FDA News and Views: Pediatric Clinical Trials, Genetic Health Risk Testing, Shared REMS, Type 2 Diabetes CE Course – Drug and Device Digest

FDA BRIEF: Week of November 6, 2017


FDA Awards Funding to Support Pediatric Clinical Trials Research

Some 60%-90%  drugs used in children and neonates not approved for pediatric use

  • Failure in clinical trials
  • Could not complete study recruitment
  • FDA to facilitate the plan, start up, conduct, close out of clinical investigations

FDA award to Institute for Advanced Clinical Trials for Children (IACT for Children) and Duke University

  • $1 million to each awardee for this year under Global Pediatric Clinical Trials Network Cooperative Agreement
  • Leverage basic science research to support extrapolation from adult data
  • Understand maturation of metabolic pathways for modeling and simulation to optimize dosing strategies
  • Develop innovative trial designs to optimize data from multiple sources
  • Leverage and standardize all sources of information
  • Develop multiple types of biomarkers
  • Develop clinically meaningful short- and long-term efficacy and safety endpoints


FDA statement on implementation of agency’s streamlined development and review pathway for consumer tests that evaluate genetic health risksCapture.JPG

Genetic health risk (GHR) testing helps understanding of  individual risk for developing diseases

  • Make more informed lifestyle choices
  • Direct-to-consumer (DTC) access to GHR tests available
  • Opportunities for  detection of additional genetic conditions and diseases

GHR Testing has its own risks

  • Incorrect or misleading information leading to health choices without medical advice
  • Adaptive regulatory framework for unique  challenges of new technology
  • Balance efficient pathway to customer access without sacrificing FDA oversight

Flexible approach for regulating novel medical advances such as GHR

  • Lessons learned from authorization of  first DTC GHR and carrier screening tests (23andMe)
  • GHR tests to be exempted from premarket review under certain conditions
  • Manufacturers would have to come to FDA for a one-time review; no furtjer review of new tests
  • Established special controls  in a separate de novo classification order

READ23andMe label

Capture.JPGFDA statement on new steps to improve FDA review of shared Risk Evaluation and Mitigation Strategies to improve generic drug access

Risk Evaluation and Mitigation Strategy (REMS) to ensure that the benefits of the drug outweigh the risks

  • Medication Guide or patient package insert
  • Communication plan
  • “Elements to Ensure Safe Use” (ETASU) e.g. prescriber training, patient counseling

Use of shared REMS to prevent use as a tool to block generic competition

  • Encourage use of shared system between innovator and generic companies
  • Reduce  likelihood for branded drug companies to use existing REMS to slow generic competition
  • Streamline submission and review process for shared system REMS
  • Use of a single Drug Master File (DMF) for shared system REMS submissions


Leveraging Health Literacy and Patient Preferences to Reduce Hypoglycemia Events in Patients with Type 2 Diabetes

CDERLearn: Health Literacy and Patient Preferences to Reduce Hypoglycemia Events in Patients with Type 2 Diabetes

  • Free one-hour Continuing Education (CE) course for healthcare providers (physicians, pharmacists, nurses, and others)
  • Describe the prevalence of hypoglycemic events among patients with type 2 diabetes mellitus and risk factors leading to an event
  • Introduce methods of assessing health literacy and numeracy of patients and caregivers
  • Review effective ways to incorporate patient preferences into care plans and differentiate A1C target values for individuals
  • List the action steps to reduce the likelihood of a hypoglycemic event for a high-risk patient


Image credit: FDA, 23andMe, IACT

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