The recent death of a Drexel University student due to a rare meningitis B strain is an unquestionable tragedy; one that is made all the more tragic because two vaccines for this strain already exist (one of which has been approved by overseas regulators). Unfortunately, the FDA has been dragging its feet in getting the drugs approved domestically.
Meningitis is a frightening disease. 4,100 new cases are diagnosed annually in the U.S., with a mortality rate of over 10 percent. The serogroup B strain (MenB), however, is encountered infrequently in the U.S. – thus, a vaccine for this strain has not been made available in the U.S. until recently (and was restricted to use in Princeton and Santa Barbara), and universities have not included vaccination for MenB among their required vaccines for new students.
Without rehashing the media coverage, both Princeton University and U.C. Santa Barbara were threatened with a potential outbreak of this deadly strain. And in a smart move, the FDA permitted the use of Novartis NVS -0.41%s vaccine, Bexsero, in the two schools to vaccinate the students. Despite a determination that the benefits outweighed the costs for these two universities, the drug still isnt available to all who need it, as the FDA is still in negotiations with Novartis over the design of Phase III trials for the vaccine.
As my colleague, Paul Howard and I, have written previously in Health Affairs, it is mind-boggling that a drug (Bexsero) which has been approved in the EU, Canada, and Australia, is unavailable to Americans. The simple solution is reciprocity with foreign regulators that require similar standards of safety and efficacy, as we argued in our Health Affairs piece.
Recent moves at the FDA, however, are even more surprising.
Novartis isnt the only company working on a MenB vaccine. Pfizer PFE -0.03% has a drug candidate that has gone through early-stage trials which scored high marks for safety and immunogenicity, and is now engaged in Phase III trials in the U.S. and Europe. In what is likely a response to the latest outbreaks, the FDA granted Pfizers candidate (bivalent rLP2086) "Breakthrough Therapy" designation.
For the uninitiated, the Breakthrough designation is designed to help speed the approval of drugs for life threatening diseases (MenB is highly deadly – check) where early evidence indicates the potential for significant improvements over existing treatments based on surrogate (non-mortality based) endpoints (no approved vaccines for MenB – check).
While Pfizers drug appears to be highly effective (achieving seroprotection rates as high as 100 percent after three vaccinations), it seems odd that the FDA would ignore a drug it has already approved for use in Princeton and Santa Barbara. Indeed, Bexsero has been shown to achieve comparable seroprotection rates (after two vaccinations), and as the CDC points out, has been administered safely to over 8,000 individuals.
Indeed, the FDAs reticence is deeply puzzling, especially since (even if Pfizers vaccine is superior) theres a viable treatment available today that could help save lives while Pfizers product is working its way through the U.S. approval process.
If the use of Bexsero in foreign countries isnt enough for FDA, then the agency should be collecting data from the vaccinations of college students in Princeton and Santa Barbara – if the drug works and doesnt cause major adverse effects, the agency should at least license Novartiss vaccine for use by college students in what is a clear health emergency (this wouldnt be out of the ordinary either; as Manhattan Institute senior fellow, Peter Huber has previously noted: "[T]he FDA does not license drugs—it licenses specified drug-patient combinations.")
This would still be a second-best solution. Ideally, the agency should pursue reciprocity with foreign regulators – it could even be optional, but would at least require the FDA to identify the reasons why it is deciding against reciprocity. Moreover, if Pfizers treatment does make it through before Bexsero does, there is still no reason for only one drug to be licensed: first of all we should embrace price competition between the two, and second, down the road we may discover that one drug works better for some populations.
Simply put, there is no question that granting Breakthrough Designation to Pfizers drug candidate is a great move. There is undoubtedly an important unmet medical need. But the only reason that this unmet need exists is because the FDA is dragging its feet in approving a comparable alternative that is widely available. A drug that is safe for Europeans and college students in California and New Jersey is certainly safe for other Americans.
Original Source: http://www.forbes.com/sites/theapothecary/2014/03/28/fda-and-meningitis-b-picking-winners-and-losers/