I noticed that Velcade got a positive response from the European Medicines Evaluation Agency’s Committee on Proprietary Medicinal products here. This virtually guarantees European approval of this product. The history of the acquisition and subsequent development and approval of this product has investment implications that in my opinion will be felt for many years,
First the history of the acquisition briefly summarized here Proscript was in essence willing to give the product away. If I remember correctly the final price for the whole company was on the order of two million dollars. At that time I was engaged to advise a potential purchaser of Proscript and to review the entire package on a scientific and clinical and marketing basis. The client had a deep and talented scientific crew who were not at all smitten with the “Not Invented Here” bug and kept an open mind throughout the entire due diligence process. Nonetheless we all felt that a) the product was too non-specific-after all the target is not called Ubiquitin for no reason and b) the side effects predicted for humans on the basis of the pre-clinical models were daunting-mainly neuropathy. The client passed on the opportunity, which was then picked up by Leuko-Site and then folded into Millennium. Not one of my greater predictions.
The approval of the product from the time of initiation of the phase I trials to the granting of the NDA was also much shorter and less stringent than many observers expected about 4.5 years here. While many believe the FDA under the new commissioner is more responsive the broader implications on investment and political bases have been less extensively examined. For as long as I can remember there has been a battle between pragmatists and the purists regarding the optimum amount of data required for approval to market a drug. In most instances there is a tradeoff between safety and efficacy that is fairly obvious and therefore the more severe the disease the less efficacy required/unit of safety. So far so good-cancer patients are now clearly the biggest beneficiaries of the ascendancy of the pragmatists. However the basis of this newfound readiness in rooted in strictly utilitarian terms and does not seem to have broader libertarian implications.
While pragmatists are ascendant in the cancer arena-the purists are holding sway absolutely in the arena of gene therapy and probably in the cardiovascular arena as well. Mandatory drug testing in children for any drug, which might ever be used in one, is also not a sign of the demise of the purists. Moreover FDA advisory panels, (made up of a group of outside experts who are quite influential in the drug approval process) vary greatly in their respective outlooks. I would like to examine the committee members in each of the therapeutic areas and see if their composition and pre-existing pronouncements have some predictive value in their recommendations. I have felt for many years that the presence of a biostatistician or too many PhD’s on any advisory panel increased the odds of a negative outcome (purist win.)
While the FDA is in my opinion still extremely productive, countervailing pressures are also necessary. Patient advocacy groups are one, and a more libertarian political atmosphere as reflected in the appointment of a commissioner is another. However to infer a more sanguine outlook to the drug approval process in general on the basis of anti-cancer agents is probably premature.
These swings are probably more cyclical than our short-term memories allow for, and the further we are from the thalidomide tragedy the more likely we are to have forgotten its lessons.
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