Tag Archives: Quality

NICE Publish Quality Standard for Antenatal Care

The majority of pregnant women in the UK will have an uncomplicated pregnancy, giving birth to a healthy baby at full term. However, common problems including miscarriage, fetal growth restrictions and premature birth still remain, and stillbirth rates have changed very little in recent years. Depression, thromboembolism, haemorrhage and sepsis are still encountered, with the most extreme cases contributing to the UK’s maternal mortality rate of around 11 out of 100,000 maternities (data from 2006-2008).

For further details, please view the document below.

EMA Publish Guideline on the Requirements for Quality Documentation Concerning Biological Investigational Medicinal Products in Clinical Trials

The “Detailed guidance on the request to the competent authorities for authorisation of a clinical trial on a medicinal product for human use, the notification of substantial amendments and the declaration of the end of the trial”1 (‘detailed guidance CT-1’) sets out the requirements as regards data related to an investigational medicinal product (IMP) to be submitted with the request for a clinical trial authorisation in the IMP Dossier (IMPD).

For further details, please view the document below.

EMA Publish Guideline on Quality, Non-Clinical and Clinical Aspects of Medicinal Products Containing Genetically Modified Cells

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans and presented for marketing authorisation. Its focus is on the quality, safety and efficacy requirements of genetically modified cells developed as medicinal products.The quality section addresses the requirements specific for the genetic modification of the target cell population and for the transduced cell product resulting from the manufacturing process.

The non-clinical section addresses the non-clinical studies required with the aim at maximising the information obtained on dose selection for the clinical trials, to support the route of administration and the application schedule. Non-clinical studies should also allow determining whether the observed effect is attributable to the transduced gene, to the transduced cells or to both.

The clinical section addresses the requirements for studying as far as possible pharmacological properties of the cells itself and the transgene. The requirements for efficacy studies emphasises that the same principles apply as for the clinical development of any other medicinal product, especially those of current guidelines relating to specific therapeutic areas. The clinical section further addresses the safety evaluation of the product as well as the principles for follow up and the pharmacovigilance requirements.

For further details, please view the document below.

EMA Publish VICH GL51: Quality: Statistical Evaluation of Stability Data

This guideline describes when and how extrapolation can be considered when proposing a retest period for a drug substance or a shelf life for a veterinary medicinal product that extends beyond the period covered by “available data from the stability study under the long-term storage condition” (hereafter referred to as long-term data). Application of this guideline is entirely optional and it is up to the Applicant to decide whether or not to use statistical analysis to support the claimed retest period/shelf-life.

For further details, please view the document below.

FDA publishes guidance for industry on Q8, Q9 and Q10 (R4) questions and answers

Since the Q8, Q9, and keep 10 guidance is were made final experiences implementing the guidance is in the ICH regions are given rise to requests for clarification. So the FDA has consolidated these requests into a structured questions and answers document that users should interrogate.

Q8 is associated with pharmaceutical development, Q9 is associated with quality risk management, and Q 10 is associated with pharmaceutical quality systems.

EMA published guidance on specifications for herbal products

This new EMA guidance document provides general principles on the setting and justification of a uniform set of specifications herbal substances and preparations. Since a simplified registration procedure was established for traditional herbal medicinal products for human use the quality medicinal products is independent of its traditional use. More specifically the addition of vitamins to herbal products need to comply with all the relevant legislation guidance.

This document is concerned with the specific definitions as a list of tests, references to analytical and biological procedures and appropriate acceptance criteria a number of limit ranges and other criteria have been as described and established.

excerpt from regulations

full text here

This guidance document provides general principles on the setting and justification, to the extent possible, of a uniform set of specifications for herbal substances/preparations and herbal medicinal products to support applications for marketing authorisation or registration according to Directives 2001/82/EC and 2001/83/EC as amended. It should be read in conjunction with the ‘Guideline on quality of herbal medicinal products’ (EMA/CPMP/QWP/2819/00, EMA/CVMP/814/00, EMA/HMPC/201116/2005, as revised).
A simplified registration procedure was established for traditional herbal medicinal products for human use under Directive 2004/24/EC. The quality of a medicinal product is independent of its traditional use; therefore all general principles of quality also apply to traditional herbal medicinal products for human use. Traditional herbal medicinal products for human use may additionally contain vitamins or minerals. Concerning these products, this guideline describes specific aspects linked to mixtures of herbal substances/herbal preparations with vitamins and/or minerals. In addition, the quality, specifications and documentation for each vitamin and mineral have to comply with all relevant legislation and guidelines.

A specification is defined as a list of tests, references to analytical and biological procedures, and appropriate acceptance criteria, which are numerical limits, ranges, or other criteria for the tests described. It establishes the set of criteria to which a herbal substance/preparation or herbal medicinal product should conform to be considered acceptable for its intended use. “Conformance to specifications” means that the herbal substance/preparation or herbal medicinal product, when tested according to the listed analytical procedures, will meet the listed acceptance criteria. Specifications are legally binding quality standards that are proposed and justified by the manufacturer and approved by regulatory authorities.
Specifications are one part of a total control strategy for the herbal substance/preparation and herbal medicinal product designed to ensure product quality and consistency. Other parts of this strategy include thorough product characterisation during development, upon which specifications are based, adherence to the ‘Guideline on Good Agricultural and Collection Practice (GACP)’ (EMEA/HMPC/246816/2005) and Good Manufacturing Practice (GMP), and a validated manufacturing process, e.g., raw material testing, in-process testing, stability testing, etc.
In the case of herbal medicinal products, specifications are generally applied to the herbal substance, to the herbal preparation and to the herbal medicinal product. Specifications are primarily intended to define the quality of the herbal substance/preparation and herbal medicinal product rather than to establish full characterisation, and should focus on those characteristics found to be useful in ensuring the safety and efficacy of the herbal substance/preparation and herbal medicinal product.

EMA Publishes Guidance on Paediatric Development of Medicinal Products

EMA Publishes Guidance on Paediatric Development of Medicinal Products

Full Text Here

Children can neither be regarded as small adults nor as a homogeneous group in themselves. As a consequence, paediatric medicines should be appropriately designed for the target age group(s).
In January 2007 Regulation EC No 1901/2006 (the “Paediatric Regulation”) entered into force. As a result of this Regulation, the number of paediatric formulations that the pharmaceutical industry will have to develop to support their clinical trials will increase. It is expected that the number of medicines applying for a marketing authorisation for paediatric use will increase as a result. Therefore, the existing regulatory documents need to be supported by specific regulatory guidance on the pharmaceutical development of medicines for use in children between birth and 18 years of age.

The physical, metabolic and psychological processes peculiar to growth from birth into adulthood reveal that children can not be regarded as small adults nor can they be regarded as a homogeneous group in themselves. As a consequence, clinical studies in adults are not necessarily predictive for children. Thus, clinical trials may be needed in children of different ages in order to demonstrate that a medicine is safe and effective in all of the indicated target age group(s).
In addition, the treatment of children with medicines poses specific pharmaceutical problems which have not been seen to the same extent in adults and which occurrence may be age dependent. For example, young children are simply unable to swallow conventionally-sized tablets whereas tablets are a favourable dosage form for elder children and adults. Especially neonates pose specific characteristics and needs. They may for example require very small volumes of a parenteral medicine in order to avoid a volume overload. Therefore, children should be treated with medicinal products of which the pharmaceutical design is tailored for use in the target age group i.e. age appropriate medicines.
Knowledge on the critical to quality aspects of paediatric medicines is still limited, especially when considering these aspects in a multidimensional approach to the best attainable and affordable paediatric medicinal products. As a consequence, the usefulness (practicality) of some of the currently paediatric medicines might be questionable / based on minimum standards and could consequently be subject to further optimisation in the interest of parents, other caregivers and children.
On the 26th of January 2007, the “Paediatric Regulation” entered into force (Regulation EC No 1901/2006 of The European Parliament and of the Council, amending regulation EEC No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation EC No 726/2004). This regulation aims to “facilitate the development and accessibility of medicinal products for use in the paediatric population, to ensure that medicinal products used to treat the paediatric population are subject to research of high quality and are appropriately authorised for use in the paediatric population, and to improve the information available on the use of medicinal products in the various paediatric populations”. As a result of this Regulation, both the number of paediatric formulations that should be developed by the pharmaceutical industry and the knowledge on the critical to quality aspects of paediatric medicines is expected to increase rapidly.
Bearing the aforementioned in mind, the current regulatory documents need to be supported with guidance on the pharmaceutical development of paediatric medicines. Therefore, this guideline aims to provide additional tools for the rationale pharmaceutical development of medicines for children between birth and 18 years of age to those already described in the current CHMP and ICH guidelines. The guideline intends to balance between predictable and consistent regulatory assessments of paediatric medicines (either generic, innovative, existing or new), the speed of development, industrial feasibility and the need to develop medicines that are better tailored for use in children than the currently authorised, but “questionable” paediatric medicines or the currently applied off-label or pharmacy compounded medicines. The outcome of this balanced approach should not necessarily result in a “gold standard” paediatric medicine.



For Assistance with Paediatric Product Development Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…


  1. (required)


  2. (valid email required)

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA Publish Draft Guidance on Development of Medicines for Paediatric Use

EMA Publish Draft Guidance on Development of Medicines for Paediatric Use

Full Text Here

Children can neither be regarded as small adults nor as a homogeneous group in themselves. As a consequence, paediatric medicines should be appropriately designed for the target age group(s).
In January 2007 Regulation EC No 1901/2006 (the “Paediatric Regulation”) entered into force. As a result of this Regulation, the number of paediatric formulations that the pharmaceutical industry will have to develop to support their clinical trials will increase. It is expected that the number of medicines applying for a marketing authorisation for paediatric use will increase as a result. Therefore, the existing regulatory documents need to be supported by specific regulatory guidance on the pharmaceutical development of medicines for use in children between birth and 18 years of age.

The physical, metabolic and psychological processes peculiar to growth from birth into adulthood reveal that children can not be regarded as small adults nor can they be regarded as a homogeneous group in themselves. As a consequence, clinical studies in adults are not necessarily predictive for children. Thus, clinical trials may be needed in children of different ages in order to demonstrate that a medicine is safe and effective in all of the indicated target age group(s).

In addition, the treatment of children with medicines poses specific pharmaceutical problems which have not been seen to the same extent in adults and which occurrence may be age dependent. For example, young children are simply unable to swallow conventionally-sized tablets whereas tablets are a favourable dosage form for elder children and adults. Especially neonates pose specific characteristics and needs. They may for example require very small volumes of a parenteral medicine in order to avoid a volume overload. Therefore, children should be treated with medicinal products of which the pharmaceutical design is tailored for use in the target age group i.e. age appropriate medicines.

Knowledge on the critical to quality aspects of paediatric medicines is still limited, especially when considering these aspects in a multidimensional approach to the best attainable and affordable paediatric medicinal products. As a consequence, the usefulness (practicality) of some of the currently paediatric medicines might be questionable / based on minimum standards and could consequently be subject to further optimisation in the interest of parents, other caregivers and children.

On the 26th of January 2007, the “Paediatric Regulation” entered into force (Regulation EC No 1901/2006 of The European Parliament and of the Council, amending regulation EEC No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation EC No 726/2004). This regulation aims to “facilitate the development and accessibility of medicinal products for use in the paediatric population, to ensure that medicinal products used to treat the paediatric population are subject to research of high quality and are appropriately authorised for use in the paediatric population, and to improve the information available on the use of medicinal products in the various paediatric populations”. As a result of this Regulation, both the number of paediatric formulations that should be developed by the pharmaceutical industry and the knowledge on the critical to quality aspects of paediatric medicines is expected to increase rapidly.

Bearing the aforementioned in mind, the current regulatory documents need to be supported with guidance on the pharmaceutical development of paediatric medicines. Therefore, this guideline aims to provide additional tools for the rationale pharmaceutical development of medicines for children between birth and 18 years of age to those already described in the current CHMP and ICH guidelines. The guideline intends to balance between predictable and consistent regulatory assessments of paediatric medicines (either generic, innovative, existing or new), the speed of development, industrial feasibility and the need to develop medicines that are better tailored for use in children than the currently authorised, but “questionable” paediatric medicines or the currently applied off-label or pharmacy compounded medicines. The outcome of this balanced approach should not necessarily result in a “gold standard” paediatric medicine.



For Assistance with Paediatric Drug Development and PIPs Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…


  1. (required)


  2. (valid email required)

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA Publishes Concept Paper on using CTD Format in Traditional Herbal Medical Products Applications

EMA Publishes Concept Paper on using CTD Format in Traditional Herbal Medical Products Applications

Full Text Here

This concept paper is concerned with the revision of the guideline on the use of the Common Technical Document (CTD) format in the preparation of a registration application for traditional herbal medicinal products (EMEA/HMPC/71049/2007).
This revision pertains to the presentation and content of the Module 3 on Quality (chemical, pharmaceutical and biological information) for traditional herbal medicinal products (THMPs) to help future applicants in their submission.

This guideline is applicable to applications for traditional use registration of THMPs for human use.
The compilation of dossiers for marketing authorisation applications for HMPs is not covered by this guideline. However, guidance provided on modules 2.3 and 3 are also applicable to HMPs applications for marketing authorisation for human and veterinary use.

In the light of experience, there is a need to update this guideline to provide further clarification on the exact location of quality data requirements in the Module 3 on Quality of the CTD and to provide further explanations on the kind of information that is required from applicants at time of application.
Minor changes in the body of the current guideline will be introduced and two annexes will be added. The first annex will be a best practice guide describing the exact location of relevant parts of the documentation and the corresponding guidelines in the CTD Module 3 sections and the second annex will be a Module 3 mock-up.

For Assistance with Registering a Herbal Product in the UK, EU or USA Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…


  1. (required)


  2. (valid email required)

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA SME Workshop, Scientific and Regulatory Advice

EMA SME Workshop, Scientific and Regulatory Advice

Full Text Here

26/05/2011 – 26/05/2011

The workshop presents the support available to SMEs during early drug development and highlight recent experience in quality, non-clinical and clinical scientific advice. In addition, the workshop covers regulatory support available prior to submission of an application for marketing authorisation.

For Assistance with Obtaining Scientific Advice or Regulatory Advice from EMA Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…


  1. (required)


  2. (valid email required)

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA Publish Reflection Paper on Stability Testing for Herbal Medicinal Products

EMA Publish Reflection Paper on Stability Testing for Herbal Medicinal Products.

Full Text Here

This reflection paper addresses the need for specific requirements for establishing the stability of herbal medicinal products (HMPs). The quality, including the stability, of HMPs should be guaranteed and demonstrated in accordance with the existing requirements as set out in Annex I of Directive 2001/83/EC, as amended, Annex I of Directive 2001/82/EC, as amended and with current EU/ICH guidance on quality. The committees of the European Medicines Agency have published several quality guidelines related to stability testing, which focus mainly on chemically defined substances. In view of the complex nature of HMPs, it is considered that further guidance is needed in order to ensure that stability of these products is addressed appropriately. The purpose of this reflection paper is to consider issues relating to the application of the existing stability guidance on HMPs and to provide additional guidance where necessary.

For Assistance with Developing and Registering Herbal Products Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…


  1. (required)


  2. (valid email required)

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA Publish Procedural Advice on Certification of Quality and Non-Clinial Data for ATMP

EMA Publish Procedural Advice on Certification of Quality and Non-Clinial Data for ATMP

Full Text Here

Article 18 of Regulation (EC) No 1394/20071 provides that Small and Medium-sized Enterprises (SMEs) developing an Advanced Therapy Medicinal Product (ATMP) may submit to the European Medicines Agency (EMA) all relevant quality and, where available, non-clinical data required in accordance with modules 3 and 4 of Annex I to Directive 2001/83/EC2 (as amended) on the Community code relating to medicinal products for human use, for scientific evaluation and certification.  Provisions for the evaluation and certification of such data are laid down by the Commission in Regulation (EC) No 668/20093.
This document gives guidance and describes the procedures, timelines and practical steps to be followed by the applicants and the EMA for the submission, evaluation of a certification application and if applicable the issuing of the certificate.

For Assistance with the Regulation and Development of Advanced Therapies Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…



  1. (required)



  2. (valid email required)


“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA Publish Concept Paper on Storage Conditions During Transport

EMA Publish Concept Paper on Storage Conditions During Transport.

Full text Here

The globalisation of the manufacture of human and veterinary medicinal products has brought both benefits and a wide range of challenges. This paper is concerned with challenges related to the maintenance of appropriate transit conditions during transport as products move through all stages of the manufacturing and wholesale distribution system from active substance through the wholesale distribution system to ensure that patients and animals receive safe and efficacious medicinal products. The current guidance (CPMP/QWP/609/96/Rev2) was written in 1996 and revised in 2003, during this time significant changes continued to occur in the globalisation of manufacture with a consequent increase in the complexity and vulnerability in the supply chain.

There is a lack of clear guidance on the regulatory expectations for ensuring that medicinal products and APIs are not damaged during transportation. In order that products are fit for their intended purpose, including the ability to tolerate the range of expected storage conditions during use by patients or use in animals, simple and risk-based guidance is required for the transport. Such guidance needs to cover cold chain and non-cold chain products and all of the different stages of manufacture, importation and distribution.

For Assistance with CMC Issues Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

Fill Out the Short Form Below…



  1. (required)



  2. (valid email required)


“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, The European Drug Regulator, Publishes Concept Paper on the Revision of the Note for Guidance on the Quality of Modified Release Oral Dosage Forms and Transdermal Dosage Forms: Section I (Quality)

EMA, The European Drug Regulator, Publishes Concept Paper on the Revision of the Note for Guidance on the Quality of Modified Release Oral Dosage Forms and Transdermal Dosage Forms: Section I (Quality)

Full Text here

The Note for Guidance on modified release products addresses specific quality requirement for modified release products, particularly the in vitro testing. The NfG focus primarily on oral dosage forms including both prolonged and delayed release formulations. However there is room for further guidance particularly in relation to the choice of the appropriate dissolution test and media, details on the development of in vivo/in vitro correlation and new technologies. The requirements for transdermal dosage forms are only briefly described in the document. As such, a more elaborated chapter on transdermal patches is needed in this guideline with special focus on the interchangeability aspects for generic transdermal patches.

Oral formulations are still the most common pharmaceutical dosage form, even among the newly introduced drugs, and will probably continue to be in the next few years. Several Modified Release (MR) technologies (e.g., hydrophilic matrix tablets, osmotic systems, and coated multiparticulates) are well established and well understood.
In vitro dissolution test is often used during development and quality control as a predictive tool to indicate changes which may have an effect on the efficacy or safety of the product and can also be used as a surrogate for in vivo testing when in vitro-in vivo correlations are developed. However the estimation of the release and dissolution of the drug in the intestinal fluid (and the permeation of the intestinal mucosa) with in vitro techniques that are not biorelevant can be misleading in some cases according to the properties of the drug and the formulation. At the same time, developments in formulation and specific types of excipients have led to more complicated MR dosage forms. On the other hand, new drug substances rarely exhibit good solubility (classified as Class II compounds in BCS) and their development as Modified Release forms can be challenging in terms of achievement of in vivo and/or in vitro release. MR products can also present unique challenges when it comes to establishing therapeutic equivalence between two formulations.
In addition, the new quality paradigm –Quality by Design- and new technologies in this respect (e.g. PAT) can be used to provide in vitro-in vivo relationships based on the performance of individual dosage form units, or to set up dissolution specifications.
As a result of the interaction of alcohol with modified release oral dosage forms containing strong opioids which lead to “dose dumping” of opioids for some (generic) products, it has become necessary to review the requirements for in vitro or in vivo data for all modified release products.
Taking into account the above it is deduced that there is a need to provide further guidance and elaborate the specific requirements in relation to these points.
Following recent scientific developments and increased number of transdermal patches applications for marketing authorisation, it has become necessary to further illustrate the specific requirements for this dosage form. Although section four of the current guideline attempts to describe the requirements for transdermal dosage forms, clear guidance on limits, e.g., for patch size and drug load versus total amount released are not specified. In addition, the specific requirements for dissolution methods for transdermal patches should be described in greater detail. Finally, skin adhesion properties are not fully addressed and no mention is made to the in vitro methodologies and in vivo testing to assess and control skin adhesion. Especially the concept of generics and interchangeability of transdermal patch formulations has generated the need for further clarification and guidance. The issue regarding interchangeability should be discussed in a separate subsection of this guideline since the objectives of pharmaceutical development differ for those type of products. A patch formulation using an active substance for the first time can hardly be evaluated based on certain standard requirements, although the methods applied to establish and describe the patch formulations characteristics should be based in general on standardized procedures. In contrast to this individual case evaluation, generic patch development needs to be focussed on comparability aspects. Certain standard requirements have to be established to facilitate the decision making process when it comes to evaluation of product equivalence between originator and the generic patch formulation. Due to the particularities of the transdermal route and its underlying principles of release from the formulation, interchangeability of transdermal patch products is not solely depending on the proof of bioequivalence as known for oral modified release products. Adhesion as well as skin irritation properties might have a significant impact on the in-vivo release characteristics of a patch formulation.

For CMC Consulting Services Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

First name

Phone #1

E-mail address

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, The European Drug Regulator, Publishes a Concept Paper on the Revision of the Note for Guidance on Quality or Modified Release Oral Dosage Forms and Transdermal Dosage Forms: Section 1 (Quality)

EMA, The European Drug Regulator, Publishes a Concept Paper on the Revision of the Note for Guidance on Quality or Modified Release Oral Dosage Forms and Transdermal Dosage Forms: Section 1 (Quality)

Full Text Click Here

The Note for Guidance on modified release products addresses specific quality requirement for modified release products, particularly the in vitro testing. The NfG focus primarily on oral dosage forms including both prolonged and delayed release formulations. However there is room for further guidance particularly in relation to the choice of the appropriate dissolution test and media, details on the development of in vivo/in vitro correlation and new technologies. The requirements for transdermal dosage forms are only briefly described in the document. As such, a more elaborated chapter on transdermal patches is needed in this guideline with special focus on the interchangeability aspects for generic transdermal patches.

Oral formulations are still the most common pharmaceutical dosage form, even among the newly introduced drugs, and will probably continue to be in the next few years. Several Modified Release (MR) technologies (e.g., hydrophilic matrix tablets, osmotic systems, and coated multiparticulates) are well established and well understood.
In vitro dissolution test is often used during development and quality control as a predictive tool to indicate changes which may have an effect on the efficacy or safety of the product and can also be used as a surrogate for in vivo testing when in vitro-in vivo correlations are developed. However the estimation of the release and dissolution of the drug in the intestinal fluid (and the permeation of the intestinal mucosa) with in vitro techniques that are not biorelevant can be misleading in some cases according to the properties of the drug and the formulation. At the same time, developments in formulation and specific types of excipients have led to more complicated MR dosage forms. On the other hand, new drug substances rarely exhibit good solubility (classified as Class II compounds in BCS) and their development as Modified Release forms can be challenging in terms of achievement of in vivo and/or in vitro release. MR products can also present unique challenges when it comes to establishing therapeutic equivalence between two formulations.
In addition, the new quality paradigm –Quality by Design- and new technologies in this respect (e.g. PAT) can be used to provide in vitro-in vivo relationships based on the performance of individual dosage form units, or to set up dissolution specifications.
As a result of the interaction of alcohol with modified release oral dosage forms containing strong opioids which lead to “dose dumping” of opioids for some (generic) products, it has become necessary to review the requirements for in vitro or in vivo data for all modified release products.
Taking into account the above it is deduced that there is a need to provide further guidance and elaborate the specific requirements in relation to these points.

Following recent scientific developments and increased number of transdermal patches applications for marketing authorisation, it has become necessary to further illustrate the specific requirements for this dosage form. Although section four of the current guideline attempts to describe the requirements for transdermal dosage forms, clear guidance on limits, e.g., for patch size and drug load versus total amount released are not specified. In addition, the specific requirements for dissolution methods for transdermal patches should be described in greater detail. Finally, skin adhesion properties are not fully addressed and no mention is made to the in vitro methodologies and in vivo testing to assess and control skin adhesion. Especially the concept of generics and interchangeability of transdermal patch formulations has generated the need for further clarification and guidance. The issue regarding interchangeability should be discussed in a separate subsection of this guideline since the objectives of pharmaceutical development differ for those type of products. A patch formulation using an active substance for the first time can hardly be evaluated based on certain standard requirements, although the methods applied to establish and describe the patch formulations characteristics should be based in general on standardized procedures. In contrast to this individual case evaluation, generic patch development needs to be focussed on comparability aspects. Certain standard requirements have to be established to facilitate the decision making process when it comes to evaluation of product equivalence between originator and the generic patch formulation. Due to the particularities of the transdermal route and its underlying principles of release from the formulation, interchangeability of transdermal patch products is not solely depending on the proof of bioequivalence as known for oral modified release products. Adhesion as well as skin irritation properties might have a significant impact on the in-vivo release characteristics of a patch formulation.

For CMC Consulting Services Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

First name

Phone #1

E-mail address

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, The European Drug Regulator, Published a Concept Paper on the Revision of the Note for Guidance on Quality of Modified Release Oral Dosage Forms and Transdermal Dosage Forms: Section I (Quality)

EMA, The European Drug Regulator, Published a Concept Paper on the Revision of the Note for Guidance on Quality of Modified Release Oral Dosage Forms and Transdermal Dosage Forms: Section I (Quality)

Full Text Here

The Note for Guidance on modified release products addresses specific quality requirement for modified release products, particularly the in vitro testing. The NfG focus primarily on oral dosage forms including both prolonged and delayed release formulations. However there is room for further guidance particularly in relation to the choice of the appropriate dissolution test and media, details on the development of in vivo/in vitro correlation and new technologies. The requirements for transdermal dosage forms are only briefly described in the document. As such, a more elaborated chapter on transdermal patches is needed in this guideline with special focus on the interchangeability aspects for generic transdermal patches.

Oral formulations are still the most common pharmaceutical dosage form, even among the newly introduced drugs, and will probably continue to be in the next few years. Several Modified Release (MR) technologies (e.g., hydrophilic matrix tablets, osmotic systems, and coated multiparticulates) are well established and well understood.
In vitro dissolution test is often used during development and quality control as a predictive tool to indicate changes which may have an effect on the efficacy or safety of the product and can also be used as a surrogate for in vivo testing when in vitro-in vivo correlations are developed. However the estimation of the release and dissolution of the drug in the intestinal fluid (and the permeation of the intestinal mucosa) with in vitro techniques that are not biorelevant can be misleading in some cases according to the properties of the drug and the formulation. At the same time, developments in formulation and specific types of excipients have led to more complicated MR dosage forms. On the other hand, new drug substances rarely exhibit good solubility (classified as Class II compounds in BCS) and their development as Modified Release forms can be challenging in terms of achievement of in vivo and/or in vitro release. MR products can also present unique challenges when it comes to establishing therapeutic equivalence between two formulations.
In addition, the new quality paradigm –Quality by Design- and new technologies in this respect (e.g. PAT) can be used to provide in vitro-in vivo relationships based on the performance of individual dosage form units, or to set up dissolution specifications.
As a result of the interaction of alcohol with modified release oral dosage forms containing strong opioids which lead to “dose dumping” of opioids for some (generic) products, it has become necessary to review the requirements for in vitro or in vivo data for all modified release products.
Taking into account the above it is deduced that there is a need to provide further guidance and elaborate the specific requirements in relation to these points.
Following recent scientific developments and increased number of transdermal patches applications for marketing authorisation, it has become necessary to further illustrate the specific requirements for this dosage form. Although section four of the current guideline attempts to describe the requirements for transdermal dosage forms, clear guidance on limits, e.g., for patch size and drug load versus total amount released are not specified. In addition, the specific requirements for dissolution methods for transdermal patches should be described in greater detail. Finally, skin adhesion properties are not fully addressed and no mention is made to the in vitro methodologies and in vivo testing to assess and control skin adhesion. Especially the concept of generics and interchangeability of transdermal patch formulations has generated the need for further clarification and guidance. The issue regarding interchangeability should be discussed in a separate subsection of this guideline since the objectives of pharmaceutical development differ for those type of products. A patch formulation using an active substance for the first time can hardly be evaluated based on certain standard requirements, although the methods applied to establish and describe the patch formulations characteristics should be based in general on standardized procedures. In contrast to this individual case evaluation, generic patch development needs to be focussed on comparability aspects. Certain standard requirements have to be established to facilitate the decision making process when it comes to evaluation of product equivalence between originator and the generic patch formulation. Due to the particularities of the transdermal route and its underlying principles of release from the formulation, interchangeability of transdermal patch products is not solely depending on the proof of bioequivalence as known for oral modified release products. Adhesion as well as skin irritation properties might have a significant impact on the in-vivo release characteristics of a patch formulation.

Click Here for a Drug Development Expert

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

First name

Phone #1

E-mail address

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, the European Drug Regulators, Publish Draft Guidance on Non-Clinical and Clinical Aspects of Medical Products Containing Geneticaly Modified Cells

EMA, the European Drug Regulators, Publish Draft Guidance on Non-Clinical and Clinical Aspects of Medical Products Containing Genetically Modified Cells.

Full Text Here

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans. Its focus is on the quality, safety and efficacy requirements of genetically modified cells developed as medicinal products.

Genetically modified cells may be developed either for therapeutic use (gene therapy medicinal products) or to use the genetic modification in the manufacturing process of a cell therapy / tissue engineering product.
The following are some examples of medicinal products containing genetically modified cells (GMC) that have been used in clinical trials:

genetically modified cells for treatment of monogeneic inherited disease;

genetically modified dendritic cells and cytotoxic lymphocytes for cancer immunotherapy;

genetically modified autologous chondrocytes for cartilage repair; genetically modified progenitor  cells for cardio-vascular disease treatment or for in vivo marking studies, particularly for in vivo biodistribution or in vivo differentiation analysis;

genetically modified osteogenic cells for bone fractures repair; genetically modified cells for 66 infectious disease treatment.
This guideline defines scientific principles and provides guidance to applicants developing medicinal products containing genetically modified cells. It is recognised that this is an area under constant development and guidance should be applied to any novel procedures as appropriate.

Click Here for Biotechnology Development Planning Services

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

First name

Phone #1

E-mail address

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, the European Drug Regulator Publishes Reflection paper on Quality, Non-Clinical and Clinical Issues related to the Development of Recombinant Adeno-Associated Viral Vectors

EMA, the European Drug Regulator Publishes Reflection paper on Quality, Non-Clinical and Clinical Issues related to the Development of Recombinant Adeno-Associated Viral Vectors

Full Text Here

Recombinant adeno-associated viral (rAAV) vectors are derived from the single stranded DNA virus adeno-associated virus which belongs to the genus dependovirus within the Parvoviridae family. As the name suggests the wild type virus is incapable of independent replication and relies on co-infection of a helper virus to enable a lytic replication cycle (Gonclaves, 2005). Adenovirus (Ad), herpes simplex virus (HSV), pseudorabies virus (PrV) and human papilloma virus (HPV) are known to support wild type AAV replication.
Infection with wild-type AAV is not associated with any pathogenic disease, and in the absence of a helper virus co-infection, the virus may integrate into the host cell genome or remain as an extrachromosomal form (Schnepp, 2005). In both situations the virus appears to remain latent. In-vitro studies suggest that wild-type viral DNA integration can occur occasionally in a site specific manner (19q13.3) (Kotin, 1990 and 1991 and 1992), but only at very high multiplicities of infection (Hüser, 2002), and this was originally considered to be a safety feature of vectors derived from this virus. However, it has been subsequently shown that site specific integration is dependent on the presence of both the inverted terminal repeats (ITR) and the Rep gene products (Weitzman, 1994; Linden, 1996), the latter of which is not present in rAAV; as such the site specific integration feature of these vectors is lost. The level of integration of DNA into the cellular chromosome in in-vivo models, however remains contentious. Nonetheless, long term protein expression (in-vivo) from the gene of interest inserted into rAAV vectors has been observed (Flotte, 1993; Kaplitt, 1994; Conrad, 1996; Monahan, 1998; Donahue, 1999; Stieger, 2006), even in the absence of identifiable genetic integration (Miller, 2004; Song, 2004; Flotte, 1994). This persistence is thought to be derived from stable concatemerized duplex genome forms (circular or linear molecules) that are transcriptionally active (Duan, 1998; Yang, 1999; Fisher, 1997).
Examples of diseases studied include haemophilia B (Manno, 2006 and 2003), cystic fibrosis (Flotte, 2003), Parkinson’s disease (Kaplitt, 2007), rheumatoid arthritis (www.targen.com [tgAAC94]), Leber’s congenital amaurosis (Bainbridge, 2008; Maguire, 2008; Jacobson, 2006), infantile neuronal ceroid lipofuscinosis (Worgall, 2008) and muscular dystrophy (Xiao, 2000). Furthermore non-clinical studies indicate rAAV expressing heterologous antigenic sequences (HPV16 – Kuck, 2006; HIV – Xin, 2001 and 2002; SIV – Johnson, 2005; malaria – Logan, 2007) can illicit both humoral and cellular immune responses, and modest immunogenicity has been reported in a phase I/II study using rAAV2 encoding HIV antigens (Mehendal, 2008). However, it has been suggested that cellular responses to the transgene products of rAAV vectors may be impaired (Lin, 2007), as such the utility of these vectors when used for prophylactic purposes needs further investigation.
There are currently 6 confirmed serotypes of adeno-associated virus (AAV-1 to -6) and 2 tentative species (AAV-7 and 8) (source: International Committee on Taxonomy of Viruses [ICTV]). However there are a number of publications describing additional serotypes (i.e. 9 and 10) which are currently not recognized by the ICTV. It is likely therefore, that there are significantly more serotypes circulating that have currently not been formally identified or recognized (Pacak, 2006; Limberis, 2006; Gao, 2004). Nonetheless, the majority of the 67 clinical trials undertaken to date using rAAV for gene delivery have used serotype 2 (Gene Therapy Clinical Trials Worldwide. J. Gene Med. March 2009 Update, http://www.wiley.co.uk/genmed/clinical ).
Evidence is accumulating which suggests that different AAV serotypes may have different tissue tropisms, for example AAV-8 is suggested to have a preferred tropism to the liver (Davidoff, 2005), while for AAV-1, -6 and -7 the preferred tropism is to skeletal muscle (Duan, 2001; Chao, 2000), AAV-4 is highly specific to the retinal pigmented epithelial cells in several animal species (Weber, 2003) and the ependymal cells (Zabner, 2000) and AAV-9 is described as being tropic to cardiacmuscle (Pacak, 2006), thought it also tranduces liver (Van den Driessche, 2007) and brain (Foust, 2009). Vectors based on these serotypes, in-vitro selected AAV with altered tropisms and hybrid vectors (i.e. ITR and Rep from AAV-2, Cap (protein coat) from another serotype i.e. 8) are being investigated (in-vitro and in animal models) to evaluate further the utility of the preferred tropisms and their potential for avoiding pre-existing immunity to AAV-2.
A new development in the field of AAV vectors is the use of self complementary (sc) AAV. Conventional rAAV vectors require 2nd strand synthesis before genes can be expressed, and it is theorized that scAAV bypass this step by delivering a duplex genome. This is achieved by deleting the nicking site of one ITR so that it no longer serves as a replication origin but still forms an AAV hairpin structure. The result is a single stranded, dimeric inverted repeat genome with the altered ITR sequence situated in the middle of the molecule and a wild-type ITR at each end. Following infection and uncoating, the DNA is folded to form a double stranded molecule. A closed hairpin end is formed from the altered ITR, and an open end formed from the two wild-type ITR’s, thus mimicking the structure of a single stranded rAAV after 2nd strand synthesis (McCarty, 2003). It is anticipated that such vectors will improve transduction efficiency and improve the level of protein expression from the transgene. The coding capacity of these vectors, however, is reduced by a factor of two.
Given the basic biology of the ‘parent’ virus as described above, the methods for manufacture and quality control of product are complicated, and the long-term fate of the administered vector is at present unknown. There are a number of manufacturing strategies that can be used to produce rAAV vectors and these are discussed further below, however the basic functional requirements for manufacture are:

The AAV ITR’s flanking the ‘gene of interest’ (this construct contains the cis elements necessary for packaging and replication of its single stranded DNA genome).

Genetic sequences (Rep and Cap) necessary for AAV replication and viral capsid proteins (generally provided in trans within a plasmid or in a packaging cell line).

Helper virus functions: either co-infection of the helper virus or co-transfection/infection of a plasmid/chimeric virus encoding the helper genes (adenovirus: E1a/1b, E2a, E4orf6, VA1 RNA; herpes simplex virus: UL5, UL8, UL52 and UL29).

A cell line capable of supporting helper virus and AAV replication.
The aim of this paper is to discuss quality, non-clinical and clinical issues that should be considered during the development of medicinal products derived from AAV, and to indicate requirements that might be expected the time of a market authorisation application (MAA). The issues raised are specific only to the development of rAAV vectors as medicinal products; general requirements for MAA are not within the scope of this paper. It is recommended that this paper is read in conjunction with the guidance documents referenced in section 4.2.

For Drug Development Services Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

First name

Phone #1

E-mail address

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, the European Drug Regulator publishes, Draft Guideline on Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.

EMA, the European Drug Regulator publishes, Draft Guideline on Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.

Full text Here

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans. Its focus is on the quality, safety and efficacy requirements of genetically modified cells developed as medicinal products.

Genetically modified cells may be developed either for therapeutic use (gene therapy medicinal products) or to use the genetic modification in the manufacturing process of a cell therapy / tissue engineering product.
The following are some examples of medicinal products containing genetically modified cells (GMC) that have been used in clinical trials:
− genetically modified cells for treatment of monogeneic inherited disease;
− genetically modified dendritic cells and cytotoxic lymphocytes for cancer immunotherapy;
− genetically modified autologous chondrocytes for cartilage repair; genetically modified progenitor cells for cardio-vascular disease treatment or for in vivo marking  studies, particularly for in vivo biodistribution or in vivo differentiation analysis;
− genetically modified osteogenic cells for bone fractures repair; genetically modified cells for  infectious disease treatment.
This guideline defines scientific principles and provides guidance to applicants developing medicinal products containing genetically modified cells. It is recognised that this is an area under constant development and guidance should be applied to any novel procedures as appropriate.

For Biotechnology Development Services Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

First name

Phone #1

E-mail address

ida consultants freestrategyconsultation 515x64 EMA, the European Drug Regulator publishes, Draft Guideline on Quality, non clinical and clinical aspects of medicinal products containing genetically modified cells.

Free Strategy Consultation - Biotech Pharma Regualtory

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

ida 100programme 515x64 LowRes EMA, the European Drug Regulator publishes, Draft Guideline on Quality, non clinical and clinical aspects of medicinal products containing genetically modified cells.

Drug Regulators, EMA (EMEA), Publish Reflection Paper on in-vitro Cultured Chondrocyte Containing Products for Cartilage Repair of the Knee

Drug Regulators, EMA (EMEA), Publish Reflection Paper on in-vitro Cultured Chondrocyte Containing Products for Cartilage Repair of the Knee

Full Text Here

This reflection paper addresses specific points related to medicinal products containing in vitro cultured autologous chondrocytes intended for the repair of cartilage lesions of the knee. This reflection paper is considered to supplement the ‘Guideline on human cell-based medicinal products’ (EMEA/CHMP/410869/2006) and therefore it should be read in conjunction with the guideline.