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FDA Recommends Early, Pre-Approval Trials in Pediatrics for Systemic Dermatitis Drugs

April 16, 2018

The FDA published a new draft guidance outlining how early Sponsors should incorporate pediatric patients, and relevant age groups, for systemic therapies for atopic dermatitis (AD).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 The new draft guidance recommends Sponsors initiate pediatric studies early in development, typically after obtaining initial evidence from early-phase adult studies.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 The draft guidance is available here: www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm603702.pdf.

CVS Planning First Clinical Trials to Develop Home Dialysis Machines
CVS Health is planning to develop in-home dialysis machines through the company’s first clinical trials and an eventual FDA submission. Following experience with at-home care through the company’s infusion provider, Coram, as well as its relationships with payers through Caremark, CVS hopes to reshape the dialysis treatment business model and kidney care space, said Alan Lotvin, executive vice president and head of CVS Specialty. CVS plans to approach the project in stages, first focusing on early disease identification and patient education, followed by the development of a comprehensive home program for hemodialysis and peritoneal dialysis. According to the company, the device’s design is intended to make home dialysis simple and safe, in order to facilitate longer, more frequent treatments.

FDA Adopts ICH Update to17-Year-Old Pediatric Clinical Trial Guideline
The FDA adopted an addendum updating the ICH’s E11 guideline on pediatric clinical trials, outlining ethical considerations, age classifications, pediatric drug formulations, practicalities in clinical trials and approaches to optimizing drug development. The new revision, issued by the ICH in 2016, recommends sponsors build early consensus with international agencies, and identify key pediatric populations and subgroups; methodologies and trial design elements; and any knowledge gaps in developmental physiology, disease pathology or data in adult populations or related compounds. According to the addendum, when obtaining child assent, relevant elements of informed consent should be provided appropriate to the child’s capability to understand. The absence of dissent or objection should not be interpreted as assent. In addition, it may be necessary for assent to be reassessed as the child matures over the course of a study — as well as obtain informed consent once the child reaches legal age. Sponsors should also consider the need for different formulations or doses across subgroups, as well as ethical considerations across international regions, the guidance said. In addition, computer simulations can fill knowledge gaps in efficacy, safety and pharmacokinetics, and help determine appropriate dosing strategies. When extrapolating data, the ICH recommends considering differences in geographic and developmental factors and warns that direct pediatric safety data may still be necessary for approval. The original E11 guideline was first published by the ICH in 2000. The addendum, E11 (R1), has since been adopted by Japan’s Ministry of Health, Labour and Welfare as well as the EMA, where it went into effect in February. The E11(R1) addendum is available here: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM530012.pdf.

NIH Publishes Detailed PanCancer Atlas to Help Inform Clinical Research
Researchers funded by the NIH published a detailed genomic analysis of molecular and clinical information from over 10,000 tumors representing 33 types of cancer, summing up the work of The Cancer Genome Atlas (TCGA). “This project is the culmination of more than a decade of groundbreaking work,” said NIH Director Francis Collins. “This analysis provides cancer researchers with unprecedented understanding of how, where and why tumors arise in humans, enabling better informed clinical trials and future treatments.” Known as the PanCancer Atlas, the work was made available as a collection of 27 papers across several Cell journals, complementing the more than 30 tumor-specific papers published by TCGA over the past 10 years. The project included cancer genome sequencing as well as gene and protein expression profiles, and associated them with clinical and imaging data, the NIH said. The NIH is planning a three-day symposium this September, focused on TCGA’s legacy and the future of large-scale cancer studies.

Henry Ford Health System to Launch Pancreatic Cancer Clinical Research Center
Henry Ford Health System plans to launch a pancreatic cancer research center following a $20 million gift from an anonymous donor, which will house investigator-initiated clinical trials. The gift will help establish a multi-institutional pancreatic consortium, led by Henry Ford, as well as endowed funds to support the hiring of an administrative director and clinical and research leaders. The center will focus on global collaborations to develop new methods for early detection, including validating biomarkers and translating them into screening tests, as well as using artificial intelligence to analyze clinical medical records and radiologic studies to identify patterns in medical records that may provide an earlier diagnosis. David Kwon, director of surgical oncology at Henry Ford Cancer Institute and director of the Multidisciplinary Pancreas Clinic at Henry Ford Hospital, will help to oversee the cancer center.

Billion-Dollar NIH Opioid Initiative to Accelerate Clinical Testing of Safer Pain Treatments
The NIH launched a new interagency effort to accelerate the clinical development of solutions to the national opioid crisis, such as non-addictive pain therapies and addiction treatments, following a near-doubling of federal funding from Congress to $1.1 billion in fiscal 2018. The HEAL Initiative, for Helping to End Addiction Long-term, plans to launch a longitudinal study to follow patients after surgery or the onset of acute pain to identify biomarkers for those more likely to transition to chronic pain. The NIH also plans to use innovative imaging and neurotechnologies developed through the BRAIN Initiative to identify new targets for treatment of chronic pain and biomarkers to predict treatment response — as well as define best practices for pain management using non-drug and integrated therapies for specific conditions. In addition, public-private partnerships to develop new pain medicines will share research data and match researchers with previously abandoned pharmaceutical compounds.