Approving Trumenba: Why Not Bexsero?

Last Wednesday, the FDA approved a vaccine for Meningitis B^1,2, the same strain of bacterial meningitis that Princeton students faced last year. Yet the newly-approved drug is not Novartis’ Bexsero, which thousands of students at Princeton and UCSB have received in the past year. Instead, the FDA approved Pfizer’s Trumenba. This forces us to wonder why the FDA decided to approve Trumenba first, even though Bexsero has now been used safely and effectively in the United States and is approved in Canada, Europe, and Australia³.

The most straightforward explanation for the earlier approval of Trumenba is that Pfizer filed for approval before Novartis⁴. Yet another possible factor for the approval of Trumenba over Bexsero could be that Bexsero is not considered to be cost-effective⁵. This idea was spread by the UK Joint Committee on Vaccination and Immunisation (JCVI), which revealed that Bexsero may not protect those vaccinated for long enough to be worth the current cost of the vaccination. Also, those who are vaccinated can still act as Meningitis B carriers. So while two doses of the vaccine can make an individual immune, this vaccination does not guarantee that the disease will not be transmitted to others, making it less effective than public health officials would hope. While Bexsero was effective at Princeton and UCSB, the JCVI still deemed that it is not cost-effective at market price, thus perhaps influencing the FDA choose Trumenba instead.

While the cost-effectiveness of Trumenba has not yet been carefully analyzed, the FDA may have decided to approve Trumenba over Bexsero in order to facilitate competition between the drugs, as Bexsero is already approved in other regions⁶. By creating this competition, the FDA may help lower the prices of both drugs since there is no longer a worldwide monopoly on the Meningitis B vaccine. This notion is supported by the fact that the FDA has recently taken other measures to lower drug costs. For example, with the initiation of the Abbreviated New Drug Application for generic drug manufacturers, the FDA has made it easier for generic manufacturers to enter the market, thus giving people access to affordable medications⁷.

Also, in relation to rare illnesses such as Meningitis B, the FDA has taken measures to make treatment for rare illnesses more affordable by offering tax benefits and other perks to manufacturers who make drugs to treat orphan diseases, which affect fewer than 200,000 people and thus are not seen as profitable by pharmaceutical companies⁸. By facilitating competition between Trumenba and Bexsero, the FDA could be supporting its existing mission to make medications for rare illnesses affordable, perhaps explaining the choice to approve Trumenba over Bexsero.

Furthermore, there have not yet been any official vaccine efficacy trials for Bexsero⁹. So while we have observed that Bexsero is effective on the college campus, Novartis has not collected any vaccine efficacy data, which is the numerical data that displays how effective Bexsero is in lowering the frequency of Meningitis B contraction in a population as compared to frequency of disease incidence in an unvaccinated population¹⁰. This lack of official evidence may have encouraged the FDA to approve the newer drug Trumenba instead, since Pfizer is currently conducting trials to assess the effectiveness of Trumenba¹¹. In addition, while Bexsero has been used in emergency situations, such as the situation at Princeton, it has not been used anywhere routinely⁵, meaning there is no long-term evidence of Bexsero’s safety and effectiveness to compensate for the lack of official efficacy trials, giving the FDA less incentive to approve Bexsero.

Therefore, while it may seem counterintuitive that the FDA decided to approve Trumenba over the tried-and-true alternative Bexsero, we can explain the decision by comparing the application timings for Novartis and Pfizer. Furthermore, the FDA may have been influenced both by past studies of the cost-effectiveness of Bexsero and by the fact that Bexsero has not been tested for vaccine efficacy. So while thousands of students have received Bexsero, the lack of official incentive to approve Bexsero helps explain why Trumenba is now the only Meningitis B vaccine approved in the US.

Endnotes:

1. Thomas, Katie. F.D.A. Approves Pfizer’s Trumenba, a Vaccine for a Rare Meningitis. Retrieved from http://www.nytimes.com/2014/10/30/business/fda-approves-vaccine-for-rare-meningitis.html?_r=0
2. First vaccine approved by FDA to prevent serogroup B Meningococcal disease. FDA. Retrieved from http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm420998.htm
3. Miller, Henry I. Meningitis Outbreaks Call for FDA Leadership. Don’t Hold your Breath. Retrieved from http://www.forbes.com/sites/henrymiller/2013/12/16/meningitis-outbreaks-call-for-fda-leadership-dont-hold-your-breath/
4. PPHR. Interview with CDC, performed by Genevieve Medina.
5. JCVI position statement on use of Bexsero® meningococcal B vaccine in the UK. Retrieved from https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/294245/JCVI_Statement_on_MenB.pdf
6. Vaccine Approved in the US Protecting Against Meningitis B. ACSH. Retrieved from http://acsh.org/2014/10/vaccine-approved-us-protecting-meningitis-b/
7. Abbreviated New Drug Application (ANDA): Generics. FDA. Retrieved from http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/AbbreviatedNewDrugApplicationANDAGenerics/
8. Developing Products for Rare Diseases and Conditions. FDA. Retrieved from http://www.fda.gov/forindustry/DevelopingProductsforrareDiseasesConditions/default.htm
9. Bexsero: Summary of Product Characteristics. European Medicines Agency. Retrieved from http://ec.europa.eu/health/documents/community-register/2013/20130114125155/anx_125155_en.pdf
10. McNeil, Shelly. Overview of Vaccine Efficacy and Vaccine Effectiveness. Retrieved from http://www.who.int/influenza_vaccines_plan/resources/Session4_VEfficacy_VEffectiveness.PDF
11. Zauzmer, Julie. First Vaccine fro Deadly Form of Meninigitis Approved in U.S. Retrieved from http://www.washingtonpost.com/local/education/first-vaccine-for-deadly-form-of-meningitis-approved-in-us/2014/10/29/9d6b693e-5fa0-11e4-91f7-5d89b5e8c251_story.html