Giuliana Miglierini, Autore a NCF International

Strategic recommendations on the future of EU Research and Innovation programmes

Manufacturing

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24 October 2024

Strategic recommendations on the future of EU Research and Innovation programmes

EU Research and Innovation programmes will play a fundamental role in supporting the competitiveness of the Union. New strategic recommendations on how to maximise their impact were published in October 2024: the Independent expert report was drafted by the high-level group on the interim evaluation of Horizon Europe, upon request by the EU Commission. The proposed actions should be implemented in the last three years of Horizon Europe, 2025-2027, and embedded in future EU R&I programmes.

The twelve expert recommendations start from the adoption of a whole-of-government approach to align R&I with the EU strategy for competitiveness and a clean, digital economy. A stronger framework programme would be needed to boost Europe’s global competitiveness, through a portfolio of actions in different fields. An experimental unit to launch disruptive innovation programmes with fast funding options is also suggested, as well as expanding funding for ERC, EIC and the Marie Skłodowska-Curie Actions. Experts also support the creation of an Industrial Competitiveness and Technology Council and a Societal Challenges Council. The attractiveness of the EU R&I ecosystem would require securing long-term investments, university alliances and Member States’ co-investment. Other recommendations include the reduction of administrative burdens, agile funding and streamlined application processes. Demand-driven solutions should inspire the development of an innovation procurement programme. Dual-use technologies and international partnerships should also be pursued.

Source: European Commission

A transatlantic partnership for the secure supply of medicines

Supply chain

Giuliana Miglierini

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19 September 2024

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<strong>A transatlantic partnership for the secure supply of medicines</strong>

The reciprocal importance of trading partnership between the US and the EU in the field of medicinal and pharmaceutical products has been once more acknowledged by the new transatlantic partnership for the secure supply of medicines signed by the Association for Accessible Medicines, the Canadian Generic Pharmaceutical Association and Medicines for Europe. The partnership will support the respective markets for generic medicines by leveraging the respective strengths. The US, for example, has initiated several projects to reduce certain manufacturing dependencies, while the EU has launched the Critical Medicines Alliance to consider the options to strengthen manufacturing in Europe and create strategic partnerships to improve security of supply.

The partnership also aims to assess how the EU, the US and Canada could cooperate to reduce dependency from other regions in terms of supply of medicines, according to the recent calls from the European Parliament to increase EU-US cooperation and the 2023 EU-Canada Summit joint statement on strategic partnership. Key topics of collaboration are expected to include the security of medicines supply, the setup of solidarity-based responses to crises, and bilateral and multilateral regulatory cooperation.

New molecules to modulate gene expression from an IIT-EMBL study

Research

Giuliana Miglierini

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31 July 2024

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New molecules to modulate gene expression from an IIT-EMBL study

Researchers at the Italian Institute of Technology (IIT) in Genoa and the European Molecular Biology Laboratory (EMBL) in Grenoble have revealed how the process of gene regulation can be modulated using small molecules.

The study, published in Nature Communications, lays the foundations for a possible future identification of new drugs that can act directly on mutations or genetic modifications that alter the process of gene expression, thus intervening, for example, on the onset of tumours or genetic diseases. The proper functioning of cells depends to a large extent on the ability to control gene expression: a complex process through which information contained in DNA is copied into RNA to give rise to all proteins and most of the cell’s regulatory molecules. The study was coordinated by Marco De Vivo, principal investigator at the Molecular Modeling & Drug Discovery Lab and associate director for Computation at IIT in Genoa, and Marco Marcia, group leader at EMBL in Grenoble.

The study brought together the expertise of EMBL and the Grenoble Structural Biology Partnership in biochemistry, biophysics and structural biology, which allowed X-ray photographs of the process to be obtained. For this purpose, the highly automated MASSIF-1 beamline of the EMBL and the European Radiation Synchrotron Facility (ESRF) was used. These were then joined by the computational simulation skills of the IIT, thanks to which it was possible to study the details of the chemical-physical interactions between the molecules involved. The study focused on splicing, one of the key control levels of the gene expression process during which molecular machines in the cell ‘cut and paste’ specific RNA sequences to create functional versions. These ‘mature’ versions of RNA perform various functions, including acting as instructions for protein production or directly as regulators of various cellular processes.
“Studying the RNA splicing reaction, i.e. “cutting and stitching”, is very complex because of both the chemical reactions and the molecular actors involved, such as RNA, proteins, ions and water molecules. Thanks to modern molecular simulation techniques, we have gained a detailed understanding of what happens, and how we can intervene to modulate splicing. Our study has already allowed us to synthesise newdrug-like molecules that can modulate splicing in a new, specific and very effective way,’ Marco De Vivo commented.

Molecules that have been the subject of a patent filed by IIT and EMBL researchers, with the support of EMBL’s technology transfer office. The future improvement of such compounds could, according to the researchers, make it possible to modulate the production of proteins that originate from defective or mutated genes. Visualising at the atomic level the modulation of splicing is exciting. It allows us to control one of the fundamental reactions that enables life. In the future, by continuing to integrate our experimental biological studies with the chemical and computational studies of our collaborators, we will aim at an ambitious goal, that of developing new antibacterial and anti-cancer drugs,’ Marco Marcia emphasised. The research is part of the Italian Institute of Technology’s RNA Flagship initiative dedicated to the development and application of new RNA-based technologies.

The EMRN designated as a WHO Listed Authority

Regulatory

Giuliana Miglierini

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27 June 2024

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The EMRN designated as a WHO Listed Authority

The European Medicines Regulatory Network (EMRN) is the first international regulator recognised as a ‘single entity’ and designated as a ”Regional Regulatory System”. The EMRN was designated in May 2024 as WHO Listed Authority (WLA) by the World Health Organization (WHO). The designation covers each individual regulatory authority of the EMRN, as well as the EMRN overall. The network is composed of the the European Commission, EMA and the 30 national authorities of the European Economic Area Member States. The designation as WHO Listed Authority is a recognition of its advanced level of performance, in compliance with all the indicators and requirements specified by WHO. The WLA initiative aims to ensure the best possible use of limited global regulatory resources by facilitating reliance on the work and decisions of trusted regulatory authorities. WLAs listed regulators can be used as a reference point by other regulatory authorities that lack the resources to perform all functions, or which have not yet reached higher maturity levels for medical product oversight.

EFPIA’s white paper on circular economy

Giuliana Miglierini

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4 June 2024

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<strong>EFPIA’s white paper on circular economy</strong>

EFPIA, representing the research-based pharmaceutical industry at the European level, released a white paper to support the principles of the EU Commission’s Circular Economy Action Plan. Implementation of a circular economy is fundamental to help limit global warming, an objective that is welcomed by the pharmaceutical industry. EFPIA wishes to work collaboratively with the EU in shaping the legislative framework and within its organizations to mitigate our impacts. The white paper addresses the changes throughout value chains needed to transition to a more circular economy, from product design, manufacturing and supply to new business and market models.According to EFPIA, the pharmaceutical industry’s approach to circularity builds on efforts to decarbonise the healthcare sector and aligns with the EU’s approach, even if challenges are present due to highly regulated environment typical of the pharma sector. Circularity and regulation of pharmaceuticals should be carefully balanced, states EFPIA. Consideration should also be made with regard to factors that may impact on pharmaceutical activities, for example on use of secondary raw materials in manufacturing. The influence that EU’s actions may exert on the approach that other countries and jurisdictions take to the issue of circular economy should also be taken into due account, so to safeguard international supply of medicinal products.

Industrial associations’ joint statement on the UE proposal for Compulsory licensing

European Union

Giuliana Miglierini

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30 May 2024

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Industrial associations’ joint statement on the UE proposal for Compulsory licensing

A coalition of several European organizations (EFPIA, Medtech Europe, Eurochambres, Digital Europe, Business Europe) released a new joint statement in view of the preparation of the Council position of the Commission’s proposal for a regulation on EU compulsory licensing for crisis management. Among amendments by the European Parliament positively judged by the associations are the greater involvement of the rights-holder, a requirement for attempted voluntary licensing, and a reasoned approach to remuneration. Nevertheless, according to the statement some problematic provisions would be still present, such as inadequate judicial review and forced technology transfer/trade secret sharing.

The industrial associations position is that the initiative would undermine innovation and competitiveness of European companies, and the resulting weakening of IP iwoulde represent a negative precedent internationally. Main points of concern include the fact compulsory license should be a last resort measure, the exclusion of trade secrets and know-how from the scope of the proposal, and the need for clearer and well-defined definitions. A thorough judicial review of a compulsory license should also be made, according to the statement, and right holders should always be given an opportunity to be heard prior to the adoption of any decision on compulsory licensing.

Source: EFPIA

Medicines for Europe, no to national stockpiling of medicines

Supply chain

Giuliana Miglierini

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16 May 2024

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Medicines for Europe, no to national stockpiling of medicines

A position paper published by Medicines for Europe is the response of the generics and biosimilars industry association to the stockpiling of various types of medicines that several European countries have put in place as a tool against drug shortages. According to the industry representative, warehousing mandates initiated by countries such as France, Germany, the Czech Republic, Austria, Greece, Denmark and Poland could actually worsen the shortage problem by undermining European solidarity mechanisms. According to the note, a six-month national stockpile of antibiotics in Germany would correspond to a quarter of the quantities for the entire EU. Such a six-month stockpile, moreover, could cover the combined supply of eleven small EU countries, the manufacturers’ association adds. There would, the note also points out, also be a problem with the affordability of many low-cost generics, due to the additional costs of stockpile management and the increased risk of unnecessary stock depreciation. Dynamics that, according to Medicines for Europe, could further strengthen the consolidation of the generics market in the hands of fewer and fewer companies. National stockpiles, moreover, could create a false sense of security about the actual ability to mitigate a serious shortage.

Alternative solutions

In its position paper, Medicines for Europe supports the EU strategic stockpile initiative announced by the European Health Emergency and Response Authority (HERA) and proposes some actions that could be included in it.
In particular, it would be necessary according to the industry representative for the EU Commission to strictly monitor national requirements in order to prevent disproportionate and excessive stockpiling actions by individual countries. The voluntary European solidarity mechanism should also allow manufacturers to reallocate stocks from one country to another efficiently in the event of a shortage. An objective that Medicines for Europe believes requires greater transparency of the EU market, together with greater use of the real-time data on demand and supply contained within the European Medicines Verification System (EMVS). As far as European strategic reserves, co-financed by the EU and Member States, are concerned, these should be agreed with manufacturing companies on the basis of stock rotation, so as to reduce wasteful cancellations and greatly reduce costs for taxpayers. These reserves should also be targeted, proportionate and transparent.

Medicines for Europe would also welcome some regulatory changes, such as the electronic package leaflet or the digitisation of the network of regulatory bodies, which could better support agile supply chain management. Finally, for the trade association, it would also be necessary to include security of supply in market policies, as well as in future EU guidelines on the procurement of medicines and the adjustment of reference pricing policies. This would, in fact, encourage companies to invest more in production and diversification activities.

The EU Parliament approved new rules on urban wastewater treatment

Giuliana Miglierini

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9 May 2024

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The EU Parliament approved new rules on urban wastewater treatment

On 10 April 2024 the plenary assembly of the European Parliament adopted the results of the negotiations reached with the European Council in January 2024 on revising the EU’s water management and urban wastewater treatment standards. The Council needs to formally approve the agreement before it can enter into force. The new legislation will greatly impact the pharmaceutical and cosmetic sectors, as a result of the introduction of the extended producer responsibility (EPR) for medicinal products for human use and cosmetic products. EPR is intended to cover the costs of quaternary treatment needed to remove micro-pollutants from urban wastewater. At least 80% of the costs will be covered by producers, complemented by national financing.

According to the approved schedule, by 2035 urban wastewater will undergo secondary treatment to remove biodegradable organic matter in all agglomerations of the size of 1,000 population equivalent or more. By 2039, tertiary treatment (i.e. the removal of nitrogen and phosphorus) will be applied in all wastewater treatment plants covering 150,000 p.e. and above, and by 2045 in those covering 10.000 p.e. and above. Quaternary treatment to remove a broad spectrum of micro-pollutants will be mandatory for all plants over 150,000 p.e. by 2045. Monitoring of various public health parameters (such as known viruses and emerging pathogens), chemical pollutants, including per- and polyfluoroalkyl substances (PFAS), microplastics and antimicrobial resistance is also included in the new legislation.

Source: European Parliament

EU Parliament approved amendments to review of pharmaceutical legislation

Giuliana Miglierini

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22 April 2024

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EU Parliament approved amendments to review of pharmaceutical legislation

The roadmap for the revision of European pharmaceutical legislation saw the EU Parliament, meeting in plenary session on 10 April, approve by a large majority a series of amendments to the Commission’s initial proposal that, in fact, appear to have largely accommodated the demands of the innovator pharmaceutical companies. The proposal for a directive was approved by 495 votes in favour, 57 against and 45 abstentions, the proposal for a regulation by 488 votes in favour, 67 against and 34 abstentions (link to the proposal for a new code for medicinal products for human use and link to the proposal for a regulation on authorisation procedures for medicinal products).

The proposed amendments had already been approved by the Parliament’s Environment, Public Health and Food Safety (ENVI) Committee on 19 March. The legislative process will now pause, to be resumed by the new members of the EU Parliament after the elections in June. “We hope the Council will take note of our ambition and commitment to create a robust legislative framework, preparing the ground for effective negotiations,” commented the directive’s rapporteur, Danish Pernille Weiss. The rapporteur of the regulation, German Tiemo Wölken, added that “We are strengthening our health infrastructure and increasing our collective resilience in anticipation of future health crises, an important milestone in our pursuit of fairer and more accessible healthcare for all Europeans”.

Seven and a half years of regulatory data protection

Among the most significant changes compared to the initial version of the proposal for a new directive, the text approved by Parliament extends the minimum period of regulatory data protection to seven and a half years (against the six proposed by the Commission). During this period, access to data by other companies, such as generic and biosimilar companies, would be prohibited. Obtaining marketing authorisation would also be linked to a two-year extension of market protection, during which no generic, hybrid or biosimilar versions of products could be marketed. The Parliament also approved a number of extensions of the data protection period upon the occurrence of certain events, but rejected the Commission’s proposal to link this to the simultaneous marketing of the medicinal product in all 27 EU countries. More specifically, pharmaceutical companies could obtain an additional year of data protection for products addressing unmet medical needs. The Parliament also approved a six-month extension in the case of conducting comparative clinical studies, i.e. if a significant part of the R&D on the product was carried out in the EU and at least partly in collaboration with EU research institutions. The maximum period of combined data protection could, in any case, not exceed eight and a half years. At the level of market protection, on the other hand, companies could obtain an additional year of (non-replicable) extension in the case of obtaining a marketing authorisation for an additional indication, and provided that this guarantees significant clinical benefits compared to existing treatments. Market exclusivity could be up to 11 years for orphan drugs suitable for treating rare diseases and addressing ‘high unmet medical need’.

OK for voucher for new antimicrobials

Actions to support the development of new antimicrobial agents include Parliament’s approval of ‘transferable data exclusivity vouchers’, which would benefit companies engaged in the development of priority antimicrobials. The approved scheme provides for the possibility to extend data protection for authorised products for up to 12 months. The voucher would also not be usable for products that have already benefited from the maximum possible period of regulatory data protection, and would only be transferable once to a second MA holder. MEPs also voted in favour of the introduction of market entry rewards and milestone payment reward schemes, based, for example, on early financial support following the achievement of certain R&D goals prior to AIC authorisation. To complement this, and to encourage the development of antimicrobials, the Parliament also envisaged the possibility of a subscription-based model based on voluntary joint procurement agreements.

New partners for Darwin EU

Giuliana Miglierini

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21 March 2024

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The Data Analysis and Real World Interrogation Network DARWIN EU has reached twenty partners (public or private institutions) from 13 European countries. The Network aims to generate real-world evidence (RWE) from sources such as hospitals, primary care, health insurance, registries and biobanks to support regulatory activities of EMA’s scientific committees and national regulators in the EU.

The current capacity of the DARWIN EU network refers to data from approx. 140 million patients across Europe, which are converted into a common data model. Data are stored and analysed locally to ensure the maximum level of protection of personal data.

Since the establishment of the Network in 2022, 14 RWD studies have been completed and eleven are ongoing. The plans for the next couple of years include the scaling up of the output of studies, and the on-boarding of some ten new data partners in 2024, and another ten in 2025. The final objective is to deliver over 70 RWD studies this year, and over 140 studies per year from 2025 onwards to enhance more data-driven decision making on medicines in the EU.

Source: EMA

Giuliana Miglierini

Alternative solutions

Seven and a half years of regulatory data protection

OK for voucher for new antimicrobials

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