NAVLIN BRIEF:
The Economic Committee for Health Products (CEPS) and pharmaceutical industry association (Leem) in France have signed off amendments to the country’s orphan drug pricing framework. The amendments in question make changes to Article 15 of the existing framework, which sets out the pricing mechanism for “particular drugs” – a scope that includes orphan medicinal products. The document stipulates that drugs meeting the orphan criteria must be assessed under the rules of the framework with “regard, in particular, to their ASMR levels” based on two specificities. Additionally, in the absence of a conventional agreement under the rules, and if the annualized cost claimed per patient exceeds the threshold amount of EUR 50,000, the Committee or the company may propose that, in return for a face price consistent with those practiced internationally, the company promises to supply the drug to all patients eligible for treatment without any restriction for a contractually negotiated limited amount of turnover.
The European Medicines Agency (EMA) and EUnetHTA have published a new joint document work plan for 2021-2023, outlining the main areas of activity and expected outcomes through the upcoming joint EU HTA initiative. The document is a product of the European Commission inviting the two organizations to establish a joint work plan for delivering on previously identified priorities. According to the plan, deliverables on the HTA side will either be actioned by EUnetHTA21 if related to their service contract delivery or alternatively through individual HTA bodies from the consortium or beyond who are from a European (EU/EEA) Member State and express interest to participate. The two groups recognize that the implementation of the activities needs to be flexible so that “such collaborative work can transition into a legislative framework on European HTA cooperation, once adopted”.
The Government of Canada announced it will be moving forward with the amendments to the Patented Medicine Prices Review Board (PMPRB) regulations, meaning they will take effect on 1 July 2022. The regulatory reforms have been stalled four times due to the COVID-19 pandemic. Following a consultation with stakeholders in March 2022, Canada will implement the new basket of comparator countries – dubbed the PMPRB11 – and reduced reporting requirements for those medicines at lowest risk of excessive pricing. As such, PMPRB will soon be relying on an updated basket of reference countries that will comprise France, Germany, Italy, Sweden, the UK, Australia, Belgium, Japan, the Netherlands and Spain. The U.S. and Switzerland were swapped out for markets where drug prices tend to be lower.
The Details
COVID-19
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the use of Moderna’s COVID-19 vaccine, Spikevax, in children between 6 and 11 years old after the drug met the required safety, quality and effectiveness standards set by the Agency.
The MHRA has granted Conditional Marketing Authorization (CMA) to Valneva’s COVID-19 vaccine. The approval makes the MHRA the first regulatory body in the world to approve the Valneva vaccine, adding it to its existing arsenal of five COVID-19 vaccines for use in England.
Pfizer and BioNTech announced new phase 2/3 data on a third dose of their COVID-19 vaccine in children from 5 through 11 years of age showing that the vaccine triggers an immune response to both the Omicron variant and the wild-type strain.
The Department of Health and Social Care (DHSC) has announced that it will expand access to Pfizer’s oral antiviral COVID-19 treatment, Paxlovid (nirmatrelvir and ritonavir), to thousands more vulnerable people by adding it to a trial to assess how best to use the drug in its highly vaccinated population. The DHSC stated that the drug has been added to the PANORAMIC national study, the UK’s fastest-ever recruiting clinical trial of its kind, which is run by the University of Oxford in close collaboration with general practitioner (GP) hubs.
Congress has agreed on a deal to provide $10 billion in funding for urgent COVID-19 needs and therapeutics that was introduced by Senate Majority Leader Chuck Schumer (D-NY) and Senator Mitt Romney (R-UT). Romney stated: “Today’s agreement [repurposes] $10 billion to provide needed domestic COVID health response tools. Half of the funding will be used for the development and purchase of therapeutics, potentially eliminating the need for future vaccine and mask mandates.” Of the $9.25 billion specifically for the Biomedical Advanced Research and Development Authority (BARDA), no less than $5 billion should be for R&D, manufacturing, producing, purchasing and administrating therapeutics, and $750 million will be for research and clinical trials for emerging coronavirus variants and supporting sustainable and expanded vaccine manufacturing capacity.
The Vaccine Alliance (GAVI) has secured an additional $4.8 billion in funding for the COVAX initiative to help ensure equitable access to COVID-19 vaccines. Speaking at the 2022 Break COVID Now Summit, Professor José Manuel Barroso, Gavi Board Chair, said: “This pandemic is not over; while gaps in coverage remain, it will continue to wreak havoc on individuals, communities and economies. Achieving equity is the key to breaking COVID now. We cannot afford to wait and risk the virus undoing all the progress that has been made.”
Taiwan’s government inked an agreement to buy 700,000 courses of Pfizer’s COVID-19 antiviral pill, Paxlovid, amid a rise in Omicron cases, reported Taiwan News. The agreement was announced by the Central Epidemic Command Center (CECC), which said Paxlovid allows for patients with mild cases to recover at home, enabling clinics to focus on those with severe symptoms.
Japan has announced that it has cancelled the purchase of around 40 million AstraZeneca COVID-19 vaccine doses it agreed to buy last year. According to Reuters, a health ministry official said the contract allowed the government to cancel a portion of the supply if it was “unneeded.”
The Swiss Agency for Therapeutic Products (Swissmedic) has announced that it has granted temporary approval to Novavax’s COVID-19 vaccine, Nuvaxovid, for people aged 18 and over, making it the fourth COVID-19 vaccine to be approved in Switzerland. The temporary approval is backed by the favourable decision of the European Medicines Agency (EMA) on 20 December 2021. According to Swissmedic, phase 3 data from the U.S., Mexico and the UK demonstrated an efficacy of roughly 90% against symptomatic COVID-19 and 100% in preventing severe COVID-19.
POLICY
According to sources seen by NAVLIN, the recently proposed German Federal Ministry of Health (BMG) legislation has been “withdrawn at short notice,” following criticism that it was “too drastic.” In March, the BMG published a draft law on the financial stabilization of the statutory health insurance (GKV-Finanzstabilisierungsgesetz) proposing a number of measures, including increased mandatory discount on patented drugs from 2023 and a new restriction in the current “orphan privilege” rule; however, since the initial announcement, local publication Gerechte Gesundheit reported on March 29 that the Ministry has officially withdrawn the law, with Federal Minister of Health Karl Lauterbach promising to “present a well-considered draft law in good time”.
The Economic Committee for Health Products (CEPS) and pharmaceutical industry association (Leem) in France have signed off amendments to the country’s orphan drug pricing framework. The amendments in question make changes to Article 15 of the existing framework, which sets out the pricing mechanism for “particular drugs” – a scope that includes orphan medicinal products. The document stipulates that drugs meeting the orphan criteria must be assessed under the rules of the framework with “regard, in particular, to their ASMR levels” based on two specificities. Additionally, in the absence of a conventional agreement under the rules, and if the annualized cost claimed per patient exceeds the threshold amount of EUR 50,000, the Committee or the company may propose that, in return for a face price consistent with those practiced internationally, the company promises to supply the drug to all patients eligible for treatment without any restriction for a contractually negotiated limited amount of turnover.
The Government of Canada announced it will be moving forward with the amendments to the Patented Medicine Prices Review Board (PMPRB) regulations, meaning they will take effect on July 1, 2022. The regulatory reforms have been stalled four times due to the COVID-19 pandemic. Following a consultation with stakeholders in March 2022, Canada will implement the new basket of comparator countries – dubbed the PMPRB11 – and reduced reporting requirements for those medicines at lowest risk of excessive pricing. As such, PMPRB will soon be relying on an updated basket of reference countries that will comprise France, Germany, Italy, Sweden, the UK, Australia, Belgium, Japan, the Netherlands and Spain. The U.S. and Switzerland were swapped out for markets where drug prices tend to be lower.
The Association of Research-Based Pharmaceutical Companies (AIFD) of Turkey is pushing for a revamp of the pharmaceutical pricing system in Turkey since Turkey currently lags when it comes to accessing new drugs that have been approved in Europe. Turkey has no access to 79% of the new drugs approved in Europe in the last three years due to the pricing policy in place that depends on foreign currency. The euro rate used in drug pricing for this year is 6.29, representing an increase of 37.4%.
Ecuador is seeking to allow patients who receive an electronic prescription at a public hospital to fill their prescription at private pharmacies free of charge, with the Ministry of Public Health (MSP) providing a list of over 300 drugs that should be available through the private pharmacy network. The plan was to take effect in March of this year in six hospitals before being implemented in specialized, general and basic hospitals and type C health centres by the end of September 2022; however, the plan did not manifest in March as announced.
HTA
The European Medicines Agency (EMA) and EUnetHTA have published a new joint document work plan for 2021-2023, outlining the main areas of activity and expected outcomes through the upcoming joint EU HTA initiative. The document is a product of the European Commission inviting the two organizations to establish a joint work plan for delivering on previously identified priorities. According to the plan, deliverables on the HTA side will either be actioned by EUnetHTA21 if related to their service contract delivery or alternatively through individual HTA bodies from the consortium or beyond who are from a European (EU/EEA) Member State and express interest to participate. The two groups recognize that the implementation of the activities needs to be flexible, so that “such collaborative work can transition into a legislative framework on European HTA cooperation, once adopted”.
The High Health Authority (HAS) of France has given an unfavourable opinion for the reimbursement of Swedish Orphan Biovitrum’s Kineret (anakinra) for the treatment of COVID-19 in adults with pneumonia requiring supplemental oxygen and who are at risk of developing severe respiratory failure, as determined by blood levels of a protein called suPAR (soluble urokinase plasminogen activator receptor) of at least 6 ng per ml. The decision is based on HAS’s Transparency Commission (CT), which stated that “although the available data suggested a modest improvement in the patient’s clinical status at 28 days”, the side effects of the drug are “poorly established” due to the absence of robust data on the reduction in mortality or progression to invasive mechanical ventilation or death.
The National Institute for Health and Care Excellence (NICE) has published a final appraisal document recommending Vumerity (diroximel fumarate) for the treatment of relapsing-remitting multiple sclerosis (RRMS), specifically for patients who do not have highly active or rapidly evolving severe RRMS, on the condition that Biogen provides the drug according to the commercial arrangement agreed on. NICE noted the list price of Vumerity as £1,471.07 per pack; however, Biogen has a commercial arrangement in place with the NHS that makes the therapy available with a confidential discount. According to the document, Vumerity is expected to be as clinically effective as Biogen’s own Tecfidera (dimethyl fumarate), which NICE already recommends for the indication.
Speaking at a recent press conference, Nathalie Schalck, Eisai’s regional oncology sales manager, confirmed that the company’s targeted therapy Lenvima (lenvatinib) is now available in France for the treatment of endometrial carcinoma (EC) patients with disease progression during or following treatment with any line of platinum-based therapy who are not eligible for curative surgery or radiotherapy.
PRICING & REIMBURSEMENT
The Institute for Clinical and Economic Review (ICER) has published a draft evidence report on bluebird bio’s Zynteglo/LentiGlobin (betibeglogene autotemcel) for the treatment of beta thalassemia. The U.S. Food and Drug Administration (FDA) set a revised Prescription Drug User Fee Act (PDUFA) target action date of August 19, 2022 for Zynteglo and the company has said pricing will involve an outcome-based payment plan of five equal yearly payments totaling $2.1 million for individuals who achieve and maintain transfusion independence. ICER has preliminarily concluded, “Despite remaining uncertainties, the evidence suggests that beti-cel provides net health benefits to patients with TDT, and given the high annual costs of standard care, traditional cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at a cumulative price of $2.1 million if paid through an outcomes-based contract for patients with sustained transfusion independence”
The National Institute for Health and Care Excellence (NICE) has published draft guidance estimating the value of Fetcroja (cefiderocol) and Zavicefta (ceftazidime–avibactam) on the country’s NHS, as part of a new initiative with NHS England, NHS Improvement and the Department of Health and Social Care (DHSC) to incentivize antimicrobial resistance (AMR) research. The drugs are set to be reimbursed via a proposed “subscription-style payment model,” which has been designed to try and address the lack of new antimicrobials being developed and the growing threat posed by antimicrobial resistance – The initiative is part of the UK’s 20-year vision for AMR, which outlines developing and testing “new models for national purchasing that de-link the price paid for antimicrobials from the volumes sold, using a NICE led healthcare technology assessment to support robust stewardship.” The NICE draft guidance on the two technologies provides an estimate of their benefits to the health of the overall population in England measured in quality-adjusted life-years (QALYs); NICE is the first health technology assessment organization anywhere in the world to attempt to estimate the full value of an antimicrobial in this way.
The National Institute for Health and Care Excellence (NICE) has published a final appraisal document recommending Merck’s Tepmetko (tepotinib) for routine use across the NHS in England through Project Orbis for adult patients with advanced non-small-cell lung cancer (NSCLC) with METex14 skipping alterations – overturning its original negative opinion.
At its March meeting, the Scottish Medicines Consortium (SMC) agreed to reimburse Orchard Therapeutics’ Libmeldy (atidarsagene autotemcel) for the treatment of pediatric metachromatic leukodystrophy (MLD). However, as clinical evidence on Libmeldy is limited due to the nature of both the drug and the disease, the Consortium granted access on the condition that Orchard gathers data on its efficacy in the real world for three years, as “despite a confidential discount provided by the company, the cost in relation to the health benefits remains high.” The organization also made recommendations on two other therapies at the March meeting, Venclyxto (venetoclax) and Rinvoq (upadacitinib), for the treatment of acute myeloid leukaemia (AML) and moderate-to-severe atopic dermatitis, respectively.
The Italian Medicines Agency (AIFA) has approved Orchard Therapeutics’ Libmeldy (atidarsagene autotemcel) for the treatment of children with metachromatic leukodystrophy (MLD). According to a release, AIFA approved the one-shot therapy at an ex-factory price of EUR 2,875,000 and a public price of EUR 4,744,936.23.
The Zorginstituut (ZIN) has advised the Netherlands’ Minister of Health, Welfare and Sport to include Bayer’s Kerendia (finerenone) in appendix 1B in the GVS, for insured person aged 18 or older with chronic kidney damage with type 2 diabetes mellitus. Kerendia was assessed through the Netherlands’ new Parallel Procedures pathway.
The European Federation of Pharmaceutical Industries and Associations (EFPIA) has announced its members have pledged to step up measures to help deliver faster, more equitable access to medicines for millions of patients across the European Union (EU). According to EFPIA, members pledge to file for reimbursement by national health systems no later than two years after EU regulatory approval, “provided that local systems allow it.” EFPIA Director General Nathalie Moll believes that industry and governments can create a step-change in the access landscape for patients across Europe “by reducing the time it takes to file for pricing and reimbursement in all 27 EU member States, by bringing greater transparency to the barriers and delays to access, by co-creating an equity-based pricing system, by adopting an efficient system of EU assessments of relative efficacy and by sharing information on the implementation of novel pricing mechanisms.”
DRUG APPROVAL
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Connoya’s CMG901 for the treatment of advanced gastric cancer and gastroesophageal junction adenocarcinoma, announced the company. In March of last year, the FDA gave Connoya approval to conduct a phase I clinical trial of the product in both cancers. CMG901 is the first antibody conjugate drug targeting Claudin 18.2 to receive IND approval in both China and the U.S.
Health Canada granted marketing authorization for Jardiance (empagliflozin) 10 mg in chronic heart failure as adjunct to standard of care therapy, announced Boehringer Ingelheim and Eli Lilly. The treatment is the first to receive approval for patients with chronic heart failure regardless of ejection fraction, which means the approval covers both reduced and preserved ejection fraction (HFrEF and HFpEF).
The National Medical Products Administration (NMPA) of China has accepted Shanghai Henlius Biotech’s New Drug Application (NDA) for Hansizhuang (serplulimab) for use in combination with chemotherapy for the first-line treatment of extensive stage small cell lung cancer (ES-SCLC).
Health Canada has approved an indication expansion for Merck’s Gardasil 9 (human papillomavirus 9-valent vaccine, recombinant) to include prevention of oropharyngeal and other head and neck cancer caused by HPV types 16, 18, 31, 33, 45, 52,and 58 in individuals aged nine through 45 years.
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