decision to approve a new drug depends not only on the question of a positive effect but also on the question if the new substance becomes better than standard therapy, a real improvement?
In the past Spectrum pharmaceuticals has hade a substance which was not accepted for approval because of week data results after a phase 3 trial with Satraplatin for hormone refractory prostate cancer as a second line therapeutical substance.
The design of the phase 3 trial for EOquin will be the most important question for the near future to produce data which is eloquent enough to convince in all relevant aspects in the question of approval by the FDA.
A poorly planed phase 3 study can damage the best pharmaceutical pipeline and bladder cancer is a multifactorial malignancy with many interfering factors influencing the outcome of patients.
