Regulatory & Commercial Guidance on PDUFA VI: Interpretation, Strategy & Implementation

December 13, 2017 Suzanne Konigsberg

By: Partha Roy, Vice President, Technical, PAREXEL Consulting

As of October 1, 2017, PDUFA VI is our new reality. And readiness matters.

PAREXEL Regulatory and Commercial Experts with prior experience at the FDA and within the industry remain totally informed, maintain insightful context on potential impacts in the US and globally, and can assist you with any level of strategy, interpretation, implementation, proactive risk management and compliance mediation and mitigation.

PDUFA VI Synthesis

Changes/Impacts

Revamped fee structure for PUFA and BsUFA fees

  • Fee increase (~20%) for prescription drug applications
  • Sponsors can no longer deduct the annual BPD fees from the application fee upon filing

Meetings regarding new surrogate endpoints

  • Classified as Type C but BD must accompany request

 

Possible draft guidance on Real World Evidence (RWE)

  • Stay tuned—likely developed by end of FY2021 on how RWE can contribute to regulatory decision-making around safety and effectiveness evaluation

Patient Voice in Drug Development: Plan to develop guidances on the range of clinical outcome assessments

  • Including possible updates to the PRO guidance.
  • Information repository on publicly available tools

Benefit-Risk Assessment for new drugs and biologics: Plan to develop guidance by FY2020

 

  • Increased FDA interaction on benefit-risk discussion throughout product development
  • Get ready for increased scrutiny on product’s benefit-risk ratio during FDA AC meeting

Drug Development Tools Qualification Pathway for Biomarkers

  • Biomarker guidances are in development, including one scheduled for FY2020 that will address general evidentiary standards for biomarker qualification
  • Maintain public website to communicate a list of biomarker qualification submissions

Innovative Trial Designs and Model-Informed Drug Development

  • Guidances and public meetings to discuss novel trial designs (e.g. Bayesian, Adaptve)
  • Develop best practices around modeling and simulation approaches to support trial designs, disease progression and predict treatment response

Postmarketing Drug Safety Evaluation

  • Public meetings, MAPPS, Training – efforts to expand the Sentinel System and integration into FDA PV activities
  • Timely and effective communication of Postmarketing Safety Signals

This document is for informational purposes only.  Please seek professional advice for your specific circumstances.

 

 

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