Trending News Today: Obama Cancer Moonshot Advances Scientific Goals

There has been recent growing concern over orphan drug spending, but new analysis in Health Affairs finds that these concerns are unjustified, reported Kaiser Health News. For the analysis, researchers at IMS Health and drug manufacturer Celgene analyzed US pharmaceutical spending from 2007 to 2013 on more than 300 drugs that had orphan approval under the Orphan Drug Act. The analysis was narrowed to examine spending on orphan drugs that were only used to treat rare diseases. The results of the review found that the orphan drug spending totaled $15 billion in 2007 and $30 billion in 2013. Furthermore, the study projects that orphan drug spending will remain fairly stable as a proportion of total drug spending, which contrasts other published reports estimating the drugs will account for 20% of worldwide spending on drugs by 2020, Kaiser reported. The rise in spending is largely caused by an increase in the number of orphan drugs approved by the FDA, which jumped from 16 in 2007 to 33 in 2013, according to the report. In fact, total orphan drug spending represented about 1% of total US health care spending. The authors noted the study’s focus was on aggregated spending, and did not include the effect of orphan drug prices on individual patients.

On Wednesday, a committee of top cancer researchers and patient advocates recommended 10 scientific goals designed to speed up progress against the disease, which represents a major step forward for the Obama administration’s “cancer moonshot,” according to The Washington Post. The 10 ideas were developed by a blue ribbon panel, and address a range of topics, including clinical trials, data mining, research, prevention, and patient engagement. Experts stated that the new recommendations are representative of areas that hold promise and are poised for relatively rapid progress. One of the most significant initiatives calls for the creation of a national network that allows patients across the US to have their tumors profiled. Another recommendation would create a clinical trial network dedicated to immunotherapy, while a third calls for an increase in testing for hereditary cancer syndromes, reported the Post. These recommendations have been sent to a task force headed by Vice President Joe Biden, which is currently working on a comprehensive report due next month regarding how the government can make further gains in the fight against cancer.

The FDA will review an immunotherapy cancer drug, pembrolizumab (Keytruda), as a first-line treatment for patients with non-small cell lung cancer (NSCLC). According to The Wall Street Journal, the FDA granted Keytruda breakthrough therapy designation and priority review status, with a target action date of December 24, 2016. Merck’s submission was based on results from a phase 3 study released in June, which showed that Keytruda monotherapy achieved superior progression-free survival and overall survival compared with standard chemotherapy. Last October, Keytruda was cleared for second-line treatments in NSCLC patients whose tumors contained a certain level of PD-L1, and whose disease continued to worsen after they received chemotherapy or other drugs.