excerpts from guidance

full text here

This guideline defines key elements necessary for the validation of bioanalytical methods. The guideline focuses on the validation of the bioanalytical methods generating quantitative concentration data used for pharmacokinetic and toxicokinetic parameter determinations. Guidance and criteria are given on the application of these validated methods in the routine analysis of study samples from animal and human studies.

Measurement of drug concentrations in biological matrices (such as serum, plasma, blood, urine, and saliva) is an important aspect of medicinal product development. Such data may be required to support applications for new actives substances and generics as well as variations to authorised drug products. The results of animal toxicokinetic studies and of clinical trials, including bioequivalence studies are used to make critical decisions supporting the safety and efficacy of a medicinal drug substance or product. It is therefore paramount that the applied bioanalytical methods used are well characterised, fully validated and documented to a satisfactory standard in order to yield reliable results.
Acceptance criteria wider than those defined in this guideline may be used in special situations. This should be prospectively defined based on the intended use of the method.

Full Text Here

This guideline provides guidance on the clinical development of compounds for the treatment of Chronic Hepatitis C (CHC), including directly acting antivirals (DAAs) as well as host targeting antivirals (HTA). It should be read in conjunction with updated and recognised clinical treatment guidelines. Various combination regimens, including a DAA or HTA together with peginterferon (pegIFN) and ribavirin, regimens with more than one DAA/HTA in combination with pegIFN + ribavirin, as well as regimens excluding either or both of these agents, are considered.
While the primary investigation of new DAA/HTA in combination with pegIFN + ribavirin in patients with genotype (GT) 1 remains important, it is recognised that other paths of drug development, focussing on wider or alternative populations, or other drug combinations (such as more than one DAA/HTA with or without ribavirin, or 2 DAA/HTAs in combination with pegIFN+ribavirin) are warranted and ongoing.
The guidelines emphasize the importance of new DAA/HTA for usage in special populations including patients with decompensated liver disease, patients pre/post transplantation, HCV/HIV co-infected patients, patients intolerant to pegIFN and/or ribavirin and patients with prior DAA experience.
When studying novel agents in combination with pegIFN and ribavirin, the comparator in pivotal trials should be a licensed first line recommended regimen; notwithstanding this, European regulators recognise the need for licensed DAA/HTAs from several classes, with different side effects profiles and resistance patterns, which is seen as a benefit per se. When studying novel drug combinations without pegIFN, it is recommended that patients previously failing therapy with pegIFN+ribavirin that do not have an immediate treatment need be avoided prior to obtaining proof-of-concept of sustained virological response (SVR), as the consequences of acquired drug resistance in terms of retreatment success has still not been investigated. For drugs to be used in combinations eschewing pegIFN, it is recognised that patients that do not tolerate pegIFN have no presently licensed therapeutic options and a probability of viral clearance close to zero. Thus, for licensure, response rates would be weighed in relation to this fact. Regarding special populations, the need to start trials as early as can safely be done for groups with an important unmet medical need (e.g., patients with decompensated liver disease or HCV/HIV coinfection) is emphasised.
Since the previous guidelines were adapted, host IL-28B genotype has emerged as a very important predictor of the efficacy of pegIFN, and it is recommended that stratification by IL28B genotype be employed whenever the studied drug regimen includes this drug.
Regarding future developments, proof-of-concept of SVR with treatment combination excluding pegIFN, as well as data on retreatment of patients that have failed therapy that has selected for DAA-resistant variants, but whose dominant population has subsequently reverted to wild-type, are eagerly awaited. Such data are likely to greatly impact regulatory considerations within the field. It is recognised that this is a rapidly moving therapeutic area, and that a further revision of these guidelines may be mandated within the foreseeable future.

For Assistance with Developing Hep C 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.

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“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.”

Full Text Here

This guideline provides guidance on the clinical development of compounds for the treatment of Chronic Hepatitis C (CHC), including directly acting antivirals (DAAs) as well as host targeting antivirals (HTA). It should be read in conjunction with updated and recognised clinical treatment guidelines. Various combination regimens, including a DAA or HTA together with peginterferon (pegIFN) and ribavirin, regimens with more than one DAA/HTA in combination with pegIFN + ribavirin, as well as regimens excluding either or both of these agents, are considered.
While the primary investigation of new DAA/HTA in combination with pegIFN + ribavirin in patients with genotype (GT) 1 remains important, it is recognised that other paths of drug development, focussing on wider or alternative populations, or other drug combinations (such as more than one DAA/HTA with or without ribavirin, or 2 DAA/HTAs in combination with pegIFN+ribavirin) are warranted and ongoing.
The guidelines emphasize the importance of new DAA/HTA for usage in special populations including patients with decompensated liver disease, patients pre/post transplantation, HCV/HIV co-infected patients, patients intolerant to pegIFN and/or ribavirin and patients with prior DAA experience.
When studying novel agents in combination with pegIFN and ribavirin, the comparator in pivotal trials should be a licensed first line recommended regimen; notwithstanding this, European regulators recognise the need for licensed DAA/HTAs from several classes, with different side effects profiles and resistance patterns, which is seen as a benefit per se. When studying novel drug combinations without pegIFN, it is recommended that patients previously failing therapy with pegIFN+ribavirin that do not have an immediate treatment need be avoided prior to obtaining proof-of-concept of sustained virological response (SVR), as the consequences of acquired drug resistance in terms of retreatment success has still not been investigated. For drugs to be used in combinations eschewing pegIFN, it is recognised that patients that do not tolerate pegIFN have no presently licensed therapeutic options and a probability of viral clearance close to zero. Thus, for licensure, response rates would be weighed in relation to this fact. Regarding special populations, the need to start trials as early as can safely be done for groups with an important unmet medical need (e.g., patients with decompensated liver disease or HCV/HIV coinfection) is emphasised, Since the previous guidelines were adapted, host IL-28B genotype has emerged as a very important predictor of the efficacy of pegIFN, and it is recommended that stratification by IL28B genotype be employed whenever the studied drug regimen includes this drug.
Regarding future developments, proof-of-concept of SVR with treatment combination excluding pegIFN, as well as data on retreatment of patients that have failed therapy that has selected for DAA-resistant variants, but whose dominant population has subsequently reverted to wild-type, are eagerly awaited. Such data are likely to greatly impact regulatory considerations within the field. It is recognised that this is a rapidly moving therapeutic area, and that a further revision of these guidelines may be mandated within the foreseeable future.

For Assistance with Planning The Development of Hepatitis C 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.

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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.

“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.”

Full Text Here

This guideline provides guidance on the clinical development of compounds for the treatment of Chronic Hepatitis C (CHC), including directly acting antivirals (DAAs) as well as host targeting antivirals (HTA). It should be read in conjunction with updated and recognised clinical treatment guidelines. Various combination regimens, including a DAA or HTA together with peginterferon (pegIFN) and ribavirin, regimens with more than one DAA/HTA in combination with pegIFN + ribavirin, as well as regimens excluding either or both of these agents, are considered.
While the primary investigation of new DAA/HTA in combination with pegIFN + ribavirin in patients with genotype (GT) remains important, it is recognised that other paths of drug development, focussing on wider or alternative populations, or other drug combinations (such as more than one DAA/HTA with or without ribavirin, or DAA/HTAs in combination with pegIFN+ribavirin) are warranted and ongoing.
The guidelines emphasize the importance of new DAA/HTA for usage in special populations including patients with decompensated liver disease, patients pre/post transplantation, HCV/HIV co-infected patients, patients intolerant to pegIFN and/or ribavirin and patients with prior DAA experience.
When studying novel agents in combination with pegIFN and ribavirin, the comparator in pivotal trials should be a licensed first line recommended regimen; notwithstanding this, European regulators recognise the need for licensed DAA/HTAs from several classes, with different side effects profiles and resistance patterns, which is seen as a benefit per se. When studying novel drug combinations without pegIFN, it is recommended that patients previously failing therapy with pegIFN+ribavirin that do not have an immediate treatment need be avoided prior to obtaining proof-of-concept of sustained virological response (SVR), as the consequences of acquired drug resistance in terms of retreatment success has still not been investigated. For drugs to be used in combinations eschewing pegIFN, it is recognised that patients that do not tolerate pegIFN have no presently licensed therapeutic options and a probability of viral clearance close to zero. Thus, for licensure, response rates would be weighed in relation to this fact. Regarding special populations, the need to start trials as early as can safely be done for groups with an important unmet medical need (e.g., patients with decompensated liver disease or HCV/HIV coinfection) is emphasised,
Since the previous guidelines were adapted, host IL-28B genotype has emerged as a very important predictor of the efficacy of pegIFN, and it is recommended that stratification by IL28B genotype be employed whenever the studied drug regimen includes this drug.
Regarding future developments, proof-of-concept of SVR with treatment combination excluding pegIFN, as well as data on retreatment of patients that have failed therapy that has selected for DAA-resistant variants, but whose dominant population has subsequently reverted to wild-type, are eagerly awaited. Such data are likely to greatly impact regulatory considerations within the field. It is recognised that this is a rapidly moving therapeutic area, and that a further revision of these guidelines may be mandated within the foreseeable future.

For Assistance with Clinical Development Planning and Regulatory Strategy 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.

“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.”

Full Text Here

Measurement of drug concentrations in biological matrices is an important aspect of medicinal product development for those products containing new active substances as well as for line extensions and generic products. Such data may be required to support new applications as well as variations to authorised drug products. The results of toxicokinetic, pharmacokinetic and bioequivalence studies are used to make critical decisions supporting the safety and efficacy of a medicinal drug substance or product. It is therefore paramount that the applied bioanalytical methods used are well characterised, fully validated and documented to a satisfactory standard in order to yield reliable results.
Acceptance criteria wider than those defined in this guideline may need to be used in special situations, such as analysis of complex matrices (e.g. solid tissues), when usual acceptance criteria cannot be met. This should be justified and prospectively defined.

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology) and regulatory consultant, we work with our 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.

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“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.”

Full Press Release Here

This communication is intended to inform all interested parties about several important changes the European Medicines Agency will be introducing shortly

New visual identity

On 8 December 2009, The EMEA will officially launch our new visual identity, comprising a new logo, new colour chart, new typography and rebranded materials based on these elements. The EMEA are giving you advance notice of this so that you are not surprised when you start to see new documents and other materials emerging from the Agency that do not bear the familiar ‘EMEA’ logo. The EMEA have created the new visual identity as part of a wider effort to improve the quality and consistency of our communications with partners, stakeholders and the public. The main benefit is that our communications materials will now be based on professionally designed templates, and have a more harmonised look and feel than our current materials. The cornerstone of their new identity is a new logo they have created that reflects more accurately the nature and character of the Agency, which has evolved significantly in the 15 years since it, and the original logo, were created. Please visit there public website on 8 December for further details about our new visual identity and materials.

Still the European Medicines Agency, but no longer ‘the EMEA’ – and not ‘the EMA’
either

While you may be familiar with us as ‘the EMEA’, many of their partners and stakeholders over the years have told tjhem they find the acronym confusing, firstly because it does not accurately reflect their name (“What does the second ‘e’ stand for?”) and secondly because it is an acronym widely used in the business community to mean ‘Europe, Middle East and Africa’, which can cause some confusion. Since it is important to them that they communicate clear and unambiguous messages about who they are, they have decided that they will no longer be using the EMEA acronym in their communications, and it does not feature in there new logo. Please note that they will not be calling ourselves ‘the EMA’ either. Although this may seem a more obvious acronym, it is not one that feels right for them yet. They may reconsider our position at a later stage, if ‘EMA’ evolves naturally into a commonly accepted and widely used shorthand for our organisation. Until then, however, they will be using only their full name (or ‘the Agency’, for short) in our communications. (For technical reasons, they need to use some kind of abbreviation in our document references and our website and e-mail addresses. They will exceptionally use ‘EMA’ in these defined cases.)

New ‘ema.europa.eu’ address for their website and e-mails

On 8 December 2009, all Agency website and e-mail addresses will change from ‘emea.europa.eu’ to ‘ema.europa.eu’, as a consequence of there decision to discontinue our use of the acronym ‘EMEA’. From that date onwards, the address of our public website will be www.ema.europa.eu and our e-mail addresses will take the form name.surname@ema.europa.eu Please update your website bookmarks, address books, contacts databases and other resources accordingly.

New organisation chart

Over the past three months, they have been implementing a series of changes to our internal organisation, aimed at improving the functioning of the Agency and the way in which they deliver their core tasks. The new structure and allocation of responsibilities will be made public on 8 December 2009. Key changes relate to the two units responsible for medicines for human use that have been re-formed and re-named to become ‘Human Medicines Development and Evaluation’ (formerly ‘Pre-authorisation Unit’) and ‘Patient Health Protection’ (formerly ‘Post-authorisation Unit’). Staff members dealing with veterinary medicines have been re-grouped in one single sector responsible for all areas of veterinary medicines. In addition, a single sector has also been formed to manage product data and documentation related to applications for both human and veterinary medicines. Overall, the changes to our organisational structure will ensure more accountability at various management levels, and will allow them to achieve greater efficiency and effectiveness in our core activities. Further information is available in a press release published on 5 October 2009.

New public website (in early 2010)

A further, major initiative they are undertaking as part of our efforts to improve our communications is to completely redesign there public website (currently at  www.emea.europa.eu). Scheduled for launch in early 2010, their new website will offer much-improved navigation and search functionality, and content will be structured more logically and intuitively. It will also be compliant with their new visual identity. In designing the new website, they have carefully considered the feedback they have received from you, their partners and stakeholders, through surveys and other channels over the past two years. Thanks to your input, they are confident that their  redesigned website will overcome many of the weaknesses identified with the current site, and will offer a greatly improved user-experience for all Agency audiences.
Damien Bové works as a drug development consultant (pharmaceutical or biotechnology) and regulatory consultant, we work with our 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.

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“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.”

full text here

Interested party (Organisations or individuals) that commented on the draft Guideline as released for consultation

Stakeholder No.                Name of Organisation or individual
1 Society for Research on Nicotine and Tobacco (SRNT)
2 Royal College of Physicians
3 Smoking Prevention Group of the Spanish Respiratory Society (SEPAR)
4 National Association of Women Pharmacists (UK)
5 EFPIA
6 Merck Sharp & Dohme (Europe) Inc.
7 Johnson & Johnson Consumer Group (JJCG)
8 Dr. Peter Hajek

If you would like more detail in this area please get in touch with Damien Bové damien.bove@idaconsultants.com

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology) and regulatory consultant, we work with our 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.

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“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.”

Guide Here

This guide has been prepared for micro, small and medium-sized enterprises (SMEs) operating in the pharmaceutical sector. Its aim is to facilitate understanding of the main aspects of medicinal product legislation. The guide is structured to follow the chronological stages of developing a medicinal product. An overview of the scientific data requirements for obtaining a marketing authorisation in the European Union (EU) is provided. The regulatory procedures in place to optimise development and obtain an EU marketing authorisation are also summarised.

The guide focuses primarily on the requirements for authorising innovative medicinal products for human or veterinary use. The guide is not intended to be an exhaustive document but rather to raise SMEs’ awareness of the various more detailed sources of information available, with links throughout the text to additional information.

In December 2005, Commission Regulation (EC) No 2049/20051 introduced provisions aimed at promoting innovation and the development of new medicinal products for human and veterinary use by SMEs. This guide is intended to fulfil the obligation laid down in Article 12 of that Regulation, which calls for a ‘User Guide’ on the administrative and procedural aspects of medicines legislation that are of particular relevance to smaller companies to be published by the European Medicines Agency (EMEA).

Pursuant to the SME regulation, companies can access financial assistance (in the form of fee reductions and fee deferrals) and administrative assistance from the agency, details of which are outlined in Section 2 of this guide. To facilitate contact with the agency, an ‘SME Office’ was launched in December 2005 and is dedicated to addressing the particular needs of smaller companies.

If you would like more detail in this area please get in touch with Damien Bové damien.bove@idaconsultants.com

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology) and regulatory consultant, we work with our 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.

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“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.”

In the case of certain categories of medicinal products,however, in order to meet unmet medical needs of patients and in the interests of public health, it may be necessary to grant marketing authorisations on the basis of less complete data than is normally the case and subject to specific obligations, hereinafter ‘conditional marketing authorisations’

The categories concerned:

1. medicinal products which aim at the treatment, prevention or medical diagnosis of seriously debilitating or life-threatening diseases,
2. medicinal products to be used in emergency situations, in response to public health threats duly recognised either by the World Health Organisation or by the Community in the framework of Decision No 2119/98/EC;
3. medicinal products designated as orphan medicinal products in accordance with Article 3 of Regulation (EC) No 141/2000.

All the following need to be met:

1. the risk-benefit balance of the medicinal product, as defined  in Article 1(28a) of Directive 2001/83/EC, is positive;
2. it is likely that the applicant will be in a position to provide the comprehensive clinical data;
3. unmet medical needs will be fulfilled
4. the benefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required.

If you would like more detail in this area please get in touch with Damien Bové damien.bove@idaconsultants.com

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology) and regulatory consultant, we work with our 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.

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“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.”

full text here

CF is a life threatening and chronically debilitating disease, impairing the Quality of life, characterised by progressive bronchiectasis and obstructive pulmonary disease (> 90% patients). The lower respiratory tract involvement is the primary cause of morbidity and mortality in patients with CF (> 90% of fatalities). Ninety percent of CF patients are colonised with Pseudomonas aeruginosa (PA), and PA infections are the cause of mortality in 80% of those patients.

CF is mainly a paediatric disease. Life expectancy is considerably shortened due to respiratory damage-induced morbidity and mortality. However, due to the advances of past 20 years in the prophylaxis of chronic respiratory infection, along with the systematic supplement of pancreatic enzyme and decrease in malnutrition CF patients reach a mean 30 to 40 years of age, and the proportion of adult CF patients increases.

The guidance will focus on:
• clinical trials design in lung involvement and clinically relevant endpoints (mucociliary clearance and broncho-pulmonary infections (mortality), in particular: treatment of PA early colonisation, chronic infection and exacerbations; prophylaxis of chronic PA infection; slowing/stopping lung damages (fibrosis, bronchiectases)
• clinical trials design in pancreatic involvement, in particular management of exocrine pancreatic insufficiency and malnutrition.