Business & Policies Archives - European Industrial Pharmacists Group (EIPG)

Lessons learnt to transition from Horizon 2020 to the new FP10


by Giuliana Miglierini The European Commission published the ex post evaluation of Horizon 2020 (H2020), the FP8 framework programme for research and innovation (R&I) run in years 2014-2020. The report identifies several areas of possible improvement, which may be taken into Read more

Approvals and flops in drug development in 2023


by Giuliana Miglierini Approvals and flops in drug development in 2023 The European Medicines Agency published its annual highlights, showing 77 medicines were recommended for marketing authorisation, and just 3 received a negative opinion (withdrawals were 19). In 2023 some highly expected Read more

Webinar: Oral Colon Drug Delivery - Design Strategies


EIPG webinar Next EIPG webinar is to be held on Wednesday 21st of February 2024 at 17.00 CET (16.00 GMT) in conjunction with PIER and University College Cork. Anastasia Foppoli, will discuss on the various approaches and the general aspects Read more

Approvals and flops in drug development in 2023

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by Giuliana Miglierini

Approvals and flops in drug development in 2023

The European Medicines Agency published its annual highlights, showing 77 medicines were recommended for marketing authorisation, and just 3 received a negative opinion (withdrawals were 19).

In 2023 some highly expected candidates under clinical development failed to meet the fixed endpoints, as reported by Fierce Biotech. The impact of the Covid-19 pandemic reduced the commercial performance of medical products launched in 2020, highlights the Trinity Annual Drug Index. We summarise the main features emerging from the three documents.

The approval of the first CRISPR/Cas-9 gene therapy

The only advanced therapy medicinal product (ATMP) recommended by EMA in 2023 represents a true innovation in the therapeutic arsenal to treat transfusion-dependent beta-thalassemia and severe sickle cell disease. Casgevy (exagamglogene autotemcel) is the first-in-class CRISPR/Cas 9 gene therapy approved, targeting specific mutations in the genome of patients that affect the production or function of haemoglobin.

EMA recommended in 2023 39 medicines based on a new active substance never authorised before in the EU. Generics and biosimilars were about a third of the approved products (14 and 8, respectively). On the other hand, 17 products received an orphan designation. Other new medicinal products followed different dedicated regulatory pathways, such as Prime (3) or accelerated assessment (3). One product received approval under exceptional circumstances, other 8 a conditional marketing authorisation.

Oncology continues to represent the most attractive therapeutic area for pharmaceutical R&D, with a total of 14 new medicinal products.

Elrexfio (elranatamab) and Talvey (talquetamab) were approved for the treatment of adult patients with relapsed or refractory multiple myeloma, a rare cancer of the bone marrow that affects plasma cells. Two other new medicines – Columvi (glofitamab) and Tepkinly (epcoritamab) – were approved for the treatment of diffuse large B-cell lymphoma, an aggressive cancer of the lymphatic system. The treatment of myelofibrosis, a rare blood cancer that affects the bone marrow, can benefit from the approval of Omjjara (momelotinib). Cerebral glioma in paediatric patients from one year of age is the target of the combination of Finlee (dabrafenib) and Spexotras (trametinib).

Among other particularly innovative products recommended for approval by EMA are two vaccines to protect against lower respiratory tract disease caused by respiratory syncytial virus, Abrysvo (bivalent, recombinant) targeting small infants via immunisation of the mother during pregnancy (and over-60 adults), and Arexvy (recombinant, adjuvanted), representing the first vaccine for active immunisation of adults aged 60 years and older.

EMA also recommended two medicines for use in countries outside the EU, under the regulatory procedure “EU-Medicines for all” (EU-M4All). Arpraziquantel (arpraziquantel) targets schistosomiasis, a neglected tropical disease caused by parasitic trematode worms and affecting an estimate of 50 million young children. Fexinidazole Winthrop (fexinidazole) is already in use from 2018 to treat human African trypanosomiasis, a disease caused by the parasite trypanosoma brucei gambiense and also known as sleeping sickness. The CHMP extended the indications to include treatment of the more acute and lethal form of the disease caused by trypanosoma rhodesiense.

The main failures in clinical R&D

Pharmaceutical R&D may also lead to failure of the clinical development for candidate products. A selection of the more significant flops in 2023 as for clinical trials has been published by Fierce Biotech on its website.

An already FDA approved gene therapy product is also included in the list, Sarepta’s Elevidy for the treatment of Duchenne muscular dystrophy, as its phase 3 Embark study didn’t meet the primary endpoint. The product is now under further scrutiny by the FDA. As for vaccines, a major failure refers to Janssen Pharmaceuticals’ HIV vaccine and its phase 3 Mosacio study, that was terminated as it was not expected to meet the primary endpoint. According to Fierce Biotech, Johnson & Johnson would have ended the development of the HIV vacci-ne and completely revised the infectious disease R&D unit. Failure to meet the expected benefit (3.5-month overall survival) in the phase 3 Sapphire trial impacted also sitravatinib, a spectrum-selective kinase inhibitor developed by Mirati Therapeutics to overcome resistance to checkpoint inhibitors in the treatment of non-small lung cell carcinoma. Tarcocimab tedromer is an anti-VEGF antibody biopolymer conjugate developed by Kodiak Sciences to treat diabetic macular edema, and that did not meet the primary endpoint in a phase 3 trial compared to the approved therapy. The same occurred to evobrutinib, a BTK inhibitor from Merck KGaA to treat multiple sclerosis, that failed the comparison with the reference product in two phase 3 studies. The failure of the potential blockbuster factor-XIa inhibitor asundexian, developed by Bayer for treatment of atrial fibrillation with stroke risk, was due to an observed “inferior efficacy” com-pared to the standard treatment Eliquis. The failure of efruxifermin (a FGF21 analog) in a phase 2b study aimed to treat fibrosis in cirrhotic MASH patients was attributed by Akero Therapeutics to the fact enrolled patients may have reached a too advanced state of disease for the treatment to be effective. The failure of Nektar Therapeutics’ phase 2 clinical trial in lupus with Rezpeg (rezpegaldesleukin) is a less typical occurrence, as it was due to errors made by the industrial partner Eli Lilly in the analysis of data from a phase 1b trial in eczema and psoriasis. Lilly admitted the errors and was then sued by Nektar.

The land of unicorns also crashed down when izokibep, a small protein developed by Acelyrin, failed the primary endpoint against placebo. The company had received a $540 million IPO, to then see its shares value decreasing by 58%. The failure was attributed to a programming error by a CRO, which according to Fierce Biotech is under investigation by the sponsor. The potential of artificial intelligence in supporting drug discovery may also be impacted by the failure of BEN-2293, a topical pan-Trk inhibitor in eczema developed by Benevolent AI which failed to meet the secondary endpoints of the safety-focused study.

The commercial performance of products approved in 2020

The commercial performances of novel drugs approved in 2020 are the focus of the Trinity Annual Drug Index.

Oncology represented in 2020 the leading indication (29% of the total 58 unique FDA drug and biologic approvals), followed by neurology (16%). The combination of the two therapeutic areas marked a net increase compared to 2017 (45% vs 34%, respectively). Half (9/17) of the new pro-ducts approved in Oncology were small molecules, mainly mutation directed. A quarter (24%) of the new medicines were monoclonal antibodies. The antibody drug conjugates Trodelvy, in particular, was the highest performing Oncology drug overall.

The strong impact of the Covid-19 pandemic on the pharmaceutical industry in 2020, with many shifts of priorities in development and the need to manage shortages and disruptions of the supply chain, led to a lower commercial performance of the new products launched com-pared to 2016-2019. Good commercial results were obtained only by new medicines addressing significant unmet need or providing very strong therapeutic benefits.

The Trinity Annual Drug Index also highlights that approx. 21% (12/58) of approved products in 2020 constituted a “first launch” for their respective companies. None of them surpassed their forecast expectations, and approx. a half significantly underperformed.


European Council’s recommendations on R&I

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by Giuliana Miglierini

The end of 2023 saw some steps forward to better support the European framework on Research and Innovation (R&I). The Council of Europe approved on 8 December 2023 its conclusions on the impact of research and innovation (R&I) in policymaking. The Council also reached a political agreement on a recommendation of a framework supporting researchers and research careers in the EU. R&I is strategically important as one of the main tools to make Europe more attractive to young talents and to create a open and sustainable European labour market for researchers, innovators and entrepreneurs. We summarise the main features of the Council’s decisions.

How to support the European R&I

The Council conclusions were proposed by the Spanish Presidency (Ministry of Science, Innovation and Universities), and they represent one of its main priorities in the area of research and innovation.

Three mutually complementary dimensions have been identified as fundamental to the success of European R&I. Science plays an important role to reinforce the political process of decision making, which in turn is key to improve life conditions of EU citizens and strengthen democracy. To this instance, the inclusion of scientific evidence and knowledge in the regulatory process and a better coherence of policy initiatives in different areas are deemed important by the Council. According to the conclusions, such an inclusion should help to improve the response capacity of the EU and member states against both structural and cyclical or circumstantial challenges. The document also recalls the ‘Science for Policy’ concept and the EU’s long-standing tradition of relying on science and evidence-based knowledge in all disciplines to support decision-making.

The availability of strong R&I ecosystems in all member states is deemed fundamental to sustain EU’s competitiveness and should be supported among others by the implementation of open-science policies and new technologies and innovation, including social innovation.

The best available scientific evidence should also always be included in impact assessments, so to improve citizens’ trust in public action, as well as the added value of the legislation. To this instance, a rigorous methodological framework would be needed, even though uncertainties are still possible. Transparent and responsible communication would support a better dissemination of scientific outcomes at all levels. The Council also recommended the mapping of the existing practices of knowledge valorisation in policymaking and the national institutional scientific advisory systems and mechanisms. The Commission should also extend the use of the Technical Support Instrument and the Policy Support Facility to support public policymakers and strengthen public structures for scientific advice.

Local and regional innovation ecosystems and ERA’s R&I

R&I may also represent a boost to enhance cooperation and territorial cohesion, reduce R&I fragmentation and disparities between and within member states and to sustain the creation of regional and local innovation ecosystems. Their design should aim to build synergies between cohesion policy and R&I funds. To this regard, according to the Council the R&I framework programme (i.e. Horizon Europe) should continue to drive research excellence in all member states.

Regional centres of excellence may represent a particularly interesting tool to support the regional dimension, with a special attention to the less innovative ecosystems. This goal is part of the New European Innovation Agenda (NEIA), as well as the Regional Innovation Valleys and the pilot project of the Partnerships for Regional Innovation. Cross-border cooperation (especially between less and more innovative member states and regions) may also be key to support better economic, social, and territorial cohesion and reinforce R&I efficiency.

The third dimension is referred to the policy impact of the Recovery and Resilience Facility (RRF) on the design of R&I policies in the European Research Area (ERA) after the pandemic crisis. This last occurrence had a positive effect in enabling many actions at the national level, allowing for targeted investments and reforms. The new ERA should be based on trust, shared responsibilities, and societal engagement and diversity.

Many sectoral and R&I policies experienced a joint approach to their improvement, including the additionality of the Facility with other EU funds. The Council invited the Commission to run a separate study that complements the mid-term evaluation of the RRF, expected by February 2024. The exercise should consider the differences between the RRF and other EU funds.

The reform of research careers

The political agreement reached by the Council on the proposal of a European framework to attract and retain research, innovation and entrepreneurial talents in Europe updates the R1- R4 profiles for researchers, introduced in 2011. It also introduces the European Charter for Re-searchers (ECR), a revision of the 2005 ECR and the Code of Conduct for the Recruitment of Researchers.

The revised definition of researcher and the related research activities are expected to widen career options, thus making European R&I framework more attractive for both internal and foreign talents.

According to the proposal, the term “researcher” would identify professionals engaged in the conception or creation of new knowledge, active in basic or applied research, experimental development, operating research equipment, or project management within any sector of the economy or society (i.e.academia, business, governmental laboratories and the public administration, and the non-profit sector). Careers in research management are also included in the definition.

Four different profiles have been identified to describe the career steps of researchers. First Stage Researcher (R1) are doing research under supervision up to the point of a PhD or equivalent level of competence and experience. Recognised Researcher (R2) hold a PhD or equivalent level of competence and experience but are not yet fully independent in their ability to develop their own research, attract funding, or lead a research group. R1 and R2 refer to researchers at the beginning of their career in science. R3 and R4 refer to senior researchers. Established Re-searcher (R3) holds a PhD or equivalent level of competence and possesses sufficient experience to independently develop and run their own research. Leading Researcher (R4) are recognised as leading their research field by their peers.

The Council recommends that these profiles are referenced to by members states in all vacancies specifically addressed to researchers. Member states are also called to promote equal esteem and reward of the different paths of research careers, regardless of the sector of employment or activity. Appropriate measuring should support comparison of careers across member states, sectors, and institutions, so enabling their full interoperability. The Council recommendation also aims to reduce the precarity of research labour by promoting adequate social protection measures. Inter-sectoral mobility is also encouraged, as well as better equality in research careers, as a tool to respond to the request of highly skilled talents. The Council expects that all organisations employing or providing funding for researchers would provide endorsement of the new “European Charter for Researchers”.


The risk of a biosimilar void in Europe

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by Giuliana Miglierini

The undergoing revision of the pharmaceutical legislation aims, among others, to redefine data protection to better support competitiveness of generics and biosimilars and to favour the timely access of patients to treatments.

While the innovator pharma industry is claiming the proposed reform would reduce the attractiveness of Europe for R&D activities, a recent report from Iqvia analysed the status of biosimilar competition. According to the document, not all biological medicines experiencing loss of exclusivity (LoE) in the next decade would automatically face competition by the corresponding biosimilars. This would result in the creation of a “biosimilar void” on the market, with many originators losing protection without seeing the parallel development of their biosimilar versions.

Competition is not guaranteed

Biosimilar competition is not necessarily guaranteed, and emerging dynamics pose a risk to conventional notions of medicines lifecycles, states the report since its very beginning. The analysis refers to biological medicines that will lose protection in the period 2023-2032.

Despite the approx. 8-fold expected increase in LoE opportunity by value between 2012 and 2032 (from €4.4 billion to €32.2 bln, as result of loss of exclusivity for 110 biological medicines), data show a declining trend for years 2021-2023 (€4.3 bln). According to Iqvia, more than a half (55%) of biologics with LoE in the period 2023-2027 might experience the lack of a biosimilar in development.

The report highlights five areas of common perception to be addressed to better define the issue. The increasing complexity of many biological medicines coupled to new barriers to entry is one of the factors making the development of biosimilars interesting only for products referred to originators with large market shares. According to Iqvia, 27% of the 26 high-sales products that will reach loss of exclusivity by the end of 2032 do not have yet a biosimilar candidate in development in Europe (vs 45% at the global level), corresponding to a potential loss of approx. €8 bln market opportunity. The number of biosimilar candidates in the pipeline for high-sales biologics is also expected to decrease from 2027 onwards.

Regulatory hurdles, therapeutic classes, and disease indication are expected to play a greater role in guiding decisions on biosimilar development, indicates the report. The attractiveness of the European market should also be considered. Oncology will remain the more interesting area, with 44% of all candidates in early to late development for LoE events occurring between 2023 and 2027. Immunology and ophthalmology are other therapeutic areas that might experience growing competition.

The current barriers to biosimilar development

According to Iqvia, the main constraints limiting the decision on biosimilars development are represented by cost and time. In the oncology area, for example, high costs have to be considered to purchase the reference comparator biologic medicine, and large patients populations are required to demonstrate relevant clinical endpoints. New therapeutic classes, i.e., PD-L1/PD-1 inhibitors, may also pose challenges for the design of pharmacokinetic and equivalence studies. From the manufacturing perspective, the increasing use of antibody-drug conjugates (ADC) would result in new barriers to entry.

According to Iqvia, the least attractive products for biosimilars development are those with less than €500 million annual sales in Europe. The report shows 93% of these products might fail to see biosimilar competition, compared to 27% of high-sales medicines. This negative trend would result in a “biosimilar void” corresponding to approx. €15 bln in lost savings. Iqvia also identified some exceptions that might experience a niche development, on the basis of specific technological and manufacturing know-how, platforms and market access excellence.

Another factor to be considered is reimbursement rate, that the report identifies in 51% for low-sales biologics with no biosimilar pipeline (approx. 30% lower than for products with a biosimilar pipeline). The management of the intellectual property referred to the originator should be also taken into consideration.

Orphan and one-off medicines

Despite the growing number of new biologics reaching marketing authorisation as orphan medicines, according to Iqvia biosimilar development is undergoing by now for only one product (eculizumab). No other orphan biologics are expected to face biosimilar competition in future, as annual sales of the 39 orphan medicines currently on the market are too low (approx. €105 mln).

A major factor limiting the development of biosimilars for orphan medicines is linked to the fact many of these therapies fall in the antibody-drug conjugates (ADC) and cell- or gene-therapies (ATMPs) categories (wave 3 biologics). This implies many challenges from the development and manufacturing point of view, higher upfront investments and a more complex setup for analytical and clinical testing.

According to Iqvia, there are currently 16 non-orphan biosimilar candidates under development, corresponding to wave 3 biologics. A limiting factor for this pipeline is identified in the still present fragmentation of the European regulatory system, e.g., reimbursement policies, incentives, and clinical standards. ATMPs, also referred to as one-off therapies, represent a particular case, being relatively young on the market. This leads to no expectation of LoE events in the next five years. The trend would then change, with some 10 products losing protection by 2040, but it should be considered together with the parallel declining of the number of eligible patients, as many of them might have been already treated with the one-off originator medicine.

Shifting standards of care

Another factor analysed by Iqvia is the impact on biosimilars development of the possible changes in the current standards of care, for example resulting from the availability of new and more user-friendly formulations of the originator (i.e., subcutaneous vs intravenous injections). The availability of second- and third generation versions of the original biologic should be considered as another factor limiting the possible market share of a biosimilar of the first-generation product. The picture is indeed furthermore complicated, as another frequent possibility, especially in the oncology area, sees the development of combination therapies based on the use of two or more biologics. As already said, some of them might be very costly (i.e. monoclonal antibodies and PD-1 inhibitors), and require a larger study population to demonstrate equivalence of the add-off effect.

The proposed solutions to fill the biosimilar void

The Iqvia report proposes several possible solutions to overcome the expected biosimilar void, starting from horizon scanning activities aimed at early identification of upcoming LoE events in order to prevent contractions in biosimilar development. Horizon scanning may also support market entry and granting of incentives based on demand. The development of biosimilars of orphan medicines might benefit of a default waiver of comparative efficacy studies, a suggested measure that according to Iqvia would not compromise the demonstration of biosimilarity. Improvements at the regulatory level might also help to streamline development, together with global convergence of regulatory guidance. Iqvia also suggests the adoption of clear regulatory pathways to incentivise the development of the next-generation, one-off biosimilar gene- and cellular treatments. Access might be improved by optimisation of market conditions, with incentives for clinicians combined with the introduction of prescription targets. New tender models would also be needed to favour multi-winner procurement practices.


EP’s draft position on Unitary SPC and SPC Regulation revision

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by Giuliana Miglierini

The Committee for Legal Affairs (JURI) of the European Parliament released the draft amendments to the Commission’s proposals aimed to establish a Unitary Supplementary Protection Certificate (SPC) (links to the document and to the procedure) and to revise the current SPC Regulation (links to the document and the procedure).

On the dedicated pages of EP’s website, you can also find the opinion issued by the Consultative Working Party, according to the Inter-institutional agreement of 28 November 2001 on a more structured use of the recasting technique for legal acts.

A document analysing the potential impact of the Unitary SPC on access to health technologies was also prepared by the Policy Department for Citizens’ Rights and Constitutional Affairs Directorate-General for Internal Policies in September 2023.

We summarise the main features of the EP’s draft positions, which were discussed in the 7 November meeting of the JURI Committee.

The revision of the current SPC Regulation

The JURI Committee (Rapporteur Tiemo Wölken) moved to Recital 2 the statement that “medicinal products, in particular those that are the result of long, costly research will not continue to be developed in the Union unless they are covered by favourable rules that provide for sufficient protection to encourage such research”. Recitals 3 and 5 of the original proposal have been deleted, the last one referring to the risk research centres located within the EU might move to countries offering greater protection. The new Recital 2 makes now reference to the difficulty of establishing a direct link between favourable protection rules and EU competitiveness. If, on the one hand, it would be true that the attractiveness of EU markets might benefit from favourable protection, on the other it should be taken into account that European incentives can be granted also to authorised medicines from third countries. Furthermore, UE-based innovative companies can equally benefit from incentives in third countries.

Recital 13, referring to the request of a marketing authorisation for a biological medicinal pro-duct identified by its International Nonproprietary Name (INN), has been amended to indicate that the protection conferred by the SPC should extend to all biosimilars (and not to therapeutically equivalent products, as previously indicated).

A reference to Article [86] of the new Directive (EU) …/… [2023/0132(COD)] to be approved has been introduced in Recital 24, concerning fees that can be charged by the European Union Intellectual Property Office (EUIPO) with reference to centralised application for SPCs for paediatric medicinal products.

The newly inserted Recital 41 a highlights the importance of the timely entry of generics and biosimilars in the EU market, as it may support competition, reduction of prices, sustainability of national healthcare systems and access to affordable medicines.

Several amendments have been proposed for Recital 45. Among the main ones is the reference to the opportunity “to restrict the protection conferred by a supplementary protection certificate in accordance with Regulation (EU) 2019/933 so as to allow making for the exclusive purpose of export to third countries and any related acts in the Union strictly necessary for making or for the actual export itself […]”. The JURI Committee referred to “related acts” as those that “could include the possession, supply, offering to supply, import, using or synthesis of an active ingredient for the purpose of making a medicinal product containing that product, or temporary storage of the product or advertising for the exclusive purpose of export to third-country destinations”.

A phrase was added to Recital 60 on the centralised SPC register to deny the possibility to use the hereby contained information to support patent linkage, regulatory or administrative decisions related to generic or biosimilars, pricing and reimbursement decisions or tender bids. Article 35 – paragraph 11 a further emphasises this concept with reference to public authorities, that should not use such information for refusal, suspension, delay, withdrawal or revocation of marketing authorisations.

The JURI Committee also modified the definition of medicinal product contained in Article 2 – paragraph 1 – point 1 of the proposed Regulation, making reference to “‘any substance or com-bination of substances that fulfils at least one of the following conditions”. These include properties for treating or preventing disease in humans, the possibility to restore, correct or modify physiological functions by exerting a pharmacological, immunological or metabolic action, or to making a medical diagnosis.

The new Article 2 – paragraph 1 – point 12 a defines the meaning of the wording ‘economically linked’ with reference to “different holders of two or more basic patents protecting the same product, that one holder, directly or indirectly through one or more intermediaries, controls, is controlled by or is under common control with another holder”.

The JURI Committee also introduced the new Article 8 – paragraph 1 – point d b, stating the need to provide information on any direct public financial support received for research related to the development of the product.

The new Article 26 – paragraph 4 – point c a mentions the inclusion of any evidence in the notice of opposition in support of the opposition itself. According to the amended Article 26 – paragraph 6, the opposition panel should communicate its decision together with the reasoning for it. The same applies to the EUIPO (Article 26 – paragraph 9). The Office should also issue a single decision with reference to several oppositions filed against an examination opinion (Article 26 – paragraph 9 a). Undue delays are repeatedly discouraged.

Article 28 – paragraph 3 – point a was amended to indicate that examiners of patents and SPCs should possess relevant expertise and sufficient experience in the assigned tasks. Article 45 – paragraph 3 adds experts shall be verified for the absence of any conflict of interest.

Amendments of the Unitary SPC proposal

Many of the amendments made by the JURI Committee to the Unitary SPC proposal correspond to the ones seen above for the SPC recast. Among the distinctive ones is the new Recital 14 a, focusing on the “digital by default” principle and consequent electronic applications for unitary and combined applications for supplementary protection certificates. Article 8 – paragraph 4 a adds that the electronic application for a unitary SPC should use the formats made available by EUIPO. Other articles regulate the entire procedure to occur by exchange of electronic documentation.

Amended Recital 22 now makes explicit reference to the possibility to produce and store in the EU “in view of entering the market of any Member State upon expiry of the corresponding certificate (‘EU Day-one entry’) and any acts related thereto”.

The new Article 22 – paragraph 1 – point c b defines cases where the applicant shall waive the SPC rights for markets where the medicinal product has not been launched, i.e., the medicinal product is not placed on all Member States or a Member State market covered by the unitary certificate or combined centralised SPCs.

Comments from Medicines for Europe

The first drafts of the EP position on the SPC and SPC Regulation recast are a step in the right direction for access to medicines across Europe, according to Medicines for Europe. The association particularly appreciated the identification of the necessary safeguards for scrutiny of the SPC application before granting, to prevent invalid (non-innovative) SPCs from delaying access to generic and biosimilar medicines. The undue use of SPC expiry dates in the register to implement unlawful and anti-competitive patent linkage strategies were also deemed positive.


EC Communication (part 2): a Critical Medicines Alliance to support European pharma supply chain

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by Giuliana Miglierini

After last week’s examination of the first part of the Commission’s Communication, specifically targeted to short-term actions to prevent and mitigate critical medicine shortages, in this second post we will address the announced mid- and long-term structural measures, focused on the creation of the Critical Medicines Alliance, the diversification of supply chains and the role of international partnerships.

The Critical Medicines Alliance

The second part of the Commission’s Communication details the structural measures to strengthen the secure supply of pharmaceuticals in the EU, with particular reference to critical medicines. An objective that, according to the Commission, may require the development of new pieces of legislation, such as the EU Critical Medicines Act. To this instance, the preparatory study should be launched by the end of 2023, and followed by the impact assessment.

In the meantime, the improved coordination of the industrial approach to the management of shortages in the EU should be pursued by the Critical Medicines Alliance, to be created in early 2024. The Alliance will bring together all involved stakeholders; its activities should start from a shared analysis of vulnerabilities in the supply chain of the critical medicines on the Union list (i.e over-dependency on a limited number of external suppliers, limited diversification possibilities, limited production capacities, etc).

The result of this exercise should be the identification of useful tools to address vulnerabilities of a limited number of critical medicines with the highest risk of shortages and impact on healthcare systems. To this regard, several lines of actions are identified in the Communication, starting from the issuing of a dedicated guidance and common criteria for the coordinated procurement of critical medicines (e.g. green production and prioritisation of supplies in Euro-pe at times of critical shortages). A better quantification of demand and the consequent possibility to compensate and incentivise industry for its effort in these directions are other expected outcomes.

Medium-term contractual incentives are proposed as a tool to improve predictability of supply and to attract new manufacturing investments in Europe, together with the use of capacity reservation contracts modelled on EU FABs. These last instruments were launched by the HERA Authority during the pandemic in order to reserve manufacturing capacities for vaccines and obtain a priority right for their manufacturing in case of a future public health emergency.

The second line of action of the Alliance would address the diversification of global supply chains for critical medicines, including the identification of priority countries to be involved in strategic partnerships on the security of supply (see also below).

The third pillar should see the Alliance involved in the coordination and harmonisation of efforts to identify security of supply needs for critical medicines, on the basis of the above-mentioned identified vulnerabilities. Actions cited by the Communication, such as the Services of General Economic Interest (SGEI) coordinated at the EU level, should be compatible with the state aid framework. The Alliance may also represent the dedicated location where member states may better discuss the possibility of a new Important Project of Common European Interest (IPCEI) focusing on sustainable manufacturing of critical medicines (including off patent medicines).

Stockpiling, skills and financial support

EU stockpiling of critical medicines is another area of activity of the Critical Medicines Alliance. The goal is to overcome current limitations typical of national stockpile programmes; the development of a common strategic approach and a Joint Action on stockpiling has been announced for the first half of 2024, based on the previously mentioned vulnerability analysis and on the experience of the Union Civil Protection Mechanism (UCPM, that will continue to be part of the EU approach) and the rescEU stockpile.

The Alliance should also address the need for new and updated skills to work in the pharmaceutical sector, so to cope with the increasing impact of digitalisation, the evolution of the regulatory environment and the green transition. Pharmacists are cited in the Communication, as their curricula could be easily adjusted to accommodate education and training on new skills. Attention should be paid to increasing STEM (Science, Technology, Engineering and Mathematics) graduates. A Pact for Skills is the measure identified to actively involve key actors in educational and training activities aimed to fill industry skills gaps.

The Alliance would also play a significant role in better leverage and align EU and national funding: a goal deemed important in order to support improved long-term investment predictability for the private sector, and to avoid duplication of efforts. Among other tools cited by the Communication to reach it, the proposed Strategic Technologies for Europe Platform (STEP) is also inclusive of pharmaceuticals, biotechnologies and medical technologies. The creation of a Sovereignty Seal to promote synergies amongst existing programmes, and the Technical Support Instrument to enhance the administrative capacity of member states in managing shortages and producing critical medicines are among other proposed tools.

Diversification of supply chains

A second, fundamental line of action identified by the Commission addresses how to better diversify the complex, global pharmaceutical supply chain, also by means of new international partnerships with third countries. According to the Communication, the EU industry needs to have access to a broad range of essential inputs; to this regard, new strategic partnerships with third countries for production of critical medicines and active ingredients should be based on concrete actions of mutual interest.

The EU has 42 preferential trade agreements in place with 74 different trading partners, and a new one is under negotiation with India. The Commission also recalled the importance of bilateral meetings with China on issues affecting access to medicines supply chains, and of the dialogue with Latin America.

An improved regulatory convergence is another main objective of the planned actions at the international level, so to increase GMP compliance of medicinal products marketed in the EU and manufactured by extra-UE partners. To this instance, the Communication mentions the work of international bodies such as the ICH (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use) and ICMRA (International Coalition of Medicines Regulatory Authorities) for the harmonisation of standards for pharmaceuticals, and the WHO support to improved regulatory convergence. Many free trade and mutual recognition agreements (MRAs) signed by the EU also contain this type of obligation, and in some cases the sharing of non-sensitive market knowledge to anticipate possible problems too.

A new network of international partners should be created by the Commission within a year, in conformity with applicable state aid and antitrust rules. The network activities would focus on crisis preparedness and supply diversification. The Communication mentions also different international initiatives already in place, such as the Global Gateway to support local manufacturing of health products and announced another Team European Initiative in Africa on health security and pandemic preparedness and response. Another ongoing initiative is the EU-Latin America and Caribbean Partnership on manufacturing and access to vaccines, medicines and health technologies. The EU will also continue to support the provision of critical medicines in humanitarian contexts.


EC Communication (part 1): How to address critical medicines shortages

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by Giuliana Miglierini

As announced on 3 October in the speech given by Commissioner Stella Kyriakides at European Parliament Plenary Session, the EU Commission has published on 24 October its Communication on medicine shortages and strategic healthcare autonomy.

The planned actions are firstly targeted to prevent and mitigate on the short-term critical medicine shortages, thus avoiding the reoccurrence of situations such as those experienced in the 2022. Mid- and long-term actions have been also addressed to support the strategic autonomy of the European pharmaceutical supply chain. Among these is the creation of a Critical Medicines Alliance, to start operations in early 2024.

Improving the management of critical shortages of medicines and ensuring a steady security of supply for the EU has been our priority since day one. We need a single market for medicines in the EU and a new approach to better tackle shortages of critical medicines. Today we are putting forward collective actions to work closer with the industry and help Member States improve the security of supply for the coming winter and in the long-term.” said Stella Kyriakides, Commissioner for Health and Food Safety.

In this first post, we will examine actions in the field of medicines shortages, leaving the medium and long-term ones to a following article (part 2).

Prepared for future winters

The first goal of the EU Commission is to avoid situations of shortages of critical antibiotics such as those that occurred last year. To this instance, the Health Emergency Preparedness and Response Authority (HERA) and the European Medicines Agency (EMA) have already identified key antibiotics potentially at risk of critical shortages in the winter season, also in future years.

Immediately after the release of the Communication by the Commission, EMA published the details of the announced new European Voluntary Solidarity Mechanism for medicines, the MSSG Solidarity Mechanism.

The mechanism was developed by EMA’s Medicines Shortages Steering Group (MSSG), on the basis of the informal experience made during the pandemic. In case of critical shortages escalated to the MSSG for coordination at European level to request assistance, other member states may be of help through the rescEU stockpile mechanism to redistribute medicines from available stocks. The activation of the Union Civil Protection Mechanism (UCPM), via its 24/7 available European Response Coordination Centre (ERCC), aims to coordinate and logistically support the voluntary transfer of medicines, and it should represent the last resort, after the interested member state had exhausted all other possibilities.

The MSSG also developed a Toolkit including recommendations on how to tackle shortages of critical medicines. Among others are the monitoring of available stocks, supply and demand, interactions with marketing authorisation holders and manufacturers for increasing the manufacturing capacity and for the fair distribution of medicinal products, the implementation of regulatory flexibilities and actions aimed to improve communication to the public and international cooperation with other regulators to early identify critical shortages.

The other actions to tackle shortages

The first version of the Union list of critical medicines is expected to be released by the end of 2023. It will allow the development of further actions, on the basis of the analysis of the vulnerabilities of the supply chain of selected medicines to occur by April 2024.

In addition to the practical recommendations relative to demand forecasting at national level, the Commission is working on an EU Mechanism for Demand Signalling that should better support the collective EU public sector in its decisions. A new European Shortages Monitoring Platform for reporting information regarding available stocks and shortages of medicines is expected to start operating in 2025. Many future actions shall be supported using artificial intelligence to extract information about trends in demand and supply from existing data.

At the regulatory level, a new Joint Action has been announced for early 2024 to promote the effective use of flexibility as well as of measures applied at national level (i.e. magistral preparations of local pharmacies). Regulatory flexibilities may include, among others, the quick authorisation of alternatives, the approval of alternative suppliers of raw materials or finished products, or the temporary extension of shelf-life.

Another initiative announced for 2024 should see the issuing of an EU guidance on procurement of medicines, better detailing the already existing tools and practices supporting the security of supply. In the meantime, an EU joint procurement for antibiotics and treatments for respiratory viruses should be activated for the incoming winter.

The Communication contains some recommendations for member states and the pharmaceutical industry. The former are called to monitor and fully enforce the supply obligations of companies, to develop effective communication plans, and to consider how national procurement rules and criteria can increase security of supply. Industrial stakeholders should continuously monitor the evolution of demand and supply of critical medicines, assuring to the full the supply obligation under EU law. Early communication of critical situations to regulators should also occur, as well as the implementation of recommendations, both on regulatory flexibilities and on the elements of the pharmaceutical revision that could already be applied.

Comments from the stakeholders

The interested pharmaceutical associations promptly reacted to the EU Commission’s Communication.

EFPIA particularly welcomed the structural measures to address the industrial dimension of medicines shortages in the medium and long term, as the Critical Medicines Alliance. The development of solutions targeting the specific root causes of shortages, and measures aimed at mitigating shortages in the short term should be “proportionate and provide efficient, workable solutions that serve public health needs”. EFPIA asks for the industry to be included in the design and implementation of new processes and highlighted the “missed opportunity” represented by sharing of the information stored in the European Medicines Verification System (EMVS).

In response to Member State and Parliament calls for a Critical Medicines Act, this communication is a positive first step for the security of supply of medicines. Medicines for Europe will partner with the EU to implement these important reforms”, said Medicines for Europe President, Elisabeth Stampa. The associations ask, among others, for a strategic EU reserve of essential medicines, and EU funds and State aid projects to incentivise investments in greener and more secure manufacturing processes for essential medicines and active pharmaceutical ingredients (APIs). Digitalisation of the regulatory system and harmonisation of pack sizes and presentations would be also helpful.

European community pharmacists also welcomed the Communication, as it may help to avoid new, severe medicine shortages like the one experienced last winter. “PGEU’s annual survey confirms that shortages exist in all EU countries across all types of medicines, causing detriment to patients’ health, waste of resources and frustration. Every day, we spend hours managing shortages and finding solutions to guarantee continuity of treatment for our patients”, commented PGEU President Koen Straetmans. As for the common strategic approach to stockpiling, according to PGEU it should be guaranteed that stocks will not be to such an extent as to jeopardize the general supply of medicines, nor they should generate unnecessary waste.

EuropaBio, representing the biotech industry, positively commented on the Communication and highlighted that EU actions should not be limited to essential medicines, but should target also the growing dependency on third countries for innovation medicines.


The UK’s statutory scheme revision for branded medicines

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by Giuliana Miglierini

The amendment of the statutory schemeregulating the increase in pricing and consequent clawback payments of branded medicines proposed by the Department of Health and Social Care (DHSC) of UK’s government attracted the strong criticism of the pharmaceutical industry. The proposal aims to supersede the current scheme, established in 2018 by The Branded Health Service Medicines (Costs) Regulations. UK’s framework for pricing and clawback payments is completed by the 2019 voluntary scheme for branded medicines pricing and access (VPAS). Under the current framework, should a pharmaceutical company decide to opt out and not to join the voluntary scheme, then it becomes automatically subject to the statutory one. The current VPAS scheme, which in its original form dates to 1957, will end in December 2023. The DHSC is thus negotiating a new deal with the industrial counterparts to be effective starting 1 January 2024. Negotiations on the voluntary scheme are independent from the consultation on the statutory scheme, claims the DHSC.

The main features of the proposal

The statutory and voluntary schemes are administered by the DHSC on behalf of England, Wales, Scotland and Northern Ireland. The so received clawback payments from the pharmaceutical industry are then annually back allocated to each of the four countries on an agreed basis. According to the DHSC’s proposal, the ongoing negotiation should result in the two schemes – statutory and voluntary – continuing to operate in a complementary way. Should the negotiation on the voluntary scheme close without results, then the statutory scheme would continue to apply from 2024 onwards to all branded medicinal products. “With negotiations ongoing, there cannot currently be a default assumption of continuing alignment with any potential voluntary scheme provisions”, wrote the government. The proposed reform of the statutory scheme is comprehensive of an increase of the allowed annual growth rate, which is expected to impact on the back payment percentages. A revision of current exemptions from scheme payments should also occur.

A structure similar to that of the current statutory scheme should be used to manage these amendments, while a new lifecycle adjustment should be put in place to rebalance the payment percentages referred to medicines at different stages of their product lifecycle. As for unbranded biological products, which should be always prescribed by brand name according to MHRA’s guidance, the government aims to clarify that the statutory scheme should be applied to all biological medicines, irrespective to the fact they are marketed or not under a brand name. The government’s goal is to achieve a balance between these three objectives, “in a way that is consistent with supporting both the life sciences sector and broader economy”.

Comments from the ABPI

According to the Association of British Pharmaceutical Industries (ABPI), there is no equivalent in the world to UK’s voluntary scheme. The version agreed for the period 2018-2023 is based on a 2% per annum rise in pricing: pharmaceutical companies are called to pay back to the NHS rebates on their sales on all expenditure above the capped limit. On their side, the current voluntary scheme also requires the DHSC and NHS England to improve medicines access environment over the period 2019-2023.

ABPI mentions the dramatic rise in payments linked to the unforeseen circumstances of increased post pandemic NHS demand, which is posing major challenges for the UK life science sector. The members of the association more specifically criticise the cap growth mechanism, as well as the proposed lifecycle adjustment. The top management of leading pharma companies operating in the UK also expressed their views (read more, on the European Pharmaceutical Review).

More details on the proposals UK’s government confirmed its commitment to working with the pharmaceutical industry to facilitate the development of medicines in the UK and to support rapid NHS’s patients access to innovative medicines. The current form of the statutory scheme allows for a growth rate of 1.1% (nominal) per year for sales of branded medicines subject to the scheme. The payment percentage for 2023 and all subsequent years was set in April 2023 at 27.5%. The main exemptions refer to sales of pharmacy only and general sales list medicines, small companies with under £5 million sales to the NHS each year, sales of low-cost presentations costing less than £2, and parallel imports. The continuation of the policy of broad commercial equivalence between the statutory and voluntary schemes is the criterion chosen by the DHSC to protect the stability and efficacy of both: payment percentages in the statutory scheme would be thus comparable (but not necessarily identical) to those in the voluntary scheme. The new payment percentages in the statutory scheme proposed for 2024 would be based on a higher allowed growth rate of 2% (nominal). According to the DHSC, the maintenance of the current growth rate of 1.1% per year, in the absence of a newly agreed VPAS, would result in an effective decrease in allowed growth for most companies and might give rise to “a commercial environment for the life sciences sector that may not fully reflect the objective of supporting the sector and the broader economy”. On the other hand, an increase above 2% per year may lead to a unsustainable budget pressure on the NHS. As for the exemptions, the proposal aims to include in the statutory scheme some additional exemptions from payment which are currently part of the VPAS, namely referred to sales of medicines containing a new active substance (NAS) for 36 months from the date of their first marketing authorisation. According to the consultation document, this would incentivise companies to launch innovative medicines in the UK more rapidly than in other countries. An exemption from scheme payments for centrally procured vaccines (CPVs) is also part of the proposed package, and it would include vaccines for national immunisation programmes recommended by the Joint Committee on Vaccination and Immunisation (JCVI), procured by a central government body, or managed by the UK Health Security Agency (UKHSA) or a successor body. The third exemption to be included in the statutory scheme refers to payments for exceptional central procurements (ECPs). The measure would cover medicines related to purposes of emergency preparedness (i.e. national stockpiles) conducted by a central government body, or managed by UKHSA or a successor body. The lifecycle adjustment Innovative medicines are typically characterised by higher prices at launch, to then lower it while approaching the end of intellectual property protection. According to the DHSC’s proposal, initial prices are typically above the opportunity cost to the NHS, and older medicines would in general also benefit from greater price competition from generics and biosimilars. In some instances, states the document, this competition would be insufficient, thus resulting in prices not low enough to reflect the later stage in the product’s lifecycle. This especially applies to single supplier markets, where no competition at all is available. The proposal aims to overcome the current one-size-fits-all approach to the statutory scheme, and to introduce additional payments for older products with no competition, or a flat, lower payment for older products in more competitive markets. The so-generated additional income would then be used to reduce the headline payment percentage paid by newer products. According to the consultation document, these latter ones would refer to any product where the active substance has been marketed in the UK for less than 12 years.


UK will participate to European research programmes

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by Giuliana Miglierini

The divergent road opened as a consequence of the Brexit, in January 2021, between the European Union (EU) and the United Kingdom (UK) is now converging again as for the possibility for UK researchers to participate to Horizon Europe (HE) and Copernicus scientific programmes. The agreement in principle reached on 7 September 2023 by the European Commission and the UK Government was facilitated by the previous Windsor Framework Agreement. It shall now be ratified by the Council of the European Union, and then adopted by the Specialised Committee on Participation in Union Programmes.

The EU and UK are key strategic partners and allies, and today’s agreement proves that point. We will continue to be at the forefront of global science and research.”, said the Presi-dent of the European Commission, Ursula von der Leyen.

The possible association of the UK to Horizon Europe animated a vigorous debate in the past couple of years among the scientific international community, as well as that of other third countries such as Switzerland (those association is still pending, see below).

The current agreements in place between the UK and the EU are not comprehensive of the participation of UK’s students to the Erasmus+ programme, participation that was cancelled by the UK government in 2020.

The financial terms of the agreement

The new association of UK to both HE and Copernicus programmes will become operative star-ting 1 January 2024, superseding the previous transitional agreement that allowed UK researchers to apply and be evaluated as other potential beneficiaries under HE calls. It will become possible for UK researcher to access HE’s 2024 Funding Programme and Work Programme (including the coordination of consortia), and to participate in the European Research Council and Marie Skłodowska-Curie Actions.

According to the European Commission, the estimated annual contribution of the UK to Horizon Europe and the Copernicus component of the Space programme should be on average €2.6 billion, in line with the terms agreed in the Trade and Cooperation Agreement. The EU Commission will issue twice a year a call for funds to the UK corresponding to the due contribution. The overall EU budget for Horizon Europe is €95.5 billion, plus contributions due by the various associated countries.

The agreement is also comprehensive of a correction mechanism referred to Horizon Europe, aimed to compensate the contribution of the UK, should its receipts in grants be higher than its contribution for grants. Under the terms of the Trade and Cooperation Agreement, an automatic correction to the UK’s contribution would occur if it reached a threshold of 8% over two successive years. A balance mechanism has also been put in place to compensate for the UK receiving significantly fewer grants than its contribution. In this instance, the level of UK participation may be improved, or (should it overpays by more than 12%), the issue may be object to scrutiny by the joint Specialised Committee on Participation in Union Programmes to agree upon the measures needed to balance the situation.

A temporary and automatic mechanism has been also agreed to address any risk of critical underperformance by the UK should the imbalance exceed 16%, based on the consideration the country did not fully participate in HE in the past two years.

The main fields of collaboration

UK’s participation to European research programmes will focus on area of mutual interest, i.e. emerging technologies, climate change and health. The participation to strategic parts of Horizon Europe – including those related to strategic assets, interests, autonomy or security – is subject to the previous assessment of UK participants on equal terms with other associated countries (Art. 22(5) of the Horizon Europe Regulation). The participation to other parts of HE will occur on equal terms with researchers and organisations from EU Member States.

Copernicus is part of the European space programme. The association will allow the UK to access a state-of-the art capacity to monitor the Earth and its services. Among the main goals of the programme is the understanding and acting on environmental and climate change related challenges. The UK will also have access to EU Space Surveillance and Tracking services.

The reactions

The announcement of the agreement on the association of the UK to European research pro-grammes found very positive reactions among the different parties interested in solving the issue.

Joining the Horizon Europe programme is a huge win for the scientific research community, who have been pushing for resolution over the past few years. UK innovation and research de-pends on international collaborations which are crucial for driving advancements in all areas of science, including the discovery and early development of new medicines and vaccines”, said Janet Valentine, ABPI Executive Director, Innovation and Research Policy. “The UK accession to Horizon enables the two sides to reinvigorate their longstanding partnership in R&D, and directly contributes to UK growth and competitiveness in the life sciences sector by making the UK an attractive destination for talented researchers.”

This decision represents a long-awaited signal for renewed international collaboration on fundamental frontier research in Europe. It will strengthen the research of all involved, both in the EU and in the UK. At the ERC, we look forward to welcoming back researchers based in the UK, after the trying last few years. They have been sorely missed, and will now be able to participate again as from our 2024 grant competitions”, said the President of the ERC, Maria Leptin.

The academic world represented by Cesaer highlighted the reintegration of UK into Horizon Eu-rope and Copernicus reaffirms the commitment of both the EU and the UK to advancing global scientific excellence. The association of the European universities of science and technology also supports further progress in building a wider international scientific community, with particular reference to Switzerland.

Today, Europe’s universities celebrate the end of a long road that began in 2016 and look for-ward to rebuilding and further developing close partnershipssaid Josep M. Garrell, President of the European Universities Association.

We are extremely grateful for the efforts of everyone in the European research community who has worked tirelessly to help secure this agreement”, added Jamie Arrowsmith, Director of Universities UK International.

Switzerland is still waiting for the association

Despite Switzerland being a very important country for research in life sciences, and location of many of the major pharmaceutical industries, the country is still waiting to restart the negotiations with the EU for its association to the European research programmes. The exclusion of Switzerland from any form of collaboration was the result, in 2021, of the political divergence with the EU on many issues.

We feel alone in the middle of Europe,” Yves Flückiger, rector of the University of Geneva, told Business|Europe.

According to the article by David Matthews, the incumbent Swiss federal elections in October 2023 and the European elections of 2024 may slow down the negotiations on the new political relationship. The association of Switzerland to EU’s research programmes might then not occur before 2025. Some explanatory talks would be already ongoing, adds the article. Sympathy for researchers in Switzerland was expressed by the ERC President, Maria Leptin. “They are not alone in the sense they are loved by all the rest of us,” she said. “There is very high-level research being done in Switzerland, same as in the UK. We all want to be one group that competes at the same level and is evaluated by the same high-level panels.


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

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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 on Supplementary Protection Certificates(SPCs) and compulsory licensing (CL) in crisis situations. It also includes a new Regulation on Standard Essential Patents(SEPs).

The proposed reform, which is part of the EU Industrial Strategy, will now undergo the scrutiny of the European Parliament and Council. It aims to improve European competitiveness, innovation and technological sovereignity, with a special attention to the role played by SMEs. The proposal is based on comments received during the consultation on the Action Plan on Intellectual Property issued in November 2020. The IP legislative framework will complement the Unitary Patent system, that will fully entry into force on 1 June 2023.

Supplementary Protection Certificates

Central to the reform of the SPC system is the creation of a unitary SPC to complement the Unitary Patent. The aim is to reduce the current fragmentation in the issuing of SPCs at the national level, which often leads to complex interpretation of patents’ expiry dates, and consequent legal uncertainty. The new system would not replace the existing national SPC schemes.

Procedures should be simplified, with a single application to be submitted to the EU Intellectual Property Office (EUIPO), which would be responsible for its central examination in close cooperation with EU national IP offices. The process would lead to national SPCs granted for each of the designated member states (MS), plus a unitary SPC if required by the applicant (here the Q&As).

According to the Commission, approx. 25% of current SPC procedures have contradictory outcomes. The mean number of annual SPC applications is 81 per MS, with a total cost of €192,000 over the 5 years of duration (compared to roughly €3,000 in the US and €4,200 in Japan). Savings from the new procedures may amount to up €137,000 for the EU27 wide, five years long SPC protection. A central SPC database is also planned in order to increase transparency.

The proposed reform is comprehensive of a Regulation specific to medicinal products and a second one focusing on plant protection products, plus parallel recasting regulations to review the current legislative provisions (i.e. Regulation (EC) No 469/2009). Innovators would be incentivised to use unitary SPCs, since otherwise a unitary patent could be extended at higher costs only by means of national SPCs. Infringements of unitary SPCs would fall under the judgement of the UPC Court.

The Commission expects the development and access to generic medicines will be facilitated. In particular, SMEs will be able to submit observations during the examination of a centralised SPC application, and to file an opposition in order to centrally challenge the validity of the SPC protection, if justified. The new framework complements the proposed pharmaceutical legislation, for example on the Bolar exception. This should allow the generic industry to perform research and testing for preparing regulatory approval also while a patent/SPC is still in force.

Compulsory licensing

Compulsory licensing may be used during crisis in order to provide access to relevant products and technologies, should result in impossible (or not adequate) to close voluntary licensing agreements with owners of IP rights. The current fragmentation of procedures at the national level results in a wide legal uncertainty (see also the published Q&As). The new framework would complement other EU crisis tools, such as the Single Market Emergency Instrument, HERA regulations and the Chips Act.

According to the proposal, a Union compulsory licence can only be granted after activation of an emergency or crisis mode at EU level. Instruments to trigger this fundamental passage are listed in an Annex, so to improve legal certainty. A remuneration scheme for IPR holders is also included, on the basis of successive steps in the activation and termination of compulsory licensing.

The existing national frameworks on compulsory licensing will continue to operate, and they may be used to manage local crisis. Compulsory licensing of exported products would not be allowed.

Standard Essential Patents

SEPs refer to technologies essential for the implementation of a technical standard adopted by a standard developing organisation. They are typical of the ITC industrial sector, and central to building the Internet of Things.

To improve the transparency and legal certainty of SEPs, the proposal aims to ensure innovation would be run in the EU by both EU SEP owners and implementers. End users would benefit from products based on the latest standardised technologies at fair and reasonable prices. SEPs licensing is based on the FRAND scheme (fair, reasonable and non-discriminatory) for the remuneration of patent holders.

Comments from the stakeholders

EFPIA granted positive feedback on the simplification and harmonisation of the SPC system and to the opportunities offered by the unitary SPC. On the other hand, the proposals on compulsory licensing didn’t find the agreement of the research-based pharmaceutical industry.

According to a note, voluntary licensing would be the preferred instrument for innovators, as it allows for the choice of the best-positioned and trusted partners to speed up production and distribution of medicinal products during health crisis. On the contrary, compulsory licensing is seen as a threat to investment stability of the EU’s IP system and to the overall innovation pipeline.

Protecting the EU’s intellectual property framework could not be more important if we are to close the investment gap between Europe, the US and increasingly China and continue to offer patients the best possible treatments. Yet we are seeing multiple proposals emerging from the European Commission in the pharmaceutical legislation and patent package which tend towards the opposite”, said EFPIA Director General Nathalie Moll.

Medicines for Europe (MfE), on behalf of the generic and biosimilar industry, said that while “voluntary licensing agreements are relevant for health crises, we will contribute constructively to the EU-wide compulsory licensing system”. The request to the Commission is to make it a remedy also for anti-competitive abuses of the patent system, according to art. 31(k) of the TRIPS Agreement.

As for new SPCs, MfE highlights the new regime would extend their geographical scope from the current 20 out of 27 MS covered on average. “The proposal for a reform in the SPC system has the potential to reduce fragmentation in Europe but the legislation must ensure improved quality and transparency of granting procedures to prevent misuse by right holders to delay competition”, said MfE Director General Adrian van den Hoven.

Critics of the proposed scheme for compulsory licensing also came from EUCOPE, representing pharmaceutical entrepreneurs. According to the Confederation, the Commission’s proposal would further weakening the value of intellectual property rights within the EU. “Together with the proposal on the revision of the general pharmaceutical legislation, it is another indicator that the development of an innovation-friendly environment is not a priority, contrary to statements in the Intellectual Property Action Plan”, it states in a note.

For EUCOPE, the proposed SPC regime would not amend the substantive elements of the current system. Furthermore, a centralised SPC application would only be possible on the basis of a European patent, including a unitary patent, and for products with a centralised marketing authorisation. EUCOPE position goes for an optional EU-wide SPC, so to allow flexibility for IP owners in deciding their strategy for the protection of IP rights.


Pioneer plan, UK’s alternative to support research and innovation

By Giuliana Miglierini

While the position of the UK with respect to the participation to Horizon Europe (HE) is still pending, the British government has announced its new Pioneer plan aimed to provide a domestic alternative framework for research and innovation.

The plan is deemed to “protect and support UK science, research, technology and innovation (SRTI) sectors, should association to Horizon Europe on fair and appropriate terms not be possible”.

The planned total budget for investment (£14.6 billion up to 2030) corresponds to the same amount the UK government would have allocated to participation of the country in Horizon Europe. According to UK science and technology minister Michelle Donelan, the government is still committed to pursue HE association but “We must ensure we have an ambitious alternative ready to go should we need it,” she said.

This means the Pioneer plan might be activated in case of failure of the still ongoing negotiations with the European Commission. The plan has been specifically developed to provide a robust reference framework supporting UK’s Science, Research, Innovation and Technology (UKSRTI) network, and it is complementary to other, already existing R&D incentives. It shall also integrate with other relevant pieces of legislation in the field of research and innovation.

Four key pillars to boost UK’s research

Four different strategic lines of action have been considered in order to build a comprehensive approach for R&D and innovation. The publication of the plan is also a message from the UK government to the EU Commission, to mark the position of the country as a ‘science and technology superpower’ in the case that UK would be unable to join Horizon Europe.

The four key areas of action identified by the Pioneer plan are the attraction of talents, innovation, global collaboration and infrastructures.

Pioneer Talent is a programme aimed at attracting both domestic and international talents to run their research in the UK. The estimated investment amounts to up £2 billion by 2027/28. According to the plan, the new programme should be delivered in partnership with UKRI and the UK’s National Academies. A new set of doctorates, fellowships and professorships would be available through Pioneer Discovery, an investigator-led research programme, set up to provide grants of longer duration, higher level and flexibility compared to HE. The framework would also provide support for specialised leadership training, international mobility, entrepreneurship and commercialization of results. According to the plan, Pioneer Discovery would make available each year up to 800 studentships, 370 international fellowships and 260 early-, mid- and late-career stage fellowships and awards.

Pioneer Innovation would invest in the creation of partnerships between all actors involved in business-led innovation across sectors and technologies, including research bodies, industry and the third sector. The planned investment is up to £3.5 billion by 2027/28, targeted to a selected number of ambitious programmes aimed to sustain the transformative effort towards new models of collaboration.

Funding of the selected projects might vary from short- to medium- and long-term, and it would be based on quick-start catalyst programmes, challenge prizes and Eureka actions. Health innovations, green industrial growth, resilient UK, and transformative technologies are the identified priorities. Pioneer Innovation should allow for fast-track commercialisation and deployment of new technologies and innovations.

Pioneer Global aims to build broaden international SRTI collaboration beyond the EU, funded with a planned budget of £3.8 billion by 2027/28. The programme should focus on bilateral, minilateral (small groups of countries collaborating on specific, urgent global challenges) and multilateral collaborations (i.e influencing standards and increasing reliance on global supply chains). Global technology transfer should also be addressed. Discovery-driven bottom-up collaborations with researchers around the globe should drive the funding of activities.

For collaboration to European research programmes, the indication coming from the plan is to make reference to funding for all eligible consortia for Third Country Participation in Horizon Europe until 31 March 2025. News on how to address this issue after that date is expected to be released by the UK government in October 2024.

Pioneer Infrastructure is the programme aimed at building a completely renewed, once-in-a-generation network of world-class national and international high-quality R&D infrastructure andlab facilities supporting the previous lines of action. The planned investment is up to £1.7 billion by 2027/28, with the final goal to fully exploit the potential of UK’s Public Sector Research Establishments (PSREs), universities, institutes, national labs and research organisations.

The pending negotiation for participation to HE

Despite the commitment of UK government to associate the country to the current European research and innovation programmes, including Horizon Europe, Copernicus and Euratom Research& Training (as established under the Trade and Cooperation Agreement, TCA), negotiations with the European Commission are still ongoing. “But association would need to be on the basis of a good deal for the UK’s researchers, businesses and taxpayers”, is written in the Pioneer plan.

According to the document, the UK government has maintained active investments during the two-years delay, committing over £1 billion through the Horizon Europe Guarantee, and £684million of direct funding to UK Science and Research, Fusion and Earth Observation. Launched in November 2021, the Horizon Europe Guarantee should remain active up to the end of June 2023; grants issued by the UKRI up to the end of February 2023 amounted to more than £882 million.

One of the last interventions in support to the closure of the negotiation came from the director of the Francis Crick Institute and Nobel Laureate Paul Nurse, during a hearing at the House of Commons science and technology committee (see here more on Science & Business).

According to Mr. Nurse, the Pioneer plan would turn to send mixed messages, and the British academic community might be not enough protected from the risk of being excluded from HE due to the yet unclear commitment of the government to positively close negotiations.

An editorial signed by Science’s Editor-in-Chief H. Holden Thorp also summarises the sentiment of the chief executive of UK Research and Innovation (UKRI) Ottoline Leyser on the long-waited definition of the adhesion procedure to Horizon Europe. “I’ve been pleasantly surprised by how much wide press interest there’s been in the UK’s association to Horizon Europe,” she said to Thorp, “and I think it signals a much wider, very positive trend of real and deep interest in research and innovation across the UK system.”

The association to Horizon Europe should had closed in March 2023, after the signature of the Windsor Framework agreement by PM Rishi Sunak and President Ursula von der Leyen. Among open issues is the UK’s financial contribution to HE, even if according to the European Commission the country should not be requested to pay the full association fee for the two years it has been excluded from the research programme.

The Windsor Framework political agreement in principle signed on 27 February 2023 focuses mainly on customs requirements for goods entering Northern Ireland (NI) from Great Britain, a fundamental passage to ensure NI’s unique access to the EU single market. Two decisions related to the operative phase of the Windsor Framework were adopted by the European Council on 21March 2023, respectively establishing the EU’s position in the Joint Committee and the Joint Consultative Working Group set up under the EU-UK Withdrawal Agreement.