CHAPTER 56 -- DRUG QUALITY ASSURANCE

SUBJECT:

Inspections of Licensed Biological Therapeutic Drug Products

 

IMPLEMENTATION DATE

 

October 20, 2003

*Revision note:

This program is being reissued as a CDER program due to the transfer of certain biological products to CDER's jurisdiction effective FY04.  This reissued program retains most of the instructions and procedures from the prior version published by CBER: 7341.001.  (CBER may continue this same program under their program number (7341.001) for products remaining under their jurisdiction.)

Products regulated as human drugs by CDER prior to the transfer of certain biological products from CBER are subject to the existing Compliance Program for these products, 7356.002, only.*

 

COMPLETION DATE

 

September 30, 2006

DATA REPORTING

PRODUCT CODES

PRODUCT/ASSIGNMENT CODES

57YY-

 

56002M   GMP Inspections

46832M   Pre-license Inspections

 

 

FIELD REPORTING REQUIREMENTS:

 

Domestic Inspections: A copy of each establishment inspection report (EIR), including endorsement and classification, should be submitted to *CDER, Office of Compliance Division of Manufacturing and Product Quality (DMPQ), Investigations & Preapproval Compliance Branch (HFD-322).*  Exhibits should not be included unless specifically requested. A copy of the coversheet and endorsement should be sent to HFC-100.  The complete original report, including exhibits, should be sent to the home district for filing.

 

Foreign Inspections: The complete original EIR, including exhibits, should be forwarded to *CDER Division of Manufacturing and Product Quality (DMPQ), Investigations & Preapproval Compliance Branch, Foreign Inspection Team (FIT), HFD-325, regardless of classification.*  A copy of the coversheet and endorsement should be sent to HFC-100.

 


PART I - BACKGROUND

 

FDA is responsible for ensuring that biological products are safe and effective and in compliance with the law and FDA regulations.  Biological products are licensed under the provisions of Section 351 of the Public Health Service Act (42 USC) (PHS Act).

 

Licensed therapeutic products are regulated under both the Federal Food, Drug and Cosmetic Act (FD&C Act) and the PHS Act.  For those therapeutic products that meet the definition of a drug under the FD&C Act, the manufacture of these products must be performed in accordance with the cGMP regulations in 21 CFR Parts 210 and 211.  As the products are also biologicals, manufacturers must also comply with the applicable regulations in 21 CFR Parts 600-680.

 

Technical advances over the last 15 years have greatly increased the ability of manufacturers to control and analyze the manufacture of many biotechnology-derived therapeutic products.  Specified biotechnology-derived products, defined in 21 CFR 601.2, are exempted from complying with certain biologics regulations and are not required to be submitted for lot release.  These products are (1) therapeutic synthetic peptides of 40 or fewer amino acids; (2) therapeutic DNA plasmid products; (3) monoclonal antibodies for in vivo use; and (4) therapeutic recombinant DNA-derived products.

 

*This program covers both specified and non-specified therapeutic biological drug products now regulated by CDER, but formerly regulated by CBER under this same program.* 

 

Technology

 

While the specifics of each manufacturing operation may be different, the manufacture of therapeutic biological products has a number of common elements.  The processes usually begin with a master cell bank (MCB), which is derived from a single cell or colony and is stored to assure genetic stability.  The MCB provides source material for the working cell bank (WCB), which is used to initiate the production batch.

 

One method of propagating sufficient cells to manufacture product is through fermentation.  Fermentation is the process of multiplying the cells from the WCB into a quantity sufficient to extract the desired product.  Cells from the WCB are inoculated into medium to begin fermentation.  After a number of passages in small vessels (usually flasks), the inoculated medium is added to a fermentation vessel, usually a bioreactor.  At the conclusion of the fermentation process, the cells are subject to a variety of purification steps, designed to remove extraneous cellular material and/or media components and inactivate or remove any adventitious agents.  Purification can include filtration, chromatography, extraction, and enzyme digestion.  The resulting final bulk product may be filled in this form, further diluted and filled, or lyophilized before filling.

 

Team Biologics

 

In October 1998, responsibility for conducting biennial inspections of therapeutic product manufacturers was transferred to the ORA Team Biologics/Core Team.  The Core Team will also be primarily responsible for handling any enforcement actions that result from inspections performed under this program, in accordance with the procedures set forth in the Team Biologics case processing procedure.  The Core Team will ensure that the home district is advised of activities related to facilities in their areas and may solicit assistance from the home district in carrying out the inspections or enforcement activities.

 

Pre-license inspections are the responsibility *of CDER*, and will include a field investigator whenever possible.


 

PART II - IMPLEMENTATION

 

A.              Objectives

 

This program has the following objectives:

 

  • To ensure the safety and effectiveness of licensed biological therapeutic drug products by determining that they are manufactured in compliance with current Good Manufacturing Practice regulations and that they comply with standards and commitments made in license applications and/or supplements.

 

  • To encourage voluntary compliance by identifying practices, which need correction or improvement, and to identify areas in which firms need to establish and implement programs.

 

  • To provide regulatory/administrative guidance to ensure that appropriate enforcement actions are initiated against those manufacturers found to be in significant noncompliance with applicable laws and regulations.

 

  • To provide information and guidance to investigators assigned to perform biennial GMP or for cause inspections of manufacturers of licensed biological therapeutic drug products.

 

B.              Program Management Instructions

 

Firms covered under this program include all licensed manufacturers of biological therapeutic drug products, including biotechnology-derived products.

 

Work planning for these inspections will be coordinated by Team Biologics.  Each facility and its biological products are to be covered in a single, comprehensive inspection that assesses the adequacy of all-significant processes and systems.  The inspections should be performed on at least a biennial basis, or more often if circumstances, such as the firm's compliance history, so warrant.

 

The inspections will be conducted using a team approach whenever possible, with an ORA Team Biologics/Core Team investigator leading and a *CDER* product reviewer participating.  The inspection team may include other ORA or *CDER* members, as necessary, to assure appropriate coverage of the facility.  If *CDER* on-site participation is not possible, the Core Team will proceed with the inspection with other ORA members, as necessary.

 

Historically, there have been individual inspections for each therapeutic class.  At some firms, this has resulted in a burdensome number of redundant inspections.  This program departs from this practice, in that there will be one inspection of a facility to cover all products.  If a company makes products reviewed by multiple divisions in *CDER*, the Center may send individuals from each division, but they should try to stagger the attendance of the additional persons to keep the number of individuals at the firm at one time to a reasonable level.


 

PART III – Inspectional

 

Every effort should be made to inspect each licensed firm at least once every two years or more frequently if the firm’s compliance history so warrants.

 

A.              Inspectional Procedures

 

Review and use applicable sections of Compliance Program 7356.002, Drug Manufacturing Inspections; Compliance Program 7356.002A, Sterile Drug Process Inspections; and Biotechnology Inspection Guide.  If there are differences between the instructions in this program and the above referenced documents, investigators should follow the instructions in this program when conducting inspections of manufacturers of licensed biological therapeutic products.

 

Manufacturers of licensed biological therapeutic drug products are subject to the regulations in 21 CFR Parts 600-680 and Parts 210 and 211.  Manufacturers of licensed products must also conform to the conditions in their approved license applications and supplements.  If it is necessary to verify the content of a license application or supplement, contact CDER for assistance.

 

B.              Inspection

 

1.               Components

 

Manufacturers who purchase components from outside sources are required to establish adequate quality requirements and specifications for such components.  The licensed manufacturer is ultimately responsible for ensuring that components conform to specifications and are acceptable for use.  This may be done through inspections, sampling and testing, and/or through certificates of analysis from the supplier.  Validity of the certificates should be established by the manufacturer through experience, historical data, testing, and/or audits of the supplier.

 

For components received from outside sources, either purchased or otherwise received, verify that:

 

  • The firm has written, approved, specified requirements for the component(s);

 

  • The firm evaluates and selects suppliers based on their ability to meet specified requirements; and

 

  • The type and extent of control needed over the component and suppliers has been defined and is based on the manufacturer's evaluation of the supplier.

 

Animal source material must meet the applicable requirements of 21 CFR 600.11.  Investigators should ensure that tests and specifications for materials of animal source that may potentially be contaminated with adventitious agents (e.g., mycoplasma, Bovine Spongiform Encephalopathy for bovine-derived products, and others) as described in a license application are performed.  If the license application includes certification supporting freedom of substances from adventitious agents, this should be confirmed during inspections.

 

Acceptance activities must be documented.  Verify that the manufacturer has defined methods, e.g., inspections, tests, and other verification tools (certificates of analysis and/or supplier audits), to ensure that components conform to all specifications prior to release for use in manufacturing and that acceptance activities are documented in the batch record.

 

Review the manufacturing SOPs and batch records for a representative number of lots to ensure that acceptance criteria are met for all components.

 

  1. Master Cell Bank (MCB) and Working Cell Bank (WCB)

 

    1. Storage conditions

 

The storage conditions for the MCB and WCB should be clearly defined.  Ensure that the storage conditions have been maintained.  If the storage requirements specify temperature limitations, ensure that there is documentation of the temperature and determine if there is a working alarm system in place if the temperature deviates from the established one.

 

    1. Identification

 

Ensure that the WCB has been characterized and met specifications before use.  If any WCB that did not meet specifications was used, determine which lot(s) of product was manufactured from the WCB and the disposition of the product.

 

Ensure the firm has records to show the origin and history (number of passages) of the MCB and WCB.

 

    1. Handling of the WCB

 

Review the records for inventory and handling of the WCB and ensure they are adequate to protect the integrity of the cells.  Ensure the firm has records to show which WCB is used to initiate a production batch.

 

    1. New MCB

 

If the firm has generated a new MCB from a WCB, ensure that there is an approved license application or supplement for this.  Ensure that the new MCB is tested and properly characterized.

 

  1. Media, buffers

 

The firm should have well-established acceptance criteria for all materials.  If buffers or media are prepared prior to use, determine if the firm has established holding times and conditions, validated the holding times and conditions, and has records to show the conditions are met.

 

  1. Containers/closures

 

Ensure that the firm has adequate written specifications and procedures describing the receipt, handling, sampling, and storage of containers and closures, especially those that need to be sterile and/or pyrogen-free.

 

Review procedures for accepting/rejecting final product containers and closures from the vendor.  Determine whether the firm conducts vendor audits and, if so, how and with what frequency.

 

Review the procedures and controls used by the firm to verify and ensure suitability of containers and closures.  Evaluate procedures used by the firm to validate the container/closure systems.  Report any changes in container/closure systems that have not been validated.

 

Review SOPs for reconciliation of final containers.

 

Ensure that depyrogenation and sterilization procedures for product containers, closures, and components are appropriately validated, and routinely followed.  Equipment used for these processes (stopper processors, tunnel sterilizers, ovens, autoclaves) should be properly maintained and re-qualified periodically.

 

2.               Manufacturing

 

a.                Aseptic/controlled process

 

Therapeutic products are manufactured in a controlled environment.  The entire process does not have to be done under aseptic conditions, but the firm must have established the point in the process where aseptic controls begin.

 

Observe the aseptic processes directly, when possible, and evaluate aseptic technique.  Evaluate all connections and transfers to ensure that they are made in an aseptic manner.

 

Review all cleaning and sanitization procedures for the aseptic core and evaluate whether the cleaning agents are used according to results of validation studies and whether surfaces are monitored to demonstrate continued efficacy.  If filling needles are re-used, the cleaning should be validated.

 

If the duration of filling is lengthy, determine if time limits have been set and validated to ensure that the duration of the fill does not affect the potency of the product and its susceptibility to microbial contamination.  An SOP should be in place for interruption of the fill, should it occur.

 

Some bulk products are held after sterile filtration prior to filling.  Determine whether the holding period and storage conditions have been validated.

 

Procedures must be in place for limiting access to controlled and classified areas.

 

Determine if the sterilizing filters were validated for product compatibility and microbial retention and are adequate for their intended use.  Evaluate validation of sterilization of filters.  Ensure that filters are evaluated prior to use to ensure they meet specifications.  Integrity testing should be performed on filters post-fill and results should be in keeping with manufacturer's and validated specifications.

 

Review the program in place for training operators.  In addition to training in the manufacturing process, the operators should also be trained in proper gowning technique.  Written procedures for gowning should be in place and followed.

 

Evaluate the firm's aseptic processing areas (filling and lyophilization) using 21 CFR 211.42(c)(10), the Guideline on Sterile Drug Products Produced by Aseptic Processing, and the *Draft Guidance for Industry – Sterile Drug Product Produced by Aseptic Processing – Current Good Manufacturing Practices as guides*.

 

Determine whether Class 100 conditions have been validated and are maintained in areas in which sterile product and components, including container/closure systems, are exposed.  When alert limits are exceeded, microbial identification should be performed.

 

Products must be maintained in a controlled environment throughout the process and have specified action and alert limits for which the firm can provide a meaningful rationale.  Bioburden alert and action levels should be established for intermediates.  Ensure that the manufacturer has established microbial specifications and a monitoring program to include identification of the types of microorganisms present.

 

Investigators should examine the system to determine if there are any ports of entry for adventitious agents.

 

b.               Endotoxin levels

 

There is often no step to remove endotoxin from the product, so it must be controlled from the beginning of the process.  If there is a step in the manufacturing process to remove endotoxin, it is usually early in the process, so the remainder of the process must be carefully controlled against contamination.  Investigators should review the manufacturing facility and process for potential opportunities for contamination.  Review the firm's procedures to monitor the bioburden level throughout the manufacturing process to ensure the level is controlled.

 

Ensure that:

 

·       Production is performed in a controlled environment that prevents an increase in the product’s microbial load beyond its design specifications;

 

·       Procedures to prevent equipment or product contamination by any substance that could reasonably be expected to have an adverse effect on product quality are in place and followed; and

 

·       Precautions are taken to prevent contamination or cross-contamination in areas in which operations for the preparation of cell banks and product manufacturing processes which are capable of promoting microbiological growth are monitored for bioburden on a routine basis.

 

c.                Fermentation/Bioreactors