After incorporating input we received from stakeholders to a draft version, we are finalizing our guidance to industry today
in order to provide a more detailed explanation of these programs and
help drug innovators determine whether their products are likely
These expedited programs include:
Fast track designation: Providing for more frequent
meetings and communications with FDA to discuss the drug’s development
plan and ensure collection of appropriate data needed to support drug
approval, including such things as the design of the proposed clinical
trials and use of biomarkers.
Accelerated Approval: Basing approval not on a clinical endpoint but on
an agreed upon surrogate marker, that is a measure such as blood test
or urine marker, that is believed to be indicative of a disease state
and treatment effect, but not demonstrative of a direct health gain to
Priority review: Acting on drug applications within 6 months instead of 10 months for standard review, and;
Breakthrough Therapy Designation: Providing all of
the benefits of Fast Track designation plus intensive guidance on an
efficient drug development program, beginning as early as Phase 1, and
the commitment from FDA’s review staff, including senior managers, to
work closely together throughout the drug development and review
The Food and Drug Administration has issued draft
recommendations for the development of direct acting antivirals (DDAs).
The revised draft guidance is important for the following reasons:
Recommendations for combining different
inhibitors (protease, polymerase and NS5a) with and without ribavirin in
the phase 2 and phase 3 studies to prevent drug resistance. These
recommendations encompass treatment-naïve and treatment-experienced
Sustained virologic response (SVR) has been
changed from 24 weeks post-treatment to 12 weeks post-treatment. This
is important because it will help to speed up drug approval.
Now that more research has been completed on
DAA’s, the FDA is encouraging more studies for people who are HIV/HCV
coinfected, pre- and post-transplant, and decompesenated cirrhosis.
These are populations that are at highest risk for complications
including severe disease progression and death.
The email from the FDA is below and
there is a direct link included to the draft recommendations. Public
comment is encouraged. -Alan Franciscus
The Food and Drug Administration (FDA) has published draft guidance
to assist sponsors in the clinical development of direct-acting
antiviral (DAA) drugs for the treatment of chronic hepatitis C (CHC)
from the initial pre-investigational new drug application (pre-IND)
through the new drug application (NDA) and postmarketing stages.
Although comments on guidance may be submitted at any time, to ensure
that the Agency considers your comment on this draft guidance before
it begins work on the final version of the guidance, please submit
either electronic or written comments on the draft guidance by December
Comments may be submitted electronically at http://www.regulations.gov.
Alternatively, written comments can be submitted to the Division of
Dockets Management (HFA-305), Food and Drug Administration, 5630
Fishers Lane, rm. 1061, Rockville, MD 20852.
This guidance revises the draft guidance for industry entitled
“Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral
Agents for Treatment” issued in September 2010. Significant changes in
this revision include:
Details on phase 2 and phase 3 trial design options for the
evaluation of interferon (IFN)-free and IFN-containing regimens in
treatment-naïve and treatment-experienced populations, including
Revised primary endpoint to sustained virologic response at 12 weeks post-treatment cessation.
Greater emphasis on DAA drug development in special populations
including trial design options for human immunodeficiency
virus/hepatitis C virus co-infected patients, patients with
decompensated cirrhosis, and patients pre- or post-liver transplant.
More details on clinical virology considerations for DAA drugs.
Office of Special Health Issues
Food and Drug Administration
Division of Antiviral Drug Products
Food and Drug Administration