NorthWest Biotherapeutics Inc (NWBO)

[aboobmove]

Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products
Guidance for Industry
DECEMBER 2019

Draft
Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

Email
Print

Submit Comments by 02/18/2020
Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the FDA considers your comment on a draft guidance before it begins work on the final version of the guidance, submit either online or written comments on the draft guidance before the close date.

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2019-D-4964

Docket Number:
FDA-2019-D-4964
Issued by:
Center for Biologics Evaluation and Research
Center for Drug Evaluation and Research
This document is intended to provide guidance to applicants planning to file new drug applications (NDAs), biologics license applications (BLAs), or applications for supplemental indications on the evidence to be provided to demonstrate effectiveness. This guidance complements and expands on the 1998 guidance entitled Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products (the 1998 guidance).

The 1998 guidance was issued in response to the Food and Drug Administration Modernization Act of 1997 (FDAMA) (Pub. L. 105–115), which stated that the substantial evidence requirement for effectiveness, which had generally been interpreted as calling for two adequate and well-controlled trials, could also be met by a single trial plus confirmatory evidence. The 1998 guidance, therefore, provided many examples of the types of evidence that could be considered confirmatory evidence, with a specific focus on adequate and well-controlled trials of the test agent in related populations or indications, as well as a number of illustrations of a single adequate and well-controlled trial supported by convincing evidence of the drug’s mechanism of action in treating a disease or condition.

FDAMA thus introduced a specific new area of flexibility in the evidence needed to support effectiveness, but there are many other characteristics of the evidence supporting effectiveness that can vary (notably, trial designs, trial endpoints, statistical methodology), and evidence that varies in such ways potentially can provide substantial evidence of effectiveness but because of these characteristics may provide greater or lesser certainty. These characteristics also deserve consideration and were not discussed in the 1998 guidance. FDA’s consideration of these various designs, endpoints, and analyses which can differ in the strength of evidence they provide, reflects the Agency’s longstanding flexibility when considering the types of data and 39 evidence that can meet the substantial evidence requirement.

Questions?
Human Drug Information
Office of Communications
10001 New Hampshire Ave
Hillandale Building, 4th Fl
Silver Spring, MD 20993
[e-mail]
[phone] Toll Free
[phone]
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
[e-mail]
[phone]
[phone]





https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-human-drug-and-biological-products