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Giuliana Miglierini

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Towards the Brexit’s transition phase

Giuliana Miglierini
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Towards the Brexit’s transition phase

The winner of the December 2019 UK’s general elections, PM Boris Johnson, is rapidly proceeding to finally make the Brexit become true on 31 January 2020; starting from this date, the negotiation period will start to find new trade agreements with the European Union, a process that should close by the end of 2020. June shall be a critical month, with an already planned summit with EU27s to monitor progress and to leave the UK the last chance to ask for an extension of the transition period (read more on the due schedule for 2020 on the Financial Times). Should the final deal not be reached by 31 December 2020, the hard-Brexit would finally occur. 

The priorities for the negotiations

The Brexit Health Alliance (BHA) published a briefing document to highlight its priority areas for the incoming negotiations between the UK and the EU, in order to safeguard the interests of patients across the two sides of the Channel. 

The Alliance – that includes the NHS and several industrial, scientific and academic associations – asks for a close cooperative relationship with the EU across trade in medicines and medical devices, health security and research. This approach is considered by BHA the best way to pursue the general goal of patient protection. 

Detailed targets to be kept in mind by negotiators include the alignment of regulations and customs arrangements to maintain availability of safe medicines and medical devices both in UK and EU. BHA also expects the UK regulatory agency MHRA may continue its participation in the European Medicines Agency and other regulatory networks and databases.

The preservation of reciprocal healthcare arrangements is considered important to ensure the simple and safe access to healthcare for travellers from UK and EU, while a full UK participation in European research programmes (including Horizon Europe) and a full researcher mobility would be important to support innovation. Alignment on pre-competitive research regulation, including the fullest UK cooperation on clinical trials and participation in European Reference Networks, are other requests advanced by the Briefing document, as well as a continued collaboration and data sharing on health security.

The comments on the results of the general elections 

The Association of the British Pharmaceutical Industry (ABPI) welcomed positively the results of the general elections, as Boris Johnson’s electoral programme included strong commitments to improve the availability of new medicines to NHS patients, the uptake of vaccines, and to place life sciences at the centre of an innovation based economy (see below). “The ABPI supports these ambitions and we look forward to working with our members to bring new investment to the UK to further strengthen our world-leading science base. The Prime Minister’s Brexit deal includes an important commitment to exploring close cooperation on medicine regulation. Achieving this will be important in prioritising patients and public health as well as the future of the UK life sciences sector”, said ABPI chief executive, Mike Thompson.

“A Conservative majority offers a new era of political stability at Westminster, which makes long term business decision making simpler for our sector”, added Steve Bates, chief executive of the UK Bioindustry Association (BIA). Expectations for the post-Brexit include the creation of the Innovative Medicines Fund, an expanded R&D tax credits, and fulfilling the PM’s “personal pledge of the ‘fastest ever increase’ in R&D spending. Our key priorities are ensuring companies can continue to access the capital and talent they need, working in partnership to deliver the life sciences strategy and ensuring NHS patients continue to access the latest innovative medicines”, said Steve Bates.

ABPI’s comments on the electoral manifestos

Both the Conservative and Labour parties published their electoral programmes including the foreseen actions in the pharmaceutical and healthcare sectors. ABPI commented the two manifesto from its website.

The Innovative Medicines Fund should represent for the Conservatives the tool to support innovative therapies in the field of tumors, rare and autoimmune diseases. The new Fund should be an expansion of the Cancer Drugs Fund, and it should be granted an investment of £ 500 million for the first year; among its duties may figure also the conditional reimbursement of medicines for some types of diseases. According to ABPI, its real impact would depend greatly on the increasing number of innovative therapies reaching the market. The industrial association disagreed with the tools proposed by the Labour’s manifesto, e.g. compulsory licensing and the creation of a “governmental” manufacturer of generics drugs. 

The Conservative’s intent to make the UK a global hub for life sciences after the Brexit, with a planned R&D expenditure reaching the challenging goal of 2.4% of GDP, has been also welcomed by ABPI. A new agency to manage high risk and high potential initiatives is another interesting feature that according to the Association might prove critical to facilitate new public-private partnerships. Support to basic R&D and the increase of tax credit up to 13% for R&D activities (and possibly also for investments in cloud computing and data management) are other positive elements of the Conservative programme. 

The current Apprenticeship Levy tool to attract talents is considered insufficient; the proposal of ABPI sees the creation of a new Life Sciences Skills Fund to fill the knowledge gaps in genomics, immunology, bioinformatics, chemo-informatics and clinical pharmacology. 

A hot issue such as drug prices were missing from the two electoral manifesto, noted Richard Staines on Pharmaphorum just after the general elections. According to the article, US and UK negotiators were preparing to expedite new trade agreements that will maximise the patent protection period for biologic drugs. 

This goal would have a double impact from the US point of view, explains Richard Staines, both on the US election in November 2020 and in shaping the future UK’s pharmaceutical market. 

EPO: Patents to boost SME’s competitiveness and success

Giuliana Miglierini
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EPO: Patents to boost SME’s competitiveness and success

Patents are the key for the success of small and medium sized companies (SMEs), says the report “Market success for inventions – Patent commercialisation scoreboard: European SMEs” recently published by the European Patent Office (EPO). SMEs use patent especially to protect high potential inventions, 67% of those have successfully reached the market (50% in partnerships with bigger companies). Patents filed by SMEs or individual entrepreneurs were roughly one fifth of the total of patents filed in 2018, according to the report.

A tremendous untapped potential

“SMEs are a key user group in the European IP system, creating new technologies, jobs and growth. And patents help them to protect their inventions,” said EPO President António Campinos. “Crucially, this new study demonstrates that SMEs are also using patents to commercialise their inventions, they are increasingly innovative with their IP strategies, and that there is tremendous untapped potential. Given the role that patents play in supporting our economy and bringing forward new technologies, efforts have to continue in finding ways to tackle successfully the challenges revealed in this study.”

The European Patent Office run a survey in June 2019 involving more than 1,100 European SMEs, focused on patents they filed in years 2008-2018 (already granted or still under examination) for inventions in the fields of chemistry, mechanical and electrical engineering, instruments. In the chemical sector, 34% of the considered patents referred to products, 43% included product, process and/or method and the remaining 23% were process- or method-based patents. The European territory was considered according to areas corresponding to Germany, UK, France, South-East Europe and North-West Europe. 

Collaborations to reach the market

Partnerships are often the preferred by SMEs to find the way to market for their innovative technologies. Roughly half (53%) of the interviewed companies closed industrial agreements within their nation, while another 56% found partners in other European countries. This last trend is highly favoured by the protection conferred in 44 different countries by the European Patent; the US resulted attractive for 26% of the interviewed SMEs, Asia for 21%. 

Lack of specific experience in intellectual property (IP) protection is still one of the main challenges SMEs have to face to efficiently protect and support their inventions. This also impacts on their ability to find potential partners interested in their commercialisation; the limited business contacts that typically characterise these companies represent another factor which may restrict the possibility to close commercial agreements, says EPO. With this respect, already known customers and clients represent the SMEs’ preferred partners in 59% of cases, followed by suppliers (26%), the academia and other research institutions (19%), and even direct competitors (15%). This last category ranked second (22%) as for the possibility to close future collaborations (clients and customers maintained the first place, 32%). And in a tenth of cases, SMEs’ industrial partners come from a completely different sector of activity. 

Licensing agreements are the preferred form of collaboration for 62% of the SMEs participating to the survey; cooperation contracts resulted also very attractive (49%), followed by spin-off creation (32%) or cross-licensing (21%). The main benefit perceived by SMEs following a collaboration agreement is the possibility to increase their revenues (85%), together with a better access to the market (73%). Shared innovation projects (56%), outsourcing of manufacturing activities (42%) or the solution of cases of patents infringement (32%) are all possible driving forces to look for new collaborations.

The search for new partners is usually run through the company’s business networks (77%) or through previous partners (67%); in more than a half of cases (60%), the SME was identified as a possible partner by the bigger industrial entity.

Many reasons to file a patent

In the pharmaceutical industry patents may protect either the active ingredients or the final medicinal products, the formulation, the production process or the use of particular reagents or solvents, etc. Patents may also refer to the clinical use of the medicinal product, with reference to single or different indications. Dosage regimens, new uses for the same compound, improved variations are other examples possible for pharmaceutical IP protection (see here more).

The desire to protect the innovative product or technology from imitation remains the main reason (83%) for  European SMEs to file patents. IP protection also represents a useful mean to safeguard the reputation of the company (69%), and it supports the freedom-to-operate on international markets (59%). The survey also mentions the possibility to close contracts (53%), licensing (46%) or financing deals (35%) as other possible causes to look for IP protection.

Patents complementary to the main one protecting a certain technology are considered important by roughly the half (48%) of SMEs participating to the survey. Brands are also often taken into consideration (45%), while less appealing is industrial design (27%).

According to EPO, SMEs highly value the importance of their inventions compared to other ones in the same industrial field; 39% of the interviewed companies perceive their inventions as being ranked in the top 10% of technical developments in their industry, another 43% place them in the top half and just 17% put their new technologies in the bottom half. The majority of SMEs considered their patented inventions very highly (44%) or highly (22%) relevant for the company’s core business. Patents in the chemistry field were more frequently judged highly relevant compared to the other industrial sectors considered by the report. 

The commercial exploitation

Deterrence is an important reason many SMEs (64%) are using their patents for. Commercial exploitation of patents is higher in the mechanical engineering field (73%) and lower in chemistry (60%). This industrial sector – that include also the pharmaceutical industry – has showed the higher propensity to collaborative exploitation of patents inventions (35%). Patents in the chemical field are often exploited through co-operation (55%) or licensing and selling (60%); the creation of spin-off is also very high in this area (41%), says the EPO report. 

How smart labelling solutions can benefit Pharma and Healthcare Industry

Giuliana Miglierini
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How smart labelling solutions can benefit Pharma and Healthcare Industry

Radio frequency identification (RFID) is increasingly becoming the new standard for tracking packages of medicinal products along the entire supply chain, from production to end users. The global RFID technology market in healthcare was valued at $ 2.58 billion in 2018 and it is expected to grow at a CAGR of 22.4% up to 2025, according to a report by Grand View Research. The Falsified Medicines Directive greatly supported the use of RFID in the pharmaceutical sector, as well as other tracking technology i.e. barcodes, with revenues of $ 778.9 million in 2018 for the pharmaceutical tracking segment.

“The pharmaceutical industry already uses temperature sensors or RFID tags, but it is limited to certain applications which only take one part of the supply chain into account without being holistically structured”, explained Frank Jäger, managing director at Faubel, in an article on European Pharmaceutical Manufacturer.

The driving forces supporting RFID

Driving forces for this expansion are, according to Grand View Research, the reduction of operative costs, the increased adoption of the technology in the healthcare sector to optimise the efficiency of the supply chain and the need to limit visits to hospitals, with the overall final goal to improve patient safety. RFID technologies also allow the real-time management of stock and inventories. 

The advent of the Internet of Things (IoT), where different products and devices are freely communicating  with one another, is deeply changing how many healthcare procedures are run and dispensed. It is now possible to track the assumption even of a single pill, enclosing an RFID microchip which communicates from the body’s interior to a remote control station the patient’s biological parameters. In hospitals, RFID may be used for example to monitor storage and transport conditions for blood bags, or to mark each device used in the surgery room, thus lowering the probability of medical errors. And each patient can receive upon admission a special wrist band including all information about his/her medical history, disease and  treatment regimen.

What is RFIDs

The term RFID encompasses a wide range of different smart technologies allowing for the easy communication of items connected to the IoT. Radio waves are used to share information between the tag and the reader. Each RFID tag can be associated to a Unique Identification Number (UIN), so to exactly track each different item. It is so possible, for example, to track every single package of a medicinal product, or each single box or pallet included in a certain shipment, without the need of the line-of-sight typically used to read barcodes: contents of an entire truck can be checked with RFID in just few seconds. 

An article from Resource Label Group describes the different types of technologies available on the market, differing upon the frequency range they use (low, high and ultra-high) or the fact they are active (powered), passive (un-powered) or semi-passive (battery-assisted). Low-frequency (LF) RFID tags operate at 30 KHz to 300 KHz, they are slower but less prone to interferences by liquid and metals. High-frequency (HF) RFID tags operate at 3 to 30 MHz and have a higher memory capacity. This category includes also the Near field communication (NFC) tags, specifically using the 13.56 MHz frequency, that requires reader and tag are at just few centimetres of distance to be read (one at a time). Ultra-high-frequency (UHF) RFID tags operate at 300 MHz to 3GHz, have a lower price and are often used to manage the supply chain.  

RFID and the pharmaceutical supply chain

Logistics is a crucial part of the pharmaceutical supply chain: the last decade saw great investments to improve the transition towards a new model based on new smart technologies, among which are RFID and the IoT, together with artificial intelligence and the blockchain. The impact of RFID on this sector has been recently examined by Usha Sharma in an article on Express Pharma.

The smart label technology can be applied to all steps along pharma development and manufacturing, including the tracking of raw materials supplies and the production and shipment of batches for clinical studies. Smart labels, for example, can be very usefully applied to avoid the need for re-labelling of investigational medicinal products during clinical trials due to changes in the stability data obtained in parallel to the study, explained Frank Jäger.

The final objective of smart warehouses’ management is to avoid both under- and over-stocking, a goal often pursued through a wide use of automation in all activities. New generation warehouses are characterised by active and passive temperature loggers for continuous temperature monitoring, directly linked to tables listing the stability properties of the different products, so to immediately activate alerts in case of deviations. Smart labelling solutions also allow for the optimisation of logistics operations and inventory management across the different manufacturing sites and warehouses typical of big pharma companies.

The Falsified Medicines Directive asks the industry to closely track each passage along the supply chain to minimise the risk of counterfeiting. With this regards, smart labels are also useful at the pharmacy level, as they support pharmacists both in checking the received shipments and in dispensing the products to patients. These too can use sealing labels containing RFID to check for the integrity of the package. 

Tracking of temperatures and times during shipments is critical to ensure the quality and efficacy of many pharmaceutical products, especially in the field of advanced therapies. RFID allows for the real-time tracking of all needed aspects, thus supporting the immediate activation of mitigation actions were appropriate. GPS technology support the exact location of each item at each time. The overall security of the supply chain is improved, limiting the risk of theft and the possible shortage of medicines due to diversion processes. 

The possible uses of RFID labels in hospitals

RFID technology has potentially limitless applications in the healthcare sector, according to Marsha Frydrychowski, Resource Label Group’s director of Marketing Services (see here the article on Packaging Europe). This technology may greatly improve the overall efficiency of services provided by hospitals, for example, since it allows for the closely monitoring of each single step or procedure, from checking for the correct storage conditions for medicines to the dispensing of diagnostic testing and medications, from surgery procedures to patient care. And greater efficiency means also reduction of general costs, an evergreen target for hospital managers called to keep costs under strict control. 

The central hospital database collects all the UIN associated to each single RFID tag, applied on each product at the moment it is received by the hospital; tags are more difficult to duplicate than barcodes, according to the article on Packaging Europe. RFID labelling also allows for an improved productivity, with up to 700 products read per second. The use of smart inventories and cabinets may also assist a more efficient management of the internal hospitals’ supply chain, reducing the risk of errors while dispensing medications and improving costs savings, as all personnel can easily locate the exact position of the product of interest, for how long it has been stored and the expiry date. Inventory management is also easier, as the database is automatically updated. 

Revised guidelines on the use of genetics and genetic testing in health care

Giuliana Miglierini
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Revised guidelines on the use of genetics and genetic testing in health care

The Reykjavik Declaration on the use of genetics and genetic testing, first issued in 2005 by the World Medical Association (WMA), has been recently revised during the 70° General Assembly to update its content to the progress of science and the new technologies now available to manipulate cells and nucleic acids (find here the document). “At a time when consumer genetic testing is growing, it is very important that we update our guidelines for the protection of patients”, said WMA President Dr. Miguel Jorge.

The WMA clearly states in the Declaration its opposition towards the possibility to use gene editing or other techniques to alter human germ-line cells, due to the many still scientifically unresolved risks. These emerging technologies can thus be only applied to somatic cells or staminal cells for regenerative medicine applications, also at the clinical level. WMA completely exclude the possibility to modify germ-line cells for reproductive cloning of humans.

A great value still coupled to many risks

Genetics is probably the most important emerging technology in the biomedical field since the beginning of the third millennium. Gene editing technologies are now very cheap and widely available; genetics proves also useful to predict the multifactorial risks at the basis of many diseases. The therapeutic application of these techniques represents a great value for both the single patient and the overall society, says the Declarations, but on the other hand there are still risks related to the protection of personal data and the privacy of patients.. 

These considerations are at the basis of WMA’s position declaring the ethical use of genetics and genetic testing only if it is directly related to the provision of new modalities to cure illness. The WMA’s ethical principles detailed into the Taipei Declaration on health databases and biobanks should guide the collection, storage and use of patients’ data, while the Helsinki Declaration should represent the reference to run medical research involving human subjects. 

Many competing interests

It is not easy to deal with the many, and often competing, interests involved in the development and exploitation of new advanced therapies. The collection of genetic profiles is at the center of very “intense” interests, and it also represents a highly sensitive field with respect to the data protection regulation, as it allows for the precise identification of a single person and of his/her health status. The final impact is still not fully understood, says the Declaration, which also states the impossibility to completely anonymise genetic data.

Genetics offers for the first time a true possibility to permanently solve many yet orphan diseases (e.g.some types of cancers). But the patients may not be the only ones impacted by the therapy or the genetic testing, as their relatives might be also asked to give access to their genetic profiles in order to evaluate the possible occurrence/prevention of the same disease. 

How to properly inform and use genetic data

The use of genetic data should be always based on the possible benefit for patients, says the Reykjavik Declaration. According to the WMA, predictive genetic testing in children represents a particularly delicate issue and it should be taken into consideration only when clear clinical indications are available , and for the only interest of the child involved. 

This also means that the use of data should be limited just to the purpose of the specific genetic testing they have been acquired for. This critical step has to be formalised through the informed consent. The Declaration details the points it should contain in a clearly understandable form. Among these are the nature, purpose and benefits of the genetic testing, its limits and the nature and meaning of the information so obtained. The informed consent should also state modalities to communicate the results and the new findings that may arise in future. Another important feature is the description of the different therapeutic choices resulting from the testing; the document should also provide the modalities for the storage of data and biological samples (where and for how long), included specifications of who is allowed to access current and future data. 

This also refers to the secondary use of genetic data or samples, a hot and still unresolved issue. Current rules limit the secondary use of genetic data for research in the case of new projects activated after the acquisition of the informed consent; this greatly impact, for example, on the possibility to run retrospective clinical studies. Secondary use of data should always reflect patients’ preferences and the meaningfulness of the so obtained results for both the health of the single patient and other interests, included those of his/her family. The scientific validity of results should also be considered, together with the strength of evidences linking them to risks for patient’s health and to the possibility to make the new results “actionable”. The informed consent should also explain how would be managed results that may impact on the family of patients, as well as how data can be shared or possibly commercialised, the intellectual property arising from them and their transfer to third parties. 

Psychological support is important 

The patient should be supported by a psychologist both before the execution of the testing and for the interpretation of results, through the activation of genetic counselling procedures. This is important to ensure the decisions assumed by patients truly reflect their values and interests, including the right to refuse genetic testing and/or therapies. Due to the possible impact on the patients’ family, it should be the patient him/herself to communicate the results of genetic testing to its relatives. Medical doctors should be only involved in this process – upon consultation with the patient and the hospital’s ethical committee. 

The psychological impact of genetics and genetic testing relates also to the possible discriminations that may occur at the level of human rights, work environment or insurances. The Declaration states the need to protect both patients and their families from this risk, with a particular attention to the more vulnerable people. 

Genetic testing still has a quite high impact of healthcare costs, that should always be carefully evaluated in comparison to the expected benefit for the patient. Informed consent should also detail information on the reliability, accuracy, quality and limits of the specific tests, an aspect particularly relevant when considering the highly growing market of direct-to-consumer genetic testing. 

This sort of testing is currently widely used in the contest of clinical trials to support the action of a certain medicinal product; this is a very delicate phase, according to the Declaration, as the testing may reveal a serious risk for patient’s health. In this case too, genetic counselling should be always provided in order to identify together with the patient the more appropriate therapeutic intervention.

The use of gene editing on somatic cells or that of gene therapy should reflect the autonomous decision of the patient, and reflect the deontology of the medical profession. With this respect, the informed consent should also list all possible risks linked to this type of intervention, that should be always activated after the running of a detailed risk-benefit analysis of the perceived efficacy of the therapy. 

How to support the diffusion of genetics in healthcare

Genomic sequencing is transforming the clinical practice, thanks to the support of a total $4 billion investments in national genomic-medicines initiatives by governments of several countries, according to a paper published in The American Journal of Human Genetics. The different approaches taken by countries e.g. the US, the UK, France and Australia have been discussed with reference to both the organisation of local healthcare systems, active initiatives in the field of genome sequencing and the infrastructures supporting them, and policies to improve the acquisition and management of data and the corresponding investments. Wider international collaborations are envisaged in order to expand the representativity of the population reference dataset needed to improve the variant interpretation at the global level and the discovery of the genes associated with still orphan diseases. Such collaborations should also prove useful for the optimisation of health priorities and healthcare systems.

Evidence generation and data sharing are the two key priorities identified, which have to face the very big amount of investments needed to further expand this sort of population profiling. There is still an insufficient body of evidence to support the clinical utility of genomic testing, according to authors, and reimbursement methods are also considered unsuited to support the transformation. Data silos are still the main barrier to the sharing of data and the development of knowledge databases. The solution suggested asks for a wider availability of technical standards and policy guidance to make possible the real transformation of how healthcare shall be provided to patients in coming years.

Genomic information and the pharmaceutical business

The NIH’s workshop “Genome-Based Therapeutics: Targeted Drug Discovery and Development” addressed issues related to the use of genomic information in the development of new therapies (see here the Workshop Summary). Genomics data are today widely used in R&D in conjunction to artificial intelligence to identify new targets, solve complex biological pathways, support the identification of possible respondents to therapies or foreseen the adverse effects of a certain treatment.

The pharmaceutical industry greatly invested towards this direction, both in the form of the  acquisition of smaller biotech companies where the technologies were created and of collaborative partnerships with academic and other  institutional partners. According to Nicholas Davies from PwC, the pharmaceutical industry invested an estimated $125 billion in R&D across the industry, 5% of which specifically on genetic and genomic research.

Reduction of costs for genomic sequencing and other technologies i.e. CRISPR-Cas9, and the development of companion diagnostics greatly improved R&D activities for the development of new therapeutic approaches. According to the FDA, more than 110 marketed drugs have pharmacogenetic biomarkers on the label, was reported during the workshop. Oncology remains the most studied area, followed by the cardiovascular system, central nervous system, and immune system. Even if genomic testing is now widely diffused, according to Nicholas Davies  many of these methods are not being employed in late-stage development due to a reluctance of pharmaceutical companies to enable genomic- or genetic-based trials. The dimension of the potential market, far less than that of a traditional medicine, is also a common worry for the industry, but it might be counterbalanced by the greater attention to cost-effectiveness and outcomes-driven therapies.

Pharmacists in Medical Affairs: roles in the pharmaceutical industry in the UK

Giuliana Miglierini
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Pharmacists in Medical Affairs: roles in the pharmaceutical industry in the UK

Pharmacists have a broad understanding of the development process of a medicine, its pharmacology and formulation, its clinical use and side effects. They are therefore able to succeed in many different roles within the pharmaceutical industry (see here for some examples). Furthermore, the ability of pharmacists to understand the basis of a certain pharmacological approach and to explain its therapeutic use and practical implications are particularly useful with respect to the many innovative therapies that have entered the market in recent years (e.g. CAR-T therapies).

Medical Affairs and Medical/Scientific Advisors

Medical Affairs is one of the three key strategic pillars of a pharmaceutical company (along with Commercial and Research & Development) (see here a McKinsey report on “A Vision for Medical Affairs in 2025”).  Medical Affairs is a critical partner in ensuring the success of the company’s medicinal products, including successful product launches based on scientific excellence.  Medical Affairs also acts as the recognized voice and conscience of the patient within the company.

Roles in Medical Affairs have traditionally been covered by medical doctors, but in recent years a new trend is emerging to include pharmacists in these highly specialised professional positions. Examples of possible interesting roles for pharmacists within the Medical Affairs structure of a pharmaceutical company include Medical Advisor, Medical Information Specialist, Medical Science Liaison, Medical Education Specialist, Medical Affairs Operations and Compliance Specialist. A very significant new development in the UK has been the expansion of the role of Nominated Signatory to include registered pharmacists.

The role of Medical Advisor, for example, involves being the scientific expert on the clinical use of a medicinal product and working closely with commercial colleagues on scientific communications with healthcare professionals.  The role also involves the management of post-authorisation clinical studies and the scientific training for the sales force. Medical Advisors also maintain peer-to-peer contacts with key opinion leaders.

The Nominated Signatory

The UK Code of Practice for the Pharmaceutical Industry, independently administered by the Prescription Medicines Code of Practice Authority (PMCPA) now includes UK registered pharmacists within the professionals entitled to assume the role of Nominated Signatory (see here and here more details). This role is involved in the certification of all promotional and educational activities and materials produced by pharmaceutical companies for use by healthcare professionals and patients.

The role of Nominated Signatory is a critically important one, that carries a significant amount of responsibility. Pharmacists who take on this role are required to have appropriate product knowledge, relevant experience, length of service and seniority plus an up-to-date and detailed knowledge of the Code. All qualifications and details of signatories have to be notified to the UK Medicines and Healthcare Products Regulatory Agency (MHRA), as well as to the PMCPA.

The Compliance Specialist

Checking for the compliance of all processes within a certain company or organisation with respects to regulatory requirements and standards is the main duty of the Compliance Specialist. This role requires a deep knowledge of pharmaceutical legislation and regulatory guidances. Internal auditing may also be a key activity to be performed, together with the identification of strategies to solve any compliance issues and the monitoring of the relevant legislation. This role is also connected to the Nominated Signatory role, as it has to confirm compliance of all promotional and marketing materials to the regulatory requirements. It may be a good transition role for a pharmacist wishing to become Nominated Signatory.

The Medical Science Liaison

This role, often described as MSL, is responsible for establishing and maintaining scientific relationships with key opinion leaders (physicians, academic researchers, etc) within a specific geographic area. The roles are focused on a specific therapeutic area and support the correct use of medicinal products within the area. MSLs also provide scientific information and advise on latest developments and new clinical data. The ability to keep up-to-date with the scientific knowledge of their therapeutic area is a key requirement to cover the MSL role. MSLs are field based to travel within their assigned geographic area to keep in contact with key opinion leaders, and the roles have a good level of autonomy and flexibility compared to other roles in medical affairs.

The Medical Information Specialist

MIS often represents an entry role for pharmacists into the pharmaceutical industry, particularly for pharmacists who have worked in other sectors (e.g. community or hospital pharmacy). Medical Information is responsible for answering clinical and scientific questions from healthcare professionals (including physicians and pharmacists), and patients. As seen for the MSL, it is important to be able to clearly understand and explain the clinical and pharmaceutical use of medicines, combined with experience of analyzing scientific data. Medical Information also involves writing scientific summaries of the data on a particular topic, which can be used to respond quickly to questions from healthcare professionals. The UK has seen the development of specialised contact centres for Medical Information services to answer first line questions coming from patients or healthcare professionals. Roles within pharmaceutical companies have become more specialized as a result, dealing with the complex questions and generating resources to be used in responding to questions.  There has also been a growth in the use of digital communication channels to provide information to healthcare professionals.

Closely related to the MSL and MSI roles are also Medical Education Specialists (MESs), whose focus is on the provision of medical education resources on a specific product or therapeutic area to both healthcare professionals and patients. This may also occur through the use of a mix of different media and communication channels, i.e. web platforms, social media, etc.

Medical Affairs Operations

This role is responsible for the coordination of all the different activities included in the Medical Affairs function and organisational structure. The development and implementation of medical affairs strategies is a typical role, together with the monitoring of performance and the procurement of all needed resources (both materials and human). Skills in project management are highly advisable to success in these positions, which involve very complex cross-functional networking with a variety of  teams and activities.

A report on the EU Public Health Policies

Giuliana Miglierini
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A report on the EU Public Health Policies

The DG Internal Policies of the European Commission has just released a report addressing the outlook on the different fields of healthcare; the document was a specific request of the Committee on the Environment, Public Health and Food Safety of the EU Parliament (ENVI) and it aims to shape the public health agenda during the new legislature. 

Despite great investments in healthcare (roughly 10% of the EU GDP), many Europeans still suffer for health needs, especially in the more vulnerable parts of the population. The report takes WHO’s definition of health, i.e. “a state of complete physical, mental and social well-being”, upon which the absence of disease is not enough to guarantee a good health condition. “The aim of public health policy is to create the enabling conditions for people to maintain their health, improve health and well-being, and prevent ill-health or the deterioration of their health”, states the report as a declaration of principles. The document analyses the different lines of action already ongoing or among the priorities listed in the Mission Letter to the Commissioner-designate for Health (Stella Kyriakides). We briefly address the main topics related to the pharmaceutical industry.

The determinants of health

Many are the factors impacting on health status, that are discussed in detail by the report; it also refers to the “Health in All Policies” (HiAP) approach, according to which health impacts should be considered in all relevant policy processes.

Social, or socio-economic, determinants of health relate to the conditions people live and work: income, employment and education are important points to be considered, also with relation to inequalities and inequities. 

Environmental determinants of health are very wide group of different factors – from pollution to occupational health, from climate change to urban environments, up to water quality and noise levels – that may act at a double level both on human and environmental health. 

The organisation of health systems and their resources are another important determinant to deliver “quality services to all people, when and where they need them” and without causing financial problems.

Activities run by the private sector refer to the commercial determinants of health; roles and rules used to run these activities may influence health, as for example different corporate practices that have been identified to have negative implications, says the report.

Individual determinants of health are listed as the last factor, and it includes biological factors and lifestyle.

Affordable and innovative medicines 

The supply of affordable medicines and an innovative pharmaceutical industry should be a major priority for the future Health Commissioner, states the report.

Sustainability of innovative medicines is quite an old issue still waiting for real solutions. Several legislative measures have been already undertaken at the EU level to facilitate access, from the 2017 Resolution on options for improving access to the Council Conclusions on strengthening the balance in the EU’s pharmaceutical systems (2016). BeNeLuxAI and the Valletta Declaration Group of countries are examples of collaborations at the member State level aimed to better negotiate with the corporates the price of medicinal products. The SPC Regulation may also be  of help in improving access, when it will fully come into force in 2022.

Another hot issue waiting for solution is shortage of medicines; EMA Task Force is still working, after the publication of a guidance on the detection and notification of shortages and good practice indications to communicate with the public.

On the side of innovative products, a Staff Working document on medicines for rare diseases is expected to be published by the Commission by the end of 2019. Personalised medicines is closely linked to digitalisation, to better characterise individuals’ phenotypes and genotypes, to identify the best therapeutic options or to prevent the insurgence of the disease. Strictly related is also genomics, which may greatly impact on diagnosis and therapy. The report underlines that genomics policy still falls under the EU Digital Single Market. “Another challenge is to ensure that developments and research demands in personalised medicine and genomics do not overshadow attention to existing effective health interventions, or compromise individual rights”, is the warning of the document.

Other relevant indications

The implementation of the Clinical Trials Regulation in 2020, new agreements and guidelines on GMP and GDP, international cooperation and falsified medicines are among the other topics to be monitored in the incoming years. 

Guido Rasi will close its mandate as EMA’s executive director in November 2020. While waiting for his substitute, the Agency is also called to recognise the remarks made by the European Ombudsman on relationship between EMA and pharmaceutical companies. “It remains to be seen which of the Ombudsman’s recommendations will be taken on board by the Agency”, is the comment of the report.

The new Medical Devices Regulation will entry into force on May 2020; the report questions about the current number of notified bodies to be enough to ensure the availability of devices. An issue to be closely monitored, as well as the implementation of the regulation; devices will  now fall under the competences of the Health Directorate of the new Commission. 

Many suggestions on how to improve the Cross-Border Healthcare directive have been advanced by the European Court of Auditors and the EU Parliament, and are waiting for decisions at the Commission level. 

How UK’s science is preparing to afford the post-Brexit

Giuliana Miglierini
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0
How UK’s science is preparing to afford the post-Brexit

The Brexit issue is still not closed after three years of complex negotiations; while waiting for the results of UK’s general elections fixed on the 12th December, the new deadline has shifted at the 31st January 2020. In the mean time, the debate on the post-Brexit future of UK’s science is flourishing. A report published by the Government compared the country research base’ statistical release with those from all G7 countries, Brazil, China, India, Russia, South Korea and other international benchmarks.

According to the report, UK’s field-weighted citation impact has ranked 1st in the G7 every year since 2007. In 2018 the country produced 7% of all publications at the global level (third after the US, 22%, and China, 19%), a percentage increasing to 14% when considering just the most high-cited ones (vs US 37%, China 20%). In a twenty years period (1998-2018) the proportion of UK’s publications based on international collaborations has doubled (55% in 2018 vs 26% in 1998). UK is the second most internationally collaborative country in the G7, after France (56%) and significantly higher than the OECD average (31%).

Ambitious goals for the future of UK’s science

The possible future scenario for British science was considered in a report commissioned by the Government to Professor Sir Adrian Smith (The Alan Turing Institute) and Professor Graeme Reid (Chair of Science and Research Policy at University College London), published at the beginning of November. 

The initiative aims to provide independent advice on the design of potential future UK funding schemes for international, innovation and curiosity-driven blue-skies research, in the case it would not be possible for the country to continue with the association to Horizon Europe. The exercise is not trivial as, for example, the authors have been struck “by evidence across regions of the UK of the ways in which strands of EU structural funds and regional development support have been combined with research and innovation funding to play a vital role in developing local economies”. 

The ability to attract and retain the best international talents is a prerequisite to maintain UK’s scientific leadership at the global level. The suggestion advanced by the two independent experts is, in the case of a no-deal Brexit, to maintain the same level of investment as the country received in the past from participation in EU programmes (around £1.5 billion/year) and to use such funds to stabilise and protect the internal R&D environment and to built new forms of international collaboration (not only at the European level). The Brexit should represent in any case an opportunity to boost UK’s ecosystem for research, without disruption of existing activities in the case of a hard exit. 

The new vision depicted by the report includes the creation of an international version of the highly successful UK Research Partnership Investment Fund to attract foreign direct investments, a coherent Global Talent Strategy, combining reforms to immigration policy with a suite of fellowship and post graduate programmes, the availability of substantial additional funding for basic research, and a flagship programme of research fellowships offering large awards over long periods of time for exceptional researchers. 

Several options have been also provided on how to manage administrative structures supporting investments, among which international partnership with funding agencies and businesses are the preferred ones. A possibility would be for the UK to create a “bigger and brighter” global rival to the European Research Council (ERC) in the case of a hard Brexit, says an article published on Science|Business. This new entity would be managed by an international organisation and would be open to both UK and international scientists.

The chair of the UK House of Common’s science and technology committee, Norman Lamb, also commented before 31st October the possibility of a no-deal exit in an interview to Science|Business, during which he said to be confident “the country will eventually gain full access to the 2021 – 2027 EU research programme, Horizon Europe“.

Comments from the pharmaceutical industry

The Smith-Reid review received comments from the Association of the British Pharmaceutical industry (ABPI), which represents one of the main investors in UK’s economy with £4.3 billion spent in R&D and 63,000 people employed. “This report is clear on some of the things that need to be done, if the UK wants to keep one of its most valuable assets”, said Dr Sheuli Porkess, Executive Director, Research, Medical and Innovation at ABPI.

The Association mentions the 13,000 project participations and around €5.9 billion in funding from Horizon 2020 (13.5% of the total, second to Germany; data June 2019). The requests of the pharmaceutical industry in view of 31st January 2020 remains the same: to negotiate a continued access to long-term European funding and collaboration programmes for science, a continued participation in the European Investment Bank and European Investments Fund (including a seat at the Board), an agreement to facilitate the movement for highly skilled talent across borders, a UK immigration system needs-based, straightforward and rapid.

The future legislative framework for medicinal products and technologies is one of the most delicate issues to be addressed in negotiations, as it may impact citizens’ health at both sides of the Channel. According to ABPI, the UK should seek to negotiate alignment and commonality with the EU for the regulation of medicines, through a regulatory cooperation agreement or a mutual recognition agreement with EMA. Focus should be paid to the continued alignment of current and future regulations as well as the continued UK participation in EU regulatory and medicines safety processes. 

The impact of the unwished scenario of a no-deal Brexit on the British healthcare system has been also deeply examined by Steve Brozak in an article published on Forbes.

A manifesto to support the biotech sector

The UK BioIndustry Association (BIA) has launched a biotech manifesto in view of the general election of 12 December, with its recommendations on how political parties can support the innovative UK life sciences sector. “This general election is an opportunity for political parties to back this exciting sector”, said Steve Bates, Chief Executive of BIA.

According to the manifesto, UK’s biotech employs over 250,000 people (two thirds outside London and the South East), with a yearly contribution of over £30 billion to the UK’s GDP. The Association asks the government to continue pursuing the target of R&D representing 2.4% of GDP by 2027 (and 3% in the long-term). A long-term investment plan to be published in 2020 would help increase the sector confidence to make R&D investment commitments in the UK. SMEs should continue to be supported by the Biomedical Catalyst, an initiative that should be re-filled by the government, that should also encourage pension schemes to invest more in the UK’s innovative young businesses.

The manifesto confirms the request for regulatory alignment already seen above, and asks for new modalities to assess medicines to enter the NHS that would not prioritise cost-effectiveness over a more holistic understanding of real-world value and the wider impact on patients’ quality of life. The appraisal system should be more flexible, suggests BIA, not to act as an undue barrier to patient access, particularly recognising the data limitations for rare diseases.

The position of the Royal Society

Many British scientific associations are working to maintain the leadership of the country in R&D, and supporting its competitiveness at the global level even in the worst scenario of an hard exit from the European Union. 

The Royal Society (RS), for example, also aims to keep highly-skilled scientists already working in the UK and attract new talents, while at the same time ensuring access to money and scientific networks and maintaining regulatory alignment to allow access to new medicines and technologies.

According to the RS, in the case of a no-deal exit the UK could lose access to over £1 billion a year in EU research funding, a sum that could decrease to half a billion with the UK government’s guarantees. The impact on research underway in the UK would be immediate, while the implementation of new alternatives may need years of time.

Costs may also have a great impact on families of foreign researchers remaining to work in the UK, should the government apply immigration charges to EU nationals, based on the current system. The Royal Society estimates that an EU academic with a partner and two children entering the country on a 3 year Tier 2 visa would have to pay upfront costs equivalent to 20% of their annual salary. Data show that currently 18% of academic researchers active in the country come from the EU, 13% from extra-EU countries; this ratio inverts (35% from non-EU countries and 13% from EU) in the case of post-doc researchers. 

EMA’s view on method validation

Giuliana Miglierini
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0
EMA’s view on method validation

In an article published in Clinical Pharmacology & Therapeutics, regulators and academics discuss factors still limiting the regulatory acceptability of clinical data coming from sources different than classical randomised trials (RCT). The authors, among which are EMA’s Executive Director Guido Rasi, the Senior Medical Officer, Hans-Georg Eichler, and the Chair and Vice-Chair of EMA’s human medicines Committee (CHMP), focused on how to adapt the design of clinical studies to consider the new sources of real-world data. The suggestion is for companies to work on the validation of innovative methods for clinical development, while looking for support from regulators to be dispensed through EMA’s methods qualification advice procedure.

The new era of not-randomised data

The increasing diffusion of new data sources (i.e. smart or wearable devices) and analytical algorithms for their interpretation is deeply changing how healthcare services are provided to patients. The new scenario also includes pharmaceutical development, where the increased diffusion of personalised medicine corresponds to the need for clinical studies to be run on smaller and more selected groups of patients. Post-authorisations phases are also often requested by the authorities in order to monitor the impact of a new treatment in real-world conditions. 

The Clin. Pharmac. & Ther. paper analyses from a regulatory perspective the current limits to acceptability of not-randomised analytical methods (e.g. statistical, epidemiological, etc.). Randomised clinical trials traditionally represent the preferred way to validate outcomes of a development project; they are based on highly selected cohorts of patients, and results are obtained through statistical analysis and interpretation of data. 

The method is not useful to validate real-world trials, where the considered population has not been selected against strict criteria, including for example the possible presence of co-morbidities. New statistical and analytical methods are thus needed to support the decision process during which regulatory authorities evaluate the data submitted with the application.

Not an easy goal to be achieved

As for every kind of analytical method, also those used for real-world data analysis need to be tested and validated before they can be applied for regulatory purposes. Data coming from electronic health records (e-HR), or from sharing results of terminated studies, from cross-trail analysis or through the combination of real-world data with the e-HR’s or insurance ones, are just few of the many possible sources yet available. 

According to the authors, two main factors are currently limiting the true usefulness of this type of data:  the availability of proper technical-operative skills and the lack of a clear governance for data. Other possible bias may result from data coming from not-randomised studies. Randomised trials should thus continue to represent the main, standard tool to develop new medicinal products, says the paper. Emerging approaches would constitute just a complementary possibility, to be used when the traditional design for the trial is not possible or acceptable, for example due to ethical reasons. 

Experts also claim that “we see various degrees of methodology aversion in all stakeholder groups within the pharmaceutical ecosystem”: a quite open criticism to those who say regulators are unwilling to adopt novel statistical (and other) methods for data analysis while, on the other hand, they would also work to “recklessly abandoning the ‘gold standard’ RCT”. “We concur that unfounded methodology aversion is a potential roadblock to making the best use of new data sources”, writes the authors.

A pre-agreed plan for validation

The suggestion made by the experts of regulatory affairs is to validate the new analytical methods in the same way it would be done for a medicinal product, in a prospectively and well‐controlled manner and following a plan pre-agreed with regulators. EMA’s procedure for methods qualification advice may be of help with this respect, together with an active role of health technology assessment bodies, payers and patients’ associations. The proposed modalities would represent, from the European regulators perspective, an efficient and transparent platform for the development and validation of study design. 

The article also includes many examples that may follow under the proposed new vision, from the “borrowing of data” from trials already terminated, to the use of external control groups and threshold crossing, from the indirect comparison of the relative efficacy to the possibility to fully replace randomised trials with the real-world model. Methodology evaluation may be run in parallel to the standard way of development (both in the pre- and post-marketing phase), is the suggestion coming from the article, and it would require investments judged by the authors “not prohibitive” if compared to costs and times typically needed to generate data by mean of interventional studies. These activities, they further suggest, may be also financed by the European Union through the IMI Initiative. 

The CHMP opinion on the method

According to the proposed approach to validation, the CHMP may also release opinions on the acceptability of a certain method on the basis of specific scientific questions. From this point of view, a hot issue is the possibility to completely substitute randomised clinical trials with real-world studies, so to allow great savings both in terms of costs and time. But the track record of not-randomised comparative trials is still not enough convincing to support this type of transition, say the experts, and many are the reported cases of divergent results obtained with the two type of experimental design on both the size and direction of the observed effect of the experimental drug. 

The prospective and structured evaluation of real-world studies would help limiting the risk of “adjusted” results, in the case outcomes of randomised trials are already available. Some bias might occur at the level of different geographical areas, warn the authors, thus making difficult, for example, the direct extrapolation in Europe of data produced in the US (or viceversa). 

An open and agnostic mind-set is the ultimate key to achieving credibility, write the regulatory experts: pharmaceutical companies should not limit to test new approaches making “dry runs with old products”. “We emphasise that if developers want trial assessors to accept novel methods, they will have to expose some of their experimental drugs to methodology development exercises”, is the call to the industry made in the conclusions. A firewall should be put in place to ensure the complete separation of method and product evaluation: a very challenging target to be achieved, that requires the strong collaboration of all different actors involved in the development and regulatory approval of a new medicine, including also funding agencies those investments are needed to support the long-term sustainability of the transition. 

The sustainability of the development model in the pharmaceutical sector

Giuliana Miglierini
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0
The sustainability of the development model in the pharmaceutical sector

The issue of the time needed for the recovery of R&D costs in the pharmaceutical industry is certainly not new; the high costs typical of last and next generation biological therapies also pose many questions on the sustainability of the current model. 

According to an article by Jonathan Kimball published on StatNews, the yet not ratified US-Mexico-Canada trade agreement (UMSCA) should continue to protect the business through a provision limiting “the ability of Congress to decrease monopoly rights for developers of new biologic drugs to less than 10 years” (it is now 8 years in Canada).

Small-molecule drugs are granted a shorter market exclusivity in the US, just 5 years (that may become around 7 due to regulatory times needed to reach approval for the generic versions). Exclusivity is currently 10 years in Europe (not considering SPCs’ extensions) and 5 years in Australia and New Zealand.

The official costs for R&D

The last estimates from PhRMA, the American association of the pharmaceutical industry, indicates an average cost of $2.6 billion and 10-15 years time to develop a new medicinal product. This amount of money might be greatly lower, according to an article by Public Citizen summarising also the many critics to the Tufts Center “official” calculations advanced some years ago by the British Medical Journal. 

Investment in R&D made by biopharmaceutical industry in 2016 was around $90 billion, says PhRMA. The following year, the Association set new R&D requirements for its members, including a 3-year average global R&D/global sales ratio of 10 percent or greater, and a 3-year average global R&D spending of at least $200 million per year.

In Europe, the European Federation of Pharmaceutical Industry Associations (EFPIA) highlights the increased value innovative medicines represent for patients. Many types of tumor, for example, are now curable thanks to advanced therapies, and hepatitis C can be defeated in more than 90% of cases using the fourth generation of therapies (vs 41% of first generation, in years 1999-2010).

According to EFPIA, “innovative medicines can put healthcare systems on a more sustainable path by reducing costs in other parts of the healthcare systems such as hospitalisations and clinician times”. Furthermore, the biopharmaceutical industry would also represent a value for the overall economy in terms of job creation, R&D investment, patients’ productivity and ability to continue contributing to the community. R&D spending represented in Europe 14,4% of net sales in 2014. The Federation claims that the combination of generic price erosion and price regulation resulted in a 24% decrease of medicines’ prices, compared to a 30% rise in consumer prices in the period 2000-2013. Many European countries are already testing innovative and flexible pricing and funding models, i.e. outcome-based reimbursement, patient access schemes or managed entry agreements. But it remains unclear, from the data presented by EFPIA here, the times and true costs afforded for the development.

Costs and times under scrutiny

Development costs estimated by the Tufts Center for the Study of Drug Development (TCSDD) are often taken as the gold reference, as they are elaborated in close collaboration with the pharmaceutical industry providing the data. An article published in JAMA Internal Medicine in November 2017 by researchers of the Memorial Sloan Kettering Cancer Center indicates that these estimates may be affected by a lack of transparency and independent replication. 

The authors calculated the median R&D spending on cancer drug development for 10 companies and drugs. Development time resulted on average 7.3 years; 50% of the examined candidates products received accelerated regulatory approval. The median cost of drug development was $648.0 million, increasing to $757.4 million when considering a 7% per annum cost of capital (opportunity costs). The 10 products under consideration produced after 4 years $67.0 billion total revenues from sales, representing almost ten times the total R&D spending ($7.2 billion, or $9.1 billion including 7% opportunity costs).

In the US, the Biologics Price Competition and Innovation of 2009 grants 12 years of market exclusivity for new biological medicines, based on the assumption of longer times needed for their development. A recent article published on Nature Biotechnology took into consideration the difference in development time for medicinal products based on small-molecules vs biologics.

According to the study, average pre-market development times for biologics are around 12 years, similar to that of small-molecule medicines. The regression analysis using the Merck Index source for data showed indeed a 2.5-2.9 years shorter time for the development of biological vs small-molecule drugs. Considering the differential exclusivity on the US market, the authors argue that the development of new biological medicines might be favoured compared to small molecules. 

R&D costs for orphan vs non-orphan medicines

Many biopharmaceutical companies in the last decade focused their attention on the development of new therapies for rare diseases, the so-called orphan medicines. These are often advanced gene or cellular therapies, and they are usually characterised by very high prices on the market, due to the small number of target patients. The development of new orphan therapies often benefits also of incentives and dedicated regulatory pathways to accelerate access. 

A research group from the University of Toronto compared costs for the development of 100 orphan medicines (approved between 2000 and 2015) to that of 100 non-orphan ones; results have been published on the Orphanet Journal of Rare Diseases. 

Researchers analysed public available data from a total of more than a thousand clinical trials, making calculations of their expected costs. Outcomes of this analysis may sound astonishing: clinical development costs for new orphan medicines are much lower than those referred to non-orphan products ($166 million vs $291 million, respectively, for out-of-pocket clinical costs;  $291 million vs $412 million, respectively, for capitalised clinical costs. 2013 USD). The capitalised cost per approved orphan drug containing a New Molecular Entity (NME) was found to be half that of a non-orphan product.

The duration of clinical trials resulted almost double for orphan medicines compared to non-orphan ones; possible causes have been identified in the lower number of patients and their geographical dispersion, the fewer information on disease prevalence and incidence, the lack of data on natural disease progression, and a lower medical expertise. 

Out-of-pocket costs were lower for phase 1 and 2 in the non-orphan group, but higher for phase 3. This last phase is much more frequent for non-orphan products (often it is not even run for orphan medicines), a possible explanation for the observed rise in costs together with the higher funding from various institutions available for orphan medicines. The authors ask for further research to obtain a consensus on cost categories to be included in the analysis. “When considering value of drugs, more discussion is required before assessing whether recouping R&D costs should be a consideration when setting prices for drugs”, they write.

The new EU Commissioners for Health and Research: proposals and suggestions for their action

Giuliana Miglierini
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0
The new EU Commissioners for Health and Research: proposals and suggestions for their action

The new European Commission is due to enter into force from 1st November, after the Commissioners-designated were heard and voted by the European Parliament. Many are also the suggestions advanced by stakeholders in view of the five years term of action of the von der Leyen Commission.

Stella Kyriakides is the new Health Commissioner

The new Commissioner for Environment, Health and Food safety is Cyprus’ representative Stella Kyriakides; she has been heard on 1st October by the ENVI Commission of the European Parliament. The new Health Commissioner is a clinical psychologist with a long experience both as a politician and in the field of patients’ advocacy, where she held several top positions in important organisations in the field of cancer (Europa Donna European Breast Cancer Coalition, Europa Donna Cyprus Breast Cancer Forum, Patient Advisory Committee (PAC) of the European Cancer Organisation, Cyprus’ National Committee on Cancer) (see here the complete CV). 

A moment of Stella Kyriakides hearing (credits: European Parliament)

In her speech to the ENVI Committee, Mrs Kyriakides fully embraced the new vision of a green new deal and sustainable revolution aimed to create a healthier, new digital world. The proposed Beating European Cancer Plan should be one of the main actions to be delivered within her agenda, to be achieved using an holistic approach. 

Research to find new treatment options for cancer – but also new diagnostics and new opportunities for survival and palliative care – should be one of the key activities of the Cancer Plan, together with the creation of a European Health Data Space and the fight of antimicrobial resistance. Innovation in digital technologies applied to the health sector shall be a fundamental enabler to reach the goal.

Answering to the questions posed during the hearing, Stella Kyriakides said screening programmes for cancer should be accredited and based on common European guidelines. Other themes touched included shortage of medicines – a hot problem on which the new Commissioner took time to deep her understanding – and the possible return on the EU territory of many pharmaceutical companies which had delocalised their activities in extra-EU countries. Digitalisation and support to scientific development are key issues to be addressed to make this possible – said Mrs Kyriakides – together with a better attention to price and reimbursement policies, health technology assessment and new business models. Stella Kyriakides told about the possibility to improve cross-border paediatric clinical studies, even if she would wait for the termination of EMA’s evaluations before any decision about the possible review of the EU regulations on paediatrics and orphan drugs.

Mariya Gabriel is the Commissioner for Innovation and Youth

The new Commissioner for Innovation and Youth is Mariya Gabriel, who served already as EU Commissioner for Digital Economy and Society in the Juncker Commission. The continuity of action with her previous role will inspire Mrs Gabriel future action, she said during the joint hearing with the Committee on Industry, Research and Energy and the Committee on Culture and Education of the European Parliament. 

Mariya Gabriel during the hearing (credits: European Parliament)

New synergies of actions across the different European programmes for research, innovation and education – i.e. Horizon Europe, Erasmus, Creative Europe, DiscoverEU – shall be one of the priority for Mariya Gabriel, in order to reach a more coherent framework “able to act on the lives of European citizens”. Mrs Gabriel too recalled the importance of the new green agenda to support ecological, social and economic transitions. 

The European Union should continue to be a global leader in research, said the Commissioner-designated; to achieve this, the EU framework programme shall remain open and attractive. Another important point to boost competitiveness is the ability to maintain in the EU territory both top scientists and young and innovative, small and medium size companies: a future target for the European Innovation Council, said Mrs Gabriel. Partenariats are another possible “win-win solution” she suggested during the hearing, as well as the possible creation of a European space for education and European universities. Furthermore, the Erasmus programme may become in future “greener” and more inclusive. 

These very ambitious goals should be pursuits, according to the candidate Commissioner, by tripling the investments available within the EU’s budget. Mariya Gabriel also suggested that Horizon Europe’s mission councils should name a person responsible to keep contacts with the EU Parliament, in order to better inform it on new evolutions and decisions. A first pilot project might be activated in 2020, she said. The candidate Commissioner also explained that Horizon Europe’s missions “will be public goods, we have to convince citizens that it is necessary and used for them”. A possible communication campaign to better link science with people and young generations may be a possible way to address this goal. 

Mariya Gabriel’s commitment to support excellent research in Europe has been welcomed with a note by Science Europe, the association representing many primary funding European institutions.

A call to higher Horizon Europe’s budget

In the meantime, on 16 September, 93 European associations in the field of research and innovation (among which EFPIA, EuropaBio, MedTech Europe and Vaccines Europe) signed a Joint Statement urging the von der Leyen Commission to make research, development and innovation (RD&I) a priority within the next Multiannual Financial Framework 2021-2027.

The request is to raise Horizon Europe’s budget to at least €120 billion (in 2018 prices); at least 60% of the sum should be dedicated to the “Global Challenges and European Industrial Competitiveness” pillar, considered essential to break down silos and promote the flow of knowledge between public research and industry. According to the Statement, this wider financial basis would allow not only to safeguard the competitiveness of the European Union, but would also support the creation of up to 100,000 jobs in RD&I activities between 2021-2027, with a €11 return of GDP for each euro invested over 25 years.

EFPIA’s priorities and the European Cancer Dashboard

EFPIA’s representative Kristene Peers summarised from the Federation’s blog the key priorities in the pharmaceutical sector, starting from an affordable and rapid access to medicines that can extend or transform the lives of patients. The industry also wishes a regulatory system able to accelerate rather than hold back access to safe and effective innovative medicines and the availability of incentives to support research and development of new treatments.

The EFPIA Oncology Platform – in collaboration with European Cancer Organisation (ECCO) and European Cancer Patient Coalition (ECPC) – has also announced the organisation of a session during the European Health Forum Gastein (EHFG) specifically designed to discuss how to approach the development of the European Cancer Plan which is one of the core proposals of the new Health Commissioner. 

The final goal, says EFPIA, is to inspire discussion about the Plan by introducing a European Cancer Dashboard based on the recently updated Comparator Report on patient access to cancer medicines in Europe. The expected outcomes to be identified are key-performance indicators (e.g. survival, time to return to work, rehabilitation, progress, quality of care, expenditure and innovation) to be used within the new Plan.

Other suggestions for health and research

According to the new vision of the circular economy, the above mentioned two EU’s Commissioners should not be the only one to deal with innovation. A major role may be also granted to the new commissioner for Internal Market still to be designated (the first candidate, France’s Sylvie Goulard, failed the hearings, blocking de facto the final vote of the Parliament), and Margrethe Vestager, EU’s vice-president for the Digital Age. This last agenda is particularly compelling, as Mrs Vestager is expected to rapidly produce a new strategy for artificial intelligence and its ethical implications (see here Science | Business).

Another hot issue waiting for the action of the new Commission is the possible competition between two important European institutions dealing with research, the European Innovation Council (EIC) and the European Institute of Innovation and Technology (EIT). This topic has been addressed by the director general of the DG Research, Jean-Eric Paquet, during a hearing at the EU Parliament (see here more). The two institutions should offer different services and funding opportunities to researchers and companies, said Paquet. The new layout for the mission of the Innovation and Youth Commissioner, that will include for the first time also education under the same umbrella, may also ask for a new design for the directorates. 

The think-tank Bruegel’s indication is to keep the ERC independent from any sort of oversight. To facilitate access to scientific information, Bruegel also suggests the new EU Commission to act in order to lower the subscription fees of scientific journals and to run negotiations with publishers on fair prices for providing open access. As for education, the think-tank suggests revamping the Marie Curie scientist training programme (see here more). 

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