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Global Pricing Insights – Last Week In Review – May 30 – June 3 , 2022

NAVLIN Brief

  • Last July, France made changes to its long-standing Authorization for Temporary Use (ATU) and Recommendation for Temporary Use (RTU) systems via the Social Security Finance Bill 2021 (PLFSS 2021) – now that the scheme has been in place for 10 months, the National Agency for the Safety of Medicines and Health Products (ANSM) has released a review of the processes, with a view to adapt and improve them going forward. Based on the review, the High Health Authority of France (HAS) wants to refine its evaluation principles, namely the criteria it uses to determine the presence or absence of appropriate treatment. Additionally, in view of the “diversity of clinical situations encountered during the first 10 months of practice,” HAS has acknowledged that it is not possible to offer a single definition of innovation. In this context, HAS will assess various components, taking into account, in particular, the specificities of certain therapeutic areas for which there is a major medical need with regard to the mortality and morbidity of the pathology.
  • The Italian Medicines Agency (AIFA) has published a cost-effectiveness report on Orchard Therapeutics’ Libmeldy (atidarsagene autotemcel) for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene resulting in a reduction in the enzyme activity of ARSA in children with late infantile or early juvenile forms, without clinical manifestations of the disease, or in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. In the scenario analysis presented by the Economic Evaluation Office (UVE), in almost all the simulations carried out, the ICER was around the base case value, with the exception of two scenarios which were associated respectively with a maximum ICER of EUR 236,526 per QALY and minimum ICER of EUR 56,139 per QALY. In April, AIFA confirmed that the SSN could pay for the drug, often called the world’s most expensive – when used for the indication. AIFA approved the one-shot gene therapy at an ex-factory price of EUR 2,875,000 and a public price of EUR 4,744,936.23.
  • In a report released on May 25, The National Institute for Health and Care Excellence underscored its ongoing efforts to place the UK as a world leader in medicines evaluation, breaking down its “lofty ambitions” and achievements so far. The organization wants to take a “proportionate approach” to technology appraisals, by piloting and implementing a “shorter and less resource-intensive technology appraisal process for topics where a highly streamlined process can be applied.” Additionally, the organization wants to use learnings from the pandemic to produce living guidance on non-COVID topics and “begin to explore how we might replicate this across the breadth of our work”.

The Details

COVID 19

A British and Argentinian-led resolution has been announced at the World Health Assembly (WHA), which aims to protect the world from future pandemics as well as existing health threats. According to the British government, by encouraging more specific funding, the resolution will help developing countries increase their capacity to run their own clinical trials – “helping them to test new drugs, vaccines and other health interventions which will benefit their populations – while working across countries to respond to future pandemics more rapidly,” read the statement. The UK government also stated that as well as helping countries respond faster in a public health emergency, the resolution will help countries tackle existing health challenges such as tuberculosis, diabetes, and mental health problems.

The European Union is holding discussions around a joint purchase agreement for Monkeypox vaccines and antivirals, following the recent outbreak. The Health Emergency Preparedness and Response Authority (HERA) – now responsible for joint procurement activities – has reached a consensus with the member states to “acquire medical countermeasures” as soon as possible. The Union is in talks to buy Bavarian Nordic’s Imvanex and SIGA Technologies’ tecovirimat; however, no contracts have yet been signed.

Moderna has announced an agreement with the European Commission (EC) to amend their originally agreed contractual delivery schedules for the Moderna COVID-19 vaccine booster product Spikevax (mRNA-1273) or updated booster vaccine candidate. The amendment allows participating Member States to have Moderna COVID-19 vaccine booster doses scheduled for delivery in the second quarter of 2022 to be delivered later in the calendar year 2022 or early calendar year 2023. Moreover, participating Member States will have the possibility to receive the expected updated bivalent booster candidate following approval by the European Medicines Agency (EMA).

The Philippines Food and Drug Administration (PFDA) has authorized use of Moderna’s COVID-19 vaccine, Spikevax, in children aged six to 11 years. The Emergency Use Authorization (EUA) was filed by Zuelling Pharma Corporation, through its commercial arm ZP Therapeutics. For children, the vaccine will be administered in two 50 μg doses.

Novavax announced it has submitted a request with the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) to expand the conditional marketing authorization (CMA) for COVID-19 vaccine, Nuvaxovid (NVX-CoV2373). The company is seeking clearance of Nuvaxovid as a booster dose for those age 18 years and older. Novavax submitted data from the Phase 2 trial of the vaccine conducted in Australia, South Africa, and the UK (COV-BOOST trial), in which a single Nuvaxovid booster was administered in healthy adults around six months after their primary two-dose vaccination with Nuvaxovid.

Policy

Chilean President Gabriel Boric has unveiled a bill to restructure Chile’s health system, including establishing a Universal Health Fund to provide greater access to services. The health system will offer zero co-payments for people in sections C and D of the National Health Fund (FONASA), benefitting more than six million people. Further, Boric’s plan will slash wait times for surgeries by launching regional support centers for surgery. “We will ensure that all oncological surgery is resolved within 90 days and all higher-risk surgery will be resolved within six months,” said Boric.

Now that COVID-19 restrictions are being lifted in Beijing and Shanghai, China plans to continue revisions to the National Essential Drug List (NEDL). Initially set to begin in September 2021, China’s plan to revise the essential drugs list were postponed due to the COVID-19 pandemic. China’s Saibailan reported that stakeholders have drafted the first shortlist of NEDL products which will be reviewed by experts, and companies will be required to submit supporting materials, including information on safety, efficacy, and pharmaco-economics. In January, the National Health Commission’s (NHC) drug administration outlined a list of key focus areas during NEDL revisions, including aligning China with World Health Organization (WHO) standards for pediatric medicines and targeting areas of great unmet medical need.

U.S. Senators Chuck Grassley (R-IA) and Marian Cantwell (D-WA) have introduced the bipartisan Pharmacy Benefit Manager Transparency Act of 2022 that would empower the Federal Trade Commission (FTC) to boost drug price transparency and go after Pharmacy Benefit Managers (PBMs) for “unfair and deceptive practices”. In summary, the Act would ban unfair pricing schemes, prohibit arbitrary clawback payments made to pharmacies, and require PBMs to report to the FTC their earnings from spread pricing and pharmacy fees. Grassley commented, “Pharmacy benefit managers and other intermediaries in the pharmaceutical supply chain must be held accountable for increasing the cost of health care in the United States.”

In a report released on May 25, The National Institute for Health and Care Excellence underscored its ongoing efforts to place the UK as a world leader in medicines evaluation, breaking down its “lofty ambitions” and achievements so far. The organization wants to take a “proportionate approach” to technology appraisals, by piloting and implementing a “shorter and less resource-intensive technology appraisal process for topics where a highly streamlined process can be applied.” Additionally, the organization wants to use learnings from the pandemic to produce living guidance on non-COVID topics and “begin to explore how we might replicate this across the breadth of our work.”

HTA

The Finnish Medicines Agency (FIMEA) has published an evaluation of Astellas’ Padcev (enfortumab vedotin) for the treatment of adults with locally advanced or metastatic urothelial carcinoma who previously received platinum-containing chemotherapy and a PD-1/PD-L1 antibody. Considering results from the Phase 3 EV-301 study, FIMEA concluded an ICER for Padcev compared to vinflunine of EUR 116,000/QALY, although FIMEA noted that the correct cost-effectiveness ratio range for Padcev compared to vinflunine is EUR 140,000 to EUR 165,000, when considering the choice of patient population. FIMEA expects 27 patients to benefit from the drug, with an annual budget impact of €1.1 million. However, the assessment is subject to uncertainty when it comes to the number of patients, so FIMEA estimated a budgetary impact for Padcev of EUR 1.5 to EUR 2.5 million for 27-45 patients.

The transparency committee (TC) of the French High Health Authority (HAS) is in favor of reimbursing Trodelvy (sacituzumab govitecan) as monotherapy for the treatment of adults with unresectable or metastatic triple-negative breast cancer (TNBC) who have already received at least two systemic treatments, including at least one for an advanced form of the disease. HAS determined that Trodelvy as monotherapy is “Important” in terms of actual benefit (SMR) in the indication of MA and in the case of improvement of the medical service rendered (ASMR), HAS found the drug to be “Moderate (III).” According to a release by the body, HAS based its decision on the results of the IMMU-132-05 study (ASCENT study).

The Italian Medicines Agency (AIFA) has published a cost-effectiveness report on Orchard Therapeutics’ Libmeldy (atidarsagene autotemcel) for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene resulting in a reduction in the enzyme activity of ARSA in children with late infantile or early juvenile forms, without clinical manifestations of the disease, or in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. In the scenario analysis presented by the Economic Evaluation Office (UVE), in almost all the simulations carried out, the ICER was around the base case value, with the exception of two scenarios which were associated respectively with a maximum ICER of EUR 236,526 per QALY and minimum ICER of EUR 56,139 per QALY. In April, AIFA confirmed that the SSN could pay for the drug, often called the world’s most expensive – when used for the indication. AIFA approved the one-shot gene therapy at an ex-factory price of EUR 2,875,000 and a public price of EUR 4,744,936.23.

The transparency committee (TC) of the French National Authority of Health (HAS) is in favor of reimbursing an extension of Keytruda (pembrolizumab) in combination with chemotherapy for the treatment of adult patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC), whose tumor expresses PD-L1 with a CPS ≥ 10 and who have not received previous chemotherapy for metastatic disease. Keytruda, in combination with chemotherapy, is the first-line treatment for locally recurrent unresectable or metastatic TNBC, whose tumors express PD-L1 with a CPS ≥ 10. In the absence of comparison, the place of this association in the first line compared to PARP inhibitors in the case of tumors with BRCA1/2 mutation remains to be determined. HAS determined that Keytruda in combination with chemotherapy is “Important” in terms of actual benefit (SMR) in the indication extension, and in the case of improvement of the medical service rendered (ASMR), HAS found the drug to be “Moderate (III).”

The High Health Authority of France (HAS) has published a favorable opinion on reimbursing an indication extension for Tagrisso (osimertinib) for the treatment of adult patients with stage IB–IIIA non-small cell lung cancer (NSCLC) with epidermal growth factor receptor activating mutations (EGFR) by deletion of exon 19 or substitution of exon 21 (L858R), only in adjuvant treatment after complete tumor resection and after adjuvant chemotherapy. In a release, the Committee also emphasized that in the adjuvant setting, patients must be treated until the recurrence of the disease or occurrence of unacceptable toxicity and that a treatment duration of more than three years has not been studied. HAS concluded that the medical service rendered (SMR) by Tagrisso in this indication is “Important” while the actual therapeutic benefit is “Minor IV.”

Pricing & Reimbursement

The Institute for Clinical and Economic Review (ICER) has published its revised Evidence Report on bluebird bio’s Zynteglo/LentiGlobin (betibeglogene autotemcel; beti-cel) for the treatment of beta thalassemia – it follows an initial report from April 2022. Though not yet approved, Zynteglo is currently under review by the U.S. Food and Drug Administration (FDA) and has a Prescription Drug User Fee Act (PDUFA) target action set for August 19, 2022. While ICER expressed doubts regarding the gene therapy’s long-term efficacy, it nevertheless judged that evidence shows Zynteglo is superior to standard of care, and traditional cost-effectiveness modelling suggests a cumulative price of $2.1 million, with an 80% payback option for patients who do not achieve and maintain transfusion independence over a five-year period.

According to an analysis led by Dr Charlotte Bee, Program Manager, Data Projects, at the National Institute for Health and Care Excellence (NICE), 86 percent of managed access agreements (MAAs) in England end in routine reimbursement on the NHS. Citing a recent study, “What Goes in Must Come Out: An Analysis of NICE Recommendations for Drugs Exiting Managed (Early) Access in England,” Bee explained how the study reviewed all MAAs negotiated since they began in 2016, when the Cancer Drugs Fund (CDF) was reformed. The study found that in six years, 22 technologies had been re-evaluated by NICE following a period of managed access – all but two of which were cancer therapies and the MAAs for 19 drugs had involved RWD collection.

Last July, France made changes to its long-standing Authorization for Temporary Use (ATU) and Recommendation for Temporary Use (RTU) systems via the Social Security Finance Bill 2021 (PLFSS 2021). Now that the scheme has been in place for 10 months, the National Agency for the Safety of Medicines and Health Products (ANSM) has released a review of the processes, with a view to adapt and improve them going forward. Based on the review, the High Health Authority of France (HAS) wants to refine its evaluation principles, namely the criteria it uses to determine the presence or absence of appropriate treatment. Additionally, in view of the “diversity of clinical situations encountered during the first 10 months of practice,” HAS has acknowledged that it is not possible to offer a single definition of innovation. In this context, HAS will assess various components, taking into account, in particular, the specificities of certain therapeutic areas for which there is a major medical need with regard to the mortality and morbidity of the pathology.

The Danish Medicines Agency has announced that from June 1, 2022, a new four-year pilot scheme for risk-sharing drug subsidies will enter into force – the Agency will have the option to include five medicines in the scheme. “Generic risk-sharing conditional reimbursement means that the public health system covers the cost of reimbursement for a specified number of patients (the target population) deemed to meet the conditions for reimbursement, while pharmaceutical companies cover the public health system’s cost of reimbursement for the number of patients that may exceed the target population,” the Agency explained. The Agency generally awards conditional reimbursement on terms of risk-sharing to medicinal products on a first-come, first-served basis based on the date of receipt of the Danish Medicines Agency’s complete application.

Drug Approval

The U.S. Food and Drug Administration (FDA) has cleared Legend’s Investigational New Drug (IND) application to evaluate investigational, autologous CAR-T cell therapy, LB1908, in a Phase 1 clinical trial. LB1908 will be investigated as treatment of adults with relapsed or refractory gastric, esophageal (including gastro-esophageal junction) or pancreatic cancers in the first-in-human, open-label, multicenter Phase 1 clinical study. Lida Pacaud, M.D., VP of Clinical Development, commented, “Based on prevailing research, we are optimistic that a CAR-T therapy targeting Claudin 18.2 can be integrated in future treatment strategies for those with relapsed or refractory gastrointestinal cancers.”

 

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorization (MA) for VBI Vaccines’ PreHevbri [Hepatitis B vaccine (recombinant, adsorbed)] for active immunization against infection caused by all known subtypes of the hepatitis B virus (HBV) in adults. “Based upon the safety and immunogenicity profiles observed in the PROTECT and CONSTANT pivotal Phase 3 studies, data from which built the foundation of our FDA, EMA, and now UK MHRA approvals, we continue to believe PreHevbri will be a meaningful new intervention in the fight against hepatitis B,” said Jeff Baxter, VBI’s President, and CEO. PreHevbri is the only approved 3-antigen hepatitis B vaccine, comprised of the three hepatitis B surface antigens of the hepatitis B virus – S, pre-S1, and pre-S2, for adults in the United Kingdom. The company informs that PreHevbri is expected to be available in the UK in early 2023.

The European Commission (EC) has approved the Marketing Authorization Application (MAA) for Foresee’s Camcevi (leuprolide) 42 mg prolonged-release suspension for injection for the treatment of hormone-dependent advanced prostate cancer and the treatment of high-risk localized prostate cancer and locally advanced hormone-dependent prostate cancer in combination with radiotherapy. The approval applies to all 27 European Union (EU) Member States, Iceland, Norway, and Liechtenstein. Camcevi is the first-ever ready-to-inject sterile formulation of leuprolide mesylate for subcutaneous injection that comes in a pre-filled syringe with no mixing required.

The Ministry of Health, Labor, and Welfare (MHLW) of Japan has approved ViiV’s Vocabria (cabotegravir injection and tablets) in combination with Janssen’s Rekambys (rilpivirine long-acting injectable suspension) and Edurant (rilpivirine tablets) as the first and only complete long-acting treatment for HIV. The cabotegravir injection-rilpivirine long-acting combination is indicated to treat HIV-1 infection in adults who are virologically suppressed, on a stable regimen with no history of treatment failure, and with no known or suspected resistance to either cabotegravir or rilpivirine. Deborah Waterhouse, CEO of ViiV Healthcare, commented, “By removing the need for daily oral treatment, the approval of cabotegravir injection and rilpivirine long-acting is an important development for the HIV community and reinforces our efforts to provide new treatment options so that no person living with HIV is left behind.”

Keytruda (pembrolizumab) has been authorized by the UAE’s Ministry of Health and Prevention (MOHAP) for the treatment of triple-negative breast cancer (TNBC). Dubai’s Hospital Consultant Medical Oncologist Dr. Dina Hamza told Khaleej Times that all TNBC patients will have access to MSD’s immunotherapy at public and private clinics. MSD’s drug plus chemotherapy has been shown to better reduce the reoccurrence rate of TNBC versus chemotherapy alone.

China’s National Medical Products Administration (NMPA) has approved a two-dose vaccine schedule for GSK’s Cervarix [Human Papillomavirus bivalent (types 16, 18) Vaccine, Recombinant)] in girls aged between 9 to 14 years for the prevention of cervical cancer, cervical intraepithelial neoplasia, and adenocarcinoma in situ (AIS). With this approval, Cervarix is the first imported two-dose HPV vaccine for the age group 9 to 14 in mainland China. Cervarix is a non-infectious recombinant, AS04-adjuvanted vaccine. Data shows that cervical cancer has the highest mortality of all malignant tumors in the female reproductive system in China, with 110,000 new cases of cervical cancer and 59,000 deaths in 2020 due to the disease. Moreover, incidence and mortality rates show an increasing trend in younger women. The potential impact of vaccines against oncogenic HPV types 16 and 18 is estimated to be high (84.5%) against total squamous cell carcinoma (SCC).

The U.S. Food and Drug Administration (FDA) has approved both Opdivo (nivolumab) (injection for intravenous use) in combination with fluoropyrimidine- and platinum-containing chemotherapy and Opdivo plus Yervoy (ipilimumab) as a first-line treatment for adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) regardless of PD-L1 status. The approvals are based on the Phase 3 CheckMate -648 trial, which evaluated Opdivo in combination with chemotherapy (n=321) and Opdivo plus Yervoy (n=325) each compared to chemotherapy alone (n=324) and was the largest Phase 3 trial of immunotherapy in first line ESCC. In the trial, Opdivo in combination with chemotherapy demonstrated superior overall survival (OS) compared to chemotherapy alone. The application was reviewed under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.

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