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EMA, new features for the PRIority Medicines (PRIME) scheme


By Giuliana Miglierini Based on the review of results obtained in the first five years of implementation of the PRIority Medicines (PRIME) scheme, the European Medicines Agency has launched a set of new features to further enhance the support to Read more

The proposals of the EU Commission for the revision of the IP legislation


By Giuliana Miglierini In parallel to the new pharmaceutical legislation, on 27 April 2023 the EU Commission issued the proposal for the new framework protecting intellectual property (IP). The reform package impacts on the pharmaceutical industry, as it contains proposals Read more

Webinar: Pharmacovigilance as a specialization and the role of the Pharmacovigilance Risk Assessment Committee (PRAC)


EIPG webinar Next EIPG webinar is to be held on Wednesday 31st of May 2023 at 17.00 CEST (16.00 BST) in conjunction with PIER and University College Cork. Sofia Trantza, a pharmacist with long experience as a Qualified Person for Pharmacovigilance Read more

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EDQM introduces a consultation phase in the management of CEP documents

January 27, 2023 , , , , , , , , , , , , , , , , , , , , , , , , , , ,

by Giuliana Miglierini

The new process implemented by the European Directorate for the Quality of Medicines and HealthCare (EDQM) for the elaboration of documents related to the Certification of Suitability  (CEP) procedure includes a newly inserted consultation phase. This new step, which may be public  or targeted to specific groups of stakeholders, aims to increase the transparency of the  elaboration process and offers stakeholders the possibility to forward comments to the draft documents  in order to optimise them.

Transparency and efficiency are also the main goals inspiring the overall new elaboration process, which covers the entire pathway of CEP documents, from development, through consultation,  up to final adoption, publication and implementation.

A dedicated page on the EDQMs’s website will host the documents open to consultation, together with the respective instructions for the stakeholders wishing to submit comments. Announcements  on new documents available for consultation will be made on EDQM Certification webpages.  The CEP Steering Committee will be responsible for the elaboration process for CEP documents, in compliance with the EDQM document CEP Terms of Reference and Rules of Procedure (PA/PH/CEP (01) 1).

The elaboration process will cover both public documents (the main part), as well as those the CEP Steering Committee would indicate as restricted for use by the bodies involved in the CEP  procedure. The new process does not cover the Resolution on the Certification procedure, which falls under another specific process established by the Council of Europe.

A guidance to understand the new process

The management of CEP guidelines and operational documents for the CEP procedure has been described in a specific guidance issued in November 2022 by the EDQM’s Certification of Substances Department.

The guidance covers a broad range of documents participating from different perspectives to the CEP procedure. The elaboration of the different types of documents may slightly differ from one another, with possible exemptions from some steps, for example in the case of minor revisions (which in any case always have to be full justified and documented). All CEP documents will be drafted in English; the guidance provides indication of the format to be used to establish the unique reference code for governance documents and technical guidelines (PA/PH/CEP (XX)  YY), as well as for the revision number (ZR) where needed.

 The EDQM specifies that the implementation date of the newly approved CEP documents will be such to allow interested parties to have enough time to comply with the new or revised requirements.

Governance documents define procedural aspects for the practical implementation of the CEP procedure. The initial draft will be prepared by the EDQM and reviewed and agreed upon by the CEP Steering Committee before entering the consultation phase. Comments collected will serve as the basis to consolidate the final version of the document. A second round of consultation may be needed in case of critical comments preventing finalisation. The adoption of the final document falls under the responsibility of the CEP Steering Committee, which may also indicate the need to improve and re-submit the draft before adoption. Once the final version of the document is available, its publication on the EDQM’s website and implementation will close the process.

Technical guidelines inform about the requirements applicants should fulfil for the submission or evaluation of CEP applications. Their drafting may be initiated also by members of the relevant Technical Advisory Board (TAB), in addition to the EDQM. The TAB is also called to review and agree upon the draft document before the assessment and approval by the CEP Steering Committee and the following consultation phase can take place. The same applies to the consolidation of comments and finalisation of the document, that has to be approved by the relevant TAB. In this case too, a second round of consultation is possible should criticalities arise during the first one, followed by adoption by the CEP Steering Committee (and a possible second round of updating and approval by the TAB, if needed), and publication and implementation.

The management of specific aspects of the procedure can be supported by the issuing of administrative or operational documents. These documents fall under the responsibility of the EDQM, that may consult the CEP Steering Committee of other parties where necessary.

The consultation phase

A specific chapter of the EDQM’s guidance describes the newly inserted consultation phase, those details (type of process and duration) will be decided on a case-by-case basis by the CEP Steering Committee.

In the case of a public consultation, the draft document will be made available at the dedicated page of the EDQM website. The draft may also be sent to identified relevant stakeholder organisations, to ensure a better awareness of the ongoing process.

Targeted consultations aim to obtain feedback from selected stakeholders on specific areas of intervention. In such instances, the forwarding of the draft document will be restricted only to identified interested parties, including regulators and relevant industrial associations or other organisations.

According to the type of document and/or the topic under consultation, the consultation phase may vary in duration. To this instance, the guidance indicates a possible range between 3 weeks and 3 months, with the effective duration to be communicated as a part of the call for consultation.  A template will also be available to submit comments, which should be always justified and contain concrete proposals for action to tackle the issue under consideration. All comments and justifications received will be transmitted to the groups in charge of approving and adopting the documents.

At the end of the elaboration process, the final approved versions of CEP documents will be published on the EDQM’s website.   


Real-world evidence for regulatory decision-making

September 23, 2022 , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , ,

by Giuliana Miglierini

Digitalisation is rapidly advancing also in the regulatory field, as a tool to improve the efficiency and accuracy of processes used for the generation and use of data to inform the regulatory decision-making. To this instance, real-world data (RWD) on the use of a certain medicine by the general population are increasingly important to monitor the impact in terms of health benefits, adverse events and costs for healthcare systems.

The trend is confirmed by the publication of a joint statement by the International Coalition of Medicines Regulatory Authorities (ICMRA) addressing the use of real-world data and real-world evidence (RWE) in the development, authorisation and monitoring of medicines to support regulatory decision-making. The document has been endorsed by the European Medicines Agency (EMA), which also published the third workplan developed by its Big Data Steering Group to set key actions to be delivered in the period 2022–2025.

The debate on real-world evidence for regulatory decision making was also stimulated by an ISPE’s position paper released in 2020, and by papers published in the scientific literature (see here a recent review).

ICMRA’s joint statement on real-world evidence

The joint statement is the final result of a ICMRA workshop on real-world evidence co-organised with EMA, the US FDA and Health Canada in June 2022, that saw the participation of representatives from more than 40 countries. Heterogeneous data sources across different geographic areas and different levels of quality, as well as various processes for data sharing and access of the data, were among the main open issues identified during the meeting. Four different areas of collaboration have been identified, representing the focus of further discussion by international regulators in order to implement concrete actions to address criticalities.

The first area addresses the harmonisation of RWD and RWE terminologies. This exercise is deemed essential in order to share common operational definitions of RWD and RWE, characterized by a clear scope and level of granularity (e.g., pertaining to RCTs and observational studies).

Already existing guidance, such as the ICH M14 on “General principles on planning and designing pharmacoepidemiological studies that utilize real-world data for safety assessment of a medicine”, may represent useful tools to support these activities.

The convergence on RWD and RWE guidance and best practice should include the development of common principles for RWD quality. Metadata are an essential feature, as they enable the characterisation and discoverability of RWD. Other targets include the definition of suitable scenarios where RWE may contribute to regulatory decision-making, and the setting up of templates for study protocols/reports that can be used in multiple regulatory jurisdictions.

Readiness shall be pursued by strengthening the international regulatory collaboration on RWE. The rapid creation of expert groups on specific topics of interest and an improved collaboration on governance and processes aimed to support the efficient conduct of RWD studies in different countries are key actions in this area.

Transparency should be the common denominator underlying all the above-mentioned activities. It should be tackled through the definition and use of common principles and practices for the systematic registration of pre-specified study protocols and study results in publicly available registries. Publication of study results in open-source, peer-reviewed journals is also expected.

The 2022-2025 workplan on Big Data use for public health

In parallel with the publication of ICMRA’s joint statement, EMA released the Big Data Workplan 2022-2025, jointly developed with the Heads of Medicines Agencies (HMA). The joint workplan represents the expansion towards the operative phase of the priority recommendations for regulators on the best approaches to use and generate data developed in 2020 by the former Big Data Task Force. The document refers mainly to medicines for human use, even if it includes some information on veterinary medicines.

Big data are the key element to be considered in order to achieve the overall objectives seen above, as they enable the extensive analysis of information available on medicinal products subject to regulatory scrutiny. According to the Big Data Steering Group, the increased evidence arising from big data analysis may accelerate medicine development, improve treatment outcomes and facilitate earlier patient access to new treatments.

The workplan details ten different priory areas and the respective recommendations for action, and it provides the expected timelines for their implementation.

Central to the overall planned system is the Data Analysis and Real-World Interrogation Network (DARWIN EU), a federated network to enable access and analysis of real-world data. After the establishment of the DARWIN EU Coordination Centre and the activation of the first studies in 2022, new activities shall focus on the support to the European Health Data Space (EHDS) and the collaboration with the European Centre for Disease Prevention and Control (ECDC), HTA bodies and payers.

The joint action Towards A European Health Data Space (TEHDAS) shall facilitate the engagement with stakeholders, a critical point to ensure the quality of data. The release of a first version of a data quality framework for the EU Regulatory Network is expected in late 2022. An agreement on metadata to describe and identify RWD sets shall also be supported by the publication of a good practice guide on metadata and a public catalogue of European RWD. Release of new analytics tools and a review of the utility of eHealth data and social media data are also part of actions targeted to data discoverability.

The reinforcement of skills of all stakeholders involved in the process of RWD and RWE generation, analysis and use is also essential to guarantee the success of the new approach. EMA and HMA aim to develop agreed training curricula on biostatistics, pharmacoepidemiology and data science, including the availability of selected modules open to patients, healthcare professionals and academics. A curriculum on genomics data shall also be adopted.

The results of pilot studies on the use of RWE by EMA scientific committees will be released in next years, with a final report and a portfolio of RWE use cases to support uptake of RWE by the ERMN expected in 2025.

More in detail, a proof-of-concept pilot assessing access to individual patient data from clinical trials (raw data) in the scientific assessment of medicines is planned for Q4 2022. The final reflection paper on artificial intelligence (AI) for use in regulatory processes shall be published in Q3 2023, while 2024 should focus on a pilot on data analysis for CMC data.

All the above-mentioned activities shall be supported by new guidance issued by the new EMA Methodologies Working Party, while expert advice will be strengthened through the European Specialised Expert Communities (ESEC), initially focusing on AI & RWE.

The governance of the process shall benefit by a review of the mandates of the Big Date Steering Group and EU Network Data Board expected for late 2022. Target actions aimed to strengthen the framework for ethics in data analysis will also be explored. Activities at the level of the international framework of regulators shall include among others the evaluation of the clinical trial conceptual model under the ICH M11.

An annual Big Data stakeholder forum is also planned, complemented by topic specific meetings and workshops and biannual industry meetings. In the veterinary field, the workplan includes the implementation of the EU Veterinary Big Data strategy adopted in 2022. Among the main deliverable are a catalogue of veterinary data sources, finalisation of the system on antimicrobials sale and use data, and the evolution of training curricula.


FAT and SAT, a critical step for the introduction of new equipment

March 25, 2022 , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , ,

by Giuliana Miglierini

There are two key moments to be faced to introduce a new piece of equipment in a pharmaceutical plant: a factory acceptance testing (FAT), usually performed by its manufacturer to verify the new equipment meets its intended purpose, prior to approve it for delivery and once arrived at its final destination and installed, a site acceptance testing (SAT) run by the purchasing company and is part of the commissioning activity.

According to an article published in Outsourced Pharma, the commissioning of a new piece of equipment poses many challenges, and criticalities needs to be considered both from the business and regulatory point of view. Pharmaceutical plants are very complex and often customised upon the specific business needs, and the delivery of a new equipment requires the interaction of many different parties, both internal and external to the purchasing company. FAT, SAT and commissioning activities require a careful planning and detailed responsibilities for all participating parties to be included within the Commissioning and Qualification Plan (CQV plan). A possible responsibility matrix is suggested by the authors to provide clarity and ensures ownership of activities.

FAT, assessing the equipment at the manufacturer site

FAT and SAT testing involve the visual inspection of the equipment and the verification of its static and/or dynamic functioning, in order to assess the actual correspondence to the user requirement specifications (URS). While FATs are usually based on simulations of the equipment’s operating environment, SAT testing occurs at the final site after installation, thus it reflects the real operating conditions and environment in order to support qualification.

There are many different elements to be considered during FAT testing, including for example verification of the existing site drainage, piping, or room dimensions, or the position of the handle for accessibility, as well as software design specification, interface, and device integration.

The FAT exercise is always highly recommended, as it is essential to solve in advance (before shipment to the final destination) any error or malfunctioning of the equipment, that otherwise might occur at the purchasing company’s site. This results in the optimisation of the delivery and commissioning process, with important savings in terms of both time and costs for the purchasing company. To ensure for the transparency of FAT testing, the entire procedure (that requires usually 1-3 days, depending on the complexity of the equipment to be verified) is usually performed in the presence of a third party inspector and customer representative.

A comprehensive set of documentation should be always available to support FAT, including URS, drawings, checklists and procedures, calibrations and certifications, data sheets, references, etc. Raw data acquired during FAT are transmitted to the customer for analysis and validation. FAT should take into consideration all aspects relevant to the evaluation of the safety and functionality of the equipment and its compliance to URS, GMPs and data integrity. To this regard, it is also important for the engineering team called to run the new equipment at its final location to learn and share knowledge with the manufacturer along the entire commissioning process, so to increase the first-hand direct experience. According to the article, this is also critical to authorise the shipment of the equipment to the final destination, a step that should always be performed by an authorised, trained, and approved subject matter expert.

 SAT acceptance testing

All criticalities emerged during the FAT exercise are then checked again at the final site, after installation and verification; additional test cases may also be added to the SAT protocol to check for potential failure modes. SAT testing is performed once all connections between the new equipment and other machines/softwares are in place, under the real operating parameters, and may be witnessed by a representative of the equipment’s manufacturer.

Results from SATs may thus differ from those obtained from the FAT previously run by the manufacturer. From the regulatory point of view, SAT testing is a key element to demonstrate the compliance of the equipment to GMP requirements and to support the overall quality and safety of pharmaceutical productions. In this case too, many are the possible elements to be inspected and verified, including interlocks, ventilation, internal box pressure, electrical/hydraulic connections and safety systems, visual checks of components, training of the operators, etc.

A plan for each testing phase

FAT planning begins at the very moment of the purchasing company placing the order for the new equipment, and it has to reflect all URS to be checked for acceptability of the manufactured apparatus. This step in the design is critical and calls for a strict and positive communication between the manufacturer and its customers, a key point to take into consideration all elements that should enter the project.

All identified items and procedures to be challenged during FAT and SAT testing are usually addressed within the CQV plan, that connects the design phase to user requirements specifications and the other elements impacting the commissioning and qualification processes (i.e. system impact assessment, design specification, functional risk assessment, hardware / software specifications, Installation / Operational / Performance Qualification), including deviations and change management. The plan specific to SAT testing should include the scope, test specifications and logs, a test summary, the Commissioning report and the final Certificate of Acceptance.

Transparency and a robust statistical approach should represent main targets along the entire commissioning and validation procedure, that may be run with the assistance of external consultants. All activities that shall enter the regulatory dossiers should always be justified and documented, also under the perspective of data integrity. The Outsourced Pharma’s article also suggests paying a particular attention to controls on data provided by the manufacturer in the case a risk-based leveraging is applied.


Steps towards the final approval of the IP action plan

December 10, 2021 , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , ,

By Giuliana Miglierini

The end of 2021 may see the final approval of many pieces of the new legislative framework announced in November 2020 by the European Commission. An important piece of this puzzle is represented by the IP Action Plan, governing the protection of intellectual property (IP); a step forward in this direction is represented by the resolution of 11 November 2021 on the Own-initiative report of the European Parliament.

The final text licensed in single reading is the result of the examination of the initial draft report – issued in May 2020 by the Committee for Legal Affairs, rapporteur Marion Walsmann – by several other Committees (IMCO, DEVE, CULT, AGRI).

The main points of the resolution

The resolution recognises the importance for the European economy of a balanced protection and enforcement of intellectual property rights (IPR). In years 2012-2016, the knowledge-intensive industries generated almost 30% of all jobs and almost 45% of total economic activity (in terms of Gross Domestic Product, GDP) in the EU; the IPR-intensive industries account for 93% of total EU exports of goods.

Europe’s recovery and resilience capacity is also highly impacted, as demonstrated by the pandemic when shortages of certain medicinal products and vaccines occurred. The EU Parliament acknowledges the role played by intellectual property in increasing the overall value of companies,especially the small-and-medium size ones (SMEs).

A current limitation to IP protection in Europe is represented by the still fragmented situation across different member states, which often leads to parallel national validation procedures and litigation for European patents. To this instance, the Parliament suggests the establishment of an IP coordinator at European level, to harmonise the approach to EU IP policy and enhance cooperation between the different bodies involved in the process (i.e. national IP authorities, Commission Directorates-General, EPO, EUIPO, WIPO, etc).

The Parliament also recognised the role IP plays in the pharmaceutical sector, where the availability of incentives greatly favours the development of new and innovative treatments. The resolution asks the Commission to support the innovative potential of European companies “on the basis of a comprehensive IP regime”, so to guarantee effective protection for R&D investments and favour fair returns through licensing. The availability of open technology standards has been valued as an important competitive element on the wider, global scenario.

Many different types of incentives are suggested by the Parliament’s resolution as useful to support micro-enterprises and SMEs in filing and managing their intellectual property, including IP vouchers, IP Scan and other Commission and EUIPO initiatives to support simple registration procedures and low administrative fees. The newly created European IP Information Centre may represents a fundamental reference point to increase knowledge in the field. The Parliament also suggests to introducing an EU-level utility model protection, not yet available, as a possible fast and low-cost protection tool to protect technical inventions.

Unitary patents and improved market competition

Still missing members states are urged to adhere to the enhanced cooperation scheme for the creation of a Unitary Patent Protection (UPP) and to ratify the Protocol to the Agreement on a Unified Patent Court on provisional application (PPA). The activation of this unique Court in charge of the examination of litigations would allow for a more efficient process and for lowering legal costs and improving legal certainty.

Fragmentation remains an issue also with respect to Supplementary Protection Certificates (SPCs): to this instance, the resolution asks the Commission to issue guidelines for member states and to provide a legislative proposal based on an exhaustive impact assessment. A major criticality to be solved is represented by the unitary patent not providing a unique SPC title valid across the EU; the own-initiative report also suggests the extension of the EPO’s mandate, so that examination of SPC applications could be carried out on the basis of unified rules.

Other important points needing attention to improve the presence of generic and biosimilar medicines in the EU are the abuse of divisional patent applications and patent linkage, which should also see an intervention by the Commission. The Parliament also opened the possibility of a revision of the Bolar exemption, which allows clinical trials on patented products needed to reach marketing authorisation of a generic or biosimilar version not to be regarded as infringements of patent rights or SPCs. This may also support the immediate market entry after the expiration of patent rights and SPCs. The Commission is called also to ensure the effectiveness and better coordination of compulsory licensing in order to provide access to medicines needed in case of health emergencies.

The resolution also addresses the theme of standard essential patents, which currently often leads to litigations, and it calls for the revision of the 20-years old system for design protection. Transparency on results obtained from publicly funded R&D is also recommended. The Parliament suggests artificial intelligence (AI) and blockchain technologies may play an important role in tackling counterfeiting practices and guarantee traceability of goods, as they may contribute to a better enforcement of intellectual property rights along the whole supply chain. The Commission should also work to establish clearer criteria for the protection of inventions created by the AI, without human intervention.

Comments from the industry

“The European Parliament has clearly voted for a strong and fair IP system by underlining the importance of timely generic and biosimilar medicine competition. The misuse of divisional patents, the need to enlarge the scope of bolar to include API and all regulatory and administrative steps, and the long overdue ban anti-competitive patent linkage are well known problems that the Commission should address in the IP Action Plan. The Parliament has voted; the Commission must act.”, said Adrian van den Hoven, Director General at Medicines for Europe.

A major point in the implementation of the new European policies is represented by the review the Commission is going to conduct in 2024 to assess the effective achievement of goals of the SPC manufacturing waiver, which entered into force in July 2019 and is expected to start producing effects in the second half of 2022.

Many of the themes discussed in the Parliament’s resolution were debated during a webinar organized by Medicines for Europe, with the participation of representatives from the European Commission and the European Patent Office.

EFPIA, representing the innovator pharmaceutical industry, focused its attention on the impact of past EU Free Trade Agreements (FTAs) on drug spending, timing of countries’ access to new medicines after global launch, investments overall and in pharmaceuticals, and clinical trial participation. A report by IQVIA published in the Federation’s website addresses the impact of IP protection on these elements. Results confirm the central role of the pharmaceutical sector as the most R&D intensive industry in the world, with R&D spending averaging over 15% of revenue. A strong IP protection framework available at the level of EU FTAs favours the attractiveness for investments in the EU and its FTA partner countries. According to the report, an expanded IP protection appears not to be linked to the generation of a higher pharmaceutical spending; drugs’ share of healthcare spending is claimed to stay flat or fall after an FTA, and prices for medicines to rise more slowly than the level of inflation. A stronger IP index, adds IQVIA, is also correlated with increased clinical trial activity in a country, bringing both clinical and economic benefits.


A new role for EMA and a pilot project for the repurposing of medicines

November 12, 2021 , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , ,

by Giuliana Miglierini

A draft agreement was reached at the end of October between the Council of the European Union and the European Parliament to reinforce the mandate of the European Medicines Agency (EMA) with reference to crisis preparedness and management for medicinal products and medical devices. “EU-level preparation and coordination are two essential ingredients to fight future health crises. Thanks to this deal we are adding an essential new building block to upgrade the EU’s health architecture. It will allow the EU’s Medicines Agency to make sure we have the medicines needed to deal with public health emergencies”, said Janez Poklukar, the Slovenian minister for health.

The revision of EMA mandate is part of the broader activities announced by the EU Commission in November 2020 to achieve the European Health Union; these also include the reinforcement of the European Centre for Disease Prevention and Control and a draft law on cross-border health threats. The establishment of the new Health Emergency Preparedness and Response Authority (HERA), announced in September 2021, is also part of the package. The draft agreement shall now be endorsed both by the Council and the Parliament before entering into force.

Three new key targets for EMA

The draft agreement reached by the Council and Parliament negotiators focuses on three main areas. The first one refers to the definition of a major event and how to recognise it: these shall be events likely to pose a serious risk to public health in relation to medicinal products, as acknowledged by a positive opinion from the Medicines Shortages Steering Group, and which may trigger specific actions such as the adoption of a list of critical medicinal products to fight the health threat.

Solid funding from the Union budget shall be also provided to EMA in order to support the work of the new steering groups, task force, working parties and expert panels. The availability of provisions for adequate data protection is important to guarantee the full compliance to the GDPR regulation and other EU data protection rules, and the safe transfer of personal data relevant to EMA’s activities (e.g. data from clinical trials).

EMA shall play an improved role in the monitoring and management of shortages of medicines and medical devices, a critical activity for the availability of the products needed during public health emergencies. Other points of the agreement include the timely development of high-quality, safe and efficacious medicinal products, and the creation of a new EMA’s structure specific for expert panels in charge of the assessment of high-risk medical devices and of essential advice on crisis preparedness and management.

How to tackle shortages of medicines

According to the EU Parliament, two “shortages steering groups” (for medicines and medical devices, respectively) shall be created by EMA; if needed, these groups may also include expert advice from relevant stakeholders (e.g. patients and medical professionals, marketing authorization holders, wholesale distributors, etc.).

Parliament negotiators highlighted the importance to achieve a high transparency of the process, including avoidance of interests related to industry sectors for members of the two groups; summaries of the proceedings and recommendations shall be also made publicly available.

A European Shortages Monitoring Platform shall be created by EMA to facilitate the collection of information on shortages, supply and demand of medicinal products; a public webpage with information on shortages of critical medicines and medical devices shall be also made available.

As already occurred during the Covid pandemic, future public health emergencies may boost the development of new medicines and medical devices. Sponsors of clinical trials conducted during health emergencies will be required to make the study protocol publicly available in the EU clinical trials register at the start of the trial, as well as a summary of the results. Following the granting of the marketing authorisation, EMA will also publish product information with details of the conditions of use and clinical data received (e.g. anonymised personal data and no commercially confidential information).

“With this agreement, Parliament makes both the Agency and all actors in the supply chain more transparent, involving them more in the process and fostering synergies between EU agencies. Moreover, we pave the way to promoting clinical trials for the development of vaccines and treatments, boosting transparency on those issues. In short, more transparency, more participation, more coordination, more effective monitoring and more prevention”, said Rapporteur Nicolás González Casares (S&D, ES).

EMA’s pilot project for the repurposing of medicines

The repurposing of already approved and marketed medicines is another key action put in place to ensure improved response capacity in case of future health emergencies.

A new pilot project to support the repurposing of off-patent medicines has been launched by EMA and the Heads of Medicines Agencies (HMA), with special focus on not-for-profit organisations and the academia as the main actors to carry out research activities needed to support the regulatory submission for the new indication. The initiative follows the outcomes reached by the European Commission’s Expert Group on Safe and Timely Access to Medicines for Patients (STAMP).

Interested sponsors may access EMA’s specific scientific advice upon submission of the drug repurposing submission form to the e-mail address [email protected] by 28 February 2022. More information is available in a Question-and-Answer document. The pilot will last until scientific advice for the selected repurposing candidate projects; filing of an application by a pharmaceutical company for the new indication is another target. Final results of the project will be published by EMA.

Comments from the industry

The European Federation of Pharmaceutical Industry Associations (EFPIA) welcomed the proposed framework for the repurposing of authorised medicines. “This pilot launch comes at a timely moment to test whether a streamlined and more transparent regulatory pathway for repurposing of off-patent established products increases the chances of including existing scientific evidence into regulatory assessment. One of the goals of the pilot is to raise awareness regarding the standards required for regulatory-ready evidence on the road to further increase availability of authorised therapeutic use”, said the chair of EFPIA’s Regulatory Strategy Committee Alan Morrison.

Innovation on existing, well-known molecules through repurposing can deliver huge benefits for patients, according to Medicines for Europe. The Association of the generic and biosimilar industry supports the pilot project as a way to generate robust data packages and to translate research into access for patients. A sustainable innovation ecosystem for off-patent medicine is the expected final outcome, possibly including also reformulation of existing medicines, new strengths or adaptation for specific patient groups (i.e. paediatric populations). “These investments must also be recognised in pricing and reimbursement policies to make access a reality for all patients”, writes Medicines for Europe.