EMA and FDA encourage use of innovative approaches in the development of medicines for Gaucher disease

Approach aims to facilitate development of medicines for rare paediatric diseases in general

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have developed a joint proposal to promote the use of innovative approaches in the development of medicines for Gaucher disease, which can apply to rare diseases in children in general.

This strategic collaborative approach from EMA and the FDA discusses possible ways to enhance the efficiency of medicine development in Gaucher disease, a rare lysosomal storage disorder, which is used as a model to reflect on recent progress made in the area of data extrapolation. The strategy document encourages medicine developers to make better use of:

• extrapolation of available clinical data, including through appropriate modelling and simulation techniques, to predict how a medicine may work in children and adolescents on the basis of studies conducted in adults or other paediatric populations;
• the possibility to test the safety and efficacy of medicines developed by different companies in one single trial, so-called multi-arm, multi-company clinical trials. As the same control arm is used to compare more than one medicine under evaluation, this approach facilitates the clinical testing of medicines while reducing the total number of children included in trials.

The approach set out by this joint proposal aims to reduce the number of patients needed for clinical trials, meaning overall less burden on children and their families, while maintaining high quality standards for medicine development.

Medicine developers who wish to apply these innovative approaches in their development plan are advised to seek scientific advice. They can approach EMA or the FDA separately, or request parallel scientific advice from the two regulatory authorities if they wish.

In addition, EMA is finalising a reflection paper which outlines a systematic approach to scientifically sound and reliable extrapolation of data to support medicine authorisation. The paper, which is expected to be published the fourth quarter of 2017, will complement the approach published today.

The strategic collaborative approach from EMA and the FDA published today is the result of extensive collaborative work with various groups of stakeholders, including patients and healthcare professionals.

The FDA will be publishing this strategy paper in a different format in the next few months.

Source: EMA

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Implementation plan for the introduction of the safety features on the packaging of centrally authorised medicinal products for human use

EMA NEWS

Certain aspects of the implementation of the Falsified Medicines Directive (Directive 2011/62/EU) and the new delegated act on the safety features (Commission Delegated Regulation (EU) 2016/161 - "the Delegated Regulation") may impact on the product information and the marketing authorisation dossier; in particular the placing of safety features, a unique identifier (UI) carried by a 2-D barcode and an anti-tampering device (ATD), on the packaging of prescription medicines and certain nonprescription medicines for the purposes of authentication and identification. 

The European Medicines Agency and the European Commission have prepared this implementation plan to guide applicants and Marketing Authorisation Holders (MAHs) through the regulatory changes necessary to accommodate the new legislative requirements. 

The European Medicines Agency and the Quality Review of Documents (QRD) Group have revised the Human Product Information templates. The updated QRD Template will facilitate the implementation of the relevant standard statements on the UI and its carrier under sections 17 and 18 of Annex IIIA, in order for the MAHs to implement the safety features by the 9th of February 2019 as required by the Delegated Regulation. 

The inclusion of the safety features standard statements under sections 17 and 18 of Annex IIIA does not indicate that the safety features have been actually implemented on the packaging placed on the market, but rather that the product information has been updated to confirm that the safety features will be implemented on the marketed packaging in line with the provisions of the Delegated Regulation (i.e. by the 9th of February 2019).

Source: EMA Click Here for more infor & Downloads


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PHARMA NEWS | EMA Builds Collaboration with African Regulators

The agency met with the representatives of the East African Community to discuss the creation of a networking agency.

The European Medicines Agency (EMA) met with a delegation from the East African Community (EAC) from May 18–19, 2017 as part of the agency’s collaboration with African regulators. The meeting offered an exchange of information and discussion on the EAC potentially creating a networking medicines agency. The meeting followed a workshop held in March as part of EMA’s Article 58 procedure for drugs intended for use outside of the European Union.

EMA’s structure and operations were used as a model for the potential EAC networking medicines agency. Discussions at the meeting included drug approval processes, pharmacovigilance, inspections, training, and internal controls.

Participants in the meeting included heads of EAC national agencies, representatives from the World Health Organization, and representatives from the World Health Bank. The EAC is an official observer of the International Council for Harmonization and is a member of the International Pharmaceutical Regulators Forum.

Published under PHARMA NEWS

Source: pharmtech

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How to inform EMA about GMP Problems or Data Integrity Issues

European Medicines Agency (EMA) released a new policy on the handling of information which has been received from external sources about serious concerns, e.g. related to GMP deviations or Data Integrity issues. The EMA stated in its press release that "since 2013 EMA has received a total of 43 reports that relate for example, to the manufacturing of medicine or the conduct of clinical trials."

So far there has been no formal procedure in place to handle these cases. This gap has now been closed with the publication of the EMA Policy/0072 entitled: "EMA's handling of information from external sources disclosing alleged improprieties concerning EMA activities related to the authorisation, supervision and maintenance of human and veterinary medicinal products". In this document the EMA describes how reports will be handled and which actions will be taken. In the US a procedure for so called "whistleblowers" is well known in public. However, in Europe very few countries have procedures for individuals to report improprieties to an authority.

The document published by the EMA intends to help especially individuals to report their observations if they notice illegal procedures within companies. This might be the case if, for example, raw data has been deleted or adulterated. Such cases of fraud might cause serious risks for patient safety e.g. if a QC test confirms that a product is out of specification and the corresponding data has been deleted or if manufacturing steps have not been performed according to the documentation in the company. In the past years more and more cases have become public about Data Integrity. The policy for external individuals will support them to ensure that the information provided will be treated in a structured manner and that the personal data of the individuals will be treated confidentially.

Source: EMA, gmp-compliance
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EMA revises Guide on Pharmaceutical Water And open for Commnets

EMA revises Guide on Pharmaceutical Water

According to the European Pharmacopoeia, it will be allowed as of the 1st of April 2017 to produce water for injection (WFI) using cold i.e. non-distillation methods (as already reported under "WFI obtained by non-distillation methods - What are the Next Steps?"). Now, the European Medicines Agency (EMA) has published a concept paper on the revision of the Note for (Click Here for Download) Guidance on Quality of water for pharmaceutical use (CPMP/QWP/158/01 EMEA/CVMP/115/01). This Guideline from 2002 describes the different qualities of water for pharmaceutical use and their use in the production of medicinal products and/ or diverse dosage forms (e.g. topical, oral or parenteral). The paper also mentions the production of the different qualities and is in line with the present regulations of the European Pharmacopoeia which only allowed distillation for the production of WFI. The document also describes Highly Purified Water (HPW) and its use. HPW meets WFI quality but can be produced by means of membrane processes. Here, an essential aspect is the proposition of EMA's Quality Working Party  to remove in the future the monograph (1927) Highly Purified Water.

The (Click here) "Concept paper on the need for revision of note for guidance on quality of water for pharmaceutical use (H+V)". Comments to this paper can be submitted until 6th June 2017.

Email address for submissions: qwp@ema.europa.eu

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European Medicines Agencies and PIC/S agree on Audit Cooperation

PIC/S has published a "Letter of Agreement between the EEA Heads of Medicines Agencies and the Pharmaceutical Inspection Co-operation Scheme" on their website. The objective of the agreement is the exchange of information after audits among authorities and joint trainings for GxP inspectors.

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European Medicines Agency’s Committee Meeting highlights on Medicinal Products for Human Use (CHMP) 23-26 January 2017

European Medicines Agency’s Committee Meeting highlights on Medicinal Products for Human Use (CHMP) 23-26 January 2017

Eight medicines recommended for approval, including two biosimilars.

The European Medicines Agency’s Committee for Medicinal Products for Human Use(CHMP) recommended eight medicines for approval at its January meeting.

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Handling of Reports of Suspected Quality Defects in Medicinal Products

GDP Compliance : EMA Active substances and medicinal products shall be distributed in accordance with good distribution practices. Member States shall enter the certificates of good distribution practices which they issue in the Union database (EudraGMDP) in accordance with Art. 111(6) of the Directive 2001/83/EC as amended. The Community formats for the GDP Certificate for Medical Products and for active substances are published in the Compilation of Community Procedures, which can be found at the following location: The EudraGMDP Database contains GMP and GDP Compliance and Non-Compliance reports. So far a lot of GMP information has been published by the EU authorities but less about GDP compliance. Every EU Member State Inspectorate is asked to publish their GMP and GDP Inspection results. This will allow other authorities and industry to check the compliance status of certain companies. A number of EU authorities have not published their GDP data information yet. However, progress can be seen also with regard to GDP compliance certificates and GDP non-compliance reports. From January 2016 until today six GDP non-compliance reports have been published. The Czech authority published three, the Danish authority two and a German inspectorate one GDP non-compliance report. The current reports are not very detailed. However, some information can be found. For instance, for the company Ya Medicare in Winsen Germany the inspectors state: "The company has in general failed to comply with good distribution practice as they did not use the described quality system. The company has written procedures but was not able to show an adequate documentation for acting according to the procedures. Further the company does not have relevant documentation for storage and transport under correct temperature, a system for ensuring that they only obtain their supplies of medicine from companies with a valid WDA/MIA or a system for ensuring that they only supply medicines to companies with a valid WDA or a valid license to supply medicinal products to the public." The information provided in the report is very helpful in order to understand the nature of the non-compliance. 1. Introduction  1.1 One of the main purposes of the GMP/GDP Inspectors Working Group is to establish and maintain a system for mutual recognition of national inspections in respect of the manufacture and, where relevant, wholesale distribution of medicinal products and for the administrative collaboration between Member States (MS) of the European Economic Area (EEA). The general requirements for national pharmaceutical inspectorates are to fulfil the requirements of national legislation and of the relevant European Directives for EEA countries. Specific obligations of inspections as contained in national law and if any European Directives must be included in the national Inspectorate’s quality systems.  1.2 This document outlines the quality system requirements for GMP pharmaceutical inspectorates. It is intended that each GMP pharmaceutical inspectorate uses the document as the basis for developing and implementing its quality system and for preparing the quality manual. In addition to providing a basis for self-assessment and a reference document for use by external assessors, establishing and maintaining an effective quality system will generate confidence within and between GMP national pharmaceutical inspectorates in the assessment of compliance with good manufacturing practice and/or good wholesale distribution practice. 1.3 National GMP pharmaceutical inspectorates, the European Commission (EC), the European Medicines Agency (EMEA) and the pharmaceutical Inspection Cooperation Scheme – (PIC/S) should co-operate with one another in exchanging experiences in the maintenance and operation of quality systems and in the further development of this document.  1.4 Only on voluntary basis, this document could be useful for (other) inspectorates assessing compliance with GXP or for the inspection of pharmacies.  1.5 In preparing this text, the working group was advised by: EN ISO/IEC 17020:2005 General criteria for the operation of various types of bodies performing inspections; EN ISO/IEC 17023:2006 General requirements for bodies operating assessment and certification/ registration of quality system; ISO 9001-2000 Quality management systems-Requirements;  ISO 9004-2000 Quality management systems: guidelines for performance improvements;  ISO 19011 : 2002 Guidelines for quality and/or environmental managerial systems auditing;  PI 002-1 : 2000 Recommendations on quality system requirements for pharmaceutical inspectorates; Compilation of Community Procedures on Inspections and Exchange of Information  EMA/572454/2014 Rev 17 Page 7/253 May 2001 Revised Compilation of Community procedures on administrative collaboration and harmonisation of inspections; 1998 Proceedings of the PIC-PIC/S seminar on quality systems for pharmaceutical inspectorates. Handling of Reports of Suspected Quality Defects in Medicinal Products Procedure for Handling Rapid Alerts Arising from Quality Defects Download:  Full guideline Quality Systems Framework for GMP Inspectorates

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EMA issues new Guideline on "Chemistry of Active Substances"

A medicinal product authorization application requires comprehensive information on origin and quality of an active substance. What information is required was defined in two Guidelines so far: the Guideline "Chemistry of Active Substances" (3AQ5a) from 1987 and the "Guideline on the Chemistry of New Active Substances" from 2004. Because both Guidelines' content do not take into account the ICH Guidelines Q8-11 issued in the meantime and do thus not meet the current state of the art in sciences and in regulatory practice, the EMA Quality Working Party (QWP) developed an updated document  entitled "Guideline on the chemistry of active substances" (EMA/454576/2016), which was issued on 21 November.

The new Guideline describes the information on new or already existing active substances required in an authorization dossier. In the context of this Guideline "already existing" ingredients are those that are used in a product already authorized in the EU.

In detail the information and data regarding the substance have to be included in the following chapters of the CTD:

3.2.S.1: Nomenclature, information on the structural formula, pharmacological relevant physicochemical properties.

3.2.S.2: Information on the manufacturer(s), contractor(s), testing facilities etc.; description of the manufacturing processes (schematic representation with flow diagram as well as narrative); where appropriate detailed information on alternative manufacturing processes, for recovering of solvents and for routine reprocessing. Information with regard to re-working should not be included in the authorization dossier.

3.2.S.2.3: Information for controlling the material used during the manufacture and for its specification (incl. identity test). This paragraph is more comprehensive in the new Guideline compared with its predecessor and takes into account the requirements of the ICH Guideline Q11. This Guideline comprises requirements for the following materials: materials from biological sources, those used for the chemical synthesis of starting materials, materials from herbal origin, excipients like solvents (incl. water), reagents, catalysts etc.

3.2.S.2.4: Information on critical process steps (the Guideline comprises examples for these critical steps) as well as on quality and control of isolated intermediates within the synthesis steps. All information has to be provided with the appropriate justifications.

3.2.S.2.5: Information on Process Validation

3.2.S.2.6: Information on the development of the manufacturing process. Here all changes have to be described that were performed during the various phases (pre-clinical, clinical, scale-up, pilot and possibly production phase) of the process for new active substances. For already existing active substances available in production scale no information on process development is needed.

3.2.S.3: Information on Characterisation. Comprehensive information on the elucidation of the structure of the active substance, its physico-chemical properties and its impurities profile have to be provided. Further, the mutagenic potential of degradation products has to be considered. The analytical methods have to be described and their suitability has to be justified.

3.2.S.4: Information on the control of active substances. The analytical procedures and their validation have to be described. Data for the analytical method development should be provided if critical aspects of the analysis regarding the active substance's specification need to be clarified. Analytical data are necessary for batches for pre-clinical and clinical studies as well as for pilot batches which are not less than 10% of the maximum production scale. The substance's specification and its control strategy have to be justified on the basis of data from the pre-clinical and clinical phase and, if available, from the production phase.

3.2.S.5: Information on reference materials. If no Chemical Reference Substances (CRS) of the European Pharmacopoeia - counting as completely qualified reference standards - are used, comprehensive information on the analytical and physico-chemical characterization are required even for established primary standards.

3.2.S.6: Information on Container Closure System. Here a brief description is sufficient. However, if a Container-/Closure System is critical for the substance's quality, its suitability has to be proven and justified. A reference to stability data can be used as supporting information.

3.2.S.7: Information on Stability. A detailed description of the stability studies carried out and the protocol used as well as a summary of the results are expected. Information on stress studies and conclusions on storage conditions and re-test dates or expiry dates are also to be made. This does not apply to substances monographed in the European Pharmacopoeia. If no re-test period or expiry date of batches on the production scale is available at the time of submission of the application, a stability commitment has to be attached with a post-approval stability protocol. The analytical methods have to be described.

The Guideline's provisions also apply to an Active Substance Master File (ASMF) or to a Certificate of Suitability (CEP). They apply to active substances that have undergone development in a "traditional" way or according to the "enhanced" approach. The provisions of the ICH Guidelines Q8-11 have to be taken into account.

The Guideline is not applicable to active substances of herbal, biological and biotechnological origin as well as to radiolabelled products and radiopharmaceuticals.

The Guideline "Guideline on the chemistry of active substances" (EMA/454576/2016) becomes effective six months after issuing, which means in May 2017.

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EMA revises Guideline on Process Validation for Finished Products

EMA revises Guideline on Process Validation for Finished Products

The EMA (European Medicines Agency) has published the revised version of its “Guideline on process validation for finished products – information and data to be provided in regulatory submissions“ on 21 November 2016.

 

The amendment is considered to be minor and only comprises an update to the definition for “on-line measurement" included in the glossary. A consultation phase was considered to be unnecessary as the document is not intended as a full revision of the guideline.

Source:

EMA: Guideline on process validation for finished products

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EMA/ FDA Mutual Recognition Agreement moving forward

EMA/ FDA Mutual Recognition Agreement moving forward

A possible agreement between the European Medicines Agency EMA and the US Food and Drug Administration FDA on mutual recognition of drug facility inspections could already be signed in January 2017. This is noted in a report of the EU Commission: "The state-of-play and the organisation of the evaluation of the US and the EU GMP inspectorates were discussed. In light of the progress achieved, the conclusion of a mutual recognition agreement of Good Manufacturing Practices (GMPs) inspections by January 2017 is under consideration."

But, according to the Commission, some issues are still not resolved - like, for example, the exchange of confidential information and the inclusion of veterinary products in the scope of the text.

The "Report of the 15th Round of Negotations for the Transatlantic Trade and Invesment Partnership" summaries the 15th round of negotiations for the Transatlantic Trade and Investment Partnership (TTIP) from 3rd to 7th October 2016 in New York.

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Comprehensive Overview of global Initiatives on Medicine Regulation published

The European Medicines Agency (EMA) has published an overview of existing international regulatory initiatives for human medicines, like for example International Council for Harmonisation (ICH), Pharmaceutical Inspection Co-operation Scheme (PIC/S), International Pharmaceutical Regulators Forum (IPRF) and International Coalition of Medicines Regulatory Authorities (ICMRA) members.

The report entitled  (click to download)  "Connecting the dots - Towards global knowledge of the international medicine regulatory landscape: mapping of international initiatives" also lists the respective international projects and details on the number and scope of global initiatives.

The aim of the mapping exercise is to raise awareness of ongoing international regulatory activities, help establish a basis for a more strategic coordination to avoid duplication of efforts, and identify possible gaps.

In the conclusion, EMA mentions that there is "a myriad of initiatives but no strategic coordination". So these mappings and analyses should "strongly support the need for a global strategy to support cooperation between international medicines regulators, to help avoid an overlap of activities and make resources available for areas where gaps still exist."

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