FILE – This April 28, 2010, file photo shows the GlaxoSmithKline offices in London. On July 20, 2018, the Food and Drug Administration approved GlaxoSmithKline’s Krintafel, a one-dose treatment to prevent relapses of malaria. AP PhotoKirsty Wigglesworth, File
The Food and Drug Administration’s FDA Priority Review Vouchers PRVs for neglected tropical diseases NTDs are aimed to incentivize pharmaceutical companies to develop drugs or vaccines for NTDs. The vouchers do this by providing a six-month expedited review of another drug within that company’s development pipeline if they receive FDA approval for such a drug or the voucher can be sold to another company. Though critics have claimed that the program doesn’t require the treatments to be novel or affordable, the program has been successful in ensuring a number of new medications for NTDs have been developed and launched in an accessible way.
Pharmaceutical companies have brought a number of already existing drugs to market and received a PRV. These are drugs that had been approved by other regulatory agencies – sometimes decades ago – and were in use and often on the World Health Organization’s Essential Drug List WHO-EDL.
High-profile examples include the artemetherlumefantrine Coartem combination targeting malaria in use as early as the 1990s, approved by FDA in 2009, benznidazole indicated for Chagas disease in use as early as the 1970s, approved by FDA in 2017, and miltefosine, a treatment for leishmaniasis. The latter was approved by the Indian regulatory agency in 2002. It was subsequently placed on the WHO-EDL. In 2014, miltefosine obtained orphan drug status in the U.S. A PRV was awarded. The PRV was then sold for $125 million without establishing a means to improve access to the drug at an affordable price.
In the case of Coartem, however, a comprehensive access plan was established. Together with public-private partners, including Medicines for Malaria Venture, Novartis established donation programs for Coartem dispersible, a sweet-tasting, cherry-flavored tablet that dissolves in water and is developed for children. Besides the donation programs, Novartis is supplying the drug at marginal cost to malaria-endemic countries.
There are also a growing number of instances of novel NTD drugs that have gained FDA approval, and for which certain guarantees of access have been promised or already established. For example, in December 2018, moxidectin became the first onchocerciasis river blindness treatment approved by the FDA in 20 years. The drug had not been registered elsewhere. Additionally, the sponsor, the not-for-profit Medicines Development for Global Health, has declared that revenue from the sale of the PRV will be supporting access to moxidectin.
According to Dr. John Reeder, director of TDR, the WHO hosted Special Program for Research and Training in Tropical Diseases, the “voucher fully meets the original spirit of the PRV program to promote research and development of affordable and accessible drugs for neglected tropical diseases. Without this program, Medicines Development for Global Health would not have been able to raise the funds to complete all of work for the new drug application.”
Another example is Krintafel tafenoquine, also named Kozenis, which was approved in 2018 for the prevention of relapse of Plasmodium vivax malaria in patients aged 16 years and older. Developed by GlaxoSmithKline and Medicines for Malaria Venture with funding from the Bill & Melinda Gates Foundation, Krintafel is the first new drug approved to eradicate Plasmodium vivax in 60 years. Given in a single dose it’s much easier to use than the previous standard of care, which often requires a two-week course. The manufacturer, GlaxoSmithKline, has promised that in malaria-endemic countries tafenoquine will be provided at an “affordable price” to maximize access to those who need it.
Yet another example is bedaquiline Sirturo. Following a fast-track, accelerated approval process, FDA gave the nod to Sirturo in 2012. Sirturo became the first novel treatment in 40 years for pulmonary multi-drug resistant tuberculosis MDR-TB. Access is available through a number of routes, including tiered pricing. The sponsor, Johnson & Johnson, has implemented an equitable pricing strategy that sets prices according to a country’s ability to pay. Initially, the price for a six-month course of bedaquiline is different for low-, middle-, and high-income countries $900, $3,000, and $30,000, respectively. Per July 2018, Johnson & Johnson announced a not-for-profit price of $400 for more than 130 eligible countries procuring through the Stop Tuberculosis Partnership’s Global Drug Facility.
In 2014 the sponsor also set up a donation program that is managed by the United States Agency for International Development USAID to increase access to the product in over 100 low- and middle-income countries. The program initially covered 30,000 treatment courses. USAID and Johnson & Johnson subsequently made an additional 75,000 courses available until March 2019. According to Johnson & Johnson, 118 countries currently have access, including all 30 countries with high MDR-TB burdens. The program incorporated physician and patient education on proper application of diagnostics, the use of bedaquiline, and activities to minimize risk to exposure to tuberculosis. Although a donation program continues in China, the USAID-managed program appears to have recently expired.
A growing number of novel NTD treatments with post-marketing access plans in place appear to be addressing critics’ concerns about the PRV program. Nonetheless, it’s imperative that sustained paths to access be coupled with NTD drug development.
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