As Pfizer's potential blockbuster arthritis drug Xeljanz (tofacitinib) prepares to come to market, a US Senator is demanding to know what US taxpayers can expect in terms of its affordability and protection of their interests, given that "taxpayer-funded research was fundamental" to the drug's development.
Xeljanz was approved by the US Food and Drug Administration (FDA) last November, and is nearing the market as the first oral medication for the treatment of rheumatoid arthritis (RA). In a letter to Francis Collins, director of the National Institutes of Health (NIH), Democratic Senator Ron Wyden writes that: "given that the research base provided by the NIH culminated in the approval of Xeljanz, citizens have the right to be concerned about the determination of its price and what return on investment they can expect."
"While it is correct that the expenses of drug discovery and preclinical and clinical development were fully undertaken by Pfizer, taxpayer-funded research was foundational to the development of Xeljanz," writes Sen Wyden, the senior US Senator for Oregon.
Moreover, with the drug’s price estimated at about $25,000 a year and some industry experts projecting its annual sales as high as $2.5 billion, "it is important to consider whether the public investment has assured accessibility and affordability," he goes on.In a recent press release, NIH reports that in 1993, shortly after NIH scientist John O'Shea and his team discovered the Janus kinase (JAK)3 protein on which Xeljanz is based and established its role in inflammation, Dr O'Shea learned that scientists at Pfizer were searching for drug targets to tackle autoimmunity and transplant rejection. Subsequent discussions led to an innovative public-private collaboration between NIH and Pfizer, through a cooperative R&D agreement (CRADA).
Such partnerships are important and can be instrumental to medical breakthroughs and innovation, writes Rep Wyden. But, he adds, there should be a mechanism that ensures the return on taxpayer investment is considered, pointing out that the NIH/Pfizer CRADA for Xeljanz occurred after NIH abandoned, in 1995, the policy requiring "a reasonable relationship between the pricing of a licensed product, the public investment in that product and the health and safety needs of the public."
The Senator asks Dr Collins to supply him with information about the CRADA and any other relevant agreements and licenses that helped to develop Xeljanz, "to gain an understanding of what the public can expect as a return on its research investment."He also asks for a list of all the major medicines and therapies that have come to market as result of research at NIH since 1995, and suggests the need for a better balance between encouraging profit, innovation, accessibility and affordability.
"NIH should convene an outside panel to re-examine the pricing of medicines and treatments developed with public funds," he tells Dr Collins.
After the New York Times ran a story on the matter this week, Pfizer issued a response in which it points out that, over the course of 20 years, it has invested more than $1 billion into the discovery, development and commercialisation of Xeljanz.This effort included screening hundreds of thousands of compounds in Pfizer's proprietary file to identify a lead molecule, preparing more than 1,000 variations of the firm's original lead molecule in order to come up with one investigational compound and, over the course of a decade, conducting one of the largest clinical trial programmes in RA at the time, encompassing more than 5,000 patients in over 550 locations in more than 40 countries.
"It was the culmination of this work undertaken by Pfizer that led to the drug now known as Xeljanz, the first new oral disease-modifying therapy in more than 10 years for adults with moderately to severely active RA," according to the company.
Pfizer also "believes it first became aware of the JAK pathways at a scientific conference in 1993," at which Dr O'Shea presented experimental findings about the JAK3 enzyme. "Dr O'Shea's findings were also published in 1994. The research was in the public domain, and thus widely accessible," it notes.Pfizer goes on: "to our knowledge, the CRADA did not yield any compounds or patentable intellectual property. Under the terms of the CRADA, the NIH was entitled to - and did - make publicly available the results of the research supported by Pfizer, including 10 publications. In this way the CRADA benefited the entire research community and the public at large."
On the issue of the drug's price, Pfizer says: “the prices of brand-name medicines are based on the value they will provide patients and healthcare providers, the scientific innovation they represent and the investment by Pfizer in comprehensive, multi-study, global clinical development programmes."