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Regulatory Update, September 2016

Thursday, September 1, 2016

FDA Publishes Several Draft Guidance Documents

Chronic HCV Infection: Developing Direct-Acting Antiviral Drugs for Treatment

In the May 4, 2016 Federal Register, the FDA announced the draft guidance titled “Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral Drugs for Treatment.” Submit comments by July 5, 2016 to Docket No. FDA-2013-D-1170. The purpose of this draft guidance is to assist sponsors in all phases of development of direct-acting antiviral (DAA) drugs for the treatment of chronic hepatitis C. This draft revises the draft of the same name that was issued on October 23, 2013.

This draft guidance addresses nonclinical development, early phases of clinical development, and phase III protocol designs. Important issues addressed in this draft include: trial design options, non-inferiority margin for active-controlled phase III trials in the evaluation of interferon (IFN)-free regimens, and trial design options and safety evaluation for specific populations, including patients with decompensated cirrhosis, patients either pre- or post-liver transplant, patients with chronic kidney disease, and clinical virology considerations. Significant changes in this draft compared to the earlier version are:

  • Modification of several sections to focus on IFN-free DAA regimens.
  • Additional details on phase II and III trial design options for the evaluation of IFN-free regimens in treatment-naïve and treatment-experienced populations, including DAA-experienced populations. Specifically, the guidance now recommends that each marketing application contain at least one active-controlled comparative trial.
  • Additional clarification on DAA drug development in specific populations, including trial design options for human immunodeficiency virus/hepatitis C virus co-infected patients, pediatric patients, patients with advanced chronic kidney disease, patients with decompensated cirrhosis, patients either pre- or post-liver transplant, and patients who failed to respond to a prior DAA-based regimen.

Chronic Obstructive Pulmonary Disease: Developing Drugs for Treatment

In the May 20, 2016 Federal Register, the FDA announced the draft guidance titled “Chronic Obstructive Pulmonary Disease: Developing Drugs for Treatment.” Submit comments by July 19, 2016 to Docket No. FDA-2007-D-0133. This guidance is intended to assist sponsors in designing a clinical development program for new drug products for the treatment of chronic obstructive pulmonary disease (COPD). This guidance revises the draft guidance of the same name, issued November 9, 2007, by adding information regarding the St. George’s Respiratory Questionnaire (SGRQ).

The emphasis of this guidance is on the assessment of efficacy of a new molecular entity (NME) in phase III clinical studies of COPD. Development of NMEs for COPD poses challenges and opportunities. Not all drugs developed for COPD will fit into the types described, and the efficacy endpoints discussed in this guidance may not fit the need for all drugs. The FDA encourages sponsors to develop clinical programs that fit their particular needs and to discuss their planned approach with the Center for Drug Evaluation and Research’s Division of Pulmonary, Allergy, and Rheumatology Products.

The FDA acknowledges the importance of assessing patient perspectives in clinical trials and therefore is interested in eliciting comment on the SGRQ, included in Appendix A of the draft. Also, this guidance outlines the FDA’s thinking based on information that was available in 2007 on the development of various types of drugs for COPD. The FDA acknowledges that the landscape of clinical trials has evolved since 2007 and therefore is encouraging public comment on the body of the guidance in addition to public comment on the SGRQ information added in Appendix A.

Use of Electronic Health Record Data in Clinical Investigations

In the May 17, 2016 Federal Register, the FDA announced the draft guidance titled “Use of Electronic Health Record Data in Clinical Investigations.” Submit comments by July 18, 2016 to Docket No. FDA-2016-D-1224. This guidance is intended to assist sponsors, clinical investigators, contract research organizations, institutional review boards (IRBs), and other interested parties on the use of electronic health record (EHR) data in FDA-regulated clinical investigations. In particular, the draft provides recommendations on the following: (1) deciding whether and how to use EHRs as a source of data in clinical investigations; (2) using EHRs that are interoperable with electronic systems supporting clinical investigations; (3) ensuring quality and integrity of EHR data that are collected and used as electronic source data in clinical investigations; and (4) ensuring that use of EHR data collected and used as electronic source data in clinical investigations meets the FDA’s inspection, recordkeeping, and record retention requirements. In an effort to modernize and streamline clinical investigations, the goals of the draft guidance are to facilitate use of EHR data in clinical investigations and to promote the interoperability of EHRs and electronic systems supporting the clinical investigation. 

The Regulatory Update is excerpted from Research Practitioner, Volume 17, Number 04, July-August 2016.

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