Courts Should Defer to FDA Pediatric Policy: Professor
Commenting on Amgen’s federal court suit challenging FDA’s denial of six-months pediatric exclusivity for its Sensipar (cinacalcet), Yale Law School professor Jeanie Kim says in a JAMA Network online article that courts should defer to the agency’s current policy that links “fairly respond” to a study’s scientific value and allows FDA to effectuate the purpose of the pediatric exclusivity program. FDA denied the pediatric exclusivity after determining that the company’s studies had been conducted inadequately and had provided inconclusive safety data.
Kim says that if Amgen succeeds in its suit, it can delay generic competition for six months for a drug that generated $1.24 billion of revenue in 2016. More broadly, she writes, a decision favoring Amgen could diminish FDA’s ability to encourage clinically meaningful pediatric studies and thus undermine the purpose of Congress in offering patent extensions to reward pediatric research.
According to the article, FDA and Amgen agreed on a written request for pediatric studies encompassing four studies. The company completed three of the four, and on the fourth failed to study the minimum number of patients for the minimum duration. The company asked FDA to amend the request, but the agency refused, Kim says, because it had already approved several amendments to accommodate recruitment difficulties in the other studies and doubted that decreasing the size of the unacceptable study would accomplish the written request’s objective to evaluate the safety of cinacalcet for the relevant pediatric population.
In its suit, Kim says, Amgen does not contest FDA’ scientific judgment that the safety data are inconclusive. Rather, it says that the statutory standard for granting pediatric exclusivity is a “purely legal judgment” that involves “ministerial check-offs, not scientific decisions.” According to Amgen, then, FDA should base pediatric exclusivity determinations on whether studies complied literally with most of the terms in the written request.
Kim says that through its written requests, FDA specifies various trial parameters, which are not equally weighted but together can yield scientifically useful information. The agency then evaluates the submitted evidence to decide whether the studies meet the objectives of the written request. “This is what Congress intended,” she writes, “‘fairly’ is a qualitative, not quantitative, concept for study design. FDA admits that companies can obtain pediatric exclusivity even if studies do not meet every term, but FDA uses its scientific expertise to distinguish inadequately designed studies from well-designed studies capable of generating meaningful evidence.”
According to the article, Amgen’s interpretation creates disincentives for rigorous pediatric research because it would allow companies to simply select a number of criteria to fulfill, instead of conducting studies in a way that is likely to meet FDA’s standards for useful clinical evidence.