NAVLIN BRIEF:
Following a review, the European Medicines Agency’s (EMA) human medicines committee (CHMP) has recommended authorization of AstraZeneca’s long-acting antibody (LAAB) combination, Evusheld (tixagevimab co-packaged with cilgavimab), for use within the European Union (EU) for the prevention of COVID-19 in a broad population of adults and adolescents aged 12 years and older weighing at least 40 kg.
The National Institute for Health and Care Excellence (NICE) has recommended BioMarin’s Vimizin (elosulfase alfa) as an option for the treatment of patients with mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome). The Institute based its decision on real-world evidence (RWE) from 69 people who received NHS-funded treatment since 2015 as part of a managed access agreement. NICE added that the health and quality-of-life benefits of Vimizin are considered to be substantial. In a release, NICE states that the list price for Vimizin is £750 per 5 mg vial; however, the company has a commercial arrangement (simple discount patient access scheme), which makes the drug available to the NHS with a discount.
U.S. Senators Susan Collins (R-ME) and Jeanne Shaheen (D-NH) are currently leading Senate talks to lower insulin costs as they seek to update their bill, introduced by the duo in July 2019 as the Insulin Price Reduction Act, that “would hold Pharmacy Benefits Managers (PBMs), pharmaceutical companies and insurers accountable for … surging prices by incentivizing reductions in list prices,” specifically for insulin, primarily by tackling rebates and deductibles. Senate Majority Leader Chuck Schumer (D-NY) said of the talks to make insulin more affordable, “The effort has my support,” adding, “Over the next few weeks … offices will work on getting that bill finalized, and I intend to put that bill on the floor as soon as possible after Easter recess.”
THE DETAILS
COVID-19
Following a review, the European Medicines Agency’s (EMA) human medicines committee (CHMP) has recommended authorization of AstraZeneca’s long-acting antibody (LAAB) combination, Evusheld (tixagevimab co-packaged with cilgavimab), for use within the European Union (EU) for the prevention of COVID-19 in a broad population of adults and adolescents aged 12 years and older weighing at least 40 kg.
Pfizer is supplying UNICEF with as many as 4 million courses of Paxlovid (nirmatrelvir + ritonavir) for the treatment of COVID-19 in 95 countries, announced the company. Pfizer said it will be able to fulfil the order starting in April of this year and continuing until the end of 2022, depending on demand and regulatory authorizations.
In response to critics, the Department of Disease Control (DDC) of Thailand emphasized on Saturday that the influenza antiviral drug favipiravir is reasonably priced and highly effective in the treatment of COVID-19, reported local press. The medication costs just 800 baht per course of treatment, compared to remesivir, which costs 1,512 baht, and molnupiravir and paxlovid, which both cost 10,000 baht, according to Opas Karnkawinpong, director-general of the DDC.
The Ministry of Food and Drug Safety (MFDS) of South Korea has granted emergency authorization to Merck’s investigational oral antiviral pill, Lagevrio (molnupiravir), to treat COVID-19. Local media reported that the MFDS reviewed the safety and efficacy of Lagevrio and concluded that the oral pill is needed to deal with the public health crisis, making it the second pill to be used to treat COVID-19 in South Korea, following Pfizer’s COVID-19 antiviral Paxlovid (nirmatrelvir/ritonavir).
The Drugs Controller General of India (DCGI) has granted emergency use to Novavax’s COVID-19 vaccine Covovax (SARS-CoV-2 rS Protein (COVID-19) recombinant spike protein nanoparticle vaccine) for adolescents aged 12 to 18. The authorization is based on the results of a Phase 2/3 observer-blinded, randomized, controlled study, which demonstrated that Covovax was well-tolerated with a reassuring safety profile in a total of 460 Indian adolescents aged 12 to 18 years. The authorization in India also references the ongoing PREVENT-19 pivotal Phase 3 paediatric expansion trial of NVX-CoV2373 in adolescents in the U.S. aged 12 to 18, results of which were shared in February.
Moderna announced it signed a new agreement with Switzerland to supply 7 million doses of its COVID-19 booster vaccine starting in 2023. The agreement also includes an option for the delivery of seven million doses in 2023 and 2024.
POLICY
The autonomous community of Andalusia, Spain, is seeking feedback on proposed modifications to Law 22/2007 on Pharmacy in Andalusia that would eliminate the drug auction process by which the region purchases medicines. The text of the law reads: “Eliminate, as unnecessary, the mechanism currently planned for the selection of medicines, which is voluntary.” President of Andalusia Juan Manuel Morena previously declared an end to drug auctions in Andalusia in January 2021, calling the auction “a system that we promised to change because it supposed a disguised cut in the rights of Andalusians to access to medicines and a clear interference with the freedom of prescription and placed Andalusians in a situation of inequality compared to other Spaniards.”
U.S. Senators Susan Collins (R-ME) and Jeanne Shaheen (D-NH) are currently leading Senate talks to lower insulin costs as they seek to update their bill, introduced by the duo in July 2019 as the Insulin Price Reduction Act, that “would hold Pharmacy Benefits Managers (PBMs), pharmaceutical companies and insurers accountable for … surging prices by incentivizing reductions in list prices,” specifically for insulin, primarily by tackling rebates and deductibles. Schumer said of the talks to make insulin more affordable, “The effort has my support,” adding, “Over the next few weeks … offices will work on getting that bill finalized, and I intend to put that bill on the floor as soon as possible after Easter recess.”
Senator Chuck Grassley (R-IA) is urging the Federal Trade Commission (FTC) to move forward with the plan to study the business practices of Pharmacy Benefit Managers (PBMs) after the commission came to a deadlocked vote in February. Highlighting bipartisan support for a deeper dive into PBM practices, the letter from Grassley and other legislators is calling for a more targeted study to gauge the impact these players have on prescription drug pricing. Grassley’s legislation, S. 1388, the Prescription Pricing for the People Act, would require the FTC to conduct a study and provide a report to Congress on the effects of consolidation and potentially anticompetitive behaviour of PBMs that may impact prescription drug pricing.
HTA
The National Institute for Health and Care Excellence (NICE) has recommended BioMarin’s Vimizin (elosulfase alfa) as an option for the treatment of patients with mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome). The Institute based its decision on real-world evidence (RWE) from 69 people who received NHS-funded treatment since 2015 as part of a managed access agreement. NICE added that the health and quality-of-life benefits of Vimizin are considered to be substantial. In a release, NICE states that the list price for Vimizin is £750 per 5 mg vial; however, the company has a commercial arrangement (simple discount patient access scheme), which makes the drug available to the NHS with a discount.
NICE has also recommended AbbVie’s Venclyxto (venetoclax) with low-dose cytarabine as an option for treating untreated acute myeloid leukaemia (AML) in adults when intensive chemotherapy is unsuitable.
In addition, NICE has published appraisal guidance recommending Bristol Myers Squibb’s Opdivo (nivolumab) plus Yervoy (ipilimumab) for untreated advanced renal cell carcinoma (RCC) in adults. According to a release, the appraisal reviews the additional evidence collected as part of the Cancer Drugs Fund (CDF) managed access agreement for the drug combination.
The Federal Joint Committee (G-BA) of Germany has reassessed Janssen’s orphan drug Darzalex (daratumumab) and found it has “considerable added benefit” for the treatment of patients with newly diagnosed multiple myeloma (MM) who are not eligible for an autologous stem cell transplant (ASCT). According to the G-BA, Darzalex, in combination with lenalidomide and dexamethasone, showed advantages in OS and life quality over one of the comparators, lenalidomide, and dexamethasone.
The High Health Authority (HAS) of France has granted early access to Merck’s anti-PD-1 therapy, Keytruda (pembrolizumab) in combination with Lenvima (lenvatinib) for the treatment of adult patients with advanced or recurrent endometrial carcinoma (EC) with disease progression during or following treatment with any line of platinum-based therapy who are not eligible for curative surgery or radiotherapy.
(HAS has also granted early access to Sanofi’s Xenpozyme (olipudase alfa), an investigational enzyme replacement therapy for the treatment of acid sphingomyelinase deficiency (ASMD).
PRICING & REIMBURSEMENT
The U.S. Department of Health and Human Services (HHS) said it suspects the use of biosimilars in Part B is limited due to the manner in which it reimburses providers. At the moment, providers are not strongly incentivized to use biosimilars. The study is expected to examine Part B 2021 expenditure and calculate the amounts spent on reference biologics and biosimilars by both Medicare and its beneficiaries. It will then look into the potential impact on costs if alternative policies are put in place or if the use of biosimilars is broadened. In other news, HHS is looking into risk-adjustment tactics that are contributing to overpayments to some Medicare Advantage (MA) plans, and the U.S. Food and Drug Administration (FDA) just revised its guidance to broaden the participation of adults aged 65 years and older in clinical trial enrolment.
Trent Twomey, National President of the Pharmacy Guild of Australia, said, “Research shows that some 900,000 Australians delayed getting prescriptions due to cost in 2019-20, and 31% of middle-income households find it hard to afford medicines” without a concession card. As it stands, the general co-payment in Australia is AU$42.50, after the maximum amount was raised on January 1 of this year, although the trajectory is that it will hit the AU$50 mark, warned the guild.
Boehringer Ingelheim’s Ofev (nintedanib) is now reimbursed in Italy for the treatment of adults with chronic fibrosing interstitial lung disease (ILD) with a progressive phenotype, announced the Italian Medicines Agency (AIFA). Ofev 60 100 mg capsules and 60 150 mg capsules have an ex-factory price (excluding VAT) of EUR 2,719.29 and a retail price (VAT included) of EUR 4,487.92, with an obligatory discount for all indications reimbursed to be applied at all public health facilities.
The High Health Authority (HAS) of France has published a favourable opinion for reimbursing Deciphera’s orphan drug, Qinlock (ripretinib), for the treatment of adult patients with advanced gastrointestinal stromal tumour (GIST) who have already been treated with three or more medicines of the kinase inhibitor class, including a medicine called imatinib.
The Pharmaceutical Benefits Agency (PHARMAC) of New Zealand has announced a bundle deal with AstraZeneca for lung cancer and ovarian cancer treatments, as well as severe eosinophilic asthma treatments. Bundle deals are negotiated by PHARMAC with one company for multiple medicines and allow PHARMAC to provide access to even more treatments at competitive rates.
DRUG APPROVAL
The Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization (CMA) to Takeda’s Exkivity (mobocertinib) as a monotherapy treatment for adult patients with epidermal growth factor receptor (EGFR) Exon 20 insertion mutation-positive (Exon20ins+) locally advanced or metastatic non-small cell lung cancer (NSCLC) who have received prior platinum-based chemotherapy. The CMA gives Exkivity an edge over Johnson & Johnson’s Rybrevant (amivantamab), an intravenous treatment that is still awaiting endorsement in England. In May of last year, the U.S. Food and Drug Administration (FDA) signed off on Rybrevant; four months later, the FDA approved Exkivity.
Aura Biosciences has announced that the European Commission (EC) has granted Orphan Drug Designation (ODD) to its AU-011 to treat uveal melanoma, which includes choroidal melanoma as well as melanomas of the iris and the ciliary body.
Merck’s Keytruda (pembrolizumab) has been approved by the U.S. FDA as a single agent for the treatment of patients with advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
The U.S. FDA has approved Bristol Myers Squibb’s first-in-class Opdualag (nivolumab and relatlimab-rmbw) in paediatric unresectable or metastatic melanoma, announced the company.
The FDA also approved a label update for Viiv & Janssen’s HIV drug, Cabenuva (cabotegravir and rilpivirine), allowing healthcare professionals and people living with HIV in the U.S. the option to start this once-monthly or every-two-months injectable treatment without the need for the oral lead-in phase.
The FDA has awarded Pfizer’s respiratory syncytial virus (RSV) vaccine candidate Breakthrough Therapy Designation for the prevention of lower respiratory tract disease caused by RSV in individuals 60 years of age or older.
EPA CONGRESS 2022
Claus Runge, Global Head of Market Access Public Affairs & Sustainability at Bayer, chaired a panel discussion on navigating the market access, RWE and pricing landscape in a pandemic. Christoph Glätzer, Chief Global Value and Access Officer at Johnson & Johnson; Saira Jan, Vice-President & Chief Pharmacy Officer at Horizon Blue Cross Blue Shield of New Jersey; Claudia Neuber, Head of Global Pricing at AstraZeneca; and Jenniffer Prescott, Programme Director – Process and Operations at NICE, discussed the existing challenges, how we can embrace opportunities and look to the future based on learnings from the pandemic.
Wim Goettsch, Special HTA-advisor at the Dutch National Health Care Institute (Zorginstituut Nederland) chaired a roundtable with Bart Van den Daele, Head of External Affairs – Director Market Access, Gilead Belux, and Inneke Van De Vijver, Strategic Advisor – Reimbursement Pharmaceuticals, RIZIV-INAMI, and a number of other industry experts. According to the table, “Working toward the future, you could say that not all the assessments thus far were successful, but it is working in the right direction and more aspects are becoming certain and established. This is the way we are going. EUnetHTA will be implemented soon, and BeNeLuxA is always maturing.”
Pascaline Faivre, Senior Director Market Access EU at Certara, presented an overview of access mechanisms for rare disease treatments in Europe, including recent changes and challenges. The orphan medicines reimbursement system cost the healthcare systems of the EU member states approximately EUR 20-25 billion between 2000 and 2017, and worldwide orphan drug sales are forecast to grow at a CAGR of 12.3% from 2019 to 2024 – essentially doubling the orphan drug market.
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