The European Union (EU) General Data Protection Regulation (GDPR) is set to become enforceable in just over a month, which is causing new work for those in clinical research, and carrying with it a brutal fine for noncompliance.

The FDA released two final guidance documents related to next-generation sequencing (NGS) and one draft guidance on investigational in vitro diagnostics (IVDs) in oncology trials. All three documents offer test developer recommendations for a more efficient path to market.

The FDA finalized guidance on its special protocol assessment (SPA) program that offers sponsors an advanced declaration from the agency that their trial designs, clinical endpoints and statistical analyses are acceptable.

The FDA published a new draft guidance outlining how early Sponsors should incorporate pediatric patients, and relevant age groups, for systemic therapies for atopic dermatitis (AD). The agency previously recommended that Sponsors submit pre-approval data on the use of topical products, but did not recommend pediatric studies of systemic drugs until after adult approval. The new draft guidance recommends Sponsors initiate pediatric studies early in development, typically after obtaining initial evidence from early-phase adult studies. In addition, Sponsors should discuss the specifics of their pediatric programs with the FDA as early as possible, as pediatric study plans are required to be submitted within 60 days after an end-of-Phase II meeting. The FDA said it is important to study all relevant age groups, including children younger than two years of age. A sequential approach may not be needed, except for safety concerns, technical issues or the need for information from older subpopulations to inform further study designs. Juvenile animal toxicity studies should also be considered before enrolling human participants. If approved, Sponsors should provide as much information as possible when labeling in regard to pediatric use. The agency said it is also planning a separate guidance to address the technical aspects of drug development for pediatric patients with AD. The draft guidance is available here: www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm603702.pdf.

Merck announced that the pivotal Phase III KEYNOTE-042 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the first-line treatment of locally advanced or metastatic non-small cell lung cancer met its primary endpoint of overall survival. KEYNOTE-042 is an international, randomized, open-label Phase III study investigating KEYTRUDA monotherapy compared to standard-of-care platinum-based chemotherapy in patients with locally advanced or metastatic PD-L1 positive (TPS ≥1 percent) NSCLC. Patients had no EGFR or ALK genomic tumor aberrations and had not previously received systemic therapy for advanced disease. The primary endpoint is OS with TPS of ≥50 percent, ≥20 percent and ≥1 percent, which were assessed sequentially. The secondary endpoints are PFS and objective response rate (ORR). The study enrolled 1,274 patients randomized 1:1 to receive either KEYTRUDA (200 mg fixed dose every three weeks). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported monotherapy studies involving patients with advanced NSCLC.

Twice monthly, CWWeekly provides featured listings of clinical research job openings, upcoming industry conferences and educational programs from JobWatch, CenterWatch’s online recruitment website for both clinical research employers and professionals.

The clinical research enterprise is “at best the sickliest link” in the chain between patients, the healthcare system, and science that can and should bring cures and better therapies, according to Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (CDER).

The clinical research business is approaching an inflection point similar to the shifts seen over the past 25 to 30 years, only now the trends are toward consolidation in larger health systems and their affiliates that can take a more patient-centric approach, according to Ken Getz, director of sponsored research programs at the Tufts University Center for the Study of Drug Development.

The FDA published a new draft guidance on the scientific and ethical considerations for including pregnant women in clinical trials, telling sponsors that data are needed to inform safe and effective treatment during pregnancy, and that it is appropriate to enroll pregnant women in certain situations. The agency said it considers it ethically justifiable to include pregnant women in clinical trials when adequate nonclinical studies have been completed, and when the trial holds the prospect of direct benefit to the women and/or the fetus that cannot be obtained by any other means. In the postmarket setting, an established safety database in nonpregnant women should be available, and inclusion should be allowed when efficacy cannot be extrapolated or safety cannot be assessed through other study methods.