On 24th November 2020, the European Commission delivered on one of the items mentioned in the mission letter of Stella Kyriakides, Commissioner for Health and Food Safety. The new Pharmaceutical Strategy for Europe forms an essential building block for the European Health Union that was announced earlier this month. The European Association of Hospital Pharmacists (EAHP) and its members welcome the publication of the Pharmaceutical Strategy and its all-encompassing nature focusing on the whole life cycle of a medicine stretching from research and authorisation over its supply to the safe distribution to patients.
EAHP President Petr Horák highlighted that “Patient-centredness, which lies at the heart of all representations made by hospital pharmacists, is a key element for the delivery of high-quality, effective and safe care. Also, cooperation and communication via a structural dialogue can contribute immensely to the improvement of health and care in Europe. Since many patients are still without access to the essential medicines they need and since the COVID-19 pandemic has uncovered several problems in our system, EAHP is committed to working together with the European institutions and other stakeholders to build the European Health Union that our patients deserve.”
The new Pharmaceutical Strategy puts forward suggestions for legislative and non-legislative changes in four areas, namely:
Ensuring access to affordable medicines for patients and addressing unmet medical needs;
Supporting competitiveness, innovation and sustainability of the EU’s pharmaceutical industry and the development of high quality, safe, effective and greener medicines;
Enhancing crisis preparedness and response mechanisms, diversified and secure supply chains as well as addressing medicines shortages; and
Ensuring a strong EU voice in the world, by promoting a high level of quality, efficacy and safety standards.
Commenting on the third pillar of the Strategy, Aida Batista (EAHP Vice President and Board member working on the topic of medicines shortages) acknowledged the intention of reinforcing supply obligations and requiring early notification. “For over ten years, hospital pharmacists have been raising awareness about the impact that medicines shortages have on patient care. For EAHP it is of uttermost importance that the proposals for the diversification of the supply chain, the adoption of preventive and mitigating measures as well as the improved notification of shortages to healthcare professionals and patients are implemented promptly in collaboration with all stakeholders. EAHP and its members are committed to continue their work linked to medicines shortages and to support European action on this issue.”
In addition to medicines shortages, the Pharmaceutical Strategy for Europe touches on several areas relevant for hospital pharmacists, including but not limited to the growing threat of antimicrobial resistance, the potential of the digital transformation in health and care, patient safety and the COVID-19 pandemic. To better serve all European patients, it is important for EAHP and its membership that different risk management tools, for example, single unit dose barcoding, quality control committees and computerised order entry systems, are widely implemented in hospitals to lower medication errors for the benefits of patients. Also, the facilitation of the delivery of pharmacy preparations and the investment into compounding services must be promoted to better respond to patient needs and extraordinary situations like pandemics and shortages of essential medicines.
For EAHP’s President-Elect András Süle, who will be heading the Association during the implementation period of most of the ambitious proposals put forward, the engagement of hospital pharmacists and their views is crucial for making the Pharmaceutical Strategy a success. He underlined that “Measures for optimising the use of antibiotics should factor in the experiences made with antimicrobial stewardship teams in hospitals. Unmet medical needs and access to medicines could be better addressed through utilising the unique compounding skills of hospital pharmacists and their expertise in medicines procurement. Also, seamless transitions between the interfaces of different health settings need to be considered during the implementation of the Pharmaceutical Strategy to ensure that the patient care started in hospitals can be continued in the community.”
FIP publishes the report of the regional conference for the European region
In autumn 2019, EAHP’s Vice President Aida Batista attended the Regional Conference for the European Region of the International Pharmaceutical Federation (FIP) to share the Association’s views on medicines shortages and to discuss the importance of seamless care for patients. The event was held in Ankara, Turkey and brought together pharmacists from many different countries.
The aim of the FIP Regional Conference for the European Region Report was to act as a regional roadmap for action to transform primary healthcare. The report describes the outcomes of the conference, including the results of a survey among all pharmacy leaders in the European Region to follow-up on national priorities and strategies to implement the Ankara Commitment to Action. The report also provides a focus chapter on the mapping of the FIP Development Goals against the Ankara Commitment to Action to outline pharmaceutical practice, service, education and workforce development needs in the context of primary healthcare.
Celebrate the European Antibiotic Awareness Day
On 18th November 2020 the European Union celebrated the European Antibiotic Awareness Day (EAAD). This year’s edition was held fully online. Pharmacists, doctors, nurses, hospital managers, farmers, veterinarians, policymakers, professional and patient organisations, governmental institutions, and the general public were encouraged to share how they #KeepAntibioticsWorking.
Like every year, the EAAD fell within the WAAW which has seen a name change to World Antimicrobial Awareness Week. The WHO decided to switch from ‘antibiotics’ to the more encompassing and inclusive term ‘antimicrobials’, which covers antibiotic, antiviral, antifungal and antiprotozoal medicines. Multiple factors – including overuse of medicines in humans, livestock, and agriculture, as well as poor access to clean water, sanitation and hygiene – have accelerated the threat of antimicrobial resistance worldwide. The theme of the WAAW in 2020, that was running from 18 to 24 November, was ‘United to preserve antimicrobials’.
As part of the EAAD, healthcare professionals were invited to join the global Twitter storm on 18 November at 2 PM CET. By tweeting about #AntibioticResistance and #WAAW they raised awareness of the issue. If you wish to participate, make sure to post this through your Twitter account at that time or during the day, together with hashtags that are relevant for you.
Moving towards a European Health Union
In November, the European Commission announced its plans for a European Health Union to better protect the health of its citizens and improve collective responses to cross-border health crises such as the COVID-19 pandemic. The proposals put forward will help the EU to better respond to Member States’ needs and to build a strong and resilient Europe Health Union that can withstand other health threats in the future.
The proposals – which take into account the lessons learnt from COVID-19 – focus on revamping the existing legal framework for serious cross-border threats to health as well as reinforcing the crisis preparedness and common coordination by strengthening the roles of two key EU agencies, the European Centre for Disease Prevention and Control (ECDC) and the European Medicines Agency (EMA). With greater support, the ECDC can help increase capacities for preparedness, surveillance, early warning, and risk assessment, while an enhanced EMA can help address the vulnerability in the supply chains for both medical products and medicines by better monitoring and mitigating these situations.
EU-JAMRAI launches its first antibiotic resistance symbol
Last year, on the occasion of the celebration of the EAAD, the Joint Action on Antimicrobial Resistance and Healthcare-Associated Infections (EU-JAMRAI) launched a design contest to find a symbol of the threat against antibiotic resistance. With the new symbol, EU-JAMRAI seeks to raise awareness of antimicrobial resistance, a great unknown to many people.
After almost a year and 600 applications from 44 different countries, EU-JAMRAI announced last week the winning symbol. The symbol combines two capsules, 2 band-aids and two hearts. In addition, it is easy to make yourself and easy to recognise. Congratulations to Swedish designer David Ljungberg!
#VaccinesWork – Social media toolkit for healthcare professionals
As part of the #VaccinesWork campaign, the European Commission has put together a social media toolkit which seeks to fight disinformation. The toolkit targets healthcare professionals since they play a key role in proving accurate information about vaccines.
Vaccine hesitancy was identified by WHO as one of the top ten threats to global health. Consequently, the toolkit on ‘Spreading authoritative information online’ tries to ensure that false narratives both in day-to-day interactions with patients and through activity on social media are countered by healthcare professionals. The document intends to guide them in that process. It lays out, in several steps, the proven approaches and strategies that can help react to this threat against vaccines and shift the narrative back towards science-based prevention.
Updates from the European Medicines Agency
The European Medicines Agency (EMA) published new communications on the first long-acting injectable antiretroviral therapy for HIV which was recommended for approval, a new gene therapy to treat the rare genetic disorder metachromatic leukodystrophy and the first treatment for the rare condition primary hyperoxaluria type 1.
First long-acting injectable antiretroviral therapy for HIV recommended for approval
EMA has recommended the granting of marketing authorisations for two new antiretroviral (ARV) medicines, Rekambys (rilpivirine) and Vocabria injection (cabotegravir), to be used together for the treatment of patients with HIV type 1 (HIV-1) infection. The two medicines are the first ARVs that come in a long-acting injectable formulation. This means that instead of daily pills, patients receive intramuscular injections monthly or every 2 months.
The combination of Rekambys and Vocabria injection is intended for maintenance treatment of adults who have undetectable HIV levels in the blood (viral load less than 50 copies/ml) with their current ARV treatment, and when the virus has not developed resistance to certain class of anti-HIV medicines called non-nucleoside reverse transcriptase inhibitors (NNRTIs) and integrase strand transfer inhibitors (INIs).
The opinion by EMA’s human medicines committee (CHMP) is based on data from three phase three randomised, open-label, multicentre clinical trials with HIV-infected, treatment naïve (ie, never taken ARV therapies) or successfully treated men and women aged 18 years or older. These studies demonstrated the safety and efficacy of Rekambys and Vocabria regimen when administered every four or 8 weeks. Because the levels of the active substances fall slowly, it is even more important than with standard ARV medicines that patients adhere to the dosing schedule of Rekambys and Vocabria to avoid the risk of the virus developing resistance.
As for all medicines, a risk management plan (RMP) will ensure rigorous safety monitoring of the medicines once authorised across the EU. Further efficacy and safety data will be collected through a prospective cohort study and a real-world 5 year drug utilisation study. The results will be included in post-marketing safety reports. The most common side effects observed in clinical trials with the injectable regimen Rekambys and Vocabria include injection site reactions followed by headache, fever, nausea, fatigue, asthenia, myalgia and dizziness.
The opinion adopted by the CHMP at its October 2020 meeting is an intermediary step on Rekambys and Vocabria’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.
New gene therapy to treat rare genetic disorder metachromatic leukodystrophy
EMA has recommended granting a marketing authorisation in the European Union for the gene therapy Libmeldy to treat metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is indicated for use in children with the ‘late infantile’ or ‘early juvenile’ forms of MLD, who have been identified as carriers of the defective gene but have not yet developed symptoms. It is also indicated in children who have been diagnosed with the early juvenile form who have started developing symptoms but still have the ability to walk independently and before the onset of cognitive decline.
There is currently no cure for MLD, a disease caused by a fault in the gene that produces an enzyme called arylsulfatase A (ARSA). This leads to a build-up of substances called sulfatides in the central and peripheral nervous system. The damage caused by this leads to the progressive loss of motor function and cognitive ability and, ultimately, death.
In its overall assessment of the available data, the Committee for Advanced Therapies ( CAT ), EMA’s expert committee for cell- and gene-based medicines, found that the benefits of Libmeldy outweighed the possible risks in patients with the late infantile or early juvenile forms of MLD who have not yet any symptoms and in patients with early juvenile form with early symptoms of the disease. In clinical studies, Libmeldy showed that it was most effective in patients who had not yet developed symptoms. Once they had received the medicine, their performance with regard to cognitive and motor function was maintained and comparable to that of their healthy peers during the observation period.
However, in patients with the early juvenile form of MLD, who already showed symptoms when they were first given Libmeldy, the effects were less pronounced. A possible slower decline in motor function was seen while cognitive function was maintained compared with what is known about the disease progression in untreated patients. The further the disease had already progressed in these patients, the fewer positive effects could be seen. Therefore, in patients with early juvenile form of MLD who have already developed symptoms, the CAT concluded that treatment success is maximised if at the time of diagnosis, screening for treatment and treatment, children can still walk independently and have no signs of cognitive decline.
The main adverse reactions observed in trials were fever and a diminished ability to fight infections (febrile neutropenia), inflammation of the mouth and lips (stomatitis) and the gastro-intestinal tract (mucosal inflammation). These side effects are related to the conditioning medicine used to prepare the child for treatment with Libmeldy. As for all haematopoietic stem cell interventions, there is a theoretical risk that patients develop malignancies such as leukaemia or lymphoma later in life. The CHMP, EMA’s human medicines committee, agreed with the CAT’s conclusions and recommended approval of Libmeldy in these patients. As part of its recommendation for marketing authorisation, the committees requested that the company uses a registry of patients to learn more about the long-term efficacy and safety of the medicine. Results from this registry study will be submitted periodically for evaluation to EMA.
The company has also been requested to propose measures to safely reduce the overall time needed to supply the patient with the individualised product. This is because treatment is more effective before the onset of symptoms or in the presence of early mild symptoms, before symptoms become more severe and disease is rapidly progressive. Treatment with Libmeldy should only be carried out in qualified specialised treatment centres, and patients and their carers should receive extra educational material to warn them of the symptoms of leukaemia and lymphoma.
The opinion adopted by the CHMP is an intermediary step on Libmeldy’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation.
First treatment for rare condition primary hyperoxaluria type 1
EMA has recommended granting a marketing authorisation in the European Union for Oxlumo (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1). Primary hyperoxaluria is a rare inherited disorder characterised by the overproduction of oxalate. Oxalate can form calcium oxalate deposits, which can cause stones in the kidney and urinary tract (structures that carry urine) as well as injury to other organs such as the heart, eyes, bones and skin. Characteristic symptoms of the disease include renal colic, blood in the urine, frequent urinary tract infections and stomach pain. PH1 is the most common and the most severe form of the disease, accounting for 80% of all cases. The condition is very rare with an estimated prevalence of 0.05 in 10 000 people in the EU.
There are currently no approved medicines for PH1 in the EU. Different treatments are used to prevent the accumulation of calcium oxalate such as dietary changes, drinking plenty of fluids and taking vitamin B6. In certain cases kidney and/or liver transplantation is required. Untreated PH1 leads to kidney failure, which is life-threatening.
Oxlumo will be available as solution for injection (189 mg/mL). The active substance of Oxlumo is lumasiran, a small interfering RNA that causes degradation of the messenger RNA involved in the synthesis of the enzyme glycolate oxidase (GO) in the liver, leading to decreased GO enzyme levels in the body. This results in reduction of plasma and urinary oxalate levels, the underlying cause for the symptoms in patients with PH1.
The benefit of Oxlumo is its ability to reduce oxalate levels in the plasma and 24 hours urinary oxalate excretion, when compared with a placebo treatment. This was observed during a 6 month clinical trial involving 38 patients aged 6–60.
Oxlumo was accepted in EMA’s PRIME scheme and has benefited from the extra support offered by the Agency to medicines that have a particular potential to address patients' unmet medical needs. EMA’s human medicines committee (CHMP) reviewed the application for Oxlumo under its accelerated assessment procedure, which allows the speeding up of patients' access to medicines.
The opinion adopted by the CHMP is an intermediary step on Oxlumo’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation.
For more information please visit EMA’s website at https://www.ema.europa.eu.
Footnotes
Provenance and peer review: Commissioned; internally peer reviewed.