FDA Watch: User Fees Dodge a Bullet, a Cancer Drug Approval Breaks New Ground and the Supreme Court Serves Up a Biosims Blockbuster

The FDA budget pot continues to boil. In March, the Trump administration proposed a controversial provision to double the medical user fees that diagnostics, medical device and pharma companies pay to have the FDA review their products. "Industries that directly benefit from FDA’s medical product premarket approval … can and should pay more to support FDA’s continued capacity," the administration argued.

But the so-called "recalibration" plan didn’t get far. First and foremost, it completely ignored the fact that user fees were the subject of previous negotiations between the FDA and industry. And The FDA Reauthorization Act includes the negotiated rather than the recalibrated fees. And that’s one of the reasons the bill enjoys bipartisan support and is expected to pass easily in July.

Undeterred, the White House has reintroduced recalibration as part of its May 23 proposed budget for 2018. In addition to being highly problematic in its timing, the new plan is even more extreme than its predecessor. Instead of doubling, the administration would triple user fees to $439 billion. Needless to say, industry has criticized the plan and called on Congress to stick with the negotiated fees which, according to AdvaMed "is beneficial to patients, FDA and American innovation." But while recalibration is likely to pass, it something that industry will have to continue monitoring in the months ahead.

A Landmark Cancer Drug Approval
On May 23, the FDA did something it had never done before by approving a cancer drug administered on the basis of the genomic features of a tumor rather than where in the patient’s body the tumor started. The drug, Merck’s Keytruda (pembrolizumab) PD-1/PD-L1 inhibitor, was approved for treating adult and pediatric patients with nonoperable or metastatic solid tumors identified as having microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) genetic alterations, a type of tumor most frequently found in colorectal, endometrial and gastrointestinal cancers affecting roughly four percent of advanced cancer cases in the U.S.

Richard Pazdur, acting director of the Office of Hematology and Oncology Products within the FDA’s drug division, called the approval "an important first for the cancer community," a sentiment echoed by the medical community. "This is the first time in the history of oncology that a cancer medicine has been approved by the FDA using a pan-tumor predictive biomarker rather than a tumor-specific approach," said Luis A. Diaz, Jr., M.D., from Memorial Sloan Kettering Cancer Center. "This is a transformational milestone in our progress toward personalized immunotherapy," added Diaz.

Biosims Supreme Court Stunner
On June 12, makers of biosimilars, i.e., biologic medical products that are nearly identical copies of original products manufactured by a different company, scored a major legal victory in the U.S. Supreme Court issued a ruling making it easier for them to launch products.

Explanation: Drug makers must give the FDA marketing notice of new products and cannot launch until the FDA issues an approval, a process that takes six months. Amgen argued that biosims makers were subject to the six-month waiting period; Sandoz claimed that imposing such a waiting period would undermine a federal law called the Biologics Price Competition and Innovation Act (BPCIA) designed to encourage biosims production by providing a relatively short product licensing process. The Supreme Court agreed with Sandoz. Bottom line: Biosims must file an FDA marketing notice but can launch right away without waiting six months for FDA approval.

Here’s a look at key FDA diagnostics approvals in May and early June:


Manufacturer(s) Product(s)
Qiagen Premarket approval of Artus CMV QS-RGQ molecular diagnostic kit for use on QiaSymphony platform to manage solid organ transplant patients undergoing anti-cytomegalovirus
Becton Dickinson Extended approval of BD MAX real-time PCR enteric bacterial panel to include molecular test that detects infectious diarrhea caused by intestinal bacteria
Quidel Approval and CLIA waiver for Sofia Influenza A+B fluorescent immunoassay operating on the Sofia 2 analyzer
Quidel Approval of molecular assay for detecting C.diff infections from stool samples performed on Quidel’s Solana platform
Grifols Investigational New Drug approval of Procleix Babesia assay for blood screening at certain U.S. blood banks
Luminex Approval of Aries Bordetella Assay to detect respiratory tract infections attributable to B. pertussis and B. parapertussis from nasopharyngeal swab specimens
GenePOC Approval Revogene molecular diagnostics instrument
GenePOC Approval of GenePOC GBS LB molecular assay for detecting Group B streptococcus
BioMérieux Approval of FilmArray Respiratory Panel 2 (RP2) tests for viruses and bacteria responsible for respiratory tract infections
Roche Approval of VENTANA PD-L1 (SP263) Assay as complementary diagnostic for PD-L1 status of patients with locally advanced or metastatic urothelial carcinoma for purposes of deciding on treatment with AstraZeneca’s anti-PD-L1 immunotherapy IMFINZI
Roche Approval to use Rydapt, acute myeloid leukemia drug, in combination with chemotherapy and testing with Invivoscribe Technologies’ LeukoStrat CDx FLT3 Mutation Assay to identify patients who can use drug

New CE Marks
Here’s a summary of notable European CE certifications:


Manufacturer(s) Product(s)
Singulex Approval of Sgx Clarity cTnl assay for quantitative measurement of troponin
EntroGen Approval of BRCA Complete kit for exome sequencing of BRCA1 and BRCA2 genes for use on Illumina’s MiniSeq, MiSeq and NextSeq platforms
Multiplicom Extended approval of BRCA Mastr Plus Dx next-generation sequencing library preparation kit, analysis software and quality control to identify mutations in coding regions of BRCA1 and BRCA2 genes
Great Basin Approval of molecular diagnostic assay to detect Bordetella pertussis, the bacterium that causes whooping cough
Agilent Technologies Approval of Dako PD-L1 IHC 28-8 pharmDx test to identify patients with squamous cell carcinoma of the head and neck likely to benefit from using cancer drug Opdivo (Bristol-Myers Squibb); previously, test was approved only as a companion diagnostic for Opdivo

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