A. Food and Drug Administration. Interestingly, in 2013 the Chinese regulatory body responsible for food and drugs (similar to US FDA) was named the China Food and Drug Administration (CFDA), but was renamed and restricted into the National Medical Products Administration (NMPA) in 2018.

B. False. For example, a small but consistent difference between 2 large populations could have a statistically significant value without a clinically meaningful effect.

B. Patients with melanoma. This description avoids dehumanizing patients.

A. The cost of filing an NDA/BLA in the US, known as the PDUFA fee. If FDA reviews your application and the product is not approved, you don’t get a refund. Products granted Orphan Drug Designation, a designation given to drugs intended for diseases/conditions affecting <200,000 people in the US, are exempt from the PDUFA fee. ITIL-168 was granted ODD for the treatment of melanoma stages IIB through IV on April 26, 2021. Note that in 1993, the first year a PDUFA fee was charged, the cost of filing was only $100k which is the equivalent of ~$206k in today's dollars.

A. Erin Tricker

A. The FDA Center for Biologics Evaluation Research. CBER regulates (certain) biological products for human use under applicable federal laws, including cell therapies. Some biologics are regulated by CDER, FDA’s Center for Drug Evaluation Research, such as monoclonal antibodies and non-vaccine therapeutic immunotherapies.

B. Poor explanation of rationale was most frequently chosen survey option (7 of 22 regulatory document reviewers). In follow-up interviews, reviewers explained that interpreting the authors intent can be time consuming and can be interpreted as a lack of transparency, flagging the application for extra investigation. Go to https://amwajournal.org/index.php/amwa/article/view/83/70 for more.

A. Clonal heterogeneity. By avoiding additional cellular selection methods, we may maintain a diverse set of T-cell clones (that hopefully have cytolytic [killer] activity)

D. All of the above. All of these have a 30-day deadline.

B. Sophia Siu

B. Oncologic Drugs Advisory Committee. ODAC is a committee of experts called on by FDA when they need independent expert advice on data concerning safety and effectiveness for drugs for cancers. FDA has advisory committees (ACs) for a range of functions. The Vaccines and Related Biological Products Advisory Committee has been particularly busy the last couple of years with COVID. AC meetings are generally webcast and open to the public to listen in. FDA does not need to follow the advice of the AC. In 2020, the AC reviewing aducanumab, Biogen’s treatment for Alzheimer’s disease, voted 10:1 that the research didn’t show the drug’s effectiveness. Following FDA’s approval of aducanumab, 3 of the AC members resigned with 1 stating, “This might be the worst approval decision that the FDA. has made that I can remember.”

C. The 95% confidence interval. It provides information on the precision of a population estimate or the precision of a difference between 2 estimates (eg, between the medians of 2 groups). It can help us determine whether the difference is noteworthy based on the size of the interval and whether 0 is included in the interval.

C. T-cell receptor. Hyphenate words that, together, modify the subsequent noun. Hyphens are not needed after adverbs that end in -ly (such as correctly-hyphenated) and we should not add hyphens in the middle of a word (such as ad-vantage).

B. PRV. FDA can award a PRV to a sponsor that gets approved for a drug to treat rare pediatric diseases, certain tropical diseases, or that is used as a material threat countermeasure to biological, chemical, radiological, or nuclear agents (eg, anthrax, ebola). PRVs are not tied to the original sponsor/product and can be bought and sold, to be redeemed by the owner during a BLA/NDA submission and results in an FDA Priority Review (6 months) instead of the Standard Review (10 months).
Gilead redeemed a PRV purchased for $80M for Priority Review of their NDA for filgotinib for the treatment of moderate-to-severe rheumatoid arthritis in 2019. Unfortunately for Gilead, filgotinib was ultimately not approved by FDA. In a win for Gilead, they were awarded a PRV upon approval of their COVID-19 drug, remdesivir in 2020.

B. Eric Backes

B. The Medicines and Healthcare products Regulatory Agency? Yes! The United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) regulates medicines, medical devices, and blood components for transfusion in the UK.

A. Subject incidence rate, which reports the number of subjects who had at least 1 adverse event within a given category. If the adverse event rate was used, multiple events reported for 1 subject would all be counted.

B. Neoplasm and tumor. The others are not completely interchangeable. Both because and since can mean “for the reason that”, but since can also mean “from the time of”.
Relationship implies cause and effect, whereas an association occurs between 2 or more variables in which the independent variable does not necessarily cause the other dependent variable(s).

B. The time a sponsor has to report to FDA a suspected AR that is both serious and unexpected in a clinical study. Unexpected fatal or life-threatening suspected AR must be reported within 7 days of initial receipt of information, and the FDA has either 14 or 21 days to respond to the request for a meeting (depending on the meeting type).

C. Diana Ibrahim

C. Cellular, Tissue, and Gene Therapies Advisory Committee, which reviews and evaluates available data relating to the safety, effectiveness, and appropriate use of human cells and tissues, gene transfer therapies, and xenotransplantation products. You can be reviewed by more then 1 FDA AC (eg, Amgen’s talimogen laherparevec for metastatic melanoma was reviewed by 2, both CTGTAC and ODAC).

A. The frequency of lung cancer in the asbestos-exposed group is 5 TIMES that in the unexposed group (10% / 2% = 5). If we were to describe a difference as “times higher”, we should look at the difference between groups (10%-2% = 8%, and 8% / 2% = 4). In this example, the frequency of lung cancer in the asbestos-exposed group was 4 TIMES HIGHER than that of the unexposed group.

A. is correct. B. has 2 issues: we should not add commas when fewer than 3 modifiers are used and when the modifier and the noun are read as one entity (eg, double-blind randomized clinical trial), and "post" means "after" only when used as a prefix. In C, the reader may wonder whether a woman is more likely to have a degree or a man by the age of 25 based on the placement of "than".

B. The fine per day for delays in reporting clinical study results to ClinicalTrials.gov. Sponsors should report clinical study results <1 year after primary completion of the study. In 2021, Acceleron Pharma became the first company to face potential fines of $10k/day from the FDA for failing to report results on time. There is no FDA fee for an IND review. There is no set fine per day for delays in reporting treatment-related fatal adverse reaction safety events, but the FDA can take enforcement action and criminal fines for violations of the Food Drug and Cosmetic Act can go up to $500k.