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The effects of new regulatory initiatives on process validation.(pharmaceutical cGMPs for the 21st century

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Journal of Validation Technology , 02/01/2006 12 2

The effects of new regulatory initiatives on process validation.(pharmaceutical cGMPs for the 21st century: A risk-based approach)(current good manufacturing practice (GMP)) Fields, Tim *~|~*

COPYRIGHT 2006 Advanstar Communications, Inc.


Following in the wake of the concept paper, "Pharmaceutical cGMPs for the 21st Century: A Risk-Based Approach, (1)" issued by the Food and Drug Administration (FDA) in August 2002, other initiatives have been published by the FDA and International Conference on Harmonization (ICH) that have the potential to impact process validation. The underlying theme of these initiatives is the application of good science and modern technology in the manufacture of drugs. The new initiatives focus on prevention of product quality and processing problems through the application of good science and risk-based approaches rather than reliance on the reactive approaches of the past. The initiatives encourage use of modern technology, such as Process Analytical Technology (PAT), for monitoring and controlling processes. The new initiatives reinforce the use of science-based risk analysis to define the aspects of the process that are critical to product quality and then to focus validation!
efforts on those critical aspects. Although documentation remains a key requirement for process validation, the paradigm is shifting from documentation focused process validation efforts to a focus on enhancing process and product understanding. While these initiatives do not replace or supersede the regulatory requirements for process validation, they are likely to alter the way companies approach validation. NEW INITIATIVES
The new initiatives addressed in this article are as follows:
* Draft Guidance for Industry: Quality Systems Approach to Pharmaceutical Current Good Manufacturing Practice (GMP) Regulations (2)
* PAT: A Framework for Innovative Pharmaceutical Development, Manufacturing, and Quality Assurance (3)
* ICH Q8: Pharmaceutical Development (4)
* ICH Q9: Quality Risk Management (5)
Historical Perspective
Articles by Tetzlaff et al., (6) and Chapman (7) provide excellent overviews of the history of validation in the U.S. including chronologies of events that eventually lead to many of the validation practices applied today in the pharmaceutical industry. These events will not be repeated here, but are important elements in understanding the validation approaches and terminology used today and formed the basis for the "old paradigm" of process validation.
Misinterpretation and misunderstanding of regulatory requirements and expectations have resulted in process validation often being viewed as a documentation exercise in which three batches of product were manufactured in the middle of the process parameter range, with limited knowledge of the science behind the establishment of the ranges. Often, upon completion of the three "validation batches," a final report was written and approved, and then the documentation was shelved and rarely used.
Additionally, process validation has frequently been viewed as an event (i.e., manufacture three batches) rather than a lifecycle that begins in development and continues until the product is no longer manufactured.
The new initiatives focus on process and product knowledge through the entire product lifecycle including the use of risk-based methodologies to define the critical process parameters and good science and modern technology to determine the effects on product quality if the critical process parameters are varied. Underlying the use of good science, modern technology, and a risk-based approach to process validation is the establishment and maintenance of a good quality management system, which includes such things as written standard operating procedures (SOPs), change control, properly trained and qualified personnel, and properly installed and maintained equipment.
Regulatory Perspective
Process validation is required by regulations in all of the major international markets (i.e., U.S., EU, Canada, and Japan) and although the regulatory approaches may vary in each of these markets, the basic requirements for process validation are similar (see Figure 1).
Regulatory agencies in all of the major markets use a multi-tiered approach involving laws, regulations, and supplementary documents to ensure the safety, efficacy, and quality of drug products. In the U.S., the underlying law enforced by the FDA is the Food, Drug, and Cosmetic Act (FD & C) better know as "The Act." A similar law, Food and Drug Act, exists in Canada. In the European Union (EU), Directives (e.g., Directive 2003/94/EC) are issued that serve as the underlying law for the pharmaceutical industry. The Pharmaceutical Affairs Law is applied in Japan.
The U.S. FD & C Act calls for the FDA Commissioner to promulgate regulations to define the requirements that firms must meet to comply with the Act. These regulations can be found in Title 21 of the Code of Federal Regulations (CFR). For example, 21CFR Parts 210-211 contain the regulations known as Good Manufacturing Practices (GMPs) for Finished Drug Products. Other parts of 21CFR contain GMPs for Blood Products (21CFR Part 606) and GMPs for Medical Devices (21CFR Part 820). These regulations establish legal requirements that must be met before product can enter into interstate commerce. Similar documents developed in the EU and Canada as Guidelines (e.g., Guide to Good Manufacturing Practice for Medicinal Products in the EU, and Good Manufacturing Guidelines in Canada) provide more detailed interpretation of the laws in their respective countries.
Supplementary documents, such as guidance documents, guides, and annexes, are also periodically issued by regulatory agencies to provide specific guidance on a particular topic. Regulations define "what must be done (e.g., what must be validated), while supplementary documents provide guidance on how to do it (e.g., how to validate). ICH Guidelines, which are approved by the participating countries (i.e., EU, U.S., and Japan) can also be considered as supplementary documents and provide useful advice on how to address an issue, but such guidelines are not regulatory requirements. Although compliance with these supplementary documents is not required, such compliance is usually a good idea.
Validation Definition
There are a variety of definitions of validation that have been developed over the years and promulgated in regulations and in supplementary documents. The definition that is probably cited most often is from the 1987 FDA Guidelines on General Principles of Process Validation, (8):</p> <pre> "Process validation is establishing documented evidence which provides a high degree of assurance that a specific process will consistently produce a product meeting its predetermined
specifications and quality attributes." </pre> <p>EU GMP Annex 15 (9) offers the following definition for process validation:</p> <pre> "The documented evidence that the process, operated within
established parameters, can perform effectively and reproducibly to produce a medicinal product meeting its predetermined specifications
and quality attributes." </pre> <p>The definition in the Canadian GMPs10 expounds slightly on these definitions:</p> <pre> "The documented act of demonstrating that any procedure, process, and activity will consistently lead to the expected results. It includes qualification of systems and equipment. Manufacturing processes are clearly defined and controlled. All critical processes are validated to ensure consistency and compliance with specifications." </pre> <p>The International Conference on Harmonization ICH Q7A11 provides the following definition for process validation of active pharmaceutical ingredients (APIs):</p> <pre> "Process Validation (PV) is the documented evidence that the process, operated within established parameters, can perform effectively and reproducibly to produce an intermediate or API meeting its predetermined specifications and quality attributes." </pre> <p>Figure 2 provides a synopsis of these definiti!
ons demonstrating the similarities in all of them.
FOUR KEY PRINCIPLES
Although each of these definitions is slightly different, there are four key principles that appear to be harmonized among these definitions:
* The process must be defined and specific
* The product must meet pre-defined specifications
* There must be documented evidence
* The process must be reproducible
Key Principle 1 -- The Process Must Be Defined and Specific
A process cannot be validated unless it is defined. The definition of a process begins in the early stages of development where the materials, equipment, critical process parameters, and critical quality attributes are initially defined. Validation is a process that follows a lifecycle, which begins with the development of the process and product. Product quality must be designed into the product during the development phase because quality cannot later be tested into the product. 21CFR 211.110(a) (12) requires that:</p> <pre> "manufacturing processes that may be responsible for causing variability in the characteristics of in-process material and the drug product" be validated. </pre> <p>The EU GMPs (5.37) (13) states, "Critical processes should be validated." Canadian GMPs likewise require validation of critical processes [C.02.011 (I 2)].
Critical process variables can be determined by applying a risk-based scientific approach during development as described in ICH Q9. Any process variable that affects product quality should be considered a critical process variable. The effects of varying the critical process variables on product quality should be determined and documented. When determining the critical processing variables, it is important to consider any variables that might affect product quality, including: process operating parameters, manufacturing materials, equipment, and container-closure systems. Defining the variables and the means for controlling them is critical to defining the process to be validated.
The Proven Acceptable Ranges (PAR) approach described by Chapman (14) in 1984, defined critical process parameter ranges based on experimental data gathered by altering one critical process parameter at a time and looking for the adverse consequence of exceeding a limit. The PAR approach has evolved into the Design Space concept described in ICH Q8. The Design Space is defined as the established range of process parameters that has been demonstrated to provide assurance of quality. Movement out of the design space is considered to be a change and would normally initiate a regulatory post approval change process. The Design Space is established by collecting knowledge about the process and the factors that affect the process generally through the use of multi-variant Design of Experiments (DOE). While the PAR approach tended to be a two-dimensional analysis for establishing acceptable operating ranges, Design Space is a multi-dimensional analysis for looking at several fact!
ors that may affect product quality.
Key Principle 2 -- The Product Must Meet Pre-Defined Specifications
Defining the product specifications should follow an approach similar to that used to define the process parameters. Information gained from experiments conducted during development is used to define the product specifications. Risk analysis and good science are used to determine which product attributes are critical to quality. The critical attributes (e.g., particle size, solubility, and moisture content) of active pharmaceutical ingredients (APIs) and excipients critical to the quality of the final product should be determined during development and included in the specifications for such materials. Finished product specifications should be defined and include attributes critical to the proper functioning of the drug throughout its shelf-life (e.g.: dissolution, sterility, moisture content). The information gained during process and product development should be used to support the product specifications.
Once it has been determined which attributes are critical to product quality, controls must be established to ensure that these critical quality attributes are met. The level of control required is based on a risk analysis.
Key Principle 3 -- There Must Be Documented Evidence
Although the focus of process validation should be on gaining process and product knowledge, the focus is usually on developing the documentation. A question often asked about validation documentation is, "How much is enough?"
Figure 3 lists some common validation documents that are described in the EU Guide, Annex 15. In addition to the documents listed in Figure 3, other key documents may play a role during validation, including: development reports, standard operating procedures (SOPs), manufacturing instructions, lab records, risk analyses, design specifications, material specifications, equipment specifications, and product specifications.
Although the focus of process validation must be on obtaining knowledge about the process and the product, this knowledge must be documented and not lost. Documentation of the knowledge is an ongoing process following the product lifecycle. Process and product knowledge gained during development must be collected, documented, and provided to those responsible for producing the product. This documented knowledge should include "common cause" variability (i.e., known variability that is inherent in the process), which will enable informed decisions to be made when events occur (e.g., out of specification batch). Validation documentation should be used as a reference when making decisions regarding process deviations, changes to the process, and changes to the materials used. Since validation follows a lifecycle, any knowledge gained in regard to changes to the process or materials, or any deviations, should also be captured and included in the validation documentation for fu!
ture use. Every batch produced provides more validation documentation and knowledge regarding the process.
Key Principle 4 -- The Process Must Be Reproducible
Under the old paradigm, a common approach to process validation was to collect data during development to establish proven acceptable ranges, and then to produce three commercial scale batches to demonstrate reproducibility by sampling at discrete points in the process. Because the product is manufactured for the market, data continues to be analyzed to provide additional support for process reproducibility. For example, to demonstrate blending times, samples may be collected at 15-minute intervals over a two-hour time span and analyzed to provide documented evidence that the established blend times will result in an acceptable product.
Use of PAT concepts and on-line continuous monitoring could eliminate the need for such "experimental" approaches to validation by providing continuous on-line monitoring and control to ensure that product is produced within established operating parameters. PAT provides improvements over the current process validation methodology by providing the opportunity for immediate feedback and control over process variability. PAT provides the ability to continuously validate the process. For example, if a new raw material supplier is used, on-line continuous monitoring may indicate that the material has some different characteristics that affect the process, even though the raw material met the release specifications. Such differences may not be detected if sampling is performed only at the end of the process.
SUMMARY
Although the objectives of the new initiatives are not directly aimed at process validation, many of the concepts addressed in these initiatives lend themselves to getting the focus of validation back onto the critical aspects of the process and not on documentation. These new initiatives should result in a paradigm shift that uses science and risk-based approaches to ensure that quality is designed into the product and that validation is addressed from a scientific view and not as art. The new initiatives address validation from a lifecycle perspective and not as a one-time event. By merging science-based risk management with an integrated quality systems approach and the use of modern technology, quality is built into the product and the process is validated and maintained in a validated state.
ABOUT THE AUTHOR
Tim Fields is the President of Drumbeat Dimensions, Inc. (DBD), a professional compliance management company located in Mystic, CT, that provides products and services designed to help firms assess and enhance their regulatory compliance.
Mr. Fields has over 22 years of experience in the Pharmaceutical Industry including 13 years with Pfizer where he was responsible for organizing and managing the corporate software quality assurance audit program.
Mr. Fields is a member of the International Society of Pharmaceutical Engineers (ISPE), Parenteral Drug Association (PDA), and GAMP Americas Manufacturing Execution Systems (MES) Special Interest Group. He has published and lectured worldwide on computer-related system validation, electronic signatures and records, and document management. Mr. Fields is a member of the Editorial Review Board for the Journal of GXP Compliance.
Mr. Fields has a B.A. in Biology from Indiana University and an M.A. in Life Sciences from Indiana State University. He may be reached via email at: tim_fields@drumkey.com
REFERENCES
1. U.S. Food and Drug Administration, "Pharmaceutical cGMPs for the 21st Century--A Risk based Approach," FDA, (http://www.fda.gov/oc/guidance/gmp.html), August 2002.
2. U.S. Food and Drug Administration, "Draft Guidance for Industry; Quality Systems Approach to Pharmaceutical Current Good Manufacturing Practice Regulations," FDA, (http://www.fda.gov/cber/gdlns/qualsystem.htm), Sept. 2004.
3. U.S. Food and Drug Administration, "PAT--A Framework for Innovative Pharmaceutical Development, Manufacturing, and Quality Assurance," FDA, (http://fda.gov/cder/guidance5882fnl.htm), Sept 2004.
4. International Conference on Harmonization, "ICH Q8, Pharmaceutical Development," ICH, (http://www.ich.org/LOB/Media/MEDIA1707.pdf), Nov. 2005.
5. International Conference on Harmonization, "ICH Q9, Quality Risk Management," Federal Register 70 (151), 45722-45723 (August 8, 2005).
6. R.F. Tetzlaff, R. E. Shepherd, and A.J. LeBlanc, "The Validation Story: Perspectives on the Systematic GMP Inspection Approach and Validation Development," Pharmaceutical Technology 17 (3), 100-112, (1993).
7. K.G. Chapman, "A History of Validation in the United States: Part I, "Pharmaceutical Technology 15 (10), 82-96 (1991), and "Part II, Validation of Computer-Related Systems," Pharmaceutical Technology 15 (11), 54-70 (1991).
8. U.S. Food and Drug Administration, "Guideline on General Principles of Process Validation," FDA, May 1987.
9. European Commission, Enterprise Directorate-General, "Final Version of Annex 15 to the EU Guide to Good Manufacturing Practice--Qualification and Validation," European Commission, (http://www.pharmacos.eudra.org/F2/eudralex/vol-4/pdfs-en/v4an15.pdf), July 2001.
10. Health Products and Food Branch Inspectorate, "Canadian Good Manufacturing Practices Guidelines, 2002 Edition Version 2," (http://www.hc-sc.gc.ca/dhp-mps/compli-conform/gmp-bpf/guide-ld-2002/index_e.html), December 31, 2002.
11. International Conference on Harmonization, "Guidance for Industry Q7A Good Manufacturing Practice Guidance for Active Pharmaceutical Ingredients," August 2001.
12. Code of Federal Regulations, Title 21, Food and Drugs, Part 211, "Current Good Manufacturing Practice for Finished Pharmaceuticals," 1 April 2004.
13. "The Rules Governing Medicinal Products in the European Community, Volume IV," Good Manufacturing Practice for Medicinal Products (Commission on the European Communities, Brussels, Luxembourg, 1992) Commission Directive 91/356/EEC.
14. K.G. Chapman, "The Par Approach to Process Validation," Pharmaceutical Technology, 8 (12), 22-36 (1984).
Article Acronym Listing
<pre>
API Active Pharmaceutical Ingredient
C Centigrade
CFR Code of Federal Regulations
DOE Design of Experiment
EU European Union
FD &amp; C Food, Drug, and Cosmetic (Act)
FDA Food and Drug Administration
GMP Good Manufacturing Practice
ICH International Conference on
Harmonization
PAR Proven Acceptable Range
PAT Process Analytical Technology
PV Process Validation
SOP Standard Operating Procedures
U.S. United States
VMP Validation Master Plan

Figure 1 Regulatory Requirements for Process Validation

U.S. (21CFR) EU Guide Canada ICH Q7A

211.100 1.3 C.02.011 8.4
5.23 Interpretations 2, 3, 4
211.110(a) 5.37
C.02.014
Interpretation 6

Figure 2 Comparison of Process Validation Definitions

U.S. Drug 1987
Process Validation
Guideline EU Guide Annex 15

Documented evidence Documented evidence
Specific process Defined process
Consistent product Consistently yields
product of the required
quality
Meets predetermined Yields product of
specifications and required quality
quality attributes

Canada GMPs ICH Q7A

Documented act Documented evidence
Processes clearly Process operated
defined within established
parameters
Ensures consistency Reproducible
Complies with Meets predetermined
specifications specifications and
quality attributes

Figure 3 Validation Documentation Requirements

Document EU Guide Annex 15

Validation Plan All validation activities should be planned. The key
elements of a validation programme should be clearly
defined and documented in a validation master plan
(VMP) or equivalent documents.
Protocol A written protocol should be established that
specifies how qualification and validation will be
conducted. The protocol should be reviewed and
approved. The protocol should specify critical steps
and acceptance criteria.
Report A report that cross-references the qualification and/
or validation protocol should be prepared, summarizing
the results obtained, commenting on any deviations
observed, and drawing the necessary conclusions,
including recommending changes necessary to correct
deficiencies. Any changes to the plan as defined in
the protocol should be documented with appropriate
justification. </pre>

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Pharmaceutical Validation Documentation Requirements

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