Special Protocol Assessment Guidance for Industry | Guideline Explorer

Description

This guidance provides information on the procedures and general policies adopted by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) for special protocol assessment (SPA).

Scope & Applicability

Product Classes

4

Biosimilar biological product

Products requiring clinical studies to prove biosimilarity; Applications under section 351(k) of the PHS Act

Companion Diagnostic Device

Device protocols excluded from SPA

Combination Product

Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.

device

premarket review of device submissions

Stakeholders

6

Sponsor

Entity responsible for submitting applications under section 524B

FDA Division Director

Authority to change or rescind SPA agreement

advisory committee

External body providing input on labeling changes

Executive Carcinogenicity Assessment Committee

Renders final judgment on protocol acceptability

Division Director

FDA official who decides whether to accept an SPA request

Applicant

Entity submitting development data and knowledge; Entity performing the work process for change

Regulatory Context

Attributes

1

Type I error rate

Statistical parameter requiring strong control when testing multiple endpoints

Identified Hazards

Hazards

2

public health concerns

Basis for Agency to alter perspective on protocol design

Substantial scientific issue

Issue essential to determining safety or effectiveness identified after testing began

Related CFR Sections (9)

21CFR314.500§ 314.500 Scope.
This subpart applies to certain new drug products that have been studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit to patients over existing treatments (e.g., ability to treat patients unresponsive to, or intoRead full regulation →
21CFR601.40§ 601.40 Scope.
This subpart applies to certain biological products that have been studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit to patients over existing treatments (e.g., ability to treat patients unresponsive to, or inRead full regulation →
21CFR314.103§ 314.103 Dispute resolution.
(a) General. FDA is committed to resolving differences between applicants and FDA reviewing divisions with respect to technical requirements for applications or abbreviated applications as quickly and amicably as possible through the cooperative exchange of information and views.Read full regulation →
21CFR312.48§ 312.48 Dispute resolution.
(a) General. The Food and Drug Administration is committed to resolving differences between sponsors and FDA reviewing divisions with respect to requirements for IND's as quickly and amicably as possible through the cooperative exchange of information and views.Read full regulation →
21CFR10.75§ 10.75 Internal agency review of decisions.
(a) A decision of an FDA employee, other than the Commissioner, on a matter, is subject to review by the employee's supervisor under the following circumstances:Read full regulation →
21CFR314.101§ 314.101 Filing an NDA and receiving an ANDA.
(a) Filing an NDA.Read full regulation →
21CFR601.2§ 601.2 Applications for biologics licenses; procedures for filing.
(a) General. To obtain a biologics license under section 351 of the Public Health Service Act for any biological product, the manufacturer shall submit an application to the Director, Center for Biologics Evaluation and Research or the Director, Center for Drug Evaluation and Research (see mailing aRead full regulation →
21CFR312.23§ 312.23 IND content and format.
(a) A sponsor who intends to conduct a clinical investigation subject to this part shall submit an “Investigational New Drug Application” (IND) including, in the following order:Read full regulation →
21CFR314.126§ 314.126 Adequate and well-controlled studies.
(a) The purpose of conducting clinical investigations of a drug is to distinguish the effect of a drug from other influences, such as spontaneous change in the course of the disease, placebo effect, or biased observation. The characteristics described in paragraph (b) of this section have been develRead full regulation →