IRIS platform Archives - European Industrial Pharmacists Group (EIPG)

A new member within EIPG


The European Industrial Pharmacists Group (EIPG) is pleased to announce the Romanian Association (AFFI) as its newest member following the annual General Assembly of EIPG in Rome (20th-21st April 2024). Commenting on the continued growth of EIPG’s membership, EIPG President Read more

The EU Parliament voted its position on the Unitary SPC


by Giuliana Miglierini The intersecting pathways of revision of the pharmaceutical and intellectual property legislations recently marked the adoption of the EU Parliament’s position on the new unitary Supplementary Protection Certificate (SPC) system, parallel to the recast of the current Read more

Reform of pharma legislation: the debate on regulatory data protection


by Giuliana Miglierini As the definition of the final contents of many new pieces of the overall revision of the pharmaceutical legislation is approaching, many voices commented the possible impact the new scheme for regulatory data protection (RDP) may have Read more

EMA’s pilot scheme for academic and non-profit development of ATMPs

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by Giuliana Miglierini

Advanced therapy medicinal products (ATMPs) are often developed by academic and non-profit organisations, because of their high level expertise in the biotechnological techniques that underpin many new therapeutic approaches. On the other hand, these organisations often lack sufficient understanding and experience to face the complexity of the regulatory development.

To improve the possibility for non-commercial developers to access regulatory and scientific support related to promising ATMPs addressing unmet medical needs, a pilot was launched by EMA in September 2022. Three projects have been already selected to participate to the pilot. ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells, developed by the Hospital Clínic de Barcelona, was the first project to access the pilot. The product was granted eligibility to the PRIME scheme in December 2021, and is targeted to treat patients older than 25 years with relapsed/refractory acute lymphoblastic leukaemia.

The second call closed in December 2023 and saw the participation of 11 candidates, among which two new academic organisations were selected. The Berlin Center for Advanced Therapies (BeCAT) – Charité is developing TregTacRes, a gene therapy based on modified T-cells, for use as add-on therapy after transplantation. Fondazione Telethon’s gene therapy Telethon 003 (etuvetidigene autotemcel) targets the Wiskott-Aldrich syndrome, a rare, life-threatening immunodeficiency.

The new phase of the project will now recruit a total of 5 new participants by the end of 2024. The first results of the pilot are expected in 2025.

How to apply
Interested academic organisations can find all information together with the ATMP Pilot Application Form on the dedicated EMA webpage; applications are open up to the end of April 2024. The email address [email protected] is also available to request more information or to express interest in participating. A guideline document on fee incentives for scientific advice, marketing authorisation (MA) applications and pre-authorisation inspections for academic participants is available, together with Q&As on the pilot.

Pre-selected candidates will be invited to a meeting with EMA’s Innovation Task Force (ITF) to provide further information on their projects before final selection. At this stage, interactions between EMA and applicants would mainly take place via the online platform IRIS. Therefore, interested organisations will need to register to the platform and request a research product identifier (RPI).

Requirements and procedure for the application
Academic developers active in the EU can apply to the pilot provided they have already generated some proof-of-principle data on the interested ATMP. The academic status of the organisation will be checked by EMA during the selection phase. Applicants may include public/not-forprofit hospitals or research organisations and hospitals, Higher Education Institutions (HEI), public-private partnerships/consortia, and international research organisations, provided they are establish in the EU. In case of projects comprehensive of non-EU participants, the principal investigator has to be located in the EU, and clinical trials must include EU patients. The academic sponsor must be free from operating agreements with any pharmaceutical company, and it can freely operate via intellectual property rights on the product.

The support provided by EMA aims to ensure that development activities would meet regulatory standards as for quality, safety and efficacy of the ATMP product. A smooth path towards the submission of the MA application based on existing regulatory procedures and tools should therefore be possible. The pilot also aims to identify potential gaps in existing tools and procedures, from the perspective of academic sector developers.

Key principles used to select the new participants are listed in the Q&As document. As for individual ATMPs under development, they must address an unmet medical need, represent a major therapeutic advantage over existing treatments, or offer a new option in orphan areas. Previous eligibility to the PRIME scheme is not a prerequisite to apply for the pilot. The Q&As also specify that there is no direct link between the product having received an hospital exemption (HE) and access to the academic pilot.

Preliminary clinical evidence in patients is needed to support the application, as well as information on the mechanism of action gained by non-clinical studies. A sufficiently mature quality development, to be assessed against the pharmaceutical process and the planned GMP manufacturing process, should be also available to better support later stage clinical development and/or a MA application in the EU.

The academic sponsor must also have full access to the data related to the development and manufacture of the product, e.g. control of critical starting materials. The knowledge needed to successfully interact with EMA may be ideally provided by a specific person (also a consultant) appointed by the sponsor and with experience in the field of product development and regulatory affairs.

Benefits and fee reductions
Selected academic organisations will benefit by a dedicated EMA’s point of contact in the relevant therapeutic area office. A EMA Support team may be also appointed to provide regulatory and scientific support depending on the stage of development and nature of the program. Activities to be part of the pilot may include preparatory teleconferences to check planning, identify potential needs for additional support and complement interaction mechanisms under existing tools. The optimisation of pre-submission meetings is another goal of the pilot, together with debriefings before and/or after regulatory interactions. A particular attention will be payed to the regular assessment of the level of maturity of the projects, including co-decisions and stopping points.

EMA will also provide financial support to the selected academic applicants for the activities concerning the five selected ATMPs. More in particular, the Agency will grant the same incentives as for micro-, small- and medium-sized enterprises, with respect to fees established by the Council Regulation (EC) No 297/95 and its Implementing Rules.

To qualify for the fee incentives, selected academic organisations must continue to fulfil all the above mentioned criteria for accessing the pilot also at the time of the request for a fee incentive related to a procedure or service to be provided as a part of the pilot itself. To this instance, applicants shall submit a declaration to EMA, inclusive of the fulfilment of requirements and establishment in the European economic area.

Incentives for academic organisations participating to the pilot include a 90% fee reduction for both initial scientific advice and follow-up, and pre-authorisation inspections. MA applications for designated orphan medicinal products for human use will benefit a 100% fee reduction, while MA applications not covered under this occurrence will see deferral of payment until the notification of the final decision on the MA for the concerned ATMP is issued.

The document on fee incentives specifies also that remuneration of national competent authorities for those activities shall not be reduced.

The granting of fee incentives will follow EMA’s verification of the documentation submitted by the applicants. After confirmation by the Agency the applicant qualifies for the fee reduction, participants to the pilot will have a six months period to submit their requests for scientific advice and/or marketing authorisation. Ex–post controls and prove of evidence confirming the fulfilment of criteria for the fee reduction may also be required at any time until the finalisation of the concerned procedure.


EMA’s Industry stakeholders group (ISG)

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by Giuliana Miglierini

The Industrial Stakeholder Group (ISG) is a new initiative recently launched by the European Medicines Agency (EMA) in order to favour the dialogue with the industrial stakeholders. The first meeting of the ISG, the 21 June 2022, focused on the mandate of the Group and on the three priority topics to be addressed during the pilot phase: the Emergency Task Force (ETF), the issue of shortages of medicines and medical devices and the medical device expert panels.

The initiative is part of the activities planned by EMA for the implementation of its extended mandated, as for Regulation (EU) 2022/123.

The mandate of the ISG

The main scope of the ISG is to provide a dedicate forum to capture the industrial point of view and proactively inform on open issues during the implementation of EMA’s extended mandate. The ISG will focus on human medicines and will complement other existing tools, such as industry platform meetings, bilateral meetings, topic or project related meetings. The outcomes obtained from the pilot phase will form the basis of an analysis to evaluate if to extend the scope to other initiatives.

The Chair of the ISG is nominated by the Agency’s Executive Director; the group is composed by one member and one alternate from selected EU industry organisations relevant to the subject of discussion, on the basis of a call for expression of interest. Additional representatives of selected organisations and observers may also participate to specific meetings, according to the topics on the agenda. Observers include the European Commission, EMA’s committees (e.g. CHMP, ETF, CMDh, SPOC WP, SMMG), the EU Network, Notified bodies; ad-hoc observers may be also invited from member states and stakeholder groups.

Appointed members will be responsible to liaise with the respective industrial rganisations, so to contribute the discussion with their point of view and to keep them updated on the outcomes of the ISG meetings. The current schedule includes four quarterly meetings per year; the next two are fixed for the 26 September and 22 November 2022. The summary report of each meeting will be available in EMA’s website.

The Emergency Task Force

The new Emergency Task Force (ETF) builds upon the experience gathered during the pandemic and acts within EMA to advise and support on medicines for public health emergencies and preparedness.

The ETF is in charge of coordinating all efforts following the declaration of a public health emergency by health authorities, in strict coordination with all other relevant bodies including the European Health Emergency preparedness and Response Authority (DG HERA), the European Centre for Disease Prevention and Control (ECDC), the WHO and the European Commission.

The new ETF started operating on the new mandate on 22 April. Its composition is based on expertise, and it includes representatives of EMA’s Scientific Committees and Working Parties as well as selected patients and healthcare professionals and clinical trials experts from various member states.

There are three distinct area of activities for the Task Force. Scientific advice and support to clinical trials for the development of medicines to be used during the emergency will be directly managed and assessed by the ETF, free of charge and flowing a fast-track procedure. The new streamlined procedure should lead to the outcome in 20 days; deceleration criteria are also considered, i.e. premature evidence to address the medical need, high workload or lack of urgency. Expected benefits include the reduction of the use of medicines with insufficient evidence of efficacy and the increase of safe and harmonised use across the EU of new products from the pipelines ahead of authorisation. Activities of the ETF will cover all stages of development, from pre-authorisation (e.g. rolling applications or paediatric plans) to post-authorisation (e.g. major changes), investigational products and compassionate use.

The systematic assessment of the available evidence on medicines will be the focus of the scientific reviews, while recommendations will target medicines not yet authorised or topics of particular scientific or public interest. These may include, for example, the monitoring of new outbreaks and epidemics and the information on potential radiological, chemical or bioterrorism agents.

All lists of medicines under assessment to address a declared emergency will be made public to increase transparency, as well as the CHMP opinions on the use of medicines not yet authorised, Product Information, EPARs end Risk Management Plans.

Two dedicated mailboxes are also available, the first for sponsors of clinical trials to request EMA/ETF support for facilitating CTA and approval and sponsors agreement to conduct larger multinational trials ([email protected]), the second for manufacturers to discuss with EMA/ETF their development programs or plans for scientific advice prior to any kind of formal submission ([email protected]).

Shortages of medicines

EMA’s extended mandate in this area include the monitoring and mitigation of shortages of critical medicines and medical devices, and the setting up, maintenance and management of the European Shortages Monitoring Platform (ESMP). The action also includes the establishment of the Medicines Shortages Steering Group (MSSG), which will be supported by the Working Party of singles points of contacts in the members states (the EU SPOC Network) and a network of contact points from pharmaceutical companies (the i-SPOC system). A corresponding Executive Steering Group on Shortages of Medical Devices (MDSSG), to be created by February 2023, will be in charge of adopting the list of categories of critical medical devices and to monitor their supply and demand.

According to Regulation (EU) 2022/123, pharmaceutical companies are required to identify a i-SPOC to act as the reference contact for EMA should the Marketing Authorisation Holder (MAH) have medicinal products be included in the lists of critical medicines. All information has to be provided through the IRIS platform; the registration process opened on 28 June 2022 and is comprehensive of two steps (the IAM preliminary requirement for the creation of the account and the following IRIS submission).

Scheduled milestones will see the establishment of a list of the main therapeutic groups for hospital care (due by 2 August 2022), the registration of i-SPOCs from MAHs (by 2 September 2022), and the definition of shortages of medical devices and in vitro diagnostics (by 2 February 2023). The ESMP platform is expected to go live by 2 February 2025, and will represent a single reference point to make information available on shortages, supply and demand of medical products, including the marketing status and cessation.

Expert panels on medical devices

Regulation (EU) 2022/123 establishes the hangover of expert panels on medical devices from the Joint Research Centre (JRC) to EMA, thus adding a completing new type of activity for the Agency.

The new Secretariat is coordinating the activities of the Screening panel composed by 70 experts in charge of the decision whether to provide a scientific opinion, eleven thematic expert panels and expert panels sub-groups (for a total of approx. 130 experts), and a Coordination Committee inclusive of the Chair and vice-Chair of all the expert panels.

The main task of the expert panels is to provide opinion to the notified bodies for certain high-risk medical devices and in-vitro diagnostic, for the assessment of their clinical and/or performance evaluation. EMA is specifically involved in the coordination of the Clinical Evaluation Consultation Procedure (CECP) for medical devices and Performance Evaluation Consultation Procedure (PECP) for in-vitro diagnostics. Further details on the procedures and their interfaces with the ETF is available here.