One year ago, the Healthyist wrote about how
Ebola could potentially bring much needed attention to the
FDA's priority review
voucher program (PVR) if it was added to WHO's list of
neglected tropical diseases (NTD). As strange as it seems, the PVR
law has come back into the limelight via another extremely serious
Unlike Ebola, Chagas (American Trypanosomiasis) isalready
classified by WHO as a NTD.
When a disease is categorized as a NTD, it generally affects a
large portion of people in third-world countries but only a small
percentage in more developed countries. Because of this,
first-world countries with the technology to develop treatments
have less incentive to invest millions on something from which they
will not profit. In steps in the
FDA's PVR program. My post from last year discusses how one of
the two FDA PVR vouchers can be sold for millions by the receiving
pharmaceutical company. This money is meant to recoup losses put
into research and development of the treatment, which can easily
reach more than 100 million. Although there has been some
misinformation about how the PVR program works, one thing is for
sure: it does not allow a company to skip clinical trials on the
road to FDA approval. Rather, the PVR program is actually just an
expedited process at the FDA review level. So, where does
benznidazole fit in with this?
Chagas was only recently
added to the NTD list of qualifying PVR diseases but already
main treatments: benznidazole and nifurtimox. One of these
treatments has recently started popping up unexpectedly in the
news. This new interest in benznidazole has brought to light
several underlying subplots that could have a positive effect on
the pharma industry (weird, I know, stay with me.) First, it renews
interest in a disease still affecting millions in poverty stricken
countries. Search queries using the term Chagas have risen by more
than 100 percent in December 2015 compared to prior months. Second,
more attention is being brought to the PVR program and is already
causing a federal systematic review of the important policy (which
is still pretty new, as FDA programs go). And finally, buried deep
within copy text, is the fact that this life-saving drug was
originally an act of good will from another pharma company.
Neither benznidazole nor nifurtimox is currently approved by the
FDA, but benznidazole is available for
free via the CDC in the United States for qualifying patients.
Benznidazole has not been approved by the FDA for sale in the
United States yet due to the fact that the number of U.S. patients
with Chagas who require treatment is relatively
low (a reason why Chagas was added to NTD list). The leader of
the CDC program that provides benznidazole reports that the CDC
only provided 60 to 70 courses of treatment of either benznidazole
or nifurtimox over the past year in the United States. But the
disease is considered to be an epidemic in South America and causes
approximately 11,000 deaths a year in Latin America.
That rate is steadily declining due to the fact benznidazole or
nifurtimox is widely available in a number of Latin American
countries--thanks to a donation.
Benznidazole was originally developed by the pharmaceutical company
Roche in the 1970s but was later donated by the company during the
beginning of the Chagas outbreak in several South American
donation transfer to the Brazilian government included the
rights, trade data, and knowledge and technical information to
manufacture its medicine.
After an agreement was made to subcontract manufacturing and
marketing to a public Brazilian agency, the Laboratorio
Farmaceutico do Estado de Pernambuco (LAFEPE), from 2003 to 2008,
Roche worked to transfer the necessary technology to develop the
drug to LAFEPE. In 2009, through a partnership with LAFEPE and
DNDi, the first pediatric formulation of benznidazole
became available in 21 countries in Central and South America.
The partnership allowed the drug to be sold at cost, with no profit
to the institutions involved, keeping the price of the two-month
treatment at $50 to $100 USD.
Roche helped to save the Brazilian government and Latin American
medical industry millions of dollars in research and development
for a disease plaguing its low income regions. Production of
generic forms of benznidazole, with the same active ingredient
founded by Roche,
has expanded to Argentina in order to meet an ever growing
demand, while still keeping pricing down.