pharmaceutical industry Archives - European Industrial Pharmacists Group (EIPG)

Steps forward towards the new framework for HTA


By Giuliana Miglierini The long-waited European regulation on Health Technology Assessment (HTA) was adopted by the Council of Europe on November 9, and it has now to pass through the final endorsement of the European Parliament as the last step Read more

Commission establishes portfolio of 10 most promising treatments for Covid-19


by Giuliana Miglierini The second phase in the development of new medicines to treat Covid-19 - a part of the EU Strategy on Covid-19 Therapeutics launched in May 2021 - has reached a cornerstone with the announcement made by the Read more

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


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 Read more

Steps forward towards the new framework for HTA

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By Giuliana Miglierini

The long-waited European regulation on Health Technology Assessment (HTA) was adopted by the Council of Europe on November 9, and it has now to pass through the final endorsement of the European Parliament as the last step before publication in the EU Official Journal. The regulation will entry into force three years and twenty days after publication.

The first proposal of a new HTA regulation was made in January 2018 by the EU Commission; the final political agreement between the Council and the EU Parliament was reached in June 2021. The position of the Council of Europe on the draft regulation at first reading was also published.

The provisions of the new HTA regulation will apply to medicinal products, medical devices (for example pacemakers, dialysis equipment or infusion pumps) or medical and surgical procedures, as well as measures for disease prevention, diagnosis or treatment used in healthcare.

The adoption of this law is another demonstration of how EU countries, when acting together, can achieve very practical results for their citizens. This new law will benefit patients, producers of health technologies and our health systems.”, said Janez Poklukar, the Slovenian minister for health.

Cooperation and joint activities

Joint clinical assessments and joint scientific consultations are central concepts of the HTA regulation: a target that would require the active cooperation of all member states in order to efficiently identify emerging health technologies. Administrative procedures shall be greatly simplified and become more cost-efficient, as manufacturers of health technologies (especially small companies) should be required to submit once-only all data and documentation for a certain technology at the EU level. These will form the basis for national competent authorities to run all joint activities, including scientific advice and clinical assessment.

The added value of new health technologies compared to the existing ones will be a main driver to guide the assessment activities, so to take informed decisions on pricing or reimbursement.

Joint scientific consultations may also include the exchange of relevant information between national authorities and manufacturers on development plans for the technology under assessment, so to favour the availability of all the evidence required to meet regulatory expectations.

The new Heads of Agencies Group

While waiting for the formal adoption of the new HTA regulation by the EU Parliament, other activities are ongoing to set up the operative framework needed to guarantee the smooth activation of all planned collaborative efforts.

The newly formed Heads of Agencies Group (HAG) is an initiative aimed to support the implementation of common joint work approach on all HTA activities at the EU level, according to the new model of cooperation among national authorities established by the regulation.

The new HTA-focused collaborative network for high-level strategic exchange and discussion was launched on 29 September 2021 by the heads of 19 European HTA agencies, which elected Prof. Rui Santos Ivo (INFARMED, Portugal) as its Chair, and Prof. Dominique Le Guludec (HAS, France) and Dr. Trygve Ottersen (NIPH, Norway) as Vice-Chairs. The secretariat of the Group has been established at the Dutch Health Care Institute (ZIN).

All HAG’s activities will be based on a joint Memorandum of Understanding. The Group will work during the next three years to support national systems to be prepared for the entry into force of the HTA regulation, including the availability of the needed capacity. HAG will also support the joint technical and scientific work performed by HTA bodies across Europe, and it will advise policymakers and other relevant institutions – both at the EU and national level – on issues related to cooperation in HTA.

Current members of the group include the following national authorities involved in HTA activities: AEMPS (Spain), AIFA (Italy), AGENAS (Italy), AIHTA (Austria), INFARMED (Portugal), KCE (Belgium), NIPH (Norway), G-BA (Germany), HAS (France), HIQA (Ireland), IQWiG (Germany), FIMEA (Finland), NCPE (Ireland), REDETS (Spain), RER (Italy), RIZIV-INAMI (Belgium), NOMA (Norway), TLV (Sweden) and ZIN (The Netherlands).

The EUnetHTA 21 consortium

After the closing of its third Joint Action (2016-2020), which paved the way to the permanent HTA working structure for Europe (encompassing more than 80 HTA bodies), the European Network for Health Technology Assessment (EUnetHTA) has published a HTA White Paper as the final document resuming lessons learned up to now that may prove relevant for the implementation of the next phase of the HTA joint cooperation.

This new phase in the life of the Network, that now goes under the name of EUnetHTA 21, is no more a Joint Action; a joint consortium has been created instead, led by the Dutch HTA body ZIN and including the following HTA agencies: AEMPS (Spain), AIFA (Italy), AIHTA (Austria), GBA (Germany), HAS (France), INFARMED (Portugal), IQWIG (Germany), KCE (Belgium), NCPE (Ireland), NIPN (Hungary), NOMA (Norway) and TLV (Sweden). The consortium will provide support to the future European HTA system to be established according to the upcoming regulation.

EUnetHTA 21 is funded by a two-years’ Service Contract for the Provision of Joint Health Technology Assessment (HTA) Work Supporting the Continuation of EU Cooperation on HTA, signed on 17 September 2021 by the European Health and Digital Executive Agency (HaDEA).

The first Stakeholder Kick-Off online meeting of the consortium is scheduled on 3 December

2021; the discussion will focus on the illustration of the governance principles, the planned interactions with stakeholders in the form of public consultations and the presentation of deliverables planned for the next two years.

The first Open Call for consultation

EUnetHTA 21 has already launched its first Open Call , targeted to the pharmaceutical industry with reference to four different Joint Scientific Consultations (JSC, previously referred to as Early Dialogues). The Call is open until 7 December 2021; some other four slots for JSC are expected to be activated during the period of activities of EUnetHTA 21.

The medicinal products to access these four first slots will be selected on the basis of the results of the Open Call, within two weeks from its closure; the following Joint Scientific Consultations are expected to start in January 2022. According to EUnetHTA, the procedure to be used for JSC shall remain essentially unchanged, with just minor adjustments; an updated guidance document should be soon available.

JSCs are a pillar of the new HTA regulation, aimed to provide non-binding scientific advice to developers of new products, after completion of the feasibility or proof of concept studies and prior to the activation of pivotal clinical trials, in order to improve the quality and appropriateness of the data to be used for future HTA assessment. This type of evaluation will run in parallel to EMA’s scientific advice procedures.

Early exchange of relevant information between applicants and both regulatory (EMA) and HTA agencies represents the core of the process, so to optimise the integration of the different requirements to be included in the study design across multiple European member states. These might refer, for example, to the choice of comparators or relevant outcomes, to the quality of life and/or patient groups (both for pivotal trials and post-launch studies), as well as to the economic evidence generation plan.


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

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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.